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/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations. (Show More)
Development Status	Active
Indication	X-Linked Retinitis Pigmentosa
Disease Ontology Term	DOID:0110414
Compound Name	AGTC-501
Compound Alias	Laruparetigene zovaparvovec
Compound Description	rAAV2tYF-GRK1-RPGR
Sponsor	Beacon Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	75
Results Posted	Not Available

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	none
Dose 1	6.8E11 vg/eye
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	21
Locations	United States,United Kingdom,Australia

Has US IND	True
FDA Designations	Fast Track, Regenerative Medicine Advanced Therapy
Recent Updates	First patient dosed in registrational trial 6/12/24, enrollment ongoing