News

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…

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

Ghana is working with Novartis to make hydroxyurea treatment available to people with sickle cell disease, and to open a clinical trial in the African country testing the company’s biologic SCD therapy Adakveo (crizanlizumab), recently approved in United States. The five-year, public-private partnership was formed in January…

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…

By 2029, Americans with sickle cell disease (SCD) could be living 10 years longer than they do today, predicts U.S. Health and Human Services Secretary Alex Azar. That’s one of the Trump administration’s long-term goals, Azar — the nation’s top health official — told more than 900 delegates attending…

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…

Oxbryta (voxelotor), a first sickle cell treatment that works to stop the sickling and destruction of red blood cells that mark this disease, has been given accelerated approval by the U.S. Food and Drug Administration (FDA) for patients ages 12 and older. Specifically, Oxbryta — an oral,…

Droplets packed with hemoglobin may briefly form tiny clusters inside red blood cells that ultimately deform their shape, and cause the pain crises that mark sickle cell disease (SCD), a study reports. The research, “A mechanism for reversible mesoscopic aggregation in liquid solutions,” appeared in the journal …

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…

CTX001 safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crises in the first severe sickle cell disease (SCD) patient receiving the therapy, according to preliminary data from a Phase 1/2 clinical trial. CTX001 is a CRISPR-based gene editing therapy developed by CRISPR Therapeutics and Vertex…