蘑菇视频APP

CAMBRIDGE, Mass.--August 27, 2019-- today announced it has received Orphan Drug Designation for SIG-001, an investigational therapy for hemophilia A that leverages Sigilon鈥檚 Shielded Living Therapeutics鈩� platform to implant cells engineered to produce stable blood plasma levels of factor VIII, a crucial clotting protein.

鈥淲e are very pleased to have received Orphan Drug Designation for SIG-001. The designation underscores the critical unmet need for effective, durable therapies for hemophilia A and reinforces our commitment to advance SIG-001 through our development program,鈥� said Rogerio Vivaldi, M.D., MBA, President and Chief Executive Officer of Sigilon. 鈥淭his is the first of what we hope will be multiple Orphan Drug Designations for Sigilon as we continue progressing novel therapies for chronic diseases through our pipeline.鈥�

Orphan Drug Designation is granted by the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA). The designation is awarded to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States. Having received the designation, Sigilon is eligible for various development incentives for SIG-001, including tax credits for qualified clinical testing.

Sigilon is currently completing IND-enabling studies for SIG-001 and expects to begin clinical trials in the first half of 2020. In preclinical studies, Sigilon has demonstrated that its engineered cells produce steady therapeutic levels of blood plasma clotting factor. The cells are protected by Sigilon鈥檚 Afibromer鈩� biomaterials matrix, which prevents the immune system from rejecting the cells and averts foreign body response, or fibrosis.

About Sigilon Therapeutics

Sigilon Therapeutics is developing functional cures for chronic diseases through its Shielded Living Therapeutics鈩� platform. Sigilon鈥檚 therapeutics consist of novel human cells engineered to produce the crucial proteins, enzymes or factors needed by patients living with chronic diseases such as hemophilia, diabetes and lysosomal storage disorders. The engineered cells are protected by Sigilon鈥檚 Afibromer鈩� biomaterials matrix, which shields them from immune rejection and fibrosis. Sigilon was founded by 蘑菇视频APP in conjunction with Daniel Anderson, Ph.D., and Robert Langer, Sc.D., of the Massachusetts Institute of Technology.