A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112

NCTID	NCT03116113 (View at clinicaltrials.gov)
Description	The objective of the study is to evaluate the safety, tolerability and efficacy of a single sub-retinal injection of BIIB112 in participants with X-linked retinitis pigmentosa (XLRP). (Show More)
Development Status	Inactive
Indication	X-Linked Retinitis Pigmentosa (XLRP)
Disease Ontology Term	DOID:0110414
Compound Name	BIIB112
Compound Alias	Cotoretigene toliparvovec
Compound Description	AAV8.coRPGR
Sponsor	Biogen
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	50 (ACTUAL)
Results Posted	View Results

Target Gene/Variant	RPGR
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type
Dose 1	5E9 vg/eye (n=3)
Dose 2	1E10 vg/eye (n=3)
Dose 3	5E10 vg/eye (n=14)
Dose 4	1E11 vg/eye (n=3)
Dose 5	2.5E11 vg/eye (n=15); Dose 6: 5E11 vg/eye (n=3)

No.of Trial Sites	8
Locations	United States,United Kingdom

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	Phase 2/3 study did not meet its primary endpoint, Company suspended development in 2021