Gene Therapy for Achromatopsia (CNGA3)

NCTID	NCT03758404 (View at clinicaltrials.gov)
Description	A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia (Show More)
Indication	Achromatopsia
Compound Name	AAV-CNGA3 (AAV8-hG1.7p.co.CNGA3)
Sponsor	MeiraGTx UK II Ltd
Funder Type	Industry
Status	Completed
Enrollment Count	11

Target Gene/Variant	CNGA3
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	Undisclosed low dose
Dose 2	Undisclosed intermediate dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

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

Has US IND	True
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