Ocugen, Inc., a biotechnology leader in gene therapies for blindness diseases, announced on June 16, 2025, that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug $(IND.AU)$ amendment, allowing the initiation of a Phase 2/3 pivotal confirmatory trial for their modifier gene therapy candidate, OCU410ST. This therapy is being developed to treat Stargardt disease and other ABCA4-associated retinopathies. The FDA previously granted OCU410ST both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation, highlighting the urgent need for treatment options for these conditions. This regulatory approval marks a significant step forward in Ocugen's efforts to provide innovative treatments for patients affected by these debilitating diseases.
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