News

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…

It may be possible to collect nearly three times as many stem cells for gene therapy for sickle cell disease (SCD) with BioLineRx’s motixafortide than with standard plerixafor, and even more when it’s combined with natalizumab, a Phase 1 study shows. The proof-of-concept clinical trial (NCT05618301) is…

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…

In a Phase 1/2 clinical trial, the gene-editing stem cell therapy BEAM-101 worked as intended, and showed early signs of efficacy in people with severe sickle cell disease (SCD), according to new early data. Developer Beam Therapeutics will present the findings at the upcoming American Society of Hematology…

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…

The U.S. Food and Drug Administration has granted orphan drug status to Kind Pharmaceuticals’ investigational oral small molecule AND017 to treat sickle cell disease (SCD). This designation is granted to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides incentives like…

Editas Medicine has announced proof-of-concept data for a new gene-editing approach for sickle cell disease (SCD) that does not involve a stem cell transplant. The strategy instead makes use of targeted lipid nanoparticles, or LNPs, which are tiny vesicles made of fatty molecules. They are used to deliver…

Patient enrollment has been completed for the Phase 3 RISE UP study, a clinical trial that’s assessing the safety and efficacy of the oral therapy mitapivat in people with sickle cell disease (SCD). More than 200 patients, 16 and older, have been enrolled, and results from the study…

Zydus Lifesciences is launching a proof-of-concept Phase 2a clinical trial to evaluate the safety and efficacy of its oral candidate desidustat in people with sickle cell disease (SCD). The study is the result of a collaboration with the Indian Council of Medical Research (ICMR). “Public-private partnerships in the…