Sigilon Therapeutics Receives Orphan Drug Designation for a Treatment of Hemophilia A

The following is a press release from Sigilon Therapeutics. Read the press release on the Sigilon website here.

Sigilon Therapeutics has announced it has received Orphan Drug Designation for SIG-001, an investigational therapy for hemophilia A that leverages Sigilon’s Shielded Living Therapeutics™^ platform to implant cells engineered to produce stable blood plasma levels of factor VIII, a crucial clotting protein.
“We 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,” said Rogerio Vivaldi, M.D., MBA, President and Chief Executive Officer of Sigilon. “This 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.”
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’s Afibromer™Â biomaterials matrix, which prevents the immune system from rejecting the cells and averts foreign body response or fibrosis.
Read the press release on the Sigilon website here.