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…
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…
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…
In the years after undergoing a stem cell transplant, people with sickle cell disease (SCD) exhibit quality-of-life scores that are similar to norms for the general population, according to a recent analysis. “In addition to the well-documented survival benefits of [stem cell transplant] for SCD, this study describes…
Children with sickle cell disease (SCD) may be more likely than their peers to have attention-deficit hyperactivity disorder (ADHD), according to a new study that found that the prevalence of ADHD in SCD kids may be more than double the expected rate in the general population. Based on these…
Enrollment has been completed in the adolescent group of the Phase 1/2/3 RUBY clinical trial testing Editas Medicine’s gene-editing therapy, renizgamglogene autogedtemcel (reni-cel), in people with sickle cell disease (SCD). “I am proud of the Editas team’s work and our advancement in the first half of 2024 as…
A bone marrow transplant may help reverse heart damage in people with sickle cell disease (SCD), according to a study. The findings were published in a letter to the editor titled, “Impact of Hematopoietic Cell Transplantation on Myocardial Fibrosis in Young Patients with Sickle Cell Disease,” in…
A novel small molecule called SR-18292, which acts to boost the production of fetal hemoglobin, was shown to reduce disease severity in a mouse model of sickle cell disease (SCD) and demonstrated benefits in other lab testing in a new study. “We’ve uncovered a promising approach that can offer…
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