Phase 1/2 Clinical Trial of AAV-CNGB3
MeiraGTx is currently conducting an open-label, dose-escalation Phase 1/2 clinical trial of AAV-CNGB3 in adult and pediatric patients with achromatopsia (ACHM) due to mutations in the CNGB3 gene. The primary endpoint of this clinical trial is the safety of delivering AAV-CNGB3 through subretinal administration. Secondary endpoints include the outcomes of a range of functional tests and detailed structural analysis of the retina, including structural analysis of individual photoreceptors.
MeiraGTx is also conducting an ongoing natural history study following over 90 patients with ACHM for up to five years to collect data on the structure and function of their eye.