Franklin Biolabs Achieves CLIA Certification for AAV NAb Testing
Learn more

Custom Potency Assays for Novel Gene Editing Platforms

PROVEN INTELLIGENCE ACCELERATING NEXT-GENERATION THERAPIES

Custom Potency Assays for Novel Gene Editing Platforms

CELL & GENE | RNA | BIOLOGICS

Proven Intelligence in Custom Assay Development.

A marketing flyer for Franklin Biolabs detailing their immunogenicity services, including various assays and support for cell and gene therapy clinical trials.

Frequently Asked Questions

Why is a custom potency assay necessary for a CRISPR-based therapeutic?

Standardized assays often fail to capture the specific mechanism of action (MOA) of novel gene editing systems like CRISPR-Cas9, base editors, or prime editors. A custom, MOA-reflective potency assay is required by regulatory bodies (FDA, EMA) to demonstrate that the product consistently produces its intended biological effect, which is fundamental for lot release and clinical use.

What are the key considerations for developing potency assays intended for global IND or IMPD submissions?

For global submissions, assays must be developed with harmonized international guidelines (ICH) in mind from the outset. This involves phase-appropriate validation, robust documentation under GxP conditions, and ensuring the assay is sensitive, specific, and reproducible across different manufacturing lots and testing sites.

How does Franklin Biolabs approach the development of a cell-based potency assay for a new gene editing platform?

We design a data-driven strategy tailored to the asset’s specific modality and target indication. This begins with selecting a biologically relevant cell line, defining the editing event, and developing quantitative endpoints, such as NGS for editing efficiency or functional readouts that measure downstream protein expression or cellular function.

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

For next-generation therapies utilizing gene editing platforms like CRISPR-Cas9 or base editors, a generic, one-size-fits-all potency assay is insufficient for regulatory submission. The development of a custom, biologically relevant, and mechanism-of-action (MOA)-reflective assay is a foundational component of a successful CMC strategy. These assays provide the definitive link between the product’s structural attributes and its intended clinical function, forming the analytical backbone for product characterization, lot release, and stability programs.

Defining Biological Activity Beyond Simple Expression

Potency assays for gene editing platforms must quantitatively measure the intended biological function. This moves beyond simple surrogate markers to directly assess the molecular event. The selection of an appropriate analytical method is determined by the specific editing strategy.

  • Nuclease Platforms (CRISPR-Cas9, Cas12a): Assays often focus on quantifying the frequency of insertions and deletions (indels) at the target locus using Next-Generation Sequencing (NGS).

  • Precision Editors (Base/Prime Editors): The analytical endpoint must confirm the specific, intended base conversion or sequence insertion while simultaneously demonstrating the absence of unintended edits.

  • Reporter Systems: While useful for early-stage screening, the choice of a reporter must be carefully considered. As demonstrated in AAV-mediated gene transfer studies, reporter expression can be highly tissue-specific and may not always correlate with the therapeutic protein’s activity, underscoring the need for biologically relevant models (PMID: 17510373).

A blue-toned image of white lab rats in their cages within a laboratory or vivarium setting, likely for scientific research or testing.

A Phase-Appropriate Path to a Validated Assay

A robust potency assay evolves with the therapeutic program. The initial research-grade assay used for candidate selection requires significant refinement and qualification before it is suitable for GxP-compliant testing. This process is managed within our >100,000 sq ft of specialized laboratory space.

Our approach ensures that the assay is developed and validated in a phase-appropriate manner, meeting the expectations for multi-jurisdictional IND and IMPD submissions. This integrated strategy is central to achieving an 18-24 month timeline to IND. The scientific leadership and core team at Franklin Biolabs, which formally launched in 2024, have maintained a 100% successful IND rate since 2019 by building these foundational analytics correctly from the start. A well-designed vector with demonstrated biological activity is directly linked to positive in vivo outcomes, a principle validated in the development of gene therapy vectors for neurological disorders.

For more information on our comprehensive analytical capabilities, please see our core services.
Vector | CMC | Analytics Services

Scientific Process Diagram

This content is for informational purposes. For guidance specific to your therapeutic program, please contact our team for a consultation.

Explore More Capabilities