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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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…
A camp in North Carolina offers children living with sickle cell disease the chance to build confidence, independence, and emotional strength while connecting with others who also live with a chronic blood disease. Each summer, Camp Hope provides kids with sickle cell to learn about themselves while participating…
The investigational gene-editing therapy OTQ923 resulted in normalization of the speed of blood flow in the brain of three adults with sickle cell disease (SCD), according to data from a Phase 1 clinical trial. Findings suggest the treatment could help lower the risk of stroke in patients. The…
The first healthy volunteer has been dosed in a Phase 1 clinical study of CLY-124 — a novel oral small molecule that Cellarity is developing to treat sickle cell disease — following clearance of the company’s investigational new drug application by the U.S. Food and Drug Administration. “Initiation…
Higher pain scores at the time of discharge from the emergency department (ED) may predict the likelihood of hospitalization in children with sickle cell disease (SCD) who seek emergency care for acute pain episodes, according to a study in the U.S. The study also found that patients with severe…
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