News

Madrigal’s resmetirom has scored a landmark FDA win, bolstering hopes for a diverse pipeline of drugs for non-alcoholic steatohepatitis.

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.

The FDA approved 55 novel therapeutics in 2023, the second highest count in the past 30 years.

The FDA could soon approve Iovance’s lifileucel for advanced melanoma, a much-anticipated milestone for the 35-year-old T cell therapy modality.

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.

Move over degraders; proximity-inducing drugs that stabilize, phosphorylate and inhibit their targets are coming through.

Clinic-ready inhibitors, degraders and molecular glues are pushing cancer frontiers by targeting KRAS variants including KRAS-G12D.

The FDA approved 37 novel drugs in 2022, the fewest to pass regulatory scrutiny since 2016.

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?

Could Vertex’s Nav1.8 blocker tap the elusive analgesic potential of voltage-gated sodium channels?

Flush with COVID-19 vaccine success, pharma and biotech companies are moving forward with mRNAs engineered to make therapeutic proteins.

Bristol Myers Squibb’s first-in-class TYK2 inhibitor and blockbuster contender deucravacitinib showcases the role for heavy hydrogen in novel drug discovery.