Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

NCTID	NCT04671433 (View at clinicaltrials.gov)
Description	A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP) (Show More)
Development Status	Active
Indication	X-Linked Retinitis Pigmentosa
Disease Ontology Term	DOID:0110414
Compound Name	Botaretigene sparoparvovec
Compound Alias	proper name
Compound Description	AAV2/5.hRKp.RPGR
Sponsor	Janssen Research & Development, LLC
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	97
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	AAV2/5
Editor Type	none
Dose 1	2.0E10 vg/mL
Dose 2	2.0E11 vg/mL
Dose 3	2.0E12 vg/mL
Dose 4
Dose 5

No.of Trial Sites	27
Locations	Canada,Netherlands,Belgium,United States,Denmark,Italy,United Kingdom,Israel,France,Switzerland,Spain

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	Phase 3 dosing complete, potential BLA filing in 2025