Ocugen Expands Focus on Inherited Retinal Disorders by Licensing Novel Modifier Gene Therapy Platform

Source: Ocugen

Wednesday, December 20, 2017 | Retina


Ocugen announced a strategic expansion to its leading ophthalmology product development pipeline that enhances the company's focus on inherited retinal disorders (IRDs). Ocugen has obtained an exclusive worldwide license to develop and commercialize future ophthalmology products based on groundbreaking technology from the Schepens Eye Research Institute of Massachusetts Eye and Ear, according to a company news release. The novel modifier gene therapy platform has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse IRDs and other degenerative retinal diseases, leading to multiple potential product opportunities.

The technology was developed by Neena Haider, PhD, Associate Professor of Ophthalmology at Harvard Medical School and Associate Scientist at Mass. Eye and Ear. It encompasses the targeted delivery and expression of certain nuclear hormone receptors (NHRs) that are expressed naturally in retinal tissue.  Therapeutic NHRs were identified by their ability to modify disease progression through regulation of key gene networks that can restore retinal homeostasis and rescue the defects caused by inherited gene mutations. This use of genetic modifiers represents a powerful and remarkably broadened means of treating a variety of retinal degenerative diseases, as compared to single-gene replacement therapy.

"Ocugen is committed to advancing innovative therapies for ocular conditions where there is significant unmet need," Shankar Munsunuri, PhD, Chairman, CEO and Co-Founder of Ocugen, said in the news release. "Therefore, we are very pleased to augment our pipeline with this cutting edge technology and to work with esteemed ophthalmic scientists at Mass. Eye and Ear. Single-gene replacements have shown tremendous promise in rare retinal diseases, but they are highly specific and cannot cover the multitude of disease-causing genetic defects. This novel approach has shown potential to rescue many of these genetic defects, and may lead to vision-sparing therapies for rare/orphan IRDs such as S-cone syndrome, Goldman-Favre syndrome, and retinitis pigmentosa, as well as other forms of retinal and macular degeneration, adding significant long-term value for Ocugen."

"Nuclear hormone receptors play a vital role in regulating retinal cell development, maturation, metabolism, visual cycle function, and survival," said Dr. Haider.  "We've shown in multiple animal models that modifier gene therapy can stabilize retinal cells by regulating the expression of key genes required for proper retinal function, potentially halting disease progression and improving outcomes for people with blinding retinal diseases. We are pleased to partner with a leading ophthalmology company such as Ocugen to continue this work and advance a lead candidate for preclinical and clinical development."


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