Pieter Cullis’ quest for a lipid-based, fusogenic delivery system for nucleic acid therapeutics: success with siRNA so what about mRNA?
Abstract
For the best part of 40 years, lipids and membrane fusion have been at the center of Pieter's research. Projects range from the purely academic quest of understanding the roles of lipids in biological membranes, to the translation of this knowledge into the most advanced lipid nanoparticle (LNP) drug delivery systems in clinical trials to-date. Pieter's pioneering work in lipid polymorphism and characterizing the unique properties of unsaturated phospatidyethanolamines (PE), together with the introduction of ionizable, dialkylamino lipids to trigger membrane fusion at acidic pH, provided the foundation on which a new generation of highly potent, well-tolerated LNPs for intravenous delivery of nucleic acid therapeutics has been built. In this contribution to the special edition honoring Pieter's achievements we highlight key research conducted in Pieter's laboratory and at several biotechnology companies, some spun out of his research group, which resulted in the development of a fusogenic delivery system for siRNA therapeutics. Patisiran®, an LNP encapsulating siRNA for hepatic gene silencing, is currently in Phase III clinical trials for treatment of Transthyretin amyloidosis as are several other siRNA products employing this delivery technology. Finally, we describe more recent work in which the platform shows real promise in the rapidly growing new field of mRNA therapeutics.
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LNP formulations optimized for siRNA delivery do not efficiently deliver large nucleic acids such as mRNAs and plasmids.
“However, LNP formulations that were optimized for small interfering RNA (siRNA) delivery do not efficiently deliver large nucleic acids (e.g., mRNAs and plasmids) (7, 8).”
From CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy (2020) · cites this paper as [7]
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- Authors
- Tam, Y. K., Madden, T. D., & Hope, M. J.
- Affiliations
- Acuitas Therapeutics (Canada) (CA)
- Corresponding author
- Tam, Ying K.
- ORCID
- Tam, Ying K.
- Venue
- Journal of Drug Targeting
- Publisher
- Informa UK Limited
- Published
- 2016-09-02
- Volume
- 24
- Issue
- 9
- Pages
- 774-779
- ISSN
- 1026-7158 (ISSN-L)
- 1061-186X
- 1029-2330
- OpenAlex
- W2485983625
- Cited by
- 20
- References
- 58
- Citations by year
- 2025: 3 · 2024: 1 · 2023: 3 · 2022: 4 · 2021: 2 · 2020: 2 · 2019: 4 · 2017: 1
- FWCI
- 0.15
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Journal of drug targeting (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Lipid Membrane Structure and Behavior
- Keywords
- lipid nanoparticles, siRNA delivery, mRNA therapeutics, lipid polymorphism, membrane fusion, intravenous delivery, patisiran, transthyretin amyloidosis, nucleic acid therapeutics
- MeSH terms
- Humans, Lipids, RNA, Messenger, Genetic Therapy, Gene Transfer Techniques, RNA, Small Interfering, Nanoparticles
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