Marisa Wexler, MS, senior science writer —

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…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on FTX-6058, an experimental therapy that Fulcrum Therapeutics is developing to treat sickle cell disease (SCD). Fulcrum announced it would be stopping dosing in an ongoing Phase 1b clinical trial (NCT05169580) that’s testing different…

The risk of pregnancy-related death is about 26 times higher among people with sickle cell disease (SCD) in the U.S. compared with the national average, a recent study reported. The rate of pregnancy-related complications among SCD patients has not improved in recent decades, the results indicate. “People with sickle…

We at Sickle Cell Anemia News have brought you coverage of the latest developments in research and advances in treatment related to sickle cell disease (SCD) in 2022. We look forward to continuing being a resource for the sickle cell community in 2023. Here we’ve compiled a list of the…

New preclinical data support the ability of the ESCAPE platform, an approach Beam Therapeutics is advancing, to more safely prepare people with sickle cell disease (SCD) for gene-editing treatments now in development. Notably, these studies represent the first in vivo data for ESCAPE — that is, the experimental platform’s first evaluation in living…

The experimental gene-editing therapy EDIT-301 appears to be working largely as expected in the first two sickle cell disease (SCD) patients treated as part of the Phase 1/2 RUBY trial, according to new data shared by the therapy’s developer, Editas Medicine. “These promising clinical results from the RUBY…

The Qatar Ministry of Public Health has approved Endari (L-glutamine) to treat adults and children, starting at age 5, with sickle cell disease (SCD), the therapy’s developer, Emmaus Life Sciences, announced. “We are grateful that Qatari officials made access to Endari easier for the sickle cell patients…