Drug Discovery Solutions and In Vitro Models
Fewer than 10% of compounds that enter Phase I clinical trials ultimately reach approval, with 75 - 80% of failures attributed to lack of efficacy or safety concerns1. For therapy developers, this underscores the need to generate more predictive, human-relevant data earlier in the drug discovery process before advancing candidates into resource-intensive later-stage studies.
Selecting the right biological system is central to making these early decisions with confidence. Whether investigating disease biology, validating targets, identifying leads, or conducting safety assessment, the cell model you choose directly impacts the significance of your data. Using systems whose biological characteristics directly align with your intended context of use can help reduce uncertainty and strengthen early decision-making.
However, developing in vitro models and cell-based assays that are fit for purpose can be challenging. To generate meaningful data, models must accurately reflect the tissue context, disease state, or functional response under investigation. When grounded in physiologically relevant biology, these in vitro systems can provide deeper insight into disease mechanisms, support more reliable evaluation of candidate activity, and help identify potential efficacy and safety liabilities earlier in development, including through new approach methodologies (NAMs).
º£½ÇÆƽâ°æ Technologies supports therapy developers with biologically relevant cell models, defined media, optimized workflows, and scientific expertise that help strengthen preclinical and translational research. As a reliable supplier of NAMs-aligned cell models across a wide range of tissues, and decades of experience in cell sourcing, cell culture, cell isolation, quality control (QC), and cryopreservation, º£½ÇÆƽâ°æ can help your team develop:
- Healthy tissue models to evaluate in vitro toxicity, and standardised absorption, distribution, metabolism, and excretion (ADME) assays during lead identification, lead optimization, and pre-clinical studies.
- Disease models to investigate disease biology and evaluate therapeutic potential across key preclinical study phases, including target discovery and validation, lead identification and optimization, and preclinical data package generation.
Frequently Asked Questions About Cell-Based Assays for Drug Discovery
What are new approach methodologies (NAMs) and why are they gaining traction in drug discovery?
New approach methodologies (NAMs) are human-relevant in vitro, in silico, and in chemico approaches that allow for rapid and effective risk assessment while reducing reliance on, or complementing, traditional animal models. They include organoids, human pluripotent stem cell (hPSC)-derived cell models, microphysiological systems, and artificial intelligence and machine learning algorithms among others.
Global health authorities such as , the (EMA), and the (FDA) has accelerated interest by removing the longstanding statutory requirement for animal testing in drug development and explicitly recognized these approaches as acceptable alternatives for Investigational New Drug (IND) submissions. NAMs are gaining traction in drug discovery because they offer therapy developers a practical path to generate more human-relevant data earlier in development and support more predictive efficacy, safety, and ADME studies.
Adopting NAMs is a progressive, iterative journey rather than a sudden displacement of legacy protocols. Rather than seeking an immediate, wholesale replacement of animal models, the most effective strategy leverages human cell-based assays within a clearly defined Context of Use (CoU) to run parallel benchmarks alongside traditional studies, augmenting the predictive accuracy of early safety and efficacy screening. º£½ÇÆƽâ°æ supports NAMs implementation by providing reliable cell sources, optimized culture media, and validated workflows that underpin reproducible, human-relevant experimental systems.
How does the choice of cell source shape outcomes in the drug discovery pipeline?
The choice of cell source is one of the earliest and most consequential decisions in designing an in vitro workflow, as it directly shapes what biological questions the model can reliably answer.
- Human primary cells are isolated directly from human tissue and retain the differentiated phenotype and functional characteristics of the source, making them valuable for physiologically grounded experiments where access to the relevant cell type is available.
- iPSC-derived cells offer scalability, genetic tractability, and the ability to model patient-specific or disease-relevant genotypes, which is particularly useful in neurological, cardiovascular, and rare disease research.
In practice, many drug discovery programs use both in parallel: human primary cells for near-term functional validation, and iPSC-derived models for longer-horizon mechanistic or patient-stratified studies. The right starting point depends on the disease biology you are modeling, and our Disease Modeling resources walk through this across several tissue contexts.
For either approach, establishing a reliable biological starting point is critical. Standardized primary cell culture, iPSC culture, differentiation, and quality control resources walk through this across several tissue contexts.
How should researchers approach model selection by complexity when implementing in vitro NAMs?
When implementing in vitro NAMs, model selection should be guided by the specific biological question the system needs to answer. In vitro NAMs range from 2D primary or human pluripotent stem cell (hPSC)-derived cell cultures to co-cultures, 3D spheroids, organoids, and organ-on-chip systems. The most effective approach is to use the least complex in vitro NAM that can reliably support your experimental goal while fitting your required scale, workflow, timeline, and funding situation.
In practice, this often means using simpler, highly reproducible models earlier in the pipeline for broader screening, assay optimization, or target validation, then moving to more complex, translationally rich systems when additional biological context is needed to assess mechanism of action, targeted efficacy, or safety risk. This fit-for-purpose strategy helps researchers adopt NAMs in a practical way, balancing human relevance with assay robustness, workflow integration, and resource efficiency.
What practices support reproducibility and scalability when working with in vitro NAMs?
Reproducibility in in vitro NAMs workflows depends on standardizing the key inputs and conditions that underpin those systems. This includes using well-characterized human cells, lot-validated materials, consistent cryopreservation practices, defined culture media, and validated protocols. These controls help reduce biological variability, limit biological drift, and make results easier to compare across experiments, operators, and sites.
As NAMs are adopted more broadly in screening, disease modeling, toxicity testing, and ADME studies, reproducible workflows are essential for building confidence in the data. º£½ÇÆƽâ°æ supports this with quality-tested primary and cultured cell products, optimized culture media, validated protocols, and workflow solutions that help researchers build more consistent and scalable cell-based assays.
How do culture media and workflow conditions influence the performance of in vitro drug discovery models?
Culture media and workflow conditions directly shape the biological performance and predictive value of in vitro drug discovery models. Cell culture media is an active signaling environment that influences cellular identity, morphology, marker expression, functional maturation, and assay stability. If culture conditions are not optimized for the relevant tissue, cells can lose key physiological traits that would be essential in generating predictive data.
For drug discovery teams, this makes media formulation a critical part of model qualification. A model may appear viable but still be unsuitable if the markers, functions, or phenotypes required for the experiment are not present or reproducible. Using optimized, tissue-specific media and standardized culture workflows helps maintain biological relevance and generate data that is better aligned with the intended application.
What should research teams look for in a cell and reagent supplier for drug discovery?
The most valuable support combines reliable biological starting materials, standardized culture systems, and the scientific expertise to help teams build workflows that are reproducible and scalable from the start, providing scientific depth and workflow flexibility at every stage.
º£½ÇÆƽâ°æ Technologies can support drug discovery workflows by supplying standardized biological inputs and integrated workflow solutions, including high-quality primary cells, human induced pluripotent stem cell (iPSC) lines, optimized cell culture media, cell isolation products, and cryopreservation solutions.
For teams seeking additional support, our Contract Research Services can provide assay development and workflow design, drawing on more than 30 years of in vitro biology expertise.
Together, these products and services help researchers reduce variability, streamline assay development, and establish more reproducible in vitro model systems throughout the drug discovery process.
Explore our scientific resources to learn how predictive in vitro models can help you generate more meaningful insights throughout drug discovery. This learning center will cover cell sourcing, alongside critical experimental domains within the drug discovery pipeline, specifically disease modeling and predictive toxicity testing, as well as technologies and emerging trends shaping more human-relevant research workflows.
References
1. Hinkson IV et al. (2020) Front Pharmacol 11: 770.
