Marisa Wexler, MS,  senior science writer—

An advisory committee to the U.S. Food and Drug Administration (FDA) has completed its meeting on exa-cel — formally exagamglogene autotemcel — ahead of the agency’s approval decision on the gene editing therapy, expected next month. The meeting was scheduled as part of the FDA’s review of exa-cel, which is…

If the U.S. Food and Drug Administration (FDA) approves the experimental sickle cell disease (SCD) gene therapy lovotibeglogene autotemcel (lovo-cel), its developer Bluebird Bio plans to sell the voucher it will receive that can be redeemed to speed up the review of a future application of a different…

The number of red blood cells that produce the abnormal hemoglobin S (HbS), which characterizes sickle cell disease (SCD), is reduced by the experimental gene therapy BCH-BB694, as is their tendency to take a sickle-like shape. That’s according to single-cell data from seven patients in an ongoing Phase…

Point-of-care tests can be used to accurately diagnose sickle cell disease (SCD) in resource-limited settings such as sub-Saharan Africa, according to a recent study. “Based on our experience and this study, we support the use of these [point-of-care] tests as standalone diagnostic tests for the early diagnosis of SCD…

HPC International has released new editions of Hope & Destiny, a book series that aims to help people with sickle cell disease (SCD) navigate life with the disorder. “We’re excited to get the new Hope & Destiny editions into the hands of the patients who need them, furthering our goal to…

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…