ADC, Gene Therapy, Cell Therapy, Oligonucleotides & Toxicology & Multi Organ & Liver

Opportunities for Microphysiological Systems in Toxicity Testing of New Drug Modalities

A comprehensive review of emerging drug- discovery approaches that leverage complex in vitro and in silico models to shift from animal- based toxicity to human- relevant predictive systems.

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Liver & Multi Organ & Toxicology

International Multisite Study of Human-Induced Pluripotent Stem Cell-Derived Cardiomyocytes for Drug Proarrhythmic Potential Assessment

A blinded, multisite study shows hiPSC-cardiomyocytes can stratify torsades de pointes risk across 28 drugs with reproducible electrophysiologic readouts, supporting use in proarrhythmia assessment.

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Multi Organ & Toxicology

Application of Microphysiological Systems to Enhance Safety Assessment in Drug Discovery

The review argues that MPS hold promise to improve drug safety prediction by offering more physiologically relevant preclinical models for key organs (liver, heart), while outlining existing technical and translational challenges to their adoption

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Heart, liver & Toxicology

Microengineered systems with iPSC-derived cardiac and hepatic cells to evaluate drug adverse effects

Outlines how iPSC-derived cardiac/hepatic microsystems model drug adverse effects and proposes qualification paths for regulatory-grade use.

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Multi Organ & ADME

Introduction to a manuscript series on the characterization and use of microphysiological systems (MPS) in pharmaceutical safety and ADME applications

Frames pharma needs and cross-cutting concepts for characterizing MPS toward defined contexts of use.

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Skin & Toxicology

Drug-induced skin toxicity: gaps in preclinical testing cascade as opportunities for complex in vitro models and assays

Maps current complex skin models, highlights missing immune/appendage/flow features, and recommends steps toward assay qualification.

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Liver & Toxicology

Liver microphysiological systems development guidelines for safety risk assessment in the pharmaceutical industry

Provides staged benchmarking, key performance metrics, and a 20-compound test set to standardize liver MPS for safety assessment.

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Kidney & Toxicology

A pharmaceutical industry perspective on microphysiological kidney systems for evaluation of safety for new therapies

Specifies attributes and context-of-use criteria to evaluate nephrotoxicity with kidney MPS in drug development.

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Multi Organ & ADME

Microphysiological systems for ADME-related applications: current status and recommendations for system development and characterization

Overview of MPS platforms for ADME: highlights progress in single- and multi-organ systems, outlines key design/validation criteria and urges industry adoption to reduce animal reliance

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GI & Toxicology

Developing in vitro assays to transform gastrointestinal safety assessment: potential for microphysiological systems

Proposes assay strategies and readouts to transform gastrointestinal safety testing, positioning GI-MPS as enabling tools.

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Liver & Toxicology & ADME

Characterizing the reproducibility in using a liver microphysiological system for assaying drug toxicity, metabolism, and accumulation

Demonstrates that a liver MPS yields reproducible toxicity, metabolism, and intracellular accumulation data across runs and labs.

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Cardiovascular & Toxicology

Cardiovascular microphysiological systems (CVMPS) for safety studies—a pharma perspective

Synthesizes how heart and vascular MPS can be integrated for CV safety, outlining practical adoption considerations.

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Heart & Toxicology

Use of Human iPSC-CMs in Nonclinical Regulatory Studies for Cardiac Safety Assessment

A review of how human-iPSC-derived cardiomyocytes (hiPSC-CMs) are increasingly used in nonclinical regulatory submissions for cardiac safety assessment, highlighting their utility and limitations.

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Multi Organ & Toxicology

IVIVE: Facilitating the Use of In Vitro Toxicity Data in Risk Assessment and Decision Making

A comprehensive review of in-vitro-to-in-vivo extrapolation (IVIVE) methods that outlines definitions, regulatory implications and the path to reducing animal testing in toxicity risk assessment.

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Liver & Toxicology & ADME

Liver-on-chips for drug discovery and development

A review of liver-on-chip (LoC) technologies highlighting their promise for improved hepatic clearance and toxicity prediction in drug development, while pinpointing translational and implementation challenges for widespread industrial use.

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Multi Organ & Toxicology

Opportunities and insights from pharmaceutical companies on the current use of new approach methodologies in nonclinical safety assessment

Industry survey reveals that pharmaceutical companies are increasingly using new approach methodologies (NAMs) to replace animal studies in nonclinical safety assessment—but adoption is hindered by regulatory harmonisation and translational-confidence gaps

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Multi Organ & Oligonucleotides

Leveraging microphysiological systems to address challenges encountered during development of oligonucleotide therapeutics

The review discusses how microphysiological systems (MPS) can help overcome key challenges in the development of oligonucleotide therapeutics (ONTs)—such as delivery, species selectivity, ADME, and toxicity- by providing human-relevant models tailored for ONTs.

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Multi Organ & Cell Therapy

Application of microphysiological systems for nonclinical evaluation of cell therapies

The review examines how microphysiological systems (MPS) can be applied to nonclinical evaluation of cell therapies, outlining opportunities (efficacy, safety, mechanistic insight) and challenges in qualification and adoption.

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Multi Organ & Gene Therapy

Exploring the synergy of CRISPR and microphysiological systems

A review exploring how combining CRISPR- based genome editing with microphysiological systems (MPS) enhances both gene therapy modelling and safety assessment by improving human relevance and reducing animal use.

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Liver

Liver Microphysiological Systems for Predicting and Evaluating Drug Effects

Reviews how liver MPS recapitulate 3D, multicellular, flow-exposed microenvironments to improve prediction of metabolism, DDI, and toxicity relevant to clinical pharmacology.

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Liver

Liver-on-chips for drug discovery and development

A review of liver-on-chip (LoC) technologies highlighting their promise for improved hepatic clearance and toxicity prediction in drug development, while pinpointing translational and implementation challenges for widespread industrial use.

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Lung

Microphysiological lung models to evaluate the safety of new pharmaceutical modalities: a biopharmaceutical perspective

Defines qualification elements and practical needs to deploy lung MPS for biopharma safety testing and de-risking new modalities.

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Immune System

Application of Immunocompetent Microphysiological Systems in Drug Development: Current Perspective and Recommendations

Summarizes status and recommendations for incorporating immune-competent MPS into pipelines, including qualification needs.

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Multi Organ

Perspectives on the evaluation and adoption of complex in vitro models in drug development: Workshop with the FDA and the pharmaceutical industry (IQ MPS Affiliate)

FDA–IQ MPS workshop consensus on evaluation, standardization, and adoption frameworks for complex in vitro models in drug development.

Open resource

Multi Organ

Applications of microphysiological systems to disease models in the biopharmaceutical industry: Opportunities and challenges

Reviews how disease-relevant MPS are used in industry and the hurdles to broader deployment and regulatory acceptance.

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Multi Organ

Considerations from an International Regulatory and Pharmaceutical Industry (IQ MPS Affiliate) Workshop on the Standardization of Complex In Vitro Models in Drug Development

Captures FDA–IQ MPS workshop outcomes on standardizing complex in vitro models and pathways for regulatory use.

Open resource

Multi Organ

Towards in vitro models for reducing or replacing the use of animals in drug testing

Pharma authors argue for in vitro model adoption to reduce animal use while pinpointing advances needed to raise readiness levels.

Open resource

Multi Organ

The Current Status and Use of Microphysiological Systems by the Pharmaceutical Industry: The International Consortium for Innovation and Quality Microphysiological Systems Affiliate Survey and Commentary.

Industry survey details how MPS are resourced and applied (safety/PK/PD/ADME) and flags barriers to broader internal adoption and regulatory inclusion.

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Multi Organ

Considerations from the pharmaceutical industry (IQ MPS Affiliate) workshop on animal microphysiological systems and 3Rs in drug development

Discusses roles for animal cell-based MPS in 3Rs strategies and how they might complement human MPS in development pipelines.

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Multi Organ

Pharmaceutical Industry Perspective on the Utility of Animal Cell-Based Microphysiological Systems to Support Human Drug Development

Presents a pharma perspective on when animal MPS can be decision-supportive for human programs and how they fit alongside human MPS.

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Multi Organ

Potential value of animal microphysiological systems

Animal-derived microphysiological systems (MPS) can help bridge species gaps, support regulatory decision-making and advance the 3Rs by mimicking organ function across species.

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Multi Organ

Advancing alternative methods to reduce animal testing: Emerging approaches show promise for regulatory use

FDA outlines a strategic framework for accelerating qualification and regulatory adoption of new-approach methodologies (NAMs) to reduce reliance on animal testing.

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Multi Organ

Gaps and challenges in nonclinical assessments of pharmaceuticals: An FDA/CDER perspective on considerations for development of new approach methodologies

FDA/CDER identifies key gaps in current nonclinical testing and advocates for context-defined new approach methodologies (NAMs) to bolster regulatory safety evaluations.

Open resource

Multi Organ

Human organs-on-chips for disease modelling, drug development and personalized medicine

Human organ-chip devices replicate organ-level human physiology and inter-organ interactions to improve disease modelling, drug development and personalised medicine.

Open resource

Multi Organ

A guide to the organ-on-a-chip

This Primer systematically maps the design, fabrication, operation and applications of organ-on-chip (OoC) systems, enabling researchers to match device architecture to biological function efficiently.

Open resource

Multi Organ

Organ-on-a-chip systems: translating concept into practice (manuscript series)

This editorial frames the transition of organ-on-chip systems from concept to practice by highlighting commercialization milestones and practical implementation challenges.

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Multi Organ

Complex in vitro models positioned for impact on drug testing in pharma: a review

A pharma-industry review defining model-omics and context-of-use frameworks to evaluate where complex in vitro models (CIVMs) like organoids and Organ-Chips are ready for impact in drug development.

Open resource