REGENXBIO Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights

Late-stage gene therapy pipeline for rare and retinal diseases advancing toward key catalysts

Published on Mar. 5, 2026

Got story updates? Submit your updates here. ›

REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the fourth quarter and year ended December 31, 2025. The company is rapidly advancing its late-stage pipeline of gene therapies to treat rare and retinal diseases with significant unmet need, with multiple near-term catalysts in 2026.

Why it matters

REGENXBIO's gene therapy programs, including RGX-202 for Duchenne muscular dystrophy and surabgene lomparvovec (sura-vec, ABBV-RGX-314) for wet age-related macular degeneration and diabetic retinopathy, have the potential to deliver meaningful new medicines to patients in need through the curative potential of gene therapy.

The details

Key highlights include positive 18-month functional data from the Phase I/II trial of RGX-202 for Duchenne, plans to share pivotal topline data in early Q2 2026, and upcoming topline data from the pivotal trials of sura-vec for wet AMD and the initiation of a pivotal Phase IIb/III trial for diabetic retinopathy. The company also provided updates on its neurodegenerative disease programs RGX-121 and RGX-111, which are on clinical hold.

  • In January 2026, positive 18-month functional data from the Phase I/II trial of RGX-202 for Duchenne were reported.
  • REGENXBIO expects to share pivotal topline data for RGX-202 in early Q2 2026.
  • REGENXBIO expects to share topline data from the ATMOSPHERE and ASCENT pivotal trials of sura-vec for wet AMD in Q4 2026.
  • REGENXBIO plans to activate U.S. clinical sites for the pivotal Phase IIb/III NAAVIGATE study of sura-vec for diabetic retinopathy, with first patient dosed expected in Q2 2026.
  • In February 2026, the FDA issued a complete response letter for the RGX-121 BLA.

The players

REGENXBIO Inc.

A biotechnology company on a mission to improve lives through the curative potential of gene therapy, advancing a late-stage pipeline of one-time treatments for rare and retinal diseases.

Curran Simpson

President and Chief Executive Officer of REGENXBIO.

AbbVie

REGENXBIO's collaboration partner for the development of surabgene lomparvovec (sura-vec) for the treatment of wet AMD and diabetic retinopathy.

Nippon Shinyaku

REGENXBIO's partner for the development of clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I.

FDA

The U.S. Food and Drug Administration, which placed clinical holds on REGENXBIO's RGX-111 and RGX-121 programs and issued a complete response letter for the RGX-121 BLA.

Got photos? Submit your photos here. ›

What they’re saying

“We are rapidly advancing our late-stage pipeline of gene therapies to treat rare and retinal diseases with significant unmet need, with multiple near-term catalysts in 2026.”

— Curran Simpson, President and Chief Executive Officer, REGENXBIO

What’s next

REGENXBIO plans to request a pre-BLA meeting in mid-2026 to align with the FDA on the BLA submission for RGX-202. The company is also working to address concerns in the FDA's complete response letter for the RGX-121 BLA with the goal of resubmitting the BLA.

The takeaway

REGENXBIO's focus on advancing its late-stage gene therapy pipeline for rare and retinal diseases, with multiple near-term catalysts expected in 2026, highlights the company's commitment to delivering meaningful new medicines to patients in need through the curative potential of gene therapy.