Preclinical and Clinical Gene Editing Analysis
Gene editing has transformed gene and cell therapy, enabling precise corrections for genetic disorders. Tools like CRISPR allow targeted modifications both ex vivo in cell therapy and in vivo, treating previously untreatable conditions. Ensuring the stability of these edits is crucial for long-term safety and efficacy. Gene editing stability analysis verifies that modifications remain consistent and functional over time, meeting regulatory standards and advancing these therapies into clinical use, providing reliable and effective treatments for patients.
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
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 and validated per FDA requirements. Following real-time PCR or ddPCR-based biodistribution studies, tissues with positive edited genes are selected for quantitative NGS amplicon sequencing. We assess the consistency of indels and point mutations at on/off-target sites and report their relative abundance over time.
Clinical Studies
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.
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.