Marisa Wexler, MS,  senior science writer—

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…

The gene editing therapy EDIT-301 appears to be working as intended in the first person with sickle cell disease (SCD) who received the experimental treatment, according to recent data from the Phase 1/2 RUBY clinical trial. Editas Medicine, which is developing EDIT-301, also announced that the U.S. Food…

Note: This story has been updated July 19, 2022, to correct that voxelotor is approved in the U.S. to treat sickle cell in patients ages 4 and older. Global Blood Therapeutics, known as GBT, has initiated an expanded access program to make voxelotor, its daily oral therapy…

Two mouse models commonly used to study sickle cell disease (SCD) are not suitable for testing certain gene editing treatment approaches, including those for fetal hemoglobin production, because of their specific genetic makeup, a study reported. “This work provides a reminder for scientists to carefully consider the genetics of…

A novel strategy called Engineered Stem Cell Antibody Paired Evasion (ESCAPE) could be used to more safely prepare people with sickle cell disease (SCD) for experimental gene-editing approaches for treating the inherited blood disorder, according to researchers. ESCAPE may help patients ready for a bone marrow transplant, a key…

Global Blood Therapeutics (GBT) has begun a Phase 2/3 trial to study the safety, tolerability, effectiveness, and pharmacological properties of its experimental oral therapy GBT601 in people with sickle cell disease (SCD). Initial data from the study (NCT05431088) are expected before the end of the year, according…

Aflac, a U.S. supplemental health insurance company, has given $100,000 to Children’s National Hospital in Washington, D.C., to support research into sickle cell disease (SCD) and its treatments. The donation was made to honor World Sickle Cell Awareness Day, observed each June 19. “We are honored for Aflac’s partnership to…

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to inclacumab and GBT601, two experimental therapies for sickle cell disease (SCD) being developed by Global Blood Therapeutics (GBT). These designations confer benefits to therapy developers that are designed to speed…

Hemex Health’s portable point-of-care device, Gazelle, is being enhanced to more precisely monitor fetal hemoglobin levels. According to Hemex, this could be useful for monitoring people with sickle cell disease (SCD) who are being treated with hydroxyurea. The company announced the software upgrade on May 8…