Ocugen announced that the FDA has granted Orphan Drug Designation, ODD, for its OCU410ST, an adeno-associated virus serotype 5 capsid protein containing gene construct encoding human retinoic acid receptor-related orphan receptor alpha, for the "treatment of ABCA4-associated retinopathies" including Stargardt, Retinitis Pigmentosa 19, and Cone-rod dystrophy 3 diseases. "There are approximately 44,000 patients in the U.S. living with ABCA4-associated retinal diseases for whom no treatment options exist," said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. "This designation acknowledges the potential for OCU410ST to fulfill a significant unmet medical need and represents an important milestone in our effort to develop innovative treatments for inherited retinal diseases." Ocugen plans to submit an IND in 2Q 2023 to initiate a Phase 1/2 clinical trial.
Published first on TheFly
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