Vertex Pharmaceuticals is betting nearly $1 billion on a company developing a stem cell-based therapy and device with the stated goal of creating a potentially curative treatment for Type 1 diabetes, though it is still preclinical.
Boston-based Vertex said Tuesday that it would pay $950 million to buy Semma Therapeuticis, a privately held company based on nearby Cambridge. The company is developing a therapy using stem cell-derived human islets.
Type 1 diabetes is an autoimmune disease that results from destruction of the insulin-producing beta cells in the endocrine pancreatic islets of Langerhans cells. A commentary published in May in the journal Diabetes by University of Pennsylvania researcher Michael Rickels stated that human embryonic stem cells differentiated into pancreatic cells have the potential to be further differentiated in vivo into functional pancreatic islets. That has the potential to lead to the development of cell therapies for treating the disease.
According to the companies, Semma has made two of what it called major scientific advances, namely the ability to produce large numbers of functional beta sells that restore insulin secretion, as well as a device that encapsulates and protects the cells from the immune system, enabling implantation without the need for immunosuppressive therapy.
“We see a substantial opportunity to transform the treatment paradigm for Type 1 diabetes, a specialty disease cared for by endocrinologists, both by advancing the development and manufacturing of the cells themselves, as well as through the highly innovative cell-device combination,” Vertex CEO Jeffrey Leiden said in a statement.
The company announced preclinical data in primates and pigs in July. In the non-human primates, the stem cell islets were delivered into diabetic animals under immunosuppression, successfully engrafting and showing high functionality, persisting over six weeks in the liver and reducing the need for insulin by 60 percent. In another study, pigs received the cells in Semma’s device, which is designed to keep the cells alive without the need for immunosuppression, with results showing immunoprotection that helped keep cells alive.
The company said it plans to initiate two human clinical trials next year.
Photo: gustavofrazao, Getty Images
