Boston Medical Center researchers say they have discovered a small molecule that increases fetal hemoglobin production in human blood stems cells which results in fewer sickled red blood cells and provides a potential new treatment for sickle cell disease. Sickle cell disease are relatively rare—affecting 100,000 people in the U.S.…

Sickle cell disease (SCD) will be the first focus of the U.S.’s new Cell and Gene Therapy (CGT) Access Model, which aims to increase access to cell and gene therapies for vulnerable populations. Gene therapies are some of the most expensive treatments in the world. The Centers for Medicare &…

In a landmark moment for sickle cell disease treatment and CRISPR, two gene therapies were approved for the condition by the U.S. FDA. One of them,Vertex and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]), is the first ever FDA approved CRISPR/Cas9 genome-edited cell therapy. Simultaneously, Bluebird Bio received the FDA OK…

Sickle cell disease (SCD) and beta thalassemia could be cured by gene therapy, but which approach would be most effective and safest? A team from St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard may have the answer. They compared five gene editing strategies in CD34+ hematopoietic stem…

Research, led by MIT, shows that a “spleen-on-a-chip” device can accurately model the human spleen and could be used to understand sickle cell disease better and help guide new therapy development. Sickle cell disease is an inherited blood disorder, thought to affect around 100,000 Americans. The condition manifests as abnormally…

Bluebird Bio said it has temporarily and voluntarily suspended two clinical trials assessing its LentiGlobin gene therapy for sickle cell disease (SCD; bb1111) following reports that two participants in the earlier-phase study developed blood cancers—one of acute myeloid leukemia (AML), the other of myelodysplastic syndrome (MDS). Bluebird said it temporarily…

Psychological stress has adverse effects on various human diseases, including those of the cardiovascular system. However, the mechanisms in which stress causes disease activity remains unclear. Using vaso-occlusive episodes (VOEs) of sickle cell disease (SCD) as a vascular disease model, researchers at Albert Einstein College of Medicine discovered that the…

Rockland Immunochemicals received a $224,473 SBIR grant from the NIH's National Heart Lung and Blood Institute to develop an antibody-based point-of-care device that can diagnose sickle cell disease. Rockland says it secured the award by demonstrating the technology's ability to develop and produce life science tools for basic and clinical…

Researchers from the Broad Institute and St. Jude Children’s Research Hospital have corrected the sickle-cell disease mutation in mice and in a human cell line using a bespoke adenine base editor, achieving phenotypic rescue with a high allele conversion rate. Conversion of the pathogenic sickle-cell disease (SCD) allele (HBB-S) to…

The CRISPR-Cas9 gene-edited therapy CTX001 has shown a consistent and sustained positive response in 10 patients treated for a pair of blood disorders—sickle-cell disease (SCD) and beta thalassemia—reported CRISPR Therapeutics and Vertex Pharmaceuticals the developers of the therapy. These are the first clinical studies of a CRISPR gene-editing candidate sponsored…

In a field bubbling with preclinical data and ideas on complex genome edits—including rewriting chunks of DNA—Beam Therapeutics has shown that just one nucleotide change can change an entire life. At the 66th Annual Meeting and Exposition for the American Society of Hematology (ASH) in San Diego, California, Beam Therapeutics…

Gene therapies received back-to-back sickle cell disease (SCD) approvals in December 2023, but uptake has been slow largely because of cost: Bluebird bio’s Lyfgenia costs $3.1 million, and Vertex’s and CRISPR Therapeutics’ partnered Casgevy has a list price of $2.2 million. Both gene therapies are one-time infusions meant to cure…

A novel technique could potentially eradicate the toxic conditioning process required to effectively transplant hematopoietic stem and progenitor cells (HSPCs), which play a crucial role in treating blood disorders like sickle cell disease and β-thalassemia. Researchers from Stanford University and the University of California, San Francisco (UCSF) utilized CRISPR-Cas9 to…

Over the last thirty years, protein-based injections and DNA therapeutics accounted for the majority of newly introduced treatments. However, these medicines remain out of reach for many around the world due to high pricing, with some therapies ranging from thousands to several millions of dollars to purchase. [caption id=”attachment_147257″ align=”alignright”…

In a big first, Vertex Pharmaceuticals and CRISPR Therapeutics announced today that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for Casgevy (exagamglogene autotemcel [exa-cel]) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Casgevy is a CRISPR/Cas9 gene editing…