Posted by Michael Wonder on 04 Mar 2018
MeiraGTx receives EMA PRIME designation for achromatopsia gene therapy candidate
2 March 2018 - MeiraGTx announced today the EMA has granted Priority Medicines (PRIME) designation to MeiraGTx’s gene therapy product candidate A002 for the treatment of patients with achromatopsia (ACHM) due to mutations in the CNGB3 gene.
Achromatopsia is a chronically debilitating inherited eye disorder that severely limits a person’s sight. Patients are legally blind from birth with very poor visual acuity as well as extreme light sensitivity, involuntary eye movement, and complete color blindness.
The PRIME application was based on non-clinical in vivo data and early clinical safety data from MeiraGTx’s ongoing Phase I/II dose escalation study.
