Steve Bryson, PhD, science writer —

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…

Exagamglogene autotemcel (exa-cel), an experimental gene-editing cell therapy, increased fetal hemoglobin levels and prevented vaso-occlusive crises (VOCs) in 31 people with severe sickle cell disease (SCD), according to new data from the CLIMB-SCD-121 trial. Vertex Pharmaceuticals and CRISPR Therapeutics, which are jointly developing the therapy, recently launched…

Graphite Bio’s GPH101, an investigational gene-editing therapy that aims to correct the genetic mutation that causes sickle cell disease (SCD) and potentially cure the condition, has been awarded fast track designation by the U.S. Food and Drug Administration (FDA). The fast track process is designed to accelerate the…

Disrupting a specific area of the gene that encodes for adult hemoglobin — which is faulty in people with sickle cell disease (SCD) — led to an increase in the production of fetal hemoglobin, a version of the protein normally produced during fetal development, which can help relieve disease…

A naturally-occurring protein called hemopexin, also an investigational therapy for acute vaso-occlusive crises (VOCs) in sickle cell disease (SCD), safely reduced the signs of such crises in cell and animal models, a study has demonstrated. These findings support an ongoing Phase 1 trial (NCT04285827), sponsored by the…

The United Arab Emirates (UAE) Ministry of Health has approved Endari (L-glutamine) for sickle cell disease (SCD), according to Emmaus Life Sciences, the therapy’s developer. During the five-month review process, which was originally expected to take up to one year, the oral therapy was available by early…

Dosing will soon start in a Phase 1/2 trial of GPH101, an experimental gene-editing therapy designed to correct the genetic mutation that causes sickle cell disease (SCD). The first participant had been enrolled in the study late last year, but due to the recent surge of the SARS-CoV-2…