With CRISPR/Cas9, we aim to target the source of genetically-defined diseases and engineer the next generation of cellular therapies
Genetically-defined disease: Many diseases have a genetic cause, including more than 10,000 monogenic diseases caused by mutations in individual genes. CRISPR/Cas9 may enable us to repair the genetic defects that underlie these diseases.
Cellular engineering: Cell therapies have begun to make a meaningful impact in certain diseases. With CRISPR/Cas9, we can potentially engineer the genomes of cellular therapies to make them more efficacious, safer and available to a broader group of patients.
At CRISPR Therapeutics, we aim to develop transformative gene-based medicines based on CRISPR/Cas9 gene editing. For genetically-defined diseases, we can use a guide RNA that directs Cas9 to cut DNA at a specific site in a disease-causing gene, or at a different site, such as a region that regulates genes, to ameliorate the genetic defect through gene disruption or correction. For cell therapies, we can target genes that when disrupted may improve the safety or efficacy of the therapy, or precisely insert new genes to give the cells new abilities. In either case, we may edit cells either ex vivo (outside the body) or in vivo (inside the body).
Our development of transformative new 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.
Our lead program in hemoglobinopathies uses ex vivo gene editing
We are pursuing both viral and non-viral approaches to deliver CRISPR/Cas9 in vivo
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