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
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)
THE STUDY INVOLVES:
- Screening before the treatment
- A single injection of the genetic therapy to the retina at the back of the eye
- Follow up for 1 year
LOCATIONS AND CONTACTS:
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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