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)
Indication	Retinitis Pigmentosa
Compound Name	RST-001 (AGN-151597)
Sponsor	AbbVie
Funder Type	Industry
Status	Completed
Enrollment Count	14

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
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

Has US IND
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