Gene Therapy for Achromatopsia (CNGB3)

An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3

A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia

CNGB3 retinal gene therapy for patients with achromatopsia

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