16. Segment and Geographical 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 December 31, 2025 and 2024, the Company’s cash, cash equivalents and marketable securities were $240.9 million and $244.9 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 income (loss) (in thousands):

 

 

Years Ended December 31,

 

 

 

2025

 

 

2024

 

Revenues

 

$

170,441

 

 

$

83,328

 

Less:

 

 

 

 

 

 

Cost of license and royalty revenues

 

 

20,298

 

 

 

33,567

 

Research and development expense

 

 

 

 

 

 

Personnel

 

 

73,508

 

 

 

65,950

 

Direct development and support (a)

 

 

112,887

 

 

 

97,951

 

Facilities

 

 

11,276

 

 

 

11,410

 

Stock-based compensation

 

 

15,977

 

 

 

17,985

 

Depreciation and amortization

 

 

14,651

 

 

 

15,226

 

Total research and development expense

 

 

228,299

 

 

 

208,522

 

General and administrative expense

 

 

 

 

 

 

Personnel

 

 

22,793

 

 

 

20,816

 

Other general and administrative (b)

 

 

35,018

 

 

 

29,480

 

Facilities

 

 

5,502

 

 

 

4,856

 

Stock-based compensation

 

 

18,586

 

 

 

20,478

 

Depreciation and amortization

 

 

964

 

 

 

989

 

Total general and administrative expense

 

 

82,863

 

 

 

76,619

 

Other segment items (c)

 

 

(32,859

)

 

 

8,278

 

Net loss

 

$

(193,878

)

 

$

(227,102

)

 

(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, commercial cost reimbursement to and from collaborators and other corporate overhead expenses.
(c)
Other segment items include credit losses (recoveries), impairment of long-lived assets, other operating expenses, interest income from licensing, investment income and interest expense.

The Company’s interest income during the years ended December 31, 2025 and 2024 included interest income from licensing as presented in the consolidated statements of operations and comprehensive loss, as well as interest income from investments of $12.2 million and $12.1 million, respectively, which is included within investment income in the consolidated statements of operations and comprehensive loss.

For the year ended December 31, 2025, 44% and 25% of the Company’s revenues were attributed to Japan and the United States, respectively, and no other countries accounted for 10% or more of the Company’s revenues. For the year ended December 31, 2024, 36%, 10% and 10% of the Company’s revenues were attributed to the United States, Germany and United Arab Emirates, respectively, and no other countries accounted for 10% or more of the Company’s revenues. The country of origin for license revenue is determined based on the country of domicile of the licensee. The country of origin for royalty revenue is determined based on the location of the underlying net sales of licensed products. The country of origin for service revenue is determined based on the location where the Company principally performs the services.

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

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Historical Timeline

Fiscal YearFiled
2025Mar 5, 2026Showing above
2024Mar 13, 2025

About Segments Disclosures

Segment disclosures break a company into its reportable operating units, revealing revenue, profit, and asset allocation that consolidated financial statements obscure. Under ASC 280, segments must match how the chief operating decision maker views the business, providing a window into internal management structure and resource allocation priorities.

Key signals: compare segment margins to identify which units drive profitability and which destroy value. Watch for changes in the number of reportable segments — segment aggregation or disaggregation often coincides with strategic shifts or attempts to obscure declining performance. Intersegment elimination patterns reveal internal pricing practices. The reconciliation between segment totals and consolidated figures exposes corporate overhead allocation and unallocated items. Geographic revenue concentration highlights regulatory and currency exposure. Compare segment-level capital expenditure against segment revenue to assess where management is investing for future growth versus harvesting existing assets.