Executive Summary

A global biopharmaceutical company specializing in immunotherapies and vaccines faced a regulatory setback when their novel immunotherapy candidate was rejected due to inconclusive animal model data. To overcome this hurdle, the company leveraged Quantiphi’s DART platform—featuring human Immuno-Microphysiological Systems and AI/ML-driven ImmunoGEN analysis—to generate mechanistic evidence with 100% human relevance. This transformation enabled regulatory approval, improved product localization insights, and provided a scalable solution for both preclinical development and routine potency testing, strengthening the company’s leadership in vaccine and immunotherapy innovation.

About the Client

Industry: Lifesciences
Size: 7,500 employees
Country: Multinational

Problem Statement

During regulatory submission of a novel immunotherapy candidate, the client’s reliance on animal model data proved inadequate. The immune responses observed in animals failed to translate effectively to humans, leading regulators to request detailed mechanistic evidence. Without human-relevant insights into mechanism of action (MOA), regulatory approval and further development were at risk.

Challenges

Inconclusive animal model data hindered regulatory submission.
Animal immune systems failed to represent human physiological conditions.
Regulatory authorities demanded mechanistic validation with human relevance.

The Solution

Quantiphi’s DART platform, developed in partnership with Transcell Biologics, provided an integrated, non-animal solution:

Human-Relevant Models: Leveraged human Immuno-Microphysiological Systems replicating immune activation and memory signatures.
AI/ML-Driven Insights (ImmunoGEN): Decoded mechanistic action, potency, and product-specific localization.
Regulatory-Endorsed NAM: Recognized as a valid New Approach Methodology (NAM) for surrogate clinical efficacy insights.
Scalable Deployment: Supported preclinical evaluation and routine potency testing within the client’s workflows.

Impacts

Obtained regulatory approval for decoded MOA data.
Achieved 100% relevance to the human immune system.
Reinforced position as a global leader in immunotherapy and vaccine innovation.
Enabled application across preclinical and routine potency testing.

Technologies Used

DART (Digital Animal Replacement Technology)

DART (Digital Animal Replacement Technology)

Human Immuno-Microphysiological Systems

AI/ML-driven data analysis (ImmunoGEN)

Results

Decoded drug candidate’s mechanism of action with 100% relevance to human immune biology.

Revealed mechanistic insights linked to potency and product-specific localization.
Enabled improved vaccine and immunotherapy formulation strategies through tailored antigen delivery.
Received positive regulatory feedback for the human-relevant data package.
Successfully transferred validated bioassay SOPs into the client’s internal testing workflow.