CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sigilon Therapeutics, a biopharmaceutical company creating functional cures for chronic diseases using its Shielded Living Therapeutics™ platform, today delivered an oral presentation featuring data on its hemophilia A program (SIG-001) at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Washington, D.C. The data presented will support the company’s planned regulatory filing of SIG-001 later this year to enable initiation of clinical trials.
In addition, the company delivered a poster presentation on data pertaining to its pipeline of Shielded Living Therapeutics™ products for lysosomal storage disorders (poster #514).
The oral presentation is entitled “Correcting Bleeding Disorders Using Blood Clotting Factors Produced by Shielded Engineered Allogenic Cells.” Data included in the presentation show that, as a result of Sigilon’s optimization of cell and matrix engineering, SIG-001 demonstrates in vivo functional FVIII secretion and plasma levels at or above the therapeutic range in mice, including a mouse model of hemophilia A. These effects show a tight dose-response relationship and have remained durable over several months of observation.
Furthermore, administration of SIG-001 was shown to normalize the disease phenotype of excessive bleeding in the mouse model of hemophilia A. Seven days after placement of SIG-001, treated mice displayed a bleeding time that was restored to normal.
“Current factor-replacement treatments for hemophilia require frequent infusions and still leave patients vulnerable to the harmful effects of therapeutic troughs,” said David Moller, M.D., chief scientific officer of Sigilon. “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.”
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.