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Unleashing potential with CRISPR technology
In the dynamic landscape of biological research, CRISPR technology has revolutionized our approach to genetic disorders and the future of personalized medicine. Of these advances, cell and gene therapy is front and center as an example of where CRISPR has been forging inroads into these novel therapeutic possibilities.
Established as a pioneer in genome engineering, EditCo Bio spun out from the CRISPR heavy weight, Synthego, in March of 2024 with a mission to redefine how CRISPR tools are used. Leveraging data from over 500,000 edits, EditCo’s automated gene editing design and application platform has handled a variety of complex genomic challenges, offering streamlined solutions from optimized gene knockout kits or custom-edited cells to high-throughput projects and advanced data analysis.
High-throughput screening in iPSCs for cell therapies
This novel gene editing platform has opened the door to a wide range of challenging projects and partnerships with companies whose research is moving the world of cell and gene therapy forward quickly. An example highlighting how EditCo Bio’s CRISPR platform has solved gene editing challenges is through a recent partnership with bit.bio, a synthetic biology company with a platform to program human induced pluripotent stem cells (iPSCs) into a wide range of cell types for discovery research and cell therapy.
Combining EditCo’s ability to perform high-throughput edits in many medically relevant cell types, including human iPSCs, with full automation integration, and bit.bio’s expertise in leveraging iPSCs for cell therapies, the team performed a high-throughput screen of hundreds of guides across 25 genomic safe harbor (GSH) sites. Additionally, EditCo’s ability to analyze many biological and technical replicates with the use of an integrated robotic workcell for next-generation sequencing (NGS) allowed the generation of a highly reproducible dataset by sequencing more than 4,600 NGS libraries with a success rate ranging from 88% to 98%.
Through meticulous optimization of editing conditions, careful validation through NGS, and automated high-throughput capacity, the project showcased the precision and flexibility made possible through EditCo’s gene editing platform. This allowed for rapid identification of hot spots for high knock-in editing levels with the most effective guides in genomic regions not typically targeted for gene editing. Ultimately, this research has the aim of maximizing safety and efficacy of CRISPR-Cas editing across the 25 genomic loci.
Addressing Alzheimer’s disease with advanced CRISPR applications
Beyond large-scale editing optimization, EditCo’s genome editing process has been important for providing a unique solution for CRISPR screening in clonal cells. In collaboration with the National Institute of Health (NIH), the Inducible Pluripotent Stem Cell Neurodegeneration Initiative (iNDI) project by EditCo Bio targeted 22 genes, generating 12 variants per gene, which resulted in 264 CRISPR iPSC clones all in less than five months.
This massive screening of 8,108 clones and the execution of 6,138 sequencing reactions highlighted the platform’s capability to handle large-scale, high-throughput operations efficiently. This project is particularly crucial for advancing understanding and treatments of complex diseases like Alzheimer’s, showcasing EditCo’s pivotal role in cutting-edge genetic research.
Shaping the future of healthcare
By marrying the precision of CRISPR with the expertise and innovation within EditCo Bio, significant progress continues to be made to elucidate and mitigate risks associated with genetic diseases. Opening up new avenues for enhancing human health is proving that EditCo’s technology will continue to be a powerful solution within the field of cell and gene therapy and healthcare overall. And, as CRISPR technology continues to evolve, EditCo Bio plans to remain at the forefront, committed to partnering with researchers and leading the charge toward transformative health solutions.
References
1. Published with permission from bit.bio
2. Figure created with BioRender.com
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