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TN-301

Duchenne Muscular Dystrophy (DMD)

PreclinicalActive

Key Facts

Indication

Duchenne Muscular Dystrophy (DMD)

Phase

Preclinical

Status

Active

Company

Tenaya Therapeutics

About Tenaya Therapeutics

Tenaya Therapeutics is a clinical-stage biotech dedicated to discovering and delivering curative therapies for the underlying genetic drivers of heart disease. The company has reported promising early clinical data for its two lead gene therapy programs, TN-201 for MYBPC3-associated HCM and TN-401 for PKP2-associated ARVC, and is advancing a preclinical small molecule, TN-301, for Duchenne muscular dystrophy. Its strategy leverages a precision medicine approach, validated genetic targets, and a multi-modal platform to address significant unmet needs in cardiovascular and neuromuscular diseases.

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Therapeutic Areas

Cardiovascular 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)