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 MYBPC3 mutation-associated nonobstructive hypertrophic cardiomyopathy (nHCM). (Show More)
Indication	Hypertrophic Cardiomyopathy
Compound Name	TN-201
Sponsor	Tenaya Therapeutics
Funder Type	Industry
Status	Recruiting
Enrollment Count	15

Target Gene/Variant	MYBPC3
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type
Dose 1	3 x 10^13 vg/kg
Dose 2	6 x 10^13 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	10
Locations	United States

Has US IND	True
Recent Updates	Initial data from cohort 1 available H2 2024; enrolling patients for high dose (Cohort 2)