Topic

Gene therapy

Coverage of gene therapy across PatientSpotlight - the developments worth watching, the access reality, and what the next twelve months may bring. 1 piece on this topic across therapy areas, newest first within each collection.

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Signals

SignalApr 29, 2026FDA

FDA approves first gene therapy for severe LAD-I

The FDA approved Kresladi (marnetegragene autotemcel), the first gene therapy indicated for severe Leukocyte Adhesion Deficiency Type I, marking the first approved treatment specifically targeting the genetic root cause of this rare, life-threatening immune disorder.

RegulatoryGene therapyTreatmentAccess