The discussion surrounding New Approach Methodologies (NAMs), organoids, and organ-on-chip technologies has accelerated rapidly across the pharmaceutical industry in recent years.[1]
While much of the public conversation focuses on reducing animal testing, the deeper transformation occurring inside major pharmaceutical companies is far more strategic. The core question is no longer whether organoids are scientifically promising, but whether these technologies can become scalable, reproducible, standardized, and ultimately integrated into real-world drug discovery and preclinical development workflows.
Increasingly, the answer appears to be yes.
Large pharmaceutical companies are no longer treating organoids as exploratory academic tools. Instead, they are building internal platforms, acquiring specialized technologies, forming translational partnerships, and integrating human-relevant systems directly into toxicology, ADME/DMPK, disease modeling, and biomarker discovery workflows.[1]
Importantly, each company is pursuing a different strategic path.
Merck KGaA: Standardization and Industrialization
Merck KGaA has adopted one of the clearest industrialization-focused strategies in the organoid field.
Its acquisition of HUB Organoids in 2024, followed by collaboration with Promega on 3D assay technologies in 2025, reflects a broader objective: transforming organoids from complex academic models into scalable industrial research tools suitable for high-throughput screening and pharmaceutical workflows.[2]
The strategic emphasis is not simply scientific novelty, but operational standardization and reproducibility. This is particularly important because one of the largest bottlenecks facing organoid adoption today is the lack of consistent protocols, standardized biological inputs, and scalable assay infrastructure.
Merck’s positioning suggests that the long-term commercial value of organoids may depend less on experimental complexity and more on the ability to industrialize and operationalize human-relevant biology within drug discovery pipelines.[1,2]
Roche: Deep Internal Integration into Drug Discovery
Roche’s strategy appears centered on internal integration and long-term ownership of human biology platforms.
The company established the Institute of Human Biology (IHB) and recruited organoid pioneer Hans Clevers in 2023 — a move widely interpreted as a major signal that organoid systems are becoming core infrastructure within pharmaceutical R&D rather than peripheral innovation projects.[3]
Rather than relying entirely on external collaborations, Roche appears focused on embedding organoid systems directly into the full translational workflow, including target discovery, disease modeling, biomarker development, and preclinical validation.[1,3]
This approach reflects a broader industry shift in which future competitive advantage may increasingly depend on proprietary human biology platforms and internally generated translational datasets.
AstraZeneca: Disease-Focused Strategic Deployment
AstraZeneca has taken a more disease-centric approach.
The company has invested heavily in kidney organoid platforms and participated in International Society of Nephrology (ISN) initiatives related to organoid applications in renal disease research. It has also explored organoid applications in chronic disease areas such as COPD.[4]
Unlike broad platform industrialization strategies, AstraZeneca’s deployment appears highly targeted: applying organoids in therapeutic areas where conventional animal models have historically struggled to deliver strong translational predictability.[1,4]
In diseases involving fibrosis, chronic inflammation, and renal biology, human-relevant organoid systems may provide significantly improved mechanistic insights and target validation opportunities compared with traditional preclinical approaches.
Novartis: Internal Platformization and “Hard Problem” Applications
Novartis has adopted one of the most technically integrated organoid strategies among major pharmaceutical companies.
Within NIBR and the DAx exploratory disease research organization, the company has developed retinal organoid toxicology systems, ADPKD kidney organoid disease models, and broader 3D organoid platforms supporting kidney disease, liver disease, and fibrosis research.[5,6,9]
Strategically, Novartis appears to prioritize organoids in areas where traditional models consistently underperform — particularly toxicity prediction, rare disease biology, fibrosis, and translational disease modeling.[1,5,6,9]
Its involvement in bone marrow organoid initiatives aligned with CAR-T development is particularly notable because it demonstrates how organoid systems may evolve beyond discovery-stage biology into operational infrastructure supporting advanced therapeutic pipelines.
Rather than treating organoids as standalone experimental models, Novartis appears to be integrating them as standardized internal research tools designed to solve some of the most difficult translational bottlenecks in drug development.
Bristol Myers Squibb (BMS): Internal Capability + Academic Ecosystem Strategy
BMS has taken a hybrid strategy that combines internal platform development with extensive academic network engagement. Internally, the company has established organoid-based ADME and toxicology capabilities to support translational and preclinical research workflows.[7,10]
Externally, BMS leverages the International Immuno-Oncology Network (II-ON), a large collaborative research ecosystem involving hundreds of researchers and multiple tumor-focused translational centers.[8]
This dual-track strategy allows BMS to simultaneously:
• address operational preclinical modeling needs internally
• maintain deep visibility into cutting-edge tumor microenvironment (TME) and immuno-oncology science externally
Importantly, tumor organoid systems are becoming increasingly valuable for biomarker discovery, immunotherapy response prediction, and combination therapy development — all areas highly relevant to BMS’s immuno-oncology portfolio.[7,8]
The Clinical Decision-Making Scenarios Where Organoids Are Gaining Strategic Importance
One of the most important industry signals is that organoids are no longer being evaluated solely as “better laboratory models.” Increasingly, they are being deployed in practical clinical and translational decision-making scenarios.
1. Drug Sensitivity and Patient Stratification
Patient-derived organoids (PDOs) are increasingly being explored as predictive systems for treatment response and precision medicine applications.
In pancreatic cancer research, the PASS-01 translational study evaluated patient-derived organoids for molecular subtyping and therapeutic response analysis in pancreatic cancer.[11] Similarly, studies in rectal cancer demonstrated that PDO systems could potentially predict chemoradiation response before treatment initiation.[12]
These developments suggest that organoids may eventually become important tools for therapy selection, biomarker-guided treatment decisions, responder/non-responder stratification, and translational clinical trial design.
This represents a significant evolution from traditional preclinical applications toward clinically actionable biological modeling.
2. Toxicity Prediction and Human-Relevant Safety Assessment
Another major application area is predictive toxicology.
Conventional animal models frequently struggle to accurately predict human-specific toxicities, particularly in liver, retinal, kidney, and immune-related adverse events.
Several pharmaceutical companies, including Novartis and BMS, are already deploying organoid systems internally for toxicity screening and translational safety assessment workflows.[6,10]
This trend is particularly important because toxicity-related failure remains one of the largest contributors to clinical attrition and late-stage development risk.[13]
As regulators increasingly encourage the integration of NAMs into drug development workflows, advanced organoid systems may become valuable complementary tools for DILI assessment, retinal toxicity prediction, fibrosis biology, immunotoxicology, and chronic disease modeling.
3. Cell Therapy and Tumor Microenvironment Modeling
A third rapidly expanding application area involves cell therapy and immuno-oncology research.
Tumor organoids and bone marrow organoid systems are increasingly being integrated into CAR-T translational studies, immune cell interaction modeling, TME biology research, and combination immunotherapy development.
For example, the comBO bone marrow organoid system was specifically designed to support preclinical modeling of hematopoietic disorders and immune interactions.[5]
At the same time, collaborative efforts involving pharmaceutical companies, academic cancer centers, and translational medicine groups are exploring how organoid systems can improve immunotherapy prediction and accelerate therapeutic development.[7,16,17]
Collectively, these developments indicate that organoids are evolving beyond static disease models into dynamic translational platforms capable of supporting clinically relevant biological decision-making.
How Smaller Biotech Companies Can Participate in the NAMs Transition
While major pharmaceutical companies are building internal organoid platforms and translational infrastructures, the broader NAMs ecosystem is also creating significant opportunities for smaller biotechnology companies, startups, CROs, and platform developers.
Importantly, not every company needs to build a fully integrated organoid platform from scratch.
In reality, the current industry landscape is increasingly driven by collaboration and specialization.
Emerging companies are contributing through:
• organ-on-chip engineering
• disease-specific organoid systems
• AI-assisted biological modeling
• translational analytics
• assay development
• microfluidics
• high-content imaging
• cell sourcing and biological standardization
This creates an ecosystem model rather than a single-platform winner-take-all environment.
In many cases, smaller biotech companies may be able to move faster than large pharmaceutical organizations in developing highly specialized translational systems, particularly in niche disease areas or emerging therapeutic modalities.
At the same time, one of the biggest challenges facing early-stage organoid and organ-on-chip developers remains biological consistency and scalability.
As these systems move closer toward industrialization and regulatory use, reproducibility becomes increasingly important.
The Emerging Importance of Upstream Biological Infrastructure
As the industry focuses heavily on organoid platforms, AI modeling, and microphysiological systems, one foundational reality is sometimes overlooked:
Advanced models are only as reliable as the biological materials used to build them.
Even the most sophisticated organoid or organ-on-chip systems remain fundamentally dependent on:
• high-quality primary cells
• donor consistency
• biological functionality
• standardized workflows
• stable preservation and recovery systems
Without reliable upstream biological inputs, downstream platform reproducibility becomes difficult to achieve.
This is where upstream biological material providers may become increasingly important strategic partners within the broader NAMs ecosystem.
Rather than directly competing as organoid platform developers, companies specializing in high-quality primary cells and biological materials may play a critical enabling role for the next generation of human-relevant in vitro systems.
A Collaborative Future for Human-Relevant Models
At MileCell Biotechnology, we believe the future of NAMs, organoids, and organ-on-chip systems will likely be built through collaboration across the broader life science ecosystem.
Large pharmaceutical companies may continue building internal translational platforms. Early-stage biotech innovators may develop disease-specific organoid systems or next-generation microphysiological technologies. CROs and translational medicine groups may focus on workflow validation and regulatory integration.
Within this evolving ecosystem, reliable upstream biological materials will remain foundational.
MileCell is interested in exploring strategic collaborations with organoid developers, organ-on-chip companies, translational medicine groups, early-stage biotechnology innovators, and CROs developing next-generation in vitro systems particularly in areas such as:
• ADME/DMPK
• toxicology
• fibrosis biology
• immunology
• cell therapy
• translational disease modeling
As industry continues transitioning toward more predictive human-relevant biology, collaboration across the biological supply chain may become increasingly important for enabling scalable and reproducible next-generation research platforms.
References
[1] Lambda Biologics. Organoids in 2025: The Year Regulation Met Reality (2025).
https://afs.lambda-bio.com/blog/organoids-in-2025-the-year-regulation-met-reality/
[2] Merck KGaA. Merck Enters Into Definitive Agreement to Acquire HUB Organoids (2024).
https://www.merckgroup.com/en/news/agreement-hub-17-12-2024.html
[3] Fierce Biotech. Roche poaches organoid pioneer Hans Clevers to lead all research and early development (2023).
https://www.fiercebiotech.com/biotech/roche-creates-organoid-research-institute-shakeup-drug-discovery-and-development
[4] AstraZeneca. Kidney organoids – advancing the science of renal diseases.
https://www.astrazeneca.com/what-science-can-do/topics/technologies/Kidney-disease-research-organoids.html
[5] Shen, Y., Benlabiod, C., Rodriguez-Romera, A., et al. comBO: A combined human bone and lympho-myeloid bone marrow organoid for pre-clinical modelling of haematopoietic disorders (2025).
https://doi.org/10.1101/2025.02.16.638505
[6] Dorgau, B., et al. Human iPSC-derived retinal organoid model for in vitro toxicity screening (2022).
https://oak.novartis.com/44981/
[7] Neal, J.T., et al. Organoid Modeling of the Tumor Immune Microenvironment. Cell (2018).
https://pmc.ncbi.nlm.nih.gov/articles/PMC6656687/
[8] Bristol Myers Squibb. Collaborating to advance cancer research: The International Immuno-Oncology Network (II-ON) (2017).
https://www.bms.com/life-and-science/science/the-international-immuno-oncology-network.html
[9] Novartis. DAx: exploratory disease research at Novartis.
https://www.novartis.com/research-and-development/research-disease-areas/dax-exploratory-disease-research-novartis
[10] Hedrich, W.D., Panzica-Kelly, J.M., Chen, S.J., et al. Development and characterization of rat duodenal organoids for ADME and toxicology applications (2020).
https://pubmed.ncbi.nlm.nih.gov/33199268/
[11] O'Reilly, E.M., et al. Pancreatic cancer subtyping using patient-derived organoids (PASS-01): a prospective, multicenter, translational study (2025).
[12] Yao, Y., et al. Patient-Derived Organoids Predict Chemoradiation Responses of Locally Advanced Rectal Cancer. Cell Stem Cell (2020).
https://doi.org/10.1016/j.stem.2019.10.010
[13] Wong, C.H., Siah, K.W., & Lo, A.W. Estimation of clinical trial success rates and related parameters. Biostatistics (2018).
https://doi.org/10.1093/biostatistics/kxx069
[16] Xilis. Xilis and MD Anderson Announce Strategic Collaboration (2023).
https://xilis.com/xilis-and-md-anderson-announce-strategic-collaboration-to-advance-novel-technology-and-accelerate-therapeutic-development/[17] German Cancer Research Center (DKFZ). 3D microtumors could revolutionize treatment decisions (2026).
https://www.dkfz.de/en/news/press-releases/detail/3d-microtumors-could-revolutionize-treatment-decisions
