Organ-on-a-chip: Thumb-sized Organs, Huge Potential For Pharma R&D

Thumb-sized devices that mimic human organs could change the way we make drugs—and benefit countless patients. Discover what impact organ-on-a-chip could have on drug development and how Pharma stakeholders are investing to realise its true potential.



11 years. It’s the average time it takes for a therapeutic to make its journey from the laboratory to our medicine cabinet. 11 years of thorough research, during which sponsors spend a significant amount of time and resources evaluating a drug candidate’s safety and efficacy. 

Most common methods used by drug developers rely on animal and cell-based models that approximate the human biology, to ultimately predict if a therapeutic is safe and effective for humans. However, while these approaches provide highly valuable insights for drug developers, it is important to recognize they also come with certain drawbacks. 

As 90% of drug candidates that enter clinical trials fail due to a lack of efficacy or unexpected safety issues, and with a cost of failed attempts to bring a single drug to market reaching € 1.5Bn—Pharma is urged to find models that better reflect the human biology. 



Holding the potential to mimic human physiology more accurately than existing models, OoC could enable more realistic and precise drug testing, reduce R&D costs and timescales to bring new therapeutics to the clinic—all while scaling back on animal testing. 

With such promising prospects, the technology keeps gaining momentum—as the whole pharma ecosystem pushes to overcome the remaining challenges and unlock its full potential. 


What is an Organ-on-a-chip?


Originating in the early 2010s with the development of the first human lung-on-a-chip, the concept of organ-on-a-chip (OoC) emerged as a response to the well-known limitations of cell and animal-based testing methods. Recognizing this gap, researchers sought a solution that could bring more accuracy and scalability all while reducing reliance on animals. 


Various models are used for pre-clinical research


Combining biology and microtechnology, organ-on-chips are micro-scale 3D systems that replicate human physiology. Using microfluidics and cells, these systems emulate physiological and mechanical conditions experienced within the human body.  

In vitro, miniature tissues—engineered or natural—are cultivated within intricately designed microfluidic chips. The latter finely control cell microenvironments, sustaining tissue-specific functions, striving for a more faithful replication of human physiology.  


Representation of a “double channel” organ-on-a-chip


OoC applications: highly transformative perspectives for Pharma R&D


13 years—and many advances in organ-on-a-chip engineering later—the possibility of seeing OoC applied to advance biomedical research and healthcare seems closer than ever. 

From pinpointing potential drug targets and conducting sophisticated toxicity assessments, to unravelling the intricacies of drug efficacy and enabling personalized medicine breakthroughs, the applications below showcase how much OoC could impact Pharma R&D operations in the future.


Target Discovery 

With its ability to model the human system, OoC could precisely identify drug targets in a controllable and traceable manner. This platform proves particularly powerful in dissecting the intricate processes and mechanisms underlying cancer progression, including cell migration and invasion, extracellular signalling, biophysical factors within the tumour microenvironment. 


Toxicology and Safety Assessments

The technology might become instrumental in toxicity and safety assessments of drugs and other compounds. By simulating the organ's microstructure and function, organ-on-a-chip devices provide a more accurate representation of how substances interact with human tissues, enabling early detection of potential toxic effects and improving the safety profile of pharmaceuticals.


Efficacy Studies

Organ-on-a-chip platforms have the potential to assess drug efficacy in a more realistic environment. Researchers can observe how a drug interacts with specific organs, replicating the complex conditions of the human body. This approach enhances the understanding of a drug's effectiveness before progressing to more resource-intensive and time-consuming clinical trials.


Absorption, Distribution, Metabolism, and Excretion (ADME) Studies

Organ-on-a-chip technology is utilized for studying the pharmacokinetics of drugs—how they are absorbed, distributed, metabolized, and excreted by the body. This information is critical in determining a drug's bioavailability and potential side effects, contributing to more informed decisions during the drug development process.


Personalized Medicine

Organ-on-a-chip technology has the potential to address personalized medicine by allowing researchers to create models that mimic the unique characteristics of an individual's organs. This enables the testing of drugs in a more patient-specific context, considering factors such as genetic variability and individual responses to treatment—ultimately paving the way for more tailored and effective therapeutic approaches.


Fostering the development and utilization of organ-on-chips 


With the promise of enabling more realistic and precise drug testing, reducing R&D costs and timescales to bring new therapeutics to the clinic—all while scaling back on animal testing—OoCs are definitely gaining momentum. 

The devices’ potential for drug testing, personalized medicine and disease modelling has captured attention from major industry players—triggering a series of initiatives. 

From research and strategic partnerships to fundings, pilot projects or consortiums. In the course of 5 years, major Big Pharma companies, Contract Services Providers, investors and organ-on-a-chip companies have undertaken collaborative efforts to accelerate the development and utilization of OoCs. 

The list of recent initiatives below—picked from the data collected via our BI platform—is by no means exhaustive. However, it highlights Pharma’s willingness to embrace and develop OoCs—and demonstrates how the technology can actually be embedded into existing R&D workflows across many drug modalities. 


Big Pharma Partnerships 


AstraZeneca and Emulate Partnership | May 2018. After becoming its first Big Pharma partner several years ago, AstraZeneca gave Emulate a major seal of approval by integrating its OoC platform into its own research team in 2018. This deal set up a scientific collaboration to further develop Emulate organ-on-a-chip platform—Human Emulation System™—and foster its use into the everyday operations of its Drug Safety labs.

Bayer and TissUse Partnership | September 2018. Bayer entered into a collaborative agreement with TissUse to develop a liver-endocrine tissues assay leveraging its Multi-Organ-Chip (MOC) technology. The partnership aimed at developing a MOC assay combining target organs and liver in an integrated microfluidic system—which could ultimately be utilized by Bayer researchers. 

Roche and MIMETAS Partnership | July 2021. Roche entered into a collaboration with MIMETAS to develop advanced OoC-based disease models in 2021. A deal that allowed Roche R&D group to access Mimetas proprietary platform—OrganoPlate—in the modelling of inflammatory bowel disease (IBD) and hepatitis B virus infections (HBV).

Bayer Pilot Project | March 2023. Bayer took part in a first-of-its-kind collaboration in the consumer health industry—aiming to develop a platform combining OoC and computational software. With the support of esqLABS, Dynamic42, Placenta Lab of Jena University Hospital, the one-year pilot project’s focus is to generate clinically relevant data—a key step in evaluating new candidates in pre-clinical research. 

Astellas and MIMETAS Partnership | March 2023. Earlier this year, MIMETAS entered into a strategic partnership with Japanese Pharma Astellas—supporting Pharma’s next generation of immune-oncological therapies by giving access to its tumour models. 


Major Contract Services Activity 


Evotec Announces 3D Cell Model Platform | March 2022. In 2022, Evotec announced the development of pre-clinical cellular models based on 3D cell culture platforms—which include OoCs. With this announcement, the German Contract Service Provider also revealed that prescribed 3D in vitro platforms were set to become key elements of its R&D service offering. 

Charles River Joins NXTGEN HIGHTECH Consortium | July 2023. The leading CRO joined a Dutch consortium bringing together more than 330 parties from industry and knowledge institutes to drive the development and standardization of organ-on-a-chip technology. In total, the NXTGEN HIGHTECH program has invested € 1Bn, with a further € 450Mn contribution from the National Growth Fund to advance next generation technologies—including OoCs. 


Private Funding 


MIMETAS Closes $ 20.5Mn Series B | April 2018. In 2018, MIMETAS secured a $20.5 million in Series B financing from investors across Asia and Europe, a substantial milestone following earlier rounds in 2014 and 2015. The funding aimed at advancing MIMETAS organ-on-a-chip and tissue production products, with a long-term goal of making the technology accessible for personalized therapies. 

Emulate Closes $ 82Mn Series E | September 2021. Having already secured close to $ 225Mn in funding since 2013, Emulate later closed a significant Series E round, spearheaded by Northpond Ventures and Perceptive Advisors. The funding was earmarked for robust research and development initiatives, focusing on creating targeted OoC applications following discussions with Big Pharma customers—including Roche, Genentech, Johnson & Johnson, and Gilead Sciences. 

CN Bio Secures Funding and Doubles Lab Size | May 2020 & July 2022. In 2020, UK OoC specialist CN Bio Innovations secured € 8Mn from Chinese investors—with the objective to boost its US market and expand European activities. Two years later, CN Bio doubled its laboratory facilities in Cambridge to meet surging demand for OoCs within the drug discovery and development sector. This move highlights the growing acceptance of OoC data by regulatory agencies and Biopharmaceutical players. 

Vivodyne Closes $ 38Mn Seed Financing | November 2023. Last major move to date, Vivodyne announced the close of $38 million in total seed financing—set to advance its discovery pipeline and clinically predictive AI stack. Through its automated platform, Vivodyne has been able to leverage the scalable interrogation of its lab-grown human OoCs to accelerate scientific advancement for top-10 pharmaceutical companies. 



Current Challenges and Future Perspectives for OoC 


The collaborative efforts and investments mentioned above have strongly backed the growth of the organ-on-a-chip market—accelerating the technology’s shift from theory to its actual utilization in labs. 

Yet, despite major advancements and an increasing acceptance among the Pharma ecosystem, key challenges remain to be solved before considering OoCs implementation on a large scale. 


4 key barriers on the path to widespread adoption 

Standardization: Achieving uniformity and standardization across various Organ-on-a-Chip (OoC) models poses a significant challenge. The diversity in designs and protocols hinders the comparability and reproducibility of results.

Complexity: Developing OoCs that accurately replicate the complexity of human organs is challenging. Mimicking the intricate physiological and mechanical aspects of organs in a microscale environment requires advanced engineering and biological understanding.

Validation: Ensuring that OoCs reliably replicate the physiology of human organs is crucial for their widespread adoption. Rigorous validation processes are needed to establish the accuracy and reliability of OoC models in predicting human responses.

Cost: The initial setup and maintenance costs of OoC technology can be high. Achieving cost-effectiveness while maintaining the sophistication of the technology remains a challenge, particularly for widespread implementation.


Riding a wave of regulatory support towards future 

As paramount tackling those challenges may be to unlock OoC’s full potential, a recent regulatory milestone might turn out to be a powerful catalyst for addressing them. 

The FDA Modernization Act 2.0, signed into law by President Biden in December 2022, marked a transformative shift, as it aims to reduce the dependence on animal models for drug discovery. The legislation promotes researchers to use animal alternatives—such as OoCs and other microphysiological systems (MPS)—when submitting Investigational New Drug (IND) filings to the FDA. 

Riding this wave of regulatory support, the future could hold even brighter perspectives for organ-on-a-chip, as it will encourage further progress within the field: