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The CACHE hit-finding competition highlights the potential of AI to identify small molecules that bind to hard-to-drug targets — and the long road ahead for these computational screening approaches.
Ventus has overcome medicinal chemistry challenges to advance a first inhibitor of cGAS-STING into the clinic, as drug developers build the case for novel anti-inflammatory agents in autoimmune diseases including lupus.
A pack of CAR T cell therapies are now in trials in systemic lupus erythematosus, as the race begins to prove the potential of autologous cell therapies in autoimmune diseases.
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.
Astellas’s claudin-18.2-targeted antibody zolbetuximab provides a benefit in hard-to-treat metastatic gastric cancer, and leads a multi-modality herd of contenders.
Moderna, BioNTech and others hope that personalized cancer vaccines will soon live up to their potential — even as they work out the wrinkles for the best use of their platform technologies.
GSK’s Arexvy and Pfizer’s Abrysvo provide older adults with a much-needed vaccine for respiratory syncytial virus (RSV) — and raise hopes for an option for infants too.
The FDA approved tofersen for amyotrophic lateral sclerosis based on the drug’s ability to lower blood levels of neurofilament light (NfL) — establishing a proof of potential for this neuroscience biomarker that could have implications for other diseases of the brain.
Vertex and CRISPR Therapeutics’s first-in-modality genome-editor exa-cel, for the treatment of two haemoglobinopathies, has entered the regulatory spotlight.
Biogen and Denali’s pivotal trial of a first-in-class LRRK2 kinase inhibitor is a long-awaited test of a target that could provide a path to disease-modifying therapies for Parkinson disease.
MYC, one of the most commonly dysregulated proteins in cancer, has long seemed ‘undruggable’. Can a clinical-stage cell-penetrating peptide — or preclinical small-molecule inhibitors and degraders — prove otherwise?