Bionano Announces Publication Demonstrating Utility of OGM to Assess Genome Integrity of CRISPR-Edited Cells as Part of Gene Therapy Development

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[{"type":"text","content":"SAN DIEGO, Nov. 29, 2023 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO) today announced a publication demonstrating the use of optical genome mapping (OGM) to identify structural variations (SVs) introduced by CRISPR-Cas9 gene editing of CD4+ T-cells that could potentially limit the therapeutic use of such edited cells. The research study authors used OGM in combination with drop-off digital PCR (ddPCR) to assess genome integrity of cells that were edited by CRISPR to restore gene function in Hyper IgM1 (HIGM1), an X-linked combined immunodeficiency caused by CD40LG mutations which can lead to liver disease, cancer and infections. The study authors describe that while ddPCR assays are suitable to characterize certain expected mutations, they may miss large SVs that are either outside the regions targeted by ddPCR primers or are too big to be detected by ddPCR. The study authors first validated OGM’s performance at high coverage to capture unexpected gene editing events and found that it outperformed karyotyping’s resolution and sensitivity. OGM then revealed the presence of concameters missed by the ddPCR assay, indicating that 20-50% of the edited cells expressing the rescued gene did not undergo precise editing. In addition, OGM detected 11 nonrecurring SVs outside of the target locus, demonstrating its genome-wide ability to detect off-target events. The study authors concluded that using OGM early in the development of the gene therapy could enable researchers to reduce the number of ddPCR assays needed in the final product quality control panel. “This study highlights how researchers can use OGM when developing cell and gene therapies. The expansion of gene therapy faces risks due to both on-target and off-target structural variations that may be introduced during CRISPR-Cas9 genome editing. Since genome aberrations caused by CRISPR-Cas9 editing could lead to unforeseen adverse effects, we believe careful and comprehensive analysis of edited genomes is important for quality control while developing these therapies and their manufacture,\" commented Erik Holmlin, PhD, president and chief executive officer of Bionano. The paper is available at: https://www.embopress.org/doi/full/10.15252/embj.2023114188. About Bionano Bionano is a provider of genome analysis solutions that can enable researchers and clinicians to r...

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