Nature Reviews Drug Discovery

Clinic-ready molecular glues and heterobifunctional PROTAC drugs are taking targeted protein degradation into uncharted territory. News News in Brief News in Brief News in Brief Biobusiness Briefs Biobusiness Briefs Jeanne Marrazzo, the director of the NIAID, discusses post-COVID pandemic preparedness, H5N1 avian flu and the tension between infectious disease protection versus profit.

Small molecules that block a key protein–protein interaction could change the treatment landscape for many patients with acute leukaemias. News News in Brief News in Brief News in Brief Biobusiness Briefs Jude Samulski, former CSO and co-founder of AskBio, discusses the future of gene therapy for complex and common diseases, including Parkinson disease and congestive heart failure.

Biohaven’s BHV-1300 lowered levels of immunoglobulin G in the blood of healthy volunteers — a first clinical demonstration of the potential of a small-molecule extracellular protein degrader. News News in Brief News in Brief News in Brief Biobusiness Briefs Cigall Kadoch, a cancer researcher at Dana-Farber Cancer Institute and co-founder of Foghorn Therapeutics, discusses the case for a new wave of epigenetic drugs, targeting chromatin remodelling complexes.

‘Pre-mRNA trans-splicing’ drugs could offer an alternative to gene replacement therapies for genetic diseases — starting with Stargardt disease. News News in Brief News in Brief News in Brief Biobusiness Briefs From the Analyst's Couch Top of page ⤴ RNA has revolutionized modern medicine, creating great expectations. However, challenges with this class of drugs persist.

ADAR-based editors that can change the mRNA code offer new opportunities in both rare genetic diseases and common complex ones. News News in Brief News in Brief News in Brief News in Brief Biobusiness Briefs Biobusiness Briefs Robert Plenge, head of Research at Bristol Myers Squibb, discusses his vision of sequential immunotherapy for autoimmune diseases — including multiple sclerosis.

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. News News in Brief News in Brief News in Brief Biobusiness Briefs Monica Bertagnolli, the newly appointed director of the NIH, discusses diversity, inclusion and access to biomedical research.

Madrigal’s resmetirom has scored a landmark FDA win, bolstering hopes for a diverse pipeline of drugs for non-alcoholic steatohepatitis. News News in Brief News in Brief News in Brief Biobusiness Briefs Biobusiness Briefs Biobusiness Briefs Stuart Schreiber discusses his plans to tackle inefficiencies in the translation of biomedical research, through the newly launched for-profit institute Arena BioWorks.

The FDA approved 55 novel therapeutics in 2023, the second highest count in the past 30 years. News News in Brief News in Brief News in Brief Biobusiness Briefs Biobusiness Briefs From the Analyst's Couch Top of page ⤴ The potential of harnessing tRNAs to treat genetic diseases has recently gained significant attention.

The FDA could soon approve Iovance’s lifileucel for advanced melanoma, a much-anticipated milestone for the 35-year-old T cell therapy modality. News News in Brief News in Brief News in Brief News in Brief Biobusiness Briefs Biobusiness Briefs Biobusiness Briefs Stuart Orkin, of Harvard Medical School and HHMI, discusses how the discovery of BCL11A paved the way for Vertex and CRISPR Therapeutics’ exa-cel therapy for haemoglobinopathies.

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. News News in Brief News in Brief News in Brief Biobusiness Briefs Biobusiness Briefs Jane Grogan, Biogen’s new head of research, talks about immunology, rare diseases and her longstanding interest in the cancer target TIGIT.