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Research to Treat Neurodegenerative Diseases Advances: URMC Start-up Acquired

Wednesday, November 18, 2020

Oscine Therapeutics -- a biotechnology company that is developing cell-based therapies for neurological disorders based on discoveries made at the University of Rochester Medical Center (URMC) -- has been acquired by Sana Biotechnology for undisclosed terms.

The research behind Oscine is based on decades of work in the lab of Steve Goldman, M.D., Ph.D., professor of Neurology and Neuroscience and co-director of the URMC Center for Translational Neuromedicine. Goldman's research has focused on understanding the basic biology and molecular function of the glial support cells in the central nervous system, devising new techniques to precisely manipulate and sort these cells, and studying how cell replacement could impact the course of neurological diseases. Goldman, who was Oscine's president and scientific founder, joins Sana as senior vice-president and head of Central Nervous System Therapy. He will also remain on the URMC faculty.

Sana Biotechnology, which has operations in Washington, Massachusetts, and California, was created in 2018 with a focus on developing and delivering engineered cells as medicines for patients. The company is led by a team of biotechnology industry veterans and supported by more than $700 million in investment. Last year, the company invested in Oscine's R&D in neurological disorders, in what remains the University of Rochester's largest-ever commercial spin-off.

The exclusive licenses for the portfolio of technologies and equity stake that the University of Rochester held with Oscine have been acquired by Sana. The University and Goldman may continue to receive significant licensing, milestone, and royalty payments from Sana going forward.

"The University of Rochester has been working closely with Dr. Goldman's lab and the Oscine team from its inception," said Scott Catlin, director of UR Ventures, the University's technology transfer office. "We are thrilled with the company's impressive progress and its acquisition by Sana and look forward to continue supporting the commercialization of Dr. Goldman's technologies."

Goldman's research focuses on support cells in the brain called glia. In many neurological diseases -- such as multiple sclerosis, Huntington's, and neuropsychiatric disorders -- these cells either disappear or malfunction. This ultimately leads to the motor, cognitive, and behavioral symptoms of these disorders. Goldman's lab has shown that replacing these sick cells with healthy ones can slow and even reverse disease progression in animal models of these diseases.

The Center for Translational Neuromedicine maintains labs in Rochester and at the University of Copenhagen in Denmark. Goldman's research for cell-based therapies has received relevant support from the National Institute of Neurological Disorders and Stroke, the National Institute of Mental Health, the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation, the Lundbeck Foundation, the Novo Nordisk Foundation, CHDI, and NYSTEM.

Animal Study Shows Human Brain Cells Repair Damage in Multiple Sclerosis

Tuesday, May 19, 2020

A new study shows that when specific human brain cells are transplanted into animal models of multiple sclerosis and other white matter diseases, the cells repair damage and restore function. The study provides one of the final pieces of scientific evidence necessary to advance this treatment strategy to clinical trials.

"These findings demonstrate that through the transplantation of human glial cells, we can effectively achieve remyelination in the adult brain, " Steve Goldman, M.D., Ph.D., professor of Neurology and Neuroscience at the University of Rochester Medical Center (URMC), co-director of the Center for Translational Neuromedicine, and lead author of the study. "These findings have significant therapeutics implications and represent a proof-of-concept for future clinical trials for multiple sclerosis and potential other neurodegenerative diseases."

The findings, which appear in the journal Cell Reports, are the culmination of more than 15 years of research at URMC understanding support cells found in the brain called glia, how the cells develop and function, and their role in neurological disorders.

Goldman's lab has developed techniques to manipulate the chemical signaling of embryonic and induced pluripotent stem cells to create glia. A subtype of these, called glial progenitor cells, gives rise to the brain's main support cells, astrocytes and oligodendrocytes, which play important roles in the health and signaling function of nerve cells.

Read More: Animal Study Shows Human Brain Cells Repair Damage in Multiple Sclerosis