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)
Development Status 🔄	Inactive
Indication	Achromatopsia
Disease Ontology Term	DOID:0110008
Drug Product Name	AAV-CNGB3
Drug Product Alias	Entacingene turiparvovec
Drug Product Description	AAV8-hCARp.hCNGB3
Sponsor	MeiraGTx UK II Ltd
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	23 (ACTUAL)
Results Posted	View Results

Target Gene/Variant	CNGB3
Regulatory Element	ARR3 (promoter)
Therapeutic Modality	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
Gene Delivery System Type	Viral transduction
Vector Type 🔄	Adeno-associated virus
Viral Vector Subtype	AAV2/8
Dose 1	0.1E12 vg/mL; 0.5 mL (n=3)
Dose 2	0.3E12 vg/mL; 0.5mL (n=12)
Dose 3	0.6E12 vg/mL; 0.5mL (n=3)
Dose 4	1.0E12 vg/mL; 0.5mL (n=5)

Eligible Age	>=3 Years
Standard Ages	Child, Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * Are aged 3 years or older * Have achromatopsia confirmed by a retinal specialist (CI or PI) Exclusion Criteria: * Are females who are pregnant or breastfeeding * Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months * Have any other condition that the CI/PI considers makes them inappropriate for entry into the trial View Inclusion and Exclusion Criteria at ClinicalTrials.gov

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

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Strategic decision to not further develop AAV8-hCARp.hCNGB3 and AAV8-hG1.7p.coCNGA3 was announced June 2025. The decision was not due to safety concerns