BEAM-101, a genetically modified cell therapy for sickle cell disease (SCD) that’s being tested in clinical trials, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The FDA gives the designation to experimental therapies designed to treat conditions that affect fewer than 200,000 people…
An algorithm that grew out of a collaboration between different specialties at the Medical University of South Carolina (MUSC) may help reduce unnecessary hospital admissions for children with sickle cell disease (SCD), according to a new study. “This algorithm safely allows our care of these patients to align with…
Bluebird Bio and Xcellbio have entered into an agreement to support the commercialization of Lyfgenia (lovotibeglogene autotemcel), Bluebird’s gene therapy for sickle cell disease, incorporating Xcellbio technology into Bluebird’s potency assay with the aim of helping to ensure the therapy’s effectiveness. “We’re humbled to play a small part in…
This year, for the first time, two teenagers in Florence, South Carolina, are going to be able to go swimming on spring break without worrying about serious complications from sickle cell disease (SCD). Ashanti and Dontrell Pickens have long wanted to swim on their school holidays, but were not…
This month, the University of Tennessee Health Science Center College of Nursing is hosting a conference that aims to educate primary care doctors about sickle cell disease (SCD). The Sickle Cell Disease Comprehensive Management for Health Care Providers conference is being funded by a two-year, $364,502 grant from the…
Scientists have discovered more than a dozen genes that regulate the production of fetal hemoglobin in people of African descent, which may be potential treatment targets for sickle cell disease (SCD). Their findings were reported in the study, “FLT1 and other candidate fetal haemoglobin modifying loci…
Undergoing a bone marrow transplant during adolescence may reduce the risk of stroke in people with sickle cell disease (SCD), while waiting until adulthood is unlikely to mitigate this risk, a mouse study suggests. “We saw that if you wait until after the vasculature is damaged to do this…
A sickle cell disease (SCD) patient in Bahrain received the gene-editing therapy Casgevy (exagamglogene autotemcel), becoming the first patient outside the U.S. to be treated with the drug. Bahrain approved Casgevy in late 2023, becoming the first country in the Middle East to approve the therapy and the…
Children’s Hospital Los Angeles (CHLA) is now offering Casgevy (exagamglogene autotemcel), an approved gene-editing therapy for sickle cell disease (SCD). “This is a transformative time for Children’s Hospital Los Angeles and the pediatric community we serve,” Alan S. Wayne, MD, pediatrician-in-chief at CHLA, said in a press…
The Bill and Melinda Gates Foundation is investing up to $50 million in Tessera Therapeutics to support the development of a new gene-editing medicine for sickle cell disease (SCD), the company said. “We look forward to advancing a genetic medicine that can potentially reshape the treatment landscape for…
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