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…
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…
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