Marisa Wexler, MS,  senior science writer—

Most sickle cell disease (SCD) patients treated with the gene therapy Lyfgenia (lovotibeglogene autotemcel) in clinical trials were free from vaso-occlusive events (VOEs) after a median of three years of follow-up. That’s according to new data from Lyfgenia’s developer, Bluebird Bio, at the 65th American Society of Hematology…

Health regulators in the Kingdom of Bahrain have approved the gene-editing therapy Casgevy (exagamglogene autotemcel) to treat people with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). This decision makes Bahrain, a small island nation in the Persian Gulf, the second country to approve Casgevy. The therapy was…

The Phase 1/2 BEACON trial, which is testing the gene-editing cell therapy BEAM-101 in people with sickle cell disease (SCD), is expected to dose its first patient before the year’s end. According to Beam Therapeutics, which is developing the therapy for fetal hemoglobin production, the trial is…

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…