Our Technology.
CRISPR gene editing: Breakthrough technology to permanently cure genetic disorders.
In 2012, a newly discovered bacterial ‘immune system’ termed CRISPR was used to edit the human genome. CRISPR gene editing has quickly become the standard for precision genetic medicines and given hope to the millions of patients suffering from previously untreatable genetic diseases. The power of CRISPR to make a permanent change in a patient’s genetic code also requires precise control to ensure that no unintended editing takes place. Indeed, unintended ‘off-target’ editing could lead to severe side effects and undermine the potential of CRISPR gene editing to save and improve the lives of patients. At Acrigen, we have developed high precision gene editing systems to dramatically improve the efficacy and safety of gene editing therapies.
We have built a robust and extensible discovery engine to identify novel CRISPR-Cas nucleases. We use our proprietary AcrTAIN™ bioinformatics discovery software platform to find CRISPR systems amenable for use in human therapeutics. We then optimize these unique enzymes by leveraging our deep expertise in protein engineering to develop highly tuned editing machines for use across in vivo and ex vivo applications.
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High precision gene editing.
The human genome contains over six billion individual nucleotides. In order to edit the genome at the correct location, the CRISPR-Cas gene editor must exactly match the ~20 nucleotides encoded in the CRISPR Guide RNA to the target genomic sequence. Unfortunately, most CRISPR-Cas systems can tolerate mismatches between the Guide and genomic DNA, leading to unintended ‘off-target’ genome editing. Acrigen is developing high precision gene editing systems to increase the efficacy and safety of gene editing for in vivo and ex vivo therapies.
Nuclease discovery engine.
ErAcr protein engineering.
Acrigen has developed a novel method to solve the ‘off-target’ editing problem. Editing at unintended sites in the genome is a serious risk for any CRISPR therapy. To eliminate editing at these ‘off-target’ sites, we have developed ErAcr proteins - Engineered Recombinant Anti-CRISPR proteins - that selectively inhibit off-target editing. ErAcr proteins are built off of the work of Acrigen Co-Founder Joseph Bondy-Denomy, who first discovered Anti-CRISPR proteins capable of fully inhibiting CRISPR-Cas systems. Acrigen has engineered these small proteins to selectively inhibit off-target editing events while maximizing therapeutic target editing. An ErAcr protein coupled with a novel CRISPR-Cas nuclease form the core of Acrigen’s high precision gene editing system. This system can be tuned and optimized for in vivo and ex vivo therapeutic editing applications.
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Let’s Work Together.
Contact us about licensing our CRISPR-Cas Nuclease Library and Precision Enhancing ErAcr Proteins.