At age 45, Dr. Lakiea Bailey said, for the longest time, that she was the oldest person with sickle cell anemia that she knew. The executive director of the nonprofit patient advocacy group the Sickle ...

The U.S. Food and Drug Administration is expected to approve exa-cel gene-editing treatment for sickle cell disease. Exa-cel would be the first approved medicine in the U.S. to use CRISPR gene-editing ...

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta ...

Please provide your email address to receive an email when new articles are posted on . Exagamglogene autotemcel helped patients with beta-thalassemia achieve transfusion independence. The drug also ...

As Vertex Therapeutics nears completion of a historic FDA submission, the rare disease specialist has depicted a rosy launch picture for what could become the first CRISPR-based gene editing therapy.

Data from the study showed that exa-cel prevented vaso-occlusive crises in all but 1 of the 30 evaluable patients for at least 12 months. Exagamglogene autotemcel (exa-cel; Casgevy) prevented ...