REGENXBIO
June 23, 2026
Theater 3
Type: Company Presentation
Focus Area:
Cell and Gene Therapy and Genome Editing
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.
Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, our product candidates are designed to provide long-lasting effects, potentially significantly altering the course of disease and delivering improved patient outcomes.
We are developing gene therapy product candidates for the treatment of rare and retinal diseases. Our product candidates utilize AAV viral vectors from our proprietary NAV® Technology Platform. In addition to our internal product candidate programs, we also selectively license our NAV® vectors, enabling the development of new medicines. Thousands of patients have been treated with investigational and approved products built on REGENXBIO’s AAV platform.
Company HQ City:
Rockville
Company HQ State:
MD
Company HQ Country:
United States
Year Founded:
2009
Lead Product in Development:
RGX-121
CEO
Curran Simpson
Development Phase of Lead Product
NDA Preparation/In Review
Number of Unlicensed Products Looking for Licensing
2
Exchange
Nasdaq
Ticker
RGNX
When you expect your next catalyst update?
Q2 2026
What is your next catalyst (value inflection) update?
REGENXBIO expects to share pivotal topline data in early Q2 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026. Following the completion of enrollment in the pivotal trial (n=30) in October 2025, the Company continues to enroll in the confirmatory trial and expects to have majority of this trial enrolled at the time of BLA filing.
Regulatory interactions with the FDA and European Medical Association (EMA) are planned for 1H 2026, supporting the global expansion of the AFFINITY DUCHENNE® trial.
Primary Speaker
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