Want More? Connect With Us!

To stay up to date on the latest news about Intellia’s technology, pipeline, data releases, and congress presence, sign up to receive email updates from Intellia Medical Affairs.

Sign up for updates
Menu

Want More? Connect With Us!

To stay up to date on the latest news about Intellia’s technology, pipeline, data releases, and congress presence, sign up to receive email updates from Intellia Medical Affairs.

Sign up for updates

CRISPR:

Seeking to Redirect the
Course of Chronic Diseases With

PINPOINT ACCURACY
An illustration of CRISPR gene editing

Therapeutic Potential of CRISPR Gene Editing

An illustration of CRISPR gene editing

Therapeutic approaches that use genetic medicine include RNA therapies, gene therapy, and gene editing.1

Based on Nobel Prize–winning research, investigational CRISPR-based gene editing therapies have been designed to precisely edit a predefined DNA sequence in a gene known to be associated with a disease.2-4

Learn more about how CRISPR-based gene editing has the potential to address unmet medical needs.

Introduction to Genetic Medicine

In Vivo Approach

Genes can be edited outside of a patient’s body and reintroduced (ex vivo), or edited directly within the body (in vivo).5

Explore how in vivo CRISPR investigational therapeutic approaches may directly edit genes within the body without the need for extensive immunosuppression or hospitalization, with the goal of treating disease.2,3,6

Intellia’s In Vivo Approach
An illustration of a silhouette of a person with a DNA spiral across their torso
Intellia’s In Vivo Approach

Scientific Exploration in Hereditary Angioedema

Hereditary angioedema (HAE) is a genetic disease that causes recurrent, unpredictable, and potentially life-threatening swelling attacks in patients.7-9

CRISPR and Hereditary Angioedema

Scientific Exploration in Transthyretin Amyloidosis

Transthyretin amyloidosis (ATTR) is a progressive and often fatal disease with unmet treatment needs.10,11

CRISPR and Transthyretin Amyloidosis
A question mark

Common Questions 

About CRISPR

Explore helpful resources and frequently asked questions about this groundbreaking technology.  

CRISPR Educational Resources

Connect With Intellia

Our goal is to equip healthcare professionals with the essential knowledge to understand the science of CRISPR and its potential as a therapeutic option to support informed decision-making.

Please submit any specific questions here:

Inquire Here
Visit Intelliatx.com to Learn More About Intellia and Its Product Pipeline.
  1. Liu F, et al. MedComm (2020). 2024;5(9):e645.
  2. Gillmore JD, et al. N Engl J Med. 2021;385(6):493-502.
  3. Longhurst HJ, et al. N Engl J Med. 2024;390(5):432-441.
  4. Press release. The Nobel Prize in Chemistry 2020. October 7, 2020. Accessed March 12, 2025.
    https://www.nobelprize.org/prizes/chemistry/2020/press-release/
  5. Li H, et al. Signal Transduct Target Ther. 2020;5(1):1.
  6. Longhurst HJ, et al. N Engl J Med. 2024;390(5):432-441 (protocol).
  7. Bork K, et al. J Allergy Clin Immunol. 2012;130(3):692-697.
  8. Farkas H. Allergy Asthma Clin Immunol. 2010;6(1):19.
  9. Mendivil J, et al. Orphanet J Rare Dis. 2021;16(1):94.
  10. Kittleson MM, et al. J Am Coll Cardiol. 2023;81(11):1076-1126.
  11. Adams D, et al. Curr Opin Neurol. 2016;29(suppl 1):S14-S26.