European Commission Grants Ocugen Orphan Medicinal Product Designation for Gene Therapy Product Candidate, OCU400, For the Treatment of Both Retinitis Pigmentosa and Leber Congenital Amaurosis - GlobeNewswire

23, 2021 (GLOBE NEWSWIRE) -- Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19, today announced that on the recommendation of the European Medicines Agency (EMA), the European Commission has granted orphan medicinal product designation for OCU400 (AAV5-hNR2E3), for the treatment of both retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).

“We believe the granting of this designation by the European Commission validates the potential of our modifier gene therapy platform to treat many inherited retinal diseases (IRDs).

IRDs associated with RP and LCA diseases are caused by mutations in over 175 genes, and it is impractical to develop therapies that are specific to each gene.

OCU400 has the remarkable potential to address a significant number of patients globally who are in desperate need of rescue from these blindness diseases and we are working diligently to move this program to clinic,” said Dr.

Nuclear Hormone Receptors such as NR2E3 are important modulators of retinal development and function acting as “master genes” in the retina.

As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration.

Preclinical results published in Nature Gene Therapy demonstrate the potency of modifier gene therapy to elicit broad-spectrum therapeutic benefits in early and advanced stages of RP including vision rescue in early and advanced stages of the disease.

OCU400 is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse IRDs.

OCU400 is the first program that Ocugen is advancing based on its breakthrough modifier gene therapy platform developed by Dr.

Consisting of a functional copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to target cells in the retina using an AAV vector.

As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, stabilizing cells and potentially rescuing photoreceptors from degeneration.

is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19.

Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy.