v3.26.1
Summary of Significant Accounting Policies (Tables)
12 Months Ended
Dec. 31, 2025
Summary of Significant Accounting Policies  
Schedule of Restricted Cash

December 31, 

December 31, 

2025

2024

Cash and cash equivalents

  ​ ​ ​

$

65,931

  ​ ​ ​

$

103,659

Restricted cash

 

2,262

 

2,009

Total cash, cash equivalents and restricted cash in the consolidated statement of cash flows

$

68,193

$

105,668

Schedule of Financial Assets and Liabilities Measured at Fair Value

Fair Value Measurement Using:

  ​ ​ ​

  ​ ​ ​

Significant

  ​ ​ ​

Significant Other

  ​ ​ ​

Significant

December 31, 

Observable Inputs

Observable Inputs

Unobservable

Description

2025

(Level 1)

(Level 2)

(Level 3)

Cash equivalents

$

60,253

$

60,253

$

$

Restricted cash

$

2,262

$

2,262

$

$

Fair Value Measurement Using:

  ​ ​ ​

  ​ ​ ​

Significant

  ​ ​ ​

Significant Other

  ​ ​ ​

Significant

December 31, 

Observable Inputs

Observable Inputs

Unobservable

Description

2024

(Level 1)

(Level 2)

(Level 3)

Cash equivalents

$

80,930

$

80,930

$

$

Restricted cash

$

2,009

$

2,009

$

$

Schedule of Estimated Useful Lives of Assets

Asset Category

  ​ ​ ​

Useful Lives

Computer and office equipment

 

3 years

Laboratory equipment

 

5 years

Furniture and fixtures

 

5 years

Manufacturing equipment

 

7 years

Leasehold improvements

 

lesser of useful life or remaining term of lease

Schedule of Change in Asset Retirement Obligations

For the Years Ended December 31, 

2025

2024

Balance at beginning of period

  ​ ​ ​

$

2,821

  ​ ​ ​

$

2,401

Change in estimate

(1,676)

345

Assignment of leases to Johnson & Johnson Innovative Medicine

(115)

Amortization of interest

 

153

 

209

Effects of exchange rate changes

 

101

 

(19)

Balance at end of period

$

1,399

$

2,821

Schedule of Securities Excluded from Computation of Diluted Net Loss per Share

  ​ ​ ​

December 31, 

  ​ ​ ​

December 31, 

  ​ ​ ​

2025

  ​ ​ ​

2024

Share options

 

7,859,338

 

8,232,587

Restricted share units

6,184,250

4,252,250

Warrants

700,000

700,000

 

14,743,588

 

13,184,837

Schedule of Net Loss for the Segment

For the Years Ended December 31, 

2025

2024

Service revenue - related party

$

6,400

  ​ ​ ​

$

33,279

License revenue

 

74,991

 

Operating expenses:

Cost of service revenue - related party

4,843

23,791

General and administrative

 

38,480

 

39,389

Clinical programs:

Botaretigene sparoparvovec

1,250

AAV-hAQP1

13,762

17,307

AAV-CNGB3 / AAV-CNGA3

(969)

AAV-GAD

14,268

6,411

Other ocular diseases

4,380

1,763

Manufacturing

44,288

53,445

Preclinical programs:

Gene regulation

9,426

10,509

Neurodegenerative diseases

1,116

1,608

Preclinical ocular diseases

3,567

2,474

Other research and development1

18,582

2,494

Share-based compensation

22,105

25,191

Depreciation and amortization

12,542

12,828

Total operating expenses

187,359

197,491

Other segment items2

(8,233)

16,421

Segment net loss

$

(114,201)

(147,791)

Net loss

$

(114,201)

$

(147,791)

1 Other research and development is comprised of all other costs including payroll and payroll related costs, travel, rent and facilities costs and other non-program specific expenses.

2 Other segment items is comprised of foreign currency gain (loss), interest income, interest expense and gain on sale of nonfinancial assets.

Schedule of Long-lived Assets by Geographical Area

  ​ ​ ​

December 31, 

  ​ ​ ​

December 31, 

2025

2024

United States

$

8,830

$

7,209

United Kingdom

 

26,092

 

28,419

European Union

107,589

100,845

$

142,511

$

136,473