Editas Medicine has expanded its partnership with Azzur Cleanrooms on Demand (COD) for the manufacturing of EDIT-301, its experimental cell-based gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. If approved, the scaling up of EDIT-301 needs to follow current good manufacturing practice (cGMP) standards — set…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EDIT-301, an experimental cell-based gene-editing therapy given as a one-time infusion for the treatment of sickle cell disease (SCD). The designation is given to spur the development of therapies for diseases affecting fewer than 200,000 people…

The experimental gene-editing therapy EDIT-301 appears to be working largely as expected in the first two sickle cell disease (SCD) patients treated as part of the Phase 1/2 RUBY trial, according to new data shared by the therapy’s developer, Editas Medicine. “These promising clinical results from the RUBY…

The gene editing therapy EDIT-301 appears to be working as intended in the first person with sickle cell disease (SCD) who received the experimental treatment, according to recent data from the Phase 1/2 RUBY clinical trial. Editas Medicine, which is developing EDIT-301, also announced that the U.S. Food…

Enrollment is underway for a Phase 1/2 trial evaluating the safety and efficacy of EDIT-301, an experimental gene editing cell therapy for people with sickle cell disease (SCD). The trial, called RUBY (NCT04853576), had been cleared for launch by the U.S. Food and Drug Administration earlier this…

The U.S. Food and Drug Administration (FDA) has cleared the initiation of a Phase 1/2 trial investigating EDIT-301, Editas Medicine’s experimental gene editing cell therapy for  sickle cell disease (SCD). The planned open-label study, to be called RUBY, will assess the safety and efficacy of a…

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to EDIT-301, a potential cell therapy for the treatment of sickle cell disease (SCD). This designation is given to therapies that aim to treat serious or life-threatening disorders that affect fewer than 200,000 people…