Sigilon, MIT Collaborating to Develop Programmable Cell Therapies for Hemophilia, Other Diseases

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by Alice Melão |

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Sigilon Therapeutics and the Massachusetts Institute of Technology will work together to develop programmable cell therapeutics for the treatment of hemophilia, lysosomal storage diseases, and other serious chronic illnesses.

The collaboration will combine Sigilon’s proprietary cell technology with synthetic biology approaches developed by Ron Weiss, PhD, a professor at the MIT Synthetic Biology Center.

Weiss and his collaborators will use their Landing Pad technology to perform chromosomal site-specific engineering of Sigilon’s proprietary cells. This approach will allow the researchers to insert large amounts of synthetic DNA-encoding therapeutic proteins into cells.

“We’re gratified to be able to apply our Landing Pad technology toward creating new therapeutics for patients with serious chronic diseases,” Weiss, also an affiliate member of the Koch Institute, said in a press release. “This collaboration will further our work to utilize programmable cell circuits in biomedical therapies and beyond.”

Sigilon’s cells were altered to have a shield made of synthetic biomaterial, called Afibromer, that can prevent triggering the immune system of the host. With this new technology, the cells can be implanted with a reduced risk of potentially damaging immune reactions, such as fibrosis (tissue scarring), while promoting long-term production of therapeutic proteins after a single intervention.

With these approaches, the company is anticipating production of the next generation of its cell-programmed Shielded Living Therapeutics.

“This powerful alliance with MIT will provide Sigilon access to best-in-class methods for in vivo delivery of therapeutic proteins, enabling our Shielded Living Therapeutics product platform to address an even broader range of chronic diseases,” David Moller, MD, chief scientific officer of Sigilon, said.

Sigilon is developing two potential cell therapies for the treatment for hemophilia, SIG-001 for hemophilia A and SIG-003 for hemophilia B.

In March 2018, the European Medicines Agency (EMA) granted Advanced Therapy Medicinal Product (ATMP) designation to SIG-003 for the long-term treatment of patients with hemophilia B. This decision followed the positive recommendation of the EMA’s Committee for Advanced Therapies specifically regarding allogeneic cells (from a matched donor) genetically modified to express human factor IX protein embedded in the Afibromer biomaterial (SIG-003) for hemophilia B.

The company also announced that it anticipates the start of clinical trials for SIG-001 in hemophilia A patients this year.