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Casgevy

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Ex-vivo CRISPR gene editing modifies a patient’s cells outside the body before reinfusion, while in-vivo CRISPR delivers gene-editing machinery directly into the patient, highlighting the trade-off between potentially simpler treatment and the harder challenge of controlling where and how editing occurs. Representative image.
Features & Analysis

The liver was the easy organ. Can in-vivo gene editing reach the rest of the human body?

Ex-vivo CRISPR edits cells outside the body before reinfusion. In-vivo editing sends the editing machinery directly into a patient, potentially making treatment simpler but far harder to control.

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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

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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Representative image of non-viral gene therapy research, showing how lipid nanoparticles, CRISPR-based editing and advanced delivery platforms are gaining attention as viral vector safety concerns reshape genetic medicine.
Features & Analysis

Can non-viral gene therapies solve the safety problem holding back genetic medicine?

Viral vectors built gene therapy’s first era. Safety, redosing and manufacturing pressure are pushing non-viral platforms into the spotlight.

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