Ocugen Announces OCU400—Modifier Gene Therapy—Phase 1/2 Data Presentation at Retinal Cell and Gene Therapy Innovation Summit 2024 - Seite 2
About RP
RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, RP is associated with mutations in more than 100
genes. There are no approved treatment options that slow or stop the progression of multiple forms of RP. Proposed treatments for RP include gene replacement therapy, retinal implant devices,
retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Current gene replacement therapies are promising but are limited to treating just a single mutation. In
addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect, which may still cause stress and toxic effects leading to retina
degeneration. Therefore, the development of gene-specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved. Thus, novel therapeutic approaches
targeting broader RP disease in a gene-agnostic manner offer greater hope for patients.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, and vaccines that improve health and offer hope for patients across
the globe. We are making an impact on patient’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 diseases to support public health and orthopedic
diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.
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