Text size A A

Achromatopsia Overview

Achromatopsia is a rare inherited retinal disorder affecting a person’s cone function. It is characterized by extreme light sensitivity (day blindness), partial or total colorblindness, decreased visual acuity and nystagmus. There are currently no FDA-approved treatments for this condition, which is caused by mutations in one of several genes. The two most common mutations occur in either the CNGB3 or CNGA3 genes.

Download Patient Brochure

AGTC’s Clarity Trials for Achromatopsia

AGTC is currently conducting two separate Phase 1 / 2 clinical trials to evaluate the safety and efficacy of AAV gene therapy product candidates in patients with achromatopsia caused by CNGB3 or CNGA3 mutations. These product candidates are administered to one eye by subretinal injection.The primary study endpoint for these studies is safety and the secondary study endpoint is efficacy.

More detailed information on these studies and the full eligibility criteria are on our study-specific pages on clinicaltrials.gov:

  • ACHMB3 Achromatopsia Phase 1/2 Clinical Trial
    (ClinicalTrials.gov Identifier: NCT02599922)
  • ACHMA3 Achromatopsia Phase 1/2 Clinical Trial
    (ClinicalTrials.gov Identifier: NCT02935517)

We are happy to discuss our trials with you or send more information for you to share with your patients.

Be a part of finding a treatment for achromatopsia Join Us