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Rebecca Ahrens-Nicklas, a physician-scientist at the Children’s Hospital of Philadelphia, discusses her groundbreaking application of a gene-editing therapy for an ultra-rare urea cycle disorder.Nature Reviews Drug Discovery, Published online: 2025-07-15; doi:10.1038/d41573-025-00119-62025-07-15 DOI: 10.1038/d41573-025-00119-6
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FDA new drug approvals in Q2 2025Nature Reviews Drug Discovery, Published online: 2025-07-15; doi:10.1038/d41573-025-00124-92025-07-15 DOI: 10.1038/d41573-025-00124-9
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Chemical modification represents an effective strategy to improve the stability, efficiency and specificity of RNA therapeutics, while reducing their immunogenicity. This Review discusses approaches for manufacturing three major categories of RNA — small RNA, translatable RNA and CRISPR guide RNA — and assesses chemical modifications being applied to their broad therapeutic applications. Clinical examples and potential future opportunities are discussed.Nature Reviews Drug Discovery, Published online: 2025-07-14; doi:10.1038/s41573-025-01237-x2025-07-14 DOI: 10.1038/s41573-025-01237-x
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CRISPR-based technologies provide a diverse set of tools to correct pathogenic mutations. This Review describes the promise and the challenges of genome editing therapeutic strategies involving nucleases, base editors and prime editors for patients with inherited haematological disorders.Nature Reviews Drug Discovery, Published online: 2025-07-14; doi:10.1038/s41573-025-01236-y2025-07-14 DOI: 10.1038/s41573-025-01236-y
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Unmet needs in vaccine developmentNature Reviews Drug Discovery, Published online: 2025-07-10; doi:10.1038/d41573-025-00100-32025-07-10 DOI: 10.1038/d41573-025-00100-3
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Lilly buys Verve and its in vivo gene editors for up to US$1.3 billionNature Reviews Drug Discovery, Published online: 2025-07-09; doi:10.1038/d41573-025-00120-z2025-07-09 DOI: 10.1038/d41573-025-00120-z
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The US biosimilars market after one decadeNature Reviews Drug Discovery, Published online: 2025-07-09; doi:10.1038/d41573-025-00101-22025-07-09 DOI: 10.1038/d41573-025-00101-2
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FDA approves twice-yearly capsid inhibitor for HIV preventionNature Reviews Drug Discovery, Published online: 2025-07-08; doi:10.1038/d41573-025-00117-82025-07-08 DOI: 10.1038/d41573-025-00117-8
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