Beta-cell findings to be commercialised

Written by Victoria English

Research by Douglas Melton of Harvard University and colleagues showing that insulin-producing pancreatic beta cells can be created in a laboratory has reached the stage of commercialisation with the launch of a new company aimed at bringing a cell therapy for diabetes into human trials.

Research by Douglas Melton of Harvard University and colleagues showing that insulin-producing pancreatic beta cells can be created in a laboratory has reached the stage of commercialisation with the launch of a new company aimed at bringing a cell therapy for diabetes into human trials.

The company, Semma Therapeutics Inc of Cambridge, Massachusetts, announced the closing of a $44 million Series A financing round on 24 March enabling it to bring the technology from the laboratory into a pharmaceutical setting. Dr Melton is the scientific founder of the company and the chief executive is Robert Millman, managing director of MPM Capital, the leader of the financing round.

Last year, Dr Melton and colleagues succeeded in creating insulin-producing pancreatic beta cells from human pluripotent stem cells in the laboratory. The scientists were able to show that the cells expressed the same markers found in mature human beta cells and were able to secrete similar quantities of insulin to that of adult beta cells. Their findings were reported in the journal Cell on 9 October 2014.

Boston-based MPM Capital was supported in the financing round by Fidelity Biosciences, ARCH Venture Partners and Medtronic Inc, the medical device company. Alongside the equity financing, Semma has entered into an undisclosed strategic agreement with Novartis Pharmaceuticals. Novartis is one of the few big pharma companies to have a separate cell and gene therapy business unit, and has invested heavily in an experimental cancer therapy that involves the genetic engineering of T cells. The combination of the Series A financing and the Novartis agreement will reportedly give Semma enough capital to bring a new cell therapy from the laboratory through clinical proof-of-concept trial in man.

Separately, AstraZeneca Plc announced the start of a new five-year collaboration with the Harvard Stem Cell Institute to use beta cells produced under the Melton protocol for research. Scientists from the UK company and the university will work together to study the biology around human beta cells and to use the laboratory-produced cells for drug screening. “We think these cells are absolutely crucial because they are the real target cells in the human body, compared with rodent cells or recombinant cells or genetically modified cells,” said Marcus Schindler, head of the company’s cardiovascular and metabolic diseases unit, in an interview. AstraZeneca will provide funding for investigators at Harvard led by Dr Melton, as well as a team at its business unit in Mölndal, Sweden.

“We are interested in finding compounds that could positively influence the properties of those cells,” the executive said. This means screening compounds against the cells to search for potential new medicines that could restore beta cell activity in diabetic patients. “What we are not aiming to do is to come up with a cell therapy for diabetes,” he added.

AstraZeneca would also like to use gene editing technology to interrogate specific pathways in the beta cells, he noted. The agreement is non-exclusive. Financial terms have not been disclosed.

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