RGX-202
Duchenne Muscular Dystrophy (DMD)
Key Facts
About REGENXBIO
REGENXBIO is a publicly traded gene therapy leader built around its foundational NAV Technology Platform, comprising exclusive rights to over 100 novel AAV vectors, including the high-performing AAV8 and AAV9. The company's strategy combines advancing its own clinical pipeline, most notably in wet AMD with RGX-314, with a lucrative licensing model that includes partnerships with major biopharma companies like Novartis, Pfizer, and Takeda. With multiple clinical readouts expected in the near term and a strong financial position, REGENXBIO is positioned to be a key player in the next generation of gene therapies.
View full company profileOther Duchenne Muscular Dystrophy (DMD) Drugs
| Drug | Company | Phase |
|---|---|---|
| AOC Program | Avidity Biosciences | Clinical |
| AGAMREE® (vamorolone) | Catalyst Pharmaceuticals | Approved/Commercial |
| SRP-9001 (delandistrogene moxeparvovec) | Sarepta Therapeutics | Approved |
| Deramiocel (CAP-1002) | Capricor Therapeutics | Phase 3 |
| PBGENE-DMD | Precision BioSciences | Phase 1/2 |
| DT-DEC01 | Dystrogen Therapeutics | Phase 1 |