Regenxbio Inc. (NASDAQ: RGNX) has reported its financial results and operational highlights for the fourth quarter and full year ended December 31, 2025. The company's cash, cash equivalents, and marketable securities were $240.9 million as of December 31, 2025, compared to $244.9 million as of December 31, 2024. Revenues for the three months and full year ended December 31, 2025, were $30.3 million and $170.4 million, respectively, compared to $21.2 million and $83.3 million for the same periods in 2024.
Research and development expenses were $59.6 million and $228.3 million for the three months and full year ended December 31, 2025, respectively, compared to $50.4 million and $208.5 million for the same periods in 2024. General and administrative expenses were $22.4 million and $82.9 million for the three months and full year ended December 31, 2025, respectively, compared to $20.1 million and $76.6 million for the same periods in 2024.
Regenxbio's net loss was $67.1 million, or $1.30 basic and diluted net loss per share, for the three months ended December 31, 2025, compared to a net loss of $51.2 million, or $1.01 basic and diluted net loss per share, for the three months ended December 31, 2024. The net loss for the full year ended December 31, 2025, was $193.9 million, or $3.76 basic and diluted net loss per share, compared to a net loss of $227.1 million, or $4.59 basic and diluted net loss per share, for the full year ended December 31, 2024.
Regenxbio expects its cash, cash equivalents, and marketable securities of $240.9 million as of December 31, 2025, to fund its operations into early 2027.
On the operational front, Regenxbio's late-stage gene therapy pipeline for rare and retinal diseases is advancing toward key catalysts. Some notable highlights include:
- Positive 18-month functional data from patients treated with the pivotal dose in the phase I/II portion of the Affinity Duchenne® trial (n=4) were reported in January 2026.
- The company plans to share additional phase I/II safety, biomarker, and functional data at the MDA Clinical and Scientific Conference on March 11, 2026.
- Regenxbio continues to manufacture RGX-202 intended for commercial supply at its in-house manufacturing innovation center.
- Regenxbio expects to share pivotal topline data for RGX-202 in early Q2 2026.
- Regenxbio plans to request a pre-BLA meeting in mid-2026 to align with the FDA on the BLA submission.
The company also provided updates on its gene therapy programs for retinal and neurodegenerative diseases.
Regenxbio will host a conference call and webcast at 8:00 a.m. ET to discuss these financial results and operational highlights.
About Regenxbio Inc. Regenxbio is a biotechnology company focused on advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne, RGX-121 for the treatment of MPS II, RGX-111 for the treatment of MPS I, and Sura-vec for the treatment of wet AMD and diabetic retinopathy. Following these announcements, the company's shares moved -1.02%, and are now trading at a price of $13.62. Check out the company's full 8-K submission here.
