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Paul Healy: UK Expands Casgevy Access to Younger Children With Sickle Cell Disease and Beta-Thalassaemia
Paul Healy/LinkedIn
Oct 31, 2026, 15:41

Paul Healy: UK Expands Casgevy Access to Younger Children With Sickle Cell Disease and Beta-Thalassaemia

Paul Healy, Founder of PHARMeMED, Co-Founder and Partner at pharmaCV, shared a post on LinkedIn:

“The UK has lowered the minimum age for Casgevy from 12 to five, opening a gene-editing treatment option to younger children with severe sickle cell disease or transfusion-dependent beta-thalassaemia.

The MHRA granted the extension on 29 October 2026.

Casgevy (exagamglogene autotemcel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses CRISPR/Cas9 to edit a patient’s own blood-forming stem cells.

The edit increases production of foetal haemoglobin, which can prevent painful sickle cell crises or reduce dependence on blood transfusions.

The paediatric evidence is promising, but deserves careful reading.

In two ongoing Phase III studies involving children aged 5–11:

  • All eight evaluable children with sickle cell disease remained free of severe crises for at least 12 months.
  • All eight evaluable children with beta-thalassaemia achieved at least 12 months without transfusions.

A further three and seven children, respectively, had insufficient follow-up for those outcomes.

The studies were small and had no control groups.

Treatment also involves intensive chemotherapy before the edited cells are infused.

All 26 children experienced a grade 3 or 4 adverse event, and one child died following severe liver complications attributed to the conditioning medicine.

Importantly, UK authorisation does not automatically extend NHS funding: existing NICE managed-access recommendations apply to patients aged 12 and over.

Our full PHARMeMED coverage examines the evidence, risks and practical access questions.

For my weekly LinkedIn Newsletter covering latest insights and top story roundups.”

Paul Healy: UK Expands Casgevy Access to Younger Children With Sickle Cell Disease and Beta-Thalassaemia

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beta thalassaemia Casgevy CRISPR Therapeutics Health Hematology Hemostasis Hemostasis News Hemostasis Today Medicine Paul Healy Sickle Cell Disease Vertex Pharmaceuticals
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