Patricia Inácio, PhD, science writer —

Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high unmet…

CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are…

Use of medical marijuana to ease pain is associated with fewer visits to the hospital in people with sickle cell disease (SCD), a study suggests. Patients given medical marijuana felt this was an effective treatment for pain relief, and also reported a reduction in the need for opioids.

The cannabis company Parallel, which aims to improve quality of life with its products, has launched a 10-year collaboration with the University of Pittsburgh, known as Pitt, to investigate the potential of medical marijuana for treating people with sickle cell disease (SCD). Pitt’s research program will receive an…

The National Heart, Lung, and Blood Institute (NHLBI) awarded nearly $1 million to Athena Starlard-Davenport, a professor at the University of Tennessee Health Science Center (UTHSC), to support work into fetal hemoglobin as a potential way of treating sickle cell disease (SCD). “For the past 100 years,…

Bluebird Bio extended a partnership with two divisions of Hitachi Chemical for the long-term development and manufacturing of LentiGlobin, its investigational gene therapy for sickle cell disease (SCD), and other potential and approved treatments. The two subsidiaries — Hitachi Chemical Advanced Therapeutics Solutions (HCATS) and Apceth Biopharma — will be in charge…

The Phase 2 STRONG-SCD trial evaluating olinciguat as a potential oral therapy for sickle cell disease is completely enrolled, Cyclerion Therapeutics said. Seventy patients with stable disease were recruited, and topline results are expected between July and September. The company is not expecting the supply of…

Global Blood Therapeutics (GBT) is giving $250,000 to support the U.S. sickle cell disease (SCD) community during the COVID-19 pandemic. Its newly created GBT Community Fund will award a total of $150,000 in grants to non-profit organizations supporting the needs of  SCD patients and their families during…

High blood levels of two pro-inflammatory molecules, called interleukin-6 (IL-6) and interleukin-8 (IL-8), are linked to more frequent complications in people with sickle cell anemia, a study reports. The study, “High levels of proinflammatory cytokines IL-6 and IL-8 are associated with a poor clinical outcome…

The Institute for Clinical and Economic Review (ICER) says three recently approved therapies for managing sickle cell disease (SCD) are too costly based on early assessments of their effectiveness, and is recommending their prices be lowered. In a new evidence report, the independent watchdog agency on drug…