Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults with PKP2 Mutation-associated ARVC

NCTID	NCT06228924 (View at clinicaltrials.gov)
Description	This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC. (Show More)
Indication	Arrhythmogenic Right Ventricular Cardiomyopathy
Compound Name	TN-401
Sponsor	Tenaya Therapeutics
Funder Type	Industry
Status	Recruiting
Enrollment Count	15

Target Gene/Variant	PKP2
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	none
Dose 1	3E13 vg/kg
Dose 2	6E13 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	7
Locations	United States

Has US IND	True
Recent Updates	First patient dosed November 2024, initial data expected 2H2025