Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)

NCTID	NCT02599922 (View at clinicaltrials.gov)
Description	This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy. (Show More)
Development Status	Inactive
Indication	Achromatopsia
Disease Ontology Term	DOID:0110008
Compound Name	AGTC-401
Compound Description	rAAV2tYF-PR1.7-hCNGB3
Sponsor	Beacon Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	32
Results Posted	Not Available

Target Gene/Variant	CNGB3
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	Cone cells
Delivery System	Viral transduction
Vector Type	AAV2tYF
Editor Type	none
Dose 1	2E11 vg/ml
Dose 2	4E10 vg/ml
Dose 3	1.2E11 vg/ml
Dose 4	3.6E11 vg/ml
Dose 5	1.1E12 vg/ml; Dose 6: 3.2E12

No.of Trial Sites	8
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Development discontinued by Sponsor