CRISPR, a potent gene editing tool, empowers scientists to enact precise modifications in ex vivo gene editing for cell therapy and in vivo gene therapy, unlocking unparalleled possibilities for addressing genetic disorders. As this revolutionary technology continues to progress, so does the need for rigorous characterization of the edited genes in compliance with regulatory requirements.
The Avance Biosciences™ team is your trusted partner in navigating the complex analytical landscape of gene editing. Boasting more than 20 years of expertise in pioneering biological assay development and validation, we deliver cutting-edge solutions for evaluating the effectiveness of on-target editing and monitoring off-target editing events in gene-edited T-cells, stem cells, iPSC, sgRNA/mRNA in LIPID, and for both preclinical and clinical research studies.
Expert On/Off Target Screening & Analysis
To meet evolving regulatory requirements, standards and to mitigate risk, researchers utilizing tools such as CRISPR and other gene editing platforms must proactively identify and address off target effects, as well as any unintended modifications at a non-target site such as translocation possibilities.
Fortunately, modern genomic assay platforms — including NGS and ddPCR— provide effective, reliable means to identify off target effects and events, including point mutations, deletions, insertions, inversions, and unexpected translocations.
As an experienced leader and partner in the field of CRISPR gene editing, Avance Biosciences™ provides a comprehensive range of off target services. Using robust GMP/GLP processes — as well as state-of-the-art NGS and ddPCR platforms that optimize scalability, speed, and cost — we help life science researchers take a proactive, strategic approach to off site screening, analysis, and intervention.
Identification and Quantification of Off-Target Effects
To meet evolving regulatory standards and ensure the safety and efficacy of gene editing-based therapies, researchers must take proactive measures to detect and mitigate off-target effects. Avance Biosciences™ offers a comprehensive range of services to support your development efforts:
On/Off-Target Sites
Screening and Verification
Our team possesses substantial experience in utilizing IDT’s rhAmpSeq™ technology to screen potential off-target sites through in silico primer design and Illumina sequencing. Additionally, we have substantial experience in employing Guide-Seq, licensed from SeQure-Dx, for cross-verifying potential off-target sites. Moreover, our proficient scientific team is well-equipped to assist you in establishing alternative or complementary on/off-target analysis methodologies to meet your specific research and regulatory requirements.
On/Off Target Sites
Characterization and Quantification
Our Next-Generation Sequencing (NGS) team has collaborated closely with a diverse array of biopharmaceutical clients engaged in gene and cell therapy. Together, we’ve developed intricate amplicon sequencing panels designed to confirm and quantify on/off-target indel events stemming from various gene editing techniques. We have meticulously validated multiple amplicon assays in full compliance with FDA GLP and CGMP standards, all aimed at bolstering our clients’ preclinical and clinical investigations, spanning in vivo and ex vivo CRISPR-based gene and cell therapies.
Gene Editing
Translocation Study
Leveraging Bio-Rad’s QX200 ddPCR platform, we specialize in crafting highly sensitive assays specifically engineered to identify potential genome translocation events following gene editing. Our team boasts extensive experience in validating novel and complex assays in accordance with ICH guidelines, thus ensuring robust support for the characterization of gene editing cell therapy products.
Data Analysis
Pipeline
Our adept teams in Bioinformatics and IT possess extensive experience in setting up diverse NGS analysis pipelines, whether internally or through technology transfer from our clients. We take measures to ensure a consistent computation environment for bioinformatics pipelines throughout our clients’ drug development lifecycle and beyond. Moreover, we have implemented a strong data integrity mechanism in our NGS analysis, encompassing raw data verification, user controls, and an audit trail, among other measures.
Other Analytical Methods
to Support Gene Editing
In addition, we offer a wide array of other assays and services to facilitate the development and production of gene and cell therapeutics.
- Preclinical biodistribution and gene/protein expression studies
- Clinical PK/PD studies using QPCR, ddPCR, NGS, and ELISA
- Cell-based potency assays for characterization and lot release
- Stability study for drug substance/product characterization
- sgRNA and mRNA ID and ratio testing
- On/Off-target rhAmpSeq panel development and validation
- On-target amplicon sequencing assay development and validation
- Various other assays for characterization and lot release including compendial assays
Improve Off Target Analysis with Avance As Your Partner
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, 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.
Contact Us
Unlock the full potential of your gene editing programs with Avance Biosciences. Contact us today to learn more about our on/off-target gene editing services and discover how we can help you navigate the evolving CRISPR gene editing landscape.
Our Customers Say…
From the preliminary conclusion of this work, we are already pleased to thank you for your customer-oriented approach and proactive communication in the course of this study. We are happy to see the profound difference in business mindset between your organization and our previous vendor…
Project Manager, Belgium
Thank you so much for successfully completing this important project for us on time. We are very satisfied with the final report and thank you for taking the extra effort to customize the report format. We will definitely use your lab for future projects.
Program Manager, California
It has been a pleasure working with you and your team. The project was carried out to the highest standard and delivered in a timely manner. I will definitely recommend Avance Biosciences™’ services to colleagues and friends.
Senior Scientist, Singapore
When you partner with Avance Biosciences™, you gain a CRO partner that is creative, collaborative and dedicated to sound science with a focus on the regulatory requirements our partners require. Contact our technical staff to discuss how we can support you in your project!
