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Exosomes — small extracellular vesicles that are shed by cells — have long offered promise as drug delivery systems for small molecules, DNA, RNA and other biologic payloads. The first of these are now in the clinic.
A wave of tau-targeted agents is progressing through the clinic, hoping to avoid the negative results from a first-in-class trial of a tau-targeted antibody.
The National Cancer Institute and Cancer Research UK’s US$380 million “Cancer Grand Challenges” programme highlights understudied knowledge gaps that could yet make big differences to patients.
A lack of novel excipients is threatening the ability of drug developers to translate progress with small molecules and biologics into therapeutic success. The FDA could soon test a new model of excipient review to foster formulation innovation.
Small-molecule integrin inhibitors are catching the attention of pharmaceutical firms, with fibrosis joining gut dysfunction as a key indication for integrin-based interventions.
Seres Therapeutics’ stool-derived treatment for recurrent Clostridium difficile infection could become the first FDA-approved microbiome therapy. Several other live biotherapeutic microbial products are close behind.
Twenty-four large and medium-sized pharmaceutical firms are backing a US$1 billion fund to steward antibiotics through phase II and III trials. Can they also break an antibiotic reimbursement impasse?
Despite recent setbacks with p53-activating small molecules including the nutlins, the cancer target keeps drug hunters coming back for more. Could immuno-oncology combinations, stapled peptides and targeted degraders unleash the therapeutic potential of the ‘guardian of the genome’?
Emerging evidence suggests that SARS-CoV-2 can drive a diverse array of immune processes, raising the risk that immunosuppressant agents that are in clinical trials might be effective for some patients but detrimental for others.
Clinical trials of genome-editing agents — including CRISPR–Cas9 editors, zinc finger nucleases and TALENs — are pushing ex vivo, immuno-oncology and in vivo treatment frontiers.
Macrophage-focused immuno-oncology programmes were in the doldrums just a year ago, but are now back on the move in the wake of Gilead Sciences’ US$4.9 billion acquisition of Forty Seven.
Drug-tolerant persisters, de novo mutations and pre-existing resistance are just some of the ways cancers evade the effects of anti-cancer drugs. Can new targets and new dosing strategies tackle evolutionary forces directly to delay the inevitable?
A massive, pioneering trial is underway to assess whether RNA-based modulation of Lp(a) — a form of bad cholesterol that narrows arteries, boosts the risk of blood clots and fans the flames of inflammation — can save lives.
