URMC Partners With Sana Biotechnology To Advance Cell-Based Therapies In Neurological Disorders

The University of Rochester Medical Center (URMC) and Sana Biotechnology have entered into an agreement which will allow Sana to expand its manufacturing footprint and create new job opportunities in Rochester. Sana, a company focused on creating and delivering engineered cells as medicines, also operates in Seattle, Cambridge, and South San Francisco. The company has programs in both in vivo and ex vivo cell engineering, targeting potential medicines for serious diseases such as cancer, type 1 diabetes, central nervous system diseases, heart disease, and various genetic disorders.

Sana has collaborated with URMC for many years, and the new agreement will enable Sana to build out its operations at URMC’s cell-based manufacturing facility to support research and development of investigational therapies for diseases such as Huntington’s disease, Pelizaeus-Merzbacher disease, and secondary progressive multiple sclerosis. This expansion also enables Sana to provide manufacturing capabilities across its portfolio, including production of cell lines for early-stage clinical trials.

The company intends to hire up to 10 roles in manufacturing, quality assurance (QA), and quality control (QC) over the next two years to operate the facility.

The partnership builds upon decades of research in the lab of Steve Goldman, M.D., Ph.D., Sana’s Head of CNS Therapy and co-director of the URMC Center for Translational Neuromedicine. Goldman’s work has helped redefine understanding of the basic biology and function of important support cells found in the central nervous system. His lab has devised new ways to manipulate the fates of embryonic and induced pluripotent stem cells to create glia.` A subtype of these, called glial progenitor cells, give rise to astrocytes and oligodendrocytes, cells that play a critical role in the health and signaling of nerve cells.

In diseases such as Huntington’s, multiple sclerosis, and childhood leukodystrophies, the development and function of glial cells are impaired or lost during the course of the illness. Goldman’s lab has shown that when these cells are replaced with healthy ones in animal models, this approach can slow or even reverse the progression of these diseases.

To advance this cell replacement strategy to the clinic, Goldman and Dr. Christina Trojel-Hansen, Ph.D., co-founded Oscine in 2017, which licensed intellectual property developed in his lab at Cornell University and the University of Rochester. In 2018, Sana and Oscine entered into a collaboration, license, and option to purchase agreement that included, among other things, support for continuing research at URMC. In 2020, Sana acquired Oscine, including Oscine’s rights to cell engineering programs focused on brain disorders, and named Goldman as Sana’s Head of CNS Therapy. Sana continues to support research in Goldman’s lab at URMC today.

Sana’s goal is to repair and control genes in cells or replace missing or damaged cells that can solve the underlying cause of many diseases. These emerging capabilities have the potential to create novel medicines that meaningfully improve patient outcomes. Cell lines, gene-edited cell lines, and differentiated cells intended for use in human clinical trials will be developed at the URMC’s Cell and Tissue Manufacturing Facility. The CTMF is designated as a good manufacturing practices (GMP) facility, as it is certified as being compliant with federal guidelines necessary for the production of human cells for scientific investigation and for their use in investigational therapeutics.