A one-time CRISPR-based gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics now has an additional FDA approval as a treatment for the rare blood disease beta thalassemia. The ...

The gene-therapy-based treatment called Casgevy was recently approved in the UK, making it the first time that a treatment based on the CRISPR-Cas9 gene editing tool has been authorized for medical ...

CS-101 is Shanghai-based CorrectSequence's lead candidate, which it is developing for treating sickle-cell disease and ...

Britain has approved the first drug based on a novel gene-editing technology, authorizing Boston’s Vertex Pharmaceuticals and CRISPR Therapeutics to sell the medicine, called Casgevy, in the United ...

Gene therapies have been approved for certain patients with beta thalassemia. After a successful phase 3 readout, Agios hopes it can reach a unique subgroup and eventually all patients. Agios’ ...

Thalassemia is an inherited disease that affects the blood. The genetic features are present from birth. Children with severe thalassemia will require regular blood transfusions. Thalassemia causes ...

The lifelong effects of rare blood disorder diagnoses in childhood are being analysed in a first-of-its-kind study led by the ...

An essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires life-long blood transfusions for survival, non-transfusion-dependent ...

Patients with beta-thalassemia major (also known as Cooley's anemia) have a reduction or absence of beta protein in their hemoglobin, resulting in severe anemia, which requires regular blood ...