Intellia is committed to working closely with healthcare professionals to improve the standard of care for patients by harnessing the power of CRISPR/Cas9-based Also 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.. We are developing potential single-adminstration therapies for people living with severe and life-threatening conditions, including cancer and genetic disorders.
How We Are Developing CRISPR/Cas9-Based Treatments
At Intellia, we utilize a modular Also 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. platform to create a diverse pipeline, including programs that apply A 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 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., where the Adapted 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. components are delivered as the therapy, and Also 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 cells to repair them to their desired functions. The engineered cells are then administered to the patient so they can treat a particular disease., where we use our CRISPR/Cas9 technology as a tool to create engineered, Type 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 a healthy 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..
Access to Our Investigational Medicines
Intellia is committed to solving the complex challenges of making CRISPR/Cas9-based medicines a reality for patients suffering with severe and life-threatening genetic diseases.
Under certain circumstances, a person suffering a serious or life-threatening disease may ask to use an experimental treatment outside a A test of a new medical treatment or procedure in human volunteers with the purpose of evaluating a medical, surgical or behavioral intervention. Clinical trials typically follow preclinical (non-human) studies and are the primary way that clinical researchers and regulatory agencies evaluate whether a new treatment is safe and effective in people., before its safety and efficacy have been fully evaluated, and before the regulatory authorities have approved it. Intellia appreciates the intent of expanded access programs but, at this time, we can best advance the development of these potential promising products by enrolling patients in clinical trials.
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