Marisa Wexler, MS,  senior science writer—

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…

A Phase 1b clinical trial testing the safety and pharmacological properties of the investigational oral medication FTX-6058 in people with sickle cell disease (SCD) is expected to continue enrolling patients through 2023, according to Fulcrum Therapeutics, the therapy’s developer. “We believe we are well positioned, with a…

A gene editing platform developed by Cellectis called TALEN was able to restore healthy hemoglobin production in preclinical models of sickle cell disease (SCD). That’s according to data presented by Arianna Moiani, PhD, senior scientist and team leader of Innovation Gene Therapy at Cellectis, at the 29th Congress of…

Forma Therapeutics, which is currently developing an experimental treatment for sickle cell disease (SCD) called etavopivat, is being acquired by Novo Nordisk, the two companies announced. “Novo Nordisk has worked for more than 40 years to develop and deliver transformative medicines to patients around the world with…