Lipid metabolism and disorders Peer reviewed

Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3

Luke J Laffin, Stephen J. Nicholls, Russell Scott, Peter Clifton and 9 more

New England Journal of Medicine | Nov 8, 2025 | 34 citations

Abstract

Abstract

Editing of ANGPTL3 was associated with few adverse events and resulted in reductions from baseline in ANGPTL3 levels. (Funded by CRISPR Therapeutics; Australia New Zealand Clinical Trials Registry number, ACTRN12623000809639.).

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Authors

Researchers on this paper

Luke J Laffin

first | Cleveland Clinic | ORCID 0000-0002-3262-792X

Stephen J. Nicholls

middle | Monash University | ORCID 0000-0002-9668-4368

Russell Scott

middle | ORCID 0000-0002-2107-6480

Peter Clifton

middle | Royal Adelaide Hospital | ORCID 0000-0002-6411-626X

John E. Baker

middle | Clinical Trials New Zealand | ORCID 0000-0001-7899-3266

Ashish Sarraju

middle | Cleveland Clinic | ORCID 0000-0003-1649-2110

Shweta Singh

middle | CRISPR Therapeutics (Switzerland) | ORCID 0009-0006-0372-3628

Qiuqing Wang

middle | Cleveland Clinic

Kathy Wolski

middle | Cleveland Clinic

Huansheng Xu

middle | CRISPR Therapeutics (Switzerland)

Jen Nielsen

middle | CRISPR Therapeutics (Switzerland)

Jason M. Duran

middle | CRISPR Therapeutics (Switzerland) | ORCID 0000-0003-1666-2319

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Citation

BibTeX

@article{Laffin2025Phase,
  title = {Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3},
  author = {Luke J Laffin and Stephen J. Nicholls and Russell Scott and Peter Clifton and John E. Baker and Ashish Sarraju and Shweta Singh and Qiuqing Wang and Kathy Wolski and Huansheng Xu and Jen Nielsen and Jason M. Duran and Steven E. Nissen},
  journal = {New England Journal of Medicine},
  year = {2025},
  doi = {10.1056/nejmoa2511778},
  url = {https://doi.org/10.1056/nejmoa2511778}
}

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