Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

NCTID	NCT03920007 (View at clinicaltrials.gov)
Description	Primary Objective: To evaluate the safety and tolerability of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (LCA) caused by autosomal recessive guanylate cyclase 2D (GUCY2D) mutations (GUCY2D-LCA). Secondary Objective: To evaluate the efficacy of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with GUCY2D-LCA. (Show More)
Indication	Leber Congenital Amaurosis - Type 1
Compound Name	ATSN-101 (AAV5-hGRK1-hGUCY2D)
Sponsor	Atsena Therapeutics Inc.
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	15

Target Gene/Variant	GUCY2D
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	AAV2/5
Editor Type
Dose 1	1.0 x 10^10 vg/eye (300ul)
Dose 2	3.0 x 10^10 vg/eye (300ul)
Dose 3	1.0 x 10^11 vg/eye (300ul); expansion dose
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
Recent Updates	FDA granted Rare Pediatric Disease Designation, ODD, RMAT