News

Fulcrum Therapeutics is planning to report, by midyear, new data from the ongoing PIONEER Phase 1b clinical trial that’s testing its oral therapy candidate pociredir (FTX-6058) in people with sickle cell disease (SCD), according to a new corporate presentation. That will include data from the study’s third-highest…

Texas Children’s Global HOPE and Baylor College of Medicine Global Health, collaborating with the independent charity Bristol Myers Squibb Foundation, have announced a pioneering program to combat sickle cell disease (SCD) in Sub-Saharan Africa. Launching first in Tanzania and Uganda, this program is intended to improve the survival and…

Orna Therapeutics is partnering with Vertex Pharmaceuticals to develop gene-editing therapies for people with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The three-year collaboration will leverage Orna’s proprietary lipid nanoparticle (LNP) delivery system. LNPs are tiny vesicles made up of fatty molecules that can be used…

The Bill and Melinda Gates Foundation is investing up to $50 million in Tessera Therapeutics to support the development of a new gene-editing medicine for sickle cell disease (SCD), the company said. “We look forward to advancing a genetic medicine that can potentially reshape the treatment landscape for…

A newborn screening program in Catalonia improved outcomes for children with sickle cell disease (SCD), with patients being diagnosed earlier and having lower rates of major disease complications. “The data presented justify screening in countries where it has not yet been implemented, as it reduces patient morbidity and…

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…