CRISPR Gene Editing Creates New Possibilities — And New Challenges

The advent of CRISPR and other gene editing technologies, which facilitate gene editing in living organisms, has created exciting new possibilities within the life sciences for therapeutic treatments of genetic diseases and cancer immunotherapy. Recent clinical validation, including an early 2020 trial that demonstrated CRISPR’s safety and utility in three cancer patients, has greatly accelerated interest in gene editing-based therapeutics.

Subsequently, governing regulatory bodies — including the FDA — are making on and off target events identification a top priority. To meet evolving product characterization standards, researchers using CRISPR are expected to understand the detailed impact of gene editing on both target and off target sites.

In light of these new expectations, bioinformatics-based off target site predictions and chromosomal translocation events are no longer sufficient. Instead, researchers should prioritize precision analysis using off target-specific testing modalities. Depending on the project requirements, these modalities should utilize rhAmp-seq and targeted amplicon sequencing on Illumina NGS platforms, as well as translocation events using ddPCR assays.

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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.

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.

Platforms & Services

Avance Biosciences has years of experience developing and validating genomic assays utilizing Illumina NGS platforms as well as ddPCR technologies.

Next-Gen Sequencing

NGS assays can be used at various stages of a genome editing workflow to:

  • Analyze CRISPR off-target effects with rhAmp™ seq panels and whole-genome sequencing.

  • Confirm CRISPR knockouts and other edits with targeted amplicon sequencing.

  • Perform follow-up studies using applications such as gene expression profiling with RNA sequencing, to assess the functional impact of a given gene edit.

  • Guide RNA GMP sequencing to support manufacturing and drug product release.

dPCR

ddPCR genome detection assays offer a fast, flexible, and precise method for detection of genome editing events, providing:

  • Rapid assessment of both homology directed repair (HDR) and nonhomologous end-joining (NHEJ) edits generated by CRISPR-Cas9 or other genome editing tools.

  • Characterization of chromosomal translocation events caused by gene editing tools.

  • Ability to distinguish between homozygous and heterozygous edits and clonal populations.

  • Ability to detect editing events present at frequencies of <0.5%.

Improve Off Target Analysis with Avance As Your Partner

Avance Biosciences has more than two decades of experience providing advanced genomics services. As a true collaborative partner, we help you create a scientific and operational roadmap that helps you meet research, clinical, and commercial objectives. We’re ready and able to help you characterize your product’s off target site impact in detail, as well as navigate the evolving CRISPR gene editing standards laid out by the FDA and other regulatory bodies worldwide.

Have questions?

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The Avance Biosciences team is committed to strict adherence to GLP and CGMP regulations enacted by the US, European, Japanese, and other international regulatory agencies.

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Avance Biosciences’ GLP/GMP compliant genomic and microbial assays make use of the advanced technologies including real-time PCR, digital PCR, Sanger sequencing, next generation sequencing, and more.