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Representative image: Vertex Pharmaceuticals’ expanded FDA approval for CASGEVY opens CRISPR gene therapy to children aged 2 and older with sickle cell disease and transfusion-dependent beta thalassemia.

Pharma & Biotech

Vertex expands CASGEVY to young children with sickle cell disease and beta thalassemia

by Pallavi Madhiraju
July 2, 2026
0 Comments

Explore how the FDA’s CASGEVY age expansion could reshape early gene therapy for sickle cell disease and beta thalassemia. Read more.

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POPULAR NEWS

Representative image of a clinician reviewing skeletal imaging, illustrating Mirum Pharmaceuticals and Incyte’s zilurgisertib Phase 2 progress in fibrodysplasia ossificans progressiva and the rare disease treatment questions facing regulators.

Pharma & Biotech

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Medical Devices & Diagnostics

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Niagen Bioscience’s NB4168 has secured U.S. FDA Rare Pediatric Disease and European orphan designations as the company advances the experimental ataxia telangiectasia therapy toward human trials. Representative image.

Pharma & Biotech

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