Publications by authors named "W Julian C Hobbs"
Article Synopsis
- Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy utilizing CRISPR-Cas9 gene editing to increase fetal hemoglobin production in patients with sickle cell disease.
- A phase 3 study involved 44 patients aged 12 to 35 with a history of severe vaso-occlusive crises; patients received edited stem cells after myeloablative conditioning.
- Results showed 97% of patients were free from vaso-occlusive crises and 100% avoided hospitalization for over 12 months, with a safety profile similar to standard treatments.
Article Synopsis
- Exagamglogene autotemcel (exa-cel) is a novel cell therapy using CRISPR-Cas9 gene editing to boost fetal hemoglobin production in patients with transfusion-dependent β-thalassemia.
- In a phase 3 study, 52 patients aged 12 to 35 underwent treatment with exa-cel after myeloablative conditioning, and 91% achieved transfusion independence for at least 12 months.
- The therapy showed promising results with high mean total and fetal hemoglobin levels, a favorable safety profile, and no serious adverse events like deaths or cancers reported during the follow-up period.
β-thalassemia is a monogenic disease that results in varying degrees of anemia. In the most severe form, known as transfusion-dependent β-thalassemia (TDT), the clinical hallmarks are ineffective erythropoiesis and a requirement of regular, life-long red blood cell transfusions, with the development of secondary clinical complications such as iron overload, end-organ damage, and a risk of early mortality. With the exception of allogeneic hematopoietic cell transplantation, current treatments for TDT address disease symptoms and not the underlying cause of disease.
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