Gene Therapy Also Effective in Younger Children With Inherited Blood Disorders

Vertex’s gene therapy shows promise in younger children with blood disorders

The company will begin filing to global regulators in the first half of 2026. Read more at straitstimes.com.

Gene Therapy Also Effective in Younger Children With Inherited Blood Disorders

(MedPage Today) -- ORLANDO -- The gene therapy exagamglogene autotemcel (exa-cel; Casgevy) -- already approved for patients 12 years and older with transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD) -- led to durable...

Vertex CRISPR therapy hits early goal in children with blood disorders

Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake.

Vertex's gene therapy shows promise in younger children with blood disorders

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Vertex’s gene therapy shows promise in younger children with blood disorders - Regional Media News

Dec 6 (Reuters) - Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease to be free of painful events and allowed those with another blood disorder that requires frequent blood transfusions to be transfusion-free for at least 12 consecutive months. This supports the potential for the therapy to treat the blood disorders in a younger patient population and will help Vertex exp…