Marisa Wexler, MS, senior science writer —

Endari (L-glutamine), an approved oral treatment for sickle cell disease (SCD), has now been added to the Preferred Drug List in Texas. The move will make Endari more accessible to people in the state who are insured through Medicaid, the government program that provides health insurance to low-income…

Emmaus Life Sciences has submitted an application seeking the approval of its oral medication Endari (L-glutamine) to treat sickle cell disease (SCD) in Kuwait. The company announced that the Kuwait Drug and Food Control administration, which is responsible for registering pharmaceutical products in the country, has accepted…

A new research collaboration between Jasper Therapeutics and Aruvant Sciences will evaluate the use of JSP191, Jasper’s anti-CD117 monoclonal antibody, as a conditioning agent for ARU-1801, Aruvant’s experimental gene therapy for sickle cell disease (SCD). “The unique attributes of ARU-1801 enable us to bring a potentially curative one-time therapy…

Bluebird Bio is set to resume trials of LentiGlobin, its investigational gene therapy for sickle cell disease, after the U.S. Food and Drug Administration (FDA) lifted its clinical hold. The company is now working closely with study investigators and clinical trial sites to resume normal activities as soon…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

A spoken word poetry contest is welcoming submissions from people with sickle cell disease (SCD) and their caregivers. The contest, “Lift Every Voice to Shine the Light on Sickle Cell,” is being jointly launched by Global Blood Therapeutics (GBT) and the Sickle Cell Disease Association of America…

LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), is unlikely to be the cause of a blood cancer that arose in one of the patients who received the therapy in an early clinical trial, according to new analyses. In February, Bluebird paused two clinical…

Red blood cells in people with sickle cell disease (SCD) flow through blood vessels in a manner that damages the walls, which may explain blood vessel problems and increased inflammation in SCD, new research indicates. The study, “Flow-induced segregation and dynamics of red blood cells in sickle cell…

The European Medicines Agency (EMA) is reviewing an application from Global Blood Therapeutics (GBT) that seeks full marketing approval for the oral therapy Oxbryta (voxelotor) for people with sickle cell disease (SCD), ages 12 and up. Oxbryta would be the first treatment targeting the root cause of…

FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), is well-tolerated in people with SCD and induces improvements in several red blood cell parameters, early clinical trial data indicate. The results observed in this initial group of patients support the efficacy of Forma Therapeutics‘ investigational medication, as…