Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

NCTID	NCT06109181 (View at clinicaltrials.gov)
Description	This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM (Show More)
Indication	Arrhythmogenic Right Ventricular Cardiomyopathy
Compound Name	LX2020
Sponsor	Lexeo Therapeutics
Funder Type	Industry
Status	Recruiting
Enrollment Count	10

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	AAVrh10
Editor Type
Dose 1	Starting dose: 2.0 x10^13 gc/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States

Has US IND	True
Recent Updates	FTD and ODD granted 12/2023; cohort 1 interim data expected 2H 2024