Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCTID	NCT05445323 (View at clinicaltrials.gov)
Description	This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment. (Show More)
Development Status	Active
Indication	Friedreich Ataxia, Cardiomyopathy, Secondary
Disease Ontology Term	DOID:0111218
Compound Name	LX2006
Compound Description	AAVrh.10hFXN
Sponsor	Lexeo Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	8
Results Posted	Not Available

Target Gene/Variant	FXN
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	none
Dose 1	1.8E11 vg/kg
Dose 2	5.6E11 vg/kg
Dose 3	1.2E12 vg/kg
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
FDA Designations	Fast Track
Recent Updates	Program update expected mid 2025, potential to initiate registrational study by end of 2025/early 2026