Press release
Ocugen Announces FDA Approval for Enrollment of Pediatric Patients in Ongoing OCU400 Phase 1/2 Clinical Trial for the Treatment of Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA)
U.S. Food & Drug Administration (FDA) approves enrolling pediatric patients in the ongoing OCU400 Phase 1/2 trial who have: 1) RP associated with NR2E3 and
About this update from Ocugen, Inc.
[{"type":"text","content":"U.S. Food & Drug Administration (FDA) approves enrolling pediatric patients in the ongoing OCU400 Phase 1/2 trial who have: 1) RP associated with NR2E3 and RHO mutations and 2) LCA associated with CEP290 gene mutationsOcugen has completed enrollment of adult RP patients with NR2E3 and RHO mutations in the Phase 1/2 trial and expanded enrollment in LCA patients with CEP290 mutations MALVERN, Pa., March 27, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the FDA approved enrolling pediatric patients in the ongoing OCU400 Phase 1/2 trial. “This approval moves us one step closer in our efforts to bring OCU400, a novel gene-agnostic modifier gene therapy, to market as a potential life-changing treatment for children afflicted with inherited retinal diseases, such as RP and LCA,” noted Arun Upadhyay, PhD, Ocugen’s Chief Scientific Officer. “This approval further demonstrates the consistent, positive, and timely progress we are making with the Phase 1/2 trial in adult patients. Since a significant number of individuals in the pediatric age group are diagnosed with RP and LCA, it is very important for us to cover this age group in our clinical trials.” Enrollment of adult RP patients in the Phase 1/2 trial is complete—per protocol—and enrollment continues among patients with LCA. The Company plans to initiate the Phase 3 trial near the end of 2023. Unlike single-gene replacement therapies, which only target one genetic mutation, Ocugen believes that its modifier gene therapy platform, through its use of Nuclear Hormone Receptors (NHRs), represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and potentially address complex diseases that are caused by imbalances in multiple gene-networks. While single-gene replacement therapies have shown tremendous promise in rare retinal diseases, they are highly specific and cannot improve a multitude of disease-causing genetic defects. For example, RP and LCA are associated with mutations in more than 100 and in more than 25 genes, respectively. Ocugen is the only company with a gene-agnostic modifier platform that aims to alter t...