Gene Editing

Realizing the promise of genome editing through Life Edit at ElevateBio.

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 library 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 offers 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 library 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 collection 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 lead 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 lead nucleases to one of our proprietary deaminases.

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.

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 Integrated Technology Platforms: Fueling the Discoveries of Tomorrow

Induced Pluripotent Stem Cells (iPSCs)

We are setting a new standard with well-characterized induced pluripotent stem cell (iPSC) lines that are suitable for research, clinical and commercial use. Click here to learn about iPSCs.

RNA, Cell, Protein, and Vector Engineering

We have centralized RNA, cell, 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. Click here to learn about engineering.

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.