Patricia Inácio, PhD, science writer —

A Phase 1/2 clinical trial evaluating investigational therapy CTX001 for severe sickle cell disease (SCD) has enrolled its first patient in the U.S., and treatment is expected to begin by mid-2019. The trial (NCT03745287), still recruiting participants in Nashville, Tennessee, is looking to enroll 45 adults, 18 to 35 years old,…

Sickle cell disease (SCD) patients were safely treated with a phosphodiesterase 9A (PDE9) inhibitor therapy called PF-04447943 with no serious side effects reported and possible evidence of fewer vaso-occlusion pain crises, a Phase 1b clinical trial reports. Safety and early signs of possible efficacy — including a drop in biomarkers linked…

The U.S. Food and Drug Administration (FDA) has awarded fast track status to investigational therapy CTX001 to treat sickle cell disease. A Phase 1/2 clinical trial (NCT03745287) is recruiting patients. CTX001 uses the CRISPR gene-editing technology to increase the production of fetal hemoglobin in patients’ red blood cells. Fetal hemoglobin is…

The amount of oxygen the blood loses when traveling through the brain could help identify children with sickle cell disease who are at risk of having a stroke, a study reports. Scientists call the measurement the oxygen extraction fraction because it refers to the percentage of oxygen that the brain…

Patients with sickle cell disease show the highest rates of readmission and hospitalization within 30 days of being discharged from an emergency department, a single-center study found. The study, “Patients Discharged From the Emergency Department After Referral for Hospitalist Admission,” was published in The American Journal of Managed…

Imara, a pharmaceutical company developing new therapies for sickle cell disease (SCD), has successfully dosed the first patient enrolled in its clinical trial evaluating escalating doses of IMR-687 in adult patients with SCD. The Phase 2 trial (NCT03401112) is currently recruiting participants and expects to enroll…

Hypersensitivity to allergens in children with sickle cell anemia may increase the risk of acute chest syndrome, a study suggests. The study “Aeroallergen sensitization predicts acute chest syndrome in children with sickle cell anaemia” was published in the British Journal of Haematology. Diagnosing asthma in children with sickle cell…

Bluebird bio announced encouraging interim results from the ongoing Phase 1 clinical trial testing its investigational gene therapy LentiGlobin for patients with severe sickle cell disease (SCD). The results were presented at the 59th Annual Meeting of the American Society of Hematology (ASH), held Dec. 9-12 in Atlanta,…

Following a review of studies published in the last decade, experts have updated their recommendations for managing a sickle cell disease-related eye complication known as sickle cell retinopathy (SCR). Their paper, “Sickle cell retinopathy: improving care with a multidisciplinary approach,” appeared in the Journal of Multidisciplinary Healthcare. SCR, a…

Sancilio Pharmaceuticals says its investigational therapy Altemia (SC411) achieved primary and secondary endpoints in a clinical trial with sickle cell patients 5 to 17 years old. Altemia is a combination of specific lipids designed to restore balance to the membrane of red blood cells that are damaged in sickle…