The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, Aruvant’s experimental gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassemia. This designation means the company will be eligible to receive a priority review voucher…
Global Blood Therapeutics (GBT) and Syros Pharmaceuticals have teamed up to discover, develop, and market new therapies for sickle cell disease (SCD) and beta thalassemia. Under the collaboration agreement, Syros will be responsible for identifying new therapeutic targets and the discovery of drugs that stimulate the…
Sickle Cell Disease News brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to sickle cell disease (SCD) throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read…
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…
California’s legislature has committed $15 million toward sweeping enhancement of adult sickle cell disease (SCD) care in a state with historically underserved patients. The initiative is intended to improve care largely through creation of a statewide network of regional adult SCD clinics modeled after the newly opened clinic…
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…
Get regular updates to your inbox.