Gene Therapy for Achromatopsia (CNGB3)

NCTID	NCT03001310 (View at clinicaltrials.gov)
Description	A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia (Show More)
Indication	Achromatopsia
Compound Name	Entacingene turiparvovec (AAV8-hCARp.hCNGB3)
Sponsor	MeiraGTx UK II Ltd
Funder Type	Industry
Status	Completed
Enrollment Count	23

Target Gene/Variant	CNGB3
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	Cone cells
Delivery System	Viral transduction
Vector Type	AAV2/8
Editor Type
Dose 1	0.1 × 10^12 vg/mL; 0.5 mL
Dose 2	0.3 × 10^12 vg/mL; 0.5mL
Dose 3	0.6 × 10^12 vg/mL; 0.5mL
Dose 4	1.0 × 10^12 vg/mL; 0.5mL
Dose 5

No.of Trial Sites	2
Locations	United States,United Kingdom

Has US IND	True
Recent Updates