MeiraGTx Holdings announced a preclinical poster on safety and efficacy of the company’s gene therapy product candidate AAV-CNGA3 for the treatment of patients with achromatopsia (ACHM) due to mutations in the CNGA3 gene will be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2019 Annual Meeting in Vancouver, British Columbia.
Details of MeiraGTx’s poster are as follows:
Title: Development and efficacy assessment of AAV2/8-hG1.7p.coCNGA3, a CNGA3 gene therapy vector
Abstract Number: 3426 – A0197
Session Name: Gene therapy for ocular disorders
Date: Tuesday, April 30, 2019, 11:45am – 1:30pm PT (2:45pm – 4:30pm ET)
Location: West Exhibition Hall
Data Summary: AAV-CNGA3 utilizes a proprietary engineered promoter (hG1.7p) that was associated with strong, pan-cone gene expression in induced pluripotent stem cell (iPSC)-derived retinal organoids driving a codon-optimized CNGA3 cDNA. In preclinical models, treatment with AAV-CNGA3 resulted in long-term visual improvements and cone photoreceptor survival at titers planned for use in a phase 1/2 clinical trial of AAV-CNGA3.
AAV-CNGA3 is a gene therapy treatment being developed as a potential treatment for patients with achromatopsia (ACHM) due to mutations in the CNGA3 gene. Delivered via subretinal injection to a patient’s cone receptors at the back of the eye, AAV-CNGA3 is designed to restore cone function by delivering a normal copy of the CNGA3 gene. AAV-CNGA3 has been granted orphan drug designation and rare pediatric disease designation by the United States Food and Drug Administration (FDA), as well as orphan drug designation by the European Medicines Agency (EMA).
AAV-CNGA3 is being developed in collaboration with Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson.
In 2019, MeiraGTx and Janssen expect to initiate a phase 1/2 dose escalation study of AAV-CNGA3 in children with ACHM due to mutations in the CNGA3 gene.