LCM™ for Gene Therapy Monitoring

Highlights:

  • TimepointDx LCM™ was implemented as a new methodology to assess the potential pathogenicity of replication competent retrovirus (RCR) and replication competent lentiviru (RCL).
  • For retroviral vector-based gene therapy patients, TimepointDx LCM™ was compared via presence/absence studies to detect the presence in RCR, whereby molecular characterization profiles were developed for both binary states via LCM™.
  • Additional correlation assessments between TimepointDx LCM™ and insertion-site analysis (ISA) was conducted for orthogonal confirmation, whereby TimepointDx LCM™ determined the exact location and quantity of positively-identified vector insertion sites within PBMCs directly from capillary samples.
  • TimepointDx LCM™ serves as a RCR detection and quantitation assay for lifelong monitoring of cell and gene therapy patients as an exploratory endpoint.
  • TimepointDx LCM™ was conducted on the supernatants of cell therapy manufacturing products in addition to capillary blood samples post-therapy of manufactured products.
  • cPBMCs were isolated via TimepointDx CSCR™, followed by TimepointDx LCM™ for RCR-specific signatures. Confirmation was obtained via genomics as part of LCM™, with additional biological insights of pathways as identified by the remainder omics of LCM™.
  • TimepointDx LCM™ is an efficient method for exploratory and supplemental monitoring of gene therapies for lifelong time-series and adverse-event monitoring needs.

See White Paper for Details.

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