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)
Indication	Retinitis Pigmentosa, X-Linked
Compound Name	FT-002 (AAV5-GRK1-hRPGRORF15)
Sponsor	Frontera Therapeutics
Funder Type	Industry
Status	Recruiting
Enrollment Count	32

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
Dose 1	5 x 10^10 vg/eye
Dose 2	10 x 10^10 vg/eye
Dose 3	20 x 10^10 vg/eye
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
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