Marisa Wexler, MS, senior science writer —

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…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Endari, an oral therapy approved to treat sickle cell disease (SCD) has been added to the Florida Medicaid Preferred Drug List (PDL), its manufacturer Emmaus Life Sciences announced. The PDL, according to the Florida Agency for Health Care Administration (AHCA), is a listing of medications that are considered…

Siklos (hydroxyurea) is now approved in the U.S. to reduce the frequency of painful crises and lower the need for blood transfusions in adults with sickle cell anemia, the most common and usually the most severe form of sickle cell disease. The medication already was approved for…