Frequently Asked Questions
How does a platform methodology differ from a standard approach to tech transfer for AAV scalable suspension?
A platform methodology utilizes a pre-established, robustly characterized manufacturing process as a starting point for all vectors within a specific class, like AAVs. Instead of developing a new process from the ground up, we adapt the platform to the specific candidate, significantly shortening timelines. This contrasts with a standard approach, which treats each new vector as a unique project, requiring extensive de novo process development and increasing variability.
Can this platform approach be applied to novel modalities like RNA therapeutics or in vivo CRISPR systems?
Yes. The principles are transferable. For RNA therapeutics delivered via LNPs, a platform approach involves standardized processes for lipid formulation, encapsulation, and purification. While the RNA payload is unique, the delivery vehicle’s manufacturing parameters are well-understood, minimizing clinical risk and accelerating process development under GxP conditions.
What is the primary benefit of this approach for IND-enabling toxicology studies?
The primary benefit is the early and consistent supply of well-characterized, representative material for IND-enabling studies. A platform approach ensures that the vector or LNP produced at small scale for toxicology is manufactured using a process that is directly comparable and scalable for later clinical phases. This consistency is fundamental for regulatory bodies evaluating product safety and quality.
The transfer of manufacturing processes from development to GxP production environments represents a significant variable in program timelines. For next-generation therapies, where each construct can possess unique biological and physicochemical properties, a bespoke, candidate-by-candidate approach to process development introduces unnecessary delays and manufacturing risks.
A platform methodology establishes a data-driven framework. It is built on a deep institutional knowledge of a specific vector or delivery system class. This involves establishing a standardized, phase-appropriate production process : from plasmid supply and cell culture to purification and final formulation : that is known to be robust and scalable for a range of similar candidates.
This foundational understanding allows for rapid adaptation rather than complete reinvention. For example, deep characterization of how AAV9 capsids interact with specific glycans informs a platform-level strategy for engineering new variants with altered tropism (PMID: 39001819). This knowledge enables the predictable modulation of vector properties, such as reducing liver uptake to enhance cardiac targeting, without requiring a full redevelopment cycle. The platform itself predicts the likely impact of specific modifications.
This principle extends across modalities.
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Lentiviral Vectors: A platform approach standardizes transduction protocols and expansion conditions for specific cell types, such as hematopoietic stem cells, allowing for rapid application to new CAR or TCR constructs.
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LNP/RNA Systems: Core platform processes for lipid stock preparation, microfluidic mixing, and purification can be established, allowing for the rapid encapsulation of different mRNA, siRNA, or gRNA payloads.
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Gene Editing: For CRISPR-Cas9 systems, a platform can define the manufacturing and purification of the nuclease and guide RNA components, creating a consistent process for different gene targets.
By starting with a well-understood and optimized process, tech transfer becomes a more defined exercise in parameter adjustment, not a high-risk exploration. This approach is central to the 100% successful IND rate achieved by our core scientific team since 2019, a track record established prior to the formal launch of Franklin Biolabs in 2024. It ensures that the material used in early nonclinical work is truly representative of the final clinical product, a key requirement for global regulatory submissions.
This methodology is a core component of our work with sponsors, including our collaboration with Moderna, to accelerate the path from discovery to clinical evaluation. For more information on our specific capabilities, please see our parent hub page on Vector | CMC | Analytics Services.
