Wednesday, November 02, 2022
Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product, Covaxin, is a killed-virus vaccine for COVID-19 in-licensed from Bharat Biotech (India). The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.
Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
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Gene Therapy In The Spotlight. Ocugen held an R&D Day to highlight its programs in gene therapy and COVID-19. The presentations discussed the novel mechanisms behind its gene therapy products and the nature of the ophthalmic disease indications in development. While the company has been best known for COVAXIN and COVID-19, we have always seen the gene therapy indications as highly promising and underappreciated.
Modifier Gene Therapy Platform. The first presentations discussed the company’s novel approach to gene therapy. In contrast to other approaches that seek to transfer a functional copy of a gene to replace a gene that has mutated or become dysfunctional, Ocugen’s Modifier Gene Therapy targets a “master control gene” that controls the expression of multiple gene pathways, allowing products to be developed for diseases caused by downstream gene pathways and combinations of genetic mutations.
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