0001193125-26-330557 | 10-Q | XBRL Viewer

Revenue

$ 9,325,000

$ 13,817,000

$ 15,554,000

$ 172,386,000

Provision for income taxes

(74,754,000)

(41,117,000)

(134,401,000)

(88,446,000)

Interest expense

(17,000)

(3,000)

(41,000)

(3,000)

Interest income

3,520,000

3,792,000

6,939,000

6,681,000

Other income

308,000

28,000

327,000

57,000

(1,278,000)

Net (loss) income

(61,618,000)

$ (50,003,000)

(23,483,000)

$ 112,879,000

(111,621,000)

89,397,000

Zorevunersen

^[1]

28,243,000

11,382,000

48,097,000

29,056,000

ADOA

^[1]

2,697,000

1,747,000

4,555,000

2,140,000

SYNGAP1

^[1]

1,073,000

818,000

1,818,000

1,160,000

MECP2

Other external program costs

^[1]

(200,000)

(53,000)

Payroll and other personnel costs - R&D

3,990,000

1,841,000

7,169,000

3,670,000

Payroll and other personnel costs - SG&A

^[2]

18,218,000

11,618,000

34,336,000

22,629,000

Other non-program costs - R&D

^[2]

13,318,000

8,979,000

24,375,000

17,908,000

Other non-program costs - SG&A

^[3]

1,628,000

1,615,000

3,308,000

3,022,000

^[3]

$ 5,587,000

$ 3,317,000

$ 10,742,000

$ 8,914,000

[1]	Includes external research and development and sales, general and administrative expenses associated with programs.
[2]	Includes salaries, benefits and other personnel related expenses, including stock based compensation expense, of relevant personnel.
[3]	Includes costs incurred for conducting non-program activities related to Company activities.

Commitments and Contingencies - Schedule of Future Minimum Lease Payments Under Non-Cancellable Leases (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Jul. 31, 2025
Future minimum lease payments under non-cancellable leases
2026	$ 1,626
2027	700
2028	532
Total lease payments	2,858
Less imputed interest	(176)
Present value of lease liabilities	$ 2,682	$ 1,800

Equity Incentive Plans - Summary of Stock-based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock based compensation expense	$ 10,802	$ 7,598	$ 19,555	$ 14,351
Location, Statement of Income, Balance [Axis]: us-gaap:ResearchAndDevelopmentExpense
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock based compensation expense	4,372	2,688	7,760	5,488
Location, Statement of Income, Balance [Axis]: us-gaap:SellingGeneralAndAdministrativeExpense
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock based compensation expense	$ 6,430	$ 4,910	$ 11,795	$ 8,863

Net (Loss) Income Per Share - Schedule of Computation of Basic and Diluted Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended				6 Months Ended
	Jun. 30, 2026	Mar. 31, 2026	Jun. 30, 2025	Mar. 31, 2025	Jun. 30, 2026	Jun. 30, 2025
Numerator:
Net (loss) income	$ (61,618)	$ (50,003)	$ (23,483)	$ 112,879	$ (111,621)	$ 89,397
Denominator:
Weighted-average number of common shares, basic	66,017,895		58,353,855		64,548,862	58,109,622
Weighted-average number of common shares, diluted	66,017,895		58,353,855		64,548,862	59,681,472
Net (loss) income per share, basic	$ (0.93)		$ (0.4)		$ (1.73)	$ 1.54
Net (loss) income per share, diluted	$ (0.93)		$ (0.4)		$ (1.73)	$ 1.5

The amount of net income (loss) for the period per each share of common stock or unit outstanding during the reporting period.

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

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Condensed consolidated balance sheets - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025
Current assets:
Cash and cash equivalents	$ 110,004	$ 84,220
Marketable securities - current	182,814	200,450
Accounts receivable	7,179	5,936
Prepaid expenses	14,688	8,736
Interest receivable	1,540	1,969
Other current assets	6,313	4,389
Total current assets	322,538	305,700
Marketable securities - long-term	61,502	106,260
Restricted cash - long-term	3,227	227
Operating right-of-use assets	1,870	3,101
Property and equipment, net	4,829	3,146
Total assets	393,966	418,434
Current liabilities:
Accounts payable	4,794	4,939
Accrued and other current liabilities	28,613	41,035
Deferred revenue - current portion	8,523	11,901
Total current liabilities	41,930	57,875
Deferred revenue - net of current portion	5,652	6,961
Other long-term liabilities	837	1,141
Total long-term liabilities	6,489	8,102
Total liabilities	48,419	65,977
Commitments and contingencies (Note 6)
Stockholders’ equity
Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 62,402,595 and 58,921,999 shares issued and outstanding as of June 30, 2026 and December 31, 2025, respectively	6	5
Additional paid-in capital	955,419	849,624
Accumulated other comprehensive (loss) income	(542)	543
Accumulated deficit	(609,336)	(497,715)
Total stockholders’ equity	345,547	352,457
Total liabilities and stockholders’ equity	$ 393,966	$ 418,434

Nature of the Business

6 Months Ended

Jun. 30, 2026

Commitments and Contingencies Disclosure [Abstract]

Nature of the business

1. Nature of the business

Organization

Stoke Therapeutics, Inc. (the "Company") was founded in June 2014 and was incorporated under the laws of the State of Delaware. The Company is a late-stage clinical company dedicated to addressing the underlying causes of severe diseases by upregulating protein expression with RNA-based medicines.

Shelf Registration

In July 2025, the Company filed a universal Shelf Registration statement on Form S-3 (the “2025 Registration Statement”) with the SEC. The 2025 Registration Statement was declared effective by the SEC on July 11, 2025, and contains two prospectuses: a base prospectus, which covers the offering, issuance and sale by the Company of up to a maximum aggregate offering price of $400.0 million of the Company’s common stock, preferred stock, debt securities, warrants to purchase common stock, preferred stock or debt securities, subscription rights to purchase common stock, preferred stock or debt securities and/or units consisting of some or all of these securities; and a sales agreement prospectus covering the offering, issuance and sale by the Company of up to a maximum aggregate offering price of $150.0 million of common stock that may be issued and sold under a Controlled Equity Offering Sales Agreement (the “Sales Agreement”). The Company does not intend to make any further offers or sales of its common stock pursuant to the Sales Agreement under the 2025 Registration Statement.

In March 2026, the Company filed an automatic universal Shelf Registration Statement on Form S-3 (the “2026 Registration Statement”) with the SEC. The 2026 Registration Statement contains two prospectuses: a base prospectus, which covers the offering, issuance and sale by the Company of its common stock, preferred stock, debt securities, warrants to purchase common stock, preferred stock or debt securities, subscription rights to purchase common stock, preferred stock or debt securities and/or units consisting of some or all of these securities; and a sales agreement prospectus covering the offering, issuance and sale by the Company of up to a maximum aggregate offering price of $150.0 million of its common stock that may be issued and sold under the Sales Agreement. The specific terms of any securities to be offered pursuant to the base prospectus will be specified in a prospectus supplement to the base prospectus. The Company intends to use the 2026 Registration Statement for any future issuances and sales of common stock.

As of December 31, 2025, the Company had issued approximately 3.2 million shares of common stock pursuant to the 2025 Registration Statement for net proceeds of $87.8 million. In addition, as of June 30, 2026, the Company had issued approximately 2.6 million shares of common stock pursuant to the Sales Agreement and the 2026 Registration Statement for net proceeds of $80.7 million. Since June 30, 2026, through the issuance of these consolidated financial statements, the Company sold approximately 2.1 million shares of our common stock pursuant to the Sales Agreement and the 2026 Registration Statement for net proceeds of $65.7 million after deducting commissions. The Company may terminate this at-the-market program under the Sales Agreement at any time, pursuant to its terms.

Uncertainties

The Company is subject to risks and uncertainties common to late-stage clinical companies in the biotechnology industry including, but not limited to, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with government regulations and ability to secure additional capital to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize significant revenue from product sales.

Liquidity

The Company expects that its operating losses and negative cash flows will continue for the foreseeable future. As of the issuance date of these unaudited condensed consolidated financial statements, the Company expects that its cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses and capital expenditure requirements through at least twelve months from the issuance date of these unaudited condensed consolidated financial statements.

Commitments and Contingencies (Tables)

6 Months Ended

Jun. 30, 2026

Schedule of Future Minimum Lease Payments Under Non-Cancellable Leases

Future minimum lease payments under non-cancellable leases as of June 30, 2026 were as follows (in thousands):


2026		1,626
2027		700
2028		532
Total lease payments		2,858
Less imputed interest		(176	)
Present value of lease liabilities	$	2,682

Summary of Lease Balances

Lease balances as of June 30, 2026 were as follows (in thousands):


Operating right-of-use assets	$	1,870
Current portion of operating lease liabilities	$	1,845
Non-current portion of operating lease liabilities		837
Total operating lease liabilities	$	2,682

Summary of Weighted Average Remaining Lease Term and Weighted Average Discount Rate of Operating Leases

The weighted average remaining lease term and weighted average discount rate of the Company’s operating leases as of June 30, 2026 were as follows:


Weighted average remaining lease term in years	1.5
Weighted average discount rate	10.05	%

Equity Incentive Plans

6 Months Ended

Jun. 30, 2026

Share-Based Payment Arrangement [Abstract]

Equity incentive plans

8. Equity incentive plans

As of June 30, 2026, there were no shares available for future issuance under the 2014 Plan, 2,078,770 shares were available under the 2019 Plan and 2,160,047 shares were available under the 2023 Plan.

During the three months ended June 30, 2026, the Company granted options to purchase 529,855 shares of common stock to certain of its employees. The options vest over a period of up to four years and are exercisable at a per share price equal to the fair value of the common stock on the grant date. During the three months ended June 30, 2026, the Company granted 12,000 RSUs and did not grant any performance stock units (“PSUs”) to its employees. The RSUs vest over a period of up to four years.

Stock-based compensation

As of June 30, 2026, there was $83.1 million of unrecognized compensation cost related to unvested stock options granted under the 2019 and 2023 Plans. Compensation expense is expected to be recognized over a weighted average period of 2.8 years as of June 30, 2026. As of June 30, 2026, there was $39.7 million of unrecognized stock-based compensation related to RSUs and $0.05 million of unrecognized stock-based compensation related to PSUs. The compensation expense for RSUs and PSUs is expected to be recognized over a weighted average period of 2.8 years.

Stock-based compensation expense recorded as research and development and sales, general and administrative expenses in the accompanying condensed consolidated statements of operations and comprehensive (loss) income is as follows (in thousands):


	Three Months Ended June 30,				Six Months Ended June 30,
	2026		2025		2026		2025
Research and development	$	4,372	$	2,688	$	7,760	$	5,488
Sales, general and administrative		6,430		4,910		11,795		8,863
Total	$	10,802	$	7,598	$	19,555	$	14,351

2019 Employee stock purchase plan

In June 2019, the Company adopted the 2019 Employee Stock Purchase Plan (the “ESPP”), which became effective on June 18, 2019. The Company initially reserved 315,000 shares of common stock for sale under the ESPP. At June 30, 2026, the Company had 2,608,136 shares available for issuance under the ESPP. The average grant date fair value per share under the ESPP was $30.90 for 2026. The total ESPP stock-based compensation expense for the three and six months ended June 30, 2026 was $0.2 million and $0.4

million, respectively, and for the three and six months ended June 30, 2025 was $0.1 million and $0.3 million, respectively. The number of shares reserved for issuance under the ESPP will increase automatically on January 1 of each of the first ten calendar years following the first offering date by the number of shares equal to the lesser of 1% of the total outstanding shares of the Company’s common stock as of the immediately preceding December 31 or a lower amount determined by the Company’s board of directors. The aggregate number of shares issued over the term of the ESPP will not exceed 3,150,000 shares of the Company’s common stock.

License and collaboration agreements - Summary of Transaction Price Allocated to Each Performance Obligation, in Addition to Revenue Activity (Details) - Biogen License Agreement - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
License And Collaboration Agreement [Line Items]
Transaction Price Allocated	$ 0	$ 243,423	$ 0	$ 243,423
Revenue Recognized	6,500	3,200	10,563	155,618
IP License
License And Collaboration Agreement [Line Items]
Transaction Price Allocated	0	150,779	0	150,779
Revenue Recognized			0	150,779
Global Development Activity
License And Collaboration Agreement [Line Items]
Transaction Price Allocated	$ 0	$ 92,644	0	92,644
Revenue Recognized			$ 10,563	$ 4,839

Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

Income Taxes - Additional Information (Details) - USD ($)	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Income Taxes [Line Items]
Income tax benefit	$ 0	$ 0	$ 0	$ 1,278,000
Federal
Income Taxes [Line Items]
Income tax benefit			1,100,000	1,100,000
State
Income Taxes [Line Items]
Income tax benefit			$ 100,000	$ 100,000

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

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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 8
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483443/250-10-50-8

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
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-Name Accounting Standards Codification
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483443/250-10-50-9

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147482685/740-10-50-10

Reference 15: http://www.xbrl.org/2003/role/disclosureRef
-Topic 944
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-Paragraph 1
-Subparagraph (SX 210.7-04(9))
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Reference 16: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
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-Paragraph 1
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-Topic 235
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-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-1

Net (loss) income per share

Condensed consolidated statements of operations and comprehensive (loss) income - USD ($)	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Income Statement [Abstract]
Revenue	$ 9,325,000	$ 13,817,000	$ 15,554,000	$ 172,386,000
Operating expenses:
Research and development	49,501,000	25,855,000	89,174,000	58,531,000
Sales, general and administrative	25,253,000	15,262,000	45,227,000	29,915,000
Total operating expenses	74,754,000	41,117,000	134,401,000	88,446,000
(Loss) income from operations	(65,429,000)	(27,300,000)	(118,847,000)	83,940,000
Other income (expense):
Interest income (expense), net	3,503,000	3,789,000	6,899,000	6,678,000
Other income	308,000	28,000	327,000	57,000
Total other income (expense)	3,811,000	3,817,000	7,226,000	6,735,000
(Loss) income before income taxes	(61,618,000)	(23,483,000)	(111,621,000)	90,675,000
Provision for income taxes	0	0	0	1,278,000
Net (loss) income	$ (61,618,000)	$ (23,483,000)	$ (111,621,000)	$ 89,397,000
Net (loss) income per share:
Basic	$ (0.93)	$ (0.4)	$ (1.73)	$ 1.54
Diluted	$ (0.93)	$ (0.4)	$ (1.73)	$ 1.5
Weighted-average common shares outstanding:
Basic	66,017,895	58,353,855	64,548,862	58,109,622
Diluted	66,017,895	58,353,855	64,548,862	59,681,472
Comprehensive (loss) income:
Net (loss) income	$ (61,618,000)	$ (23,483,000)	$ (111,621,000)	$ 89,397,000
Other comprehensive (loss) gain:
Unrealized (loss) gain on marketable securities	(427,000)	200,000	(1,085,000)	247,000
Total other comprehensive (loss) gain	(427,000)	200,000	(1,085,000)	247,000
Comprehensive (loss) income	$ (62,045,000)	$ (23,283,000)	$ (112,706,000)	$ 89,644,000

Equity Incentive Plans - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands	1 Months Ended	3 Months Ended		6 Months Ended
	Jun. 30, 2019	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Option granted to purchase shares		529,855
Unrecognized compensation cost		$ 83,100		$ 83,100
Weighted average period of unrecognized compensation cost expected to be recognized				2 years 9 months 18 days
Stock based compensation expense		$ 10,802	$ 7,598	$ 19,555	$ 14,351
Restricted Stock Units
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Restricted stock units granted		12,000
Unrecognized compensation cost		$ 39,700		$ 39,700
Weighted average period of unrecognized compensation cost expected to be recognized				2 years 9 months 18 days
Performance Stock Units
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Restricted stock units granted		0
Unrecognized compensation cost		$ 50		$ 50
Weighted average period of unrecognized compensation cost expected to be recognized				2 years 9 months 18 days
Maximum \| Employee Stock Option Member
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Vesting period		4 years
Maximum \| Restricted Stock Units
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Vesting period		4 years
2019 Plan
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Number of common shares available for future issuance		2,078,770		2,078,770
2014 Plan
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Number of common shares available for future issuance		0		0
2019 ESPP
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Number of shares of common stock reserved for issuance	315,000
Number of common shares available for future issuance		2,608,136		2,608,136
Average grant date fair value per share under the plan				$ 30.9
Stock based compensation expense		$ 200	$ 100	$ 400	$ 300
2019 ESPP \| Maximum
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Number of shares of common stock reserved for issuance	3,150,000
Percentage of increase in reserved common shares	1.00%
2023 Inducement Plan
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Number of common shares available for future issuance		2,160,047		2,160,047

Net (Loss) Income Per Share - Additional Information (Details) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Earnings Per Share [Abstract]
Weighted-average shares outstanding	3,703,730	3,703,730	3,703,730	3,703,730

Net (Loss) Income Per Share

6 Months Ended

Jun. 30, 2026

Earnings Per Share [Abstract]

9. Net (loss) income per share

The following table summarizes the computation of basic and diluted net loss per share of the Company (in thousands except share and per share amounts):


	Three Months Ended June 30,						Six Months Ended June 30,
	2026			2025			2026			2025
Numerator:
Net (loss) income	$	(61,618	)	$	(23,483	)	$	(111,621	)	$	89,397
Denominator:
Weighted-average number of common shares, basic		66,017,895			58,353,855			64,548,862			58,109,622
Weighted-average number of common shares, diluted		66,017,895			58,353,855			64,548,862			59,681,472
Net (loss) income per share, basic	$	(0.93	)	$	(0.40	)	$	(1.73	)	$	1.54
Net (loss) income per share, diluted	$	(0.93	)	$	(0.40	)	$	(1.73	)	$	1.50

As the Company had net income during the six months ended June 30, 2025, the Company’s potential dilutive securities, which include stock options, RSUs and PSUs, were included in the calculation of diluted net income per share attributable to common stockholders.

For the three and six months ended June 30, 2026 and the three months ended June 30, 2025, the Company incurred a net loss. Weighted-average shares outstanding used to calculate basic net loss per share includes 3,703,730 warrants outstanding during the period. The Company’s potential dilutive securities, which include stock options, restricted stock units and performance stock units have been excluded from the computation of diluted net loss per share as the effect would be to reduce the net loss per share. Therefore, the weighted-average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same for the three and six months ended June 30, 2026 and the three months ended June 30, 2025.

The Company excluded the following potential common stock, presented based on amounts outstanding at June 30, 2026 and 2025 from the computation of diluted net (loss) income per share attributable to common stockholders because including them would have had an anti-dilutive effect.


	Three Months Ended June 30,				Six Months Ended June 30,
	2026		2025		2026		2025
Outstanding options to purchase common stock		11,532,638		9,523,757		11,532,638		7,408,212
Restricted stock units		1,938,698		1,797,643		1,938,698		544,976
Performance stock units		219,000		219,000		219,000		—
Total		13,690,336		11,540,400		13,690,336		7,953,188

Subsequent Events - Additional Information (Details) - 2026 Registration Statement - USD ($) $ in Millions	1 Months Ended	6 Months Ended
	Aug. 03, 2026	Jun. 30, 2026
Subsequent Event [Line Items]
Issuance of common stock, Shares		2,600,000
Proceeds from issuance of common stock net of commissions		$ 80.7
Subsequent Event
Subsequent Event [Line Items]
Issuance of common stock, Shares	2,100,000
Proceeds from issuance of common stock net of commissions	$ 65.7

Number of new stock issued during the period.

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-2

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.3-04)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480008/505-10-S99-1

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section S50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480070/505-10-S50-1

Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section S45
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480100/505-10-S45-3

Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section S45
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480100/505-10-S45-2

Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
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-Section S99
-Paragraph 1
-Subparagraph (SX 210.5-02(28))
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-URI https://asc.fasb.org/1943274/2147480566/210-10-S99-1

Reference 7: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
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-Name Accounting Standards Codification
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-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478478/942-210-S50-1

Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 942
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-Name Accounting Standards Codification
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-Paragraph 1
-Subparagraph (SX 210.9-03(20))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478546/942-210-S99-1

Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 942
-SubTopic 210
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-03(19))
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-URI https://asc.fasb.org/1943274/2147478546/942-210-S99-1

Reference 11: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 210
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.5-02(29))
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-URI https://asc.fasb.org/1943274/2147480566/210-10-S99-1

Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 210
-SubTopic 10
-Name Accounting Standards Codification
-Section S50
-Paragraph 11
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480657/210-10-S50-11

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 505
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478448/946-505-50-2

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
-Section S99
-Paragraph 3
-Subparagraph (SX 210.6-09(4)(b))
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-URI https://asc.fasb.org/1943274/2147479134/946-220-S99-3

Reference 15: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147479886/946-10-S99-3

Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Policies)

6 Months Ended

Jun. 30, 2026

Basis of Presentation and Consolidation

Basis of presentation and consolidation

The accompanying condensed consolidated financial statements have been prepared in conformity with generally accepted accounting principles in the United States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standard Updates of the Financial Accounting Standards Board (“FASB”). All intercompany accounts and transactions have been eliminated in consolidation.

The significant accounting policies used in preparation of these condensed consolidated financial statements for the three and six months ended June 30, 2026 are consistent with those discussed in Note 2 to the consolidated financial statements included in the Company's 2025 Annual Report on Form 10-K, other than as noted below.

Unaudited Interim Financial Information

Unaudited interim condensed consolidated financial information

The accompanying interim unaudited condensed consolidated financial statements and related disclosures are unaudited and have been prepared in accordance with GAAP for interim financial information and the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all the information and footnotes required by GAAP for complete financial statements and should be read in conjunction with the Company’s condensed consolidated financial statements and related notes as of and for the year ended December 31, 2025, which was filed with the SEC on March 16, 2026. The Company’s financial information as of June 30, 2026 and for the three and six months ended June 30, 2026 and 2025 is unaudited, but in the opinion of management, all adjustments, consisting only of normal recurring adjustments, considered necessary for a fair presentation of the financial position, results of operations and cash flows at the dates and for the periods presented of the results of these interim periods have been included. The balance sheet information as of December 31, 2025 was derived from audited condensed consolidated financial statements. The results of the Company’s operations for any interim period are not necessarily indicative of the results that may be expected for any other interim period or for a full fiscal year.

Cash, Cash Equivalents and Restricted Cash

Cash, cash equivalents and restricted cash

The Company considers all highly liquid investments with an original maturity of three months or less at the date of purchase to be cash equivalents. The Company deposits its cash and cash equivalents in checking, sweep and money market accounts. Cash equivalents are carried at cost which approximates their fair market value.

At June 30, 2026, restricted cash consisted of money market accounts collateralizing letters of credit issued as security deposits in connection with the Company’s leases of its corporate facilities.

Cash and cash equivalents, and restricted cash in the condensed consolidated statements of cash flows consists of the following (in thousands):


	As of June 30,
	2026		2025
Cash and cash equivalents	$	110,004	$	101,472
Restricted cash - current		494		75
Restricted cash - long-term		3,227		721
Total cash, cash equivalents and restricted cash	$	113,725	$	102,268

Marketable Securities

6 Months Ended

Jun. 30, 2026

Investments, Debt and Equity Securities [Abstract]

Marketable securities

4. Marketable securities

The following table summarizes the Company’s marketable securities as of June 30, 2026 (in thousands):


	As of June 30, 2026
	Amortized Cost		Unrealized Gain		Unrealized Loss			Fair Value
Marketable securities:
Corporate bonds	$	60,545	$	14	$	(78	)	$	60,481
Commercial paper		22,773		—		(14	)		22,759
US Government and US Government agency debt securities		173,474		20		(485	)		173,009
Total	$	256,792	$	34	$	(577	)	$	256,249

The following table summarizes the Company’s marketable securities as of December 31, 2025 (in thousands):


	As of December 31, 2025
	Amortized Cost		Unrealized Gain		Unrealized Loss			Fair Value
Marketable securities:
Corporate bonds	$	84,568	$	183	$	(1	)	$	84,750
Commercial paper		23,959		7		(5	)		23,961
US Government and US Government agency debt securities		197,638		365		(4	)		197,999
Total	$	306,165	$	555	$	(10	)	$	306,710

As of June 30, 2026, the weighted average maturity of the Company’s short term marketable securities is 0.52 years and the weighted average maturity of the Company's long term marketable securities is 1.3 years.

The Company did not record an allowance for credit losses as of June 30, 2026 related to its marketable securities. Further, given the lack of significant change in the credit risk of these investments, the Company did not recognize any other-than-temporary impairment losses.

License and collaboration agreements (Tables)

6 Months Ended

Jun. 30, 2026

License And Collaboration Agreement [Abstract]

Summary of Transaction Price Allocated to Each Performance Obligation, in Addition to Revenue Activity

The following table provides a summary of the transaction price allocated to each performance obligation, in addition to revenue activity for the six months ended June 30, 2026 and June 30, 2025 (in thousands):


	2026				2025
	Transaction Price Allocated		Revenue Recognized		Transaction Price Allocated		Revenue Recognized
Performance obligations
IP License	$	—	$	—	$	150,779	$	150,779
Global Development Activity		—		10,563		92,644		4,839
	$	—	$	10,563	$	243,423	$	155,618

Marketable Securities - Additional Information (Details)	6 Months Ended
	Jun. 30, 2026 USD ($)
Marketable Securities [Line Items]
Weighted average maturity short term marketable securities period	6 months 7 days
Weighted average maturity long term marketable securities period	1 year 3 months 18 days
Allowance for credit losses	$ 0

Fair Value Measurements

6 Months Ended

Jun. 30, 2026

Fair Value Disclosures [Abstract]

Fair value measurements

3. Fair value measurements


	Fair value measurements as of June 30, 2026
	Level 1		Level 2		Level 3		Total
Cash equivalents:
Money market funds	$	89,966	$	—	$	—	$	89,966
Total	$	89,966	$	—	$	—	$	89,966

Marketable Securities:
Corporate bonds	$	—	$	60,481	$	—	$	60,481
Commercial paper		—		22,759		—		22,759
US Government and US Government agency debt securities		169,904		3,105		—		173,009
Total	$	169,904	$	86,345	$	—	$	256,249


	Fair value measurements as of December 31, 2025
	Level 1		Level 2		Level 3		Total
Cash equivalents:
Money market funds	$	72,890	$	—	$	—	$	72,890
Total	$	72,890	$	—	$	—	$	72,890

Marketable Securities:
Corporate bonds	$	—	$	84,750	$	—	$	84,750
Commercial paper		—		23,961		—		23,961
US Government and US Government agency debt securities		—		197,999		—		197,999
Total	$	—	$	306,710	$	—	$	306,710

The Company’s cash equivalents and marketable securities are carried at fair value, determined according to the fair value hierarchy. The carrying value of the Company’s accounts payable and accrued expenses approximate their fair values due to the short-term nature of these liabilities.

The Company’s assets with fair value categorized as Level 1 within the fair value hierarchy include money market funds. Money market funds are publicly traded mutual funds and are presented as cash equivalents on the condensed consolidated balance sheets as of June 30, 2026 and December 31, 2025.

The Company measures its marketable securities at fair value on a recurring basis and classifies those instruments within Level 2 of the fair value hierarchy. Marketable securities are valued using models or other valuation methodologies that use Level 2 inputs. These models are primarily industry-standard models that consider various assumptions, including time value, yield curve, volatility factors, default rates, current market and contractual prices for the underlying financial instruments, as well as other economic measures. Substantially all of these assumptions are observable in the marketplace, can be derived from observable data or are supported by observable levels at which transactions are executed in the marketplace.

There were no transfers into or out of Level 3 in the periods presented.

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.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Reference 2: http://www.xbrl.org/2003/role/exampleRef
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Reference 3: http://www.xbrl.org/2003/role/exampleRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 11: http://www.xbrl.org/2003/role/disclosureRef
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Condensed consolidated statements of stockholders' equity - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-in Capital	Accumulated Other Comprehensive Gain (Loss)	Accumulated Deficit
Balance at Dec. 31, 2024	$ 229,021	$ 5	$ 719,997	$ (151)	$ (490,830)
Balance, Shares at Dec. 31, 2024		54,032,826
Unrealized gain (loss) on marketable securities	47			47
Stock-based compensation	6,753		6,753
Issuance of common stock upon exercise of stock options	1,004		1,004
Issuance of common stock upon exercise of stock options, Shares		214,996
Issuance of common stock related to employee stock purchase plan	370		370
Issuance of common stock related to employee stock purchase plan, Shares		39,467
Issuance of vested restricted stock units and performance stock units, Shares		308,425
us-gaap:NetIncomeLoss	112,879				112,879
Balance at Mar. 31, 2025	350,074	$ 5	728,124	(104)	(377,951)
Balance, Shares at Mar. 31, 2025		54,595,714
Balance at Dec. 31, 2024	229,021	$ 5	719,997	(151)	(490,830)
Balance, Shares at Dec. 31, 2024		54,032,826
Unrealized gain (loss) on marketable securities	247
us-gaap:NetIncomeLoss	89,397
Balance at Jun. 30, 2025	334,943	$ 5	736,276	96	(401,434)
Balance, Shares at Jun. 30, 2025		54,723,455
Balance at Mar. 31, 2025	350,074	$ 5	728,124	(104)	(377,951)
Balance, Shares at Mar. 31, 2025		54,595,714
Unrealized gain (loss) on marketable securities	200			200
Stock-based compensation	7,598		7,598
Issuance of common stock upon exercise of stock options	554		554
Issuance of common stock upon exercise of stock options, Shares		126,531
Issuance of vested restricted stock units and performance stock units, Shares		1,210
us-gaap:NetIncomeLoss	(23,483)				(23,483)
Balance at Jun. 30, 2025	334,943	$ 5	736,276	96	(401,434)
Balance, Shares at Jun. 30, 2025		54,723,455
Balance at Dec. 31, 2025	352,457	$ 5	849,624	543	(497,715)
Balance, Shares at Dec. 31, 2025		58,921,999
Unrealized gain (loss) on marketable securities	(658)			(658)
Stock-based compensation	8,753		8,753
Issuance of common stock upon exercise of stock options	3,205		3,205
Issuance of common stock upon exercise of stock options, Shares		260,554
Issuance of common stock related to employee stock purchase plan	465		465
Issuance of common stock related to employee stock purchase plan, Shares		46,994
Issuance of vested restricted stock units and performance stock units, Shares		443,786
Shares sold as part of controlled equity offering sales agreement	80,658	$ 1	80,657
Shares sold as part of controlled equity offering sales agreement, Shares		2,567,014
us-gaap:NetIncomeLoss	(50,003)				(50,003)
Balance at Mar. 31, 2026	394,877	$ 6	942,704	(115)	(547,718)
Balance, Shares at Mar. 31, 2026		62,240,347
Balance at Dec. 31, 2025	352,457	$ 5	849,624	543	(497,715)
Balance, Shares at Dec. 31, 2025		58,921,999
Unrealized gain (loss) on marketable securities	(1,085)
us-gaap:NetIncomeLoss	(111,621)
Balance at Jun. 30, 2026	345,547	$ 6	955,419	(542)	(609,336)
Balance, Shares at Jun. 30, 2026		62,402,595
Balance at Mar. 31, 2026	394,877	$ 6	942,704	(115)	(547,718)
Balance, Shares at Mar. 31, 2026		62,240,347
Unrealized gain (loss) on marketable securities	(427)			(427)
Stock-based compensation	10,802		10,802
Issuance of common stock upon exercise of stock options	1,913		1,913
Issuance of common stock upon exercise of stock options, Shares		162,248
us-gaap:NetIncomeLoss	(61,618)				(61,618)
Balance at Jun. 30, 2026	$ 345,547	$ 6	$ 955,419	$ (542)	$ (609,336)
Balance, Shares at Jun. 30, 2026		62,402,595

Document and Entity Information - shares	6 Months Ended
Document and Entity Information - shares	Jun. 30, 2026	Jul. 30, 2026
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Jun. 30, 2026
Document Fiscal Year Focus	2026
Document Fiscal Period Focus	Q2
Current Fiscal Year End Date	--12-31
Entity File Number	001-38938
Entity Registrant Name	Stoke Therapeutics, Inc.
Entity Central Index Key	0001623526
Entity Tax Identification Number	47-1144582
Entity Address, Address Line One	45 Wiggins Ave
Entity Address City Or Town	Bedford
Entity Address State Or Province	MA
Entity Address Postal Zip Code	01730
City Area Code	781
Local Phone Number	430-8200
Trading Symbol	STOK
Entity Current Reporting Status	Yes
Entity Filer Category	Non-accelerated Filer
Entity Small Business	true
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		64,526,242
Entity Incorporation State Country Code	DE
Title of 12(b) Security	Common Stock, $0.0001 par value per share
Security Exchange Name	NASDAQ
Entity Interactive Data Current	Yes
Document Quarterly Report	true
Document Transition Report	false

Nature of the Business - Additional Information (Details) - USD ($) $ in Millions	1 Months Ended			6 Months Ended	12 Months Ended
	Aug. 03, 2026	Mar. 31, 2026	Jul. 31, 2025	Jun. 30, 2026	Dec. 31, 2025
Nature Of Business [Line Items]
Entity incorporation state name				DE
Base Prospectus
Nature Of Business [Line Items]
Maximum aggregate offering price			$ 400.0
Sales Agreement Prospectus
Nature Of Business [Line Items]
Maximum aggregate offering price		$ 150.0	$ 150.0
2025 Registration Statement
Nature Of Business [Line Items]
Issuance of common stock, Shares					3,200,000
Proceeds from issuance of common stock net of commissions					$ 87.8
2026 Registration Statement
Nature Of Business [Line Items]
Issuance of common stock, Shares				2,600,000
Proceeds from issuance of common stock net of commissions				$ 80.7
2026 Registration Statement \| Subsequent Event
Nature Of Business [Line Items]
Issuance of common stock, Shares	2,100,000
Proceeds from issuance of common stock net of commissions	$ 65.7

Fair Value Measurements - Additional Information (Details) - USD ($)	6 Months Ended	12 Months Ended
	Jun. 30, 2026	Dec. 31, 2025
Fair Value Disclosures [Abstract]
Fair value assets, transfers into level 3, amount	$ 0	$ 0
Fair value assets, transfers out of level 3, amount	$ 0	$ 0

Condensed consolidated statements of cash flows - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025
Cash flows from operating activities:
Net (loss) income	$ (111,621)	$ 89,397
Adjustments to reconcile net (loss) income to net cash provided by (used in) operating activities:
Depreciation	897	923
Amortization and accretion of marketable securities	(734)	33
Stock-based compensation expense	19,555	14,351
Reduction in the carrying amount of right of use assets	1,288	1,211
Changes in assets and liabilities:
Accounts receivable	(1,243)	3,753
Prepaid expenses and other current assets	(7,501)	(10,158)
Accounts payable, accrued liabilities and lease liabilities	(13,121)	7,506
Deferred revenue	(4,687)	(611)
Net cash (used in) provided by operating activities	(117,167)	106,405
Cash flows from investing activities:
Purchases of property and equipment	(2,329)	(306)
Purchases of marketable securities	(45,959)	(200,569)
Proceeds from maturity of marketable securities	108,000	66,030
Net cash provided by (used in) investing activities	59,712	(134,845)
Cash flows from financing activities:
Proceeds from Employee Stock Purchase Plan	465	370
Proceeds from issuance of common stock upon exercise of stock options	5,117	1,558
Proceeds from issuance of common stock in controlled equity offering sales agreements	80,657	0
Net cash provided by financing activities	86,239	1,928
Net increase in cash, cash equivalents and restricted cash	28,784	(26,512)
Cash, cash equivalents and restricted cash—beginning of period	84,941	128,780
Cash, cash equivalents and restricted cash—end of period	113,725	102,268
Supplemental Disclosure of Non-Cash Investing and Financing Activities:
Property and equipment included in accrued expense and accounts payable	$ 250	$ 51

Segment Information

6 Months Ended

Jun. 30, 2026

Segment Reporting [Abstract]

Segment Information

11. Segment Information

The Company comprises one reportable segment, Stoke. As of June 30, 2026, this segment has not generated any product revenue since inception, as it does not yet have approved products for sale.

The Company primarily generates revenue in North America, with its long-lived assets also concentrated in this region, and manages its business activities on a consolidated basis. Decisions concerning the allocation of the Company’s resources are made by the Company’s Chief Operating Decision Maker (“CODM”), which is the Company’s Chief Executive Officer. The CODM views the Company’s operations as a single operating segment which is the business of discovering and developing RNA-based medicines, using the Company's proprietary protein upregulation platform and developing ASOs to selectively restore protein levels.

The CODM assesses performance for the Company's operations segment and decides how to allocate resources based on net income or loss that is also reported on the income statement as consolidated net income or loss and the measure of segment assets as reported on the balance sheet as total consolidated assets. Net income or loss is used to monitor budget versus actual results as well as assess the general performance of the segment in a given period.

The following table reconciles segment direct profit or loss to the Company’s condensed consolidated results (in thousands):


	Three Months Ended June 30,						Six Months Ended June 30,
	2026			2025			2026			2025
Revenue	$	9,325		$	13,817		$	15,554		$	172,386
Zorevunersen(1)		28,243			11,382			48,097			29,056
ADOA(1)		2,697			1,747			4,555			2,140
SYNGAP1(1)		1,073			818			1,818			1,160
MECP2(1)		—			(200	)		—			(53	)
Other external program costs		3,990			1,841			7,169			3,670
Payroll and other personnel costs - R&D (2)		18,218			11,618			34,336			22,629
Payroll and other personnel costs - SG&A (2)		13,318			8,979			24,375			17,908
Other non-program costs - R&D (3)		1,628			1,615			3,308			3,022
Other non-program costs - SG&A (3)		5,587			3,317			10,742			8,914
Interest expense		(17	)		(3	)		(41	)		(3	)
Interest income		3,520			3,792			6,939			6,681
Other income		308			28			327			57
Provision for income taxes		—			—			—			(1,278	)
Segment net income (loss)	$	(61,618	)	$	(23,483	)	$	(111,621	)	$	89,397

(1) Includes external research and development and sales, general and administrative expenses associated with programs.

(2) Includes salaries, benefits and other personnel related expenses, including stock based compensation expense, of relevant personnel.

(3) Includes costs incurred for conducting non-program activities related to Company activities.

The entire disclosure for segment reporting.

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Schedule of Computation of Basic and Diluted Net Loss Per Share

Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Schedule of Cash and Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025	Jun. 30, 2025	Dec. 31, 2024
Accounting Policies [Abstract]
Cash and cash equivalents	$ 110,004	$ 84,220	$ 101,472
Restricted cash - current	494		75
Restricted cash - long-term	3,227	227	721
Total cash, cash equivalents and restricted cash	$ 113,725	$ 84,941	$ 102,268	$ 128,780

Net (Loss) Income Per Share - Schedule of Antidilutive Securities Excluded from Computation of Diluted Net Loss (Income) Per Share (Details) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2026	Jun. 30, 2025	Jun. 30, 2026	Jun. 30, 2025
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities excluded from computation of earnings per share, amount	13,690,336	11,540,400	13,690,336	7,953,188
Employee Stock Option
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities excluded from computation of earnings per share, amount	11,532,638	9,523,757	11,532,638	7,408,212
Restricted Stock Units
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities excluded from computation of earnings per share, amount	1,938,698	1,797,643	1,938,698	544,976
Performance Stock Units
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities excluded from computation of earnings per share, amount	219,000	219,000	219,000

Net (Loss) Income Per Share (Tables)

6 Months Ended

Jun. 30, 2026

Earnings Per Share [Abstract]

The following table summarizes the computation of basic and diluted net loss per share of the Company (in thousands except share and per share amounts):


	Three Months Ended June 30,						Six Months Ended June 30,
	2026			2025			2026			2025
Numerator:
Net (loss) income	$	(61,618	)	$	(23,483	)	$	(111,621	)	$	89,397
Denominator:
Weighted-average number of common shares, basic		66,017,895			58,353,855			64,548,862			58,109,622
Weighted-average number of common shares, diluted		66,017,895			58,353,855			64,548,862			59,681,472
Net (loss) income per share, basic	$	(0.93	)	$	(0.40	)	$	(1.73	)	$	1.54
Net (loss) income per share, diluted	$	(0.93	)	$	(0.40	)	$	(1.73	)	$	1.50

Schedule of Antidilutive Securities Excluded from Computation of Diluted Net Loss (Income) Per Share


	Three Months Ended June 30,				Six Months Ended June 30,
	2026		2025		2026		2025
Outstanding options to purchase common stock		11,532,638		9,523,757		11,532,638		7,408,212
Restricted stock units		1,938,698		1,797,643		1,938,698		544,976
Performance stock units		219,000		219,000		219,000		—
Total		13,690,336		11,540,400		13,690,336		7,953,188

Segment Information - Additional Information (Details)	6 Months Ended
Segment Information - Additional Information (Details)	Jun. 30, 2026 Segment
Segment Reporting [Abstract]
Number of reportable segments	1
Segment Reporting, CODM, Individual Title and Position or Group Name [Extensible Enumeration]	srt:ChiefExecutiveOfficerMember
Segment Reporting, CODM, Profit (Loss) Measure, How Used, Description	The CODM assesses performance for the Company's operations segment and decides how to allocate resources based on net income or loss that is also reported on the income statement as consolidated net income or loss and the measure of segment assets as reported on the balance sheet as total consolidated assets.
Segment Reporting, Expense Information Used by CODM, Type [Extensible Enumeration]	us-gaap:NetIncomeLoss

Equity Incentive Plans (Tables)

6 Months Ended

Jun. 30, 2026

Share-Based Payment Arrangement [Abstract]

Summary of Stock-based Compensation Expense


	Three Months Ended June 30,				Six Months Ended June 30,
	2026		2025		2026		2025
Research and development	$	4,372	$	2,688	$	7,760	$	5,488
Sales, general and administrative		6,430		4,910		11,795		8,863
Total	$	10,802	$	7,598	$	19,555	$	14,351

Summary of Significant Accounting Policies and Recent Accounting Pronouncements

6 Months Ended

Jun. 30, 2026

Summary of significant accounting policies and recent accounting pronouncements

2. Summary of significant accounting policies and recent accounting pronouncements

Basis of presentation and consolidation

Unaudited interim condensed consolidated financial information

Cash, cash equivalents and restricted cash

Cash and cash equivalents, and restricted cash in the condensed consolidated statements of cash flows consists of the following (in thousands):


	As of June 30,
	2026		2025
Cash and cash equivalents	$	110,004	$	101,472
Restricted cash - current		494		75
Restricted cash - long-term		3,227		721
Total cash, cash equivalents and restricted cash	$	113,725	$	102,268

Accrued and Other Current Liabilities (Tables)

6 Months Ended

Jun. 30, 2026

Payables and Accruals [Abstract]

Schedule of Accrued and Other Current Liabilities

Accrued and other current liabilities consisted of the following (in thousands):


	As of June 30,		As of December 31,
	2026		2025
Accrued employee compensation costs	$	6,665	$	13,975
Accrued professional costs		4,011		965
Accrued research and development costs		14,474		22,028
Current portion of operating lease liabilities		1,845		3,007
Other current liabilities		1,618		1,060
	$	28,613	$	41,035

Segment Information (Tables)

6 Months Ended

Jun. 30, 2026

Segment Reporting [Abstract]

Summary of Segment Information

The following table reconciles segment direct profit or loss to the Company’s condensed consolidated results (in thousands):


	Three Months Ended June 30,						Six Months Ended June 30,
	2026			2025			2026			2025
Revenue	$	9,325		$	13,817		$	15,554		$	172,386
Zorevunersen(1)		28,243			11,382			48,097			29,056
ADOA(1)		2,697			1,747			4,555			2,140
SYNGAP1(1)		1,073			818			1,818			1,160
MECP2(1)		—			(200	)		—			(53	)
Other external program costs		3,990			1,841			7,169			3,670
Payroll and other personnel costs - R&D (2)		18,218			11,618			34,336			22,629
Payroll and other personnel costs - SG&A (2)		13,318			8,979			24,375			17,908
Other non-program costs - R&D (3)		1,628			1,615			3,308			3,022
Other non-program costs - SG&A (3)		5,587			3,317			10,742			8,914
Interest expense		(17	)		(3	)		(41	)		(3	)
Interest income		3,520			3,792			6,939			6,681
Other income		308			28			327			57
Provision for income taxes		—			—			—			(1,278	)
Segment net income (loss)	$	(61,618	)	$	(23,483	)	$	(111,621	)	$	89,397

(1) Includes external research and development and sales, general and administrative expenses associated with programs.

(2) Includes salaries, benefits and other personnel related expenses, including stock based compensation expense, of relevant personnel.

(3) Includes costs incurred for conducting non-program activities related to Company activities.

Tabular disclosure of segment reporting. Includes, but is not limited to, reconciliation of reportable segment amount to corresponding consolidated total.

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
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-URI https://asc.fasb.org/1943274/2147482810/280-10-50-15

Condensed consolidated balance sheets (Parenthetical) - $ / shares	Jun. 30, 2026	Dec. 31, 2025
Statement of Financial Position [Abstract]
Common stock, par value	$ 0.0001	$ 0.0001
Common stock, shares authorized	300,000,000	300,000,000
Common stock, shares issued	62,402,595	58,921,999
Common stock, shares outstanding	62,402,595	58,921,999

Number of shares of common stock outstanding. Common stock represent the ownership interest in a corporation.

Subsequent Events

6 Months Ended

Jun. 30, 2026

Subsequent Events [Abstract]

Subsequent events

12. Subsequent events

Since June 30, 2026, through the issuance of these condensed consolidated financial statements, the Company sold approximately 2.1 million shares of our common stock pursuant to the Sales Agreement and the 2026 Registration Statement for net proceeds of $65.7 million after deducting commissions.

Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Tables)

6 Months Ended

Jun. 30, 2026

Schedule of Cash and Cash Equivalents and Restricted Cash

Cash and cash equivalents, and restricted cash in the condensed consolidated statements of cash flows consists of the following (in thousands):


	As of June 30,
	2026		2025
Cash and cash equivalents	$	110,004	$	101,472
Restricted cash - current		494		75
Restricted cash - long-term		3,227		721
Total cash, cash equivalents and restricted cash	$	113,725	$	102,268

Accrued and Other Current Liabilities - Schedule of Accrued and Other Current Liabilities (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025
Payables and Accruals [Abstract]
Accrued employee compensation costs	$ 6,665	$ 13,975
Accrued professional costs	4,011	965
Accrued research and development costs	14,474	22,028
Current portion of operating lease liabilities	1,845	3,007
Other current liabilities	1,618	1,060
Total accrued and other current liabilities	$ 28,613	$ 41,035

Income Taxes

6 Months Ended

Jun. 30, 2026

Income Tax Disclosure [Abstract]

Income taxes

10. Income taxes

For the six months ended June 30, 2026, the Company did not have any income tax expense recorded in its condensed consolidated statements of operations and comprehensive (loss) income. For the six months ended June 30, 2025, the Company recorded federal and state tax expense of $1.1 million and $0.1 million, respectively due to recognizing income from the Biogen collaboration, offset by net operating loss carryforwards that were able to be utilized to the extent allowable by tax law. Prior recorded income tax expense of $1.1 million and $0.1 million for federal and state, respectively, were reversed due to the passage on July 4, 2025 of the One Big Beautiful Bill Act. The Company has provided a valuation allowance for the full amount of its net deferred tax assets as of June 30, 2026 and December 31, 2025, as management has determined it is more likely than not that any future benefit from deductible temporary differences and net operating loss and tax credit carryforwards would not be realized.

The entire disclosure for income tax.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
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Commitments and Contingencies Disclosure [Abstract]

Commitments and Contingencies - Summary of Lease Balances (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025	Jul. 31, 2025
Commitments and Contingencies Disclosure [Abstract]
Operating right-of-use assets	$ 1,870	$ 3,101	$ 1,800
Current portion of operating lease liabilities	$ 1,845	$ 3,007
Operating Lease Liability Current Statement Of Financial Position Extensible List	Liabilities, Current
Non-current portion of operating lease liabilities	$ 837
Operating Lease Liability Noncurrent Statement Of Financial Position Extensible List	Other long-term liabilities
Total operating lease liabilities	$ 2,682

Commitments and Contingencies

6 Months Ended

Jun. 30, 2026

Commitments and contingencies

6. Commitments and contingencies

Operating lease

The Company determines whether an arrangement is a lease at inception. The Company accounts for a lease when it has the right to control the leased asset for a period of time while obtaining substantially all of the assets’ economic benefits. Operating lease right-of-use assets and operating lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term at the lease commencement date. The discount rate used to determine the present value of the lease payments is the Company’s incremental borrowing rate based on the information available at lease inception, as the Company did not have information to determine the rate implicit in the leases. Lease expense for operating leases is recognized on a straight-line basis over the reasonably assured lease term based on the total lease payments (which include initial direct costs and lease incentives). Lease expense is included in operating expenses in the condensed consolidated statements of operations and comprehensive (loss) income. The Company’s lease agreements also contain variable payments, primarily maintenance-related costs, which are expensed as incurred and not included in the measurement of the right-of-use assets and lease liabilities.

In August 2018, the Company entered into an agreement to lease approximately 23,000 square feet of space for a term of three years. Lease terms are triple net lease commencing at $0.9 million per year, then with 3% annual base rent increases plus operating expenses, real estate taxes, utilities and janitorial fees. The lease commencement date was December 10, 2018.

In September 2021, the Company entered into an agreement to extend the initial term of the 23,000 square foot lease for a period of three years commencing on December 15, 2021 and ending December 31, 2024. In addition, this lease provides for the lease of an additional 15,000 square feet of rentable space beginning on April 1, 2022 and ending on December 31, 2024. In December 2021, the Company recognized a right-of-use asset and operating lease liability of $3.5 million for the 23,000 square feet. On April 1, 2022, the Company recognized a right-of-use asset and operating lease liability of $1.8 million for the 15,000 square feet.

In December 2023, the Company entered into an agreement to extend the term of the 38,000 square foot lease for a period of two years commencing on January 1, 2025 and ending on December 31, 2026. In December 2023, the Company recognized a right-of-use asset and operating lease liability of $4.1 million.

In January 2025, the Company entered into an agreement to lease 7,581 square feet of space for an initial term of three years and three months. The lease includes two renewal options for an additional three years each. Lease terms commence at $0.7 million per annum, with 2.0% annual base rent increases plus operating expenses, real estate taxes, and utilities. The lease commencement date was July 8, 2025. In July 2025, the Company recognized a right-of-use asset and operating lease liability of $1.8 million. In September 2025, the Company entered into a sublease agreement for the 7,581 square feet of space for a term equal to the initial term of the original lease. Sublease terms commence at $0.4 million per annum with 2% annual rent increases. As a result of this sublease, the Company recognized a right of use asset impairment of $0.7 million.

On January 21, 2026, the Company entered into a new lease for its corporate headquarters and laboratory space with NWALP PHOP Property Owner LLC, located at 245 Fifth Avenue, Waltham, Massachusetts (the “Lease Agreement”), in order to expand its office and laboratory space. The Lease Agreement premises include approximately 98,500 square feet. The Lease Agreement will expire on March 31, 2038, and includes two options to further extend the Lease Agreement for an additional five years at the then-prevailing market rate. The aggregate estimated base rent payments due over the term of the Lease Agreement is approximately $86.6 million. The Company paid a security deposit of $3.0 million in the form of an irrevocable standby letter of credit, which may be reduced over time in accordance with the terms of the Lease Agreement. The Company expects to occupy the space late in 2026 and has evaluated build to suit considerations as of June 30, 2026.

Future minimum lease payments under non-cancellable leases as of June 30, 2026 were as follows (in thousands):


2026		1,626
2027		700
2028		532
Total lease payments		2,858
Less imputed interest		(176	)
Present value of lease liabilities	$	2,682

Lease balances as of June 30, 2026 were as follows (in thousands):


Operating right-of-use assets	$	1,870
Current portion of operating lease liabilities	$	1,845
Non-current portion of operating lease liabilities		837
Total operating lease liabilities	$	2,682

The weighted average remaining lease term and weighted average discount rate of the Company’s operating leases as of June 30, 2026 were as follows:


Weighted average remaining lease term in years	1.5
Weighted average discount rate	10.05	%

Lease expense incurred under operating leases was $0.6 million and $0.7 million for the three months ended June 30, 2026 and 2025, respectively, and was $1.2 million and $1.4 million for the six months ended June 30, 2026 and 2025, respectively.

License and research agreements

In April 2016, the Company entered into an exclusive, worldwide license agreement with the University of Southampton (the “Southampton Agreement”), whereby the Company acquired rights to foundational technologies related to the Company’s proprietary protein upregulation platform technology. Under the Southampton Agreement, the Company receives an exclusive, worldwide license under certain licensed patents and applications relating to the Company's proprietary protein upregulation platform. Under the Southampton Agreement, the Company may be obligated to make additional payments that are contingent upon certain milestones being achieved, as well as royalties on future product sales. These royalty obligations survive until the latest of (i) the expiration of the last valid claim of a licensed patent covering a subject product or (ii) the expiration of any regulatory exclusivity for the subject product in a country. In addition, if the Company sublicenses its rights under the Southampton Agreement, the Company is required to pay a mid-single digit percentage of the sublicense revenue to the University of Southampton. As of June 30, 2026, the Company had paid $8.9 million under the Southampton Agreement as a result of the Acadia license and collaboration agreement and the Biogen license and collaboration agreement. Additionally, certain licenses under the Southampton Agreement require the Company to reimburse the University of Southampton for certain past and ongoing patent related expenses. For the three and six months ended June 30, 2026 and June 30, 2025 these expenses were immaterial.

Litigation

The Company may periodically become subject to legal proceedings and claims arising in connection with ongoing business activities, including claims or disputes related to patents that have been issued or that are pending in the field of research on which it is focused. As of June 30, 2026 and December 31, 2025, the Company was not subject to any legal proceedings and no legal proceedings are currently pending or threatened to which it was a party or to which its property was subject that, in the opinion of management, would have a material adverse effect on its business.

Marketable Securities - Summary of Marketable Securities (Details) - USD ($) $ in Thousands	Jun. 30, 2026	Dec. 31, 2025
Marketable Securities [Line Items]
Marketable Securities, Amortized Cost	$ 256,792	$ 306,165
Marketable Securities, Unrealized Gain	34	555
Marketable Securities, Unrealized Loss	(577)	(10)
Marketable Securities, Fair Value	256,249	306,710
Corporate Bonds
Marketable Securities [Line Items]
Marketable Securities, Amortized Cost	60,545	84,568
Marketable Securities, Unrealized Gain	14	183
Marketable Securities, Unrealized Loss	(78)	(1)
Marketable Securities, Fair Value	60,481	84,750
Commercial Paper
Marketable Securities [Line Items]
Marketable Securities, Amortized Cost	22,773	23,959
Marketable Securities, Unrealized Gain	0	7
Marketable Securities, Unrealized Loss	(14)	(5)
Marketable Securities, Fair Value	22,759	23,961
US Government Debt Securities
Marketable Securities [Line Items]
Marketable Securities, Amortized Cost	173,474	197,638
Marketable Securities, Unrealized Gain	20	365
Marketable Securities, Unrealized Loss	(485)	(4)
Marketable Securities, Fair Value	$ 173,009	$ 197,999

Amortized cost of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

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