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

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 expand the use of Casgevy, which is currently approved for patients 12 years and older with sickle cell disease or transfusion-dependent beta thalassemia (TDT).

“These results — the first clinical data ever presented on any genetic therapy for children ages 5-11 years with SCD — again demonstrate the transformative potential of Casgevy,” said Vertex