RARE Daily

Ocugen and CanSino Biologics Enter Gene Therapy Strategic Partnership

September 30, 2019

Rare Daily Staff

Ocugen said it has entered into a strategic partnership with CanSino Biologics to develop and manufacture Ocugen’s gene therapy pipeline product candidates for inherited retinal diseases.

Ocugen’s pipeline is currently in development with Schepens Eye Research Institute of Massachusetts Eye and Ear, an affiliate of Harvard Medical School.

Under the strategic collaboration, CanSino will provide all development and clinical supplies for the development of OCU400, Ocugen’s first gene therapy product candidate in its modifier gene therapy platform. CanSino maintains the option to support commercial manufacturing for Ocugen. The agreement also provides commercialization rights to CanSino in Greater China.

OCU400 is a novel gene therapy that Ocugen says has the potential to be effective in restoring retinal integrity and function across a range of genetically diverse inherited degenerative retinal diseases. It consists of a functional copy of the nuclear hormone receptor gene NR2E3. OCU400 is delivered to target cells in the retina using an adeno-associated viral (AAV) vector. Expression of NR2E3 within the retina may help reset retinal homeostasis, stabilizing cells, and potentially rescuing photoreceptors from degeneration.

“We believe our modifier gene therapy platform, and OCU400 as its first product candidate, has the potential to treat many inherited retinal diseases with one product,” said Shankar Musunuri, chairman, CEO and co-founder of Ocugen. “A reliable manufacturing partnership is critical for gene therapy clinical trials and commercialization. Partnership with CanSino Bio, with their state-of-the-art facilities and world class team, provides us a clear path to advance our development and manufacturing processes to reach the clinic.”

OCU400 has received two Orphan Drug designations from the U.S. Food and Drug Administration. The first, for the treatment of NR3E3 mutation-associated retinal degeneration and, most recently, for the treatment of CEP290 mutation-associated retinal disease.

Photo: Shankar Musunuri, chairman, CEO and co-founder of Ocugen

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