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New drug approvals reached an all-time high in 2023, with five gene therapies, the first CRISPR–Cas9-edited therapy and a disease-modifying Alzheimer’s drug.
Researchers in low- and middle-income countries are developing their own IP, scaling up local manufacturing, and looking for biomarkers — all in the hope of bringing costs down and getting therapies to people who need them.
Nature Biotechnology’s annual survey highlights academic start ups that are, among other things, correcting misfolded or disordered proteins, creating second-generation GPCR agonists, building a new gene delivery platform and mining cancer genomes for novel targets.
As new technologies hit the market, synthetic DNA is available faster and cheaper than ever before. Regulators are preparing to step in to limit opportunities for misuse.
Some 700 approved therapies in the United States — roughly a third of all drugs on the market — target G-protein-coupled receptors (GPCRs). Now advances in high-throughput and structure-based screening are sparking a second golden age of GPCR-based drug discovery.
Small molecules that target messenger RNA have therapeutic potential, but the field still lacks an unqualified success. Companies differ on how to move the resurgent field forward.
From made-to-order genetic therapies to model organisms engineered to be ‘patient avatars’, the technology exists right now to save patients with rare diseases.
Tumors are rife with bacteria and fungi. Their ubiquity is proving useful in detecting cancers, categorizing them, and even determining whether certain interventions will work.