Gene Editing Technology Platform
An unmatched editing toolbox to expand what’s possible in gene editing.
// Gene Editing and
R&D Technologies
Gene Editing Technologies to Accelerate your Therapeutics
We empower our partners by unlocking the full potential of gene editing. Our gene editing platform is designed to match the optimal editing modality to each therapeutic challenge, combining the industry’s most comprehensive technology portfolio with depth of expertise to expand what’s possible with gene editing. Our diverse collection of CRISPR systems and editing approaches can be tailored and engineered to specific genes or targets, enabling precision, potency, and specificity for previously inaccessible genetic disorders.
Advanced Editing with the Industry’s Largest Gene Editing Toolbox
We have versatile editing options for targeting genetic disorders, enabling us to match the right modality to best address the target.
Nuclease Editing
Nuclease-based editing involves cutting both strands of DNA, enabling gene insertion (knock-in) or deletion (knockout) at the cut site. Our collection of compact RNA-guided nucleases allows us to introduce knockout or knock-in edits with precision across the genome, significantly expanding the range of addressable genetic targets and enabling therapeutic approaches for complex mutations.
Base Editing
Base editing converts one nucleotide into another without cutting both DNA strands, achieved by coupling a modified nuclease to a deaminase that edits the target nucleotide. Our modular approach includes A and C base editors for both ex vivo and in vivo applications with demonstrated multiplex editing capabilities. This technology enables precise nucleotide-level correction of disease-causing mutations or strategic disruption of coding sequences to modulate gene expression.
Reverse Transcriptase (RT) Editing
RT editing – also known as prime editing or DNA writing –creates a targeted single-strand break, then replaces existing DNA sequence with new sequence encoded by the guide RNA. Our approach leverages an extensive panel of RNA-guided nucleases coupled with expertise in target screening, analysis, and optimization to achieve optimal editing outcomes at each target locus.
Broader Genomic Access Through Diverse Recognition Sequences
Our nuclease collection features diverse DNA recognition sequences – protospacer adjacent motifs or PAMs – that determine where gene editing tools can bind and operate. This expanded recognition diversity provides crucial targeting advantages: greater flexibility in positioning editing tools, access to tighter editing windows, and the ability to reach substantially more genomic sites than conventional systems. For editing approaches with stringent targeting parameters, this diversity enables us to access previously unreachable genetic targets.
Compact Design for Enhanced Therapeutic Delivery
Our RNA-guided nucleases, sourced from non-pathogenic microbes, are significantly smaller (~800-1,100 amino acids) compared to conventional nucleases while offering higher fidelity and novel functionality. This compact size enables greater versatility in packaging our systems for therapeutic delivery, potentially overcoming key limitations in getting gene editing tools to target tissues.
The Breadth of our Platform and Technology Offers Unmatched Editing Capabilities
Therapeutic Applications
We have broad therapeutic delivery capabilities, including viral and non-viral delivery platforms to provide flexibility in therapeutic design and application, unlocking new possibilities for addressing a wide range of diseases and disorders.
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AI & Protein Engineering
We harness artificial intelligence to accelerate CRISPR discovery, combining our protein engineering expertise with a vast library of over 20 billion proteins to design optimal editing systems for thousands of previously untreatable genetic disorders.
Partner with ElevateBio®
We are looking to form strategic partnerships with biotechnology and biopharmaceutical companies to advance their life-changing and curative therapies with our Life Edit platform. And by integrating with ElevateBio BaseCamp’s manufacturing infrastructure, we can accelerate your program’s path from editing technology to patient-ready medicines.
Our flexible partnership options include broad R&D collaborations, exclusive and non-exclusive licenses for specific assets, and strategic partnerships to access our technology platforms designed to accelerate the pipelines of our partners.
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