URMC spinoff’s pioneering research gets a boost

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A new University of Rochester Medical Center biotech spinoff focused on cell-based treatments for degenerative nerve disorders like multiple sclerosis and Huntington’s chorea has received a significant funding boost.

A multiyear investment in the spinoff, Oscine Therapeutics, by Sana Biotechnology will advance Oscine’s research and help move the therapies it is developing through clinical trials and into clinical practice, says Oscine CEO Christina Trojel-Hansen. Exact terms of the investment are not being publicly stated. 

Based in Seattle, San Francisco and Cambridge, Mass., Sana was founded by Steve Harr and Hans Bishop, a pair of former executives of Juno Therapeutics, a Seattle-based biotech firm that was acquired by Celegene Corp. last year for $9 billion. Investors in Sana include the biotech venture capital firms Arch Venture Partners, Flagship Pioneering, and F-Prime Capital Partners.

Degenerative nerve diseases like MS and Huntington’s eat away at the central nervous system, robbing sufferers of motor functions. Ultimately, they completely incapacitate sufferers and can affect the ability to speak as well as mental abilities and personality. 

Some MS sufferers have a relapsing form in which they sporadically lose and regain abilities. Others have a more steadily advancing progressive form. Huntington’s inexorably kills brain cells, ultimately inducing a form of dementia as well as physical symptoms such as loss of ability to speak. In end stages, such diseases require 24-hour care.

The therapies Oscine is working on derive from the research of URMC scientist and Oscine president and co-founder Stephen Goldman. They aim to slow or halt the course of such diseases by promoting patients’ ability to generate new glia, cells whose function is to protect the nerve cells that carry sensations to the brain and carry orders from the brain to muscles.

“In many instances it appears that faulty support cells of the brain are driving the (neurodegenerative) disease process. 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,”says Goldman, distinguished professor of neuroscience at URMC’s Center for Translational Neuromedicine.

The new biotech company’s name—the Latin designation for passerine birds, a class that includes songbirds—is Goldman’s nod to the avian ability that led him to pursue the line of research the company is based on. 

Songbirds use their distinctive songs to mark territory, communicate warnings and attract mates. They develop complex new songs annually. Some peal out an increasingly complicated series of notes as each season develops.

Goldman explains: “That turns out to be associated with a part of the brain that controls song enlarging in size and so we started looking at that a long time ago from the standpoint of: what are the cells that mediate that process, and it turned out that new neurons, new brain cells were being generated.”

He wondered: Where are these new neurons coming from? 

It turns out that they come from progenitor cells, which are something like stem cells. But where stem cells can develop into virtually any type of cell, progenitor cells are programmed to only develop into a specific kind of cell. Adult humans lose progenitor cells. Adult songbirds still have them. 

“That’s what hadn’t been realized,” Goldman says. “Progenitor cells that persisted into adulthood (in birds) didn’t go away.”

That realization led Goldman to pursue years of research that in turn led to the development of techniques that now hold out a possibility of relief for sufferers of degenerative nerve disorders. While promotion of new glial cells would not directly address the causes of degenerative nerve disorders, the technique could not only halt the progression of such diseases but actually reverse harm they have already done, Goldman believes.

“Finally, an uplifting report that addresses MS reversal as opposed to years of your reports addressed to treating symptoms and watching ourselves in hopeless decline. I pray to the Archangels for these scientists in Rochester,” wrote a commenter on a mid-June article describing Oscine’s work in Multiple Sclerosis News Today, a newsletter aimed at MS sufferers and their families.

Goldman’s research has focused on stem cells including a type called pluripotent stem cells that reverse course to go from an already differentiated cell type such as a skin cell back to a stem cell state in which they have the potential to develop into a variety of cell types.

Under a sponsored research agreement between Oscine and URMC, up to 21 researchers will staff Goldman’s URMC lab to do research and development to get Oscine’s treatment ready for clinical trials. 

Oscine plans to have Waisman Biotechnologies, a firm that specializes in contract manufacturing for early-stage clinical trials and helps move young biotech firms through regulatory hurdles, produce material for trials. 

Oscine’s business operations are in Seattle, where Trojel-Hansen is based. The startup licenses intellectual property from UR and Cornell University. Goldman and UR have equity stakes in the company.

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