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CRISPR Educational Resources

Medicine is advancing rapidly, with new therapeutic innovations and biotechnologies shaping the future of patient care every day. We understand that staying informed in this rapidly evolving landscape can be challenging. Our goal is to empower healthcare professionals with the latest, most relevant information, ensuring you have the tools you need to help patients make well-informed decisions about their care. To support this, we’ve developed educational videos and compiled a collection of common patient and provider questions about CRISPR.

FAQ

We’ve compiled frequently asked questions by healthcare professionals about CRISPR. 

CRISPR is a gene editing method that has been studied and used in a variety of applications since its discovery in 1987.1-3 In addition to its agricultural use in ensuring global food security,4 scientists have been investigating CRISPR for its potential therapeutic value for over 10 years, achieving the first in vitro CRISPR-mediated gene editing in human cells in 2013.5 Intellia’s cofounder, Jennifer Doudna, was awarded the Nobel Prize in Chemistry in 2020 for the development of CRISPR/Cas9 as a therapeutic gene editing modality.3,6
CRISPR continues to be studied for its therapeutic potential, with Intellia being the first to optimize this technology for in vivo clinical applications.7,8 Intellia is evaluating the safety and efficacy of two investigational in vivo CRISPR-based therapies currently in late-stage clinical studies, with more than 600 patients already dosed.9-14
CRISPR technology is being investigated to treat both genetically inherited diseases and acquired diseases caused by an aberrantly behaving wild-type protein.15,16
DSBs occur naturally and commonly in human cells.17,18 Under normal conditions, dividing mammalian cells experience about 10 to 50 DSBs per day per cell, many caused by standard DNA replication and subsequently repaired by natural DNA repair processes.17,19
Nonhomologous end joining is one of the cell’s natural DNA repair processes and can introduce small DNA insertions and deletions (indels).19 CRISPR-based therapies leverage this natural DNA repair process to potentially modify the target gene and the associated protein by introducing indels, which may address the disease at the genetic level.15,16,18,20
No. Intellia’s CRISPR platform is designed to target somatic cells, not germline cells. Gene edits in somatic cells only affect patients being treated and do not get passed on to their children.21 Intellia is not currently pursuing germline gene editing research.22
Intellia is conducting several clinical studies that evaluate the safety of their CRISPR-based therapies.10-12 More than 600 patients have already dosed with Intellia’s investigational in vivo CRISPR-based therapies.10-14 Intellia will continue to monitor safety as part of long-term follow-up studies.23-25 We expect to follow patients enrolled in the studies for up to 15 years.23,24
Intellia’s proprietary nonviral lipid nanoparticle delivery system delivers the CRISPR cargo that is designed to precisely target cells/tissues of interest. The therapies are being investigated with the objective to maximize efficacy and minimize systemic toxic effects.15,16
Before initiating clinical trials for its investigational CRISPR-based therapies, Intellia conducts comprehensive computational and empirical testing to identify gRNA sequences with the goal of developing therapies with high efficacy and specificity.15,16,26 All potential off-target locations are identified from multiple genome-wide studies and aggregated to understand a candidate gRNA’s editing precision landscape. Candidate gRNAs are then tested at supratherapeutic doses to sensitively verify which locations may harbor unintended editing activity.15,26
Intellia’s CRISPR-based therapies are designed with components that have been shown to clear from circulation within 5 days following single administration in nonhuman primates.27,* 
*These data are from an animal model; the relevance to humans has not been established.

Intellia’s investigational in vivo CRISPR-based therapies are designed to be administered as a one-time treatment via intravenous infusion in an outpatient setting, and may require some pre-medication.15,16,28,29 Ex vivo CRISPR-based therapies require initial cell removal, followed by infusion of the therapy/cell mixture into the body, and may require extensive hospitalization and immunosuppression.30

Intellia’s investigational in vivo CRISPR-based therapies are designed to be administered as a one-time treatment via intravenous infusion in an outpatient setting, and may require some premedication.15,16,28,29 Intellia’s current investigational therapies aim to provide a durable effect on disease activity with a single treatment, with the goal to not require multiple doses.15,16
CRISPR creates precise DNA edits by inducing targeted double-strand breaks in a gene.16,31 CRISPR-mediated gene editing has been studied for several decades, culminating in the first in vivo late-stage clinical trial of a CRISPR-based therapy.1,32,33 Intellia has developed a rigorous workflow designed to optimize precise on-target editing with CRISPR.15,16,26
Base and prime editing are derivative CRISPR technologies that introduce single-strand breaks instead of DSBs and use variations in DNA-modifying biochemistry to edit.33 Base editing chemically changes the identity of nucleotides within the DNA strand, and prime editing uses a template to synthesize a new DNA strand at the target location.33 These and other approaches to develop CRISPR-based therapies are the subject of ongoing investigations.29

Communicating About CRISPR to Patients

Watch the following videos for ideas on how to potentially address common patient questions about CRISPR.
Communicating CRISPR to Patients
Communicating CRISPR/Cas9 to Patients video
View Transcript
Communicating the Potential Outcomes of Genetic Modification to Patients
Communicating the Potential Outcomes of Genetic Modification to Patients video
View Transcript
Addressing Common Patient Questions on CRISPR
Addressing Common Patient Questions on CRISPR/Cas9 video
View Transcript

Introduction to CRISPR

Rationale for CRISPR-Based Therapies
Rationale for CRISPR-based Therapies: Unmet Needs in Standard of Care Treatment video
View Transcript
Understanding CRISPR Components
Understanding CRISPR/Cas9 Components video
View Transcript
Gene Editing Exploration with CRISPR
Gene Editing Exploration with CRISPR video

Intellia’s Approach to CRISPR

Understanding CRISPR Gene Editing and Intellia Therapeutics’ Approach
Understanding CRISPR/Cas9 Gene Editing and Intellia Therapeutics' Approach video
View Transcript
Intellia’s Approach to CRISPR
Intellia's Approach to CRISPR/Cas9 video
View Transcript
Intellia’s Approach to Responsible Innovation in CRISPR
Intellia's Approach to Responsible Innovation in CRISPR/Cas9 video
View Transcript

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. Gostimskaya I. Biochemistry (Mosc). 2022;87(8):777-788.
  2. Ishino Y, et al. J Bacteriol. 1987;169(12):5429-5433.
  3. Press release. The Nobel Prize in Chemistry 2020. October 7, 2020. Accessed December 14, 2025.
    https://www.nobelprize.org/prizes/chemistry/2020/press-release/
  4. Ahmad A, et al. Front Plant Sci. 2023;14:1232938.
  5. Jinek M, et al. eLife. 2013;2:e00471.
  6. The Nobel Prize. Jennifer A. Doudna facts. Updated 2020. Accessed December 14, 2025.
    https://www.nobelprize.org/prizes/chemistry/2020/doudna/facts/
  7. Press release. Intellia Therapeutics. November 9, 2020. Accessed December 14, 2025. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-doses-first-patient-landmark-crisprcas9
  8. Press release. Intellia Therapeutics. March 18, 2024. Accessed December 14, 2025. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-first-patient-dosed-phase-3
  9. ClinicalTrials.gov identifier: NCT06128629. Updated December 2, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT06128629.
  10. ClinicalTrials.gov identifier: NCT04601051. Updated May 1, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT04601051
  11. ClinicalTrials.gov identifier: NCT05120830. Updated September 19, 2024. Accessed December 12, 2025. https://clinicaltrials.gov/study/NCT05120830
  12. ClinicalTrials.gov identifier: NCT06634420. Updated December 2, 2025. Accessed December 12, 2025. https://clinicaltrials.gov/study/NCT06634420
  13. Press release. Intellia Therapeutics. September 18, 2025. Accessed November 11. 2025. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-completes-enrollment-global-phase-3-haelo
  14. Press release. Intellia Therapeutics. October 27, 2025. Accessed November 11, 2025. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-provides-update-magnitude-clinical-trials
  15. Gillmore JD, et al. N Engl J Med. 2021;385(6):493-502.
  16. Longhurst HJ, et al. N Engl J Med. 2024;390(5):432-441.
  17. Chang HHY, et al. Nat Rev Mol Cell Biol. 2017;18(8):495-506.
  18. Lieber MR. Annu Rev Biochem. 2010;79(1):181-211.
  19. Cannan WJ, Pederson DS. J Cell Physiol. 2016;231(1):3-14.
  20. Varga T, Aplan PD. DNA Repair (Amst). 2005;4(9):1038-1046.
  21. Saha K, et al. Nature. 2021;592(7853):195-204.
  22. Intellia Company Overview. Intellia Therapeutics. June 2024.
  23. ClinicalTrials.gov identifier: NCT05697861. Updated August 26, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT05697861
  24. ClinicalTrials.gov identifier: NCT06262399. Updated November 28, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT06262399
  25. ClinicalTrials.gov identifier: NCT06672237. Updated November 26, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT06672237
  26. O’Connell DJ. Presented at: 24th Annual Meeting of the American Society of Gene and Cell Therapy; May 10, 2021 (Virtual).
  27. Wood K, et al. Poster presented at: 2nd European Congress for ATTR Amyloidosis; September 1-3, 2019; Berlin, Germany.
  28. Gillmore JD, et al. Protocol. N Engl J Med. 2021;385(6):493-502.
  29. Longhurst HJ, et al. Protocol. N Engl J Med. 2024;390(5):432-441.
  30. Exagamglogene autotemcel. Package insert. Vertex Pharmaceuticals Incorporated; 2023.
  31. Nambiar TS, et al. Mol Cell. 2022;82(2):348-388.
  32. Press release. Intellia Therapeutics. May 9, 2024. Accessed December 14, 2025. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-hold-conference-call-discuss-first-4
  33. Li T, et al. Signal Transduct Target Ther. 2023;8(1):36.