Advancing Drug Discovery with Human-Relevant Models and New Approach Methodologies
Advancing Drug Discovery with Human-Relevant Models and New Approach Methodologies
New approach methodologies (NAMs) are reshaping drug discovery and safety assessment by prioritizing human-relevant systems that improve predictive power and reduce reliance on animal models. These methodologies include in vitro assays, in chemico assays, ex vivo assays, and in silico approaches, including those leveraging artificial intelligence and machine learning algorithms. Reflecting this momentum, global health authorities are encouraging NAM adoption. In 2025, the (FDA) announced a roadmap to phase out certain animal testing in favor of in vitro systems, computational modeling, and other related NAMs. has been working on identifying opportunities for using NAMs to support risk assessment, and the (EMA) supports the regulatory acceptance of NAMs. Together, these positions signal a global shift toward human-relevant models.
However, implementing NAMs is not simply a matter of adopting new tools. A key subset of NAMs are based on in vitro human tissue models, and their successful implementation depends on establishing a solid foundation of biologically relevant systems, reproducible workflows, and clear connections between in vitro data and human outcomes.
Building In Vitro NAMs on Strong Biological Foundations
The predictive power of in vitro NAMs depends on having a strong understanding of human biology and the ability to meaningfully reflect its complexity in in vitro systems. Cell source, differentiation strategy, and culture conditions directly influence functional performance, toxicity signals, and the degree to which a system can provide mechanistic insight. Variability in these factors can compromise the integrity of even the most sophisticated models, the quality of the resulting data, and their value in informing confident decisions. Developing robust NAMs therefore requires well-defined, carefully optimized culture conditions and standardized workflows that support physiological relevance, scalability, and experimental reproducibility.
Enabling NAM Development Across the Drug Discovery Pipeline
Human-relevant in vitro systems are playing an increasingly impactful role throughout drug discovery processes. When thoughtfully implemented as NAMs, they enable earlier insight into tissue-specific responses, including toxicity liabilities and structure-activity and structure-toxicity relationships. By generating functional data in controlled human-relevant systems, researchers can reduce uncertainty before advancing drug candidates into later stages of development.
Human in vitro tissue models can be effectively deployed at multiple stages of drug discovery, including:
- Biomarker discovery and measurement
- Disease modeling
- Mechanistic toxicity assessment and liability screening
- Functional performance testing
- Immunomodulation and inflammatory profiling
By integrating scalable, organ-specific systems into your workflows, you can identify risks earlier, strengthen decision-making, and refine candidates with greater confidence.
Partnering with 海角破解版 Technologies
As research shifts toward more predictive, human-relevant approaches, developing and implementing NAMs require reproducible systems with reliable biology and informed experimental design. With more than 30 years of expertise in cell culture innovation, 海角破解版 Technologies can support your research with NAM-enabled models, reagents and entire workflows across organ systems. For teams seeking additional support, our Contract Research Services can provide assay development with our optimized cell culture media and tools to advance your programs with confidence.
Explore our tools for building human in vitro tissue models, organized by organ system.
Circulatory System Tools for NAMs
The circulatory system distributes therapeutic agents and is a frequent site of off-target effects. Human CD34+ cells can be used to model hematotoxicity, differentiation, and engraftment, supporting research in hematopoiesis and blood disorders such as myelodysplastic syndromes (MDS), sickle cell disease, and cytopenias. Induced pluripotent stem cell (iPSC)-derived cardiomyocytes and endothelial models support toxicity testing and disease modeling of arrhythmias, heart failure, and atherosclerosis for efficacy studies or disease biology research.
Gastrointestinal Tools for NAMs
The digestive system鈥攑articularly the intestine and liver鈥攊s central to drug absorption and metabolism. Intestinal models enable evaluation of absorption and first-pass metabolism, while hepatic systems support assessment of metabolic functions, drug-induced liver injury (DILI), and cytochrome P450 (CYP)-mediated drug-drug interactions. Human-relevant models capturing these processes are essential for predicting pharmacokinetics and identifying liabilities early enough to be de-risked within a program.
Immunology Tools for NAMs
The immune system plays a central role across therapeutic areas鈥攆rom immuno-oncology to autoimmunity鈥攁nd is commonly implicated in adverse effects such as cytokine release or immunosuppression. Human immune cell models support evaluation of immune activation, suppression, and modulation. These systems are also essential for disease modeling, target validation, and assessing immunotoxicity in both biologic and small-molecule drug programs.
Muscular System Tools for NAMs
Skeletal and cardiac muscle tissues are key to evaluating both target-specific activity and off-target toxicity. Cardiotoxicity is a critical concern for many drug classes, while skeletal muscle can be affected by metabolic or neuromuscular side effects. Human iPSC-derived muscle models enable assessment of functional contractility, structural integrity, and cellular stress. These platforms also support disease modeling for conditions like muscular dystrophies and cardiomyopathies.
Nervous System Tools for NAMs
The nervous system is highly sensitive to off-target effects, including neurotoxicity and peripheral neuropathy, making early assessment critical. Human neural models enable evaluation of neuronal viability, function, and network activity. Beyond safety profiling, these systems also support disease modeling for neurodegenerative, neurodevelopmental, neuropsychiatric, and neuroinflammatory disorders, providing platforms for target validation and phenotypic screening in complex neurological contexts.
Respiratory System Tools for NAMs
The respiratory system is a key site of exposure for inhaled or systemically administered therapeutics and a target in diseases such as asthma, chronic obstructive pulmonary disease (COPD), and pulmonary fibrosis. Human airway and alveolar models support evaluation of respiratory toxicity, barrier integrity, and inflammatory responses. These platforms also enable disease modeling and screening in lung-relevant contexts.
Sensory System Tools for NAMs
The skin and eye are highly accessible yet vulnerable tissues, commonly affected by off-target effects such as irritation, inflammation, or degeneration. Human-relevant in vitro models support assessment of barrier integrity, immune response, and cytotoxicity. These systems also enable disease modeling for conditions like dermatitis, psoriasis, and retinal disorders, supporting the development of topical, ophthalmic, and systemically administered therapeutics.
Stromal System Tools for NAMs
Stromal cells鈥攊ncluding mesenchymal stem cells and their progeny鈥攑lay critical roles in tissue homeostasis, repair, fibrosis, and tumor microenvironment dynamics. Their signaling and structural support functions influence drug response across tissues. In vitro stromal models enable investigation of cell-cell and cell-matrix interactions, modulation of immune or epithelial behavior, and target validation in contexts such as fibrosis, oncology, and regenerative medicine.
Urogenital System Tools for NAMs
The urogenital system is a key consideration for both reproductive toxicity and renal clearance. Kidney models support evaluation of nephrotoxicity, transporter activity, and metabolic handling, while ovarian and uterine models help assess reproductive safety and enable disease modeling in areas like endometriosis or infertility.
