Also known as AML. Cancer of the blood and bone marrow that is rapidly fatal without immediate treatment, and is the most common type of acute leukemia in adults.

Also known as AAV. AAVs are small viruses used to transport genetic code to target cells and are commonly used in traditional gene therapy. Intellia’s partner Regeneron is combining AAVs with LNPs to develop a CRISPR/Cas9-based approach for targeted gene insertion, which could overcome challenges of AAV gene therapy.

Cell therapy comprising immune cells collected from healthy donors, rather than the patient, and then modified ex vivo (or outside the donors or patient’s bodies) to restore their function or add therapeutic properties, for example, to target specific cancer cells. After modification, cells are administered to the patient.

A protein component of the immune system that circulates in the blood and recognizes and fights pathogens. Over time or through administration of a vaccine, the body can build immunity through the production of antibodies to protect against pathogens in the future.

Antigens are unique to different pathogens, which include viruses, fungi and abnormal cells such as tumor cells. Antigens alert the immune system to foreign substances not recognized by the body, leading to immune responses like destruction of abnormal cells or production of antibodies.

Also known as B lymphocytes, B-cells are a type of white blood cell that is responsible for making antibodies. These cells are part of the immune system and develop from stem cells in the bone marrow.

Also known as biological markers, biomarkers are utilized to measure biological processes or responses to an exposure or intervention such as a therapeutic agent.

A biologics license application (BLA) is a formal submission to the U.S. Food and Drug Administration requesting approval to distribute and market a new biologic.

A peptide released from the biological pathway involving the KLKB1 gene and kallikrein activity. A reduction of kallikrein activity achieved through knockout of the KLKB1 gene is expected to correlate with a decrease in bradykinin activity. In turn, this decrease in bradykinin activity is expected to prevent the activation of endothelial cells that causes vascular leakage and angioedema in HAE patients.

CAR-T cell stands for chimeric antigen receptor (CAR) T cell therapy. This therapy type modifies  immune cells (T cells) to express a receptor on their surface that recognizes and binds to molecules (antigens) on the surface of malignant or cancerous cells. Once the receptor binds to a tumor antigen, the T cell is activated to attack the malignant cells.