Achromatopsia - Healing Genes


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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 With Achromatopsia Owing to Defects in CNGA3

Phase 1/2


Doctors at the Kellogg Eye Center in Michigan are seeking pediatric patients with Achromatopsia due to specific genetic mutations in the CNGA3 gene. The drug uses a genetic editing tool, delivered only within the retina, designed to eliminate the harmful mutation in the CNGA3 gene. It uses a harmless virus that can selectively pass the corrected gene into the cells of the retina .

Treatment will involve a single injection surgery to the retina at the back of the eye, then follow up for 6 months


  • Be 3 to 15 years of age
  • Meet genetic screening requirements specific to the study aim
  • Have achromatopsia confirmed by a retinal specialist (CI or PI)


  1. Screening before the treatment
  2. A single injection of the genetic therapy to the retina at the back of the eye
  3. Follow up for 1 year


The study location is at the Kellogg Eye Center in Ann Arbor, MI. Map.

Contact: MeiraGTx UK II Ltd (International)  |  +44 (0)02 3866 4320 (international) |  [email protected]



MeiraGTx UK II Ltd


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