History

The development in 2012 of CRISPR/Cas9 as a genome editing tool by a research team co-led by one of Intellia’s founders, Jennifer Doudna, triggered unprecedented scientific research on the application of this technology to revolutionize medical care and treatment. This technology is rapidly leading to the development of a new class of therapies for patients with genetic and oncological diseases.

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“We may be nearing the beginning of the end of genetic diseases.”

Jennifer Doudna, Ph.D., Professor of Chemistry and Molecular and Cell Biology, University of California; Chemistry Nobel Laureate, 2020; Co-founder, Intellia Therapeutics

CRISPR/Cas9: The Ideal Genome Editing Technology

Genome editing is efficient, precise and scalable.

CRISPR/Cas9 genome editing can make permanent, precisely targeted changes in patients’ chromosomes and edit the underlying genetic mutation, whereas more traditional gene therapy typically involves introducing a non-permanent copy of a gene into patients’ cells. These attributes of CRISPR/Cas9 provide a significant therapeutic edge over other gene therapy and costly earlier-generation genome editing technologies, such as zinc finger nucleases and transcription activator-like effector nucleases.

Intellia’s proprietary CRISPR/Cas9 system could potentially address diseases with a single dose of treatment because it permanently edits the defective DNA. This represents a breakthrough improvement over current therapies, most of which require lifelong administration because they cannot correct underlying causes of the disease.

Genome Editing Drug Development: Disease & Target Selection

How Does CRISPR/Cas9 Work?

There are two main components to the CRISPR/Cas9 genome editing system:

  • The Cas9 protein, which initially recognizes the DNA and also acts like a pair of “molecular scissors” that precisely cleaves the targeted DNA sequence.
  • The guide RNA, which guides the Cas9 scissors to the desired target DNA sequence and activates the scissors so they cut.

CRISPR’s Origin: Found in Nature as Part of Bacteria’s Immune System

Intellia has co-opted the bacterial immune system by identifying the key components (pictured above) and applying them to human DNA. Upon DNA cleavage, the natural cellular repair processes come into play to result in knockout, repair or insertion of genetic material. Click here to watch how CRISPR/Cas9 works.