Pioneering Precision: Cellectis Develops Immunotherapies for Genetic Diseases and Disorders Through Cutting-edge Gene Editing
André Choulika, the Chairman and CEO of Cellectis, talks to Lonza about the company's clinical trials of their revolutionary gene editing technology.
Since the arrival of Crispr in 2009, gene editing has made its way into labs across the globe, and its groundbreaking importance was further acknowledged through the 2020 Nobel Prize in Chemistry. Gene editing corrects within the DNA exactly as one edits a text: cutting, copying, replacing and deleting. Taking up the mantle of gene editing for medical treatments, the biopharmaceutical company Cellectis focuses their 20 years of experience on developing cancer immunotherapies. Safety through precision is the ultimate priority for Cellectis. The gene-editing company turns to Crispr for research but has found TALEN to be more precise in therapeutic uses. First, the gene cutter is introduced into a healthy donor T cell to destroy its receptor. This makes it blind so that the T cell doesn't attack the patient's cells (also known as graft versus host disease), and only the CAR carries the information about which cells to attack. The TALEN then travels to the cell via the CAR-T cell and cleaves the gene where it needs to be corrected. Cellectis has several products in the clinical trial stage that are based on allogenic gene-edited CAR-T cells: U CAR-T 22 for acute lymphoblastic leukemia, U CAR-T 123 for acute myeloid leukemia, and U CAR-CS1 for multiple myeloma.
Curious to Know More?
Cellectis CEO André Choulika explains the technology of allogenic gene editing they invented in this episode of the podcast "A View On."KEY TERMS:
TALEN: Transcription Activator-Like Effector Nuclease, or a hybrid protein that is engineered to cut specific sequences of DNA
T cell: an immune cell that circulates in the body and recognizes any foreign body
CAR T cells: T cells engineered with a receptor protein for the purpose of targeting a specific protein on a cell. A CAR protein recognizes the antigen associated with the cancer cells and signals the T cell to fire at the cancer cell.
Allogenic gene therapy: donor T cells are genetically engineered to target a specific protein within the patient to attack the associated antigen of a gene mutation or genetic disease