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Growing access to diverse 'real-world' data sources is enabling new approaches to close persistent evidence gaps about the optimal use of medical products in real-world practice. Here, we argue that contrary to widespread impressions, existing FDA regulations embody sufficient flexibility to accommodate the emerging tools and methods needed to achieve this goal.
A lipid-modulating protein that exemplifies the value of human genetics for target validation has provided a fertile testing ground for new drug modalities including long-acting RNA interference drugs, vaccines against self-antigens, CRISPR therapeutics and small molecules that control ribosomal activity.
The FDA could soon approve the first chimeric antigen receptor (CAR) T therapies for blood cancers, but this young field is still working on how to address solid tumours, safety concerns and manufacturing issues.
Thomas Lynch, Bristol-Myers Squibb's new Chief Scientific Officer, discusses his R&D priorities, genomics diagnostics and the need for faster development of novel cancer drugs in earlier stages of disease.
This analysis provides an overview of novel antihypertensive products, many of which are fixed-dose combinations of different drug classes, and provides an outlook on their likely impact on the market.
Antibody–drug conjugate (ADCs), which aim to target highly cytotoxic drugs specifically to cancer cells, are one of the fastest growing classes of anticancer therapeutics, with more than 50 such agents currently in clinical trials. This Review discusses lessons learned and emerging strategies in the development of ADCs, including aspects such as target selection, the development of warheads, the optimization of linkers and new conjugation chemistries, and provides an overview of agents that are currently in clinical trials.
Islet transplantation can be an effective therapy for patients with type 1 diabetes, but its widespread use is limited by the need for lifelong immunosuppression. Here, Desai and Shea discuss the emerging potential of islet cell encapsulation including new strategies, assess key challenges facing the human translation of this technology and highlight encapsulation devices that have entered the clinic.
Lipid second messengers such as phosphatidic acid (PtdOH) have a role in a wide range of pathological processes, and phospholipase D (PLD) enzymes are one of the major sources of signal-activated PtdOH generation. In this Review, Brown, Thomas and Lindsley discuss the development of PLD inhibitors, with a focus on isoform-specific inhibitors, and their potential applications in the treatment of cancer, neurodegeneration and infection.
