CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are…
Graphite Bio, a startup biotech company, has raised $45 million in an initial financing round to advance its gene editing technology to treat sickle cell disease and other genetic conditions. The technology being developed at Graphite is designed to target a specific gene and repair it in living cells…
The Bill & Melinda Gates Foundation has awarded a $3.3 million grant to GreenLight Biosciences to develop gene therapies revolving around messenger RNA (mRNA) — the intermediate molecule between DNA and proteins — for sickle cell disease (SCD)…
Using a newly developed assay, researchers identified seven U.S. Food and Drug Administration (FDA)-approved medications that may be effective, with minimal safety issues, in treating sickle cell disease (SCD). Their findings were reported in the Journal of Clinical Medicine, in the study, “Large-Scale Drug Screen Identifies…
As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to bring attention to newborn testing. Baby’s First Test is a program of Expecting Health, an organization focused on pregnancy and newborn health. The…
Genetic analyses of African patients identified several genes and biological pathways that are associated with disease severity and longevity, and that might serve as new therapeutic targets in sickle cell disease (SCD). The study, “Genetic modifiers of long‐term survival in sickle cell anemia,” was published in…
Microbes in the gut play a key role in inducing vaso-occlusive crises (VOCs) in response to stress, a study in mice suggests. Stress is known to trigger VOCs in people with sickle cell disease (SCD) and inflammation in those with cardiovascular disease, but in ways not fully understood.
The SECURE-SCD registry, which is collecting de-identified data on individuals with sickle cell disease (SCD) who develop COVID-19, will be expanding to include information on children and adults in Africa. The expansion was funded by a $100,000 grant from the Doris Duke Charitable Foundation (DDCF) awarded to…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…
September is National Sickle Cell Awareness Month, set aside annually to bring attention to the blood disorder and the needs of the community. From securing local government proclamations and hosting awareness events, to distributing information and lighting public spaces red, supporters have begun observing the monthlong event. The theme…
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