Innovations in Preclinical Drug Development: Efficacy, Safety, and Translational Strategies
A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Drug Targeting and Design".
Deadline for manuscript submissions: 31 January 2026 | Viewed by 45
Special Issue Editors
Interests: drug design; molecular glue degrader; antibody-drug conjugate; glutamine metabolism; innovative drug development
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Preclinical drug development is undergoing transformative innovation, focused on enhancing the predictive accuracy of candidate compound efficacy, safety assessment, and clinical translation success. In efficacy optimization, multi-omics technologies (genomics, proteomics, metabolomics) combined with AI-driven target identification and compound design significantly accelerate the discovery of highly selective and potent molecules. Advanced disease models (e.g., organoids, 3D co-cultures, humanized animal models) more accurately replicate the human microenvironment, improving the clinical relevance of efficacy evaluations. Safety assessment innovations emphasize early detection of off-target effects and mechanistic toxicity analysis. This is achieved through high-content screening, microphysiological systems (MPS), and computational toxicology models (e.g., QSAR, deep learning), enabling early warning of critical risks such as hepatotoxicity and cardiotoxicity. Key translational strategies include: (1) Integrated biomarker development to establish quantitative links between pharmacodynamics (PD) and disease progression; (2) Application of the Three Rs principles (Replacement, Reduction, Refinement) to optimize experimental design, reducing animal use while enhancing data quality; and (3) Implementation of fail-fast decision-making based on robust, quantifiable safety margins with strong translational relevance. These systematic innovations aim to bridge the gap between preclinical and clinical research, reduce late-stage attrition, and deliver safer, more effective therapeutic options to patients.
We look forward to receiving your contributions
Dr. Xi Xu
Dr. Hongxi Wu
Guest Editors
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Keywords
- preclinical drug development
- drug design
- advanced disease models
- three Rs principles
- molecular glue degrader
- antibody-drug conjugate
- glutamine metabolism
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