Patricia Inácio, PhD,  science writer—

Adding Cell Source’s special class of immune cell, Veto T-cells, to blood stem cells from an unrelated donor led to a successful transplant and to the production of normal red blood cells in a mouse model of sickle cell disease (SCD), the company announced. The preclinical Cell Source-sponsored…

A recent manufacturing agreement between Aruvant Sciences and Lonza will advance the development of ARU-1801, an investigational gene therapy for the treatment of sickle cell disease (SCD). Under the agreement, Lonza will be responsible for producing Aruvant’s gene therapy for an upcoming pivotal clinical trial. The company already has…

A clinical trial will assess a combination of MGTA-145 plus plerixafor as a new regimen for aiding in the collection of blood stem cells from adults and adolescents with sickle cell disease (SCD). The proof-of-concept trial, a collaboration between Magenta Therapeutics and Bluebird Bio, may establish this…

Treatment with Oxbryta (voxelotor) may be a feasible alternative to blood transfusions for sickle cell disease (SCD) patients who are experiencing respiratory distress and pneumonia related to COVID-19, a case report suggests. These findings are particularly important during the ongoing pandemic in the U.S., in which rising blood…

FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, is entering a Phase 1 clinical trial in healthy volunteers, the company announced. The trial (NCT04586985), not yet enrolling, aims to recruit 88 adults at its single Altasciences Clinical Kansas site. The…

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,…