Performing more than one gene edit, in sequence as part of a single process or therapy – for example, multiple knockout edits or a knockout edit followed by a targeted gene insertion.
Intellia has developed a proprietary sequential genome editing process in primary human T cells that can lead to the knockout of multiple genes.
Type of gene editing performed in somatic cells to induce non-heritable changes. These edits alter the cells that cannot contribute to the germline and thus cannot be passed down to offspring.
Non-reproductive cells in the body. Most genetic diseases manifest in somatic cells. The DNA in these cells is non-heritable, which means that somatic cell genome editing affects only the patient and will not be genetically transmitted to future children. Examples of these types of cells are cells that make up the retina, liver, heart, etc.
Cells from which all other cells with specialized functions are generated. Under the right conditions (in the body or laboratory), stem cells divide to form more specialized cells. Stem cells can be guided into becoming specific cells that can be used to regenerate and repair diseased or damaged cells in people.
Refers to a drug or therapy that uses substances that are injected into the body and travel through the bloodstream to deliver the therapy to the area of interest. The NTLA-2001 Phase 1 clinical trial is the first time a CRISPR/Cas9 gene editing treatment will be delivered through a vein of the human body to edit genes.
Type of white blood cell, or leukocyte, essential to the immune system. Intellia is engineering T cells against specific cancer antigens.
A gene introduced into the genome by artificial (e.g. not found in nature) means. To perform targeted gene insertion, a transgene must be delivered to the cell nucleus and once there, fulfill the intended role of the gene, such as restoring protein production or recognizing an immune antigen.
Translocation is when a portion of a chromosome fuses to a different chromosome. Genetic translocations are typically unintended and, in specific circumstances, may have negative consequences, such as causing serious disorders like cancer.
Also known as ATTR amyloidosis. A rare genetic disease caused by accumulation of misfolded transthyretin (TTR) protein, which affects the nerves, heart, kidneys and eyes. Patients can develop amyloidosis by inheriting the faulty TTR gene from a parent (hereditary ATTR amyloidosis, ATTRv or hATTR) or due to a natural form of this protein, without genetic mutation (wild-type ATTR amyloidosis, ATTRwt or wtATTR).
Transthyretin (ATTR) amyloidosis resulting in heart muscle disease, and manifested through symptoms that include shortness of breath, palpitations and abnormal heart rhythm, ankle swelling (edema), fainting, fatigue and chest pain (angina). Can also lead to heart failure.
