REGENXBIO

NASDAQ: RGNX ↗ $7.80 USD ~$700M MCAP

#Gene Therapy (AAV)#Duchenne Muscular Dystrophy (DMD)#Hunter Syndrome (MPS II)#Wet Age-Related Macular Degeneration (Wet AMD)#Rare Diseases

REGENXBIO is a clinical-stage biotechnology company developing gene therapies using its proprietary AAV (adeno-associated virus) NAV technology platform. The company is advancing a pipeline of one-time gene therapies for rare and serious diseases including Duchenne muscular dystrophy (RGX-202), Hunter syndrome (RGX-121), and wet age-related macular degeneration (RGX-314, partnered with AbbVie).

Company Info

Sector: Biotechnology / Gene Therapy

Headquarters: Rockville, Maryland, USA

Founded: 2009

Website: www.regenxbio.com/

Upcoming Milestones & Key Dates

Mid-2026

FDA Meeting

FDA Type A meeting to discuss CRL and path forward for RGX-121 in Hunter syndrome

Q2 2026

Earnings

Q1 2026 Earnings — Pipeline update and RGX-202 pivotal trial enrollment

Clinical Development Pipeline

RGX-202

Target: Microdystrophin transgene delivered via AAV8

Duchenne muscular dystrophy (DMD)

Preclinical

Phase 1

Phase 2

Phase 3

Approved

RGX-121

Target: Iduronate-2-sulfatase (IDS) gene via AAV9

Hunter syndrome (MPS II) — first one-time therapy for the underlying genetic cause

Preclinical

Phase 1

Phase 2

Phase 3

Approved

RGX-314

Target: Anti-VEGF Fab delivered via AAV8 (subretinal or suprachoroidal)

Wet age-related macular degeneration (wet AMD) and diabetic retinopathy

Preclinical

Phase 1

Phase 2

Phase 3

Approved

Kenneth T. Mills

President and Chief Executive Officer

Dr. Olivier Danos, Ph.D.

Chief Scientific Officer

Mitchell Chan

Senior Vice President, Chief Financial Officer

Careers & Job Openings

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Multiple Departments

See careers page

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