第3期OCU400 liMeliGhT临床试验目前正在进行,目标是在2026年上半年提交BLA。 https://www.globenewswire.com/news-release/2025/01/13/3008252/0/en/Ocugen-Inc-Announces-Positive-2-Year-Data-Across-Multiple-Mutations-from-Phase-1-2-Clinical-Trial-of-OCU400-A-Novel-Modifier-Gene-Therapy-for-Retinitis-Pi...
We report the safety and efficacy of OCU400 gene modifier therapy from the phase 1/2 clinical trial for NR2E3 and RHO associated RP (NCT05203939). Methods : The multicenter phase 1/2 open-label trial was a 3+3 dose escalation and dose expansion study. Participants included adults with ...
OCU400已经获得孤儿药的称号,并且在FDA对3期研究设计的关键方面达成一致后,Ocugen有信心推进OCU400的产品开发和许可【8】。 目前,OCU400的研究和开发正在积极推进中,其临床前研究成果曾在Nature Gene Therapy杂志上发表【2】。此外,全球有多家公司正在进行针对遗传性视网膜疾病的基因疗法的临床研究,国内也有企业在眼科A...
OCU400, as an Advanced Therapy Medicinal Product (ATMP) by the European Medicines Agency’s Committee for Advanced Therapies. OCU400 is the first gene therapy to reach Phase 3 trials for retinitis pigmentosa (RP), a disease affecting approximately...
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 (AAV) vector. ...
s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious dise...
s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and w...