Proven Intelligence Accelerating Next-Generation Therapies.

Executive Summary

Transitioning an adeno-associated virus (AAV) program from an academic research setting to a scale suitable for UK clinical trials requires a significant shift in process development, analytical rigor, and manufacturing strategy. This involves moving beyond research-grade materials to a GxP-comparable framework capable of generating the robust data package required for an Investigational Medicinal Product Dossier (IMPD) submission to the MHRA. Franklin Biolabs provides scalable suspension and adherent AAV production platforms, from 2L to 500L+, supported by a team with direct lineage from the Penn Vector Core. Our approach is designed to achieve program milestones within an 18-24 month IND timeline, building on a history that includes a 100% successful IND rate since 2019 for programs we have supported (Franklin Biolabs as a brand launched in 2024).

Bridging the Academic-to-Clinical Manufacturing Gap

Many promising viral vector programs originate in academic labs, where production is often sufficient for initial in vivo proof-of-concept. The transition to a clinical-grade manufacturing process, however, presents a distinct set of technical and regulatory challenges. The objective is to establish a robust, scalable process that consistently produces high-quality AAV vectors suitable for formal preclinical toxicology and human trials.

This transition is a core competency at Franklin Biolabs. Key scientific and operational personnel from the Penn Vector Core have transitioned to Franklin Biolabs, ensuring continuity of expertise for programs that began at the university. This lineage provides an established foundation for advancing AAV candidates.

“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 provides the critical manufacturing and scientific support for our AAV-vector based gene therapy candidate development, and we hope to continue the partnership for years to come.”
— Biotech Partner

Phase-Appropriate Process Development for IMPD Submissions

For UK-based sponsors, the CMC section of an IMPD is a point of intense regulatory scrutiny by the MHRA. A manufacturing process that is not well-characterized or scalable can introduce significant delays. We focus on developing a manufacturing process that is appropriate for the stage of development.

• Platform Selection: We offer both adherent (iCELLis®, scale-X™) and suspension (Sartorius single-use) platforms, allowing us to select the optimal system based on the specific AAV serotype, target yield, and client’s long-term manufacturing strategy.

• Process Optimization: We refine upstream parameters like transfection conditions and media selection, alongside downstream purification methods, to enhance vector packaging efficiency, titer, and purity.

• Analytical Rigor: A comprehensive suite of qualified QC assays provides the data needed to demonstrate product consistency, including vector genome titer, capsid purity, and percent full capsids.

Data-Driven Vector Characterization

A well-defined manufacturing process is directly linked to predictable vector performance. As demonstrated in preclinical evaluations of AAV-based candidates, a consistent production platform is foundational for achieving predictable immunogenicity profiles (PMID: 33442684). This consistency is a primary requirement for regulatory bodies like the MHRA.

Determining the minimally effective dose of a vector requires precise and reproducible analytical characterization (PMID: 30112420). Inaccurate titer or impurity measurements can confound preclinical results and complicate clinical dose selection. Our analytical development team specializes in creating and qualifying gene-of-interest-specific ddPCR and cell-based potency assays to support these activities.

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