CRISPR/Cas9 is a simple but powerful gene-editing technology that we can harness to precisely modify, delete or correct disease-causing abnormalities at their genetic sources. “CRISPR” refers to Clustered Regularly Interspaced Short Palindromic Repeats of genetic information, which some bacterial species use as an antiviral mechanism in combination with the Cas9 enzyme. Cas9 – a CRISPR-Associated endonuclease – acts as “molecular scissors” to cut DNA at a specific location. The location at which Cas9 cuts DNA is specified by a guide RNA comprised of a crRNA component and a tracrRNA component, either individually or combined together as a single guide RNA (sgRNA). For uses in therapeutic gene editing, a guide RNA can direct the molecular scissors to cut the DNA at the exact site of the mutation present in the genome of patients with a particular genetic disease. Once the molecular scissors make a cut in the DNA, the cell’s own robust natural repair machinery repairs the cut, a process that can disrupt or delete a disease-causing gene, or correct that gene if the desired DNA is added as a template. Dr. Emmanuelle Charpentier, one of CRISPR Therapeutics’ scientific founders, co-invented the application of CRISPR/Cas9 in gene editing.
CRISPR/Cas9 gene editing has transformed many areas of research. Thousands of academic laboratories across the world have adopted CRISPR/Cas9, using the technology to conduct a wide range of new experiments. The rapid adoption of CRISPR/Cas9 and the resulting groundbreaking research have driven tremendous progress in the field, informing our work and others’.
There are more than 10,000 known single-gene (or monogenic) diseases, occurring in about 1 out of every 100 births1. Scientists and clinicians are now conducting pioneering research using CRISPR/Cas9 to address both recessive and dominant genetic defects, opening up the potential of gene editing to provide novel transformative gene-based medicines for patients with a large number of both rare and common diseases.
We have established a portfolio of programs by selecting disease targets based on a number of criteria, including high unmet medical need, advantages of CRISPR/Cas9 relative to alternative approaches, technical feasibility and the time required to advance the product candidate into and through clinical trials. For CRISPR/Cas9-based therapeutics, technical feasibility is primarily determined by the delivery modality and by the editing strategy required to treat the disease.
CRISPR Therapeutics’ mission is to develop transformative gene-based medicines that offer the possibility of curative treatments. Our therapeutic approach is to cure diseases at the molecular level using CRISPR/Cas9 gene editing.
Our portfolio of therapeutic candidates spans three main categories: ex vivo treatments for hemoglobinopathies, such as sickle cell disease and β-thalassemia, allogeneic CAR-T cell therapies for cancer, and in vivo treatments for diseases of the liver, lung, and muscle.
We are focused on the treatment of somatic (non-germline) cells – cells that are not heritable. We are not using human germline modifications and we support the current recommendations of the International Society for Stem Cell Research in this regard. Together with other gene-editing companies, we have issued a Joint Statement - Position Regarding Human Germline Gene Editing. Our development of transformative new medicines will involve working closely with patients and families, healthcare professionals, regulatory agencies and other groups dedicated to improving healthcare.