As a leader in life-science research, Brandeis is fueling innovation in biotechnology in areas where there is unmet need for more effective treatment or diagnosis. One of the university’s success stories is the biopharmaceutical start-up Syntonix, formed by Brandeis, Brigham and Women’s Hospital, and Children’s Hospital Boston to commercialize novel drug-delivery methods. In 2007 Biogen Idec bought Syntonix and it became the first Brandeis spinout to be acquired by a biotech giant.Syntonix was started by Brandeis biologist Neil Simister, Dr. Wayne Lencer of Children’s Hospital; Dr. Richard S. Blumberg of Brigham and Women’s; and Blumberg’s brother Dr. Laurence Blumberg ’83, a member of the Brandeis University Scientific Advisory Council and a prominent biotechnology investor.
Syntonix’ technologies harness the human body’s natural immunological pathways to provide a novel method of drug delivery. Many pharmaceuticals consist of molecules too large to be absorbed through the mucous membranes, meaning that patients with chronic conditions like hemophilia, anemia, multiple sclerosis and autoimmune disorders must take drugs intravenously, by injection. Frequent dosing is typically needed, because the drugs break down quickly in the bloodstream.
In the early- to mid-1990s, Simister, Lencer, Blumberg and their colleagues discovered that the molecular receptor that carries immunoglobulin G antibodies from mother to fetus across the placenta is also found in the mucous membranes lining the intestines, airways, and lungs. This discovery led to the idea that the receptor, known as FcRn, could be used to carry large-protein drugs across mucous membranes into the bloodstream, suggesting the possibility of replacing these injection drugs with inhaled or oral versions.
Then the scientists discovered that the FcRn receptor also prevents antibodies from breaking down quickly in the bloodstream, the normal fate of other molecules. This rescue capability made FcRn part of a so-called salvage pathway. To take advantage of this pathway, Syntonix scientists designed pharmaceutical proteins that bind to FcRn, extending the lifetimes of these drugs in the bloodstream.
“We founded Syntonix with the hope of translating our basic discoveries into improvements in health care. The company developed and expanded our technologies to the stage where they have preclinical drug candidates for treating hemophilia and infertility,” says Simister, adding, “Biogen Idec’s acquisition is an excellent outcome because they have the expertise in manufacturing and development to bring these drugs to the clinic.”
In early 2008, Syntonix filed an application to begin clinical trials of their first product, Factor IXFc for the treatment of hemophilia B.
“It’s a very good outcome; the underlying biology is sound, and in three to four years we could have a life-saving drug on the market,” says Laurence Blumberg. “It’s all about innovation.”