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CRISPR-based genome editing has the potential to treat many human genetic diseases, but achieving stable, efficient and safe in vivo delivery remains a challenge. This Review assesses current delivery systems for genome editors—focusing on adeno-associated viruses and lipid nanoparticles—and highlights data from recent clinical trials. Emerging delivery systems and ongoing challenges in the field are discussed.
Advances in computational omics technologies are enabling access to the hidden diversity of natural products, and artificial intelligence approaches are facilitating key steps in harnessing the therapeutic potential of such compounds, including biological activity prediction. This article discusses synergies between these fields to effectively identify drug candidates from the plethora of molecules produced by nature, and how to address the challenges in realizing the potential of these synergies.
Merck & Co.’s PCSK9 inhibitor MK-0616 showcases the potential of macrocycles as oral drugs for extracellular targets, even as several companies explore intracellular and macrocycle–drug conjugate possibilities.
The total number of rare conditions is debated, partly because of the variety of definitions of what constitutes rare. A broader consensus view of what rare means, based on improved understanding of individual group and patient clinicopathological characteristics, will help maximize the impact of technological advances in therapeutic development programmes.
