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The FDA's rejection of UniQure's gene therapy AMT-130 for Huntington's disease has been overturned after the ouster of Trump officials, including regulator Vinay Prasad, who had demanded sham surgeries for a placebo control.
First person treated in a gene therapy trial using partial reprogramming to rejuvenate cells, aiming to treat glaucoma by regenerating optic nerve neurons.
New Limit's cellular reprogramming therapy, delivered via mRNA in lipid nanoparticles, restores liver resilience and improves survival in aged mice subjected to repeated alcohol cycles, with human trials planned next year in Australia.
Early clinical trial results show that repurposing CAR-T cell therapy, originally used for cancer, can help control HIV long-term. Two patients achieved undetectable HIV levels after a single infusion and stopped medications, suggesting a potential functional cure.