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Skeletal Muscle Regeneration Program

Duchenne Muscular Dystrophy (DMD)

Pre-clinicalActive

Key Facts

Indication

Duchenne Muscular Dystrophy (DMD)

Phase

Pre-clinical

Status

Active

Company

IPS HEART

About IPS HEART

IPS HEART is a private, pre-clinical stage biotech founded in 2015 and based in San Diego, California. The company is pioneering iPSC-derived cellular therapies designed to regenerate functional muscle tissue for cardiac and skeletal muscle disorders, moving beyond symptomatic treatment to potential cures. It has garnered regulatory recognition from the FDA, including Orphan Drug and Rare Pediatric Disease Designations. The company is currently pre-revenue and is advancing a pipeline focused on two lead programs for heart failure and DMD.

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Therapeutic Areas

Cardiology Neuromuscular Diseases

Other Duchenne Muscular Dystrophy (DMD) Drugs

Drug	Company	Phase	Status
DT-DEC01	Dystrogen Therapeutics	Phase 1	Active (Dose Escalation)
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)
EXONDYS 51 (eteplirsen)	Sarepta Therapeutics	Marketed	FDA Approved