Gene Editing Technology Platform
An unmatched editing toolbox to expand what’s possible in gene editing.
// Gene Editing
Technology Platform
CRISPR Systems and Novel Editing Technologies
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 (knock-out) at the cut site. Our collection of compact RNA-guided nucleases with diverse PAMs allows us to introduce knock-out 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, polymerase 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 screening, analysis, and optimization to achieve optimal editing outcomes at each target locus.
Epigenetic Editing
Epigenetic editing enables precise control of gene expression without altering the underlying DNA sequence. Our platform can selectively turn genes on, off, or finely tune their expression levels in a programmable manner. These changes can be durable or transient, enabling therapies designed for long-lasting benefit or reversible control. When delivered by lipid nanoparticles (LNP), epigenetic modulation supports repeat dosing, allowing gene expression to be adjusted over time based on disease biology. This flexibility makes epigenetic editing well suited for indications requiring correction of gene dosage and modulations of gene networks or pathways, where permanent DNA alterations may be unnecessary or undesirable.
Targeted Gene Insertion
Targeted gene insertion enables precise placement of large (>2 kb) therapeutic DNA sequences into the genome, supporting integrations ranging in size from single exons to full-length genes for durable expression of functional proteins. This approach can broaden access to genetic medicines by enabling a single therapeutic to address an indication with multiple causative mutations. It is particularly attractive for chimeric antigen receptor (CAR) therapeutics, combining the durability associated with viral delivery with the safety profile of non-viral methods. Our platform leverages proprietary engineered retrotransposons and large serine recombinases (LSRs), providing flexibility for diverse downstream applications.
Broader Genomic Access Through Diverse Recognition Sequences
Our nuclease collection features diverse DNA recognition sequence requirements – protospacer adjacent motifs or PAMs – that determine where gene editing tools can bind and operate. This expanded recognition diversity provides crucial advantages: greater flexibility in positioning editing tools, access to tighter editing windows, and the ability to reach greater than 99% of genomic sites. For editing approaches with stringent targeting parameters, this diversity enables us to access previously unreachable genetic targets.
Delivery Flexibility Enabled by Compact CRISPR Design
Our RNA-guided CRISPR systems are compact (~800-1,100 amino acids) while offering high fidelity and novel functionality. Their size enables greater versatility in packaging our systems for both viral and non-viral therapeutic delivery, potentially overcoming key limitations in getting gene editing tools to target tissues.
Our in-house lipid nanoparticle (LNP) platform represents a cutting-edge delivery system, and contains a library of proprietary ionizable lipids. This flexible platform enables us to reach diverse tissue types, including robust liver targeting, and opens new therapeutic possibilities beyond the constraints of viral delivery.
Partner with ElevateBio®
Wherever you are in your advanced therapy product lifecycle, we can strengthen and accelerate your development journey with our enabling technologies and unmatched process development and manufacturing capabilities.
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