v3.26.3
Segment Information
 9 Months Ended
Sep. 30, 2026
Segment Reporting [Abstract]  
Segment Information

14. Segment Information

Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker (CODM), or decision-making group, in making decisions on how to allocate resources and assess performance. The Company’s CODM, its Chief Executive Officer, views the Company’s operations and manages the business as one operating segment focused on the development and commercialization of gene therapies to treat an array of diseases. The determination of a single operating segment is consistent with the consolidated financial information regularly provided to the CODM.

The CODM reviews and evaluates consolidated net income (loss) for purposes of assessing performance, making operating decisions and allocating resources. The CODM uses net income (loss) to assess performance versus operating budgets and in the preparation of near-term and long-range operating plans to inform decisions on resource and capital allocation. The CODM reviews consolidated cash, cash equivalents and marketable securities as a measure of segment assets. As of September 30, 2026 and December 31, 2025, the Company’s cash, cash equivalents and marketable securities were $251.0 million and $240.9 million, respectively.

The following table presents information about the Company's segment revenues, significant segment expenses regularly provided to the CODM, other segment items and consolidated net loss (in thousands):

 

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2026

 

 

2025

 

 

2026

 

 

2025

 

Revenues

 

$

9,162

 

 

$

29,733

 

 

$

123,574

 

 

$

140,104

 

Less:

 

 

 

 

 

 

 

 

 

 

 

 

Cost of license and royalty revenues

 

 

1,141

 

 

 

5,725

 

 

 

13,257

 

 

 

14,370

 

Research and development expense

 

 

 

 

 

 

 

 

 

 

 

 

Personnel

 

 

18,544

 

 

 

18,464

 

 

 

57,483

 

 

 

55,153

 

Direct development and support (a)

 

 

32,478

 

 

 

26,800

 

 

 

86,500

 

 

 

81,860

 

Facilities

 

 

3,293

 

 

 

2,946

 

 

 

8,996

 

 

 

8,467

 

Stock-based compensation

 

 

3,377

 

 

 

4,245

 

 

 

10,988

 

 

 

12,181

 

Depreciation and amortization

 

 

3,317

 

 

 

3,646

 

 

 

10,467

 

 

 

11,027

 

Total research and development expense

 

 

61,009

 

 

 

56,101

 

 

 

174,434

 

 

 

168,688

 

General and administrative expense

 

 

 

 

 

 

 

 

 

 

 

 

Personnel

 

 

5,815

 

 

 

5,567

 

 

 

17,812

 

 

 

17,055

 

Other general and administrative (b)

 

 

8,560

 

 

 

8,331

 

 

 

26,233

 

 

 

24,751

 

Facilities

 

 

1,263

 

 

 

1,362

 

 

 

3,992

 

 

 

3,951

 

Stock-based compensation

 

 

4,743

 

 

 

4,753

 

 

 

14,802

 

 

 

14,007

 

Depreciation and amortization

 

 

198

 

 

 

240

 

 

 

662

 

 

 

719

 

Total general and administrative expense

 

 

20,579

 

 

 

20,253

 

 

 

63,501

 

 

 

60,483

 

Other segment items (c)

 

 

32,829

 

 

 

(9,595

)

 

 

19,538

 

 

 

(23,292

)

Net loss

 

$

(40,738

)

 

$

(61,941

)

 

$

(108,080

)

 

$

(126,729

)

 

(a)
Direct development and support includes external goods and services for the development of product candidates and early-stage research activities, laboratory costs, consulting, development cost reimbursement to and from collaborators and other expenses in support of research and development activities.
(b)
Other general and administrative expenses include professional and administrative services, consulting, commercialization expenses, commercial cost reimbursement to and from collaborators and other corporate overhead expenses.
(c)
Other segment items include gain on litigation settlement, other operating expenses (income), interest income from licensing, investment income and interest expense.

 

The Company’s interest income included interest income from licensing as presented in the consolidated statements of operations and comprehensive loss, as well as interest income from investments of $2.3 million and $5.5 million during the three and nine months ended September 30, 2026, respectively, and $3.6 million and $9.4 million during the three and nine months ended September 30, 2025, respectively, which is included within investment income in the consolidated statements of operations and comprehensive loss.

The substantial majority of the Company’s assets reside in the United States.