European Commission grants gene therapy candidate OCU400 orphan medicinal product designation for treatment ofinherited retinal diseases, retinitis pigmentosa and Leber Congenital amaurosis
OCU400 is a novel investigational gene therapy based on Ocugen’s modifier gene therapy platform. It consists of a functional copy of the nuclear hormone receptor gene NR2E3 and is delivered to target cells in the retina using an adeno-associated viral vector.
Source:
PharmaTimes