An experimental, single-dose gene-editing therapy from Tessera Therapeutics successfully corrected the genetic mutation that causes sickle cell disease (SCD) in the majority of blood-forming stem cells in nonhuman primates, reaching levels that surpass what is expected to cure the disease. The new preclinical data were presented by Tessera…
News
Nearly every person with sickle cell disease (SCD) who underwent a new regimen for stem cell transplant, developed by doctors at Johns Hopkins in the U.S., is alive and has been free of disease activity in the years following the procedure, a study reports. “The survival rate…
Agios Pharmaceuticals has filed an application with the U.S. Food and Drug Administration (FDA) seeking accelerated approval of mitapivat, its oral therapy for sickle cell disease (SCD). The developer said it expects to receive notice of acceptance of the application and the anticipated review timeline following the FDA’s…
Newer oral anticoagulants, or blood thinners, were linked to a lower risk of clinically relevant nonmajor bleeding than warfarin, an older blood thinner, in adults with sickle cell disease (SCD) treated for dangerous blood clots, according to a study led by researchers in Saudi Arabia. However, no significant differences…
Casgevy (exagamglogene autotemcel) will be reimbursed by Germany’s public health system, enabling people with severe sickle cell disease (SCD) in the country, ages 12 and older, to access the one-time gene-editing therapy at low or no cost. The reimbursement agreement, established with Vertex Pharmaceuticals — which developed…
Sickle cell disease (SCD) hospitalization patterns vary across New York State, with New York City showing the largest share of hospitalizations and Long Island reporting the highest charges for care, a recent study showed. In addition, longer hospital stays were seen in regions where access to specialized care may…
A discovery about red blood cells could have implications for sickle cell disease (SCD) and other blood disorders marked by problems with hemoglobin, the protein red blood cells use to carry oxygen through the bloodstream. Scientists found that red blood cells growing under stressful conditions can import heme, an…
Women with sickle cell anemia (SCA), the most common and severe form of sickle cell disease (SCD), have significantly lower ovarian reserve relative to women without SCA and with proven fertility, a study in Nigeria shows. Ovarian reserve is a marker-based estimate of the remaining supply of eggs in the…
One of the $3 million 2026 Breakthrough Prizes — in Life Sciences — has been awarded to two researchers whose discoveries reshaped the treatment of sickle cell disease (SCD). The award from the Breakthrough Prize Foundation, which counts its mission as “honoring scientists as heroes of our society,” is…
A global, late-stage clinical trial testing etavopivat in people with sickle cell disease (SCD) has hit its main goals, showing that the experimental oral therapy improved hemoglobin levels and reduced the risk of painful episodes compared with a placebo. Based on these positive results from the Phase 3…
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