Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

NCTID	NCT05836259 (View at clinicaltrials.gov)
Description	This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene. (Show More)
Development Status	Active
Indication	Hypertrophic Cardiomyopathy
Disease Ontology Term	DOID:0110310
Compound Name	TN-201
Sponsor	Tenaya Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	30 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	MYBPC3
Therapy Type	Gene transfer
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Delivery System	Viral transduction
Vector Type	AAV9
Dose 1 🔄	Dose escalation: 3E13 vg/kg, 6E13 vg/kg; n=3/group
Dose 2 🔄	Dose expansion: 6E13 vg/kg

Eligible Age	18 Years - 75 Years
Standard Ages	Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * MYBPC3 mutation * Hypertrophic Cardiomyopathy (obstructive and nonobstructive) * Left Ventricular Ejection Fraction ≥45% * NYHA Functional Class II or III symptoms * NT-proBNP ≥160pg/ml Exclusion Criteria: * High AAV9 neutralizing antibody titer View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	10
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Interim results announced June 2026