10-Q

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 10-Q


☒	QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended March 31, 2025


☐	TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from to .

Commission File Number: 001-37923

CRISPR THERAPEUTICS AG

(Exact name of registrant as specified in its charter)


Switzerland	Not Applicable
(State or other jurisdiction of incorporation or organization)	(I.R.S. Employer Identification No.)

Baarerstrasse 14 6300 Zug, Switzerland	Not Applicable
(Address of principal executive offices)	(Zip Code)

+41 (0)41 561 32 77

(Registrant’s telephone number, including area code)

Securities registered pursuant to Section 12(b) of the Act:


Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Shares, nominal value CHF 0.03	CRSP	The Nasdaq Global Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or emerging growth company. See definition of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.


Large Accelerated filer	☒	Accelerated filer	☐

Non-accelerated filer	☐	Smaller reporting company	☐

		Emerging growth company	☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Indicate by check mark whether registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒

As of May 2, 2025, there were 86,363,684 shares of registrant’s common shares outstanding.

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Throughout this Quarterly Report on Form 10-Q, the “Company,” “CRISPR,” “CRISPR Therapeutics,” “we,” “us,” and “our,” except where the context requires otherwise, refer to CRISPR Therapeutics AG and its consolidated subsidiaries; “our board of directors” refers to the board of directors of CRISPR Therapeutics AG; and we generally refer to CASGEVY® (exagamglogene autotemcel [exa-cel]), as “CASGEVY”.

“CRISPR Therapeutics®” standard character mark and design logo, “CRISPRXTM,” “CRISPR TXTM,” “CTX112TM,” “CTX131TM,” “CTX211TM,” “CTX213TM,” “CTX310TM,” “CTX320TM,” “CTX330TM,” “CTX340TM,” and “CTX450TM,” are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated, and Vertex Pharmaceuticals Incorporated is the manufacturer and exclusive license holder of CASGEVY. All other trademarks and registered trademarks contained in this Quarterly Report on Form 10-Q are the property of their respective owners. Solely for convenience, trademarks, service marks and trade names referred to in this Quarterly Report on Form 10-Q may appear without the ® or ™ symbols and any such omission is not intended to indicate waiver of any such rights.

FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q contains “forward-looking statements” that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Quarterly Report on Form 10-Q are forward-looking statements. These statements are often identified by the use of words such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “potential,” “will,” “would” or the negative or plural of these words or similar expressions or variations, although not all forward-looking statements contain these identifying words. Forward-looking statements in this Quarterly Report on Form 10-Q include, but are not limited to, statements about:

•

our strategic plans to develop and, if approved, subsequently commercialize any product candidates we may develop, including plans and expectations for the commercialization of, and anticipated benefits of, CASGEVY, including plans for patient access to CASGEVY;

•

the safety, efficacy and clinical progress of various clinical programs, including those for CASGEVY, CTX112, CTX131, CTX211, CTX310 and CTX320;

•

the status of clinical trials, including development timelines and discussions with regulatory authorities related to product candidates under development by us and our collaborators;

•

the results of our preclinical studies and clinical trials, including our ongoing clinical trials and any planned clinical trials, and our research and development programs;

•

the actual or potential benefits of regulatory designations, such as orphan drug, fast track and regenerative medicine advanced therapy in the United States or such European equivalents, including the PRIority MEdicines designation;

•

our ability to advance product candidates into, and successfully complete, clinical trials;

•

the size and growth potential of the markets for our product candidates and our ability to serve those markets, including our estimates regarding the addressable patient population and potential market opportunity for our current and future product candidates;

•

the rate and degree of market acceptance of our product candidates and the success of competing therapies that are or become available;

•

our internal manufacturing capabilities and operation of our cell therapy manufacturing facility;

•

our intellectual property coverage and positions, including those of our licensors and third parties as well as the status and potential outcome of proceedings involving any such intellectual property;

•

the expected benefits of our collaborations;

•

our strategy, goals, and anticipated financial performance;

•

our anticipated expenses, ability to obtain funding for our operations and the sufficiency of our cash resources;

•

the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9, as well as other technologies we develop; and

•

the volatility of capital markets and unfavorable macroeconomic conditions resulting from factors including rising inflation, international tariffs, interest rate and currency rate fluctuations, economic sanctions and economic slowdown or recession, banking instability, monetary policy changes, geopolitical tensions or the outbreak of hostilities or war.

Any forward-looking statements in this Quarterly Report on Form 10-Q reflect our current views with respect to future events or to our future financial performance and involve known and unknown risks, uncertainties and assumptions that could cause our actual results and the timing of certain events to differ materially from future results expressed or implied by the forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those identified herein, and those discussed in the section titled “Risk Factors,” set forth in Part II, Item 1A of this Quarterly Report on Form 10-Q, if any, our Annual Report on Form 10-K filed with the Securities and Exchange Commission, or SEC, on February 11, 2025, and in other SEC filings. You should not rely upon forward-looking statements as predictions of future events. Such forward-looking statements speak only as of the date of this report. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures or investments we may make or enter into.

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You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q completely and with the understanding that our actual future results, performance or achievements may be materially different from what we expect. Except as required by law, we undertake no obligation to update any forward-looking statements to reflect events or circumstances after the date of such statements.

Investors and others should note that we announce material information to our investors using our investor relations website (https://crisprtx.gcs-web.com/), SEC filings, press releases, public conference calls and webcasts. We use these channels as well as social media to communicate with the public about our Company, our business, our product candidates and other matters. It is possible that the information we post on social media could be deemed to be material information. Therefore, we encourage investors, the media, and others interested in our Company to review the information we post on the social media channels listed on our investor relations website.

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Index


	Page Number
PART I: FINANCIAL INFORMATION

Item 1. Condensed Consolidated Financial Statements (unaudited)	2

Condensed Consolidated Balance Sheets as of March 31, 2025 and December 31, 2024	2

Condensed Consolidated Statements of Operations and Comprehensive Loss for the three months ended March 31, 2025 and 2024	3

Condensed Consolidated Statements of Shareholders’ Equity for the three months ended March 31, 2025 and 2024	4

Condensed Consolidated Statements of Cash Flows for the three months ended March 31, 2025 and 2024	5

Notes to Condensed Consolidated Financial Statements	6

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations	16

Item 3. Quantitative and Qualitative Disclosures about Market Risk	23

Item 4. Controls and Procedures	24

PART II: OTHER INFORMATION

Item 1. Legal Proceedings	25

Item 1A. Risk Factors	25

Item 2. Unregistered Sales of Equity Securities and Use of Proceeds	26

Item 3. Defaults Upon Senior Securities	26

Item 4. Mine Safety Disclosures	26

Item 5. Other Information	26

Item 6. Exhibits	27

SIGNATURES	28

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PART I—FINANCIAL INFORMATION

Item 1. Financial Statements

CRISPR Therapeutics AG

Condensed Consolidated Balance Sheets

(unaudited, in thousands, except share and per share data)


	As of
	March 31,			December 31,
	2025			2024
Assets
Current assets:
Cash and cash equivalents	$	235,184		$	298,257
Marketable securities		1,620,101			1,605,569
Accounts receivable		—			25,000
Prepaid expenses and other current assets		12,326			8,306
Total current assets		1,867,611			1,937,132
Property and equipment, net		129,426			134,093
Restricted cash		8,006			11,519
Operating lease assets		140,277			143,461
Other non-current assets		20,782			15,829
Total assets	$	2,166,102		$	2,242,034
Liabilities and shareholders’ equity
Current liabilities:
Accounts payable	$	13,064		$	14,709
Accrued expenses		84,615			41,072
Deferred revenue, current		3,021			3,845
Accrued tax liabilities		1,241			451
Operating lease liabilities		17,506			17,288
Other current liabilities		—			10,417
Total current liabilities		119,447			87,782
Deferred revenue, non-current		12,323			12,323
Operating lease liabilities, net of current portion		201,852			206,405
Other non-current liabilities		3,320			3,444
Total liabilities		336,942			309,954
Commitments and contingencies, see Note 7
Shareholders’ equity:
Common shares, CHF 0.03 nominal value, 132,477,166 shares authorized at March 31, 2025 and at December 31, 2024, 86,531,240 and 85,912,297 shares issued at March 31, 2025 and December 31, 2024, respectively, 86,360,924 and 85,741,981 shares outstanding at March 31, 2025 and December 31, 2024, respectively		2,721			2,698
Treasury shares, at cost, 170,316 shares at March 31, 2025 and at December 31, 2024		(62	)		(62	)
Additional paid-in capital		3,324,314			3,293,556
Accumulated deficit		(1,501,948	)		(1,365,952	)
Accumulated other comprehensive income		4,135			1,840
Total shareholders' equity		1,829,160			1,932,080
Total liabilities and shareholders’ equity	$	2,166,102		$	2,242,034

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

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CRISPR Therapeutics AG

Condensed Consolidated Statements of Operations and Comprehensive Loss

(unaudited, in thousands, except share and per share data)


	Three Months Ended
	March 31,
	2025			2024
Revenue:
Collaboration revenue	$	—		$	—
Grant revenue		865			504
Total revenue		865			504
Operating expenses:
Research and development		72,484			76,172
General and administrative		19,296			17,953
Collaboration expense, net		57,509			46,966
Total operating expenses		149,289			141,091
Loss from operations		(148,424	)		(140,587	)
Other income:
Other income, net		13,537			24,720
Total other income, net		13,537			24,720
Net loss before income taxes		(134,887	)		(115,867	)
Provision for income taxes		(1,109	)		(724	)
Net loss		(135,996	)		(116,591	)
Foreign currency translation adjustment		41			(11	)
Unrealized gain (loss) on marketable securities		2,254			(3,454	)
Comprehensive loss	$	(133,701	)	$	(120,056	)

Net loss per common share — basic	$	(1.58	)	$	(1.43	)
Basic weighted-average common shares outstanding		85,938,720			81,794,630
Net loss per common share — diluted	$	(1.58	)	$	(1.43	)
Diluted weighted-average common shares outstanding		85,938,720			81,794,630

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

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CRISPR Therapeutics AG

Condensed Consolidated Statements of Shareholders’ Equity

(unaudited, in thousands, except share and per share data)


	Common Shares				Treasury Shares
	Shares		CHF 0.03 Nominal Value		Shares		Amount, at cost			Additional Paid-in Capital		Accumulated Deficit			Accumulated Other Comprehensive Gain/(Loss)			Total Shareholders’ Equity
Balance at December 31, 2023		80,044,378	$	2,497		170,316	$	(62	)	$	2,878,155	$	(999,700	)	$	1,913		$	1,882,803
Issuance of common stock, net of issuance costs of $3.8 million		3,929,610		132		—		—			277,015		—			—			277,147
Vesting of restricted shares		214,913		7		—		—			—		—			—			7
Exercise of vested options, net of issuance costs of $0.6 million		632,683		22		—		—			23,844		—			—			23,866
Purchase of common stock under ESPP		16,026		—		—		—			764		—			—			764
Stock-based compensation expense		—		—		—		—			19,405		—			—			19,405
Other comprehensive loss		—		—		—		—			—		—			(3,465	)		(3,465	)
Net loss		—		—		—		—			—		(116,591	)		—			(116,591	)
Balance at March 31, 2024		84,837,610	$	2,658		170,316	$	(62	)	$	3,199,183	$	(1,116,291	)	$	(1,552	)	$	2,083,936

Balance at December 31, 2024		85,741,981	$	2,698		170,316	$	(62	)	$	3,293,556	$	(1,365,952	)	$	1,840		$	1,932,080
Issuance of common stock, net of issuance costs of $0.1 million		162,482		5		—		—			8,632		—			—			8,637
Vesting of restricted shares		396,943		15		—		—			—		—			—			15
Exercise of vested options, net of issuance costs of $0.2 million		39,996		3		—		—			1,261		—			—			1,264
Purchase of common stock under ESPP		19,522		—		—		—			653		—			—			653
Stock-based compensation expense		—		—		—		—			20,212		—			—			20,212
Other comprehensive income		—		—		—		—			—		—			2,295			2,295
Net loss		—		—		—		—			—		(135,996	)		—			(135,996	)
Balance at March 31, 2025		86,360,924	$	2,721		170,316	$	(62	)	$	3,324,314	$	(1,501,948	)	$	4,135		$	1,829,160

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

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CRISPR Therapeutics AG

Condensed Consolidated Statements of Cash Flows

(unaudited, in thousands)


	Three Months Ended March 31,
	2025			2024
Operating activities:
Net loss	$	(135,996	)	$	(116,591	)
Reconciliation of net loss to net cash used in operating activities:
Depreciation and amortization		4,736			4,833
Equity-based compensation		20,212			19,405
Other non-cash items, net		2,253			(8,385	)
Changes in:
Accounts receivable		25,000			200,000
Prepaid expenses and other assets		(634	)		2,949
Accounts payable and accrued expenses		42,998			9,443
Deferred revenue		(824	)		(504	)
Operating lease assets and liabilities		(1,151	)		(966	)
Other liabilities, net		(10,541	)		(432	)
Net cash (used in) provided by operating activities		(53,947	)		109,752
Investing activities:
Purchase of property, plant and equipment		(206	)		(721	)
Investment in equity securities		(5,000	)		(7,500	)
Purchases of marketable securities		(200,730	)		(326,915	)
Maturities of marketable securities		186,181			237,336
Net cash used in investing activities		(19,755	)		(97,800	)
Financing activities:
Proceeds from issuance of common shares, net of issuance costs		8,516			280,745
Proceeds from exercise of options and ESPP contributions, net of issuance costs		2,072			25,189
Net cash provided by financing activities		10,588			305,934
Effect of exchange rate changes on cash		41			(11	)
(Decrease) increase in cash		(63,073	)		317,875
Cash, cash equivalents and restricted cash, beginning of period		309,776			401,068
Cash, cash equivalents and restricted cash, end of period	$	246,703		$	718,943
Supplemental disclosure of non-cash investing and financing activities
Equity issuance costs in accounts payable, accrued expenses, and other long-term liabilities	$	3,219		$	4,522


	As of March 31,
Reconciliation to amounts within the condensed consolidated balance sheets	2025		2024
Cash and cash equivalents	$	235,184	$	707,427
Prepaid expenses and other current assets		3,513		—
Restricted cash		8,006		11,516
Cash, cash equivalents and restricted cash at end of period	$	246,703	$	718,943

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

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CRISPR Therapeutics AG

Notes to Condensed Consolidated Financial Statements

(unaudited)

1. Basis of Presentation and Significant Accounting Policies

Basis of Presentation

The accompanying condensed consolidated financial statements are unaudited and have been prepared by the Company in accordance with accounting principles generally accepted in the United States of America, or U.S. GAAP.

The accompanying condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The Company views its operations and manages its business in one operating segment, which is the business of discovering, developing and commercializing therapies derived from or incorporating genome-editing technology. Certain information and footnote disclosures normally included in the Company’s annual financial statements have been condensed or omitted. These interim financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the financial position and results of operations for the three-month interim periods ended March 31, 2025 and 2024.

The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year. These interim financial statements should be read in conjunction with the audited financial statements for the year ended December 31, 2024, which are contained in the 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission, or the SEC, on February 11, 2025.

Use of Estimates

The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, revenue recognition, equity-based compensation expense and reported amounts of expenses during the period. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Changes in estimates are reflected in reported results in the period in which they become known.

Significant Accounting Policies

The significant accounting policies used in preparation of these condensed consolidated financial statements for the three months ended March 31, 2025 are consistent with those discussed in Note 2 to the consolidated financial statements in the Company’s 2024 Annual Report on Form 10-K filed with the SEC on February 11, 2025, with the exception of changes to the below policy.

Collaboration Arrangements

The Company records the elements of its collaboration agreements that represent joint operating activities in accordance with ASC, Accounting Standards Codification, or ASC, 808, Collaborative Agreements, or ASC 808. Accordingly, the elements of the collaboration agreements that represent activities in which both parties are active participants and to which both parties are exposed to the significant risks and rewards that are dependent on the commercial success of the activities, are recorded as collaborative arrangements.

The Company evaluates the proper presentation of the commercial activities and the proﬁt and loss sharing associated with the collaboration agreements. ASC 808 states that when payments between parties in a collaborative arrangement are not within the scope of other authoritative accounting literature, the income statement classification should be based on the nature of the arrangement, the nature of its business operations and the contractual terms of the arrangement. To the extent that these payments are not within the scope of other authoritative accounting literature, the income statement classification for the payments shall be based on an analogy to authoritative accounting literature or if there is no appropriate analogy, a reasonable, rational and consistently applied accounting policy election.

Collaboration costs specific to the Vertex Hemoglobinopathy Agreements (as defined in Note 6) accounted for under ASC 808 are presented within “collaboration expense, net” in the condensed consolidated statements of operations and comprehensive loss. Refer to Note 6 to these condensed consolidated financial statements for further discussion on the Vertex Hemoglobinopathy Agreements.

Recent Accounting Pronouncements

In December 2023, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, 2023-09, Improvements to Income Tax Disclosures, which requires entities, on an annual basis, to disclose disaggregated information about their effective tax rate reconciliation and income taxes paid. The disclosure requirements will be applied on a prospective basis, with the option to apply them retrospectively. The standard is effective for fiscal years beginning after December 15, 2024, with early

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adoption permitted. The Company has adopted ASU 2023-09 and is currently evaluating the guidance, which is expected to only impact annual disclosures with no impact to the Company’s results of operations.

In November 2024, the FASB issued ASU 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures, which requires disclosure of additional information about specific expense categories in the notes to the financial statements on an interim and annual basis. The standard is effective for fiscal years beginning after December 15, 2026, and for interim periods beginning after December 15, 2027, with early adoption permitted. The Company is currently evaluating the disclosure requirements related to this new standard.

2. Marketable Securities

The following table summarizes cash equivalents and marketable securities held at March 31, 2025 and December 31, 2024 (in thousands), which are recorded at fair value. The table below excludes $137.7 million and $193.9 million of cash at March 31, 2025 and December 31, 2024, respectively.


	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses			Fair Value
March 31, 2025
Cash equivalents:
Money market funds	$	66,748	$	—	$	—		$	66,748
Commercial paper		23,475		—		(2	)		23,473
Corporate debt securities		914		—		—			914
U.S. Treasury securities		6,339		—		—			6,339
Total cash equivalents		97,476		—		(2	)		97,474
Marketable securities:
Corporate debt securities		1,141,075		4,263		(614	)		1,144,724
Certificates of deposit		107,242		—		—			107,242
Government-sponsored enterprise securities		269,836		596		(176	)		270,256
Commercial paper		91,587		21		(4	)		91,604
Total marketable debt securities		1,609,740		4,880		(794	)		1,613,826
Corporate equity securities		10,387		—		(4,112	)		6,275
Total marketable securities		1,620,127		4,880		(4,906	)		1,620,101
Total cash equivalents and marketable securities	$	1,717,603	$	4,880	$	(4,908	)	$	1,717,575
December 31, 2024
Cash equivalents:
Money market funds	$	74,155	$	—	$	—		$	74,155
Corporate debt securities		882		—		—			882
Commercial paper		29,271		—		(9	)		29,262
Total cash equivalents		104,308		—		(9	)		104,299
Marketable securities:
U.S. Treasury securities		5,936		2		—			5,938
Corporate debt securities		1,136,255		3,442		(1,592	)		1,138,105
Certificates of deposit		52,372		—		—			52,372
Government-sponsored enterprise securities		266,877		482		(497	)		266,862
Commercial paper		127,805		34		(39	)		127,800
Total marketable debt securities		1,589,245		3,960		(2,128	)		1,591,077
Corporate equity securities		10,387		4,600		(495	)		14,492
Total marketable securities		1,599,632		8,560		(2,623	)		1,605,569
Total cash equivalents and marketable securities	$	1,703,940	$	8,560	$	(2,632	)	$	1,709,868

As of March 31, 2025, marketable debt securities were in a net unrealized gain position of $4.1 million. As of December 31, 2024, marketable debt securities were in a net unrealized gain position of $1.8 million. The Company has recorded a net unrealized gain of $2.3 million and a net unrealized loss of $3.5 million during the three months ended March 31, 2025 and 2024, respectively, related to its debt securities, which is included in comprehensive loss on the condensed consolidated statements of operations and comprehensive loss.

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As of March 31, 2025 and December 31, 2024, the aggregate fair value of marketable securities that were in an unrealized loss position for less than twelve months was $288.8 million and $451.9 million, respectively. As of March 31, 2025 and December 31, 2024, the aggregate fair value of marketable securities that were in an unrealized loss position for more than twelve months was $3.5 million and $31.8 million, respectively. As of March 31, 2025, no securities in an unrealized loss position for more than twelve months will mature beyond one year. As of December 31, 2024, no securities in an unrealized loss position for more than twelve months had maturities beyond one year.

As of March 31, 2025, marketable equity securities were in a net unrealized loss position of $4.1 million. As of December 31, 2024, marketable equity securities were in a net unrealized gain position of $4.1 million. The Company has recorded a net unrealized loss of $8.2 million during the three months ended March 31, 2025, related to its equity securities. The Company did not hold marketable equity securities as of March 31, 2024. These amounts are included in comprehensive loss on the condensed consolidated statements of operations and comprehensive loss.

The Company determined that there is no material credit risk associated with the above investments as of March 31, 2025. The Company has the intent and ability to hold such securities until recovery. As a result, the Company did not record any charges for credit-related impairments for its marketable securities for the three months ended March 31, 2025 and 2024. No available-for-sale debt securities held as of March 31, 2025 had remaining maturities greater than thirty months.

3. Fair Value Measurements

The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the fair value hierarchy classification of such fair values as of March 31, 2025 and December 31, 2024 (in thousands):


	Fair Value Measurements at
	March 31, 2025
	Total		Level 1		Level 2		Level 3
Cash and cash equivalents:
Cash	$	137,710	$	137,710	$	—	$	—
Money market funds		66,748		66,748		—		—
Commercial paper		23,473		—		23,473		—
Corporate debt securities		914		—		914		—
U.S. Treasury securities		6,339		—		6,339		—
Marketable securities:
Corporate debt securities		1,144,724		—		1,144,724		—
Certificates and term deposits		107,242		—		107,242		—
Government-sponsored enterprise securities		270,256		—		270,256		—
Commercial paper		91,604		—		91,604		—
Corporate equity securities		6,275		875		5,400		—
Total	$	1,855,285	$	205,333	$	1,649,952	$	—

	Fair Value Measurements at
	December 31, 2024
	Total		Level 1		Level 2		Level 3
Cash and cash equivalents:
Cash	$	193,958	$	193,958	$	—	$	—
Money market funds		74,155		74,155		—		—
Corporate debt securities		882		—		882		—
Commercial paper		29,262		—		29,262		—
Marketable securities:
U.S. Treasury securities		5,938		—		5,938		—
Corporate debt securities		1,138,105		—		1,138,105		—
Certificates of deposit		52,372		—		52,372		—
Government-sponsored enterprise securities		266,862		—		266,862		—
Commercial paper		127,800		—		127,800		—
Corporate equity securities		14,492		2,391		12,101		—
Total	$	1,903,826	$	270,504	$	1,633,322	$	—

Marketable securities classified as Level 1 within the valuation hierarchy consist of corporate equity securities with quoted prices in active markets. Marketable securities classified as Level 2 within the valuation hierarchy generally consist of U.S. treasury securities and government agency securities, certificates of deposit, corporate bonds, commercial paper and warrants to purchase

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common shares of publicly traded companies. The Company estimates the fair value of these marketable securities by taking into consideration valuations obtained from third-party pricing sources.

4. Property and Equipment, net

Property and equipment, net, consists of the following (in thousands):


	As of
	March 31,			December 31,
	2025			2024
Computer equipment	$	4,248		$	3,833
Furniture, fixtures and other		8,554			8,554
Laboratory equipment		42,204			42,008
Leasehold improvements		145,852			145,852
Construction work in process		5,536			6,118
Total property and equipment, gross		206,394			206,365
Accumulated depreciation		(76,968	)		(72,272	)
Total property and equipment, net	$	129,426		$	134,093

Depreciation expense for the three months ended March 31, 2025 and 2024 $4.7 million and $4.8 million, respectively.

5. Accrued Expenses

Accrued expenses consist of the following (in thousands):


	As of
	March 31,		December 31,
	2025		2024
Payroll and employee-related costs	$	6,086	$	18,443
Research and development costs		17,508		15,549
Collaboration costs		55,297		—
Licensing fees		806		1,850
Professional fees		3,426		3,086
Intellectual property costs		1,031		1,513
Other		461		631
Total	$	84,615	$	41,072

6. Significant Contracts

Agreements with Vertex

For purposes of this Note 6 and Note 7, CASGEVY (exagamglogene autotemcel [exa-cel]) is referred to as “CASGEVY”.

2015 collaboration

In 2015, the Company entered into a strategic collaboration, option and license agreement, or the 2015 Collaboration Agreement, with Vertex Pharmaceuticals Incorporated, or Vertex. The 2015 Collaboration Agreement is focused on the use of the Company’s CRISPR/Cas9 gene editing technology to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. The Company and Vertex amended the 2015 Collaboration Agreement in 2017 and 2019 with Amendment No. 1 and Amendment No. 2, respectively, namely to clarify Vertex’s option rights under the 2015 Collaboration Agreement and to modify certain definitions and provisions of the 2015 Collaboration Agreement to make them consistent with the JDA (as defined below) and the 2019 Collaboration Agreement (as defined below). In 2017, Vertex exercised an option granted to it under the 2015 Collaboration Agreement to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, and in 2019, Vertex exercised the remaining options granted to it under the 2015 Collaboration Agreement to exclusively license certain collaboration targets developed under the 2015 Collaboration Agreement.

Hemoglobinopathies collaboration

In 2017, following Vertex's exercise of its option to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, the Company and Vertex entered into a joint development and commercialization agreement, or the JDA, and agreed for potential hemoglobinopathy treatments, including CASGEVY, the Company and Vertex would share equally all research and development costs and worldwide revenues. In 2021, the Company and Vertex amended and restated the JDA, or the A&R Vertex JDCA (as amended and in effect, from time to time), pursuant to which the parties agreed to, among other things, (a) adjust the governance structure for the collaboration and adjust the responsibilities of each party thereunder, whereby Vertex leads and has all decision making (i.e., control) in relation to the CASGEVY program prospectively; (b) adjust the allocation

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of net profits and net losses between the parties with respect to CASGEVY only, which will be allocated 40% to the Company and 60% to Vertex, prospectively; and (c) exclusively license (subject to the Company’s reserved rights to conduct certain activities) certain intellectual property rights to Vertex relating to the specified product candidates and products (including CASGEVY) that may be researched, developed, manufactured and commercialized on a worldwide basis under the A&R Vertex JDCA. Additionally, for 2022, 2023, and 2024, the Company had an option to defer a portion of its share of costs under the A&R Vertex JDCA if spending on the CASGEVY program exceeded specified amounts. Any deferred amounts under the A&R Vertex JDCA, as amended, are only payable to Vertex as an offset against future profitability of the CASGEVY program and the amounts payable are capped at a specified maximum amount per year.

In December 2023, the Company entered into an amendment to the A&R Vertex JDCA, or Amendment No. 1 to the A&R Vertex JDCA, with Vertex related to the global development, manufacturing, and commercialization of CASGEVY. Pursuant to Amendment No. 1 to the A&R Vertex JDCA, among other things, the Company and Vertex agreed to (a) allocate certain costs arising from a license agreement with a third party, resulting in a current payment due to Vertex by the Company of $20.0 million upon an event specified in Amendment No. 1 to the A&R Vertex JDCA, and (b) adjust, under certain specified circumstances, the timing of and portion of the Company’s share of costs it is permitted to defer under the agreement.

Letter Agreement

In May 2024, Vertex and the Company entered into a letter agreement, or the Letter Agreement, with respect to the priority review voucher issued by the FDA to Vertex as the sponsor of the rare pediatric disease product application for CASGEVY. Vertex and the Company agreed that if Vertex utilizes or transfers the priority review voucher prior to the first calendar year in which the CASGEVY program generates a net profit, Vertex will pay the Company $43.0 million or an amount equal to 42% of the net proceeds from such transfer, as applicable. If the CASGEVY program begins generating calendar-year net profits prior to such utilization or transfer, Vertex will instead pay the Company up to $43.0 million set-off by deductions Vertex would otherwise be eligible to take against the CASGEVY program’s net profits due to the Company related to amounts deferred previously by the Company.

Collaboration in the field of diabetes

In 2021, the Company and ViaCyte, Inc., or ViaCyte, entered into a joint development and commercialization agreement, or the ViaCyte JDCA, to jointly develop and commercialize product candidates and shared products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. In the third quarter of 2022, Vertex acquired ViaCyte, and ViaCyte became a wholly-owned subsidiary of Vertex. In March 2023, (1) the Company and ViaCyte entered into an amendment to the ViaCyte JDCA, or the ViaCyte JDCA Amendment, and adjusted certain rights and obligations of the Company and ViaCyte under the ViaCyte JDCA, and (2) the Company and Vertex entered into a non-exclusive license agreement, or the Non-Ex License Agreement, pursuant to which the Company agreed to license to Vertex, on a non-exclusive basis, certain of its gene editing intellectual property to exploit certain products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. Subsequently, ViaCyte elected to opt-out of the ViaCyte JDCA. Per the opt-out terms, the on-going collaboration assets are now wholly owned by the Company, subject to a royalty on future sales owed to ViaCyte. The opt-out became effective in early February 2024.

In 2024, the Company received a $10.0 million research milestone achieved in the fourth quarter of 2024 and recorded a receivable of $25.0 million as of December 31, 2024 related to an additional research milestone achieved under the Non-Ex License Agreement in the fourth quarter of 2024. The Company is eligible to receive additional milestone payments under the Non-Ex License Agreement of up to $125.0 million in aggregate, which are dependent on the achievement of pre-determined research, development and commercial milestones for certain products utilizing the licensed intellectual property. Additionally, the Company is eligible to receive tiered royalties on the sales of certain products in the low to mid-single digits.

Accounting Analysis

For purposes of this Note 6, the 2015 Collaboration Agreement, Amendment No. 1, Amendment No. 2, A&R Vertex JDCA, Amendment No. 1 to the A&R Vertex JDCA are collectively referred to as the “Vertex Hemoglobinopathy Agreements” and the Non-Ex License Agreement and ViaCyte JDCA Amendment are collectively referred to as the “March 2023 Diabetes Agreements.”

The Vertex Hemoglobinopathy Agreements and the March 2023 Diabetes Agreements include components of a customer-vendor relationship as defined under ASC 606, Revenue from Contracts with Customers, or ASC 606, collaborative arrangements as defined under ASC 808, and research and development costs as defined under ASC 730, Research and Development, or ASC 730. Specifically, with regards to the March 2023 Diabetes Agreements, the Company concluded that the non-exclusive license is a performance obligation under ASC 606 and the ongoing research and development services under the ViaCyte JDCA Amendment are a unit of account under ASC 808.

The Company has determined that recognition criteria for the Letter Agreement has not been met and will not be met until the priority review voucher is (i) utilized or (ii) there is sufficient profitability such that Vertex is obligated to pay the Company under the Letter Agreement.

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Accounting Analysis Under ASC 606

Accounting for the March 2023 Diabetes Agreements

Recognition of Revenue

No revenue was recognized under the March 2023 Diabetes Agreements for the three months ended March 31, 2025 and 2024, respectively.

Milestones under the Non-Ex License Agreement

As of March 31, 2025, the Company is eligible to receive potential future milestone payments from Vertex of up to $125.0 million in the aggregate under the Non-Ex License Agreement depending on the achievement of pre-determined research, development and commercial milestones for certain products utilizing the licensed intellectual property. Additionally, the Company is eligible to receive tiered royalties on the sales of certain products in the low to mid-single digits.

Each of the remaining milestones under the Non-Ex License Agreement are fully constrained as of March 31, 2025. There is uncertainty as to whether the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.

Accounting for the Vertex Hemoglobinopathy Agreements

Recognition of Revenue

No revenue was recognized under the Vertex Hemoglobinopathy Agreements for the three months ended March 31, 2025 and 2024.

Deferred revenue

As of March 31, 2025 and December 31, 2024, there was no current deferred revenue related to the Vertex Hemoglobinopathy Agreements. As of March 31, 2025, there was $12.3 million of non-current deferred revenue related to the Vertex Hemoglobinopathy Agreements, which is unchanged from December 31, 2024. The transaction price allocated to the remaining performance obligations was $12.3 million.

Milestones under the Vertex Hemoglobinopathy Agreements

The Company has evaluated the milestones that may be received in connection with the Vertex Hemoglobinopathy Agreements.

Under the 2015 Collaboration Agreement and subsequent amendments, the Company is eligible to receive up to $410.0 million in additional development, regulatory and commercial milestones and royalties on net product sales for each of the three collaboration targets that Vertex licensed in 2019. Each milestone is payable only once per collaboration target, regardless of the number of products directed to such collaboration target that achieve the relevant milestone event.

The Company has the option to conduct research at its own cost in certain defined areas. If such research is beneficial to a program under the Vertex Hemoglobinopathy Agreements and the applicable product ultimately achieves regulatory approval in such areas, the Company could be entitled to receive from Vertex certain milestone payments aggregating to high eight digits.

In addition to the milestones described under the Vertex Hemoglobinopathy Agreements, the Company is eligible to receive potential future payments of up to $775.0 million upon the successful achievement of specified development, regulatory and commercial milestones under a strategic collaboration and license agreement with Vertex from 2019 for the development and commercialization of products for the treatment of Duchenne muscular dystrophy and myotonic dystrophy Type 1, or DM1. The Company is also eligible to receive tiered royalties on future net sales on any products that may result from this collaboration; however, the Company has the option to forego the DM1 milestones and royalties to co-develop and co-commercialize all DM1 products globally.

Each of the remaining milestones described above are fully constrained as of March 31, 2025. There is uncertainty that the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.

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Accounting Analysis under ASC 808

Vertex Hemoglobinopathy Agreements

In connection with the Vertex Hemoglobinopathy Agreements, the Company identified the following collaborative elements, which are accounted for under ASC 808: (i) development and commercialization services for shared products, including any transition services related to CASGEVY under the A&R Vertex JDCA; (ii) R&D Services for follow-on products; and (iii) committee participation. The related impact of the cost sharing under the Vertex Hemoglobinopathy Agreements is included within collaboration expense, net, in the condensed consolidated statements of operations and comprehensive loss. During the three months ended March 31, 2025 and 2024, the Company recognized $57.5 million and $47.0 million of collaboration expense, net, respectively. Collaboration expense, net, during the three months ended March 31, 2025 and 2024 was net of $0.1 million and $0.8 million of reimbursements from Vertex, respectively.

7. Commitments and Contingencies

Leases

Refer to Note 7 to the consolidated financial statements in the Company’s 2024 Annual Report on Form 10-K filed with the SEC on February 11, 2025 for discussion of the Company’s lease arrangements.

Litigation

In the ordinary course of business, the Company is or has been from time to time involved in lawsuits, investigations, proceedings and threats of litigation related to, among other things, the Company’s intellectual property estate (including certain in-licensed intellectual property), commercial arrangements and other matters. Such proceedings may include quasi-litigation, inter partes administrative proceedings in the U.S. Patent and Trademark Office and the European Patent Office or patent offices in other countries, involving the Company’s intellectual property estate including certain in-licensed intellectual property. The outcome of any of the foregoing, regardless of the merits, is inherently uncertain. In addition, litigation and related matters are costly and may divert the attention of Company’s management and other resources that would otherwise be engaged in other activities. If the Company is unable to prevail in any such proceedings, the Company’s business, results of operations, liquidity and financial condition could be adversely affected.

Letters of Credit

As of March 31, 2025, the Company had restricted cash of $11.5 million, representing letters of credit securing the Company’s obligations under certain leased facilities. The letters of credit are secured by cash held in a restricted depository account, with $3.5 million included in “Prepaid expenses and other current assets” and $8.0 million included in “Restricted cash” on the Company's condensed consolidated balance sheets as of March 31, 2025.

Research, Manufacturing, License and Intellectual Property Agreements

The Company has engaged several research institutions and companies to identify new delivery strategies and applications of the Company’s gene editing technology. The Company is also a party to a number of license agreements which require significant upfront payments and may be required to make future royalty payments and potential milestone payments from time to time. In addition, the Company is also a party to intellectual property agreements, which require maintenance and milestone payments from time to time. Further, the Company is a party to a number of manufacturing agreements that require upfront payments for the future performance of services.

In connection with these agreements, on a product-by-product basis, the counterparties are eligible to receive up to low eight-digit potential payments upon specified research, development and regulatory milestones. In addition, on a product-by-product basis, the counterparties are eligible to receive potential commercial milestone payments based on specified annual sales thresholds. The potential payments are low-single digit percentages of the specified annual sales thresholds. The counterparties are also eligible to receive low single-digit royalties on future net sales.

Under certain circumstances and if certain contingent future events occur, Vertex is eligible to receive up to $395.0 million in potential specified research, development, regulatory and commercial milestones and tiered single-digit percentage royalties on future net sales related to a specified target under an amendment to the 2015 Collaboration Agreement (as such term is defined in Note 6 above). In addition, Vertex has the option to conduct research at its own cost in certain defined areas that, if beneficial to a program under the Vertex Hemoglobinopathy Agreements and the applicable product ultimately achieves regulatory approval, could result in the Company owing Vertex certain milestone payments aggregating to high eight digits, subject to certain limitations on the profitability of the program.

Under the A&R Vertex JDCA, for 2022, 2023 and 2024, the Company had an option to defer a portion of its share of costs if spending on the CASGEVY program exceeded $110.3 million, which the Company exercised in each such year, resulting in deferred costs of $221.8 million, in the aggregate. Any deferred amounts under the A&R Vertex JDCA, as amended, are only payable to Vertex as an offset against future profitability of the CASGEVY program and the amounts payable are capped at a specified maximum

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amount per year. These deferred costs on the CASGEVY program will be recognized by us when recoverability of such deferred amounts by Vertex is probable and the amount can be reasonably estimated. As of March 31, 2025, no contingent payments have been accrued to date.

8. Share Capital

All of the Company’s common shares are authorized under Swiss corporate law with a nominal value of 0.03 CHF per share. Though the nominal value of common shares is stated in Swiss francs, the Company continues to use U.S. dollars as its reporting currency for preparing the condensed consolidated financial statements.

As of March 31, 2025, the Company's share capital consists of 89,905,892 registered common shares with a nominal value of CHF 0.03 per share, 8,202,832 registered common shares reserved for potential issuance of bonds or similar instruments and 19,537,850 registered common shares reserved for the Company’s employee equity incentive plans. In addition, the Board of Directors is authorized to conduct one or more increases of the share capital at any time until June 8, 2028, or the expiration of the capital band if earlier, up to an upper limit of CHF 3,142,094.52 by issuing a corresponding number of registered shares with a nominal value of CHF 0.03 each to be fully paid in. As of March 31, 2025, the number of shares that may be issued under the capital band is 14,830,592 registered common shares.

Common Share Issuances

At-the-Market Offerings

In August 2019, the Company entered into an Open Market Sale AgreementSM with Jefferies LLC, or Jefferies, under which the Company was able to offer and sell, from time to time at its sole discretion through Jefferies, as its sales agent, its common shares, par value of CHF 0.03 per share, or the August 2019 Sales Agreement. In January 2021, in connection with the August 2019 Sales Agreement, the Company filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $600.0 million. In August 2024, the Company filed a new prospectus supplement with the SEC, which replaced the previous prospectus supplements filed in January 2021 and July 2021, respectively, to offer and sell, from time to time, the common shares remaining under the original prospectus supplement and July 2021 prospectus supplement having aggregate gross proceeds of up to $378.6 million, or, together with the January 2021 prospectus supplement and July 2021 prospectus supplement, the 2021 ATM.

For the three months ended March 31, 2025, the Company issued and sold an aggregate of 0.2 million common shares under the 2021 ATM at an average price of $54.33 per share for aggregate proceeds of $8.7 million. Activity under the 2021 ATM was not material for the three months ended March 31, 2024. Equity issuance costs, excluding stamp taxes, were not material for the three months ended March 31, 2025 and 2024.

As of March 31, 2025, the Company has issued and sold an aggregate of 2.1 million common shares under the 2021 ATM at an average price of $117.41 per share for aggregate proceeds of $241.9 million, which were net of equity issuance costs of $3.3 million, excluding stamp taxes. As of March 31, 2025, common shares having aggregate gross proceeds up to $354.8 million remain available under the 2021 ATM.

Additional Financings

In February 2024, the Company entered into an investment agreement for the sale of approximately $280.0 million of its common shares to a group of institutional investors in a registered direct offering, at a price per share of $71.50. The Company received net proceeds of $279.0 million, excluding stamp taxes due of $2.8 million.

9. Stock-based Compensation

During the three months ended March 31, 2025 and 2024, the Company recognized the following stock-based compensation expense (in thousands):


	Three Months Ended March 31,
	2025		2024
Research and development	$	9,732	$	11,182
General and administrative		10,480		8,223
Total	$	20,212	$	19,405

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Stock option activity

The following table summarizes stock option activity for the three months ended March 31, 2025:


	Shares			Weighted- average exercise price per share
Outstanding at December 31, 2024		7,288,883		$	58.07
Granted		894,655			42.10
Exercised		(39,996	)		36.65
Cancelled or forfeited		(257,062	)		58.84
Outstanding at March 31, 2025		7,886,480		$	56.34
Exercisable at March 31, 2025		5,039,236		$	58.98
Vested and expected to vest at March 31, 2025		7,886,480		$	56.34

As of March 31, 2025, total unrecognized compensation expense related to stock options was $85.2 million, which the Company expects to recognize over a remaining weighted-average period of 2.8 years.

Restricted stock activity

The following table summarizes restricted stock activity for the three months ended March 31, 2025:


	Shares			Weighted- Average Grant Date Fair Value
Unvested balance at December 31, 2024		2,397,643		$	59.21
Granted		656,721			42.11
Vested		(396,943	)		67.98
Cancelled or forfeited		(129,989	)		61.44
Unvested balance at March 31, 2025		2,527,432		$	53.28

As of March 31, 2025, total unrecognized compensation expense related to unvested restricted common shares was $103.6 million, which the Company expects to recognize over a remaining weighted-average vesting period of 3.0 years.

10. Net Loss Per Share Attributable to Common Shareholders

Basic net loss per share is calculated by dividing net loss attributable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net loss per share is calculated by adjusting weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period using the treasury stock method. For purposes of the diluted net loss per share calculation, stock options, unvested restricted common shares and ESPP shares are considered to be common stock equivalents but are excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive; therefore, basic and diluted net loss per share were the same for all periods presented as a results of the Company’s net loss. The Company’s net loss is net loss attributable to common shareholders for all periods presented.

The Company did not include the securities in the following table in the computation of the net loss per share calculations because the effect would have been anti-dilutive during each period:


	Three Months Ended March 31,
	2025		2024
Outstanding options		7,886,480		7,164,349
Unvested restricted common shares		2,527,432		2,082,032
ESPP		13,330		8,630
Total		10,427,242		9,255,011

11. Income Taxes

During the three months ended March 31, 2025 and 2024, the Company recorded an income tax provision of $1.1 million and $0.7 million, respectively, representing an effective tax rate of (0.8%) and (0.6%), respectively. The income tax provision for the three months ended March 31, 2025 is primarily attributable to the income generated by the Company's U.S. subsidiaries. The difference in the statutory tax rate and effective tax rate is primarily a result of the jurisdictional mix of earnings, research credits generated, and the valuation allowance recorded against certain deferred tax assets. The Company maintains a valuation allowance against certain deferred tax assets that are not more-likely-than-not realizable.

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12. Segment Information

The Company operates as one operating segment, which is the business of discovering, developing and commercializing therapies derived from or incorporating genome-editing technology. The determination of a single business segment is consistent with the consolidated financial information regularly provided to the Company’s chief operating decision maker, or CODM. The Company’s chief executive officer, as the CODM, uses consolidated, single-segment financial information for purposes of evaluating performance, making operating decisions, allocating resources and planning and forecasting for future periods.

The CODM assesses performance and decides how to allocate resources based on consolidated net loss. The measure is used to monitor budget verses actual results to evaluate the performance of the segment.

The measure of segment assets is reported on the condensed consolidated balance sheets as total consolidated assets. All material long-lived assets are located in the United States. Long-lived assets consist of property and equipment, net, and operating lease right-of-use assets.

The following table illustrates information about segment revenue, significant segment expenses and segment operating loss for the periods presented (in thousands):


	Three Months Ended March 31,
	2025			2024
Revenue:	$	865		$	504
Less1:
Research and development expense2		60,333			62,350
General and administrative expense3		6,517			7,551
Collaboration expense, net		57,509			46,966
Stock-based compensation expense		20,212			19,405
Depreciation expense		4,718			4,819
Other segment items4		(12,428	)		(23,996	)
Segment net loss		(135,996	)		(116,591	)
Reconciliation of profit or loss:
Adjustments or reconciling items		—			—
Consolidated net loss		(135,996	)		(116,591	)

(1) The significant expense categories and amounts align with the segment-level information that is regularly provided to the CODM.

(2) Research and development expense for the three months ended March 31, 2025 and 2024 excludes $9.7 million and $11.2 million of stock-based compensation expense, respectively, and $2.4 million and $2.6 million of depreciation expense, respectively.

(3) General and administrative expense for the three months ended March 31, 2025 and 2024 excludes $10.5 million and $8.2 million of stock-based compensation expense, respectively, and $2.3 million and $2.2 million of depreciation expense, respectively.

(4) Other segment items include interest income, net, the change in fair value of corporate equity securities and income tax expense.

The Company operates in the United States and Switzerland. Collaboration revenue is attributed to the CRISPR Therapeutics AG entity, which is domiciled in Switzerland.

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Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with (i) our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and (ii) our audited consolidated financial statements and related notes and management’s discussion and analysis of financial condition and results of operations included in our Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission, or the SEC, on February 11, 2025. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and impact and potential impacts on our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including, without limitation, those factors set forth in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2024 and the “Risk Factors” section of subsequent Quarterly Reports on Form 10-Q, our actual results or timing of certain events could differ materially from the results or timing described in, or implied by, these forward-looking statements.

Overview

Our mission is to create transformative gene-based medicines for serious human diseases. We are a leading gene editing company focused on the development of CRISPR-based therapeutics, including by using CRISPR/Cas9 technology. CRISPR/Cas9 is a revolutionary technology for gene editing, the process of precisely altering specific sequences of genomic DNA. We aim to apply this technology to disrupt, delete, correct and insert genes to treat genetic diseases and to engineer advanced cellular therapies. We have advanced this technology from discovery to an approved medicine with unparalleled speed, culminating in the landmark first approval of a CRISPR-based therapy, CASGEVY (exagamglogene autotemcel [exa-cel]), in 2023 with our collaborators at Vertex Pharmaceuticals Incorporated, or Vertex. We have established a portfolio of therapeutic programs spanning four core franchises: hemoglobinopathies, CAR T, in vivo approaches and type 1 diabetes, or T1D. Depending on the program, we take either an ex vivo approach, in which we edit cells outside of the human body before administering them to the patient, or an in vivo editing approach, where we deliver the CRISPR-based therapeutic directly to target cells within the human body.

We continue to innovate on our platform to develop next-generation technologies that can enable new therapies. Through our efforts, we aim to unlock the full potential of CRISPR-based therapeutics to create medicines that can transform people’s lives. We believe that our innovative research, translational expertise, and clinical development experience, position us as a leader in the development of CRISPR-based therapeutics and may enable us to create an entirely new class of highly effective and potentially curative therapies for patients with both rare and common diseases for whom current biopharmaceutical approaches have had limited success.

Hemoglobinopathies

CASGEVY

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin in red blood cells, which can compensate for the defective adult hemoglobin in patients with SCD and TDT. CASGEVY is the first therapy to emerge from our strategic partnership with Vertex and is being advanced under a joint development and commercialization agreement between us and Vertex and certain of its affiliates.

In 2023, CASGEVY became the first-ever approved CRISPR-based gene-editing therapy in the world. To date, CASGEVY has been approved in the United States, European Union, Great Britain, Canada, Switzerland, Kingdom of Saudi Arabia, Kingdom of Bahrain and the United Arab Emirates for the treatment of eligible patients 12 years and older with SCD or TDT. We and Vertex continue to investigate CASGEVY, including (1) three clinical trials designed to assess the safety and efficacy of a single dose of CASGEVY in patients 12 to 35 years of age with severe SCD and TDT, respectively, (2) two clinical trials in patients 5 to 11 years of age, one in severe SCD and a second in TDT, and (3) long-term follow-up clinical trials designed to follow participants for up to 15 years after CASGEVY infusion. CASGEVY safety data presented to date is generally consistent with an autologous stem cell transplant and myeloablative conditioning. Efficacy data presented to date support the profile of this therapy as a potential one-time functional cure for people with severe SCD and TDT.

Additional candidates

We continue to advance our internally developed targeted conditioning program, as well as in vivo hematopoietic stem cell, or HSC, editing approaches utilizing lipid nanoparticle-mediated delivery through preclinical studies. Both initiatives could significantly expand the addressable patient populations for SCD and TDT.

CAR T

We believe CRISPR/Cas9 has the potential to create the next generation of CAR T cell therapies that may have a superior product profile and allow broader patient access compared to current autologous therapies. We are advancing several cell therapy programs for oncology and/or autoimmune indications, including two next-generation allogeneic CAR T programs, CTX112 targeting

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Cluster of Differentiation 19, or CD19, and CTX131 targeting Cluster of Differentiation 70, or CD70. These product candidates incorporate edits designed to enhance CAR T potency, reduce CAR T exhaustion and evade the immune system. An additional edit in CTX131 is designed to prevent the CAR T cells from killing other CAR T cells. In addition, these next-generation candidates exhibit increased manufacturing robustness, with a higher and more consistent number of CAR T cells produced per batch. We are producing CTX112 and CTX131 for clinical trials at our internal GMP manufacturing facility in Framingham, Massachusetts.

CD19 candidates

CTX112 is being developed for both hematologic malignancies and autoimmune indications. It is being investigated in an ongoing clinical trial designed to assess the safety and efficacy of the product candidate in adult patients with relapsed or refractory B-cell malignancies who have received at least two prior lines of therapy, as well as an ongoing clinical trial in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis. CTX112 has been granted RMAT designation by the FDA for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma.

CD70 candidates

CTX131 is being developed for both solid tumors and hematologic malignancies. It is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the candidate in adult patients with relapsed or refractory solid tumors and hematologic malignancies, including T cell lymphomas, or TCL.We believe allogeneic CAR T approaches for TCL may have greater potential to meet the unmet need in this patient population given the patients’ own T cells are not suitable for autologous manufacturing.

Additional candidates

Our CRISPR/Cas9 platform enables us to innovate continuously by incorporating incremental edits into next-generation products. We are advancing several additional investigational CAR T programs, including an autologous, gene-edited CAR T program targeting glypican-3 for the potential treatment of solid tumors.

In Vivo

Our in vivo gene editing strategy focuses on gene disruption and whole gene correction – the two technologies required to address the vast majority of the most prevalent severe monogenic diseases as well as many common diseases. We have established a leading platform for in vivo gene editing and are rapidly advancing a broad portfolio of in vivo programs, supported by an internal lipid nanoparticle, or LNP, team to enable liver-directed and extrahepatic programs with novel lipids, formulations and targeting moieties. Our first in vivo programs target the liver, taking advantage of validated LNP delivery technologies, and aim to treat diseases where we can produce a strong therapeutic effect by safely disrupting a gene with well-understood genetic association.

Cardiovascular disease

Our first two in vivo programs utilizing our proprietary LNP platform, CTX310 and CTX320, aim to address cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3, or ANGPTL3, and lipoprotein (a), or Lp(a), respectively. CTX310 is being investigated in an ongoing clinical trial targeting ANGPTL3, a gene that encodes for key protein involved in the regulation of low-density lipoprotein and triglyceride levels, in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia. In addition, CTX320 is being investigated in an ongoing clinical trial targeting LPA, the gene encoding apo(a), a critical component of Lp(a) in patients with elevated Lp(a), which has shown to have an independent association with major adverse cardiovascular events.

Additional candidates

Building upon CTX310 and CTX320, we are progressing CTX340, targeting angiotensinogen for refractory hypertension, as well as CTX450, targeting 5’-aminolevulinate synthase 1 for acute hepatic porphyria, through preclinical studies. In addition, we have programs focused on gene correction in the liver, including programs leveraging technologies developed by our CRISPR-X research team. Finally, we are pursuing additional delivery technologies, including LNPs, for delivery to tissues beyond the liver, including hematopoietic stem cells.

Type 1 Diabetes

We are developing gene-edited stem cell-derived therapies for the treatment of T1D. We believe our gene editing capabilities have the potential to enable a beta-cell replacement product candidate that may deliver durable benefit to patients without the need for long-term immunosuppression. We have three parallel efforts to achieve this goal: (1) CTX211, an allogeneic, gene-edited, hypoimmune, stem cell derived product candidate in a device that is implanted into patients and intended to produce insulin in a glucose-dependent manner, and which is in an ongoing clinical trial; (2) CTX213, a research stage deviceless beta cell replacement product candidate consisting of unencapsulated precursor islet cells derived from edited stem cells; and (3) we have granted a non-exclusive license to certain of our CRISPR/Cas9 intellectual property to Vertex to accelerate Vertex’s development of hypoimmune cell therapies for T1D in exchange for certain milestones and royalties.

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CRISPR-X

While we have made significant progress with our current portfolio of programs, we recognize that we need to continue to innovate to unlock the full power of gene editing and bring potentially transformative therapies to even more patients. We have a dedicated early-stage research team called CRISPR-X that focuses on innovating next-generation editing modalities. CRISPR-X is developing technologies to enable whole gene correction and insertion via non-viral DNA delivery and all-RNA systems, without requiring homology-directed repair or viral delivery of DNA.

Partnerships

Given the numerous potential therapeutic applications for CRISPR/Cas9, we have partnered strategically to broaden the indications we can pursue and accelerate development of programs by accessing specific technologies and/or disease-area expertise. We maintain broad partnerships to develop gene editing-based therapeutics in specific disease areas.

Hemoglobinopathies

In 2015, we partnered with Vertex and entered into a strategic collaboration, option and license agreement, which focused on the discovery and development of gene-based treatments for hemoglobinopathies and cystic fibrosis using CRISPR/Cas9 gene-editing technology. In 2017, Vertex exercised its option to co-develop and co-commercialize the hemoglobinopathies program and we entered into a joint development and commercialization agreement with Vertex, which we amended and restated in 2021, pursuant to which, among other things, we are co-developing and co-commercializing CASGEVY for TDT and SCD.

Diabetes

Beginning in 2018, we partnered with ViaCyte, Inc., or ViaCyte (now a wholly-owned subsidiary of Vertex), to pursue the discovery, development and commercialization of gene-edited allogeneic stem cell therapies for the treatment of diabetes. In 2023, ViaCyte elected to opt-out of the collaboration with us for the co-development and co-commercialization of gene-edited stem cell therapies for the treatment of diabetes. Per the opt-out terms, the on-going collaboration assets will be wholly owned by us, subject to a royalty on future sales owed to ViaCyte. Our product candidate, CTX211, being developed for the potential treatment of T1D, resulted from this collaboration, and which we are continuing to advance in a Phase 1 clinical trial. Additionally, in 2023, we entered into a non-exclusive license agreement with Vertex for Vertex to utilize certain of our gene-editing intellectual property to exploit certain products for the diagnosis, treatment or prevention of T1D, diabetes type 2 or insulin dependent/requiring diabetes throughout the world. To date, we have recognized revenue of $205.0 million in upfront and milestone payments and remain eligible to receive additional research and development milestones and royalties on future products under the license.

Other Partnerships

We have entered into a number of additional collaborations and license agreements in other therapeutic areas, including an additional agreement with Vertex for the treatment of Duchenne muscular dystrophy, or DMD, and myotonic dystrophy type 1, or DM1, and others to support and complement our hematopoietic stem cell, CAR T, in vivo and T1D programs and platform, including agreements with: Nkarta, Inc., or Nkarta, to develop and commercialize products leveraging donor-derived, gene-edited CAR-NK cells; Capsida Biotherapeutics, Inc. to develop in vivo gene editing therapies delivered with engineered adeno-associated virus, or AAV, vectors; Roswell Park Comprehensive Cancer Center to advance a gene-edited autologous CAR T program against a new target; MaxCyte, Inc. on ex vivo delivery for our hemoglobinopathy and CAR T programs; CureVac AG on optimized mRNA constructs and manufacturing for certain in vivo programs; and KSQ Therapeutics, Inc. on intellectual property for our allogeneic immuno-oncology programs.

Financial Overview

Since our inception in October 2013, we have devoted substantially all of our resources to our research and development efforts, identifying potential product candidates, undertaking drug discovery and preclinical development activities, building and protecting our intellectual property estate, establishing internal manufacturing capabilities, organizing and staffing our company, business planning, raising capital and providing general and administrative support for these operations. To date, we have primarily financed our operations through private placements of our preferred shares, common share issuances, convertible loans and payments related to certain of our license and collaboration agreements with strategic partners.

We have a history of recurring losses and expect to continue to incur losses for the foreseeable future; however, we have been in a net income position in certain previous years due to certain payments associated with our collaboration and license agreements with Vertex. Our net losses may fluctuate significantly from quarter to quarter and year to year. We anticipate that our expenses will increase as we continue our current research programs and development activities; seek to identify additional research programs and additional product candidates; conduct initial drug application supporting preclinical studies and initiate clinical trials for our product candidates; initiate preclinical testing and clinical trials for any other product candidates we identify and develop; seek regulatory approval for our product candidates; maintain, defend, protect and expand our intellectual property estate; further develop our gene editing platform; hire additional research, clinical and scientific personnel; incur facilities costs associated with such personnel

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growth; continue to develop internal manufacturing capabilities and infrastructure; and incur additional costs associated with operating as a public company.

Revenue Recognition

We have not generated any revenue to date from sales of any wholly-owned product and do not expect to do so in the near future. Revenue recognized for the three months ended March 31, 2025 and 2024 was not material. For additional information about our revenue recognition policy, see Note 2, “Summary of Significant Accounting Policies,” in our Annual Report on Form 10-K for the year ended December 31, 2024 filed with the SEC on February 11, 2025, as well as Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q.

Research and Development Expenses

Research and development expenses consist primarily of costs incurred for our research activities, including our product discovery efforts and the development of our product candidates, which include:

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employee-related expenses, including salaries, benefits and equity-based compensation expense;

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costs of services performed by third parties that conduct research and development and preclinical and clinical activities on our behalf;

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costs of purchasing lab supplies and non-capital equipment used in our preclinical activities and in manufacturing preclinical study materials, as well as supplies and materials used to manufacture clinical drug material;

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consultant fees;

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facility costs, including rent, depreciation and maintenance expenses; and

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fees and other payments related to acquiring and maintaining licenses under our third-party licensing agreements.

Our external research and development expenses support our various preclinical and clinical programs, and, as such, we do not break down external research and development expenses further. Our internal research and development expenses consist of payroll and benefits expenses, facilities expense, and other indirect research and development expenses incurred in support of overall research and development activities and, as such, are not allocated to a specific development stage or therapeutic area. Research and development costs are expensed as incurred. Nonrefundable advance payments for research and development goods or services to be received in the future are deferred and capitalized. The capitalized amounts are expensed as the related goods are delivered or the services are performed. At this time, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of any product candidates we may identify and develop. This is due to the numerous risks and uncertainties associated with developing such product candidates, including the uncertainty of:

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successful completion of preclinical studies and IND-enabling studies;

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successful enrollment in, and completion of, clinical trials;

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receipt of marketing approvals from applicable regulatory authorities;

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establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers;

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obtaining and maintaining patent and trade secret protection and non-patent exclusivity;

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launching commercial sales of the product, if and when approved, whether alone or in collaboration with others;

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acceptance of the product, if and when approved, by patients, the medical community and third-party payors;

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effectively competing with other therapies and treatment options;

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a continued acceptable safety profile following approval;

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enforcing and defending intellectual property and proprietary rights and claims; and

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achieving desirable medicinal properties for the intended indications.

A change in the outcome of any of these variables with respect to the development of any product candidates or the subsequent commercialization of any product candidates we may successfully develop could significantly change the costs, timing and viability associated with the development of that product candidate.

Research and development activities are central to our business model. We expect to continue to incur research and development costs consistent with research and development at companies of our size and stage of development, which may increase in the foreseeable future as our current development programs progress, new programs are added and we continue to prepare regulatory filings. These increases will likely include the costs related to the implementation and expansion of clinical trial sites and related patient enrollment, monitoring, program management and manufacturing expenses for current and future clinical trials.

General and Administrative Expenses

General and administrative expenses consist primarily of employee-related expenses, including salaries, benefits and equity-based compensation, for personnel in executive, finance, accounting, business development, human resources and other general and

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administrative functions. Other significant costs include facility costs not otherwise included in research and development expenses, legal fees relating to patent and corporate matters and fees for accounting and consulting services.

We expect to continue to incur general and administrative expenses consistent with general and administrative functions at research and development companies of our size and stage of development, which may increase in the future to support continued research and development activities, and potential commercialization of our product candidates. In addition, we anticipate ongoing expenses related to the reimbursements of third-party patent related expenses in connection with certain of our in-licensed intellectual property.

Collaboration Expense, Net

Collaboration expense, net, consists of operating expense under our collaboration with Vertex for the hemoglobinopathies program.

Other Income (Expense), Net

Other income (expense), net consists primarily of interest income earned on investments, as well as the change in fair value of corporate equity securities.

Results of Operations

Comparison of three months ended March 31, 2025 and 2024 (in thousands):


	Three Months Ended March 31,						Period to Period
	2025			2024			Change
Revenue:
Collaboration revenue	$	—		$	—		$	—
Grant revenue		865			504			361
Total revenue		865			504			361
Operating expenses:
Research and development		72,484			76,172			(3,688	)
General and administrative		19,296			17,953			1,343
Collaboration expense, net		57,509			46,966			10,543
Total operating expenses		149,289			141,091			8,198
Loss from operations		(148,424	)		(140,587	)		(7,837	)
Other income, net		13,537			24,720			(11,183	)
Loss before income taxes		(134,887	)		(115,867	)		(19,020	)
Provision for income taxes		(1,109	)		(724	)		(385	)
Net loss	$	(135,996	)	$	(116,591	)	$	(19,405	)

Collaboration Revenue

There was no collaboration revenue for the three months ended March 31, 2025 and 2024. Please refer to Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for further information.

Research and Development Expenses

Research and development expenses were $72.5 million for the three months ended March 31, 2025, compared to $76.2 million for the three months ended March 31, 2024. The following table summarizes our research and development expenses for the three months ended March 31, 2025 and 2024, together with the changes in those items in dollars (in thousands):


	Three Months Ended March 31,				Period to Period
	2025		2024		Change
External research and development expenses	$	17,852	$	18,669	$	(817	)
Employee-related expenses		19,735		20,991		(1,256	)
Facility expenses		23,959		22,769		1,190
Stock-based compensation expenses		9,732		11,182		(1,450	)
Other expenses		408		369		39
Sublicense and license fees		798		2,192		(1,394	)
Total research and development expenses	$	72,484	$	76,172	$	(3,688	)

The decrease of approximately $3.7 million was primarily attributable to a decrease in employee-related expenses, including stock-based compensation expenses.

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General and Administrative Expenses

General and administrative expenses were $19.3 million for the three months ended March 31, 2025, compared to general and administrative expenses of $18.0 million for the three months ended March 31, 2024. The increase of approximately $1.3 million was primarily associated with an increase in stock-based compensation expense, offset by a reduction in intellectual property costs and employee-related expenses.

Collaboration Expense, Net

Collaboration expense, net, was $57.5 million for the three months ended March 31, 2025, compared to $47.0 million for the three months ended March 31, 2024. The increase of approximately $10.5 million in collaboration expense, net, was primarily attributable to an increase in cost of goods sold and other commercial costs related to CASGEVY and collaboration expenses related to in vivo HSC editing, offset by our share of product sales.

Other Income, Net

Other income was $13.5 million for the three months ended March 31, 2025, compared to $24.7 million of income for the three months ended March 31, 2024. The decrease of approximately $11.2 million was primarily due to the change in fair value of corporate equity securities in the first quarter of 2025, as well as a decrease in interest income earned on cash, cash equivalents and marketable securities for the three months ended March 31, 2025.

Liquidity and Capital Resources

We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of March 31, 2025, we had $1,855.3 million in cash, cash equivalents and marketable securities, of which approximately $108.7 million was held outside of the United States, and an accumulated deficit of $1,501.9 million. We anticipate that we will continue to incur losses for at least the next several years. We expect to continue to incur research and development costs and general and administrative expenses consistent with costs associated with research and development at companies of our size and stage of development, and, as a result, we will need additional capital to fund our operations, which we may raise through public or private equity or debt financings, strategic collaborations, or other sources.

At-the-Market Offerings

In August 2019, we entered into an Open Market Sale AgreementSM with Jefferies under which we were able to offer and sell, from time to time at our sole discretion through Jefferies, as our sales agent, our common shares, par value of CHF 0.03 per share, or the August 2019 Sales Agreement. In January 2021, in connection with the August 2019 Sales Agreement, we filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $600.0 million. In August 2024, we filed a new prospectus supplement with the SEC, which replaced the previous prospectus supplements filed in January 2021 and July 2021, respectively, to offer and sell, from time to time, the common shares remaining under the original prospectus supplement and July 2021 prospectus supplement having aggregate gross proceeds of up to $378.6 million, or, together with the January 2021 prospectus supplement and July 2021 prospectus supplement, the 2021 ATM.

For the three months ended March 31, 2025, we issued and sold an aggregate of 0.2 million common shares under the 2021 ATM at an average price of $54.33 per share for aggregate proceeds of $8.7 million. Activity under the 2021 ATM was not material for the three months ended March 31, 2024. Equity issuance costs, excluding stamp taxes, were not material for the three months ended March 31, 2025 and 2024.

As of March 31, 2025, we have common shares having aggregate gross proceeds up to $354.8 million remaining under our current prospectus supplement, and we have issued and sold an aggregate of 2.1 million common shares at an average price of $117.41 per share for aggregate proceeds of $241.9 million, which were net of equity issuance costs of $3.3 million, excluding stamp taxes.

Additional Financings

In February 2024, we entered into an investment agreement for the sale of approximately $280.0 million of our common shares to a group of institutional investors in a registered direct offering, at a price per share of $71.50. We received net proceeds of $279.0 million, excluding stamp taxes due of $2.8 million.

Funding Requirements

Our primary uses of capital are, and we expect will continue to be, research and development activities, manufacturing activities, compensation and related expenses, laboratory and related supplies, legal and other regulatory expenses, patent prosecution, filing, defense and intellectual property maintenance costs, and general overhead costs, including costs associated with operating as a public company. We expect to continue to incur operating expenses consistent with costs associated with research and development at companies of our size and stage of development, which may increase in the future to support continued research and development activities and potential commercialization of our product candidates.

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Although we and our partner, Vertex, have received marketing approvals for CASGEVY in certain jurisdictions, we cannot guarantee we and Vertex will receive additional marketing approvals for CASGEVY or we will receive marketing approvals for our other product candidates in the future. Most of our programs are still in early stages of development and the outcome of our efforts is uncertain, and we cannot estimate the actual amounts necessary to successfully complete the development, manufacture and commercialization of any current or future product candidates, if approved, or whether, or when, we may achieve profitability. Until such time as we can generate substantial product revenues, if ever, we expect to finance our cash needs through a combination of equity financings, debt financings and payments received in connection with our collaboration and license agreements. We intend to consider opportunities to raise additional funds through the sale of equity or debt securities when market conditions are favorable to us to do so. However, the trading prices for our common shares and other biopharmaceutical companies have been highly volatile. As a result, we may face difficulties raising capital through sales of our common shares or such sales may be on unfavorable terms. In addition, a recession, depression or other sustained adverse market event, including resulting from the uncertain geopolitical environment, global trade policy and global economic conditions, could materially and adversely affect our business and the value of our common shares. To the extent that we raise additional capital through the future sale of equity or debt securities, the ownership interests of our shareholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing shareholders. If we raise additional funds through license or collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Outlook

Based on our research and development plans and our timing expectations related to the progress of our programs, we expect our existing cash, cash equivalents and marketable securities will enable us to fund our operating expenses and capital expenditures for at least the next 24 months without giving effect to any additional proceeds we may receive under our license agreements and collaborations, including with Vertex, and any other capital raising transactions we may complete. We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we expect. Given our need for additional financing to support the long-term clinical development and future commercialization of our programs, as applicable, we intend to consider additional financing opportunities when market terms are favorable to us.

Our ability to generate revenue and achieve profitability depends significantly on our success in many areas, including: developing our delivery technologies and our gene editing technology platform; selecting appropriate product candidates to develop; completing research and preclinical and clinical development of selected product candidates; obtaining regulatory approvals and marketing authorizations for product candidates for which we complete clinical trials; developing a sustainable and scalable manufacturing process for product candidates; launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; obtaining market acceptance of our product candidates, either directly or with a collaborator or distributor, if approved, including for CASGEVY; addressing any competing technological and market developments; negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter; maintaining good relationships with our collaborators and licensors; maintaining, defending, protecting and expanding our estate of intellectual property rights, including patents, trade secrets and know-how; and attracting, hiring and retaining qualified personnel.

Cash Flows

The following table provides information regarding our cash flows for each of the periods below (in thousands):


	Three Months Ended March 31,						Period to Period
	2025			2024			Change
Net cash (used in) provided by operating activities	$	(53,947	)	$	109,752		$	(163,699	)
Net cash used in investing activities		(19,755	)		(97,800	)		78,045
Net cash provided by financing activities		10,588			305,934			(295,346	)
Effect of exchange rate changes on cash		41			(11	)		52
Net (decrease) increase in cash	$	(63,073	)	$	317,875		$	(380,948	)

Operating Activities

Net cash used in operating activities was $53.9 million for the three months ended March 31, 2025, compared to cash provided by operating activities of $109.8 million for the three months ended March 31, 2024. The $163.7 million increase in net cash used in operating activities was primarily driven by a decrease in net changes of operating assets and liabilities of $155.6 million, primarily due to the impact of receivables from Vertex which were paid during the quarter ($200.0 million paid in the first quarter of 2024 compared to $25.0 million paid in the first quarter of 2025). Additionally, non-cash expense increased by $11.3 million, primarily driven by the change in fair value of corporate equity securities, and our net loss position increased by $19.4 million, from a net loss of $116.6 million for the first quarter of 2024 compared to a net loss of $136.0 million for the first quarter of 2025.

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Investing Activities

Net cash used in investing activities for the three months ended March 31, 2025 was $19.8 million, compared to net cash used in investing activities of $97.8 million for the three months ended March 31, 2024. The decrease in net cash used in investing activities was primarily driven by a net decrease in purchases of our marketable securities.

Financing Activities

Net cash provided by financing activities for the three months ended March 31, 2025 was $10.6 million, compared with $305.9 million for the three months ended March 31, 2024. Net cash provided by financing activities for the three months ended March 31, 2025 consisted primarily of $8.7 million in net proceeds from the sale of common shares issued in connection with our 2021 ATM, as well as option exercise proceeds, net of issuance costs. Net cash provided by financing activities for the three months ended March 31, 2024 consisted of proceeds from the sale of approximately $280.0 million of our common shares to a group of institutional investors in a registered direct offering, at a price per share of $71.50. We received net proceeds of $279.0 million, excluding stamp taxes due of $2.8 million.

Critical Accounting Policies and Estimates

There have been no material changes in our critical accounting policies and estimates in the preparation of our condensed consolidated financial statements during the three months ended March 31, 2025 compared to those discussed in our Annual Report on Form 10-K for the year ended December 31, 2024 filed with the SEC on February 11, 2025, with the exception of changes to the below policy.

Collaboration Arrangements

We record the elements of our collaboration agreements that represent joint operating activities in accordance with ASC 808. Accordingly, the elements of the collaboration agreements that represent activities in which both parties are active participants and to which both parties are exposed to the significant risks and rewards that are dependent on the commercial success of the activities, are recorded as collaborative arrangements.

We evaluate the proper presentation of the commercial activities and the proﬁt and loss sharing associated with the collaboration agreements. ASC 808 states that when payments between parties in a collaborative arrangement are not within the scope of other authoritative accounting literature, the income statement classification should be based on the nature of the arrangement, the nature of its business operations and the contractual terms of the arrangement. To the extent that these payments are not within the scope of other authoritative accounting literature, the income statement classification for the payments shall be based on an analogy to authoritative accounting literature or if there is no appropriate analogy, a reasonable, rational and consistently applied accounting policy election.

Collaboration costs specific to the Vertex Hemoglobinopathy Agreements accounted for under ASC 808 are presented within “collaboration expense, net” in the condensed consolidated statements of operations and comprehensive loss. Refer to Note 6 to these condensed consolidated financial statements for further discussion on the Vertex Hemoglobinopathy Agreements.

Recent Accounting Pronouncements

Refer to Note 1 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for a discussion of recent accounting pronouncements.

Item 3. Qualitative and Quantitative Disclosures about Market Risk

Interest Rate Sensitivity

We are exposed to market risk related to changes in interest rates. As of March 31, 2025, we had cash, cash equivalents and marketable securities of $1,855.3 million, primarily invested in U.S. treasury securities and government agency securities, corporate bonds, commercial paper and money market accounts invested in U.S. government agency securities. Due to the conservative nature of these instruments, we do not believe that we have a material exposure to interest rate risk. If interest rates were to increase or decrease by 1%, the fair value of our investment portfolio would increase or decrease by an immaterial amount.

Foreign Currency Exchange Rate Risk

As a result of our foreign operations, we face exposure to movements in foreign currency exchange rates, primarily the Swiss Franc and British Pound, against the U.S. dollar. The current exposures arise primarily from cash, accounts payable and intercompany receivables and payables. Changes in foreign exchange rates affect our consolidated statement of operations and distort comparisons between periods. To date, foreign currency transaction gains and losses have not been material to our financial statements, and we have not engaged in any foreign currency hedging transactions.

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Inflation

Inflation generally affects us by increasing our cost of labor, clinical trial and manufacturing costs. We do not believe that inflation had a material effect on our business, financial condition or results of operations during the three months ended March 31, 2025 and 2024.

Item 4. Controls and Procedures.

Management’s Evaluation of our Disclosure Controls and Procedures

We maintain disclosure controls and procedures that are designed to ensure that information required to be disclosed in the reports that we file or submit under the Securities and Exchange Act of 1934 is (1) recorded, processed, summarized, and reported within the time periods specified in the SEC’s rules and forms and (2) accumulated and communicated to our management, including our principal executive officer and principal financial officer, to allow timely decisions regarding required disclosure.

As of March 31, 2025, our management, with the participation of our principal executive officer and principal financial officer, evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities and Exchange Act of 1934). Our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives, and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Our principal executive officer and principal financial officer have concluded based upon the evaluation described above that, as of March 31, 2025, our disclosure controls and procedures were effective at the reasonable assurance level.

Changes in Internal Control over Financial Reporting

During the quarter ended March 31, 2025, there were no changes in our internal control over financial reporting, as such term is defined in Rules 13a-15(f) and 15(d)-15(f) promulgated under the Securities Exchange Act of 1934, that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

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PART II—OTHER INFORMATION

Item 1. Legal Proceedings.

In the ordinary course of business, we are from time to time involved in lawsuits, investigations, proceedings and threats of litigation related to, among other things, our intellectual property estate (including certain in-licensed intellectual property), commercial arrangements and other matters. Such proceedings may include quasi-litigation, inter partes administrative proceedings in the U.S. Patent and Trademark Office and the European Patent Office involving our intellectual property estate including certain in-licensed intellectual property. There are currently no claims or actions pending against us that, in the opinion of our management, are likely to have a material adverse effect on our business.

There have been no material developments with respect to the legal proceedings previously disclosed in “Item 3. Legal Proceedings” of our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 filed with the SEC on February 11, 2025.

Item 1A. Risk Factors.

We are updating and supplementing our risk factors previously disclosed in Item 1A of our Annual Report on Form 10-K for the year ended December 31, 2024 filed with the SEC on February 11, 2025 to revise the following risk factors:

Unfavorable Global Economic Conditions and Uncertain Geopolitical Environment Could Adversely Affect Our Business, Financial Condition Or Results Of Operations.

We are a multinational company and we conduct our clinical trials domestically and internationally. Our business, financial condition and results of operations could be adversely affected by general conditions in the global economy, disruption of global financial markets and a recession or market correction, including, for example, as a result of the coronavirus pandemic, uncertain trade policy, political unrest, including as a result of geopolitical tension such as a deterioration in the relationship between the United States and China, escalation of tensions between China and Taiwan, the ongoing military conflict between Russia and Ukraine and related sanctions imposed against Russia, or the Israel-Hamas war, and other global macroeconomic factors such as inflation, interest rate and currency rate fluctuations. Such conditions could reduce our ability to access capital and cause more frequent or acute fluctuations in our share price or that of our sector, which could in the future negatively affect our liquidity and could materially affect our business and the value of our common stock. In addition, domestic and international political uncertainty, including implementation of aggressive trade policies, could impact our ability to conduct our clinical trials. For example, on April 2, 2025, the United States imposed substantial tariffs on most countries throughout the world. The current U.S. administration has threatened to continue to broadly impose tariffs, which could lead to corresponding punitive actions by countries with which the United States trades. Tariffs levied by the United States and other countries may lead to uncertainty in our ability to run clinical trials in preferred jurisdictions, impact our ability to import drug product, as well as raw materials to manufacture drug product domestically, and lead to uncertainty in our ability to supply drug product for our clinical trials, and otherwise increase the costs for us to run our business.

Disruptions At The FDA, The SEC and Other Government Agencies Could Hinder Their Ability To Hire And Retain Key Leadership And Other Personnel, Prevent New Products And Services From Being Developed Or Commercialized In A Timely Manner, Or Otherwise Prevent Those Agencies From Performing Normal Business Functions, Which Could Negatively Impact Our Business And Our Timelines.

Currently, federal agencies in the United States are operating under a continuing resolution that is set to expire on September 30, 2025. Without appropriation of additional funding to federal agencies, our business operations related to our product development for the U.S. market could be impacted. Inadequate funding for the FDA, the SEC and other government agencies, including from government shut downs, or other disruptions to these agencies’ operations, could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business. For example, the current U.S. administration has issued executive orders seeking to greatly reduce the size of the federal workforce, including through layoffs and severance packages offered to employees of federal agencies within the executive branch and independent agencies, including the FDA. Any such reduction in personnel may result in longer review times by the FDA, the SEC and other agencies.

The ability of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, ability to hire and retain key personnel and accept the payment of user fees, shifting policy priorities as a result of changes in the Presidential administration and political appointees tasked to oversee the agency, and statutory, regulatory, and policy changes. Average review times at the agency have fluctuated in recent years as a result. Moreover, disruptions and personnel turnover, as a result of leadership changes, staff reductions or otherwise, at the FDA and other agencies may slow the time necessary for new drugs to be reviewed and/or approved by necessary government agencies, which could adversely affect our business. For example, changes and cuts in FDA staffing also could result in delays in the FDA’s responsiveness or in its ability to review IND submissions or applications, issue regulations or guidance, or implement or enforce regulatory requirements in a timely fashion or at all.

Over the last several years, the U.S. government has shut down several times and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical FDA, SEC and other government employees and stop critical activities. If a prolonged

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government shutdown occurs, or if staffing changes prevent the FDA, the SEC or other regulatory authorities from conducting their regular inspections, reviews, or other regulatory activities, including formal and informal interactions with product developers, it could significantly impact the ability of the FDA, the SEC or other regulatory authority to timely review and process our regulatory submissions, which could have a material adverse effect on our business and our timelines. In addition, government funding of the SEC, and other government agencies on which our operations may rely is subject to the impacts of political events, which are inherently fluid and unpredictable.

There have been no other material changes to the risk factors previously disclosed in Part I, Item 1A (Risk Factors) of our Annual Report on Form 10-K for the year ended December 31, 2024. Please refer to the complete Part I, Item 1A of our Annual Report for additional risks and uncertainties we are facing that may have a material adverse effect on our business prospects, financial condition and results of operations. In addition to the risks described in our Annual Report on Form 10-K, you should carefully consider the other information set forth in this Form 10-Q and the information in our other filings with the SEC, as they could materially affect our business, financial condition or future results of operations.

Item 2. Unregistered Sales of Equity Securities and Use of Proceeds.

None.

Item 3. Defaults Upon Senior Securities.

Not applicable.

Item 4. Mine Safety Disclosures.

Not applicable.

Item 5. Other Information.

(a) On May 6, 2025, we announced initial top-line data from our ongoing clinical trial of CTX310.

CTX310 targets ANGPTL3, a gene that encodes for key protein involved in the regulation of low-density lipoprotein, or LDL, and triglyceride, or TG, levels – both well-established risk factors for atherosclerotic heart disease, or ASCVD. Loss-of-function mutations in ANGPTL3 are associated with significantly reduced levels of LDL and TGs, as well as reduced risk of ASCVD, without adverse effects on overall health. In the United States alone, more than 40 million patients are affected by elevated LDL, severely elevated TGs or both – representing a large addressable patient population. CTX310 is initially focused on a high-risk subset of this group with the greatest unmet medical need and limited effective treatment options.

CTX310 is in an ongoing Phase 1 first-in-human dose escalation clinical trial targeting ANGPTL3 in four patient groups with elevated LDL, TG or both, including homozygous familial hypercholesterolemia , or HoFH, severe hypertriglyceridemia, or sHTG, heterozygous familial hypercholesterolemia, or HeFH, or mixed dyslipidemias, or MDL, with levels of TG (>300 mg/dL) and/or LDL-C (>100 mg/dL); >70 mg/dL for subjects with ASCVD. TG and LDL, both of which are validated as surrogate endpoints for clinical benefit and accepted by regulatory agencies, were assessed at various timepoints.

The top-line data are from the first 10 patients across the first four cohorts (lean body weight-based doses of dose level 1 [0.1 mg/kg], dose level 2 [0.3 mg/kg], dose level 3 [0.6 mg/kg] and dose level 4 [0.8 mg/kg] with at least 30 days of follow-up for each participant as of a data cutoff date of April 16, 2025.

A single dose of CTX310 demonstrated dose-dependent decreases in ANGPTL3, TGs, and LDL. Based upon ANGPTL3 knockdown, dose level 1 and dose level 2 were minimally active doses, whereas treatment at dose level 3 and dose level 4 resulted in reductions of up to 75% of baseline levels in ANGPTL3. CTX310 has been well-tolerated, with no treatment-related severe adverse events and no grade ≥3 adverse events reported. No clinically significant changes in alanine aminotransferase, aspartate aminotransferase, bilirubin or platelets were observed at any dose level. There were no dose-dependent trends in any of these laboratory measurements.

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Individual patient responses highlight the therapeutic potential of CTX310: a dose level 4 patient with sHTG had an 82% reduction in triglycerides from a baseline of 1073 mg/dL at day 30, and a dose level 3 patient with HeFH had an 81% reduction in LDL-C from a baseline of 256 mg/dL at day 90 – supporting the potential for targeted efficacy in high-risk populations.

(b) Not applicable.

(c) From time to time, our officers (as defined in Rule 16a–1(f)) and directors may enter into Rule 10b5-1 or non-Rule 10b5-1 trading arrangements (as each such term is defined in Item 408 of Regulation S-K). During the three months ended March 31, 2025, none of our officers and directors adopted, modified or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement.

Item 6. Exhibits

The exhibits filed as part of this Quarterly Report on Form 10-Q are set forth in the Exhibit Index below.


Exhibit Number		Description of Document

31.1*		Certification of Chief Executive Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002

31.2*		Certification of Chief Financial Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002

32.1*+		Certification pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002

101.INS*		Inline XBRL Instance Document – the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the inline XBRL document.

101.SCH*		Inline XBRL Taxonomy Extension Schema With Embedded Linkbase Documents

104*		Cover Page Interactive Data File (formatted as inline XBRL with applicable taxonomy extension information contained in Exhibits 101)

* Filed herewith.

+ The certification attached as Exhibit 32.1 that accompany this Quarterly Report on Form 10-Q are not deemed filed with the Securities and Exchange Commission and are not to be incorporated by reference into any filing of CRISPR Therapeutics AG under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date of this Form 10-Q, irrespective of any general incorporation language contained in such filing.

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SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.


	CRISPR Therapeutics AG

Dated: May 6, 2025	By:	/s/ Samarth Kulkarni
		Samarth Kulkarni, Ph.D.
		Chief Executive Officer
		(Principal Executive Officer)

Dated: May 6, 2025	By:	/s/ Raju Prasad
		Raju Prasad, Ph.D.
		Chief Financial Officer
		(Principal Financial Officer)