OCU410ST

OCU410ST is an investigational modifier gene therapy developed by Ocugen for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). It utilizes an adeno-associated virus serotype 5 (AAV5) vector to deliver the RAR Related Orphan Receptor A (RORA) gene via subretinal injection. The nuclear hormone receptor RORA regulates multiple pathophysiological pathways implicated in these retinal diseases, such as lipofuscin formation, oxidative stress, complement activation, inflammation, and cell survival networks. OCU410ST has received orphan drug designation from both the FDA and EMA for these indications. Clinical trials have shown a favorable safety profile and early evidence of efficacy in slowing lesion growth and improving visual function in Stargardt disease[1][2][4][5][6][8].

Other names

AAV5-hRORAAAV-5-hRORAAAV 5-hRORA