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Deramiocel (CAP-1002)

Duchenne Muscular Dystrophy (DMD)

Phase 3BLA Submitted, PDUFA date establishedNCT05126758 (HOPE-3)

Key Facts

Indication

Duchenne Muscular Dystrophy (DMD)

Phase

Phase 3

Status

BLA Submitted, PDUFA date established

Company

Capricor Therapeutics

About Capricor Therapeutics

Capricor Therapeutics is dedicated to advancing a new class of medicines using its core expertise in cardiosphere-derived cells (CDCs) and exosome science. The company is on the cusp of a potential regulatory milestone with Deramiocel for DMD, supported by positive late-breaking HOPE-3 trial data. Beyond its lead program, Capricor is building a broader pipeline through its StealthX™ platform, targeting the delivery of oligonucleotides, proteins, and small molecules, positioning it at the forefront of precision-engineered exosome therapeutics.

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Therapeutic Areas

Rare Diseases Muscular Dystrophy Cardiology

Other Duchenne Muscular Dystrophy (DMD) Drugs

Drug	Company	Phase	Status
AOC Program	Avidity Biosciences	Clinical	Active
AGAMREE® (vamorolone)	Catalyst Pharmaceuticals	Approved/Commercial	Marketed; Long-term safety study (SUMMIT Registry) ongoing
SRP-9001 (delandistrogene moxeparvovec)	Sarepta Therapeutics	Approved	Commercial
RGX-202	REGENXBIO	Phase 1/2	Active
PBGENE-DMD	Precision BioSciences	Phase 1/2	Active - IND Cleared; Clinical trial initiation
DT-DEC01	Dystrogen Therapeutics	Phase 1	Active (Dose Escalation)