Clinical Gene Editing Analysis
Gene editing therapies administered in clinical trials require rigorous molecular characterization to evaluate editing efficiency, on- and off-target activity, persistence, and genomic stability in human subjects. Avance Biosciences provides comprehensive analytical support for clinical-stage gene editing programs, generating quantitative data to support pharmacokinetic (PK), pharmacodynamic (PD), and safety assessments.
Our workflows are designed to characterize editing outcomes in blood and relevant clinical matrices following ex vivo or in vivo administration of gene editing therapeutics.
Clinical Gene Editing Analysis
Gene editing therapies administered in clinical trials require rigorous molecular characterization to evaluate editing efficiency, on- and off-target activity, persistence, and genomic stability in human subjects. Avance Biosciences provides comprehensive analytical support for clinical-stage gene editing programs, generating quantitative data to support pharmacokinetic (PK), pharmacodynamic (PD), and safety assessments.
Our workflows are designed to characterize editing outcomes in blood and relevant clinical matrices following ex vivo or in vivo administration of gene editing therapeutics.
Our Expertise
With deep expertise in supporting both preclinical and clinical studies of gene and cell therapies using CRISPR, Zinc Finger Nucleases, and other gene editing tools, we employ amplicon sequencing to analyze edited genes in animal tissues post real-time PCR confirmation. In clinical settings, our methods include NGS amplicon sequencing, ddPCR, and qPCR on patient blood samples, crucial for robust pharmacokinetic (PK) and pharmacodynamic (PD) evaluations. This comprehensive approach ensures precise validation of gene edits, supporting the advancement of cutting-edge gene therapies.
In vivo On/Off Target Characterization
We possess extensive expertise in supporting clinical studies as part of human trial PK/PD studies by monitoring gene editing profiles for consistency and excretion using advanced techniques. NGS is employed to track on/off-target consistency over time, ensuring that indels and mutations remain stable without additional changes. Additionally, ddPCR is utilized to detect translocation events and identify low levels of cells with edited genes in patient blood. Our methodologies provide accurate, reliable data to support the safe and effective application of gene editing in clinical settings.
Ex Vivo Gene Editing Programs
For ex vivo edited cell therapies, we monitor editing profiles following administration into patients. NGS amplicon sequencing is used to quantify indel distributions and targeted modifications at defined loci. Longitudinal sampling enables assessment of editing durability and clonal stability over time.
ddPCR and qPCR assays are used to quantify edited allele frequency in peripheral blood and other relevant clinical samples, supporting PK and PD analysis. These measurements provide quantitative insight into persistence, expansion, and clearance of edited cells.
In Vivo Gene Editing Programs
For in vivo editing approaches, including lipid nanoparticle (LNP)-mediated delivery and engineered delivery vehicles (EDVs), we evaluate editing activity directly in patient samples. Following confirmation of therapeutic presence, NGS amplicon sequencing is performed to quantify on-target editing rates and assess candidate off-target loci.
Longitudinal monitoring allows evaluation of editing stability and identification of any emergent sequence alterations over time. ddPCR methodologies may be applied to detect rare events, including translocations or low-frequency edited populations.
Editing Stability and Safety Monitoring
Clinical safety assessment requires continued monitoring of editing outcomes following dosing. Our analytical workflows support:
- Quantitative tracking of edited alleles over time
- Stability assessment of indel patterns
- Detection of structural rearrangements or translocations
- Evaluation of editing consistency across patient cohorts
These analyses generate reproducible molecular data suitable for inclusion in regulatory submissions and clinical study reports.
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.