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PEI-compliant preclinical safety and PK studies for gene therapy vectors

PROVEN INTELLIGENCE ACCELERATING NEXT-GENERATION THERAPIES

PEI-compliant preclinical safety and PK studies for gene therapy vectors

PEI-Compliant Pharmacokinetics for Gene Therapy Vectors

CELL & GENE | RNA | BIOLOGICS

    What bioanalytical methods support your gene therapy PK studies?

    A: We employ a suite of validated qPCR and ddPCR assays for quantifying vector copy number in blood, plasma, and tissues. For protein expression, we utilize qualified ELISA and MSD platforms to measure transgene product levels, providing a comprehensive view of both vector distribution and pharmacodynamic effect.

    How does Franklin Biolabs ensure PEI compliance for studies targeting the German market?

    A: Our study designs are aligned with Paul-Ehrlich-Institut (PEI) guidelines from inception. This includes specific recommendations for vector shedding analysis, germline transmission risk assessment, and long-term expression studies. All protocols undergo rigorous review for compliance with both PEI and broader EMA requirements.

    Can PK, biodistribution, and toxicology endpoints be integrated into a single study?

    A: Yes. We specialize in designing integrated, IND-enabling studies that combine PK, non-target tissue biodistribution, and safety toxicology endpoints. This approach optimizes animal use in accordance with the 3Rs and generates a cohesive dataset for regulatory submission, supporting an 18-24 month IND timeline.

    What is the standard sample collection schedule for a non-human primate PK study?

    A: A typical schedule involves intensive sampling within the first 24-72 hours post-dose to capture the distribution phase, followed by less frequent sampling (e.g., weekly, bi-weekly, monthly) to characterize the elimination phase and long-term vector persistence. Schedules are customized based on vector serotype, dose, and route of administration.

To support regulatory submissions to the Paul-Ehrlich-Institut (PEI) and other global authorities, gene therapy programs require robust preclinical pharmacokinetic (PK) and safety data. Franklin Biolabs provides comprehensive, GxP-compliant in vivo studies that characterize the absorption, distribution, metabolism, and excretion (ADME) of AAV vectors. Our integrated approach combines bioanalytical quantification of vector DNA with safety and biodistribution endpoints to build a complete profile for IND submission. We leverage our >100,000 sq ft facility to execute these complex studies, contributing to our 100% IND-enabling success rate for programs initiated since 2019 (the Franklin Biolabs brand launched in 2024).

Defining the Pharmacokinetic Profile for AAV Therapeutics

Characterizing the in vivo behavior of an AAV vector is fundamental to de-risking its clinical development. The PK profile informs dose selection, predicts tissue exposure, and provides a framework for interpreting toxicology findings. Unlike conventional biologics, AAV PK studies must account for vector clearance from circulation, tissue-specific transduction efficiency, and the potential for immune-mediated clearance.

Our study designs focus on generating precise data to answer these questions. We develop customized sampling schedules and bioanalytical strategies to accurately quantify vector copy number in relevant matrices over time. This allows for the construction of a clear PK model that supports clinical translation.

A 3D rendering of Y-shaped antibody molecules against a blue, abstract background.

A close-up of a scientist in a lab, wearing blue gloves and examining the results of a gel electrophoresis or Western blot.

Integrated Safety and PK Study Design

A successful preclinical program generates a cohesive dataset where PK, biodistribution, and safety findings are clearly linked. We design IND-enabling toxicology studies that integrate all necessary endpoints, providing a comprehensive assessment of the therapeutic candidate. This strategy, which incorporates rapid PK profiling to optimize dose selection, supports the development of a strong regulatory package.

Scientific literature reinforces the value of using translational data to establish a favorable safety profile. As demonstrated in efficacy and safety evaluations of AAV-based therapies, generating consistent data across relevant species provides high confidence in the vector platform’s behavior ahead of human trials (PMID: 35333110). This principle guides our approach to designing studies that directly address regulatory expectations for safety and exposure.

“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

Commitment to Animal Welfare and the 3Rs

Our programs are designed to meet the highest ethical standards. All in vivo studies are conducted in USDA-registered facilities and, through our network of strategic partners, are executed in AAALAC-accredited environments.

Our scientific and veterinary staff actively implement the 3Rs (Replacement, Reduction, and Refinement) principles in every study. By designing integrated protocols that capture multiple data streams from a single cohort, we reduce the total number of animals required while maximizing the scientific value of the data.

Scientific Process Diagram

This content is for informational purposes. For guidance specific to your therapeutic program, please contact our team for a consultation.

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