Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

NCTID	NCT00749957 (View at clinicaltrials.gov)
Description	The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene. Funding Source - FDA OOPD (Show More)
Development Status	Inactive
Indication	Leber Congenital Amaurosis-Type 2
Disease Ontology Term	DOID:0110016
Compound Name	RAAV2-CB-hRPE65
Sponsor	Beacon Therapeutics
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	12
Results Posted	View Results

Target Gene/Variant	RPE65
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	1.8E11 vg/eye
Dose 2	6E11 vg/eye
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Company discontinued development due to competing therapies for the same indication