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…
Women with sickle cell disease who undergo a stem cell transplant can become pregnant and give birth to healthy children, a new study shows. “The results of this study show that female [stem cell transplant] recipients can achieve successful and safe pregnancies,” Katja Sockel, MD, lead study author at University…
TALEN, a gene-editing technology being used by the clinical-stage biopharmaceutical company Cellectis to develop potentially life-changing therapies, was able to correct the mutation that causes sickle cell disease (SCD) in patient cells. That’s according to a new study published by researchers from the company, along with colleagues from…
Treatment with the gene-editing therapy Casgevy (exagamglogene autotemcel) substantially decreased the rates of painful vaso-occlusive crises (VOCs) among people with sickle cell disease taking part in a Phase 2/3 clinical trial. Full results from the trial, which supported the recent approvals of Casgevy in the U.S.,…
England’s National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending the country’s National Health Service (NHS) cover Pfizer’s Oxbryta (voxelotor) to treat sickle cell disease. “Today’s approval by NICE marks a significant milestone in sickle cell care, addressing decades of limited access to…
A nurse clinician-scientist and professor at the University of Toronto, in Canada, is being honored for her work in developing iCanCope, a mobile app that aims to help in sickle cell disease (SCD) pain management. Jennifer Stinson, PhD, was awarded the 2023 Barer-Flood Prize from the Canadian Institutes…
The American Red Cross and the Sickle Cell Disease Association of Illinois (SCDAI) recently joined a national distributor of propane, natural gas, and other energy-related products to host a blood donation event to raise awareness about sickle cell disease and encourage blood donations to support transfusions for sickle cell…
Bluebird Bio has struck a deal with Medicaid in the state of Michigan to allow eligible people with sickle cell disease access to the now-approved gene therapy Lyfgenia (lovotibeglogene autotemcel). The new deal will provide Lyfgenia through an outcomes-based agreement — a financial structure in…
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