Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia
A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 (rAAV2tYF-PR1.7-hCNGB3) in Patients With Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene
Doctors at research centers in FL, MA, NY, OH, and OR seek pediatric patients with Achromatopsia due to specific genetic mutations in the CNGB3 gene. The drug uses a genetic editing tool, delivered only within the retina, designed to eliminate the harmful mutation in the CNGB3 gene. It uses a harmless virus that can selectively pass the corrected gene into the cells of the retina to enable cones to carry out phototransduction.
Treatment will involve a single injection surgery to the retina at the back of the eye, then follow up continues for 1 year.
- Be 6 years of age or older
- Meet genetic screening requirements specific to the study aim
- Have achromatopsia confirmed by a retinal specialist (CI or PI)
- Not be pregnant
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 has 6 U.S. locations:
VitreoRetinal Associates in Gainesville, FL, Map.
Bascom Palmer Eye Institute in Miami, FL, Map.
Massachusetts Eye and Ear Infirmary in Boston, MA, Map.
Columbia College of Physicians and Surgeons in New York, NY, Map.
Duke Eye Center, Duke University Medical Center in Durham, NC. Map.
Cincinnati Eye Institute in Cincinnati, OH, Map.
Casey Eye Institute, Oregon Health and Sciences University in Portland, OR. Map.
Jill Dolgin, PharmD | 6178435781 | [email protected]
Applied Genetic Technologies Corp
National Eye Institute (NEI)
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