MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) announced that the FDA recently granted Orphan Drug Designation for its OCU410ST (AAV5-hRORA), 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 (RP19), and Cone-rod dystrophy 3 (CORD3) 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.”
Orphan drug designation is granted by the FDA to certain products that show promise in the treatment, prevention, or diagnosis of rare and serious diseases affecting fewer than 200,000 people in the United States.
OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene and represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks.
The ABCA4 gene produces an ATP-binding cassette (ABC) superfamily transmembrane protein and expressed exclusively in retinal photoreceptors. It is involved in clearance of all-trans-retinal aldehyde, a byproduct of the retinoid cycle, from photoreceptor cells. Mutation in ABCA4 gene results in Stargardt disease, a rare genetically inherited disease that directly affects the retina of the eye, often resulting in the slow progression of vision loss in children. In addition, different ABCA4 alleles have been identified to cause other retinopathies such as CORD3 and RP19.
Ocugen plans to submit an IND in 2Q 2023 to initiate a Phase 1/2 clinical trial.
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