One familys' Christmas wishes came true after their two sons who suffer from a rare inherited eye disorder were given the green light to receive an experimental
Variant, a company dedicated to developing innovative treatments for inherited eye orphan diseases, announces the decision of the European Commission to grant Orphan Drug Designation (ODD) for VAR002
European Commission Grants Ocugen Orphan Medicinal Product Designation for Gene Therapy Product Candidate, OCU400, For the Treatment of Both Retinitis Pigmentosa and Leber Congenital Amaurosis
NR2E3), for the treatment of both retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
The prevalence of RP in Europe is estimated at approximately 165,000 patients and the prevalence of LCA in Europe is estimated at approximately 40,000 patients. Globally, the number of people suffering from RP and LCA is estimated to be around 2.0 million and 0.2 million, respectively. We believe the granting of this designation by the European Commission validates the potential of our modifier gene therapy platform to treat many inherited retinal diseases, Chairman of the Board, Chief Executive Officer, and Co-founder of Ocugen.