CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sigilon Therapeutics today presented preclinical data demonstrating the feasibility of cellular therapies for bleeding disorders developed with the company’s Shielded Living Therapeutics™ platform, which was designed to deliver functional cures for chronic diseases. The data were shared in an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress in Melbourne, Australia.
Wild-type mice and knockout disease models were dosed with human cells genetically engineered to produce stable blood plasma levels of the clotting factors missing in patients with hemophilia. The engineered cells were protected by Sigilon’s Afibromer™ biomaterials matrix, which prevents the immune system from rejecting the cells and averts foreign body response (fibrosis), thus enabling a sustained therapeutic effect.
Intraperitoneal implantation in mice resulted in steady therapeutic levels of blood plasma clotting factor levels. Additional studies in murine models of hemophilia A found dose-dependent levels of functional hFVIII in plasma, resulting in phenotype correction, including normalizing bleeding time.
“These data demonstrate that we have effectively optimized our Shielded Living Therapeutics™ platform to deliver sustained, functional protein production after a single administration,” said David Moller, M.D., Sigilon’s Chief Scientific Officer. “By shielding the cells from fibrosis and immune rejection, we believe we can deliver a durable therapy that will liberate patients from the burden of frequent infusions and the peaks and troughs of factor production that accompany standard factor replacement therapy. We’re excited to take the next step in advancing these programs.”
Sigilon is completing IND-enabling studies for SIG-001, its therapy for hemophilia A, and expects to begin first-in-human studies in the first half of 2020. Data presented at ISTH and earlier this year at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting will support Sigilon’s planned regulatory filing.
The ISTH abstract was entitled: “Correcting Bleeding Disorders Using Blood Clotting Factors Produced In Vivo By Shielded Engineered Allogeneic Cells” and was presented by David Peritt, Ph.D., Sigilon’s Chief Technology Officer.
About Sigilon Therapeutics
Sigilon Therapeutics is a biopharmaceutical company creating functional cures for chronic diseases using its Shielded Living Therapeutics™ platform. To create a Shielded Living Therapeutics™ product, we engineer novel human cells that we encase in a proprietary immune-shielding matrix and place in the body. These Shielded Living Therapeutics™ products then produce therapeutic proteins in a programmable and durable fashion, without generating fibrosis or immune rejection. Sigilon was founded and created by Flagship Pioneering in conjunction with Daniel Anderson, Ph.D., and Robert Langer, Sc.D., of the Massachusetts Institute of Technology.