Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

NCTID	NCT03326336 (View at clinicaltrials.gov)
Description	The objective of this study is to evaluate the safety and tolerability of escalating doses of a gene therapy called GS030-DP (injected study treatment) administered via a single intravitreal injection and repeated light stimulation using a medical device called GS030-MD (stimulating glasses) in subjects with documented diagnosis of non-syndromic Retinitis Pigmentosa (Show More)
Indication	Retinitis Pigmentosa, Non-Syndromic
Compound Name	GS030 (rAAV2.7m8-CAG-ChrimsonR-tdTomato)
Sponsor	GenSight Biologics
Funder Type	Industry
Status	Recruiting
Enrollment Count	15

Target Gene/Variant	ChR-tdT
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravitreal
Drug Product Type	Viral vector
Target Tissue/Cell	Retinal ganglion
Delivery System	Viral transduction
Vector Type	AAV2.7m8
Editor Type
Dose 1	5 x 10^10 vg/eye
Dose 2	1.5 x 10^11 vg/eye
Dose 3	5 x 10^11 vg/eye
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States,United Kingdom,France

Has US IND	True
Recent Updates