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 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 RUBYÂ 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…
A hospital with the University of Rochester’s Medical Center has received a multi-million-dollar grant to conduct a study comparing the efficacy of some available treatments for sickle cell disease (SCD), helping families to make better-informed treatment decisions. Particularly, the study by scientists with Golisano Children’s Hospital will compare…
AB Science said it received a notice of allowance from the European Patent Office for a patent covering the use of the experimental oral therapy masitinib to treat sickle cell disease (SCD). The decision was based on preclinical data demonstrating that masitinib was able to prevent vaso-occlusive crises…
A Nigerian hematologist will pursue a PhD in health policy at the University of Tennessee Health Science Center to help advance her work on programs for sickle cell disease (SCD) in her home country. Aisha Galadanci Akilu, MD, helped implement a newborn screening program in Kano City, Nigeria, which…
Sigma Gamma Rho Sorority, a national collegiate women’s organization, has committed to raising $2 million by 2028 to support St. Jude Children’s Research Hospital in its efforts to treat children with sickle cell disease (SCD), cancer, and other serious conditions at no cost to their families. The…
Reproductive health issues were found to be common after a stem cell transplant among young people with sickle cell disease (SCD), most of whom underwent the procedure at approximately 11 years of age, according to a recent analysis of STELLAR data. The observed reproductive health issues, which included abnormalities in…
The use of eculizumab, an immunosuppressive medication approved for several autoimmune diseases, was found to be safe and effective for treating hyperhemolysis syndrome or HHS — a serious complication of blood transfusions — in two pediatric patients with sickle cell disease (SCD), a study showed. HHS, which can be…
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