Ocugen’s Gene Therapy Candidates for GA and Stargardt Gain Advanced Therapy Status in Europe

Ocugen announced March 3 that European regulators had granted advanced therapy medicinal product status to OCU410 and OCU410ST, the company’s subretinal gene therapy candidates for geographic atrophy (GA) and Stargardt disease, respectively.

Both OCU410 and OCU410ST utilize an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. Ocugen’s modifier gene therapy approach relies on nuclear hormone receptors that regulate diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival networks.

Europe’s advanced therapy classification accelerates the regulatory review timeline and allows the developing company to interact with the European Medicines Agency more frequently for scientific advice and protocol assistance. OCU410ST has orphan drug status in the US and EU.

Ocugen, of Malvern, Pennsylvania, is also developing OCU400, a gene therapy candidate targeting retinitis pigmentosa.