v3.26.1
Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Tables)
6 Months Ended
Jun. 30, 2026
Summary of Significant Accounting Policies and Recent Accounting Pronouncements  
Schedule of Restricted Cash The following table provides a reconciliation of the components of cash and cash equivalents and restricted cash reported in the Company’s condensed consolidated balance sheets to the total of the amount presented in the condensed consolidated statements of cash flows (in thousands):

June 30, 

December 31, 

2026

2025

Cash and cash equivalents

  ​ ​ ​

$

143,166

  ​ ​ ​

$

65,931

Restricted cash

 

2,200

 

2,262

Total cash, cash equivalents and restricted cash in the condensed consolidated statements of cash flows

$

145,366

$

68,193

Schedule of Financial Assets and Liabilities Measured at Fair Value

The table below represents the values of the Company's financial assets and liabilities that are required to be measured at fair value on a recurring basis (in thousands):

Financial Asset (Liability)

Fair Value Measurement Using:

  ​ ​ ​

  ​ ​ ​

Significant

  ​ ​ ​

Significant Other

  ​ ​ ​

Significant

June 30, 

Observable Inputs

Observable Inputs

Unobservable Inputs

Description

2026

(Level 1)

(Level 2)

(Level 3)

Cash equivalents

$

97,147

$

97,147

$

$

Contingent forward instrument (Note 5)

$

21,800

$

$

$

21,800

Royalty Notes (Note 12)

$

(100,000)

$

$

$

(100,000)

Derivative liability (Note 5)

$

(5,223)

$

$

$

(5,223)

Equity option instrument (Note 5)

$

(2,450)

$

$

$

(2,450)

Financial Asset (Liability)

Fair Value Measurement Using:

  ​ ​ ​

  ​ ​ ​

Significant

  ​ ​ ​

Significant Other

  ​ ​ ​

Significant

December 31, 

Observable Inputs

Observable Inputs

Unobservable Inputs

Description

2025

(Level 1)

(Level 2)

(Level 3)

Cash equivalents

$

60,253

$

60,253

$

$

Schedule of estimate fair value

June 30, 

Instrument

Valuation Technique

Significant Unobservable Input

  ​ ​ ​

2026

Contingent forward instrument

Monte Carlo simulation

Expected volatility
Expected term
Discount rate

 

47.5%
1 year
3.6% - 6.0%

Royalty Notes

Scenario-based analysis

Discount rate

Derivative liability

Monte Carlo simulation

Expected volatility
Risk-free interest rate

80%
4.1%

Equity option instrument

Monte Carlo simulation

Expected volatility
Expected term
Discount rate

47.5%
2 years
3.6% - 6.1%

Schedule of Change in Asset Retirement Obligations

The change in asset retirement obligations is as follows (in thousands):

For the Six-Month Periods Ended June 30, 

2026

2025

Balance at beginning of period

  ​ ​ ​

$

1,399

  ​ ​ ​

$

2,821

Change in estimate

(1,576)

Amortization of interest

 

74

 

98

Effects of exchange rate changes

 

(23)

 

111

Balance at end of period

$

1,450

$

1,454

Schedule of Net income (loss) for the Segment

The following table sets forth information about the Company’s single reportable segment and the significant expenses reviewed by the CODM, including a reconciliation to net income (loss) (in thousands):

For the Three-Month Periods Ended June 30, 

For the Six-Month Periods Ended June 30, 

2026

2025

2026

2025

Service revenue - related party

$

104,906

  ​ ​ ​

$

3,691

  ​ ​ ​

$

105,199

  ​ ​ ​

$

5,617

Service revenue

 

11,885

  ​ ​ ​

 

11,885

 

License revenue - related party

204,643

  ​ ​ ​

 

204,643

 

Operating expenses:

Cost of service revenue

1,401

1,401

Cost of service revenue - related party

5,763

  ​ ​ ​

2,676

5,961

4,054

General and administrative

 

9,146

  ​ ​ ​

8,607

 

15,316

 

14,951

Clinical programs:

Botaretigene sparoparvovec

31,302

31,331

AAV-hAQP1

7,349

2,754

9,704

7,606

AAV-GAD

3,098

8,970

4,010

10,430

Other ocular diseases

20

108

1,465

Manufacturing

5,046

8,888

21,409

22,989

Preclinical programs:

Gene regulation

1,252

3,241

2,673

5,527

Neurodegenerative diseases

328

298

729

724

Ocular diseases

180

1,162

1,003

1,787

Other research and development1

5,540

3,019

11,210

5,651

Share-based compensation

3,779

5,659

7,433

10,551

Depreciation and amortization

2,825

3,190

5,831

6,271

Total operating expenses

77,009

48,484

118,119

92,006

Other segment items2

(83,756)

5,998

(89,252)

7,613

Segment net income (loss)

$

160,669

$

(38,795)

$

114,356

(78,776)

Net income (loss)

$

160,669

$

(38,795)

$

114,356

$

(78,776)

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 (loss) gain, interest income, interest expense, income tax expense, loss on derivative liability and loss on equity method investee.

Schedule of Long-lived Assets by Geographical Area

The following table summarizes long-lived assets by geographical area (in thousands):

  ​ ​ ​

June 30, 

  ​ ​ ​

December 31, 

2026

2025

United States

$

8,189

$

8,830

United Kingdom

 

25,229

 

26,092

European Union

101,284

107,589

$

134,702

$

142,511