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Scientific Exploration in Transthyretin Amyloidosis

Aiming to address the underlying cause of transthyretin amyloidosis1

About Transthyretin Amyloidosis

Transthyretin amyloidosis (ATTR) is a progressive and often fatal disease,2-4 caused by the misfolding of transthyretin (TTR) protein into amyloid fibrils.5 Aggregation of these amyloid fibrils results in ATTR with cardiomyopathy (CM) and/or polyneuropathy (PN).6,7

An illustration showing transthyretin amyloidosis (ATTR)

mRNA, messenger RNA; TTR, transthyretin; TTR, transthyretin gene that encodes transthyretin protein.

Therapeutic Landscape

Currently, two distinct therapeutic modalities are indicated for ATTR-CM: one targets TTR at the protein level, and the other at the RNA level.3,8 Available modalities for ATTR-PN target TTR at the RNA level.3
Investigational approaches aim to expand treatment options for ATTR by targeting TTR at the protein, RNA, and DNA levels.9-20 
An illustration showing the therapeutic landscape of transthyretin amyloidosis (ATTR)

DNA, deoxyribonucleic acid; FDA, US Food and Drug Administration; mRNA, messenger RNA; RNA, ribonucleic acid; TTR, transthyretin; TTR, transthyretin gene that encodes transthyretin protein.

Rationale for CRISPR Research

ATTR is a monogenic disease with both hereditary and acquired forms resulting from TTR protein misfolding1,5:

  • Hereditary ATTR: mutation in the gene that codes for TTR protein leads to misaggregation of TTR protein
  • Acquired ATTR: spontaneous, nongenetic misaggregation of wild-type TTR protein
Circulating TTR protein is produced almost exclusively (>99%) by the liver.1

Treatment innovation for ATTR continues to progress with multiple investigational therapy studies that target the root cause of disease.9-16 ATTR may be a good therapeutic target for CRISPR-based gene editing because it involves a single gene (TTR) and TTR protein is primarily produced in the liver.1

Learn More About How CRISPR Works

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.

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  1. Gillmore JD, et al. N Engl J Med. 2021;385(6):493-502.
  2. Nativi-Nicolau J, et al. ESC Heart Fail. 2021;8(5):3875-3884.
  3. Kittleson MM, et al. J Am Coll Cardiol. 2023;81(11):1076-1126.
  4. Adams D, et al. J Neurol. 2021;268(6):2109-2122.
  5. Tschöpe C, Elsanhoury A. J Clin Med. 2022;11(8):2148.
  6. Witteles RM, et al. JACC Heart Fail. 2019;7(8):709-716.
  7. Shin SC, Robinson-Papp J. Mt Sinai J Med. 2012;79(6):733-748.
  8. Vutrisiran. Package insert. Alnylam Pharmaceuticals; 2025.
  9. ClinicalTrials.gov identifier: NCT04601051. Updated May 1, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT04601051
  10. ClinicalTrials.gov identifier: NCT06128629. Updated December 2, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT06128629
  11. ClinicalTrials.gov identifier: NCT06539208. Updated August 6, 2024. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT06539208
  12. ClinicalTrials.gov identifier: NCT04153149. Updated November 20, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT04153149
  13. ClinicalTrials.gov identifier: NCT03860935. Updated June 27, 2024. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT03860935
  14. ClinicalTrials.gov identifier: NCT06183931. Updated November 17, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT06183931
  15. ClinicalTrials.gov identifier: NCT06260709. Updated November 25, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT06260709
  16. ClinicalTrials.gov identifier: NCT04360434. Updated November 11, 2025. Accessed December 14, 2025. https://clinicaltrials.gov/study/NCT04360434
  17. Press release. YolTech Therapeutics. July 11, 2024. Accessed March 11, 2025. https://www.yoltx.com/news/press-release/65
  18. Press release. Alnylam Pharmaceuticals. October 9, 2024. Accessed March 11, 2025. https://investors.alnylam.com/press-release?id=28446
  19. Press release. BridgeBio Pharma. February 5, 2024. Accessed March 11, 2025. https://investor.bridgebio.com/news-releases/news-release-details/bridgebio-pharma-announces-us-food-and-drug-administration-fda
  20. Fontana M, et al. Eur Heart J. 2022;43(suppl 2):ehac544.1767.