Gene Therapy for Achromatopsia (CNGA3)

An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Children and Adults With Achromatopsia Owing to Defects in CNGA3

A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia

CNGA3 retinal gene therapy for patients with achromatopsia

Inclusion Criteria:

• Are aged years or over
• Have achromatopsia confirmed by a retinal specialist investigator

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 investigator considers makes them inappropriate for entry into the trial