Cited by 269Amyloidosis: Diagnosis, Treatment, OutcomesAlzheimer's disease research and treatmentsAmyotrophic Lateral Sclerosis Research

Patisiran, an RNAi Therapeutic for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis

Kristen, Arnt V, Ajroud-Driss, Senda, Conceição, Isabel, Gorevic, Peter, Kyriakides, Theodoros, Obici, Laura·2019·Neurodegenerative Disease Management

Abstract

Hereditary transthyretin-mediated amyloidosis is a rapidly progressive, heterogeneous disease caused by the accumulation of misfolded transthyretin protein as amyloid fibrils at multiple sites, and is characterized by peripheral sensorimotor neuropathy, autonomic neuropathy and/or cardiomyopathy. Current treatment options have limited efficacy and often do not prevent disease progression. Patisiran is a novel RNA interference therapeutic that specifically reduces production of both wild-type and mutant transthyretin protein. In Phase II, III and long-term extension studies in patients with hereditary transthyretin-mediated amyloidosis, patisiran has consistently slowed or improved progression of neuropathy. In addition, the Phase III trial demonstrated significant improvements in quality of life measures and indicators of cardiomyopathy. Here, we highlight efficacy and safety data from the patisiran clinical trial programme.

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Crossref · 10.2217/nmt-2018-0033

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104 of 104 references verified0 citation errors · 0 not checkable

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  • For liver targets, lipid nanoparticles and N-acetylgalactosamine conjugates are two clinically validated delivery strategies with multiple regulatory approvals.

    “For liver targets, lipid nanoparticles (LNPs) and N-acetylgalactosamine (GalNAc) conjugates are two clinically validated delivery strategies, with multiple regulatory approvals that include Onpattro® (patisiran) 2,3 , Givlaari® (givosiran) 4 , Oxlumo® (lumaisran) 5 , and Leqvio® (inclisiran) [6][7][8] , and numerous additional programs in various stages of clinical development across several thera”

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Authors
Kristen, A. V., Ajroud-Driss, S., Conceição, I., Gorevic, P., Kyriakides, T., & Obici, L.
Affiliations
  • Heidelberg University (DE)
  • Department of Neurology Northwestern University Feinberg School of Medicine Chicago IL 60611 USA
  • University of Lisbon (PT)
  • Mount Sinai Health System (US)
  • Cyprus Institute of Neurology and Genetics (CY)
  • Policlinico San Matteo Fondazione (IT)
Venue
Neurodegenerative Disease Management
Published
2018-11-27
Volume
9
Issue
1
Pages
5-23
ISSN
  • 1758-2024 (ISSN-L)
  • 1758-2032
OpenAlex
W2903444830
Cited by
268
References
104
Citations by year
2026: 19 · 2025: 35 · 2024: 49 · 2023: 40 · 2022: 47 · 2021: 32 · 2020: 23 · 2019: 23
FWCI
1.91
Type
journal article
Language
en
Open access
closed
Locations
  • Neurodegenerative Disease Management (publishedVersion)
  • PubMed (publishedVersion)
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Amyloidosis: Diagnosis, Treatment, Outcomes
  • Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
  • Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
Keywords
patisiran, hereditary transthyretin amyloidosis, prealbumin, RNA interference, amyloidosis, neuropathy, cardiomyopathy, quality of life, drug efficacy, drug safety, clinical trials
MeSH terms
RNAi Therapeutics, Clinical Trials as Topic, Humans, Amyloid Neuropathies, Familial, RNA, Small Interfering

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