Home/Pipeline/RGX-202

RGX-202

Duchenne Muscular Dystrophy (DMD)

Phase I/IIRecruitingNCT06256810

Key Facts

Indication

Duchenne Muscular Dystrophy (DMD)

Phase

Phase I/II

Status

Recruiting

Company

REGENXBIO

About REGENXBIO

REGENXBIO is a clinical-stage biotech with a mission to deliver life-changing gene therapies through its foundational NAV Technology Platform. Its achievements include enabling the blockbuster therapy ZOLGENSMA® and building a diverse internal pipeline, most notably in MPS II and Duchenne muscular dystrophy. The company's strategy combines internal development of wholly-owned assets with a high-margin, capital-efficient licensing model that provides recurring revenue and validates its technology.

View full company profile

Therapeutic Areas

Rare Genetic Diseases Ophthalmology Neuromuscular

Other Duchenne Muscular Dystrophy (DMD) Drugs

Drug	Company	Phase	Status
DT-DEC01	Dystrogen Therapeutics	Phase 1	Active (Dose Escalation)
Skeletal Muscle Regeneration Program	IPS HEART	Pre-clinical	Active
KYMBEE (deflazacort)	Upsher-Smith	Approved	Active
ALY688ER	Allysta Pharmaceuticals	Phase 1	Active
AVGN7 + AVGND	AAVogen	Pre-clinical	Active
Myomatrix Stabilization Program	Strykagen	Preclinical	Active
RIPPLE™ (implied)	SonoThera	Preclinical	Active
Vamorolone (AGAMREE)	ReveraGen BioPharma	Approved	Active
AGAMREE (vamorolone)	Catalyst Pharmaceuticals	Marketed	Commercial; Long-term safety monitored via SUMMIT Registry
EDG-5506	Edgewise Therapeutics	Phase 2	Active
ELEVIDYS (delandistrogene moxeparvovec-rokl)	Sarepta Therapeutics	Marketed / Phase 3	FDA Accelerated Approval; Confirmatory trial ongoing
SRP-5051 (vesleteplirsen)	Sarepta Therapeutics	Phase 2	Active (Next-gen PPMO)