Overview

Developing curative genome editingAlso called gene editing. Genome editing collectively refers to a set of technologies, including CRISPR/Cas9, that can be used to cut and modify DNA. Genome editing uses systems to make the DNA change inside the cell. These cells can be edited in the body (in vivo) or outside the body (ex vivo) from a patient or donor. treatments that can positively transform the lives of people living with severe and life-threatening diseases.

Our researchers work tirelessly to harness the genomeA genome is an organism’s complete set of DNA, including all of its genes. Each genome contains all of the information needed to build and maintain that organism. In humans, a copy of the entire genome—more than three billion DNA base pairs—is contained in all cells that have a nucleus. editing technology CRISPR/Cas9Adapted from a naturally occurring bacterial immune system, CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. One of the proteins in the CRISPR system is known as CRISPR-associated 9 protein or Cas9 protein, which acts as a pair of ‘molecular scissors’ to cleave DNA. Researchers have co-opted the bacterial CRISPR/Cas9 system to make specific changes in the DNA of humans, other animals and plants. CRISPR/Cas9 was first harnessed in 2012 as a genome editing tool in the lab. More recently, scientists have begun engineering and testing CRISPR systems to be very specific to a desired genetic target. for human therapeutic use. Jennifer Doudna, an Intellia co-founder, and Emmanuelle Charpentier were awarded the 2020 Nobel Prize in Chemistry for their pioneering work in CRISPR. We at Intellia are humbled to have a hand in making what we believe to be medical history. As a leader in this space, we take this responsibility to patients seriously.

We are employing a modular genome editing platform to create diverse in vivoMeaning “within the living”, this type of therapy is administered directly into the patient, targeting the cells and editing the genome from inside the body. and ex vivoAlso referred to as a cell therapy. In an ex vivo therapy, cells are removed from the body for modification. Modification is done by administering therapy directly to the cells before they are returned to the body. In the case of ex vivo CRISPR/Cas9 therapies, CRISPR/Cas9 is used to modify the extracted cells to repair them back to their proper function or add desired functions. The engineered cells are then administered to the patient so they can treat a particular disease. pipelines, spanning a range of therapeutic indications. Guided by this full-spectrum approach, we are committed to making CRISPR/Cas9-based medicines a reality for patients suffering from genetic diseases and to creating novel engineered cell therapiesType of therapy where engineered cells are transferred into a patient’s body to grow, replace or repair damaged tissue, or perform another desired function. Cells used in these therapies may originate from the patient (autologous cells) or from a donor (allogeneic cells). A common type of cell therapy is blood transfusions, where red blood cells, white blood cells and platelets from one or more donors are transferred into the body of a patient. for various cancers and autoimmune diseases.