The 2nd CRISPR Medicine Conference convenes key stakeholders across the cell and gene therapy ecosystem to address critical challenges in advancing genome editing technologies, from the research bench to clinical application. With a comprehensive focus on translational science, clinical development, regulatory strategy and patient access, the meeting is designed to foster interdisciplinary collaboration and tackle the most significant bottlenecks in the field.
MaxCyte® is pleased to participate in this year’s meeting, where we will highlight our GMP-compliant, non-viral Flow Electroporation® technology, which enables precise, scalable and high-efficiency delivery of genetic material into a wide range of cell types. Our platform is designed to support the entire therapeutic development continuum — from discovery through clinical and commercial manufacturing. In addition, we will feature SeQure DX™, MaxCyte’s variant-aware, off-target analysis service. SeQure DX integrates computational, biochemical and population-scale genomic data to provide a high-resolution assessment of off-target activity, supporting safety and regulatory readiness in gene editing programs.
Together, MaxCyte and SeQure DX offer an integrated solution to help accelerate the development of safe, effective and scalable cell and gene therapies.
Featured presentation
Variant-Aware Off-Target Analysis for Informed Guide RNA Selection
Tuesday, April 8, 2025, from 7:30 to 8:30 p.m. CET
Poster ID 126
MaxCyte is excited to highlight SeQure DX at the CRISPR Medicine Conference. Stop by the poster session below to see how researchers are applying SeQure DX’s variant-aware analytics to support safer, more precise gene editing.
Presenter
Poster abstract
The specificity of CRISPR-based gene editing is vital for therapeutic safety and efficacy. Off-target activity can lead to unintended genetic alterations, making it essential to account for both off-target risk and human genetic diversity when designing guide RNAs (gRNAs). In this study, we applied a variant-aware strategy, integrating computational and biochemical methods to select gRNAs for editing the PCSK9 gene. Using SeQure Dx’s Guide Profiler™ tool, we screened candidate gRNAs against the human reference genome and 3,502 diverse genomes, identifying PCSK9-1 as the lead candidate. Guide Select™, a multiplexed biochemical assay, confirmed PCSK9-1's low off-target activity across diverse genetic backgrounds. Finally, ONE-seq™, a high-sensitivity, variant-aware assay, provided a genome-wide assessment that confirmed PCSK9-1’s suitability for therapeutic development by detecting minimal cleavage at high-risk sites. This approach demonstrates how combining variant-aware analysis with robust in-silico and biochemical tools can reduce off-target risk and improve confidence in therapeutic genome editing.
