Intellia Therapeutics is committed to developing genome editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are currently investigating our CRISPR/Cas9 technology in a clinical trial as a single-dose treatment.

Overview of Intellia’s CRISPR/Cas9 Technology

Utilizing Intellia’s in vivo liver knockout editing approach, Intellia is developing several experimental treatments for patients living with genetic diseases. Each treatment consists of a particle specifically designed to deliver to the liver a simple, two-part genome editing system: the messenger RNA that encodes the Cas9 protein and guide RNA specific to the target gene.

These individual components by themselves are inactive but, when combined together and delivered to liver cells, they modify a specific DNA sequence within the target gene. If this process happens in enough cells, the target gene may be permanently changed, leading to an impact on disease. The study currently underway is the first time a CRISPR/Cas9 genome editing treatment will be delivered through a vein of the human body to edit genes.

Genetics and CRISPR: 101

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How Intellia's CRISPR/Cas9 Technology Works

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What is a Clinical Trial?

A clinical trial is a research study conducted in human volunteers to test a potential treatment for safety, efficacy, dosing and more. A clinical trial also is the primary method to evaluate if a potential new therapy is safe and effective.

The number of participants is usually smaller in clinical trials for drugs that treat rare diseases. The clinical trial process typically follows four phases:

Why Volunteer to Participate?

Clinical trials are required in order to bring new treatments and therapies to market. These studies evaluate the effects of experimental treatment on patients and help researchers to better understand the disease, as well as its diagnosis, prevention and potential treatment options. Data from clinical trials also allow regulatory agencies to determine whether the drug or therapy is safe, effective and, ultimately, ready to be made available to the public.

Volunteer participants — whether healthy or patients with the disease — are vital to the success of clinical trials, especially those with rare diseases, as there may be a small number of people diagnosed with the disease. Clinical trial participation offers an opportunity to advance research and help bring new treatments one step closer to patients.

Enrollment

Clinical trials follow a defined protocol for how researchers will conduct the study based on criteria such as age, gender, disease severity and more. These protocols are reviewed and approved by health regulatory authorities in each country where a trial takes place. A screening process will be used to assess if potential participants meet the criteria for any Intellia-sponsored clinical trial. Some volunteers may not meet the eligibility criteria.

Informed Consent

Informed consent is a process used by researchers to ensure potential and enrolled patients are provided ample information about the clinical study. It is intended to protect volunteers by providing enough information so that they can understand all the risks, potential benefits and alternatives associated with the clinical study. The local coordinating investigator of each Intellia clinical trial will review this process with you.

Frequently Asked Questions

  • Is CRISPR/Cas9 new?

    CRISPR/Cas9 was first harnessed in 2012 as a genome editing tool in the lab. More recently, scientists have begun to make breakthroughs in the developments of new therapeutic treatment options for patients with chronic illnesses. Even though no treatments have yet to be approved as marketed products, several are in clinical trials.

  • Can CRISPR/Cas9 edit genes all throughout my body?

    Intellia is developing treatments with CRISPR/Cas9 that will focus on genome editing in somatic cells in the liver. In preclinical (non-human) studies, we observed that the target gene in the liver was edited, resulting in reduced production of the target protein. We also observed that circulating levels of protein produced by the target gene were permanently reduced throughout the body, except for in the eye and brain, which are closed systems.

    Furthermore, the genes in somatic cells can only affect the cells of an individual’s own body. Our investigational treatments are focused on improving the health of the individual and not on future generations.

  • Is CRISPR/Cas9 safe?

    CRISPR/Cas9-based therapies are relatively new, with Intellia’s investigational treatment being the first systemically administered treatment studied in humans. In non-human preclinical studies (e.g., rodents and monkeys), the treatment was evaluated for safe and effective dosing ranges, and Intellia has carefully considered the data from these studies to maximize safety in the clinical trial. In addition, regulatory authorities review these data before allowing us to commence with clinical trials.

  • How long will the effects of the therapy last?

    If there are therapeutic effects with CRISPR/Cas9, successful treatment means a single dose of treatment with potentially permanent changes to bring relief for patients.