Gene Editing
CRISPR/Cas9 – a revolutionary gene-editing technology that can be used to modify or correct precise regions of our DNA to treat serious diseases
Dr. Emmanuelle Charpentier, one of our scientific founders, co-invented CRISPR/Cas9 gene editing. She and her collaborator, Dr. Jennifer Doudna, won a Nobel Prize for this groundbreaking work. As a gene-editing tool, CRISPR/Cas9 has revolutionized biomedical research and enabled medical breakthroughs.
CRISPR/Cas9 edits genes by precisely cutting DNA and then harnessing natural DNA repair processes to modify the gene in the desired manner. The system has two components: the Cas9 enzyme and a guide RNA.
CRISPR Lexicon
- CRISPR: “Clustered Regularly Interspaced Short Palindromic Repeats” of genetic information that some bacteria use as part of an antiviral system and that Dr. Charpentier and others discovered how to use as a gene-editing tool
- Cas9: a CRISPR-associated (Cas) endonuclease, or enzyme, that acts as “molecular scissors” to cut DNA at a location specified by a guide RNA
- Guide RNA (gRNA): a type of ribonucleic acid (RNA) molecule that binds to Cas9 and specifies, based on the sequence of the gRNA, the location at which Cas9 will cut DNA
A variety of genetic edits can be performed with CRISPR/Cas9, including:
CRISPR/Cas9 Gene Editing
DISRUPT
CRISPR/Cas9 can make a single cut using one guide RNA; the cut is then repaired through natural processes, which can result in the addition or deletion of base pairs, leading to gene inactivation.
DELETE
A segment of DNA can be removed by using two guide RNAs that target separate sites; after cleavage the two separate ends are joined together while the intervening sequence is removed.
CORRECT OR INSERT
Adding a genetic template alongside the CRISPR/Cas9 machinery can enable the cell to correct a gene or insert a new gene.
Therapeutic Approach
With innovation at our core, we aim to develop potentially transformative gene-based medicines. We are applying gene editing to treat disease in two primary ways:
Genetic diseases: Targeting specific genes that cause or influence the course of a disease
Many diseases, both rare and common, have a genetic basis. The scientific understanding of how specific genes are involved in disease is advancing rapidly, offering the opportunity to use gene editing technologies to disrupt or correct disease-related genes.
Cell therapies: Engineering the next generation of cell therapies that can address an array of diseases
Cell therapies have begun to make a meaningful impact in numerous diseases, including cancers. With our technologies, we can engineer cell therapies with the aim of making them more effective, safe and widely available.
In either case, we may edit cells ex vivo (outside the body) or in vivo (inside the body).
We’re also pushing the bounds of science as we continue to advance new gene editing technologies. Learn more here.
Our development of transformative new investigational medicines will involve working closely with patients, families, healthcare professionals, regulatory agencies and other groups dedicated to improving healthcare. We are focused on the treatment of somatic cells, which do not pass DNA on to children. We are not using human germline modifications, which could be passed from parents to children, and we support the current recommendations of the International Society for Stem Cell Research in this regard.
To learn more about the full range of investigational therapies we are developing visit our pipeline page
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