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Numerous challenges arise when developing targeted therapies for diseases comprising low-frequency molecular subtypes. In this article, we describe a pragmatic, science-based regulatory policy for the development and approval of targeted therapies in such cases.
Morgan Sheng, Vice President of Neuroscience at Genentech, discusses his continued faith in the amyloid hypothesis, the promise of the genetics of neurodegenerative disorders and the overlap between psychiatric disease and neurodegeneration.
Advances in areas such as microfluidics-assisted chemical synthesis and biological testing, as well as in artificial intelligence systems, are increasingly providing opportunities to introduce greater automation into the small-molecule drug discovery process. Schneider highlights approaches and technologies that seem poised to be implemented robustly by medicinal chemists in the near future and analyses the challenges for their more widespread application
Understanding the role of enzymes in disease states and the implementation of strategies to modulate their activities for therapeutic benefit remains a key focus for drug discovery. Here, Holdgate and colleagues assess the benefits of conducting and applying high-quality mechanistic enzymology studies throughout a drug-discovery programme and assess the value of combining such knowledge with orthogonal biophysical methods.
Lysosomal storage disorders (LSDs) are rare, inherited metabolic disorders that result from defects in lysosomal function, for which treatment options are limited. Here, Platt provides an overview of LSDs, approved and potential therapeutic approaches and agents currently in development. The challenges associated with LSD diagnosis, drug development and treatment are discussed.
