Investigational Therapy for Hemophilia A Granted Orphan Drug Designation

The Food and Drug Administration (FDA) has granted Orphan Drug designation to SIG-001 (Sigilon Therapeutics) for the treatment of hemophilia A.

SIG-001 utilizes Sigilon’s Shielded Living Therapeutics platform to implant engineered human cells to produce stable blood plasma levels of factor VIII. The cells are also shielded by Sigilon’s proprietary Afibromer biomaterials matrix that minimizes immune rejection and fibrosis. 

The Company is currently assessing SIG-001 in IND-enabling studies and expects to begin clinical trials in the first half of 2020. Preclinical studies of SIG-001 have demonstrated a consistent delivery of blood plasma clotting factor at therapeutic levels. 

“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 CEO of Sigilon. 

For more information visit sigilon.com.