Crispr Therapeutics is translating the revolutionary gene-editing technology into transformative therapies to treat hemoglobinopathies, cancer, diabetes, and other diseases.
The gene-editing tool CRISPR/Cas9 is a leading exponent of ex-vivo applications. Among others this approach involves taking cells from a patient, correcting disease-causing genes, or even inserting new genes and re-administering the edited cells to the patient.
The biotechnology company is pursuing two clinical trials in sickle cell anemia and beta thalassemia in cooperation with Vertex Pharma. Patients who have sickle cell disease have red blood cells that take on a crescent or sickle shape under certain circumstances. Patients who have beta thalassemia have less hemoglobin. This disease can result in fewer red blood cells than normal, or in anemia. Both diseases are inherited genetic disorders and Crispr Therapeutics’ gene-editing approach is designed to edit blood cells to increase hemoglobin.
Beyond these two clinical trials, the company is pursuing three cell-based projects in immune-oncology on its own. CRISPR/Cas9 aims to repair the genetic defects that underlie genetically-defined diseases and engineer the genomes of cellular therapies to make them more efficacious.