BEAM-101, a genetically modified cell therapy for sickle cell disease (SCD) that’s being tested in clinical trials, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The FDA gives the designation to experimental therapies designed to treat conditions that affect fewer than 200,000 people…

The American Institutes for Research (AIR) has been awarded a $28 million federal contract to help make cell and gene therapy (CGT) treatments more accessible to Medicaid recipients, initially focusing on sickle cell disease (SCD). The contract, from the Center for Medicare and Medicaid Innovation in the Centers for…

Starting in 2025, the U.S. Department of Health and Human Services (HHS) will introduce a pricing model that aims to make new cell and gene therapies more accessible for people with sickle cell disease who are on Medicaid, the health program for lower income residents jointly funded by the…

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…

The California Institute for Regenerative Medicine (CIRM) and the National Heart, Lung and Blood Institute (NHLBI) have entered into a “landmark” collaboration to co-fund and help speed the development of cell and gene therapies to cure sickle cell disease (SCD), according to a press release.