Steve Bryson, PhD,  science writer—

Adding cyclosporin H to a new lentivirus-delivered gene therapy with enhanced anti-sickling properties boosted the uptake of genetic material in blood stem cells from newborns at risk of sickle cell disease (SCD), a preclinical study reports. With more genetic material inside stem cells, the therapy generated therapeutic levels of…

Zydus Lifesciences is launching a proof-of-concept Phase 2a clinical trial to evaluate the safety and efficacy of its oral candidate desidustat in people with sickle cell disease (SCD). The study is the result of a collaboration with the Indian Council of Medical Research (ICMR). “Public-private partnerships in the…

Pfizer has voluntarily withdrawn Oxbryta (voxelotor), a conditionally approved oral therapy for sickle cell disease (SCD), from worldwide markets, saying clinical data shows the treatment’s benefit “no longer outweighs the risk.” Citing an imbalance in vaso-occlusive crises (VOCs) and deaths occurring in patients given Oxbryta in postmarketing…

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…

The U.S. Food and Drug Administration (FDA) gave the greenlight for Iomab-ACT, a potentially less toxic conditioning regimen, to be tested in people with sickle cell disease (SCD) undergoing a stem cell transplant. The study will be conducted in collaboration between Actinium Pharmaceuticals, which is developing Iomab-ACT,…

Hydroxyurea does not reduce ovarian reserve, or the quantity and quality of eggs in the ovaries, in girls and young women with sickle cell disease (SCD), according to a direct analysis of ovarian tissue. These findings suggest measures to preserve fertility may be unnecessary before hydroxyurea treatment. “This…

All 18 adults with severe sickle cell disease (SCD) treated with the gene-editing therapy renizgamglogene autogedtemcel — known as reni-cel — remained free of vaso-occlusive events, or VOEs, for up to nearly two years, according to new interim data from the Phase 1/2/3 RUBY clinical trial. The therapy, being…

Nearly all people with sickle cell disease (SCD) treated with the U.S.-approved gene-editing therapy Casgevy (exagamglogene autotemcel) were free of painful vaso-occlusive crises (VOCs) for at least one year, according to new data from a Phase 2/3 clinical trial. These updated results were presented at the annual…

Short-term exposure to air pollutants may be associated with a higher risk of hospitalization among children with sickle cell disease (SCD), according to a new study examining data from the southeastern U.S. state of South Carolina. In particular, more so-called hospital encounters were seen in SCD children within two…

A nurse training program at the University of Tennessee Health Science Center (UTHSC) is expanding to train more nurses to care for people with sickle cell disease (SCD). Called the Sickle Cell Boot Camp to Promote Nursing Excellence Nationally and Globally Utilizing a Train the Trainer Model,…