The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

被引：1195

作者：

Akinc, Akin ^{[1
]}

Maier, Martin A. ^{[1
]}

Manoharan, Muthiah ^{[1
]}

Fitzgerald, Kevin ^{[1
]}

Jayaraman, Muthusamy ^{[1
]}

Barros, Scott ^{[1
]}

Ansell, Steven ^{[2
]}

Du, Xinyao ^{[2
]}

Hope, Michael J. ^{[2
]}

Madden, Thomas D. ^{[2
]}

Mui, Barbara L. ^{[2
]}

Semple, Sean C. ^{[2
]}

Tam, Ying K. ^{[2
]}

Ciufolini, Marco ^{[3
]}

Witzigmann, Dominik ^{[3
]}

Kulkarni, Jayesh A. ^{[3
]}

van der Meel, Roy ^{[3
]}

Cullis, Pieter R. ^{[3
,4
]}

机构：

[1] Alnylam Pharmaceut, Cambridge, MA USA

[2] Acuitas Therapeut, Vancouver, BC, Canada

[3] Univ British Columbia, Vancouver, BC, Canada

[4] Univ British Columbia, NanoMed Innovat Network, Vancouver, BC, Canada

来源：

NATURE NANOTECHNOLOGY | 2019年 / 14卷 / 12期

关键词：

LIPID NANOPARTICLES; MESSENGER-RNA; CATIONIC LIPIDS; DELIVERY; THERAPY;

D O I：

10.1038/s41565-019-0591-y

中图分类号：

TB3 [工程材料学];

学科分类号：

082905 [生物质能源与材料];

摘要：

The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary transthyretin amyloidosis, paves the way for clinical development of many nucleic acid-based therapies enabled by nanoparticle delivery.

引用

页码：1084 / 1087

页数：4

共 22 条

[1]

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis [J].

Adams, D. ;

Gonzalez-Duarte, A. ;

O'Riordan, W. D. ;

Yang, C. -C. ;

Ueda, M. ;

Kristen, A. V. ;

Tournev, I. ;

Schmidt, H. H. ;

Coelho, T. ;

Berk, J. L. ;

Lin, K. -P. ;

Vita, G. ;

Attarian, S. ;

Plante-Bordeneuve, V. ;

Mezei, M. M. ;

Campistol, J. M. ;

Buades, J. ;

Brannagan, T. H., III ;

Kim, B. J. ;

Oh, J. ;

Parman, Y. ;

Sekijima, Y. ;

Hawkins, P. N. ;

Solomon, S. D. ;

Polydefkis, M. ;

Dyck, P. J. ;

Gandhi, P. J. ;

Goyal, S. ;

Chen, J. ;

Strahs, A. L. ;

Nochur, S. V. ;

Sweetser, M. T. ;

Garg, P. P. ;

Vaishnaw, A. K. ;

Gollob, J. A. ;

Suhr, O. B. .

NEW ENGLAND JOURNAL OF MEDICINE, 2018, 379 (01) :11-21

[2]

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms [J].

Akinc, Akin ;

Querbes, William ;

De, Soma ;

Qin, June ;

Frank-Kamenetsky, Maria ;

Jayaprakash, K. Narayanannair ;

Jayaraman, Muthusamy ;

Rajeev, Kallanthottathil G. ;

Cantley, William L. ;

Dorkin, J. Robert ;

Butler, James S. ;

Qin, LiuLiang ;

Racie, Timothy ;

Sprague, Andrew ;

Fava, Eugenio ;

Zeigerer, Anja ;

Hope, Michael J. ;

Zerial, Marino ;

Sah, Dinah W. Y. ;

Fitzgerald, Kevin ;

Tracy, Mark A. ;

Manoharan, Muthiah ;

Koteliansky, Victor ;

de Fougerolles, Antonin ;

Maier, Martin A. .

MOLECULAR THERAPY, 2010, 18 (07) :1357-1364

[3]

Liposomal drug delivery systems: From concept to clinical applications [J].

Allen, Theresa M. ;

Cullis, Pieter R. .

ADVANCED DRUG DELIVERY REVIEWS, 2013, 65 (01) :36-48

[4]

Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA [J].

Belliveau, Nathan M. ;

Huft, Jens ;

Lin, Paulo J. C. ;

Chen, Sam ;

Leung, Alex K. K. ;

Leaver, Timothy J. ;

Wild, Andre W. ;

Lee, Justin B. ;

Taylor, Robert J. ;

Tam, Ying K. ;

Hansen, Carl L. ;

Cullis, Pieter R. .

MOLECULAR THERAPY-NUCLEIC ACIDS, 2012, 1 :e37

[5]

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis [J].

Coelho, Teresa ;

Adams, David ;

Silva, Ana ;

Lozeron, Pierre ;

Hawkins, Philip N. ;

Mant, Timothy ;

Perez, Javier ;

Chiesa, Joseph ;

Warrington, Steve ;

Tranter, Elizabeth ;

Munisamy, Malathy ;

Falzone, Rick ;

Harrop, Jamie ;

Cehelsky, Jeffrey ;

Bettencourt, Brian R. ;

Geissler, Mary ;

Butler, James S. ;

Sehgal, Alfica ;

Meyers, Rachel E. ;

Chen, Qingmin ;

Borland, Todd ;

Hutabarat, Renta M. ;

Clausen, Valerie A. ;

Alvarez, Rene ;

Fitzgerald, Kevin ;

Gamba-Vitalo, Christina ;

Nochur, Saraswathy V. ;

Vaishnaw, Akshay K. ;

Sah, Dinah W. Y. ;

Gollob, Jared A. ;

Suhr, Ole B. .

NEW ENGLAND JOURNAL OF MEDICINE, 2013, 369 (09) :819-829

[6]

Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets [J].

Conway, Anthony ;

Mendel, Matthew ;

Kim, Kenneth ;

McGovern, Kyle ;

Boyko, Alisa ;

Zhang, Lei ;

Miller, Jeffrey C. ;

DeKelver, Russell C. ;

Paschon, David E. ;

Mui, Barbara L. ;

Lin, Paulo J. C. ;

Tam, Ying K. ;

Barbosa, Chris ;

Redelmeier, Tom ;

Holmes, Michael C. ;

Lee, Gary .

MOLECULAR THERAPY, 2019, 27 (04) :866-877

[7]

CULLIS P R, 1989, Advanced Drug Delivery Reviews, V3, P267, DOI 10.1016/0169-409X(89)90024-0

[8]

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing [J].

Finn, Jonathan D. ;

Smith, Amy Rhoden ;

Patel, Mihir C. ;

Shaw, Lucinda ;

Youniss, Madeleine R. ;

van Heteren, Jane ;

Dirstine, Tanner ;

Ciullo, Corey ;

Lescarbeau, Reynald ;

Seitzer, Jessica ;

Shah, Ruchi R. ;

Shah, Aalok ;

Ling, Dandan ;

Growe, Jacqueline ;

Pink, Melissa ;

Rohde, Ellen ;

Wood, Kristy M. ;

Salomon, William E. ;

Harrington, William F. ;

Dombrowski, Christian ;

Strapps, Walter R. ;

Chang, Yong ;

Morrissey, David V. .

CELL REPORTS, 2018, 22 (09) :2227-2235

[9]

On the mechanism whereby cationic lipids promote intracellular delivery of polynucleic acids [J].

Hafez, IM ;

Maurer, N ;

Cullis, PR .

GENE THERAPY, 2001, 8 (15) :1188-1196

[10]

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo [J].

Jayaraman, Muthusamy ;

Ansell, Steven M. ;

Mui, Barbara L. ;

Tam, Ying K. ;

Chen, Jianxin ;

Du, Xinyao ;

Butler, David ;

Eltepu, Laxman ;

Matsuda, Shigeo ;

Narayanannair, Jayaprakash K. ;

Rajeev, Kallanthottathil G. ;

Hafez, Ismail M. ;

Akinc, Akin ;

Maier, Martin A. ;

Tracy, Mark A. ;

Cullis, Pieter R. ;

Madden, Thomas D. ;

Manoharan, Muthiah ;

Hope, Michael J. .

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION, 2012, 51 (34) :8529-8533

← 1 2 3 →