CMC Analytics Consulting for Gene Editing Systems

PROVEN INTELLIGENCE IN CMC ANALYTICS FOR GENE EDITING SYSTEMS.

Consultation on CMC Analytics for Gene Editing Systems in the Swiss Market

CELL & GENE | RNA | BIOLOGICS

Frequently Asked Questions

What are the primary CMC analytical challenges for CRISPR-based therapeutics targeting the Swiss market?

For Swissmedic and broader EMA submissions, the analytical focus for CRISPR systems is twofold: definitively characterizing on-target potency and quantifying any potential for off-target editing events. This requires a matrix of orthogonal assays, including next-generation sequencing (NGS) to confirm gRNA integrity, mass spectrometry for nuclease characterization, and validated cell-based assays to measure functional protein knockout or correction.

How does Franklin Biolabs align its analytical strategy with international guidelines like ICH for a global submission?

We develop analytical control strategies with global regulatory expectations in mind from the outset. By adhering to ICH guidelines (e.g., Q2 for validation, Q5E for comparability), we ensure the data package for your gene editing system is robust enough for simultaneous or sequential submissions to Swissmedic, the EMA, and the FDA, minimizing redundant testing and accelerating global timelines.

For an early-stage gene editing program, how do you approach phase-appropriate assay development under GxP conditions?

A phase-appropriate strategy is fundamental. For early preclinical and IND-enabling work, we focus on developing and qualifying robust assays that provide clear data on product identity, purity, and potency. These methods are designed with future validation in mind, ensuring a smooth transition as the program advances toward clinical phases without requiring a complete redevelopment of the core analytical framework.

A stylized, 3D rendering of a DNA double helix in light blue and white, set against a soft-focus, light gray background.

Executive Summary

For sponsors of gene editing systems in Switzerland and the EU, establishing a robust Chemistry, Manufacturing, and Controls (CMC) analytical package is the foundation for a successful Investigational Medicinal Product Dossier (IMPD) submission. The analytical strategy must rigorously characterize the nuclease platform (e.g., CRISPR-Cas9, Base/Prime Editors), the guide RNA, and the delivery vehicle. Key considerations include quantifying on-target editing efficiency, ensuring the absence of significant off-target events, and developing phase-appropriate potency assays that are predictive of the therapeutic mechanism of action.

A Tailored Analytical Framework for Gene Editing Systems

A standard analytical template does not apply to the complexity of gene editing platforms. Each system : comprising the nuclease, the guide RNA, and the delivery vector (viral or non-viral) : presents a unique characterization challenge. Our approach is to build a bespoke analytical control strategy that directly addresses the specific risks and mechanism of action for your therapeutic candidate.

This involves a multi-faceted approach to characterization:

  • Nuclease & Guide RNA Integrity: Verifying the identity, purity, and stability of the core editing components using methods like mass spectrometry and next-generation sequencing.

  • On-Target Potency: Developing and qualifying functional, cell-based potency assays that accurately reflect the intended biological outcome, whether it is gene knockout, correction, or insertion.

  • Off-Target Event Quantification: Employing unbiased, sensitive methods to detect and quantify potential guide-dependent and guide-independent off-target editing in non-target tissue biodistribution assessments.

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

Translating Preclinical Data into a Coherent Regulatory Narrative

Demonstrating long-term safety and durable efficacy in relevant preclinical models is a key objective supported by the CMC analytical package. Data from nonhuman primate models showing stable, long-term target engagement, such as the sustained reduction of PCSK9 observed in AAV-delivered meganuclease studies (PMID: 33609733), provides a powerful proof-of-concept. The underlying analytical methods that characterize the vector and its payload are what give regulators confidence in the translation of these results.

Our scientific team possesses deep experience in this area. As one biotech partner noted, we provide “Vast knowledge in all aspects of vector production and analytics.” This expertise is housed within our >100,000 sq ft of dedicated laboratory and preclinical facilities. While Franklin Biolabs was formally launched in 2024, our core scientific leadership and principal scientists have achieved a 100% successful IND rate since 2019, consistently delivering data packages that meet the stringent requirements of global health authorities, including Swissmedic. This track record is built on a foundation of rigorous, well-characterized analytical science that de-risks the path to clinical evaluation.

This specialized work is a core component of our broader 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.

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