Executive Summary

For European biotech sponsors, generating definitive preclinical data for an Investigational Medicinal Product Dossier (IMPD) submission requires research-grade AAV vectors of high quality. This asset details the production of vectors for non-target tissue biodistribution and safety characterization, ensuring the data generated is reproducible and suitable for regulatory review under GxP principles. Our process, refined over decades and now operating within our >100,000 sq ft facility, is designed to move candidates toward IND-enabling studies on an 18-24 month timeline.

Vector Production for Preclinical Regulatory Success

Preclinical AAV studies must build a comprehensive safety and biodistribution profile to support regulatory submissions. Regulatory bodies in the EMEA region require clear data on vector sequestration and potential immunogenicity before authorizing first-in-human trials. The quality of the research vector used in these foundational studies directly impacts the integrity of the data submitted.

A well-characterized vector provides a clear understanding of its biological activity. For instance, studies have established that certain AAV serotypes exhibit predominant liver sequestration, a factor that informs the safety profile and potential for off-target effects (PMID: 24070336). Similarly, understanding how AAV vectors can avoid inflammatory signals that trigger destructive T-cell responses is a key consideration in designing clinical trials and interpreting preclinical immune response data (PMID: 20234342).

A Legacy of AAV Manufacturing Expertise

The Franklin Biolabs research vector team continues the work of the Penn Vector Core, bringing decades of institutional knowledge in AAV, lentivirus, and adenovirus production. This continuity ensures that programs initiated at the university transition without interruption, maintaining scientific momentum. Our established processes and deep expertise in packaging novel and refractory capsids provide the technical foundation for successful preclinical programs.

Our team provides comprehensive scientific and technical support, leveraging deep expertise in all aspects of vector production and analytics.

Commitment to Animal Welfare in Preclinical Research

All in vivo applications of our research vectors are predicated on a commitment to the highest standards of animal welfare. Our approach is aligned with AAALAC and USDA guidelines, strictly adhering to the 3Rs Principle (Replacement, Reduction, and Refinement) to ensure ethical and responsible research conduct.

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For a deeper look into our animal welfare programs and facility standards, .

Our production services include:

• Flexible Plasmid Services: We offer a full range of DNA support, including cloning, amplification, and characterization for your specific gene of interest.

• Scalable Production: Manufacturing scales are customized to meet the specific needs of your in vivo or in vitro research budget and experimental design.

• Advanced Purification: Vectors are purified using iodixanol gradient ultracentrifugation or chromatography to achieve the high purity required for in vivo experiments with low endotoxin levels.

• Comprehensive Analytics: A wide range of QC analytics options are available to characterize vector titer, purity, and identity, providing a robust data package for your records.