Movers and SHAKERS
Gene Therapy Trial Reaches Progress Milestone
Ocugen Inc is a clinical stage biopharmaceutical company. It is focused on discovering, developing and commercializing a pipeline of innovative therapies that address rare and underserved eye diseases. Ocugen offers a diversified ophthalmology portfolio that includes novel gene therapies, biologics, and small molecules and targets a broad range of high-need retinal and ocular surface diseases.
Robert LeBoyer, Vice President, Research Analyst, Life Sciences, Noble Capital Markets, Inc.
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Ocugen Meets A Pipeline Milestone Ocugen announced treatment of the first patient in its Phase 1/2 trial for OCU400, the first product from its gene therapy platform to reach clinical trials. OCU400 delivers genes to correct the genetic mutations that lead to retinitis pigmentosa, an ophthalmic condition that leads to vision loss and blindness. Patient screening began in 1Q22, meeting our expected timeframe for the trial. We see this milestone as good news for the product and the development pipeline.
Phase 1/2 Trial Is Designed To Show Safety and Efficacy. The OCU400 Phase 1/2 trial is designed with three cohorts at increasing dose levels. After establishing basic safety at the low dose, two additional cohorts will receive higher doses. The treatment delivers functional copies of the NR2E3 and RHO genes, regulatory genes that affect expression of other genes in the disease pathway. Preliminary ...
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