Modifying multiple genes simultaneously.

Researchers often use a murine, or mouse, model for early preclinical testing of experimental treatments. Studies typically progress from a murine model to testing in either a higher rodent species, such as rats, or to NHPs.

Also known as NHP. Preclinical studies advance from rodent to higher species, such as NHP, studies prior to entering clinical (human) testing. Due to their physiological similarity to humans, NHPs are used for research because they are usually the next best model to humans to investigate whether an experimental treatment is ready to enter clinical trials.

A pathogen is any of any foreign substance that the immune system does not recognize and tries to fight off by producing an immune response against it. The immune system identifies these foreign substances through antigens unique to each pathogen.

Sometimes paired with pharmacokinetics to form the abbreviation, PKPD. Pharmacodynamics is how your body reacts to the experimental treatment. PKPD is often part of an objective or endpoint of a clinical trial and is usually analyzed through a blood sample.

Sometimes paired with pharmacodynamics to form the abbreviation, PKPD. Pharmacokinetics is how an experimental treatment moves through the body. PKPD is often part of an objective or endpoint of a clinical trial and is usually analyzed through a blood sample.

Also known as KLKB1 gene. Knocking out this gene is expected to reduce kallikrein activity, which is involved in the biological pathway for release of bradykinin. Intellia expects this reduction to correlate with a decrease in bradykinin activity, thus, preventing the activation of endothelial cells that causes vascular leakage and angioedema in HAE patients.

Using a combination of tissue engineering and molecular biology to treat genes – the root causes of gene-based diseases and disorders. This new field brings together biology, chemistry, computer science, engineering, genetics, medicine, robotics and other fields to find the solution to some of the most challenging health issues in medicine.

Also known as a regulatory agency. The U.S. FDA and the U.K. MHRA are examples of local regulatory authorities responsible for protecting human health. Drug developers like Intellia must seek regulatory approval prior to starting a clinical trial and subsequent product approval.

Correction of “misspelled” disease-driving DNA sequence using a CRISPR/Cas9-based gene editing therapy.