Are you facing these Eficacy Screening Challenges?

Species-Specific Immunology: Animals and humans differ vastly in innate and adaptive immune cell signaling, making animal models to be designed specifically for testing modern immunotherapies, monoclonal antibodies, and vaccines.

Metabolic Rate Discrepancies: Laboratory animals possess significantly higher basal metabolic rates than humans, leading to accelerated drug clearance, altered pharmacokinetics (PK), and distorted dosing calculations that needs to be calculated.

Target Receptor Variation: Human drug targets (receptors, enzymes, or ion channels) often exhibit structural differences or altered tissue distribution patterns in animals, causing unexpected off-target or weak on-target efficacy which requires the use of humanized models.

Epigenetic Model Design: Animals do not naturally develop human diseases like Alzheimer’s, HIV, or specific cancers, forcing researchers to use genetically modified variants that omit the full human genetic background and therefore a epigenic model design needs to be considered

Get a Robust Study Design to match your clinical trial outcomes

The key is to treat your preclinical screening as a "Phase 0" simulation that strictly mirrors the patient selection, exposure metrics, and clinical endpoints of a human trial.

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Choose the right model background for your target market

Match the Molecular Architecture (Mechanism of Mutation)
• Gain-of-Function (GoF): If the human disease is driven by an overactive protein (e.g., BRAF V600E in melanoma), use Knock-In (KI) models. Avoid transgenic over-expression models, which insert random copy numbers and cause artificial, non-human phenotypes.
• Loss-of-Function (LoF): If the disease is driven by a tumor suppressor or inactivated enzyme, use Knock-Out (KO) or conditional KO models.
• Gene Fusions: Replicate human chromosomal translocations (e.g., BCR-ABL1 or EML4-ALK) using CRISPR-Cas9 to induce targeted double-strand breaks that force endogenous genomic rearrangements

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Muscle Weakness and Spasticity

A broad range of endpoints to match your clinical trial success

1. Know your Clinical-Grade Endpoints

Multiplexed Immunohistochemistry (mIHC/mIF): Use cycles of antibody staining and stripping (or metal-conjugated antibodies via Imaging Mass Cytometry) to evaluate 10 to 40+ markers on a single tissue section.
Clinical Phenotyping: Mirror clinical pathology by mapping structural spatial relationships (e.g., measuring the exact distance in microns between cytotoxic T-cells and tumor cells to predict clinical immunotherapeutic response) rather than just counting cells.

2. For your Neural Circuit assays measure Network-Level Screening

In Vivo Local Field Potentials (LFPs): Implant chronic, multi-channel depth electrodes into targeted brain regions (e.g., hippocampus, prefrontal cortex) of freely moving genetic animal models.
Translational Metric: Measure specific frequency bands (alpha, beta, gamma, theta oscillations). For example, screen for Parkinson's therapeutics by tracking the reduction of pathological beta-band oscillations, directly mirroring human deep brain stimulation (DBS) clinical readouts.
Continuous EEG Telemetry: Replicate human clinical electroencephalography (EEG) using fully implantable wireless telemetry transmitters in freely moving animals.
Translational Metric: Track Sleep-Wake Architecture (REM/non-REM tracking), Spike-Wave Discharges (SWD), and Event-Related Potentials (ERPs) over weeks to match clinical epilepsy or sleep disorder trial protocols.

3. Add credibility to your Data with Fluid and Liquid Biopsy Biomarkers

• Circulating Tumor DNA (ctDNA) & Cell-Free DNA (cfDNA): Track disease burden longitudinally via simple blood draws using digital droplet PCR (ddPCR). A drop in cfDNA/ctDNA serves as an immediate surrogate endpoint for clinical molecular response.
• Multiplex Protein/Cytokine Panels: Measure serum biomarkers that match human trial entry or efficacy criteria (e.g., serum creatinine for renal function, troponin for cardiac safety, or specific interleukins for inflammatory diseases).
• Surrogate Organ Function Panels: Run automated clinical chemistry panels (ALT, AST, ALP, Bilirubin) to monitor liver and systemic metabolic health exactly like a Phase I/II safety and efficacy protocol.

4. Do you need Tissue-Level Molecular Endpoints that can validate mechanisms?

When scaling your animal study to simulate a clinical trial, biological validation at the tissue level must match the regulatory requirements of human biopsies.
• Quantitative Digital Pathology: Replace manual "blinded scoring" with AI-driven, automated immunohistochemistry (IHC) quantification. Measure exact positive cell percentages, spatial distribution, and co-localization of drug-target interactions.
• Spatial Transcriptomics / Single-Cell RNA-Seq: Perform punch biopsies on treated animal tissues to verify that the drug is driving the exact downstream gene expression shifts observed in successful human pilot studies.

5. Validate your compounds through Functional and Behavioral Endpoints

For neurological, muscular, or metabolic diseases, survival is an inadequate endpoint. You must measure quality-of-life and physical function.
• Neurological / Motor Trials: Use automated, video-tracked gait analysis (e.g., CatWalk systems), grip strength meters, or rotarod testing to establish a baseline and track functional recovery.
• Cognitive Assessment: Use touch-screen operant chambers or automated mazes to test memory and executive function, eliminating human handling bias.
• Cardiorespiratory Fitness: Utilize metabolic cages (CLAMS) to measure oxygen consumption (VO₂ max), carbon dioxide production, and energy expenditure under therapeutic duress.

Custom Screening! Social Isolation during a group activity for major depressive disorder

Identifying your Clinically Relevant Compounds using our HTS system

A molecule is highly likely to fail Phase II clinical trials if you cannot objectively prove three fundamental pharmacology milestones during preclinical development.

1. Pillar 1 (Exposure): The molecule reaches the target site of action (verified via tissue biodistribution studies, quantitative whole-body autoradiography, or microdialysis) over a time window that matches the intended human dosing frequency.
2. Pillar 2 (Binding): The molecule binds to the target human protein in the intact physiological environment (verified via ex vivo receptor occupancy assays, thermal shift assays, or live-cell imaging).
3. Pillar 3 (Functional Activity): Target binding drives the exact downstream molecular expression shifts or physiological changes required to alter the human disease state (verified via spatial transcriptomics or serial biomarker tracking).

Be worry free by Establishing the Preclinical "Three Pillars" of Efficacy