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Challenges and opportunities in delivering gene therapies for sickle cell disease and beta thalassemia
Challenges and opportunities in delivering gene therapies for sickle cell disease and beta thalassemia

The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers prepare to meet growing demand for gene therapies in the coming years. Read more

Medical News - Dec 07
Exa-cel shows complete success in early pediatric trials for sickle cell disease and beta-thalassemia
Exa-cel shows complete success in early pediatric trials for sickle cell disease and beta-thalassemia

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger than 12. Read more

Medical News - Dec 06