Patricia Inácio, PhD,  science writer—

Oryzon Genomics said it received European Medicines Agency approval to start a Phase 1b clinical trial of iadademstat in people with sickle cell disease (SCD). The trial — RESTORE (2025-521838-29-00), short for REgulation of Sickling ThrOugh Reprogramming Epigenetics — will enroll 40 SCD patients, ages 18 and…

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to Beam Therapeutic’s BEAM-101, a one-time, gene-edited cell therapy for severe sickle cell disease (SCD). RMAT status is granted to therapies that have shown potential for treating serious or life-threatening conditions. It provides a…

Women with sickle cell disease (SCD) experience nearly double the rate of acute pain associated with the disease in the third trimester of pregnancy and in the first weeks following childbirth than in the 6- to 9-month period after childbirth. That’s according to a single-center study from Ghana, which…

Zynext Ventures is investing in Illexcor Therapeutics to advance the clinical development of ILX002, its lead, novel oral candidate therapy for sickle cell disease (SCD), the companies announced. The financial details of the investment by Zynext Ventures, the venture capital arm of Zydus Lifesciences, were not disclosed.

Despite advancements in medical care, pregnant women with sickle cell disease (SCD) are at a higher risk of complications and require close monitoring, a nationwide French study has found. SCD is associated with a significantly higher likelihood of maternal and fetal complications, preterm birth, and caesarean sections (C-sections), data…

The incidence of vaso-occlusive episodes and acute chest syndrome in children with sickle cell disease (SCD) is elevated during January to March, a large nationwide, retrospective study in the U.S. suggests. The findings reinforce the need for “recommendations for patients with SCD to take appropriate precautions in cold weather,”…

Texas Children’s Global HOPE and Baylor College of Medicine Global Health, collaborating with the independent charity Bristol Myers Squibb Foundation, have announced a pioneering program to combat sickle cell disease (SCD) in Sub-Saharan Africa. Launching first in Tanzania and Uganda, this program is intended to improve the survival and…

Mitem Pharma has acquired worldwide rights to Desferal (deferoxamine), a therapy commonly used to lower excessive and damaging iron levels induced by blood transfusions in people with sickle cell disease (SCD) or other anemias. The acquisition was reached in an agreement between Mitem and Novartis, which…

The NewYork-Presbyterian Dalio Center for Health Justice is using basketball to raise awareness about sickle cell disease (SCD). The Sickle Cell Awareness Ball campaign features a special-edition basketball with one crescent-shaped red panel, representing the cell that’s a hallmark feature of the disease. The black-and-red ball is featured…

Supplementing with vitamin D for six months safely improved bone health and functional capacity in children and adolescents with sickle cell disease (SCD), a study finds. More studies are required to assess the “optimal dose, duration of supplementation, long-term [adverse events], and efficacy in different types of SCD, wrote…