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Advances in understanding of the cause of autoimmune diseases and clinical data from novel therapeutic modalities such as chimeric antigen receptor T cells are providing evidence that it may be possible to re-establish immune homeostasis and, potentially, prolong remission or even cure autoimmune diseases. This article proposes a three-step ‘sequential immunotherapy’ framework for immune system modulation to help achieve this ambitious goal, and discusses existing drugs and those in development for each of the three steps.
Drugs that modulate fundamental mechanisms of ageing offer the promise of substantially improving the health of ageing populations, but innovative approaches to identify and evaluate such ‘gerotherapeutics’ are needed.
Accelerating Clinical Trials in the EU (ACT EU) is an initiative developed by the European Commission, Heads of Medicines Agencies and the European Medicines Agency to transform the clinical trials landscape in the European Union. It aims to foster regulatory, technological and process innovation to support better, faster and optimized clinical trials.
Drug discovery and development for inflammatory bowel disease (IBD) is hampered by various challenges including the insufficient mechanistic understanding of IBD immunopathology, disease heterogeneity, inadequate preclinical models and clinical trial inefficiencies. This Perspective assesses these limitations and presents strategies to overcome them, including the integration of artificial intelligence and machine learning approaches, organoid technology and innovative trial designs.
Secondary pharmacology screening of investigational small-molecule drugs for potentially adverse off-target activities is now standard practice in pharmaceutical research and development. This article uses a survey across 18 companies as the basis for discussing strategies for implementing secondary pharmacology screening programmes, approaches for interpreting off-target activities and which targets to include in screening panels, including several targets not commonly included in existing panels.
The rise of drug resistance presents a significant challenge in the treatment of infectious diseases. This Review summarizes the status and mechanisms of drug resistance in malaria, human immunodeficiency virus (HIV) infection and tuberculosis, and explores strategies to address resistance that could be incorporated into drug development across these disease areas.
The passage of the FDA Modernization Act 2.0 in 2022 has provided additional impetus to develop new approach methods for predicting the effects of drug candidates in humans from models such as microphysiological systems based on human-derived induced pluripotent stem cells. Here, we highlight progress in the field and strategies to address various challenges, including the application of artificial intelligence tools.
RNA has revolutionized modern medicine, creating great expectations. However, challenges with this class of drugs persist. In this Review, John Androsavich analyses lessons learnt from RNA modalities such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNA), and discusses strategies to develop RNA drug platforms, improving their applicability, efficacy and safety profiles.