Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy

NCTID	NCT05858983 (View at clinicaltrials.gov)
Description	The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy. (Show More)
Development Status	Active
Indication	Biallelic RPE65 Mutation-associated Retinal Dystrophy
Disease Ontology Term	DOID:0110016; DOID:0110353
Compound Name	FT-001
Compound Description	AAV2-hRPE65
Sponsor	Frontera Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	9
Results Posted	Not Available

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	none
Dose 1	1.5E10 vg/eye
Dose 2	7.5E10 vg/eye
Dose 3	15E10 vg/eye
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China