Steve Bryson, PhD, science writer —

A Sickle Cell Carrier Status Awareness program has been launched to help identify people who carry genetic variants associated with sickle cell disease (SCD). Established through a collaboration between the Sickle Cell Foundation of Georgia (SCFG), the Morehouse School of Medicine, and the human genetic testing company 23andMe,…

Bluebird Bio has provided the U.S. Food and Drug Administration (FDA) with information regarding the commercial manufacturing of its experimental gene therapy for sickle cell disease (SCD) in preparation for requesting its approval. The company had expected to submit a biologics license application seeking the approval of…

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…