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Ocugen announces FDA approval for enrollment of pediatric patients in ongoing OCU400 Phase 1/2 clinical trial


The Phase 1/2 Clinical Trial is for the treatment of retinitis pigmentosa and Leber congenital amaurosis.

A doctor holding a clipboard full of medical data (Image Credit: AdobeStock/toeytoey)

(Image Credit: AdobeStock/toeytoey)

Ocugen Inc. announced 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,” Arun Upadhyay, PhD, Ocugen’s chief scientific officer, said in a news release. “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 retinitis pigmentosa (RPPand Leber congenital amaurosis (LCA), it is very important for us to cover this age group in our clinical trials.”

According to the company, 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.

The company also noted 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.

According to Ocugen, it is the only company with a gene-agnostic modifier platform that aims to alter this single-gene therapy paradigm through the introduction of a functional gene to modify the expression of multiple genes and gene-networks.

“We believe that patient prevalence in the United States alone would provide significant long-term value, with RP and LCA affecting 110,000 and 15,000 people, respectively,” the company said in its news release.

OCU400 is the company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its diverse functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.

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