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Swissmedic-Aligned IND-Enabling Toxicology Packages for Novel Gene Therapy Vectors

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Swissmedic-Aligned IND-Enabling Toxicology Packages for Novel Gene Therapy Vectors

Swissmedic-Aligned IND-Enabling Toxicology for Gene Therapy Vectors

CELL & GENE | RNA | BIOLOGICS

    How do you align toxicology study design with Swissmedic’s specific requirements for AAV vectors?

    A: Our study designs are built upon direct experience with Swissmedic submissions. We focus on endpoints relevant to the vector serotype and transgene, including comprehensive biodistribution, shedding analysis, and immunogenicity assessments tailored to European agency expectations.

    What is the typical scope of a non-human primate (NHP) study for a novel AAV vector?

    A: The scope for an NHP study includes dose-range finding followed by a pivotal GxP-compliant study. This involves detailed clinical observations, clinical pathology, and terminal collection of a full tissue set for biodistribution (qPCR) and Histology to evaluate potential on-target and non-target tissue toxicities.

    How do you address potential species-specific differences in biodistribution?

    A: We incorporate early, multi-species comparative studies and leverage advanced vector engineering insights. Understanding how biological factors differ between species allows us to select the most translationally relevant model and interpret biodistribution and safety data with higher confidence for human clinical trials.

    What are the key components of the data package for a Swissmedic IND submission?

    A: The package includes the full study protocol, all raw and analyzed data from in-life and terminal phases, a comprehensive toxicology report with a safety assessment, and detailed reports on biodistribution, shedding, and any required immunogenicity or biomarker analysis.

Executing a Swissmedic-aligned Investigational New Drug (IND) enabling toxicology package for novel adeno-associated virus (AAV) vectors requires a specific framework. We provide comprehensive, GxP-compliant in vivo studies within our >100,000 sq ft facility, designed to generate a robust data package for regulatory submission. The typical program is structured to support an 18-24 month timeline from vector production to final IND dossier compilation.

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De-Risking AAV Development for Swiss Regulatory Submission

Navigating the regulatory requirements for gene therapy vectors demands a toxicology program that is both scientifically robust and precisely aligned with agency expectations. For sponsors targeting Swissmedic approval, this requires a deep understanding of the agency’s perspective on vector safety, biodistribution, and potential immunogenicity. The objective is to build a data package that proactively addresses potential clinical risks.

A core element of this process is characterizing the vector itself. Minor modifications to the AAV capsid can significantly alter its biological activity and safety profile. High-Fidelity In Vivo Intelligence for Complex Modalities. Our programs are designed to elucidate these properties early, ensuring the toxicology studies are relevant to the final clinical candidate.

Translating Vector Biodistribution Across Species

A primary challenge in preclinical development is the translation of biodistribution data from animal models to humans. Research into AAV9 vector engineering highlights the importance of this step. Studies show that modifying capsid domains to alter properties like galactose binding can successfully detarget the liver and improve uptake in other tissues, such as the heart (PMID: 39001819).

This same research also reveals that the glycan presentation on tissues can differ significantly between species. Such findings underscore the need for careful model selection and data interpretation. A toxicology program must account for these potential cross-species differences to generate a data set that is truly predictive of vector behavior in patients. Our approach integrates these biological principles into study design, strengthening the rationale for the chosen animal model and the interpretation of non-target tissue biodistribution data.

“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

Since 2019, programs led by our scientific teams have maintained a 100% IND success rate, a record of quality and regulatory expertise we continue at Franklin Biolabs, which launched as a new brand in 2024.

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Animal Welfare and Regulatory Adherence

Our programs operate under strict ethical guidelines, adhering to standards set forth by the USDA and aligning with the principles of animal welfare championed by organizations such as AAALAC.

We rigorously apply the 3Rs principles (Reduction, Refinement, and Replacement) to every study. This includes using advanced statistical methods to minimize animal numbers, refining procedures to enhance welfare, and continuously evaluating alternative methods.

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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