A longtime recipient of funding from the New York State Department of Health’s NYSTEM program has launched a new biotech company that aims to develop a treatment for Parkinson’s disease using human embryonic stem cells. The company is supported by $225 million in Series A financing from Bayer AG in Germany and Versant Ventures in the U.S., considered one of the largest-ever Series A financings for a biotech company.
Dr. Lorenz Studer, Director of the Center for Stem Cell Biology at Memorial Sloan Kettering Cancer Center (MSKCC), and his collaborator and spouse, neurosurgeon Dr. Viviane Tabar, have teamed up with Dr. Gordon Keller of the University of Toronto and the McEwen Centre for Generative Medicine to form BlueRock Therapeutics. With current NYSTEM funding, Dr. Studer and colleagues expect to seek approval from the U.S. Food and Drug Administration within the year to begin clinical trials. If approved, BlueRock will fund this work.
Dr. Studer’s research uses human embryonic stem cells to produce the specific subset of dopamine neurons that are lost in patients with Parkinson’s disease, a motor system disorder that produces tremors, rigidity, slow movements and impaired balance and coordination. Parkinson’s, affects half a million people in the United States and up to five million worldwide.
“The Department of Health is proud to sponsor the work of Dr. Studer, whose pioneering research has led to important breakthroughs in Parkinson’s disease,” said Commissioner of Health Dr. Howard Zucker. “Dr. Studer’s work exemplifies our goals for NYSTEM, which are to build a strong stem cell research community, advance scientific knowledge about stem cell biology and develop therapies and diagnostic methods to alleviate disease and improve human health.”
In 2008, Dr. Studer was among the first to receive an award from New York’s Stem Cell Science program, known as NYSTEM. He has since received six NYSTEM awards in all, for a total of approximately $23 million. In 2012, his awards culminated in a $14.9 million consortium award, which was dedicated to the preclinical research needed to bring the technology into clinical use. In 2015, Dr. Studer received a MacArthur Foundation “Genius” Award, and credited his work to NYSTEM, “an important partner throughout (his) career.”
NYSTEM was created in 2007 to support stem cell research in the state. NYSTEM funds basic, applied, translational and other research and development activities that advance scientific discoveries in stem cell biology. It also supports infrastructure, training and education. Since its founding, NYSTEM has issued 323 awards for $354 million to more than 35 institutions across the state.
BlueRock Therapeutics will be based in New York, as well as Toronto and Boston. In addition to Parkinson’s research, the company is working to develop the technology to regenerate heart muscle in patients who have had heart attacks or who have chronic heart failure.
The company has also licensed intellectual property from a Japanese group that will enable BlueRock to develop induced pluripotent stem cells (iPSCs). iPSCs are similar to human embryonic stem cells, but can be made from any person through simple skin biopsies or cells in blood. iPSCs allow stem cells to be generated from individual patients, which reduces the risk of immune rejection when stem cells come from another person.