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Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended				6 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Jun. 30, 2024	Mar. 31, 2024	Jun. 30, 2023	Mar. 31, 2023	Jun. 30, 2024	Jun. 30, 2023
Pay vs Performance Disclosure
Net Income (Loss)	$ (126,408)	$ (116,591)	$ (77,740)	$ (53,065)	$ (242,999)	$ (130,805)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

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+ Details

Share Capital	6 Months Ended
Jun. 30, 2024
Equity [Abstract]
Share Capital	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 June 30, 2024, the Company's share capital consists of 88,517,810 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 20,925,932 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 3,100,452.06 by issuing a corresponding number of registered shares with a nominal value of CHF 0.03 each to be fully paid in. As of June 30, 2024, 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 Offering 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, 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 July 2021, the Company filed a new prospectus supplement with the SEC, which replaced the previous prospectus supplement filed in January 2021, to offer and sell, from time to time, the common shares remaining under the original prospectus supplement having aggregate gross proceeds of up to $419.8 million, or, together with the January 2021 prospectus supplement, the 2021 ATM. As of June 30, 2024, the Company has issued and sold an aggregate of 1.5 million common shares under the 2021 ATM at an average price of $139.28 per share for aggregate proceeds of $212.4 million, which were net of equity issuance costs of $2.9 million, excluding stamp taxes. As of June 30, 2024, common shares having aggregate gross proceeds up to $384.7 million remain under the 2021 ATM. Registered Direct Offering 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.

Share Capital

6 Months Ended

Jun. 30, 2024

Equity [Abstract]

Share Capital

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 June 30, 2024, the Company's share capital consists of 88,517,810 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 20,925,932 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 3,100,452.06 by issuing a corresponding number of registered shares with a nominal value of CHF 0.03 each to be fully paid in. As of June 30, 2024, 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 Offering

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, 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 July 2021, the Company filed a new prospectus supplement with the SEC, which replaced the previous prospectus supplement filed in January 2021, to offer and sell, from time to time, the common shares remaining under the original prospectus supplement having aggregate gross proceeds of up to $419.8 million, or, together with the January 2021 prospectus supplement, the 2021 ATM.

As of June 30, 2024, the Company has issued and sold an aggregate of 1.5 million common shares under the 2021 ATM at an average price of $139.28 per share for aggregate proceeds of $212.4 million, which were net of equity issuance costs of $2.9 million, excluding stamp taxes. As of June 30, 2024, common shares having aggregate gross proceeds up to $384.7 million remain under the 2021 ATM.

Registered Direct Offering

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.

- Definition

The entire disclosure for equity.

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+ Details

Stock-based Compensation - Additional Information (Detail) $ in Millions	6 Months Ended
	Jun. 30, 2024 USD ($)
Employee Stock Option
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total unrecognized compensation expense related to stock options	$ 95.1
Unrecognized compensation expense, remaining weighted-average period for recognition	2 years 8 months 12 days
Restricted Common Shares [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation expense, remaining weighted-average period for recognition	2 years 9 months 18 days
Total unrecognized compensation expense	$ 96.2

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

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+ Details

Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 484,472	$ 389,477
Marketable securities	1,517,147	1,304,215
Accounts receivable	0	200,000
Prepaid expenses and other current assets	8,788	14,386
Total current assets	2,010,407	1,908,078
Property and equipment, net	143,031	151,945
Marketable securities, non-current	11,216	1,973
Intangible assets, net	0	16
Restricted cash	11,520	11,591
Operating lease assets	148,538	153,993
Other non-current assets	15,141	1,975
Total assets	2,339,853	2,229,571
Current liabilities:
Accounts payable	9,452	38,147
Accrued expenses	91,883	45,335
Deferred revenue, current	4,759	4,105
Accrued tax liabilities	454	438
Operating lease liabilities	16,451	15,625
Other current liabilities	4,824	5,141
Total current liabilities	127,823	108,791
Deferred revenue, non-current	12,583	14,012
Operating lease liabilities, net of current portion	214,852	223,007
Other non-current liabilities	3,646	958
Total liabilities	358,904	346,768
Commitments and contingencies, see Note 7
Shareholders’ equity:
Common shares, CHF 0.03 nominal value, 132,477,166 and 126,536,183 shares authorized at June 30, 2024 and December 31, 2023, respectively, 85,215,817 and 80,214,694 shares issued at June 30, 2024 and December 31, 2023, respectively, 85,045,501 and 80,044,378 shares outstanding at June 30, 2024 and December 31, 2023, respectively	2,665	2,497
Treasury shares, at cost, 170,316 shares at June 30, 2024 and at December 31, 2023	(62)	(62)
Additional paid-in capital	3,223,924	2,878,155
Accumulated deficit	(1,242,699)	(999,700)
Accumulated other comprehensive (loss) income	(2,879)	1,913
Total shareholders' equity	1,980,949	1,882,803
Total liabilities and shareholders’ equity	$ 2,339,853	$ 2,229,571

Net Loss Per Share Attributable to Common Shareholders

6 Months Ended

Jun. 30, 2024

Earnings Per Share [Abstract]

Net Loss Per Share Attributable to Common Shareholders

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 dividing the net loss attributable to common shareholders by the weighted-average number of common share equivalents outstanding for the period, including any dilutive effect from outstanding stock options and warrants using the treasury stock method. The Company’s net loss is net loss attributable to common shareholders for all periods presented.

The following common stock equivalents were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect:


	Three Months Ended June 30,				Six Months Ended June 30,
	2024		2023		2024		2023
Outstanding options		7,304,423		7,328,032		7,304,423		7,328,032
Unvested restricted common shares		1,990,014		1,524,987		1,990,014		1,524,987
ESPP		21,219		34,177		21,219		34,177
Total		9,315,656		8,887,196		9,315,656		8,887,196

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Jun. 30, 2024 shares
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	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 June 30, 2024, our officers and directors took the following actions with respect to 10b5-1 trading arrangements: On June 11, 2024, Raju Prasad, Ph.D., our Chief Financial Officer, adopted a Rule 10b5-1 trading arrangement with respect to the sale of up to an aggregate of 30,476 common shares of the Company pursuant to the terms of such trading plan. Dr. Prasad's Rule 10b5-1 trading arrangement is active through December 4, 2025. On June 13, 2024, Samarth Kulkarni, Ph.D., our Chief Executive Officer, adopted a Rule 10b5-1 trading arrangement with respect to the sale of up to an aggregate of 90,000 common shares of the Company pursuant to the terms of such trading plan. Dr. Kulkarni's Rule 10b5-1 trading arrangement is active through March 31, 2025.
Dr. Samarth Kulkarni [Member]
Trading Arrangements, by Individual
Name	Samarth Kulkarni
Title	Chief Executive Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	June 13, 2024
Arrangement Duration	291 days
Aggregate Available	90,000
Raju Prasad [Member]
Trading Arrangements, by Individual
Name	Raju Prasad
Title	Chief Financial Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	June 11, 2024
Arrangement Duration	541 days
Aggregate Available	30,476

Basis of Presentation and Significant Accounting Policies (Policies)	6 Months Ended
	Jun. 30, 2024
Accounting Policies [Abstract]
Basis of Presentation	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 and six-month interim periods ended June 30, 2024 and 2023. 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, 2023, which are contained in the 2023 Annual Report on Form 10-K filed with the Securities and Exchange Commission, or the SEC, on February 21, 2024.
Use of Estimates	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. Significant estimates in these consolidated financial statements have been made in connection with revenue recognition and equity-based compensation expense. 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.
New Accounting Pronouncements - Recently Adopted	New Accounting Pronouncements – Recently Adopted From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board, or FASB, or other standard setting bodies that the Company adopts as of the specified effective date. The Company does not believe that the adoption of recently issued standards has or may have a material impact on its condensed consolidated financial statements and disclosures.

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023
Operating activities:
Net loss	$ (242,999)	$ (130,805)
Reconciliation of net loss to net cash used in operating activities:
Depreciation and amortization	9,678	10,012
Equity-based compensation	43,077	42,640
Other non-cash items, net	(16,566)	(5,620)
Acquired in-process research and development	0	1,500
Changes in:
Accounts receivable	200,000	(70,000)
Prepaid expenses and other assets	5,297	15,952
Accounts payable and accrued expenses	18,724	13,637
Deferred revenue	(775)	0
Operating lease assets and liabilities	(1,874)	(1,277)
Other liabilities, net	(392)	(485)
Net cash provided by (used in) operating activities	14,170	(124,446)
Investing activities:
Purchase of property, plant and equipment	(1,428)	(6,614)
Purchase of in-process research and development	0	(1,500)
Investment in equity securities	(12,885)	0
Purchases of marketable securities	(797,509)	(452,363)
Maturities of marketable securities	587,117	795,229
Net cash (used in) provided by investing activities	(224,705)	334,752
Financing activities:
Proceeds from issuance of common shares, net of issuance costs	279,733	0
Proceeds from exercise of options and ESPP contributions, net of issuance costs	25,735	22,169
Net cash provided by financing activities	305,468	22,169
Effect of exchange rate changes on cash	(9)	60
Increase in cash	94,924	232,535
Cash, cash equivalents and restricted cash, beginning of period	401,068	224,060
Cash, cash equivalents and restricted cash, end of period	495,992	456,595
Supplemental disclosure of non-cash investing and financing activities
Property and equipment purchases in accounts payable and accrued expenses	20	1,029
Equity issuance costs in accounts payable, accrued expenses, and other long term liabilities	$ 3,029	$ 293

Marketable Securities

6 Months Ended

Jun. 30, 2024

Investments, Debt and Equity Securities [Abstract]

Marketable Securities

2. Marketable Securities

The following table summarizes cash equivalents and marketable securities held at June 30, 2024 and December 31, 2023 (in thousands), which are recorded at fair value. The table below excludes $188.0 million and $197.8 million of cash at June 30, 2024 and December 31, 2023, respectively.


	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses			Fair Value
June 30, 2024
Cash equivalents:
Money market funds	$	139,868	$	—	$	—		$	139,868
Certificates and term deposits		25,351		—		—			25,351
Commercial paper		125,953		—		(58	)		125,895
Corporate debt securities		1,424		—		—			1,424
U.S. Treasury securities		3,974		—		—			3,974
Total cash equivalents		296,570		—		(58	)		296,512
Marketable securities:
U.S. Treasury securities		11,883		—		(9	)		11,874
Corporate debt securities		1,013,028		669		(2,517	)		1,011,180
Certificates and term deposits		132,499		—		—			132,499
Government-sponsored enterprise securities		243,521		22		(927	)		242,616
Commercial paper		130,273		—		(79	)		130,194
Total marketable securities		1,531,204		691		(3,532	)		1,528,363
Total cash equivalents and marketable securities	$	1,827,774	$	691	$	(3,590	)	$	1,824,875
December 31, 2023
Cash equivalents:
Money market funds	$	185,990	$	—	$	—		$	185,990
U.S. Treasury securities		5,731		—		—			5,731
Total cash equivalents		191,721		—		—			191,721
Marketable securities:
U.S. Treasury securities		22,963		45		—			23,008
Corporate debt securities		883,550		3,367		(1,559	)		885,358
Certificates and term deposits		47,282		—		—			47,282
Government-sponsored enterprise securities		195,106		377		(352	)		195,131
Commercial paper		155,403		32		(26	)		155,409
Total marketable securities		1,304,304		3,821		(1,937	)		1,306,188
Total cash equivalents and marketable securities	$	1,496,025	$	3,821	$	(1,937	)	$	1,497,909

As of June 30, 2024, marketable securities were in a net unrealized loss position of $2.9 million. As of December 31, 2023, marketable securities were in a net unrealized gain position of $1.9 million. The Company has recorded a net unrealized loss of $1.3 million and $4.8 million during the three and six months ended June 30, 2024, respectively, related to its debt securities, which is included in comprehensive loss on the condensed consolidated statements of operations and comprehensive loss. The Company recorded a net unrealized gain of $0.5 million and $6.7 million during the three and six months ended June 30, 2023, respectively, related to its debt securities, which is included in comprehensive loss on the condensed consolidated statements of operations and comprehensive loss.

As of June 30, 2024 and December 31, 2023, the aggregate fair value of marketable securities that were in an unrealized loss position for less than twelve months was $995.5 million and $463.5 million, respectively. As of June 30, 2024 and December 31, 2023, the aggregate fair value of marketable securities that were in an unrealized loss position for more than twelve months was $229.2 million and $138.4 million, respectively. As of June 30, 2024, securities in an unrealized loss position for more than twelve months totaling $11.2 million had maturities beyond one year, which is included in marketable securities, non-current, on the condensed consolidated balance sheet. As of December 31, 2023, securities in an unrealized loss position for more than twelve months totaling $2.0 million had maturities beyond one year, which is included in marketable securities, non-current, on the condensed consolidated balance sheet.

The Company determined that there is no material credit risk associated with the above investments as of June 30, 2024. 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 and six months ended June 30, 2024 and 2023. No available-for-sale debt securities held as of June 30, 2024 had remaining maturities greater than thirty months.

Property and Equipment, net

6 Months Ended

Jun. 30, 2024

Property, Plant and Equipment [Abstract]

Property and Equipment, net

4. Property and Equipment, net

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


	As of
	June 30,			December 31,
	2024			2023
Computer equipment	$	3,739		$	3,739
Furniture, fixtures and other		8,545			8,109
Laboratory equipment		41,903			41,411
Leasehold improvements		143,260			143,260
Construction work in process		8,654			8,859
Total property and equipment, gross		206,101			205,378
Accumulated depreciation		(63,070	)		(53,433	)
Total property and equipment, net	$	143,031		$	151,945

Depreciation expense for the three and six months ended June 30, 2024 was $4.8 million and $9.6 million, respectively. Depreciation expense for the three and six months ended June 30, 2023 was $5.0 million and $10.0 million, respectively.

Document and Entity Information - shares	6 Months Ended
Document and Entity Information - shares	Jun. 30, 2024	Aug. 01, 2024
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Jun. 30, 2024
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q2
Title of 12(b) Security	Common Shares, nominal value CHF 0.03
Trading Symbol	CRSP
Security Exchange Name	NASDAQ
Entity Registrant Name	CRISPR THERAPEUTICS AG
Entity Central Index Key	0001674416
Entity Tax Identification Number	00-0000000
Current Fiscal Year End Date	--12-31
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Common Stock, Shares Outstanding		85,168,158
Entity Current Reporting Status	Yes
Entity Shell Company	false
Entity File Number	001-37923
Entity Address, Address Line One	Baarerstrasse 14
Entity Address, City or Town	Zug
Entity Address, Country	CH
Entity Address, Postal Zip Code	6300
City Area Code	41 (0)41
Local Phone Number	561 32 77
Entity Interactive Data Current	Yes
Entity Incorporation, State or Country Code	V8
Document Quarterly Report	true
Document Transition Report	false

Condensed Consolidated Statements of Shareholders' Equity (Unaudited) - USD ($) $ in Thousands	Total	Common Shares [Member]	Treasury Shares [Member]	Additional Paid-in Capital [Member]	Accumulated Deficit [Member]	Accumulated Other Comprehensive Loss [Member]
Beginning balance at Dec. 31, 2022	$ 1,875,479	$ 2,441	$ (63)	$ 2,734,838	$ (846,090)	$ (15,647)
Beginning balance, Shares at Dec. 31, 2022		78,512,450	180,316
Vesting of restricted shares, Value	5	$ 5
Vesting of restricted shares, Shares		172,995
Exercise of vested options, net of issuance costs, Value	4,683	$ 6		4,677
Exercise of vested options, net of issuance costs, Shares		159,184
Purchase of common stock under ESPP, Value	660			660
Purchase of common stock under ESPP, Shares		19,105
Stock-based compensation expense	20,875			20,875
Other comprehensive income (loss)	6,259					6,259
Net Income (Loss)	(53,065)				(53,065)
Ending balance at Mar. 31, 2023	1,854,896	$ 2,452	$ (63)	2,761,050	(899,155)	(9,388)
Ending balance, Shares at Mar. 31, 2023		78,863,734	180,316
Beginning balance at Dec. 31, 2022	1,875,479	$ 2,441	$ (63)	2,734,838	(846,090)	(15,647)
Beginning balance, Shares at Dec. 31, 2022		78,512,450	180,316
Net Income (Loss)	(130,805)
Ending balance at Jun. 30, 2023	1,816,028	$ 2,474	$ (63)	2,799,420	(976,895)	(8,908)
Ending balance, Shares at Jun. 30, 2023		79,372,366	180,316
Beginning balance at Mar. 31, 2023	1,854,896	$ 2,452	$ (63)	2,761,050	(899,155)	(9,388)
Beginning balance, Shares at Mar. 31, 2023		78,863,734	180,316
Vesting of restricted shares, Value	4	$ 4
Vesting of restricted shares, Shares		97,631
Exercise of vested options, net of issuance costs, Value	16,623	$ 18		16,605
Exercise of vested options, net of issuance costs, Shares		411,001
Stock-based compensation expense	21,765			21,765
Other comprehensive income (loss)	480					480
Net Income (Loss)	(77,740)				(77,740)
Ending balance at Jun. 30, 2023	1,816,028	$ 2,474	$ (63)	2,799,420	(976,895)	(8,908)
Ending balance, Shares at Jun. 30, 2023		79,372,366	180,316
Beginning balance at Dec. 31, 2023	1,882,803	$ 2,497	$ (62)	2,878,155	(999,700)	1,913
Beginning balance, Shares at Dec. 31, 2023		80,044,378	170,316
Issuance of common shares, Value	277,147	$ 132		277,015
Issuance of common shares, Shares		3,929,610
Vesting of restricted shares, Value	7	$ 7
Vesting of restricted shares, Shares		214,913
Exercise of vested options, net of issuance costs, Value	23,866	$ 22		23,844
Exercise of vested options, net of issuance costs, Shares		632,683
Purchase of common stock under ESPP, Value	764			764
Purchase of common stock under ESPP, Shares		16,026
Stock-based compensation expense	19,405			19,405
Other comprehensive income (loss)	(3,465)					(3,465)
Net Income (Loss)	(116,591)				(116,591)
Ending balance at Mar. 31, 2024	2,083,936	$ 2,658	$ (62)	3,199,183	(1,116,291)	(1,552)
Ending balance, Shares at Mar. 31, 2024		84,837,610	170,316
Beginning balance at Dec. 31, 2023	$ 1,882,803	$ 2,497	$ (62)	2,878,155	(999,700)	1,913
Beginning balance, Shares at Dec. 31, 2023		80,044,378	170,316
Exercise of vested options, net of issuance costs, Shares	677,561
Net Income (Loss)	$ (242,999)
Ending balance at Jun. 30, 2024	1,980,949	$ 2,665	$ (62)	3,223,924	(1,242,699)	(2,879)
Ending balance, Shares at Jun. 30, 2024		85,045,501	170,316
Beginning balance at Mar. 31, 2024	2,083,936	$ 2,658	$ (62)	3,199,183	(1,116,291)	(1,552)
Beginning balance, Shares at Mar. 31, 2024		84,837,610	170,316
Vesting of restricted shares, Value	6	$ 6
Vesting of restricted shares, Shares		163,013
Exercise of vested options, net of issuance costs, Value	1,070	$ 1		1,069
Exercise of vested options, net of issuance costs, Shares		44,878
Stock-based compensation expense	23,672			23,672
Other comprehensive income (loss)	(1,327)					(1,327)
Net Income (Loss)	(126,408)				(126,408)
Ending balance at Jun. 30, 2024	$ 1,980,949	$ 2,665	$ (62)	$ 3,223,924	$ (1,242,699)	$ (2,879)
Ending balance, Shares at Jun. 30, 2024		85,045,501	170,316

Accrued Expenses (Tables)

6 Months Ended

Jun. 30, 2024

Payables and Accruals [Abstract]

Schedule of Accrued Expenses

Accrued expenses consist of the following (in thousands):


	As of
	June 30,		December 31,
	2024		2023
Payroll and employee-related costs	$	14,373	$	17,347
Research and development costs		19,182		16,962
Collaboration costs		51,455		2,395
Licensing fees		2,325		3,143
Professional fees		2,661		2,515
Intellectual property costs		1,379		1,642
Accrued property and equipment		—		630
Other		508		701
Total	$	91,883	$	45,335

Stock-based Compensation

6 Months Ended

Jun. 30, 2024

Share-Based Payment Arrangement [Abstract]

Stock-based Compensation

9. Stock-based Compensation

During the three and six months ended June 30, 2024 and 2023, the Company recognized the following stock-based compensation expense (in thousands):


	Three Months Ended June 30,				Six Months Ended June 30,
	2024		2023		2024		2023
Research and development	$	13,411	$	13,322	$	24,593	$	24,998
General and administrative		10,261		8,443		18,484		17,642
Total	$	23,672	$	21,765	$	43,077	$	42,640

Stock option activity

The following table summarizes stock option activity for the six months ended June 30, 2024:


	Shares			Weighted- average exercise price per share
Outstanding at December 31, 2023		7,204,372		$	55.05
Granted		1,056,224			68.76
Exercised		(677,561	)		37.92
Cancelled or forfeited		(278,612	)		72.96
Outstanding at June 30, 2024		7,304,423		$	57.94
Exercisable at June 30, 2024		4,714,519		$	56.50
Vested and expected to vest at June 30, 2024		7,304,423		$	57.94

As of June 30, 2024, total unrecognized compensation expense related to stock options was $95.1 million, which the Company expects to recognize over a remaining weighted-average period of 2.7 years.

Restricted stock activity

The following table summarizes restricted stock activity for the six months ended June 30, 2024:


	Shares			Weighted- Average Grant Date Fair Value
Unvested balance at December 31, 2023		1,781,415		$	61.00
Granted		706,844			70.50
Vested		(377,926	)		71.41
Cancelled or forfeited		(120,319	)		54.48
Unvested balance at June 30, 2024		1,990,014		$	62.79

As of June 30, 2024, total unrecognized compensation expense related to unvested restricted common shares was $96.2 million, which the Company expects to recognize over a remaining weighted-average vesting period of 2.8 years.

Marketable Securities - Additional Information (Detail) - USD ($)	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023	Dec. 31, 2023
Marketable Securities [Line Items]
Marketable securities	$ 188,000,000		$ 188,000,000		$ 197,800,000
Marketable securities unrealized loss, Less than twelve months	995,500,000		995,500,000		463,500,000
Marketable securities unrealized loss, more than twelve months	229,200,000		229,200,000		138,400,000
Marketable securities totaling	11,216,000		11,216,000		1,973,000
Marketable securities unrealized loss	2,900,000		2,900,000
Marketable securities unrealized gain					$ 1,900,000
Available-for-sale debt securities remaining maturities greater than thirty months	0		0
Other Comprehensive Income (Loss) [Member]
Marketable Securities [Line Items]
Unrealized loss, net	$ (1,300,000)	$ 500,000	$ (4,800,000)	$ 6,700,000

Stock-based Compensation (Tables)

6 Months Ended

Jun. 30, 2024

Share-Based Payment Arrangement [Abstract]

Schedule of Stock-Based Compensation Expense

During the three and six months ended June 30, 2024 and 2023, the Company recognized the following stock-based compensation expense (in thousands):


	Three Months Ended June 30,				Six Months Ended June 30,
	2024		2023		2024		2023
Research and development	$	13,411	$	13,322	$	24,593	$	24,998
General and administrative		10,261		8,443		18,484		17,642
Total	$	23,672	$	21,765	$	43,077	$	42,640

Summary of Stock Option Activity

The following table summarizes stock option activity for the six months ended June 30, 2024:


	Shares			Weighted- average exercise price per share
Outstanding at December 31, 2023		7,204,372		$	55.05
Granted		1,056,224			68.76
Exercised		(677,561	)		37.92
Cancelled or forfeited		(278,612	)		72.96
Outstanding at June 30, 2024		7,304,423		$	57.94
Exercisable at June 30, 2024		4,714,519		$	56.50
Vested and expected to vest at June 30, 2024		7,304,423		$	57.94

Summary of Restricted Stock Activity

The following table summarizes restricted stock activity for the six months ended June 30, 2024:


	Shares			Weighted- Average Grant Date Fair Value
Unvested balance at December 31, 2023		1,781,415		$	61.00
Granted		706,844			70.50
Vested		(377,926	)		71.41
Cancelled or forfeited		(120,319	)		54.48
Unvested balance at June 30, 2024		1,990,014		$	62.79

Significant Contracts - Additional Information (Detail) - USD ($)	1 Months Ended	3 Months Ended			6 Months Ended		12 Months Ended
	May 31, 2024	Jun. 30, 2024	Jun. 30, 2023	Mar. 31, 2023	Jun. 30, 2024	Jun. 30, 2023	Dec. 31, 2023	Dec. 31, 2019	Dec. 31, 2021
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Non-current deferred revenue		$ 12,583,000			$ 12,583,000		$ 14,012,000
Current deferred revenue		4,759,000			4,759,000		4,105,000
Collaboration Expense		52,131,000	$ 44,636,000		99,097,000	$ 86,828,000
A&R Vertex JDCA [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Percentage of net profits and net losses									40.00%
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Business Combination, Consideration Transferred			70,000,000
Milestone payment receivable							200,000,000
Research milestone payment			70,000,000
Collaboration Expense		52,100,000	44,600,000		99,100,000	86,800,000
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] \| Collaboration Target Options [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Milestone payment receivable		410,000,000			410,000,000
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] \| 2019 Collaboration Agreement [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Percentage of exchange payment of research and development costs								50.00%
Maximum potential future payments					775,000,000
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] \| A&R Vertex JDCA [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Up-front payment received							900,000,000
Percentage of net profits and net losses									60.00%
Current payment due							20,000,000
Revenue		0	70,000,000		0	170,000,000	170,000,000
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] \| Non Ex License Agreement [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Up-front payment received				$ 100,000,000
Milestone payment receivable		160,000,000			160,000,000				$ 160,000,000
Research milestone payment			70,000,000
Revenue							100,000,000
Vertex Pharmaceuticals Inc [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Proceeds from letter agreement	$ 43,000,000
Net proceeds from transfer	42.00%
Non-current deferred revenue		12,300,000			12,300,000
Transaction price allocated to remaining performance obligations							12,300,000
Current deferred revenue		0			0		$ 0
Reimbursements from research and license agreements		$ 500,000	$ 2,400,000		1,300,000	$ 5,200,000
Vertex Pharmaceuticals Inc [Member] \| A&R Vertex JDCA [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Up-front payment received					$ 100,000,000

Stock-based Compensation - Schedule of Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Stock-based compensation expense	$ 23,672	$ 21,765	$ 43,077	$ 42,640
Research and Development [Member]
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Stock-based compensation expense	13,411	13,322	24,593	24,998
General and Administrative [Member]
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Stock-based compensation expense	$ 10,261	$ 8,443	$ 18,484	$ 17,642

Fair Value Measurements - Schedule of Financial Assets Measured at Fair Value on Recurring Basis (Detail) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Marketable securities, fair value	$ 1,528,363	$ 1,306,188
Recurring Basis [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total	2,012,835	1,695,665
Recurring Basis [Member] \| Cash [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	187,960	197,756
Recurring Basis [Member] \| Money Market Funds [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	139,868	185,990
Recurring Basis [Member] \| Commercial Paper [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	125,895
Marketable securities, fair value	130,194	155,409
Recurring Basis [Member] \| US Treasury Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	3,974	5,731
Marketable securities, fair value	11,874	23,008
Recurring Basis [Member] \| Corporate Debt Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	1,424
Marketable securities, fair value	1,011,180	885,358
Recurring Basis [Member] \| Certificates and Term Deposits [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	25,351
Marketable securities, fair value	132,499	47,282
Recurring Basis [Member] \| Government-sponsored Enterprise Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Marketable securities, fair value	242,616	195,131
Recurring Basis [Member] \| Level 1 [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total	327,828	383,746
Recurring Basis [Member] \| Level 1 [Member] \| Cash [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	187,960	197,756
Recurring Basis [Member] \| Level 1 [Member] \| Money Market Funds [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	139,868	185,990
Recurring Basis [Member] \| Level 2 [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total	1,685,007	1,311,919
Recurring Basis [Member] \| Level 2 [Member] \| Commercial Paper [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	125,895
Marketable securities, fair value	130,194	155,409
Recurring Basis [Member] \| Level 2 [Member] \| US Treasury Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	3,974	5,731
Marketable securities, fair value	11,874	23,008
Recurring Basis [Member] \| Level 2 [Member] \| Corporate Debt Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	1,424
Marketable securities, fair value	1,011,180	885,358
Recurring Basis [Member] \| Level 2 [Member] \| Certificates and Term Deposits [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	25,351
Marketable securities, fair value	132,499	47,282
Recurring Basis [Member] \| Level 2 [Member] \| Government-sponsored Enterprise Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Marketable securities, fair value	242,616	$ 195,131
Recurring Basis [Member] \| Level 3 [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total	$ 0

Significant Contracts

6 Months Ended

Jun. 30, 2024

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Significant Contracts

6. Significant Contracts

Agreements with Vertex

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 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, the A&R Vertex JDCA allows the Company to defer a portion of its share of costs under the arrangement if spending on the CASGEVY program exceeds specified amounts through 2024. 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. 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 connection with the closing of the transaction contemplated by the A&R Vertex JDCA, the Company received a $900.0 million up-front payment from Vertex. Additionally, in December 2023, the Company and Vertex received approval of CASGEVY by the U.S. Food and Drug Administration, or the FDA. The FDA’s approval of CASGEVY triggered Vertex’s obligation to make a $200.0 million milestone payment to the Company, which was recognized in December 2023 and for which payment was received in January 2024 and was included in accounts receivable in the accompanying condensed consolidated balance sheets as of December 31, 2023.

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.

DMD and DM1 exclusive license

In 2019, the Company and Vertex entered into a series of agreements, including a strategic collaboration and license agreement, or the 2019 Collaboration Agreement, for the development and commercialization of products for the treatment of Duchenne muscular dystrophy, or DMD, and myotonic dystrophy Type 1, or DM1. For the DMD and DM1 programs, Vertex is responsible for all research, development, manufacturing and commercialization activities and all related costs. Upon IND filing, the Company has the option to forego the DM1 milestones and royalties, and instead, co-develop and co-commercialize all DM1 products globally in exchange for payment of 50% of research and development costs incurred by Vertex from the effective date of the agreement through IND filing.

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 connection with entering into the Non-Ex License Agreement, the Company received a $100.0 million up front payment from Vertex in the first quarter of 2023 and subsequently received a $70.0 million research milestone achieved in the second quarter of 2023. The Company is eligible to receive additional milestone payments of up to $160.0 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 and 2019 Collaboration Agreement are collectively referred to as the “Vertex Agreements” and the Non-Ex License Agreement and ViaCyte JDCA Amendment are collectively referred to as the “March 2023 Agreements.”

The Vertex Agreements and the March 2023 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, Collaborative Agreements, or 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 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.

Accounting Analysis Under ASC 606

March 2023 Agreements

Identification of the Contract

The March 2023 Agreements were negotiated as a package with a single commercial objective and, as such, the March 2023 Agreements were combined for accounting purposes and treated as a single arrangement. The Company determined for accounting purposes that the combined contract terminates the original ViaCyte JDCA and created a new contract.

Identification of Performance Obligations

The Company concluded the transfer of the non-exclusive license, including certain modified rights and obligations provided as part of the ViaCyte JDCA Amendment to support the delivery of the license, was both capable of being distinct and distinct within the context of the contract.

Determination of Transaction Price

The initial transaction price was comprised of the upfront payment of $100.0 million.

In the second quarter of 2023, the Company adjusted the transaction price to include $70.0 million in previously constrained variable consideration related to a research milestone which was achieved in the second quarter of 2023. The Company determined that all other possible variable consideration resulting from milestones and royalties discussed below was fully constrained as of June 30, 2024. The Company will re-evaluate the transaction price in each reporting period.

Allocation of Transaction Price to Performance Obligations

The Company identified one performance obligation for the March 2023 Agreements and, as a result, no allocation of the transaction price was required.

Recognition of Revenue

The Company determined the non-exclusive license, including certain modified rights and obligations provided as part of the ViaCyte JDCA Amendment to support the delivery of the license, represented functional intellectual property, as the intellectual property provides Vertex with the ability to perform a function or task in the form of research and development in the field of diabetes. In 2023, the Company recognized revenue of $100.0 million for the non-exclusive license at the onset of the arrangement, as this was the point in time in which the non-exclusive license was delivered.

In 2023, revenue from variable consideration of $70.0 million was recognized related to a research milestone that was achieved during the second quarter of 2023. Revenue recognized under the March 2023 Agreements for year ended December 31, 2023 was $170.0 million in aggregate.

No revenue was recognized under the March 2023 Agreements for the three and six months ended June 30, 2024. Revenue recognized under the March 2023 Agreements for the three and six months ended June 30, 2023 was $70.0 million and $170.0 million, respectively.

Milestones under the Non-Ex License Agreement

As of June 30, 2024, the Company is eligible to receive potential future milestone payments from Vertex of up to $160.0 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 June 30, 2024. 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.

Vertex Agreements

Deferred revenue

As of June 30, 2024 and December 31, 2023, there was no current deferred revenue related to the Vertex Agreements. As of June 30, 2024, there was $12.3 million of non-current deferred revenue related to the Vertex Agreements, which is unchanged from December 31, 2023. The transaction price allocated to the remaining performance obligations was $12.3 million.

Milestones

The Company has evaluated the milestones that may be received in connection with the Vertex 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 is eligible to receive potential future payments of up to $775.0 million under the 2019 Collaboration Agreement based upon the successful achievement of specified development, regulatory and commercial milestones for the DMD and DM1 programs. 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.

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

Each of the remaining milestones described above are fully constrained as of June 30, 2024. 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.

Accounting Analysis under ASC 808

Vertex Agreements

In connection with the Vertex 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 is included within collaboration expense, net, in the condensed consolidated statements of operations and comprehensive loss. During the three and six months ended June 30, 2024, the Company recognized $52.1 million and $99.1 million of collaboration expense, net, related to the CASGEVY program, respectively. Collaboration expense, net, during the three and six months ended June 30, 2024 was net of $0.5 million and $1.3 million of reimbursements from Vertex related to the CASGEVY program, respectively. During the three and six months ended June 30, 2023, the Company recognized $44.6 million and $86.8 million of collaboration expense, net, related to the CASGEVY program, respectively. Collaboration expense, net, during the three

and six months ended June 30, 2023 was net of $2.4 million and $5.2 million of reimbursements from Vertex related to the CASGEVY program, respectively.

Marketable Securities (Tables)

6 Months Ended

Jun. 30, 2024

Investments, Debt and Equity Securities [Abstract]

Summary of Cash Equivalents and Marketable Securities Recorded at Fair Value


	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses			Fair Value
June 30, 2024
Cash equivalents:
Money market funds	$	139,868	$	—	$	—		$	139,868
Certificates and term deposits		25,351		—		—			25,351
Commercial paper		125,953		—		(58	)		125,895
Corporate debt securities		1,424		—		—			1,424
U.S. Treasury securities		3,974		—		—			3,974
Total cash equivalents		296,570		—		(58	)		296,512
Marketable securities:
U.S. Treasury securities		11,883		—		(9	)		11,874
Corporate debt securities		1,013,028		669		(2,517	)		1,011,180
Certificates and term deposits		132,499		—		—			132,499
Government-sponsored enterprise securities		243,521		22		(927	)		242,616
Commercial paper		130,273		—		(79	)		130,194
Total marketable securities		1,531,204		691		(3,532	)		1,528,363
Total cash equivalents and marketable securities	$	1,827,774	$	691	$	(3,590	)	$	1,824,875
December 31, 2023
Cash equivalents:
Money market funds	$	185,990	$	—	$	—		$	185,990
U.S. Treasury securities		5,731		—		—			5,731
Total cash equivalents		191,721		—		—			191,721
Marketable securities:
U.S. Treasury securities		22,963		45		—			23,008
Corporate debt securities		883,550		3,367		(1,559	)		885,358
Certificates and term deposits		47,282		—		—			47,282
Government-sponsored enterprise securities		195,106		377		(352	)		195,131
Commercial paper		155,403		32		(26	)		155,409
Total marketable securities		1,304,304		3,821		(1,937	)		1,306,188
Total cash equivalents and marketable securities	$	1,496,025	$	3,821	$	(1,937	)	$	1,497,909

Income Taxes - Additional Information (Detail) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Income Tax Disclosure [Abstract]
Income tax provision (benefit)	$ 1,287	$ 923	$ 2,011	$ 2,243
Effective income tax rate	(1.10%)	(1.20%)	(0.90%)	(1.70%)

- Definition

Amount of current income tax expense (benefit) and deferred income tax expense (benefit) pertaining to continuing operations.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 270
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (i)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482964/270-10-50-1

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (ee)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482810/280-10-50-32

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-12

Reference 4: http://www.xbrl.org/2003/role/exampleRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 231
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482663/740-10-55-231

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483443/250-10-50-9

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 6.I.7)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479360/740-10-S99-1

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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-Paragraph 8
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-URI https://asc.fasb.org/1943274/2147483443/250-10-50-8

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
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-Name Accounting Standards Codification
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-10

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 22
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482810/280-10-50-22

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 944
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.7-04(9))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477250/944-220-S99-1

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
-Section 45
-Paragraph 2
-Subparagraph (a)
-SubTopic 20
-Topic 740
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482659/740-20-45-2

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(h))
-SubTopic 10
-Topic 235
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-1

+ Details

Fair Value Measurements (Tables)

6 Months Ended

Jun. 30, 2024

Fair Value Disclosures [Abstract]

Schedule of Financial Assets Measured at Fair Value on Recurring Basis

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 June 30, 2024 and December 31, 2023 (in thousands):


	Fair Value Measurements at
	June 30, 2024
	Total		Level 1		Level 2		Level 3
Cash and cash equivalents:
Cash	$	187,960	$	187,960	$	—	$	—
Money market funds		139,868		139,868		—		—
Certificates and term deposits		25,351		—		25,351		—
Commercial paper		125,895		—		125,895		—
Corporate debt securities		1,424		—		1,424		—
U.S. Treasury securities		3,974		—		3,974		—
Marketable securities:
U.S. Treasury securities		11,874		—		11,874		—
Corporate debt securities		1,011,180		—		1,011,180		—
Certificates and term deposits		132,499		—		132,499		—
Government-sponsored enterprise securities		242,616		—		242,616		—
Commercial paper		130,194		—		130,194		—
Total	$	2,012,835	$	327,828	$	1,685,007	$	—

	Fair Value Measurements at
	December 31, 2023
	Total		Level 1		Level 2		Level 3
Cash and cash equivalents:
Cash	$	197,756	$	197,756	$	—	$	—
Money market funds		185,990		185,990		—		—
U.S. Treasury securities		5,731		—		5,731		—
Marketable securities:
U.S. Treasury securities		23,008		—		23,008		—
Corporate debt securities		885,358		—		885,358		—
Certificates and term deposits		47,282		—		47,282		—
Government-sponsored enterprise securities		195,131		—		195,131		—
Commercial paper		155,409		—		155,409		—
Total	$	1,695,665	$	383,746	$	1,311,919	$	—

Share Capital - Additional Information (Detail) SFr / shares in Units, $ / shares in Units, $ in Thousands	1 Months Ended			6 Months Ended
	Feb. 29, 2024 USD ($) $ / shares	Jul. 31, 2021 USD ($)	Jan. 31, 2021 USD ($)	Jun. 30, 2024 USD ($) $ / shares	Jun. 30, 2023 USD ($)	Jun. 30, 2024 SFr / shares shares	Dec. 31, 2023 SFr / shares	Jun. 08, 2023 CHF (SFr) SFr / shares
Class of Stock [Line Items]
Common shares, nominal value \| (per share)	$ 71.5					SFr 0.03	SFr 0.03
Proceeds from issuance of common shares	$ 280,000			$ 279,733	$ 0
Common shares employee equity incentive plans \| shares						20,925,932
Received net proceeds	279,000
Stamp taxes due on issuance of common stock	$ 2,800
Bond [Member]
Class of Stock [Line Items]
Registered common shares \| shares						8,202,832
Common Shares [Member]
Class of Stock [Line Items]
Common shares, nominal value \| SFr / shares						SFr 0.03		SFr 0.03
Registered share capital \| shares						88,517,810
Capital Band [Member]
Class of Stock [Line Items]
Shares, Issued \| shares						14,830,592
Maximum [Member] \| Common Shares [Member]
Class of Stock [Line Items]
Common Stock Share Capital Value \| SFr								SFr 3,100,452.06
Sales Agreement With Jefferies LLC [Member] \| 2021 ATM [Member]
Class of Stock [Line Items]
Average issue price of common shares \| $ / shares				$ 139.28
Proceeds from issuance of common shares				$ 212,400
Equity issuance costs				2,900
Shares, Issued \| shares						1,500,000
Sales Agreement With Jefferies LLC [Member] \| Maximum [Member] \| 2021 ATM [Member]
Class of Stock [Line Items]
Aggregate estimated gross proceeds		$ 419,800	$ 600,000	$ 384,700

Fair Value Measurements

6 Months Ended

Jun. 30, 2024

Fair Value Disclosures [Abstract]

Fair Value Measurements

3. Fair Value Measurements


	Fair Value Measurements at
	June 30, 2024
	Total		Level 1		Level 2		Level 3
Cash and cash equivalents:
Cash	$	187,960	$	187,960	$	—	$	—
Money market funds		139,868		139,868		—		—
Certificates and term deposits		25,351		—		25,351		—
Commercial paper		125,895		—		125,895		—
Corporate debt securities		1,424		—		1,424		—
U.S. Treasury securities		3,974		—		3,974		—
Marketable securities:
U.S. Treasury securities		11,874		—		11,874		—
Corporate debt securities		1,011,180		—		1,011,180		—
Certificates and term deposits		132,499		—		132,499		—
Government-sponsored enterprise securities		242,616		—		242,616		—
Commercial paper		130,194		—		130,194		—
Total	$	2,012,835	$	327,828	$	1,685,007	$	—

	Fair Value Measurements at
	December 31, 2023
	Total		Level 1		Level 2		Level 3
Cash and cash equivalents:
Cash	$	197,756	$	197,756	$	—	$	—
Money market funds		185,990		185,990		—		—
U.S. Treasury securities		5,731		—		5,731		—
Marketable securities:
U.S. Treasury securities		23,008		—		23,008		—
Corporate debt securities		885,358		—		885,358		—
Certificates and term deposits		47,282		—		47,282		—
Government-sponsored enterprise securities		195,131		—		195,131		—
Commercial paper		155,409		—		155,409		—
Total	$	1,695,665	$	383,746	$	1,311,919	$	—

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 and commercial paper. The Company estimates the fair value of these marketable securities by taking into consideration valuations obtained from third-party pricing sources.

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References

Reference 1: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 107
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-107

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 100
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-100

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(3)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2E
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2E

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (g)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 940
-SubTopic 820
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478119/940-820-50-1

+ Details

Commitments and Contingencies - Additional Information (Detail) - USD ($) $ in Millions	6 Months Ended	12 Months Ended
	Jun. 30, 2024	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Contingencies And Commitments [Line Items]
Defer option specified cost			$ 110.3	$ 110.3
Deferred Finance Costs, Share Lending Arrangement, Issuance Costs			80.9	$ 36.1
Scenario Forecast [Member]
Contingencies And Commitments [Line Items]
Defer option specified cost		$ 110.3
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member]
Contingencies And Commitments [Line Items]
Milestone payment receivable			$ 200.0
Cost excluded from license agreement	$ 4.0
Letter of Credit [Member]
Contingencies And Commitments [Line Items]
Letters of credit secured by cash held in restricted depository account	11.5
2015 Collaboration Agreement [Member] \| Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member]
Contingencies And Commitments [Line Items]
Milestone payment receivable	$ 395.0

Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Accrued Liabilities, Current [Abstract]
Payroll and employee-related costs	$ 14,373	$ 17,347
Research costs	19,182	16,962
Collaboration costs	51,455	2,395
Licensing fees	2,325	3,143
Professional fees	2,661	2,515
Intellectual property costs	1,379	1,642
Accrued property and equipment	0	630
Other	508	701
Total	$ 91,883	$ 45,335

Condensed Consolidated Balance Sheets (Parenthetical)	Jun. 30, 2024 SFr / shares shares	Dec. 31, 2023 SFr / shares shares
Statement of Financial Position [Abstract]
Common stock, par value \| (per share)	SFr 0.03	SFr 0.03
Common stock, shares authorized	132,477,166	126,536,183
Common stock, shares issued	85,215,817	80,214,694
Common stock, shares outstanding	85,045,501	80,044,378
Treasury stock, shares	170,316	170,316

Condensed Consolidated Statements of Shareholders' Equity (Unaudited) (Parenthetical) - USD ($) $ in Millions	3 Months Ended
	Jun. 30, 2024	Mar. 31, 2024	Jun. 30, 2023	Mar. 31, 2023
Common Shares [Member]
Issuance costs		$ 3.8
Employee Stock Option
Issuance costs	$ 0.2	$ 0.6	$ 0.3	$ 0.2

Basis of Presentation and Significant Accounting Policies

6 Months Ended

Jun. 30, 2024

Accounting Policies [Abstract]

Basis of Presentation and Significant Accounting Policies

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 and six-month interim periods ended June 30, 2024 and 2023.

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, 2023, which are contained in the 2023 Annual Report on Form 10-K filed with the Securities and Exchange Commission, or the SEC, on February 21, 2024.

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. Significant estimates in these consolidated financial statements have been made in connection with revenue recognition and equity-based compensation expense. 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 and six months ended June 30, 2024 are consistent with those discussed in Note 2 to the consolidated financial statements in the Company’s 2023 Annual Report on Form 10-K filed with the SEC on February 21, 2024.

New Accounting Pronouncements – Recently Adopted

From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board, or FASB, or other standard setting bodies that the Company adopts as of the specified effective date. The Company does not believe that the adoption of recently issued standards has or may have a material impact on its condensed consolidated financial statements and disclosures.

Property and Equipment, net (Tables)

6 Months Ended

Jun. 30, 2024

Property, Plant and Equipment [Abstract]

Summary of Property and Equipment, Net

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


	As of
	June 30,			December 31,
	2024			2023
Computer equipment	$	3,739		$	3,739
Furniture, fixtures and other		8,545			8,109
Laboratory equipment		41,903			41,411
Leasehold improvements		143,260			143,260
Construction work in process		8,654			8,859
Total property and equipment, gross		206,101			205,378
Accumulated depreciation		(63,070	)		(53,433	)
Total property and equipment, net	$	143,031		$	151,945

Property and Equipment, net - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	$ 206,101	$ 205,378
Accumulated depreciation	(63,070)	(53,433)
Total property and equipment, net	143,031	151,945
Computer Equipment [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	3,739	3,739
Furniture, Fixtures and Other [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	8,545	8,109
Laboratory Equipment [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	41,903	41,411
Leasehold Improvements [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	143,260	143,260
Construction Work in Process [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	$ 8,654	$ 8,859

Stock-based Compensation - Summary of Restricted Stock Activity (Detail) - Restricted Stock [Member]	6 Months Ended
	Jun. 30, 2024 $ / shares shares
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unvested restricted common shares, Number of Shares, Beginning Balance \| shares	1,781,415
Unvested restricted common shares, Number of Shares, Granted \| shares	706,844
Unvested restricted common shares, Number of Shares, Vested \| shares	(377,926)
Unvested restricted common shares, Number of Shares, Cancelled or forfeited \| shares	(120,319)
Unvested restricted common shares, Number of Shares, Ending Balance \| shares	1,990,014
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Beginning Balance \| $ / shares	$ 61
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Granted \| $ / shares	70.5
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Vested \| $ / shares	71.41
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Cancelled or forfeited \| $ / shares	54.48
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Ending Balance \| $ / shares	$ 62.79

Accrued Expenses

6 Months Ended

Jun. 30, 2024

Payables and Accruals [Abstract]

Accrued Expenses

5. Accrued Expenses

Accrued expenses consist of the following (in thousands):


	As of
	June 30,		December 31,
	2024		2023
Payroll and employee-related costs	$	14,373	$	17,347
Research and development costs		19,182		16,962
Collaboration costs		51,455		2,395
Licensing fees		2,325		3,143
Professional fees		2,661		2,515
Intellectual property costs		1,379		1,642
Accrued property and equipment		—		630
Other		508		701
Total	$	91,883	$	45,335

Income Taxes	6 Months Ended
Income Taxes	Jun. 30, 2024
Income Tax Disclosure [Abstract]
Income Taxes	11. Income Taxes During the three and six months ended June 30, 2024, the Company recorded an income tax provision of $1.3 million and $2.0 million, respectively, representing an effective tax rate of (1.1%) and (0.9%), respectively. During the three and six months ended June 30, 2023, the Company recorded an income tax provision of $0.9 million and $2.2 million, respectively, representing an effective tax rate of (1.2%) and (1.7%), respectively. The income tax provision for the three and six months ended June 30, 2024 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.

- Definition

The entire disclosure for income tax.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-12

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 231
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482663/740-10-55-231

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12C
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-12C

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12B
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-12B

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 270
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477891/740-270-50-1

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 6.I.5.Q1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479360/740-10-S99-1

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480990/946-20-50-13

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(h)(2))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-Name Accounting Standards Codification
-Publisher FASB
-URI https://asc.fasb.org/740/tableOfContent

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 14
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-14

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 21
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-21

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 17
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-17

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 2
-Subparagraph (SAB Topic 11.C)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479360/740-10-S99-2

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 30
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482603/740-30-50-2

+ Details

Marketable Securities - Summary of Cash Equivalents and Marketable Securities Recorded at Fair Value (Detail) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023
Marketable Securities [Line Items]
Cash equivalents, amortized cost	$ 296,570	$ 191,721
Cash equivalents, gross unrealized gains	0	0
Cash equivalents, gross unrealized losses	(58)	0
Cash equivalents, fair value	296,512	191,721
Marketable securities, amortized cost	1,531,204	1,304,304
Marketable securities, gross unrealized gains	691	3,821
Marketable securities, gross unrealized losses	(3,532)	(1,937)
Marketable securities, fair value	1,528,363	1,306,188
Cash equivalents and marketable securities, amortized cost	1,827,774	1,496,025
Cash equivalents and marketable securities, gross unrealized gains	691	3,821
Cash equivalents and marketable securities, gross unrealized losses	(3,590)	(1,937)
Cash equivalents and marketable securities, fair value	1,824,875	1,497,909
Money Market Funds [Member]
Marketable Securities [Line Items]
Cash equivalents, amortized cost	139,868	185,990
Cash equivalents, gross unrealized gains	0	0
Cash equivalents, gross unrealized losses	0	0
Cash equivalents, fair value	139,868	185,990
Certificates and Term Deposits [Member]
Marketable Securities [Line Items]
Cash equivalents, amortized cost	25,351
Cash equivalents, gross unrealized gains	0
Cash equivalents, gross unrealized losses	0
Cash equivalents, fair value	25,351
Marketable securities, amortized cost	132,499	47,282
Marketable securities, gross unrealized gains	0	0
Marketable securities, gross unrealized losses	0	0
Marketable securities, fair value	132,499	47,282
Commercial Paper [Member]
Marketable Securities [Line Items]
Cash equivalents, amortized cost	125,953
Cash equivalents, gross unrealized gains	0
Cash equivalents, gross unrealized losses	(58)
Cash equivalents, fair value	125,895
Marketable securities, amortized cost	130,273	155,403
Marketable securities, gross unrealized gains	0	32
Marketable securities, gross unrealized losses	(79)	(26)
Marketable securities, fair value	130,194	155,409
US Treasury Securities [Member]
Marketable Securities [Line Items]
Cash equivalents, amortized cost	3,974	5,731
Cash equivalents, gross unrealized gains	0	0
Cash equivalents, gross unrealized losses	0	0
Cash equivalents, fair value	3,974	5,731
Marketable securities, amortized cost	11,883	22,963
Marketable securities, gross unrealized gains	0	45
Marketable securities, gross unrealized losses	(9)	0
Marketable securities, fair value	11,874	23,008
Corporate Debt Securities [Member]
Marketable Securities [Line Items]
Cash equivalents, amortized cost	1,424
Cash equivalents, gross unrealized gains	0
Cash equivalents, gross unrealized losses	0
Cash equivalents, fair value	1,424
Marketable securities, amortized cost	1,013,028	883,550
Marketable securities, gross unrealized gains	669	3,367
Marketable securities, gross unrealized losses	(2,517)	(1,559)
Marketable securities, fair value	1,011,180	885,358
Government-sponsored Enterprise Securities [Member]
Marketable Securities [Line Items]
Marketable securities, amortized cost	243,521	195,106
Marketable securities, gross unrealized gains	22	377
Marketable securities, gross unrealized losses	(927)	(352)
Marketable securities, fair value	$ 242,616	$ 195,131

Net Loss Per Share Attributable to Common Shareholders - Schedule of Antidilutive Securities not Include in Computation of Net (Loss) Income per Share (Detail) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities not include in computation of net income (loss) per share	9,315,656	8,887,196	9,315,656	8,887,196
Outstanding Options [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities not include in computation of net income (loss) per share	7,304,423	7,328,032	7,304,423	7,328,032
Unvested Restricted Common Shares [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities not include in computation of net income (loss) per share	1,990,014	1,524,987	1,990,014	1,524,987
ESPP [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities not include in computation of net income (loss) per share	21,219	34,177	21,219	34,177

Basis of Presentation and Significant Accounting Policies - Additional Information (Detail)	6 Months Ended
	Jun. 30, 2024 Segment
Accounting Policies [Abstract]
Number of operating segments	1

Condensed Consolidated Statements of Cash Flows (Parenthetical) - USD ($) $ in Thousands	Jun. 30, 2024	Dec. 31, 2023	Jun. 30, 2023	Dec. 31, 2022
Cash and cash equivalents	$ 484,472	$ 389,477	$ 444,796
Restricted cash	11,520	11,591	11,799
Cash, cash equivalents and restricted cash at end of period	$ 495,992	$ 401,068	$ 456,595	$ 224,060

Property and Equipment, net - Additional Information (Detail) - USD ($) $ in Millions	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Property, Plant and Equipment [Abstract]
Depreciation expense	$ 4.8	$ 5.0	$ 9.6	$ 10.0

Commitments and Contingencies

6 Months Ended

Jun. 30, 2024

Commitments and Contingencies Disclosure [Abstract]

Commitments and Contingencies

7. Commitments and Contingencies

Leases

Refer to Note 7 to the consolidated financial statements in the Company’s 2023 Annual Report on Form 10-K filed with the SEC on February 21, 2024 for discussion of the Company’s lease arrangements.

Litigation

In the ordinary course of business, the Company is 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 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 June 30, 2024, 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 and included in “Restricted cash” on the Company's condensed consolidated balance sheets as of June 30, 2024.

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 association 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 the CASGEVY program and 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 CASGEVY program.

Under the A&R Vertex JDCA, the Company has an option to defer specified costs on the CASGEVY program in excess of $110.3 million for the years ended December 31, 2022, 2023 and 2024. Additionally, the Company is permitted, under certain specified circumstances as set forth in Amendment No. 1 to the A&R Vertex JDCA, to adjust the timing of and portion of the Company's share of costs it is permitted to defer under the agreement.

The Company exercised its option to defer its share of costs incurred in 2022 and 2023 on the CASGEVY program in excess of the deferral limit under the A&R Vertex JDCA, which resulted in a deferral of $36.1 million and $80.9 million, respectively. As of June 30, 2024, $4.0 million of costs incurred in 2024 under the A&R Vertex JDCA, as amended, have been deferred. Any deferred amounts 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, subject to potential specified offsets. These deferred costs on the CASGEVY program will be accrued for when it is probable that a liability has been incurred and the amount can be reasonably estimated. As of June 30, 2024, no contingent payments have been accrued to date.

Stock-based Compensation - Summary of Stock Option Activity (Detail) - $ / shares	6 Months Ended
	Jun. 30, 2024
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]
Shares, Outstanding, Beginning Balance	7,204,372
Shares, Granted	1,056,224
Shares, Exercised	(677,561)
Shares, Cancelled or forfeited	(278,612)
Shares, Outstanding, Ending Balance	7,304,423
Shares, Exercisable	4,714,519
Shares, Vested and expected to vest	7,304,423
Weighted-Average Exercise Price, Outstanding, Beginning Balance	$ 55.05
Weighted-Average Exercise Price, Granted	68.76
Weighted-Average Exercise Price, Exercised	37.92
Weighted-Average Exercise Price, Cancelled or forfeited	72.96
Weighted-Average Exercise Price, Outstanding, Ending Balance	57.94
Weighted-Average Exercise Price, Exercisable	56.5
Weighted-Average Exercise Price, Vested and expected to vest	$ 57.94

Net Loss Per Share Attributable to Common Shareholders (Tables)

6 Months Ended

Jun. 30, 2024

Earnings Per Share [Abstract]

Schedule of Antidilutive Securities not Include in Computation of Net (Loss) Income per Share

The following common stock equivalents were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect:


	Three Months Ended June 30,				Six Months Ended June 30,
	2024		2023		2024		2023
Outstanding options		7,304,423		7,328,032		7,304,423		7,328,032
Unvested restricted common shares		1,990,014		1,524,987		1,990,014		1,524,987
ESPP		21,219		34,177		21,219		34,177
Total		9,315,656		8,887,196		9,315,656		8,887,196

Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2024	Jun. 30, 2023	Jun. 30, 2024	Jun. 30, 2023
Revenue:
Total revenue	$ 517	$ 70,000	$ 1,021	$ 170,000
Operating expenses:
Research and development	80,165	101,555	156,338	201,490
General and administrative	19,481	19,032	37,434	41,392
Collaboration expense, net	52,131	44,636	99,097	86,828
Total operating expenses	151,777	165,223	292,869	329,710
Loss from operations	(151,260)	(95,223)	(291,848)	(159,710)
Other income:
Other income, net	26,139	18,406	50,860	31,148
Total other income, net	26,139	18,406	50,860	31,148
Net loss before income taxes	(125,121)	(76,817)	(240,988)	(128,562)
Provision for income taxes	(1,287)	(923)	(2,011)	(2,243)
Net loss	(126,408)	(77,740)	(242,999)	(130,805)
Foreign currency translation adjustment	2	28	(9)	60
Unrealized (loss) gain on marketable securities	(1,329)	452	(4,783)	6,679
Comprehensive loss	$ (127,735)	$ (77,260)	$ (247,791)	$ (124,066)
Net loss per common share - basic	$ (1.49)	$ (0.98)	$ (2.92)	$ (1.66)
Basic weighted-average common shares outstanding	84,920,929	79,091,061	83,357,780	78,885,168
Net loss per common share - diluted	$ (1.49)	$ (0.98)	$ (2.92)	$ (1.66)
Diluted weighted-average common shares outstanding	84,920,929	79,091,061	83,357,780	78,885,168
Collaboration Revenue [Member]
Revenue:
Total revenue	$ 0	$ 70,000	$ 0	$ 170,000
Grant Revenue [Member]
Revenue:
Total revenue	$ 517	$ 0	$ 1,021	$ 0