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A Highly Durable RNAi Therapeutic Inhibitor of PCSK9
Open accessAbstract
BACKGROUND: Inclisiran (ALN-PCSsc) is a long-acting RNA interference (RNAi) therapeutic agent that inhibits the synthesis of proprotein convertase subtilisin-kexin type 9 (PCSK9), a target for the lowering of low-density lipoprotein (LDL) cholesterol. METHODS: In this phase 1 trial, we randomly assigned healthy volunteers with an LDL cholesterol level of at least 100 mg per deciliter in a 3:1 ratio to receive a subcutaneous injection of inclisiran or placebo in either a single-ascending-dose phase (at a dose of 25, 100, 300, 500, or 800 mg) or a multiple-dose phase (125 mg weekly for four doses, 250 mg every other week for two doses, or 300 or 500 mg monthly for two doses, with or without concurrent statin therapy); each dose cohort included four to eight participants. Safety, the side-effect profile, and pharmacodynamic measures (PCSK9 level, LDL cholesterol level, and exploratory lipid variables) were evaluated. RESULTS: The most common adverse events were cough, musculoskeletal pain, nasopharyngitis, headache, back pain, and diarrhea. All the adverse events were mild or moderate in severity. There were no serious adverse events or discontinuations due to adverse events. There was one grade 3 elevation in the γ-glutamyltransferase level, which was considered by the investigator to be related to statin therapy. In the single-dose phase, inclisiran doses of 300 mg or more reduced the PCSK9 level (up to a least-squares mean reduction of 74.5% from baseline to day 84), and doses of 100 mg or more reduced the LDL cholesterol level (up to a least-squares mean reduction of 50.6% from baseline). Reductions in the levels of PCSK9 and LDL cholesterol were maintained at day 180 for doses of 300 mg or more. All multiple-dose regimens reduced the levels of PCSK9 (up to a least-squares mean reduction of 83.8% from baseline to day 84) and LDL cholesterol (up to a least-squares mean reduction of 59.7% from baseline to day 84). CONCLUSIONS: In this phase 1 trial, no serious adverse events were observed with inclisiran. Doses of 300 mg or more (in single or multiple doses) significantly reduced levels of PCSK9 and LDL cholesterol for at least 6 months. (Funded by Alnylam Pharmaceuticals and the Medicines Company; ClinicalTrials.gov number, NCT02314442 .).
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“Inclisiran is the first siRNA approved by both US FDA and EMA for the treatment of hypercholesterolemia; the subcutaneous injection of a lipid nanoparticle formulation of the drug, which specifically binds hepatocyte asialoglycoprotein receptors, displays high potency in lowering liver PCSK9 and plasma LDL-C levels [161,162].”
From The Emerging Role of PCSK9 in the Pathogenesis of Alzheimer’s Disease: A Possible Target for the Disease Treatment (2024) · cites this paper as [161]
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“A single subcutaneous injection of trivalent GalNAc-conjugated siRNA can achieve efficacy for >12 months22, 2324.”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [22]
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“However, a fibrate-induced increase in homocysteine may blunt elevation of both HDL-C and ApoA1 levels, and this effect may contribute to the smaller than estimated benefits of fenofibrate in CV outcome parameters [322].”
From 2019 ESC/EAS guidelines for the management of dyslipidaemias: lipid modification to reduce cardiovascular risk (2019) · cites this paper as [322]
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Stone, N. J., Robinson, J. G., Lichtenstein, A. H., Bairey Merz, C. N., Blum, C. B., Eckel, R. H., Goldberg, A. C., Gordon, D., Levy, D., Lloyd-Jones, D. M., et al. (2014) · Circulation · doi:10.1161/01.cir.0000437738.63853.7a · Cited by 7,081
Stone, N. J., Robinson, J. G., Lichtenstein, A. H., Bairey Merz, C. N., Blum, C. B., Eckel, R. H., Goldberg, A. C., Gordon, D., Levy, D., Lloyd-Jones, D. M., et al. (2014) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2013.11.002 · Cited by 3,845
Efficacy and Safety of Alirocumab in Reducing Lipids and Cardiovascular Events
Robinson, J. G., Farnier, M., Krempf, M., Bergeron, J., Luc, G., Averna, M., Stroes, E. S., Langslet, G., Raal, F. J., El Shahawy, M., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1501031 · Cited by 2,296
Efficacy and Safety of Evolocumab in Reducing Lipids and Cardiovascular Events
Sabatine, M. S., Giugliano, R. P., Wiviott, S. D., Raal, F. J., Blom, D. J., Robinson, J., Ballantyne, C. M., Somaratne, R., Legg, J., Wasserman, S. M., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1500858 · Cited by 1,509
Nair, J. K., Willoughby, J. L. S., Chan, A., Charisse, K., Alam, M. R., Wang, Q., Hoekstra, M., Kandasamy, P., Kel’in, A. V., Milstein, S., et al. (2014) · Journal of the American Chemical Society · doi:10.1021/ja505986a · Cited by 1,141
Self-citationSafety and efficacy of RNAi therapy for transthyretin amyloidosis
Coelho, T., Adams, D., Silva, A., Lozeron, P., Hawkins, P. N., Mant, T., Perez, J., Chiesa, J., Warrington, S., Tranter, E., et al. (2013) · New England Journal of Medicine · doi:10.1056/nejmoa1208760 · Cited by 971
Self-citationTabernero, J. M., Shapiro, G. I., LoRusso, P. M., Cervantes, A. M. R., Schwartz, G. K., Weiss, G. J., Paz-Ares, L. G., Cho, D. C., Infante, J. R., Alsina, M., et al. (2013) · Cancer Discovery · doi:10.1158/2159-8290.cd-12-0429 · Cited by 717
Self-citationEffect of a Monoclonal Antibody to PCSK9 on LDL Cholesterol
Stein, E. A., Mellis, S., Yancopoulos, G. D., Stahl, N., Logan, D., Smith, W. B., Lisbon, E., Gutierrez, M., Webb, C., Wu, R., et al. (2012) · New England Journal of Medicine · doi:10.1056/nejmoa1105803 · Cited by 664
Frank-Kamenetsky, M. D., Grefhorst, A., Anderson, N. N., Racie, T., Bramlage, B., Akinc, A., Butler, D., Charissé, K., Dorkin, J. R., Fan, Y., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0805434105 · Cited by 645
Self-citationZhao, Z., Tuakli-Wosornu, Y., Lagace, T. A., Kinch, L. N., Grishin, N. V., Horton, J. D., Cohen, J. C., & Hobbs, H. H. (2006) · The American Journal of Human Genetics · doi:10.1086/507488 · Cited by 642
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Simion, V., Henriet, E., Jurić, V., Aquino, R., Loussouarn, C., Laurent, Y., Martı́n, F., Midoux, P., Garcion, E., Pichon, C., et al. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.08.028 · Cited by 30
The Promise of PCSK9 and Lipoprotein(a) as Targets for Gene Silencing Therapies
Chan, D. C., & Watts, G. F. (2023) · Clinical Therapeutics · doi:10.1016/j.clinthera.2023.07.008 · Cited by 30
Katsiki, N., Filippatos, T. D., Vlachopoulos, C. V., Panagiotakos, D. B., Milionis, H. J., Tselepis, A. D., Garoufi, A., Rallidis, L. S., Richter, D. J., Nomikos, T., et al. (2024) · Atherosclerosis Plus · doi:10.1016/j.athplu.2024.01.004 · Cited by 27
Targeting low‐density lipoprotein cholesterol with PCSK9 inhibitors
Scherer, D. J., Nelson, A. J., Psaltis, P. J., & Nicholls, S. J. (2017) · Internal Medicine Journal · doi:10.1111/imj.13451 · Cited by 27
Ng, B., Cash-Mason, T., Wang, Y., Seitzer, J. L., Burchard, J., Brown, D., Dudkin, V., Davide, J. P., Jadhav, V., Sepp‐Lorenzino, L., et al. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.02.013 · Cited by 27
Wu, C., Xi, C., Tong, J., Zhao, J. Z., Jiang, H., Wang, J., Wang, Y., & Liu, H. (2019) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2019.06.006 · Cited by 27
Garber, K. (2017) · Nature Biotechnology · doi:10.1038/nbt.3810 · Cited by 27
Zhang, L., Lou, W., & Wang, J. (2024) · Clinical and Experimental Medicine · doi:10.1007/s10238-024-01463-4 · Cited by 30
Page, M. M., & Watts, G. F. (2017) · Diabetes Obesity and Metabolism · doi:10.1111/dom.13070 · Cited by 27
Gankyrin: a novel promising therapeutic target for hepatocellular carcinoma
Zamani, P., Riahi, M. M., Momtazi‐Borojeni, A. A., & Jamialahmadi, K. (2017) · Artificial Cells Nanomedicine and Biotechnology · doi:10.1080/21691401.2017.1388250 · Cited by 26
Efficacy, Safety, and Pharmacokinetics of Inclisiran in Japanese Patients: Results from ORION-15
Yamashita, S., Kiyosue, A., Maheux, P., Mena-Madrazo, J., Lesogor, A., Shao, Q., Tamaki, Y., Nakamura, H., Akahori, M., & Kajinami, K. (2024) · Journal of Atherosclerosis and Thrombosis · doi:10.5551/jat.64454 · Cited by 26
Carbon nanotubes exhibit fibrillar pharmacology in primates
Alidori, S., Thorek, D. L. J., Beattie, B. J., Ulmert, D., Almeida, B. A., Monette, S., Scheinberg, D. A., & McDevitt, M. R. (2017) · PLoS ONE · doi:10.1371/journal.pone.0183902 · Cited by 26
A New Approach to PCSK9 Therapeutics
Wang, N., & Tall, A. R. (2017) · Circulation Research · doi:10.1161/circresaha.117.310610 · Cited by 27
Non-coding RNAs – A primer for the laboratory scientist
Waller, P., & Blann, A. D. (2019) · British Journal of Biomedical Science · doi:10.1080/09674845.2019.1675847 · Cited by 25
Dyslipidemia Management in 2020: An Update on Diagnosis and Therapeutic Perspectives
Karantas, I. D., Okur, M. E., Okur, N. Ü., & Siafaka, P. I. (2020) · Endocrine Metabolic & Immune Disorders - Drug Targets · doi:10.2174/1871530320666200810144004 · Cited by 25
Chemical strategies for strand selection in short-interfering RNAs
Varley, A., & Desaulniers, J. (2021) · RSC Advances · doi:10.1039/d0ra07747j · Cited by 26
Managing dyslipidemia in patients with Type 2 diabetes
Tomlinson, B., Patil, N. G., Fok, M., & Lam, C. W. K. (2021) · Expert Opinion on Pharmacotherapy · doi:10.1080/14656566.2021.1912734 · Cited by 25
Gogate, A., Belcourt, J., Shah, M., Wang, A., Frankel, A., Kolmel, H., Chalon, M., Stephen, P., Kolli, A., Tawfik, S. M., et al. (2023) · Pharmacological Reviews · doi:10.1124/pharmrev.123.000815 · Cited by 27
Pendergraff, H. M., Krishnamurthy, P. M., Debacker, A. J., Moazami, M. P., Sharma, V. K., Niitsoo, L., Yu, Y., Tan, Y. N., Haitchi, H. M., & Watts, J. K. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.06.012 · Cited by 25
New Therapeutic Approaches for Familial Hypercholesterolemia
Ajufo, E., & Rader, D. J. (2018) · Annual Review of Medicine · doi:10.1146/annurev-med-051215-030943 · Cited by 26
RNAi therapeutic strategies for acute respiratory distress syndrome
Jagrosse, M. L., Dean, D. A., Rahman, A., & Nilsson, B. L. (2019) · Translational research · doi:10.1016/j.trsl.2019.07.011 · Cited by 25
The Role of RNA-Targeted Therapeutics to Reduce ASCVD Risk: What Have We Learned Recently?
Miname, M. H., Rocha, V. Z., & Santos, R. D. (2021) · Current Atherosclerosis Reports · doi:10.1007/s11883-021-00936-1 · Cited by 25
Ray, R. M., & Morris, K. V. (2020) · Non-Coding RNA · doi:10.3390/ncrna6010012 · Cited by 24
Geiger, M. A., Guillaumon, A. T., Paneni, F., Matter, C. M., & Stein, S. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.569358 · Cited by 24
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Chmielnicka, K., Heleniak, Z., & Dębska-Ślizień, A. M. (2022) · Transplantology · doi:10.3390/transplantology3020020 · Cited by 24
Inclisiran: A New Pharmacological Approach for Hypercholesterolemia
Fusco, S. A. D., Maggioni, A. P., Bernelli, C., Perone, F., Marzo, V. D., Conte, E., Musella, F., Uccello, G., Luca, L. D., Gabrielli, D., et al. (2022) · Reviews in Cardiovascular Medicine · doi:10.31083/j.rcm2311375 · Cited by 24
Vahdat‐Lasemi, F., Mahjoubin‐Tehran, M., Aghaee‐Bakhtiari, S. H., Jalili, A., Jaafari, M. R., & Sahebkar, A. (2019) · Drug Discovery Today · doi:10.1016/j.drudis.2019.04.007 · Cited by 24
Asbeutah, A. A., Asbeutah, S. A., & Abu-Assi, M. A. (2020) · The American Journal of Cardiology · doi:10.1016/j.amjcard.2020.05.024 · Cited by 24
Mikami, A., Erande, N. D., Matsuda, S., Kel’in, A. V., Woods, L. B., Chickering, T., Pallan, P. S., Schlegel, M. K., Zlatev, I., Egli, M., et al. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa750 · Cited by 24
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Jiang, L., Yuan, C., & Huang, M. (2021) · The FASEB Journal · doi:10.1096/fj.202002139rr · Cited by 24
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Cho, K. I., Sakuma, I., Sohn, I. S., Hayashi, T., Shimada, K., & Koh, K. K. (2018) · Circulation Journal · doi:10.1253/circj.cj-17-1445 · Cited by 23
Emerging lipid lowering agents targeting LDL cholesterol
Wójcik, C. (2020) · Postgraduate Medicine · doi:10.1080/00325481.2020.1751422 · Cited by 23
Inhaled siRNA Formulations for Respiratory Diseases: From Basic Research to Clinical Application
Fan, Y., & Yang, Z. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14061193 · Cited by 23
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Kim, K. A., & Park, H. (2023) · Journal of Lipid and Atherosclerosis · doi:10.12997/jla.2023.12.1.37 · Cited by 23
Sokolov, V. V., Helmlinger, G., Nilsson, C. A. M., Zhudenkov, K., Skrtic, S., Hamrén, B., Peskov, K. V., Hurt‐Camejo, E., & Jansson‐Löfmark, R. (2019) · Journal of Lipid Research · doi:10.1194/jlr.m092486 · Cited by 23
Aguilar-Ballester, M., Hurtado-Genovés, G., Taberner-Cortés, A., Herrero-Cervera, A., Martínez‐Hervás, S., & Navarro, H. G. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22020660 · Cited by 25
Alsayed, N., Almahmeed, W. A., Alnouri, F., Al-Waili, K., Sabbour, H. M., Sulaiman, K., Zubaid, M., Ray, K. K., & Al‐Rasadi, K. (2021) · Atherosclerosis · doi:10.1016/j.atherosclerosis.2021.11.022 · Cited by 23
Kim, E. J., & Wierzbicki, A. S. (2020) · Therapeutic Advances in Chronic Disease · doi:10.1177/2040622320924569 · Cited by 22
Review of recent clinical trials and their impact on the treatment of hypercholesterolemia
Parham, J. S., & Goldberg, A. C. (2022) · Progress in Cardiovascular Diseases · doi:10.1016/j.pcad.2022.11.011 · Cited by 22
Sun, C., Zhu, G., Shen, C., Huang, S., Li, R., Li, J., Ma, Z., & Wang, Z. (2023) · Frontiers in Oncology · doi:10.3389/fonc.2023.1134063 · Cited by 22
Dyslipidemias and Cardiovascular Prevention: Tailoring Treatment According to Lipid Phenotype
Sanin, V., Pfetsch, V., & Köenig, W. (2017) · Current Cardiology Reports · doi:10.1007/s11886-017-0869-3 · Cited by 24
O′Shea, J., Theile, C. S., Das, R. S., Babu, I. R., Charissé, K., Manoharan, M., Maier, M. A., & Zlatev, I. (2018) · Tetrahedron · doi:10.1016/j.tet.2018.09.008 · Cited by 22
A Review of PCSK9 Inhibitors and their Effects on Cardiovascular Diseases
Chen, B., Shi, X., Cui, Y., Hou, A., & Zhao, P. (2019) · Current Topics in Medicinal Chemistry · doi:10.2174/1568026619666190809094203 · Cited by 22
Function and Therapeutic Implications of tRNA Derived Small RNAs
Wilson, B., & Dutta, A. (2022) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2022.888424 · Cited by 22
Petroglou, D., Kanellos, I., Savopoulos, C. G., Kaiafa, G. D., Chrysochoou, A., Skantzis, P., Daios, S., Hatzitolios, A. I., & Giannoglou, G. (2018) · Current Medicinal Chemistry · doi:10.2174/0929867325666180604114819 · Cited by 21
Yu, Y., Dong, J., Ma, B., Jiang, X., Guo, C., Liu, Z., Chai, Y., Wang, L., Sun, L., Ou, L., et al. (2021) · Journal of Materials Chemistry B · doi:10.1039/d1tb01220g · Cited by 21
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Cesaro, A., Fimiani, F., Gragnano, F., Moscarella, E., Schiavo, A., Vergara, A., Akioyamen, L. E., D’Erasmo, L., Averna, M. R., Arca, M., et al. (2021) · Heart Failure Clinics · doi:10.1016/j.hfc.2021.07.008 · Cited by 21
Maraganore, J. M. (2022) · Nature Biotechnology · doi:10.1038/s41587-022-01304-3 · Cited by 21
Clinical pharmacology of siRNA therapeutics: current status and future prospects
Abosalha, A. K., Boyajian, J. L., Ahmad, W., Islam, P., Ghebretatios, M., Schaly, S., Thareja, R., Arora, K., & Prakash, S. (2022) · Expert Review of Clinical Pharmacology · doi:10.1080/17512433.2022.2136166 · Cited by 22
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Carugo, S., Sirtori, C. R., Gelpi, G., Corsini, A., Tokgözoğlu, L. S., & Ruscica, M. (2023) · Current Atherosclerosis Reports · doi:10.1007/s11883-023-01156-5 · Cited by 22
Gambari, R., Waziri, A. D., Goonasekera, H. W. W., & Peprah, E. K. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25084263 · Cited by 21
RNA interference efficiently targets human leukemia driven by a fusion oncogene in vivo
Jyotsana, N., Sharma, A. K., Chaturvedi, A., Scherr, M., Kuchenbauer, F. C., Sajti, L., Barchanski, A., Lindner, R. M., Noyan, F., Sühs, K., et al. (2017) · Leukemia · doi:10.1038/leu.2017.269 · Cited by 23
The Role of Metabolite-Sensing G Protein-Coupled Receptors in Inflammation and Metabolic Disease
Recio, C., Lucy, D., Iveson, P., Iqbal, A. J., Valaris, S., Wynne, G. M., Russell, A. J., Choudhury, R. P., O’Callaghan, C. A., Monaco, C. G., et al. (2017) · Antioxidants and Redox Signaling · doi:10.1089/ars.2017.7168 · Cited by 22
Ghosh, G. C., Bandyopadhyay, D., Ghosh, R. K., Mondal, S., & Herzog, E. (2018) · The American Journal of Cardiology · doi:10.1016/j.amjcard.2018.06.023 · Cited by 21
PCSK9 inhibitors and LDL reduction: pharmacology, clinical implications, and future perspectives
Fitzgerald, G., & Kiernan, T. (2018) · Expert Review of Cardiovascular Therapy · doi:10.1080/14779072.2018.1497975 · Cited by 21
Design of Organo-Peptides As Bipartite PCSK9 Antagonists
Burdick, D. J., Skelton, N. J., Ultsch, M. H., Beresini, M. H., Eigenbrot, C. W., Li, W., Zhang, Y., Nguyen, H. Q., Kong-Beltran, M., Quinn, J. G., et al. (2020) · ACS Chemical Biology · doi:10.1021/acschembio.9b00899 · Cited by 21
Busuioc, R., Covic, A. C., Kanbay, M., Banach, M., Burlacu, A., & Mircescu, G. (2019) · Nephrology Dialysis Transplantation · doi:10.1093/ndt/gfz108 · Cited by 20
Murray, J. K., Long, J., Liu, L., Singh, S., Pruitt, D., Ollmann, M. M., Swearingen, E., Hardy, M., Homann, O. R., Wu, B., et al. (2021) · Nucleic Acid Therapeutics · doi:10.1089/nat.2021.0026 · Cited by 20
New Insights into Pathophysiology and New Risk Factors for ACS
Nardin, M., Verdoia, M., Laera, N., Cao, D., & Luca, G. D. (2023) · Journal of Clinical Medicine · doi:10.3390/jcm12082883 · Cited by 21
Akabane‐Nakata, M., Erande, N. D., Kumar, P., Degaonkar, R., Gilbert, J. A., Qin, J., Mendez, M., Woods, L. B., Jiang, Y., Janas, M. M., et al. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkab050 · Cited by 20
Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs
Osborn, M. F., Coles, A. H., Golebiowski, D., Echeverria, D., Moazami, M. P., Watts, J. K., Sena‐Esteves, M., & Khvorova, A. (2018) · Molecular Cancer Therapeutics · doi:10.1158/1535-7163.mct-17-1144 · Cited by 20
Nanomedicine-based strategies for the treatment of vein graft disease
Zhou, Z., Chen, W., Cao, Y., Abdi, R., & Tao, W. (2024) · Nature Reviews Cardiology · doi:10.1038/s41569-024-01094-y · Cited by 19
Goksøyr, L., Skrzypczak, M., Sampson, M. L., Nielsen, M. A., Salanti, A., Theander, T. G., Remaley, A. T., de Jongh, W. A., & Sander, A. F. (2022) · Vaccines · doi:10.3390/vaccines11010002 · Cited by 19
Recent advances in the management and implementation of care for familial hypercholesterolaemia
Lan, N. S. R., Bajaj, A., Watts, G. F., & Cuchel, M. (2023) · Pharmacological Research · doi:10.1016/j.phrs.2023.106857 · Cited by 19
Cheng, S.-Y., Caiazzi, J., Biscans, A., Alterman, J. F., Echeverria, D., McHugh, N., Hassler, M. R., Jolly, S. M., Giguere, D., Cipi, J., et al. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.102088 · Cited by 20
New approaches to address dyslipidemia
Parhofer, K. G. (2017) · Current Opinion in Lipidology · doi:10.1097/mol.0000000000000456 · Cited by 19
Yamada, K., Hildebrand, S. R., Davis, S. M., Miller, R., Conroy, F., Sapp, E., Caiazzi, J., Alterman, J. F., Roux, L., Echeverria, D., et al. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkab1126 · Cited by 19
Non-coding RNA therapeutics in cardiovascular diseases and risk factors: Systematic review
Ardiana, M., Fadila, A. N., Zuhra, Z., Kusuma, N. M., Rurus, M. E. S. E., & Oceandy, D. (2023) · Non-coding RNA Research · doi:10.1016/j.ncrna.2023.06.002 · Cited by 18
Effective low-density lipoprotein-lowering therapy: Implementation in clinical practice
Sinning, D., & Landmesser, U. (2017) · European Journal of Preventive Cardiology · doi:10.1177/2047487317708349 · Cited by 18
Emerging role of proprotein convertase subtilisin/kexin type-9 (PCSK-9) in inflammation and diseases
Brown, M., & Ahmed, S. (2019) · Toxicology and Applied Pharmacology · doi:10.1016/j.taap.2019.03.018 · Cited by 18
Systemic Delivery of Small Interfering RNA Therapeutics: Obstacles and Advances
Chandela, A., & Ueno, Y. (2019) · Reviews in Agricultural Science · doi:10.7831/ras.7.10 · Cited by 19
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Ray, R. M., Hansen, A. H., Slott, S., Taskova, M., Astakhova, K., & Morris, K. V. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.05.024 · Cited by 18
Wong, N. D., Rosenblit, P. D., & Greenfield, R. S. (2017) · Cardiovascular Diagnosis and Therapy · doi:10.21037/cdt.2017.03.02 · Cited by 19
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Stewart, W., Hu, B., Li, F., Huang, J., Liu, Z., Zhang, C., Tang, M., Zhang, X., & Xu, X. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.113857 · Cited by 17
Aguchem, R. N., Okagu, I. U., Okorigwe, E. M., Uzoechina, J. O., Nnemolisa, S. C., & Ezeorba, T. P. C. (2024) · Life Sciences · doi:10.1016/j.lfs.2024.122823 · Cited by 17
Luna, S. E., Camarena, J., Hampton, J. P., Majeti, K. R., Charlesworth, C. T., Soupène, E., Selvaraj, S., Jia, K., Sheehan, V., Cromer, M. K., et al. (2024) · Nature Biomedical Engineering · doi:10.1038/s41551-024-01222-6 · Cited by 17
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Yokoi, H., Kasahara, Y., Obika, S., Doi, T., & Kamada, H. (2018) · Rapid Communications in Mass Spectrometry · doi:10.1002/rcm.8274 · Cited by 17
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Schrör, K., Trenk, D., & Verheugt, F. W. A. (2022) · Thrombosis and Haemostasis · doi:10.1055/s-0042-1758654 · Cited by 17
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Jiang, Y., Wang, Y., Ma, S., Qian, L., Jing, Y., Chen, X., & Yang, J. (2025) · Frontiers in Cardiovascular Medicine · doi:10.3389/fcvm.2024.1415668 · Cited by 16
Cicero, A. F. G., Bove, M., & Borghi, C. (2017) · Expert Opinion on Drug Metabolism & Toxicology · doi:10.1080/17425255.2018.1416094 · Cited by 17
Banda, N. K., Desai, D., Scheinman, R. I., Pihl, R., Sekine, H., Fujita, T., Sharma, V., Hansen, A. G., Garred, P., Thiel, S., et al. (2018) · ImmunoHorizons · doi:10.4049/immunohorizons.1800053 · Cited by 17
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Moore, M. P., Wang, X., Shi, H., Meroni, M., Cherubini, A., Ronzoni, L., Parks, E. J., Ibdah, J. A., Rector, R. S., Valenti, L. V. C., et al. (2023) · JHEP Reports · doi:10.1016/j.jhepr.2023.100716 · Cited by 16
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Pinto, R. D., Grassi, D., Properzi, G., Desideri, G., & Ferri, C. (2019) · High Blood Pressure & Cardiovascular Prevention · doi:10.1007/s40292-019-00323-7 · Cited by 16
Wang, Y. R., Guo, Y., Guo, S., Qi, L., Li, B., Jiang, L., Xu, C., An, M., & Wu, Y. (2024) · Pest Management Science · doi:10.1002/ps.7962 · Cited by 15
Metadata
- Authors
- Fitzgerald, K., White, S., Borodovsky, A., Bettencourt, B. R., Strahs, A., Clausen, V., Wijngaard, P., Horton, J. D., Taubel, J., Brooks, A., Fernando, C., Kauffman, R. S., Kallend, D., Vaishnaw, A., & Simon, A.
- Affiliations
- Alnylam Pharmaceuticals (United States) (US)
- St George's, University of London (GB)
- ORCID
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2017-01-05
- Volume
- 376
- Issue
- 1
- Pages
- 41-51
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- PMC
- PMC5778873
- OpenAlex
- W2555483953
- Cited by
- 768
- References
- 29
- Citations by year
- 2026: 43 · 2025: 61 · 2024: 84 · 2023: 89 · 2022: 85 · 2021: 68 · 2020: 88 · 2019: 76 · 2018: 95 · 2017: 74
- FWCI
- 91.52
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- New England Journal of Medicine (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Heart, Lung, and Blood Institute (P01HL020948)
- Topics
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Lipoproteins and Cardiovascular Health
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Biochemistry › Cholesterol and Lipid Metabolism
- Keywords
- inclisiran, PCSK9 inhibition, RNA interference therapy, LDL cholesterol lowering, hypercholesterolemia, statin therapy, pharmacokinetics, pharmacodynamics, safety, multiple-dose regimen, single ascending dose, healthy volunteers, lipid-lowering drugs, hypertriglyceridemia, hyperuricemia
- MeSH terms
- RNAi Therapeutics, Proprotein Convertase 9, PCSK9 Inhibitors, Acetylgalactosamine, Adolescent, Adult, Aged, Anticholesteremic Agents, Female, Humans, Injections, Subcutaneous, Cholesterol, LDL, Male, Middle Aged, Single-Blind Method, RNA, Small Interfering, RNA-Induced Silencing Complex, Young Adult, Healthy Volunteers
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