People with severe sickle cell disease (SCD) have reported significant improvements in their quality of life after receiving the approved gene-editing therapy Casgevy (exagamglogene autotemcel). That’s according to a new analysis of data from patients involved in a clinical trial that had tested the SCD treatment in individuals…
Correctsequence Therapeutics’ experimental gene-editing therapy CS-101 has been used to treat a person with sickle cell disease (SCD) for the first time. Prior to treatment with CS-101, the patient — a 21-year-old woman from Nigeria — had been experiencing frequent vaso-occlusive crises (VOCs), or painful episodes caused by…
Fundraising walks, a golf tournament, and a virtual blood drive are among activities planned to raise awareness of sickle cell disease (SCD) in September, National Sickle Cell Awareness Month. The campaign aims to raise funding for research into the inherited blood disorder and to support the approximately 100,000 U.S. residents…
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…
Pfizer’s investigational therapy inclacumab failed to outperform a placebo in reducing the rate of vaso-occlusive crises — episodes marked by painful inflammatory responses — in people with sickle cell disease (SCD), according to new data from the Phase 3 THRIVE-131 study. “We recognize this news is disappointing…
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…
People with sickle cell disease (SCD) may have a higher risk of developing blood cancer, but a lower risk of certain types of solid tumor malignancies, such as breast and prostate cancer, a systematic review study suggests. The study, “Risk of cancer in patients with thalassemia…
Children with sickle cell disease (SCD) who have sleep problems, such as sustained low blood oxygen during sleep, or nocturnal hypoxemia, require more healthcare resources than children with normal sleep parameters, a study reports. “These findings suggest that targeting nocturnal hypoxemia and related sleep disturbances … through appropriate screening…
Pociredir, an investigational oral therapy for sickle cell disease (SCD), was well tolerated and showed signs of promising activity in a group of patients treated with the 12  mg dose in a Phase 1b clinical trial, its developer Fulcrum Therapeutics announced. The ongoing trial, called PIONEER (NCT05169580), aims to…
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