News

The European Commission has granted orphan medicinal product status to mitapivat, Agios Pharmaceuticals’ oral investigational therapy for sickle cell disease (SCD). This designation is granted to treatments for rare diseases, or those affecting fewer than five in 10,000 people in the European Union, that have the potential…

Nearly all the patients with severe sickle cell disease (SCD) who were treated with renizgamglogene autogedtemcel (reni-cel) remained free of vaso-occlusive events (VOEs) for up to two years, new data from the Phase 1/2/3 RUBY  clinical trial shows. The treatment also increased total hemoglobin levels and was well tolerated,…

Etavopivat, Novo Nordisk’s investigational oral therapy for sickle cell disease (SCD), may reduce the incidence of vaso-occlusive crises (VOCs) and increase hemoglobin levels. That’s according to data from the phase 2 part of the Phase 2/3 HIBISCUS trial (NCT04624659), which is assessing the treatment’s safety and…

Most people with sickle cell disease (SCD) who received the gene-editing therapy Casgevy (exagamglogene autotemcel) in clinical trials have been free from vaso-occlusive crises (VOCs) over several years of follow-up, according to new data. Vertex Pharmaceuticals, which developed Casgevy alongside CRISPR Therapeutics, shared the findings…

The vast majority of people with sickle cell disease (SCD) who received the now-approved gene therapy Lyfgenia (lovotibeglogene autotemcel) in clinical trials have been free from vaso-occlusive events — simply called VOEs — in the years following treatment. That’s according to new data that Lyfgenia’s developer Bluebird…

Kanglin Biotechnology has raised $20 million in financing to advance the development of KL003, an experimental gene therapy for sickle cell disease (SCD) and beta thalassemia. The China-based company plans to launch a pivotal Phase 2 trial next year to test KL003 in people with beta thalassemia, a…

Two centers in California are enrolling nine people with severe sickle cell disease (SCD) for a clinical trial of a new gene editing therapy called CRISPR_SCD001. The Phase 1/2 trial (NCT04774536) is open to adolescents and adults, ages 12 through 35, who in the two years before enrollment…

Cognitive behavioral therapy (CBT) and pain education, each delivered using digital mobile systems and supported by a peer health coach, were equally effective at lessening the impact of sickle cell disease (SCD) pain, a study found. The U.S.-based clinical trial, CaRISMA (NCT04419168), involved more than 300 adults with…

Adding cyclosporin H to a new lentivirus-delivered gene therapy with enhanced anti-sickling properties boosted the uptake of genetic material in blood stem cells from newborns at risk of sickle cell disease (SCD), a preclinical study reports. With more genetic material inside stem cells, the therapy generated therapeutic levels of…

The Clinton Health Access Initiative (CHAI) is leading a project that aims to expand access to diagnostic tests and treatments for sickle cell disease (SCD). The project is being funded by a three-year, $8 million grant from Open Philanthropy, an advised fund of the Silicon Valley Community Foundation, the…