Researchers have developed a new way of delivering gene-editing therapies for people with blood disorders such as sickle cell disease (SCD), which could improve treatment safety and lower costs if eventually adopted in the clinic. Instead of a standard method that requires a round of chemotherapy before externally-edited cells…
The European Commission has decided to revoke its conditional approval of Adakveo (crizanlizumab) for sickle cell disease (SCD) — meaning its developer, Novartis, will have to remove the antibody-based therapy from the market in all 27 countries that are part of the European Union. The decision came after…
A gene editing technology called adenosine base editing (ABE), wherein a single DNA building block is changed to increase fetal hemoglobin, may be more useful for treating sickle cell disease (SCD) than other gene editing techniques, a study suggests. “We showed that not all genetic approaches are equal. Base…
Pfizer has decided to end a treatment discovery program with Syros Pharmaceuticals that it inherited after acquiring Global Blood Therapeutics (GBT) last year, leaving Syros to seek another partner for the program, which targets two blood disorders, including sickle cell disease (SCD). The agreement termination was announced…
Genetic medicines company Scribe Therapeutics has expanded its collaboration with Sanofi to advance the development of a new wave of in vivo treatments for sickle cell disease (SCD) and other disorders. The collaboration leverages Scribe’s proprietary CRISPR X-Editing technologies and Sanofi’s targeted non-viral delivery technologies for the…
Researchers at Rice University in Texas have been awarded $2.6 million to further investigate questions surrounding the safety and efficacy of gene editing as a potentially curative treatment for sickle cell disease (SCD). The four-year grant from the National Institutes of Health (NIH) was awarded to Gang Bao, PhD,…
Sickle Cell 101 is teaming up this summer with Cleveland Browns player Donovan Peoples-Jones — known to fans as DPJ — and his Limitless Foundation Football Camp to raise awareness about sickle cell trait and hydration, both key issues for people living with disorders like sickle cell disease…
The Oman Ministry of Health has approved Endari (oral L-glutamine) for children, ages 5 and older, and adults with sickle cell disease (SCD), its developer Emmaus Life Sciences has announced. The disease affects around 3,000 people in Oman, with about 6% of the population estimated to carry…
The fourth annual Cheek Week, an effort to include more blood stem cell donors in the Be The Match Registry will be featured as part of July’s African American Bone Marrow Awareness Month. The registry seeks to identify people who might be candidates to donate blood stem cells, which…
For nearly half of people with sickle cell disease who took part in the Phase 2 part of a clinical trial called RISE UP, treatment with the oral medication mitapivat brought about a significant increase in levels of hemoglobin — the oxygen-carrying protein in red blood cells — new…
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