Marisa Wexler, MS, senior science writer —

New preclinical data support the ability of the ESCAPE platform, an approach Beam Therapeutics is advancing, to more safely prepare people with sickle cell disease (SCD) for gene-editing treatments now in development. Notably, these studies represent the first in vivo data for ESCAPE — that is, the experimental platform’s first evaluation in living…

The experimental gene-editing therapy EDIT-301 appears to be working largely as expected in the first two sickle cell disease (SCD) patients treated as part of the Phase 1/2 RUBY trial, according to new data shared by the therapy’s developer, Editas Medicine. “These promising clinical results from the RUBY…

The Qatar Ministry of Public Health has approved Endari (L-glutamine) to treat adults and children, starting at age 5, with sickle cell disease (SCD), the therapy’s developer, Emmaus Life Sciences, announced. “We are grateful that Qatari officials made access to Endari easier for the sickle cell patients…

A Phase 1b clinical trial testing the safety and pharmacological properties of the investigational oral medication FTX-6058 in people with sickle cell disease (SCD) is expected to continue enrolling patients through 2023, according to Fulcrum Therapeutics, the therapy’s developer. “We believe we are well positioned, with a…

A gene editing platform developed by Cellectis called TALEN was able to restore healthy hemoglobin production in preclinical models of sickle cell disease (SCD). That’s according to data presented by Arianna Moiani, PhD, senior scientist and team leader of Innovation Gene Therapy at Cellectis, at the 29th Congress of…

Forma Therapeutics, which is currently developing an experimental treatment for sickle cell disease (SCD) called etavopivat, is being acquired by Novo Nordisk, the two companies announced. “Novo Nordisk has worked for more than 40 years to develop and deliver transformative medicines to patients around the world with…

The National Institutes of Health (NIH) has awarded a five-year, $7.7-million grant to support a new research project that seeks to lower barriers to healthcare for people with sickle cell disease (SCD). The project, titled “Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle…

People with sickle cell anemia (SCA) — the most common and the most severe form of sickle cell disease (SCD) — are more commonly hospitalized in the cooler winter and spring seasons than in the hotter summer or fall, according to a study conducted in Brazil. Better understanding of…

Intravenous bisphosphonate therapy can ease bone pain in children with sickle cell disease (SCD), a new study reports. The study, “Sickle cell bone disease and response to intravenous bisphosphonates in children,” was published in the journal Osteoporosis International. Many people with SCD experience…

Children with sickle cell disease (SCD) exposed to higher doses of the oral therapy hydroxyurea report better blood-related clinical parameters than those with lower exposure, according to a new study from the U.S. The favorable outcomes were linked to higher adherence to treatment, suggesting that “adherence interventions have…