Gene Editing
Realizing the promise of genome editing through
Life Edit at ElevateBio.
// Technology
Platforms
Diverse Tools for Unlimited Gene Editing
Genome editing technologies allow genetic material to be removed, added, or altered at a specific location in the genome to develop life-changing therapies for the most challenging genetic disorders.
Our next-generation Life Edit gene editing platform offers one of the world’s largest and most diverse libraries of RNA-guided nucleases (RGNs), base editors, and reverse transcriptase editors, that provide flexible editing and unprecedented access to the genome. They were developed using a proprietary collection of non-pathogenic microbes, which offer gene editing tools with higher fidelity, novel functionality, and easier delivery.
RNA-Guided Gene Editing
We have one of the world’s largest and most diverse libraries of RNA-guided nucleases that encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with one of our proprietary deaminases.
Our lead RNA-guided nucleases are smaller in size when compared to conventional nucleases, allowing for greater versatility in packaging our systems for therapeutic delivery.
PAM Diversity is Key to Making Any Edit Possible
The key advantage of our nuclease collections is in the diversity of Protospacer Adjacent Motifs (PAMs)that offers unprecedented access to the genome. PAMs are short sequences that determine the DNA segments in the genome to which a nuclease can bind to edit a gene.
Our broad range of PAMs increase the number of specific sites where therapeutically meaningful edits can be made, granting our nucleases access to virtually any region of the genome.
Unprecedented Flexibility in Gene Editing
The diversity of PAMs in our systems offer unprecedented flexibility in three types of gene editing:
Double-Strand Breaks
Double-strand breaks involve the nuclease cutting both strands of the DNA, enabling gene deletion (knock-out) or insertion (knock-in) at the cut site. The PAM diversity of our nucleases allows the introduction of knockout or knock-in edits virtually anywhere in the genome.
Base Editing
Base editing converts one nucleotide (base) into another, without cutting both strands of DNA. This is achieved by coupling a nuclease, modified to cut only one DNA strand, to a deaminase that edits the target nucleotide. Our modular approach to base editing couples our proprietary nucleases and deaminases to one another.
The PAM diversity of our nucleases addresses a critical base editing parameter in which the target mutation must fall within a tighter “window” than required for double-strand breaks. Our PAM diversity enables base editing at more sites than any one nuclease could access.
Our base editing systems includes A and C base editors for ex vivo and in vivo applications with demonstrated multiplex editing capabilities. Base editing technology allows precise nucleotide-level correction of harmful mutations or disruption of coding sequences to reduce gene expression.
Reverse Transcriptase (RT) Editing
RT editing involves cutting one DNA strand, then replacing an existing DNA sequence – from the cut site to the target editing site – with a new sequence that is encoded by the guide RNA (gRNA). Our approach takes advantage of our panel of RGNs – coupled with our expertise in target screening, analysis, and optimization – to enable optimal editing of the target locus.
The PAM diversity of our platform is also able to position the nuclease as close to the editing site as possible, reducing the size of the required gRNA and the challenges associated with lengthy gRNAs. RT editing allows the most precise and flexible editing outcomes for many different types of gene correction, insertion, or knock-out.
Novel Delivery Technologies
We have broad therapeutic delivery capabilities, including viral lentiviral and AAV delivery systems and a non-viral delivery platform, unlocking new possibilities for addressing a wide range of diseases and disorders.
Our novel lipid nanoparticles (LNP) platform has both liver targeting and de-targeting potential, enhancing our ability to effectively deliver gene therapies and unlocking new possibilities for addressing a wide range of diseases and disorders. The platform’s adaptability also has to potential to expand its targeting capabilities beyond the liver.
Harnessing Artificial Intelligence to Accelerate CRISPR Discovery and Design
We believe we have one of the largest CRISPR data sets in the industry with a wide range of CRISPR-Cas systems, including known subtypes identified, which engineered select systems into a variety of editing modalities, and with bases of assembled and formatted sequences. We also have an extensive catalog of more than 8 billion proteins for rapid homology searching, enabling our scientists to efficiently identify new targets.
Harnessing the power of artificial intelligence (AI), we propel CRISPR discovery and engineering efforts at an unprecedented pace to enable the development of promising therapeutic options for the broadest range of genetic disorders and diseases that were once deemed untreatable. To achieve this, we believe we have to look beyond nature-derived CRISPR systems to the design of synthetic CRISPRs that can be tailored for each disease, and that leveraging AI will accelerate the path towards this goal.
Discover how Life Edit is transforming the future of medicine with the power of AI in our latest Elevated Insights.
Our Integrated Technology Platforms Fueling the Discoveries of Tomorrow
Cell Therapy Enabling Technologies
Our technologies and capabilities can enable a range of cell-based therapies, including those derived from T cells, NK cells, induced pluripotent stem cells, and more.
RNA, Protein, and Vector Engineering
We have centralized RNA, protein, and vector engineering to improve productivity and scalability and to ensure external partners and internal programs have access to the necessary tools to build novel therapies.
Our Therapeutic Approaches
We are enabling the development of a range of ex vivo and in vivo cell and gene therapies, leveraging our in-house capabilities and expertise to design and develop an array of modalities.
End-to-End Capabilities
We help our partners scale new heights in cell and gene therapies by providing a full spectrum of viral vector and cell therapy capabilities to bring concepts to commercialization.
Partner with ElevateBio®
We are looking to form strategic partnerships with biotech and pharmaceutical companies to advance their life-changing and curative therapies. Our flexible partnership options span non-exclusive licensing of our gene editing systems, exclusive licenses to assets, and collaborations as starting points for strategic partnerships.
To provide access to genome editing tools and cure disease, we’re seeking partnerships with biotech and pharmaceutical companies including those interested in ex vivo therapies. We will also continue to support our broad portfolio of innovative biotech companies.
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