Meaning “within the living,” this type of therapy is administered directly into the patient, targeting the cells and editing the genome from inside the body.

In a clinical trial setting, informed consent means that patients considering volunteering to enroll receive information and consent to the purpose of the research, including their role and how the trial will work. Several healthcare team members will review an informed consent form with a volunteer prior to conducting study procedures.

Insertion of a new DNA sequence into the genome to manufacture a desired protein using a gene editing technology, such as the CRISPR/Cas9 system.

In a research or clinical setting, investigational means that the drug has not been approved or authorized for use in patients outside of a clinical trial by any authority that regulates new treatments, such as the U.S. Food and Drug Administration (FDA) or United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA).

Also known as an IND or IND application. Companies like Intellia submit an IND to begin testing of investigational therapies in humans in the U.S. An IND is equivalent to a CTA, the regulatory application required in many countries outside the U.S. to begin clinical testing.

Reduction of protein, with the goal of achieving a therapeutic effect, as a result of a gene or genome editing therapy is often known as protein knockdown.

Inactivation/deletion of a DNA sequence using a gene editing technology, such as the CRISPR/Cas9 system. This is the type of gene edit employed by NTLA-2001.

Also known as LNP. LNPs are fat-based molecules that are the basis of Intellia’s lead product candidates. In those product candidates, an LNP delivers a CRISPR/Cas9 system to liver cells, where it edits the target gene of interest.

Also known as blood cancers or hematological malignancies. Cancers like leukemia, lymphoma and myeloma that originate in blood, bone marrow and lymph.

Also known as mRNA. mRNA, which encodes the Cas9 protein, is one of the components comprising Intellia’s CRISPR/Cas9 LNP delivery system. Intellia researchers are applying this modular LNP system to several experimental treatments by changing only the gRNA sequence.