Joana Carvalho, PhD, managing science editor —

The National Institutes of Health (NIH) has awarded a grant to researchers at Carnegie Mellon University’s College of Engineering to support their project to develop a non-invasive, non-toxic and non-addictive pain treatment for people with sickle cell disease (SCD). “There is an urgent unmet need to…

LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), continues to show promising results in SCD patients participating in the company’s Phase 1/2 HGB-206 clinical trial, according to the latest study data. The new findings — which included data from additional patients treated in the…

A Phase 3 trial is testing a higher dose of Adakveo (crizanlizumab), the first approved targeted treatment for sickle cell disease, to explore if that dose might be more effective — while still safe — in easing the painful vaso-occlusive crises (VOCs) patients can have each…

Oxbryta (voxelotor), the first treatment for sickle cell disease that addresses a root cause, will continue to be tested in a post-approval confirmatory study opening soon in children as young as age 2, according to its developer, Global Blood Therapeutics (GBT). The new study, called HOPE-KIDS 2, is…

Adakveo (crizanlizumab) is an “important addition” to the care of sickle cell disease, a safe and effective therapy that works against painful vaso-occlusive crises (VOCs) in SCD patients and is likely do so over the long term, said the doctor who led the clinical trial that…

A first targeted treatment for sickle cell disease (SCD) — Adakveo (crizanlizumab), by Novartis, that works to lower the frequency of the painful vaso-occlusive crises (VOCs) common in this disease — has been approved by the U.S. Food and Drug Administration (FDA). Adakveo…

Scientists have found a way to increase the effectiveness of the gene-editing tool CRISPR/Cas9 that one day may be used to treat people with sickle cell disease (SCD) and HIV. The findings were reported in the study, “Improved Cas9 activity by specific modifications of the tracrRNA,”…

A technique called zinc finger nuclease (ZFN) gene editing technology can be used to modify immature red blood cells — called precursor cells — to  boost the production of fetal hemoglobin and help ensure red blood cells maintain a normal shape in people with sickle cell disease (SCD),…

The prenatal test UNITY, which uses maternal blood samples, is effective in screening for sickle cell disease (SCD), cystic fibrosis (CF), spinal muscular atrophy (SMA), and thalassemias, a study has found. The study, “A novel high-throughput molecular counting method with single base-pair resolution enables accurate…

The U.S. Food and Drug Administration (FDA) is giving priority review, as requested, to Global Blood Therapeutics‘ application to approve voxelotor as a treatment for sickle cell disease (SCD). Voxelotor, formerly known as GBT440, is as an oral and once daily potential sickle cell therapy. It works by…