Nature Reviews Drug Discovery
Nature Portfolio · General Science & Medicine · ISSN 1474-1776, 1474-1784
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A decade after their discovery in 2016, base editors have evolved into versatile tools used in clinical trials for multiple human diseases, including sickle cell disease and leukaemias.
Early clinical reports of CD19- and B cell maturation antigen-directed immune cell engagers show sustained improvements in several systemic autoimmune diseases.
Targeting T follicular helper cells offers a promising strategy to enhance lifelong health by modulating immune responses across various diseases and aging.
Novel in vivo delivery systems and immune-silent mRNA formulations are advancing mRNA therapeutics 2.0, enabling repeated administration and targeted organ delivery.
Simulation-guided design enhances complex clinical trial planning by evaluating operating characteristics and refining protocols for accelerated drug development.
Soft drugs are deliberately designed to undergo rapid metabolic inactivation, enhancing safety without reducing efficacy, especially in topical and systemic applications.
Cell surface RNA-binding proteins are identified with disease-specific and cell-type specificities, offering new avenues for immunotherapy and biomarker development.
Selective targeting of IL-6 trans-signalling may improve safety and efficacy in treating inflammatory diseases by differentiating from classic signalling pathways.
The number of drug targets has expanded to 686 biomolecules modulated by 1,702 drugs over the past 25 years, reflecting a significant evolution in therapeutic options.
Pharmacological strategies targeting multiple cellular pathways show promise in combating sarcopenia, a major cause of frailty in aging populations, with recent clinical trials indicating progress.
Targeting claudins offers a promising new approach, with zolbetuximab becoming the first FDA-approved CLDN-18.2-targeted antibody for gastric cancer treatment.
Dysregulated solute carrier (SLC) transporters are emerging as promising therapeutic targets for CNS disorders, with many linked to epilepsy, neurodegeneration, and autism.
Autophagy plays a complex, dual role in cancer, supporting tumor survival and immune responses, making it a challenging but promising therapeutic target.
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Recent mechanistic insights into agonistic antibodies have the potential to enhance their clinical efficacy and safety in immunotherapy.
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