RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa

NCTID	NCT02556736 (View at clinicaltrials.gov)
Description	Currently enrolling a total of 12 patients for Phase 2a of the study: 6 patients must have VA of no-better-than hand motion in the study eye, and 6 patients must have VA in the study eye to range from no-worse-than count fingers to 20/200 vision. (Show More)
Development Status	Active
Indication	Advanced Retinitis Pigmentosa
Disease Ontology Term	DOID:10584
Compound Name	RST-001
Compound Alias	AGN-151597
Sponsor	AbbVie
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	14
Results Posted	View Results

Target Gene/Variant	ChR2
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	3 undisclosed dose levels
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States