Business
Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative Disease
Third orphan drug designation for the same product, OCU400, is unique in Ophthalmology gene therapy and demonstrates its potential to treat many Inherited
About this update from Ocugen, Inc.
[{"type":"text","content":"Third orphan drug designation for the same product, OCU400, is unique in Ophthalmology gene therapy and demonstrates its potential to treat many Inherited Retinal Degenerative diseases (IRDs)\nMALVERN, Pa., July 27, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced the U.S. Food and Drug Administration (FDA) granted the third Orphan Drug Designation (ODD) for OCU400 in the treatment of RHO mutation-associated retinal degeneration. The RHO mutation is part of the Retinitis Pigmentosa (RP) group of rare, genetic disorders that involve a breakdown and loss of cells in the retina and can lead to visual impairment and blindness. This is one of the larger mutations within the RP class, representing about 12% of RP patients in the US.\n A novel gene therapy product candidate, OCU400 has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor (NHR) gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis and potentially offer longer benefit, stabilizing cells and rescuing photoreceptor degeneration and vision loss. Adding to ODDs for OCU400 for NR2E3 and CEP290 mutation-associated retinal degeneration, RHO gene mutation-associated retinal degeneration further supports Ocugen’s breakthrough modifier gene therapy platform’s potential to treat multiple blindness diseases with a single product. RP is a group of heterogenic inherited retinal diseases associated with over 150 gene mutations, affecting over 1.5 million individuals worldwide. In addition, ~40% of RP patients cannot be genetically diagnosed, confounding the ability to develop personalized RP therapies. Traditional gene therapy or gene editing approaches may require more than 150 products to rescue these patients from vision loss. OCU400, a single product candidate, has potential to address broad-spectrum RP. “OCU400, comprising the nuclear hormone receptor gene NR2E3, has the potential to help modulate numerous biological pathways that fun...