Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

NCTID	NCT06492850 (View at clinicaltrials.gov)
Description	The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase). (Show More)
Development Status	Active
Indication	X-Linked Retinitis Pigmentosa (XLRP)
Disease Ontology Term	DOID:0110414
Compound Name	FT-002
Compound Description	AAV5-GRK1-hRPGRORF15
Sponsor	Frontera Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	32
Results Posted	Not Available

Target Gene/Variant	RPGRORF15
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intraocular
Drug Product Type	Viral vector
Target Tissue/Cell	Photoreceptors
Delivery System	Viral transduction
Vector Type	AAV5
Editor Type	none
Dose 1	5E10 vg/eye
Dose 2	10E10 vg/eye
Dose 3	20E10 vg/eye
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	FDA clears IND for Phase 2 clinical trials 9/23/24