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Genomic Medicine · global

Sickle Cell Gene Therapy Moves Into Young Children, but Approval Is Only the Start of a Long Care Challenge

The U.S. FDA has allowed Vertex's sickle cell disease gene therapy to be used in children as young as two, signaling that a one-time treatment is entering the disease course earlier; but in pediatric care, the real test lies not only in efficacy, but also in pre-treatment conditioning, long-term follow-up, and family decision-making.

Biopharma · eu

European Drug Review’s June Meeting Opens the Door With One Hand and Hits the Brakes With the Other

Six new medicines were recommended for approval, moving once-weekly basal insulin, an influenza vaccine for older adults, and a rare-disease neurology drug closer to market; but negative opinions for a cell therapy and microbiome products also underscored that innovative platforms cannot bypass the thresholds of clinical credibility and manufacturing quality.