Intellia Therapeutics is committed to developing 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. We are currently investigating our 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. technology in a clinical trialA 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. as a single-dose treatment.
For questions about Intellia’s clinical trial, please call us at 833-888-0387 or email us at clinicalscience@intelliatx.com.
Overview of Intellia’s CRISPR/Cas9 Technology
Utilizing Intellia’s 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. liver knockoutInactivation/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. 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 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. system: the messenger RNAAlso 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. that encodes the Cas9 protein and guide RNAAlso known as gRNA. gRNA is one of two components comprising Intellia’s CRISPR/Cas9 gene editing system. Intellia researchers are applying this modular system to several experimental medicines by changing only the gRNA sequence to reprogram for a specific genetic target. 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 DNAAcronym for deoxyribonucleic acid, the hereditary material in humans and almost all other organisms. DNA can be found in the cell nucleus and contains the genetic instructions for the development, functioning, growth and reproduction of all known organisms. Nearly every cell in a person’s body has the same 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/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. 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 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 trialA 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. 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 trialA 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. 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 trialA 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.. Some volunteers may not meet the eligibility criteria.
Informed Consent
Informed consentIn 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. 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 trialA 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. will review this process with you.
Frequently Asked Questions
Is CRISPR/Cas9 new?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. was first harnessed in 2012 as a 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. 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/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. that will focus on 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. in somatic cellsNon-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. 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 investigationalIn 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). 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 investigationalIn 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). 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 trialA 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.. 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/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., successful treatment means a single dose of treatment with potentially permanent changes to bring relief for patients.