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STING, RIG-I and NLRP3 agonists might increase the effectiveness of immuno-oncology checkpoint inhibitors, while antagonists of these targets offer an anti-inflammatory bonus.
Novartis, Merck, Pfizer and biotech entrepreneurs have started working on small-molecule drugs that act on an assortment of RNA targets, hoping to unlock once undruggable targets and new biology.
CARB-X, a public–private partnership aimed at bolstering the antibiotic pipeline, funded a diverse set of 17 early-stage drug development projects in its first year.
The FDA could soon approve the first bispecific antibody for a non-oncology indication, but clinical applications that make full use of the biological opportunity afforded by the nascent modality remain rare.
Results from Novartis's huge trial of the interleukin-1β blocker canakinumab could revitalize efforts to target inflammation in atherosclerosis, and have demonstrated unanticipated activity in lung cancer.
A resurgence in synthetic lethality screening, enabled by CRISPR gene editing, is unlocking targeted drugs for patients with cancerous loss-of-function mutations.
In vitrodata can be used to accelerate the approval of drugs that target specific disease-causing mutations for additional subpopulations of patients with rare diseases such as cystic fibrosis.
In the search for the first disease-modifying therapy for Parkinson disease, drug developers are advancing α-synuclein-targeted agents into proof-of-concept clinical trials.
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.
Libraries of functionalized small-molecule fragments that can be screened in whole cells could take phenotypic drug discovery to the next level, providing new opportunities against undertargeted proteins.
The European Medicines Agency's PRIME scheme to accelerate the development of promising drugs that address unmet medical needs has enrolled 19 products in its first year, showing considerable overlap with FDA breakthrough therapy designees but also key differences.