CNGB3 gene therapy AGTC

Unassigned

New Medicines

Achromatopsia due to mutations in the CNGB 3 gene

Information

Advanced therapy medicinal product (ATMP)
Applied Genetic Technologies Corporation
Applied Genetic Technologies Corporation

Development and Regulatory status

None
None
Phase II Clinical Trials
Yes
Yes

Category

An adeno-associated virus (AAV) gene therapy
This condition causes complete loss of cone function, while rod function is normal throughout the course of disease. Its prevalence is estimated to be about 1 per 30,000. Since cones are concentrated at the fovea then the macula and fovea are disproportionately affected [1].
Achromatopsia due to mutations in the CNGB 3 gene
Subretinal injection
Parenteral