Disease Biology

Only 1-2% of drug candidates make it from preclinical studies to a marketed drug. Not because of a lack of ideas or effort, but because the drug discovery industry is optimized to manage failure rather than enable success. As a preclinical pharmacology services CRO, we empower every innovator with an ecosystem of success designed to deliver the fastest path to the best decision.

Through integrated preclinical drug efficacy, target validation and engagement, and translational biomarker and pharmacology services, we help you move beyond momentum‑driven science toward confident, biology‑led success. We quickly assess the effectiveness of drug action in the right disease context, combining smart data generation, deep disease understanding and expertise, and translational models and services.

Drug Pharmacology Services

Oncology & Immuno-Oncology Pharmacology Services

End-to-end portfolio of 2D/3D cell models and clinically meaningful in vivo systems, from PDX to humanized models, designed to enhance translational relevance and confidence in outcomes.

Neuroscience Pharmacology Services

Tailored neuroscience studies combine comprehensive in vitro and in vivo models and state-of-the-art methods and readouts.

Immunology and Autoimmune Pharmacology Services

Immunomodulatory therapies require translational models that accurately reflect human disease, appropriate biomarkers, and a deep understanding of complex immune biology.

Infectious Disease Pharmacology Services

An extensive suite of methods and models support biology-led development of antimicrobials, antivirals and vaccines for infectious diseases.

Cardiovascular & Metabolic Pharmacology Services

Comprehensive models and assays across key cardiovascular and metabolic disorders empower your drug discovery.

Rare Disease Pharmacology Services

Therapeutic development strategies for rare and N of 1 disorders are as unique and varied as the 7000+ identified rare diseases.

Preclinical Drug Efficacy & Pharmacology Services

Target Engagement in Drug Discovery

Target Validation Services

Integrating Pharmacodynamics and Pharmacokinetics

Proving Therapeutic Potential
A candidate that cannot translate is not a candidate; it is a sunk cost. Drug efficacy studies and pharmacology services must demonstrate not just activity, but meaningful therapeutic impact in biological models that translate. Our efficacy studies are explicitly designed to support decision making, ensuring your candidate has the right pharmacological profile to succeed, not just advance.

In Vitro and Ex Vivo Drug Efficacy

Translational human cell models, including 2D, 3D, complex co-culture, iPSC, patient and donor derived cells
Deep expertise in complex cell culture, including genetic engineering, primary and patient-derived cell lines
Disease-relevant functional and phenotypic cell assays
AI and ML-enabled imaging analysis workflows
Systematic design of experiment (DoE) to optimize cell-based assay cascades

In Vivo Pharmacology and Efficacy Services

Extensive predictive in vivo disease models across key therapeutic areas
- Industry-leading cancer model database and pediatric PDX collection
- Humanized models bridge the preclinical to clinical translation gap
- Best in class neurodegenerative, neuroimmune, mental and pain disorder models
Forerunner in translational in vivo imaging in drug discovery
Integration of pharmacodynamic (PD) and pharmacokinetic (PK) observations
- Repeated in vivo analyte sampling by microdialysis enables measurement of endogenous and exogenous small and large molecules
- LCMS for highly sensitive detection of biomarkers and test articles in samples
Formulation, dose schedule, and route of administration optimization
Time course studies to understand durability and reversibility of response
Complex ex vivo endpoints and tissue analysis
Mechanism anchored biomarkers to support translational relevance
IND-enabling study formats with reporting ready for submission

Proving Biological Mechanism, Not Just Exposure

Target engagement confirms that a compound is doing what it was designed to do, at the right dose, in the right tissue, at the right time. Without it, efficacy data lacks context and credibility. Our target engagement studies are designed to create a clear mechanistic chain from exposure to biology to therapeutic outcome.

Target Engagement Strategies

Direct engagement in biochemical and biophysical binding and affinity assays, as well as cellular target occupancy measurements
- Biophysical assays to measure drug-target engagement such as isothermal titration calorimetry, surface plasmon resonance (SPR), and thermal shift assays (TSA)
- Ligand binding assays to measure interaction, affinity, and binding kinetics between therapeutics and their target
Indirect engagement indicators such as pathway modulation, downstream biomarkers, and functional signaling pathway readouts
- Gene and protein measurements
- Omics data generation, including RNAseq and proteomics
In vivo engagement by tissue specific measurements and integrated PK/PD modeling

Confirm the Right Target

Target validation is not about checking a box. It is about answering the most important early question in drug discovery: Is the drug target involved in disease biology? We combine deep disease expertise with human led, data superpowered analysis to assess biological relevance, challenge assumptions, and validate targets before time, capital, and momentum are misdirected.

Target Validation Capabilities

Genetic validation
- CRISPR/Cas9 knockout and knock in
- RNAi knockdown
- Target overexpression systems
Disease relevant in vitro models
- 2D and 3D systems, iPSC derived cells
- Co culture and complex human relevant platforms
In vivo proof of biology
- Predictive disease models aligned to human pathology

Understanding the Relationship Between Exposure and Response

Our pharmacology studies are designed for translation and are built around predictive biology, not convenience models. We combine PK (pharmacokinetics; what the body does to a drug) and PD (pharmacodynamics; what a drug does to the body) readouts in expertly designed studies to quantify the relationship between drug exposure and therapeutic effect over time.

Our capabilities include:

Pharmacokinetic profiling across relevant dose routes and schedules
PK/PD modeling to define exposure–response relationships
Integration with translational biomarkers and efficacy endpoints

Why Work With Us For Drug Pharmacology Services?

Drug discovery should be more accessible, affordable, and productive. Yet for decades, the industry has operated within an orthodoxy that scales and perpetuates failure, encouraging innovators to “fail faster” rather than questioning why failure is so prevalent in the first place. We are breaking with that orthodoxy.

We design pharmacology studies with integrated pharmacokinetics and pharmacodynamics to directly link exposure, dose, target engagement, and biological effect
We combine human-relevant cell models, fit for purpose in vivo models, cutting-edge technologies, rigorous experimental design, and deep expertise
We offer diverse models, technologies, and expertise across many therapeutic areas, modalities and targets
Deep translational biology expertise across oncology, neuroscience, immunology, infectious disease, cardiovascular and metabolic diseases, and rare disorders
Platforms supporting small molecules, biologics, and novel modalities, with flexibility to adapt to first in class and high uncertainty programs
We combine expertise with human-led application of AI and machine learning, reducing timelines for generation of decision-making data

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Innovation in Pharmacology Services

Progress in drug discovery and drug pharmacology demands innovation. Our teams are committed to the development of new models, technologies, readouts and biomarkers to change the pace of drug discovery. Some of our areas of innovation include:

Development of AI and ML-enabled workflows to ensure refined, robust and reproducible data analysis
Image analysis workflows for in vitro and in vivo imaging technologies
Automated behavioral data analysis pipeline
Expansion of radiopharmaceutical capabilities, combining extensive expertise in oncology models and nuclear imaging, to enable fully in-house pipelines from radio-labeling of your test articles, through in vivo imaging, to complex imaging data analysis
Continued development of humanized models for oncology, immuno-oncology and autoimmune disorders, to bridge the translational gap between in vitro assays and human trials
Continuous expansion of tumor model to reflect patient populations and disease biology, including pediatric PDX models
Development of complex 2D and 3D cell models, including immune competent models, to support improved translational disease modeling

What is target validation in drug discovery?

Target validation studies confirm that your drug target, a specific gene or protein, is directly involved in disease biology and pathology, giving an indication that modulating it will have a therapeutic benefit for patients. In drug discovery, core approaches such as genetic manipulation, pharmacological profiling, and target and biomarker expression profiling de-risk your target and ensure your drug candidate demonstrates efficacy.

What is target engagement in drug discovery?

Target engagement is the measurable binding of a compound and its intended biological target, to verify interaction and modulation of the desired biochemical pathways. Techniques verify that a drug interacts with its target and measure binding affinity. Insufficient target exposure and engagement are leading causes of drug discovery failure.

What is drug efficacy in drug discovery?

Preclinical drug efficacy studies assess a drug's effectiveness in the appropriate disease context, indicating whether it will be efficacious in its target patient population. This goes beyond the physical binding of the drug to its target, to measure biological and therapeutic response.