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)
Development Status	Active
Indication	Non-syndromic Retinitis Pigmentosa
Disease Ontology Term	DOID:10584
Compound Name	GS030
Compound Description	rAAV2.7m8-CAG-ChrimsonR-tdTomato
Sponsor	GenSight Biologics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	15
Results Posted	Not Available

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	none
Dose 1	5E10 vg/eye
Dose 2	1.5E11 vg/eye
Dose 3	5E11 vg/eye
Dose 4
Dose 5

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