Marisa Wexler, MS, senior science writer —

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on FTX-6058, an investigational oral treatment for sickle cell disease (SCD) that’s being developed by Fulcrum Therapeutics. With the hold lifted, Fulcrum is planning to resume enrolling patients in a Phase 1b clinical trial…

A gene editing technology called adenosine base editing (ABE), wherein a single DNA building block is changed to increase fetal hemoglobin, may be more useful for treating sickle cell disease (SCD) than other gene editing techniques, a study suggests. “We showed that not all genetic approaches are equal. Base…

The fourth annual Cheek Week, an effort to include more blood stem cell donors in the Be The Match Registry will be featured as part of July’s African American Bone Marrow Awareness Month. The registry seeks to identify people who might be candidates to donate blood stem cells, which…

The U.S. Food and Drug Administration (FDA) is expected to decide before year’s end on whether or not to approve the experimental gene therapy lovotibeglogene autotemcel — known as lovo-cel — for people with sickle cell disease (SCD) who have a history of painful vaso-occlusive crises (VOCs). The…

The Committee for Medicinal Products for Human Use (CHMP) is recommending the conditional approval of Adakveo (crizanlizumab) for sickle cell disease (SCD) be revoked in Europe. According to the therapy’s developer Novartis, a final decision from the European Commission is expected in about two months. In the…

The 5th annual Kier’s Hope 5K Run and Fun Walk — a fundraiser launched by comedian Kier “Junior” Spates to support people with sickle cell disease (SCD) — is set to go this Saturday, June 3, in Dallas. It’s also Kier’s Hope Day in the City of Dallas.

Hillhurst Biopharmaceuticals announced that the final healthy volunteer has completed the study protocol in a Phase 1 clinical trial testing HBI-002, an oral carbon monoxide treatment being developed for sickle cell disease (SCD). Data are now being analyzed, and Hillhurst expects to announce top-line results before the end…

A Phase 1/2 clinical trial testing Beam Therapeutics’ gene-editing therapy BEAM-101 is continuing to enroll young adults with sickle cell disease (SCD). The study, called BEACON (NCT05456880), is active at five sites, with a waiting list for enrollment, according to Beam, which is sponsoring the trial.

Kier “Junior” Spates is hosting a new podcast to empower and educate people living with sickle cell disease. Called “Living Your Life,” the show can be found on YouTube or on the podcast website. The first episode, which premieres April 18, features comedian Nathaniel Stroman, better known as…

Health Canada has approved an extended-release formulation of Ferriprox (deferiprone) to treat iron overload in people with sickle cell disease (SCD) and other blood disorders. The key benefit of the new formulation, according to the therapy’s maker, Chiesi Global Rare Diseases, is that it reduces the medication’s dosing…