News in 2019

Continued VC largesse and a maturing biotech industry in China helped boost 2019’s IPO totals.

Immunotherapies that activate costimulatory receptors on T cells have failed so far in the clinic, but the first phase III trial of an agonist antibody in cancer has just begun and second-generation candidates are advancing.

Five anti-cancer KRAS inhibitors, with three different modes of action, are in the clinic. More are on the way.

RNA epigenetic drug development enters a new era, as companies start revealing targets and evidence of preclinical efficacy.

Nearly ten compounds from zebrafish screens are in or about to enter the clinic, and zebrafish 'avatars' are gaining traction as a tool to guide treatment plans for patients with cancers and rare diseases.

Target 2035 — an ambitious open-science proposal to develop a suite of chemical genomics tools to modulate every protein in the proteome — faces many challenges ahead.

Recursion, Janssen, insitro and others are combining high-content phenotypic screening with machine learning to zoom in on emerging opportunities in target discovery, hit identification, toxicity testing and more.

Small-molecule drugs and biologics that alter the microbiome or its interaction with host tissues are poised to change the face of microbiome therapeutics.

A long and growing list of anti-BCMA candidates — including chimeric antigen receptor-T cell therapies, antibody–drug conjugates and bispecific antibodies — are contending to transform multiple myeloma treatment.

The FDA has approved a first nanobody, lifting hopes for companies that are exploring innovative uses for domain antibodies.

Inhibitors of the innate immune system’s NLRP3 inflammasome promise potential in Parkinson disease, Alzheimer disease, non-alcoholic steatohepatitis, gout and much more, catching the eye of Novartis, Genentech and others.

Despite industry’s past setbacks with autophagy inhibitors, preclinical and clinical findings are starting to revitalize a once written-off strategy.

Transient liquid-like droplets made up of proteins and RNA are scattered throughout the cell — with potentially broad drug discovery implications.

Despite compelling genetic validation, drug developers are struggling to unlock the therapeutic promise of the Nav1.7 sodium channel as a pain target.

New therapeutics that harness cellular machinery to degrade targets are entering clinical trials, led by a PROTAC anticancer candidate developed by Arvinas.

Five years in, the US$360 million Accelerating Medicines Partnership is yielding tools to speed up drug discovery for rheumatoid arthritis, lupus, diabetes, Alzheimer disease and Parkinson disease.

Gene-silencing therapies promise progress in neurodegenerative diseases, with Roche and Ionis Pharmaceuticals testing whether such a drug can delay the progression of Huntington disease.

The FDA approved a record 59 drugs last year, but the commercial potential of these drugs is lacklustre.