Safi secures FDA fillip in work on sickle cell disease

Safi Biotherapeutics, a specialist in stem-cell derived red blood cell products, has received Rare Pediatric Disease designation and Orphan Drug designation from the US regulator.

The firm is developing blood products for use in chronic transfusion therapy for sickle cell disease (SCD) patients, helping the estimated 10% of SCD patients that need regular transfusions.

These patients often face complications such as alloimmunization or difficulty finding suitable donor matches. Safi plans to advance its manufacturing processes and begin clinical trials by 2027.