BS01 in Patients With Retinitis Pigmentosa

NCTID	NCT04278131 (View at clinicaltrials.gov)
Description	Non-randomized, open label, Phase 1/2 dose escalation study of BS01, a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP). (Show More)
Development Status	Active
Indication	Retinitis Pigmentosa
Disease Ontology Term	DOID:10584
Compound Name	BS01
Compound Description	AAV2-CAG-ChronosFP-WPRE
Sponsor	Bionic Sight LLC
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	20
Results Posted	Not Available

Target Gene/Variant	ChronosFP
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
Editor Type
Dose 1	Dose escalation with 4 levels, unknown concentrations
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Regenerative Medicine Advanced Therapy
Recent Updates	Gene therapy product is coupled with a neural circuit, retinal prosthesis