A gene therapy intended as a one-time treatment for Huntington’s disease significantly slowed progression of the disorder in a pivotal Phase 1/2 trial, paving the way for a regulatory submission in the first quarter of 2026. The developer, uniQure NV, showed that it is possible to safely impact the disease by targeting both the disease-causing huntingtin protein and the normal protein using a vector-based gene therapy and a gene encoding a microRNA (miRNA).
Pfizer Inc is to pay $4.9 billion to acquire a three-year old company, Metsera Inc, which is developing drugs for weight management giving it a selection of candidate products that can be delivered orally and by injection and which include both amylin and incretin mimetics. The amylin-based treatments restore sensitivity to leptin, a satiety hormone that enables people to feel naturally full after eating. The incretins, which include the glucagon-like peptide-1 (GLP-1) drugs, mimic hormones in the gut that help regulate blood sugar and appetite.
The European Medicines Agency has issued a positive opinion for a new indication of an antibody therapeutic that targets B cells in order to treat immunoglobulin G4 related diseases. These are autoimmune disorders caused when the body’s own defence system attacks normal tissues. This can lead to fibrosis and inflammation in one or multiple organs of the body. The therapy, Uplizna (inebilizumab), has already been approved for the treatment of neuromyelitis optica spectrum disorder, an inflammatory disorder of the central nervous system.
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