Sigilon Therapeutics recently announced that it has received Orphan Drug Designation (ODD) for SIG-001, the company’s investigational therapy for hemophilia A. ODD, which is granted by the Office of Orphan Products Development of the U.S. Food and Drug Administration, is awarded to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States.
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 currently 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. SIG-001 is designed to be delivered directly to the abdomen via a minimally invasive laparoscopic procedure, whereby 2-4 small incisions allow for an entry point in which capsules carrying the therapy may be infused into the abdominal cavity.
“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, MD, 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.”
Sigilon expects to begin clinical trials in the first half of 2020.
Source: Sigilon press release dated August 27, 2019
