A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

NCTID	NCT04850118 (View at clinicaltrials.gov)
Description	This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations. (Show More)
Indication	Retinitis Pigmentosa, X-Linked
Compound Name	Laruparetigene zovaparvovec (AGTC-501)
Sponsor	Beacon Therapeutics
Funder Type	Industry
Status	Recruiting
Enrollment Count	75

Target Gene/Variant	RPGR
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	AAV2tYF
Editor Type
Dose 1	6.8 x 10^11 vg/eye
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	13
Locations	United States,United Kingdom,Australia

Has US IND	True
Recent Updates	First patient dosed in registrational trial 6/12/24