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Bristol Myers Squibb’s first-in-class TYK2 inhibitor and blockbuster contender deucravacitinib showcases the role for heavy hydrogen in novel drug discovery.
As a surge of cancer drugs with new synthetic lethality targets enter the clinic, the opportunities and challenges of the underlying discovery strategies are coming into focus.
A PARP1-selective drug and a pipeline of ATR inhibitors demonstrate the near-term opportunities and long-term challenges for synthetic lethality in anticancer drug development.
With the FDA’s approval of the first TCR-based bispecific T cell engager, an emerging biological modality aims to take on new targets for solid cancers.
BMS’s LAG3-targeted antibody relatlimab is poised to expand the checkpoint inhibitor class beyond PD1 and CTLA4, but questions remain about the target’s future.
The NIH and FDA’s newly launched 27-member public–private partnership will spend US$76 million addressing the hurdles of AAV-based gene therapies for ultra-rare diseases.
Biogen and Ionis’s SOD1-antisense oligonucleotide tofersen failed a first phase III trial, raising questions about the next steps for this drug and for future ALS trials.
Cancer, hepatotoxicity, haematological and neurotoxicity concerns cause experts to call for more transparency and better manufacturing standards for AAV-based candidates.
AlphaFold and RoseTTAFold have delivered a revolutionary advance for protein structure predictions, but the implications for drug discovery are more incremental. For now.
Despite a regulatory setback for Provention Bio’s diabetes prevention candidate teplizumab, hopes remain high for T cell-regulating therapies in autoimmune indications including multiple sclerosis, systemic lupus erythematosus and coeliac disease.
As small activating RNAs gear up to enter phase II testing, other noncoding RNAs that upregulate gene expression are also progressing towards the clinic. But it’s early days for a complex field.