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Introduction to Genetic Medicine 

Therapeutic and investigational strategies to treat life-threatening diseases have advanced over time.¹ Innovation has progressed from small molecule drugs and biologics to RNA- and DNA-targeted therapies.²

Genetic Medicine Delivery Approaches

RNA therapies, gene therapy, and gene editing are different approaches of genetic medicine that may enable disease modification.3
RNA Therapies
Examples of RNA therapy

RNA therapies target a specific mRNA, not DNA, to decrease expression of a target protein in a patient.4,5 This approach has demonstrated effectiveness in various diseases, with treatment regimens typically involving dosing intervals ranging from 1 to 6 months.6,7

DNA, deoxyribonucleic acid; mRNA, messenger RNA; RNA, ribonucleic acid.

Gene Therapy
Examples of gene therapy

Gene therapy introduces a modified gene into the nucleus of cells with the goal of partially restoring expression of a functional protein.3

With gene transfer, the modified gene is typically delivered via a viral vector, such as adeno-associated virus (AAV), which has been shown to be effective for various diseases.8

DNA, deoxyribonucleic acid.

Gene Editing
Examples of gene editing
Gene editing aims to precisely target a predefined DNA sequence to make a specific, permanent edit to a gene, typically using CRISPR technology.9,10
Examples of RNA therapy, gene therapy, and gene editing

DNA, deoxyribonucleic acid; mRNA, messenger RNA; RNA, ribonucleic acid.

Why CRISPR?

Potential to Revolutionize Treatment of Diseases

Gene editing with CRISPR is being investigated as a one-time treatment with the goal to durably address disease activity and underlying mechanisms.9-11

Investigational CRISPR-Based Therapies Have the Potential to9,10:

Address the underlying mechanisms of a variety of human diseases by precisely inserting a functional gene or knocking out a gene underlying a disease from a patient’s DNA.11
Provide a one-time treatment intended to have a permanent effect on disease activity.7,10
Reduce healthcare and treatment burden over a patient’s lifetime.11
Address the underlying mechanisms of a variety of human diseases by precisely inserting a functional gene or knocking out a gene underlying a disease from a patient's DNA; Provide a one-time treatment intended to have a permanent effect on disease activity; Reduce healthcare and treatment burden over a patient's lifetime

DNA, deoxyribonucleic acid.

Dr. Jim Januzzi, a cardiologist, shares how he communicates the potential outcomes of gene editing with CRISPR to his patients.

Communicating the Potential Outcomes of Genetic Modification to Patients video
View Transcript
Rationale for CRISPR-based Therapies: Unmet Needs in Standard of Care Treatment video
View Transcript
Dr. Padmalal Gurugama, an allergist and immunologist, discusses unmet needs in standard-of-care treatments.

Introduction to CRISPR

Investigational CRISPR-based therapies are designed to precisely edit genes and alter protein production.12

Editing a Gene Could Alter Production of a Protein Involved in Disease13-17 

An illustration showing how gene editing could alter production of a protein involved in disease
Originally discovered as part of a naturally occurring process in bacteria, CRISPR/Cas9 is a Nobel Prize–winning gene editing technology that precisely edits DNA.10,18-20

Since its discovery in 1987, CRISPR has been an established gene editing method studied and used in a variety of applications.20,21

Cas9, CRISPR-associated protein 9; DNA, deoxyribonucleic acid.

Methods of Gene Editing With CRISPR

Gene editing with CRISPR can alter the production of proteins associated with disease through two mechanisms. It can precisely inactivate a gene that produces a protein associated with disease. Additionally, in instances where a functional protein is lacking, this technology can be used for targeted insertion or repair of a desired gene, potentially resulting in the production of a functional protein.22
Inactivate
An illustration of an inactivated DNA strand
Production of disease-associated protein from target gene is halted
Insert
An illustration of a DNA strand being restored
Production of correct protein is restored
Repair
An illustration of a repaired DNA strand
Production of correct protein is restored

In Vivo

CRISPR can be delivered directly into a patient’s body, also known as in vivo CRISPR.9,10 Investigational in vivo CRISPR therapies are being studied as a one-time intravenous infusion for real-time targeted gene editing.9,10 Depending on the investigational therapy, the in vivo approach may require some premedication, and is intended to be administered in the outpatient setting within a day, with subsequent follow-up and monitoring.23,24

CRISPR, clustered regularly interspaced short palindromic repeats; IV, intravenous.

An illustration showing in vivo CRISPR

CRISPR, clustered regularly interspaced short palindromic repeats; IV, intravenous.

Ex Vivo

CRISPR can be used to create a therapy outside a patient’s body, also known as ex vivo CRISPR.25 This involves retrieving patient or donor cells, editing them using CRISPR, and then reintroducing them into the patient.25 This method can take months, requires hospitalization, and necessitates immunosuppression prior to therapy.26

CRISPR, clustered regularly interspaced short palindromic repeats; IV, intravenous.

An illustration showing ex vivo CRISPR

CRISPR, clustered regularly interspaced short palindromic repeats; IV, intravenous.

For an overview of CRISPR, its rationale for therapeutic investigation, and how to discuss this information with patients, please watch the following video: 
Communicating CRISPR/Cas9 to Patients video
View Transcript

The History of CRISPR Technology

In 2012, scientists demonstrated CRISPR’s potential as a programmable gene editing tool, successfully editing DNA in human cells.33,34 Following thorough preclinical and clinical studies, the first ex vivo CRISPR-based therapy received FDA approval in 2023.26 Ongoing Phase 3 clinical trials are investigating the safety and efficacy of in vivo CRISPR therapies.35-37

The Journey of CRISPR: From Discovery to Innovation

A historical timeline of CRISPR from discovery to innovation

Cas9, CRISPR-associated protein 9; CRISPR, clustered regularly interspaced short palindromic repeats; FDA, US Food and Drug Administration; SCD, sickle cell disease; TDT, transfusion-dependent β-thalassemia.

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. Stern LK, Patel J. Methodist Debakey Cardiovasc J. 2022;18(2):59-72.
  2. Smith TD, Riedl MA. Ann Allergy Asthma Immunol. 2024;133(4):380-390.
  3. Riedl MA, et al. J Allergy Clin Immunol Pract. 2024;12(4):911-918.
  4. Fijen LM, et al. N Engl J Med. 2022;386(11):1026-1033.
  5. Lauffer MC, et al. Commun Med (Lond). 2024;4(1):6.
  6. Inclisiran. Package insert. Novartis; 2024.
  7. Givosiran. Package insert. Alnylam Pharmaceuticals; 2024.
  8. Liu F, et al. MedComm (2020). 2024;5(9):e645.
  9. Longhurst HJ, et al. N Engl J Med. 2024;390(5):432-441.
  10. Gillmore JD, et al. N Engl J Med. 2021;385(6):493-502.
  11. Doudna JA. Nature. 2020;578(7794):229-236.
  12. Hsu PD, et al. Cell. 2014;157(6):1262-1278.
  13. Ay C, Reinisch A. Wien Klin Wochenschr. 2024;137(9-10):261-271.
  14. Bergendahl LT, et al. Protein Sci. 2019;28(8):1400-1411.
  15. Saito Y, et al. Int J Mol Sci. 2021;23(1):25.
  16. Bezerra F, et al. Front Mol Neurosci. 2020;13:592644.
  17. Caccia S, et al. Pediatr Allergy Immunol Pulmonol. 2014;27(4):159-163.
  18. Hillary VE, Ceasar SA. Mol Biotechnol. 2023;65(3):311-325.
  19. Press release. The Nobel Prize in Chemistry 2020. October 7, 2020. Accessed March 12, 2025. https://www.nobelprize.org/prizes/chemistry
    /2020/press-release/
  20. Gostimskaya I. Biochemistry (Mosc). 2022;87(8):777-788.
  21. Ishino Y, et al. J Bacteriol. 1987;169(12):5429-5433.
  22. Our Science. Intellia Therapeutics. Accessed March 12, 2025. https://www.intelliatx.com/our-science/
  23. Gillmore JD, et al. Protocol. N Engl J Med. 2021;385(6):493-502.
  24. Longhurst HJ, et al. Protocol. N Engl J Med. 2024;390(5):432-441.
  25. Li Y, et al. Biomaterials. 2020;234:119711.
  26. Exagamglogene autotemcel. Package insert. Vertex Pharmaceuticals Incorporated; 2023.
  27. ClinicalTrials.gov identifier: NCT05565248. Updated May 23, 2024. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT05565248
  28. ClinicalTrials.gov identifier: NCT05885464. Updated January 13, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT05885464
  29. ClinicalTrials.gov identifier: NCT05643742. Updated November 14, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT05643742
  30. ClinicalTrials.gov identifier: NCT05795595. Updated November 26, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT05795595
  31. Martínez Bedoya D, et al. Front Immunol. 2021;12:640082.
  32. Li H, et al. Signal Transduct Target Ther. 2020;5(1):1-23.
  33. Jinek M, et al. Science. 2012;337(6096):816-821.
  34. Jinek M, et al. Elife. 2013;2:e00471.
  35. Press release. Intellia Therapeutics. October 18, 2023. Accessed March 12, 2025. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-fda-clearance-investigational-0
  36. Press release. Intellia Therapeutics. October 7, 2024. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-initiation-haelo-phase-3-study
  37. Press release. Intellia Therapeutics. November 7, 2024. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-third-quarter-2024-financial
  38. Press Release. CRISPR Therapeutics. February 25, 2019. Accessed September 23, 2025. https://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-progress-clinical
  39. Jakobsen RS. First Chinese CRISPR gene therapy trial demonstrates safety. CRISPR Medicine News. April 28, 2020. Accessed November 11, 2025. https://crisprmedicinenews.com/news/first-chinese-crispr-gene-therapy-trial-demonstrates-safety/
  40. Press Release. Intellia Therapeutics. November 9, 2020. Accessed November 11, 2025. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-doses-first-patient-landmark-crisprcas9