Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)

NCTID	NCT02416622 (View at clinicaltrials.gov)
Description	This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with X-linked retinoschisis. Up to 27 participants will be enrolled and 3 dose levels will be evaluated in a dose escalation format. (Show More)
Development Status	Active
Indication	X-linked Retinoschisis
Disease Ontology Term	DOID:0060763
Compound Name	RAAV2tYF-CB-hRS1
Sponsor	Beacon Therapeutics
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	27
Results Posted	View Results

Target Gene/Variant	RS1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravitreal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2tYF
Editor Type
Dose 1	1E11 vg/eye
Dose 2	3E11 vg/eye
Dose 3	6E11 vg/eye
Dose 4
Dose 5

No.of Trial Sites	9
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	The product was well tolerated, but no signs of clinical activity were observed at six months post treatment, development discontinued in 2018; product was transferred to TeamedOn