Preclinical Gene Editing Analysis
Gene editing has transformed gene and cell therapy research by enabling targeted genetic modifications in both ex vivo and in vivo settings. In preclinical development, ensuring the stability, distribution, and consistency of edits is critical for evaluating safety and therapeutic performance. Gene editing stability analysis verifies that modifications remain consistent and measurable over time, supporting regulatory-enabling studies and advancement toward clinical translation.
Preclinical Gene Editing Analysis
Gene editing has transformed gene and cell therapy research by enabling targeted genetic modifications in both ex vivo and in vivo settings. In preclinical development, ensuring the stability, distribution, and consistency of edits is critical for evaluating safety and therapeutic performance. Gene editing stability analysis verifies that modifications remain consistent and measurable over time, supporting regulatory-enabling studies and advancement toward clinical translation.
Our Expertise
With deep expertise supporting preclinical gene and cell therapy programs utilizing CRISPR, Zinc Finger Nucleases, and other gene editing technologies, Avance Biosciences applies a combination of NGS amplicon sequencing, ddPCR, and qPCR to characterize editing outcomes in animal models.
We analyze edited genes in tissues following real-time PCR confirmation of biodistribution. Our approach integrates molecular characterization with quantitative tissue analysis to support pharmacokinetic and pharmacodynamic studies. This comprehensive strategy enables precise validation of gene edits in both ex vivo–modified cell therapies and in vivo editing programs.
Preclinical Safety Studies
We have extensive experience supporting clients with gene editing site characterization using NGS amplicon sequencing for preclinical PK/PD studies. Our NGS amplicon assays are developed with defined analytical performance criteria suitable for regulatory submissions.
Following qPCR or ddPCR-based biodistribution studies, tissues demonstrating presence of edited material are selected for quantitative NGS amplicon sequencing. We assess the consistency of indels and point mutations at defined on-target and candidate off-target loci and report their relative abundance across tissues and timepoints.
For in vivo editing programs, including lipid nanoparticle (LNP)-mediated delivery and engineered delivery vehicles (EDVs), editing activity is evaluated directly within harvested tissues. This enables quantitative assessment of editing distribution and tissue specificity.
Ex Vivo Edited Cell Tracking in Animal Models
For ex vivo–edited cell therapies administered into animal models, we support characterization of persistence and tissue localization. Edited allele frequency can be quantified across organs using qPCR and ddPCR methodologies.
In xenograft or humanized models, human-specific ALU assays enable detection and quantification of human cells in animal tissues, supporting biodistribution and trafficking assessment. This approach provides insight into cell persistence and distribution patterns following administration.
When editing targets hematopoietic or immune cell populations, additional analyses may be performed to evaluate lineage distribution and tissue localization. For example, CD14-positive monocyte populations and differentiated cell types can be assessed to understand where edited cells reside within tissues over time.
Biodistribution and Genome Stability Assessment
Preclinical safety evaluation requires quantitative assessment of construct distribution and editing stability.
We support:
- Detection and quantification of edited alleles across multiple tissues
- Measurement of vector or editing construct biodistribution
- Monitoring of editing consistency over time
- Detection of potential translocation events using ddPCR
These analyses provide reproducible molecular data to support dose selection, safety evaluation, and IND-enabling preclinical studies.
Why Choose Avance Biosciences?
Experience
With over 20 years in the industry, our team brings unparalleled expertise to your gene editing projects.
Regulatory Compliance
We operate in compliance with GLP and GMP guidelines, including Part 11 regulation, ensuring your data stands up to regulatory scrutiny.
Cutting-Edge Technology
Our use of NGS, ddPCR, and advanced bioinformatics allows for precise and reliable analysis.
Collaborative Partnership
We work closely with you to develop scientific and operational roadmaps aligned with your research, clinical, and commercial objectives.
Technical Information
This guide provides an in-depth look at GUIDE-Seq/iGuide, a key technique for assessing CRISPR-Cas9 on- and off-target effects in gene and cell therapy development. It explains how the method informs the selection of gRNAs, nucleases, and CRISPR conditions to maximize specificity and safety. Readers will also gain insights into the full workflow, from Cas9 cleavage to NGS analysis, supported by Avance Biosciences’ expertise as a licensed GUIDE-Seq service provider...
Genome editing offers unprecedented precision, but off-target effects remain a key safety concern. GUIDE-Seq provides a high-throughput, genome-wide method to map CRISPR-Cas activity in living cells, enabling researchers to assess specificity and ensure safer gene-editing therapies...
To support IND-enabling studies, a biotech company needed precise measurement of in vivo gene-editing efficiency in preclinical rat tissues. Avance Biosciences delivered a fully validated NGS amplicon sequencing assay capable of detecting edits as low as 1%, enabling accurate quantification across multiple tissues and guiding data-driven development decisions.