News

Clinical failures of antisense candidates from two companies highlight the challenges for huntingtin-lowering approaches, but a diverse pipeline could yet provide a disease-modifying therapy.

Thirty-five years on from the FDA’s approval of a first monoclonal antibody, these biologics account for nearly a fifth of the agency’s new drug approvals each year.

As researchers work to understand the biology and epidemiology of post-acute COVID-19, a pioneering platform trial is now testing treatments to try to address the long-term complications of infection in previously hospitalized individuals.

At least 15 targeted degraders — from heterobifunctional PROTACs to molecular glues — should be in patients by the end of the year.

Before there was PD1, there was IL-2. Can drug developers channel the effects of this pleiotropic cytokine to take back the lead?

The FDA approved 53 novel drugs in 2020, the second highest count in over 20 years.

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.

Fragment-based screening methodologies are proliferating, fuelled by interest in novel target space and targeted degraders.

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.

Sanofi and others are testing whether trispecific antibodies might have applications in cancer and infectious disease indications.

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?