Promising ROd-cone DYstrophy Gene TherapY

NCTID	NCT05748873 (View at clinicaltrials.gov)
Description	This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm, controlled, double-masked, randomized extension phase (Step 2), in subjects with advanced RCD due to a mutation in the RHO, PDE6A, or PDE6B gene. (Show More)
Indication	Retinitis Pigmentosa
Compound Name	SPVN06
Sponsor	SparingVision
Funder Type	Industry
Status	Recruiting
Enrollment Count	33

Target Gene/Variant	NXNL1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type
Dose 1	Undisclosed low dose
Dose 2	Undisclosed medium dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States,France

Has US IND	True
Recent Updates	Enrollment of highest dose cohort is underway