Emerging Biotech Enhances CNS Focus Through a Strategic Acquisition

Sana CEO_Sana

Steve Harr, CEO of Sana, pictured above. Photo courtesy of Sana. 

Sana Biotechnology,  a biotechnology company focused on creating and providing engineered cells as accessible medicines for patients, is taking a deeper dive into its CNS therapeutic focus. On October 30, the company announced the acquisition of long-time collaborator Oscine Corp.

Oscine is developing potentially curative or disease-modifying cell therapies for diseases of the brain and central nervous system (CNS) through its glial progenitor cell (GPC) program and underlying technologies. Sana will combine this program with its existing in vivo cell engineering platform for the treatment of CNS diseases with unmet needs.

GPCs become either astrocytes, which provide regulatory and structural support for the brain, or oligodendrocytes, which make a protective layer called the myelin sheaf and enable information to move more quickly across axons. Together, they make up the majority of the cells in the brain. Their dysregulation is responsible for a number of CNS disorders.

“They’ve [Oscine] been pushing forward in a host of different disorders,” Sana president and CEO Steve Harr told BioSpace. “A few of the adrenoleukodystrophies are congenital, or genetic, disorders of oligodendrocytes in the brain white matter. The most advanced of those is something called PMD [Pelizaeus-Merzbacher disease] where these kids are born without oligodendrocytes sites and they die very young. The second is progressive multiple sclerosis, where it’s no longer really about the immune response. You just don’t have oligodendrocytes sites to support the brain.”

With operations in Seattle, Cambridge and South San Francisco, Sana’s vision is to repair and control genes, replace missing or damaged cells and develop the processes and technology to ensure widespread access to the resulting therapies.

Harr has an ambitious and altruistic purpose for the company. 

“We’re really excited about the data that others have produced and the opportunity inside of cell therapy, but one of the things that’s striking is that we have to do a lot more to ensure broad accessibility to these therapies,” Harr said. “So our three aspirations are to be able to repair and control the genes of any cell in the body, replace the cells in the body, and expand access to this class of medicines. It is said most succinctly by having an aspiration that access to our medicines will never be decided by where you’re born.”

Harr is working toward this goal by recruiting the best scientific minds in each of Sana’s priority therapeutic target areas: Oncology, genetic disorders, cardiometabolic diseases (CMDs) such as Type 1 diabetes and myocardial ischemia, and CNS disorders.   

Sana has already hooked some pretty impressive names. Senior Vice President and Head of T Cell Therapeutics, Dr. Terry Fry, previously served as the Head of the Hematologic Malignancies Section in the Pediatric Oncology Branch at the National Institutes of Health (NIH). There, he led efforts in cellular immunotherapy for pediatric leukemia including CAR T cell programs against CD19 and CD22.  CSO of Cell Therapy, Dr. Chuck Murry, conducted research that pioneered the use of human pluripotent stem cells for heart regeneration.

Now, they’ve scored the dedicated services of Oscine’s scientific founder, Dr. Steve Goldman, who joins the company as Head of CNS Therapy.

“In order to go after these things, these really complex areas of biology, you have to have one of a couple of world’s experts because it’s really hard. And so much of the devil and the angel are in the detail. Those details are understood by only a few people and Steve happens to be one of them,” Harr said. “Oscine has done a really nice job, and Steve Goldman in his research before this, of showing that in animal models, replacing these GPC cells or putting in new ones, has a profound impact in animal models on the progression of the disease.”

Goldman, for his part, is excited by the commitment and resources Sana is putting into advancing this research. 

"I am delighted to be joining Sana at this critical juncture in bringing stem cell-based therapeutics to the clinic," Goldman said in a statement. “After three decades of research into how to repair the cellular structure of the diseased brain, it is heartening to know that Sana plans to urgently drive these therapies to the clinic to explore their potential benefit for the many patients and their families stricken with these largely incurable diseases.”

Harr explained that the CNS is an area where he believes cell therapy can make a pivotal difference.

“It’s been really great to see the impact that it’s having on a number of orphan or rare diseases, but to really make this something impactful, we need to go after the diseases that most of our loved ones will die from. And the CNS is such an unmet need,” Harr said. “One of the reasons is that single pills or antibodies don’t really deal with the complexity of the brain, and we need these better cells to really kind of grapple with fixing the network of the brain. This is where cell therapy can have its most profound impact.”

The excitement for Sana in developing these “better cells” to help patients suffering from diseases like PMD, progressive multiple sclerosis and Huntington’s disease is clear. 

“Hopefully we’ll have an opportunity to treat some of these patients in the not too distant future,” Harr said, adding that he hesitated to be any more precise on a target date until Sana has alignment with regulators. 

“A lot of it has to do with, these are really unmet needs and we want to make sure that we set out dates that we can live up to, because setting expectations for these patients is something that we take really seriously.”

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