Steve Bryson, PhD, science writer —

Jasper Therapeutics’ investigational conditioning therapy briquilimab safely improved the success of stem cell transplants for three people with sickle cell disease (SCD), early Phase 1/2 trial data suggest. Stem cell transplant success was observed in all three patients treated with briquilimab, plus a less-aggressive non-myeloablative conditioning regimen,…

Treatment with Adakveo (crizanlizumab) did not reduce the yearly occurrence of painful vaso-occlusive crises (VOCs) leading to a medical visit, as compared with a placebo, in people with sickle cell disease (SCD), according to preliminary data from an ongoing Phase 3 trial. The study, called STAND (NCT03814746), is…

An imbalance of microbes in the digestive tract was found to contribute to bone disease in a mouse model study of sickle cell disease. Researchers discovered that transplanting healthy gut microbes protected the animals against SCD-related bone loss by increasing the levels of a bone growth hormone, which rose in…

A lung ultrasound may help in diagnosing acute chest syndrome (ACS), a serious lung complication in sickle cell disease (SCD), a pooled analysis of studies suggests. In fact, according to researchers, a lung ultrasound may be a better option than repeated chest X-rays to support an ACS diagnosis. An…

Reduced dietary iron eased blood vessel blockages and lessened organ damage in a mouse model of sickle cell disease (SCD), a new U.S.-based study reported. Low iron also reduced gut inflammation in SCD mice and restored a healthy balance of gut microbes. “Our study demonstrates for the…

Recruitment is now underway in BEACON, a Phase 1/2 U.S. trial testing BEAM-101, a transplant-based gene-editing cell therapy for sickle cell disease (SCD). The interventional study, in which all participants will be given a single dose of the experimental treatment, is expected to enroll up to 15 adults with…

Researchers have developed a method to detect and measure large unintended DNA modifications created by CRISPR-Cas9 gene-editing methods currently being investigated as treatments for sickle cell disease (SCD). High levels of unintended gene modifications that occurred at selected target sites demonstrate the need for a more careful evaluation of…

Contraception practices and counseling for adolescent and young adult women with sickle cell disease (SCD) vary by the healthcare provider, especially among clinicians with more or less experience, a survey reported. Established clinicians were found to be more likely to provide contraceptive counseling than those still in training, by…

Oxbryta (voxelotor) has been granted marketing approval in Great Britain to treat hemolytic anemia in people ages 12 and older with sickle cell disease (SCD). Eligible patients may take the once-daily oral therapy alone or in combination with hydroxyurea, a standard SCD therapy. The approval was granted…

To mark World Sickle Cell Day, Marja Hurley, a University of Connecticut (UConn) physician-researcher, has been awarded nearly $3 million in research funding to investigate bone loss in people with sickle cell disease (SCD). A four-year $2.21 million endowment comes from the National Institutes of Health (NIH) to Hurley…