Translational Research: Bridging Preclinical Discoveries to Human Trials
Accelerating Drug Development from Bench to Bedside
At 3Biotech, we specialize in translational research, which is the critical process of transforming preclinical discoveries - whether from in vitro assays or animal models - into viable therapeutic candidates for human trials. Our expertise ensures that promising molecules are effective in controlled experimental settings and demonstrate safety, efficacy, and manufacturability in humans.
Translational research is a high-risk, high-impact phase where inadequate planning can lead to clinical failure. Our multidisciplinary approach integrates preclinical pharmacology, toxicology, biomarker identification, and regulatory strategy to optimize the transition from laboratory research to first-in-human (FIH) studies.
From Small Molecule or Biologic Discovery to Human Trials
Translating Small Molecule Candidates to the Clinic
For small molecules derived from chemical synthesis, our translational process is designed so lead compounds can be safely and effectively administered to humans. Our services include:
Predictive Pharmacokinetics & Pharmacodynamics (PK/PD). We use physiologically based PK (PBPK) modeling to extrapolate animal data to human dosing regimens, estimating absorption, distribution, metabolism, and excretion (ADME).
Preclinical Efficacy & Dose Selection. Using validated animal models and ex vivo systems, we determine the minimum effective dose (MED) and maximum tolerated dose (MTD), helping design a safe and effective first-in-human (FIH) trial.
Translational Biomarkers & Mechanism of Action (MoA) Validation. We identify and validate biomarkers that correlate with drug response, providing a quantitative bridge between preclinical and clinical findings.
Safety & Toxicology Studies (Regulatory-Ready). We conduct in vivo GLP toxicology studies, evaluating organ toxicity, genotoxicity, reproductive toxicity, and off-target effects to ensure compliance with EMA, FDA, and ICH M3(R2) guidelines.
Formulation & Drug Delivery Optimization. We develop bioavailable formulations tailored for oral, intravenous, subcutaneous, or novel delivery systems (e.g., nanoparticles, liposomes), ensuring optimal exposure in first-in-human studies.
Regulatory & IND/IMPD Strategy. We compile IND-enabling and IMPD data packages, addressing preclinical pharmacology, CMC development, and clinical trial design, securing smooth regulatory approvals.
By integrating preclinical pharmacology with human modeling, we reduce translation gaps, accelerating the transition from animal models to first-in-human trials with higher confidence.
Translating Biologics and Cell/Gene Therapies to the Clinic
For biologic drugs, including monoclonal antibodies (mAbs), recombinant proteins, cell and gene therapies, and RNA-based therapeutics, translational challenges include immunogenicity, bioprocess scalability, and human-specific efficacy. Our biologics-focused translational research includes:
Species Selection & Human-Relevant Models. Unlike small molecules, biologics often exhibit species-specific pharmacology. We employ humanized models, ex vivo tissue cultures, and organ-on-a-chip technologies to enhance clinical predictability.
Immunogenicity & Safety Risk Assessment. We perform in silico epitope mapping, in vitro T-cell assays, and cytokine release assays to anticipate and mitigate anti-drug immune responses in humans.
PK/PD & Dosing Predictions for Biologics. Biologics exhibit nonlinear pharmacokinetics, requiring population-based PK modeling and target-mediated drug disposition (TMDD) analyses to establish human dosing projections.
Manufacturability & Developability Screening
Cell Line & Expression System Optimization (CHO, HEK, microbial platforms)
Aggregation, stability, and PTM characterization to ensure scalable biomanufacturing
Formulation studies (e.g., liquid vs. lyophilized, buffer selection) to optimize stability for clinical supply
Non-Human Primate (NHP) & Humanized Mouse Studies. Since many biologics lack cross-reactivity in rodents, we conduct non-human primate (NHP) toxicology or humanized mouse studies to simulate human-specific interactions before clinical trials.
First-in-Human Study Design & Regulatory Strategy. We guide FIH trial design, including adaptive dose-escalation plans, patient stratification using biomarkers, and regulatory submission for IND/IMPD approval, ensuring a seamless transition from preclinical to clinical development.
Why Choose 3Biotech for Translational Research?
Integrated Expertise in Both Small Molecules & Biologics
Cutting-Edge Translational Models & Biomarker Strategies
Regulatory Alignment (FDA, EMA, ICH) for IND/IMPD Success
De-Risking First-in-Human Studies with Advanced PK/PD Modeling
At 3Biotech, we bridge the preclinical-clinical gap, ensuring that your promising drug candidates have the best chance of clinical success with a rational, data-driven, and regulatory-compliant approach.
What is translational research in drug development?
Translational research bridges preclinical discoveries to human trials by optimizing drug candidates for clinical evaluation. It includes dose selection, biomarker identification, and first-in-human study design to ensure successful clinical translation.
What is the minimum effective dose (MED) and how is it determined?
The minimum effective dose (MED) is the lowest drug dose that produces a therapeutic effect. It is determined through preclinical dose-response studies, pharmacokinetic modeling, and biomarker analysis.
What are the challenges of translating biologics to the clinic?
Challenges include immunogenicity risks, complex manufacturing, stability issues, and the need for specialized delivery mechanisms. Humanized models and advanced formulation techniques help overcome these barriers.
Why are humanized models important for biologics translation?
Humanized models improve the prediction of a biologic’s efficacy and safety in humans by mimicking human immune responses and metabolism. They reduce the gap between preclinical and clinical outcomes.
How does 3Biotech help de-risk first-in-human studies?
3Biotech helps de-risk first-in-human studies by leveraging deep expertise in preclinical and clinical translation, robust Chemistry, Manufacturing, and Control (CMC) strategies, and meticulous regulatory planning. Our approach includes:
Comprehensive Safety Profiling: Early identification of potential safety concerns through rigorous ADME-Tox assessments, predictive in vitro assays, and relevant animal models.
Strategic Dose Selection: Utilizing pharmacokinetic/pharmacodynamic (PK/PD) modeling, physiologically based pharmacokinetic (PBPK) modeling, and biomarker-driven approaches to establish the safest and most effective starting dose.
Advanced Analytical and Manufacturing Strategies: Ensuring robust, scalable, and compliant manufacturing processes, supported by thorough analytical characterization to guarantee consistent product quality and stability.
Regulatory Expertise: Preparation and management of regulatory submissions (e.g., IND, IMPD), engaging proactively with health authorities to align expectations and anticipate potential issues.
By integrating these strategies, 3Biotech reduces uncertainty and enhances the likelihood of successful and safe clinical translation of your innovative therapies.
What biomarkers are used to bridge preclinical and clinical studies?
Common biomarkers include pharmacodynamic markers, genetic indicators, and surrogate endpoints that help predict clinical efficacy and safety. Biomarkers support dose selection and patient stratification.
How is physiologically based pharmacokinetic (PBPK) modeling used to predict human dosing?
PBPK modeling integrates biological and physicochemical data to simulate drug absorption, distribution, metabolism, and excretion, guiding first-in-human dose selection.