A Framework for Preclinical to Clinical Translation
A robust immunogenicity risk assessment integrates data from multiple bioanalytical platforms. This approach provides the Immunogenicity Intelligence for Advanced Therapies needed to inform clinical trial design.
Key components of this framework include:
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Neutralizing Antibody (NAb) Screening: Establishing the prevalence of pre-existing NAbs in the target patient population.
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Anti-Drug Antibody (ADA) Assessment: Developing and validating tiered assays to detect and characterize binding antibodies against both the AAV capsid and the transgene protein.
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T-cell Response Evaluation: Using functional assays like ELISpot or intracellular cytokine staining to measure antigen-specific T-cell activation.
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Data Integration: Correlating bioanalytical findings with non-target tissue biodistribution and Histology data to build a complete safety profile.
Long-term monitoring in preclinical studies is necessary to evaluate the durability of immune responses, a principle underscored in AAV platform development where sustained immunogenicity is a key characteristic (PMID: 33442684). This long-term data directly informs the design of the clinical monitoring plan.
“We started collaborating with UPenn Vector core in 2023 and the AAV vector which they manufactured laid a foundation for development of a gene therapy candidate which will enter soon preclinical studies. The key people from UPenn Vector Core joined Franklin Biolabs and our partnership transitioned without interruption from UPenn Vecor Core to Franklin Biolabs Research Vector Division. Franklin Biolabs is our trusted partner in our AAV-vector based gene therapy candidate development and we hope to continue the partnership for years to come.”
– Biotech Partner
Our programs have supported a 100% IND success rate for our partners since 2019. The Franklin Biolabs brand, launched in 2024, continues this legacy of scientific and regulatory excellence from our >100,000 sq ft GxP-compliant facility.
