News

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…

The first participant with sickle cell disease (SCD) has been dosed in a clinical trial of HBI-002, an experimental, low-dose, oral liquid formulation of carbon monoxide being developed by Hillhurst Biopharmaceuticals, which expects to report initial data by the end of this year. “Dosing the first subject…

A student at Kennesaw State University in Georgia is helping to develop a low-cost, light-based device to screen for stroke in children with sickle cell disease (SCD) by monitoring how well their blood flows and how much oxygen is carried to their brain. Aster Cheung is working with Paul…

The Centers for Medicare & Medicaid Services (CMS) has announced that 33 states, along with the District of Columbia and Puerto Rico, have agreed to participate in the Cell and Gene Therapy (CGT) Access Model, which is intended to boost access to cell and gene therapies for people with…

Acute chest syndrome (ACS), a form of lung injury associated with sickle cell disease (SCD), is driven by the abnormal activation of a part of the immune system known as the complement cascade, a study found. The researchers found elevated complement activation in SCD patients, especially in those…

Fentanyl may be a feasible, safe, and effective option when administered intranasally (into the nose), to manage acute moderate to severe pain episodes in adults with sickle cell disease (SCD), a study in Canada reports. For faster and non-invasive pain relief, it has been widely used in children with…