At the recent 2019 American Society of Gene and Cell Therapy Annual Meeting in Washington, DC, Sigilon Therapeutics presented data on an investigational cell therapy the company is developing to treat hemophilia A.
The therapy, known as SIG-001, includes human cells that are modified to carry significant amounts of synthetic DNA material that can illicit the production of the therapeutic protein factor VIII (FVIII). The therapy is further enhanced with a “shield” composed of a synthetic biomaterial called Afibromer which effectively blocks an unwanted immune response. Sigilon is developing the Shielded Living Therapeutics platform with the help of researchers from the Massachusetts Institute of Technology (MIT). Both Sigilon and MIT are based in Cambridge, Massachusetts.
Investigators are evaluating the therapy in mouse models with hemophilia A. A single dose of SIG-001 administered via the abdomen of the mouse subjects controlled bleeding and sustained therapeutic levels of FVIII for more than six months.
“These data illustrate the potential of this Shielded Living Therapeutics product to provide consistent, lasting delivery of factor VIII, and they form the basis of our planned regulatory filing for SIG-001 later this year,” said David Moller, MD, chief scientific officer of Sigilon.
Source: Hemophilia News Today, May 17, 2019