URMC biotech start-up receives funding to develop cell-based therapies for neurological disorders

Reviewed by James Ives, M.Psych. (Editor)Jun 11 2019

Oscine Therapeutics, a new biotechnology company based on discoveries made and developed at the University of Rochester Medical Center (URMC) has received a significant multi-year investment to support both research and development of cell-based therapies for neurological disorders. The funding represents the largest-ever investment in a URMC start-up company.

The new venture is based on decades of research in the lab of Steve Goldman, M.D., Ph.D., co-director of the URMC Center for Translational Neuromedicine. Goldman's work has focused on understanding the basic biology and molecular function of 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 has developed techniques to manipulate the chemical signaling of embryonic and induced pluripotent stem cells to create the brain's support cells, called 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.

"Neurological disorders are complex diseases, but in many instances it appears that faulty support cells of the brain are driving the disease process," said Goldman, the URMC Distinguished Professor of Neuroscience and Neurology. "These diseases represent promising targets for cell-replacement therapies because we know a great deal about the role these cells play, how to create them, and how to get them to the areas of the brain where they are needed."

The investment in Oscine is being made by Sana Biotechnology, a new company focused on creating and delivering engineered cells as medicines for patients. The company is led by a team of biotechnology industry veterans with extensive experience in cell therapy, gene therapy, and gene editing. The company is backed by visionary investors including Arch Venture Partners, Flagship Pioneering, and F-Prime Capital Partners.

In many neurological diseases, such as multiple sclerosis and Huntington's, glial cells have impaired development and function, or are simply lost during the course of the disease. This results in the disruption of communication between nerve cells, leading to the motor, cognitive, and behavioral symptoms of these disorders.

Goldman's lab has developed new methods to replace the sick glial cells found in these diseases with healthy ones. In research involving animal models of these diseases, this approach has slowed, and for some disorders even reversed, disease progression.

The new investment - the terms of which were not publicly disclosed - will support R&D by Oscine focused on bringing these cell-based therapies to the clinic. The research will be conducted at URMC under a sponsored research agreement and will support 21 full-time staff, with researchers in Rochester and additional staff in Seattle and New York City. The manufacturing of cells for clinical delivery will use protocols developed at Rochester.

"Cell-based therapies hold significant promise, and while progress has been made in areas such as cancer, there is a significant unmet need in diseases of the central nervous system," said Christina Trojel-Hansen, Ph.D., the CEO of Oscine, who co-founded the company along with Goldman and spearheaded its organization and fundraising. "The support from Sana will enable us to advance important research in this field and work with an established team that has experience in bringing cell-based therapies through clinical trials and into clinical practice. I am also deeply grateful for the team at the University of Rochester for their efforts to ensure that these important scientific discoveries can now advance toward a clinical application."

"Our bread and butter at ARCH is in starting companies with top researchers in world-class universities across the globe who are working on transformative discoveries, and pairing those seminal innovations with hard-charging entrepreneurs," said Paul S. Thurk, managing director with Arch Venture Partners. "We found that golden mix with Dr. Goldman and Dr. Trojel-Hansen at Oscine."

The University of Rochester and Cornell University have licensed intellectual property to Oscine, and both the University of Rochester and Goldman hold equity stakes in the company. Goldman also serves as Oscine's President. The negotiation of the license and sponsored research agreement terms was led by Matan Rapoport, Ph.D., M.B.A., senior licensing manager with UR Ventures, the University of Rochester's technology transfer office.

This investment underscores the cutting-edge nature of the neurological cell therapy research program at URMC. We are excited that the basic science discoveries made here at the Medical Center are the basis for innovative, first-in-class therapies that hold the potential to change the lives of people with these devastating neurodegenerative diseases."

Steve Dewhurst, Ph.D., vice dean for Research at the University of Rochester School of Medicine and Dentistry