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AAV Suspension Platforms for Preclinical and Phase I Supply

PROVEN INTELLIGENCE IN SCALABLE VECTOR PRODUCTION.

AAV Suspension Platforms for Preclinical and Phase I Supply

CELL & GENE | RNA | BIOLOGICS

A scientist in a sterile laboratory setting uses a multichannel pipette to transfer pink liquid into a multi-well plate for a high-throughput experiment.

Executive Summary: The transition from research-scale vector production to a scalable platform capable of supplying IND-enabling toxicology studies and Phase I clinical trials presents a significant operational bottleneck. Franklin Biolabs provides robust, high-yield AAV suspension production platforms, leveraging a deep history of process development from the core scientific team that previously operated the Penn Vector Core. Our approach focuses on generating GxP-compliant, well-characterized vector material in single-use bioreactors, ensuring the batch-to-batch consistency required for multi-jurisdictional regulatory submissions, including those aligned with Swissmedic and broader ICH guidelines.

Frequently Asked Questions

How does Franklin Biolabs ensure a smooth transition from research-grade AAV to a GxP-compliant vector suitable for IND-enabling toxicology studies?

Our process begins with the clinical application in mind. We utilize a platform-based approach with established protocols for both upstream and downstream processing that are designed for scalability. By employing the same core methodologies and analytics for research material as we do for preclinical supply, we minimize process-related variability and accelerate the timeline to generate vector under the appropriate GxP conditions required for formal safety and toxicology programs.

What are the primary challenges in developing an AAV scalable suspension process for novel or engineered capsids?

Novel capsids often require specific optimization of transfection parameters, media selection, and purification strategies. Our process development team has extensive experience refining these variables for a wide range of serotypes, including AAV8, AAV9, and custom-engineered vectors. We conduct scale-up confirmation runs to ensure that yield and key quality attributes, such as the percentage of full capsids, remain consistent from 2L development batches to 500L+ production runs.

How does your vector production process align with international regulatory submissions for programs targeting both FDA and European approvals?

We develop manufacturing and analytical documentation to meet harmonized international guidelines (ICH). This ensures that the data package, including vector characterization, purity, and potency assays, is suitable for inclusion in Investigational New Drug (IND) applications as well as Investigational Medicinal Product Dossier (IMPD) submissions for European authorities like Swissmedic or the MHRA.

A reproducible and scalable vector manufacturing process is the foundation of any successful advanced therapy program. For preclinical and early-phase clinical development, AAV suspension culture systems provide the necessary consistency and volume to support comprehensive IND-enabling toxicology studies and subsequent Phase I trials.

Franklin Biolabs utilizes single-use bioreactors, a system designed to mitigate contamination risk while enabling rapid turnaround between production runs. This platform is highly adaptable for batch sizes ranging from 2L to over 500L, providing the flexibility needed to supply programs as they advance.

Process Development for Clinical Readiness

A standard manufacturing template does not exist for AAV vectors. Each serotype and transgene combination requires a tailored, data-driven process development strategy. Our scientific team focuses on key upstream and downstream parameters to enhance vector packaging efficiency and final titer.

  • Upstream Process Development: Optimization of transfection parameters and media feed strategies to maximize vector production in suspension cell culture.

  • Downstream Process Development: Implementation of platform-based or client-specific purification methods to meet stringent quality and regulatory requirements for in vivo use.

  • QC Analytics: A comprehensive suite of platform assays to characterize vector titer, purity, percentage of full capsids, and residual impurities.

This rigorous characterization ensures that the vector supplied for preclinical evaluation is of high quality. The ability to produce vector that mediates long-term, stable transgene expression in nonhuman primate models is a direct indicator of manufacturing robustness, a principle demonstrated in foundational work showing sustained expression for nearly four years post-administration (PMID: 31017018).

The continuity of this expertise is a direct benefit to our clients, as one biotech partner noted:

This collaborative model provides clients with direct access to our team’s deep, integrated expertise across all aspects of vector production and analytics.

This history is reflected in the core team’s 100% successful IND rate since 2019. While Franklin Biolabs as a brand was formally launched in 2024, this track record represents the deep institutional knowledge of our founding scientists and principal investigators in navigating the path to clinical evaluation. For a deeper look at our approach, our webinar on initiating AAV programscovers these topics in detail.

Our work is performed within a >100,000 sq ft facility, providing the infrastructure to support multiple concurrent programs and accelerate timelines, consistently moving candidates toward IND within an 18-24 month window.

For more information on our overarching manufacturing capabilities, please see our primary service page on Large-Scale AAV Manufacturing and Process Development.

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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