FDA godkänner Crispr-behandling från två års ålder

FDA har utökat godkännandet för genterapin Casgevy (exagamglogene autotemcel) till att omfatta patienter från två års ålder.

By Lotta Naesenius

July 2, 2026 10:26 AM

Casgevy, som utvecklats av Vertex Pharmaceuticals, godkändes ursprungligen i USA för patienter från 12 års ålder med blodsjukdomarna transfusionsberoende β-talassemi och sicklecellanemi. Läkemedlet beskrivs som världens första behandling baserad på gensaxen Crispr/Cas9.

Även i EU är genterapin sedan 2024 godkänd för samma patientgrupper från 12 års ålder.

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