| OPPOSITION | - Anonymous, "Assessment report - Onpattro - International non-proprietary name: patisiran", European Medicines Agency (EMA); EMA/554262/2018; Committee for Medicinal Products for Human Use (CHMP), (20180726), European Medicines Agency (EMA); EMA/554262/2018; Committee for Medicinal Products for Human Use (CHMP), URL: https://www.ema.europa.eu/en/documents/assessment-report/onpattro-epar-public-assessment-report_en.pdf, XP093269621 | - |
| OPPOSITION | - D2a - Belliveau et al., Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA. Molecular Therapy, Nucleic Acids, 2012, with supplementary figures S5 and S7 | - |
| OPPOSITION | - D34 - Dynamic Light Scattering: An Introduction in 30 Minutes; filed by proprietor during examination (cannot find date) | - |
| OPPOSITION | - D35 - Standard Operating Procedure for dynamic light scattering; filed by proprietor during examination (cannot find date) | - |
| OPPOSITION | - D44 - Technical Report, 2025 | - |
| OPPOSITION | - D8a - Supplementary methods of D8 | - |
| OPPOSITION | - D8b - Supplementary figures of D8 | - |
| OPPOSITION | - D9 - Zetasizer | - |
| OPPOSITION | - "ISO 22412:2017 Particle size analysis -- Dynamic light scattering (DLS)", INTERNATIONAL STANDARD - ISO, ZUERICH, CH, CH , (20170201), vol. ISO 22412:2017, pages 1 - 38, XP008183842 | - |
| OPPOSITION | - MacLachlan I., "Liposomal formulations for nucleic acid delivery", MacLachlan I., CROOKE STANLEY T, ANTISENSE DRUG TECHNOLOGY: PRINCIPLES, STRATEGIES, AND APPLICATIONS, 2ND ED., US , CRC PRESS , (20070101), pages 237 - 270, ISBN 978-0-8493-8796-8, XP002584305 | - |
| OPPOSITION | - Muthusamy Jayaraman, Steven M. Ansell, Barbara L. Mui, Ying K. Tam, Jianxin Chen, Xinyao Du, David Butler, Laxman Eltepu, Shigeo Matsuda, Jayaprakash K. Narayanannair, Kallanthottathil G. Rajeev, Ismail M. Hafez, Akin Akinc, Martin A. Maier, Mark A. Tracy, Pieter R. Cullis, Thomas D. Madden, Muthiah Manoharan, Michael J. Hope, "Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo", Angewandte Chemie International Edition, ¬Verlag Chemie| :, (20120820), vol. 51, no. 34, doi:10.1002/anie.201203263, ISSN 14337851, pages 8529 - 8533, XP055063645 | |
| OPPOSITION | - Evers Martijn J. W., Kulkarni Jayesh A., Van Der Meel Roy, Cullis Pieter R., Vader Pieter, Schiffelers Raymond M., "State-of-the-Art Design and Rapid-Mixing Production Techniques of Lipid Nanoparticles for Nucleic Acid Delivery", Small Methods, Wiley - V C H Verlag GmbH & Co. KGaA, DE, DE , (20180901), vol. 2, no. 9, doi:10.1002/smtd.201700375, ISSN 2366-9608, page 1700375, XP055924365 | |
| OPPOSITION | - Jyotsana Nidhi; Sharma Amit; Chaturvedi Anuhar; Budida Ramachandramouli; Scherr Michaela; Kuchenbauer Florian; Lindner Robert; Noyan Fatih; Sühs Kurt-Wolfram; Stangel Martin; Grote-Koska Denis; Brand Korbinian; Vornlocher Hans-Peter; Eder Matthias; Thol Felicitas; Ganser Arnold; Humphries R. Keith; Ramsay Euan; Cullis Pieter; Heuser Michael, "Lipid nanoparticle-mediated siRNA delivery for safe targeting of human CML in vivo", ANNALS OF HEMATOLOGY, BERLIN, DE, DE , (20190518), vol. 98, no. 8, doi:10.1007/s00277-019-03713-y, ISSN 0939-5555, pages 1905 - 1918, XP036833260 | |
| OPPOSITION | - Cauvin Annick J., Peters Christopher, Brennan Frank, "Advantages and Limitations of Commonly Used Nonhuman Primate Species in Research and Development of Biopharmaceuticals", The Nonhuman Primate in Nonclinical Drug Development and Safety Assessment, Elsevier, (20150101), pages 379 - 395, doi:10.1016/B978-0-12-417144-2.00019-6, ISBN 978-0-12-417144-2, XP093269642 | |
| OPPOSITION | - Scott A. Barros, Gollob Jared A., "Safety profile of RNAi nanomedicines", Advanced Drug Delivery Reviews, Elsevier, (20121201), vol. 64, no. 15, doi:10.1016/j.addr.2012.06.007, ISSN 0169409X, pages 1730 - 1737, XP055078049 | |
| OPPOSITION | - Cheng Xinwei; Lee Robert J., "The role of helper lipids in lipid nanoparticles (LNPs) designed for oligonucleotide delivery", Advanced Drug Delivery Reviews, Elsevier, Amsterdam , NL, Amsterdam , NL , (20160218), vol. 99, doi:10.1016/j.addr.2016.01.022, ISSN 0169-409X, pages 129 - 137, XP029445785 | |
| OPPOSITION | - Witzigmann Dominik; Kulkarni Jayesh A.; Leung Jerry; Chen Sam; Cullis Pieter R.; van der Meel Roy, "Lipid nanoparticle technology for therapeutic gene regulation in the liver", Advanced Drug Delivery Reviews, Elsevier, Amsterdam , NL, Amsterdam , NL , (20200101), vol. 159, doi:10.1016/j.addr.2020.06.026, ISSN 0169-409X, pages 344 - 363, XP086398235 | |
| OPPOSITION | - Chen Sam; Tam Yuen Yi C.; Lin Paulo J.C.; Leung Alex K.K.; Tam Ying K.; Cullis Pieter R., "Development of lipid nanoparticle formulations of siRNA for hepatocyte gene silencing following subcutaneous administration", Journal of Controlled Release, Elsevier, NL, NL , (20141005), vol. 196, doi:10.1016/j.jconrel.2014.09.025, ISSN 0168-3659, pages 106 - 112, XP029112393 | |
| OPPOSITION | - Chen Sam; Tam Yuen Yi C.; Lin Paulo J.C.; Sung Molly M.H.; Tam Ying K.; Cullis Pieter R., "Influence of particle size on thein vivopotency of lipid nanoparticle formulations of siRNA", Journal of Controlled Release, Elsevier, NL, NL , (20160526), vol. 235, doi:10.1016/j.jconrel.2016.05.059, ISSN 0168-3659, pages 236 - 244, XP029633339 | |
| OPPOSITION | - Hassett Kimberly J.; Higgins Jaclyn; Woods Angela; Levy Becca; Xia Yan; Hsiao Chiaowen Joyce; Acosta Edward; Almarsson Örn; Moore Melissa J.; Brito Luis A., "Impact of lipid nanoparticle size on mRNA vaccine immunogenicity", Journal of Controlled Release, Elsevier, NL, NL , (20210518), vol. 335, doi:10.1016/j.jconrel.2021.05.021, ISSN 0168-3659, pages 237 - 246, XP086685076 | |
| OPPOSITION | - Hassett Kimberly J., Benenato Kerry E., Jacquinet Eric, Lee Aisha, Woods Angela, Yuzhakov Olga, Himansu Sunny, Deterling Jessica, Geilich Benjamin M., Ketova Tatiana, Mihai Cosmin, Lynn Andy, Mcfadyen Iain, Moore Melissa J., Senn Joseph J., Stanton Matthew G., Almarsson Örn, Ciaramella Giuseppe, Brito Luis A., "Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines", Molecular Therapy-Nucleic Acids, Cell Press, US, US , (20190415), vol. 15, doi:10.1016/j.omtn.2019.01.013, ISSN 2162-2531, pages 1 - 11, XP055815007 | |
| OPPOSITION | - Pieter R. Cullis, Michael J. Hope, "Lipid Nanoparticle Systems for Enabling Gene Therapies", Molecular Therapy, Elsevier Inc, United States, United States, (20170701), vol. 25, no. 7, doi:10.1016/j.ymthe.2017.03.013, ISSN 1525-0016, pages 1467 - 1475, XP055540439 | |
| OPPOSITION | - Scherphof Gerrit L., Velinova Maria, Kamps Jan, Donga Jan, Van Der Want Han, Kuipers Folkert, Havekes Louis, Daemen Toos, "Modulation of pharmacokinetic behavior of liposomes", Advanced Drug Delivery Reviews, Elsevier, Amsterdam , NL, Amsterdam , NL , (19970301), vol. 24, no. 2-3, doi:10.1016/S0169-409X(96)00457-7, ISSN 0169-409X, pages 179 - 191, XP093269644 | |
| OPPOSITION | - Kulkarni Jayesh A., Witzigmann Dominik, Chen Sam, Cullis Pieter R., Van Der Meel Roy, "Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics", Accounts of Chemical Research, ACS , Washington , DC, US, US , (20190917), vol. 52, no. 9, doi:10.1021/acs.accounts.9b00368, ISSN 0001-4842, pages 2435 - 2444, XP055878486 | |
| OPPOSITION | - E Wisse, Jacobs F, Topal B, Frederik P, De Geest B, "The size of endothelial fenestrae in human liver sinusoids: implications for hepatocyte-directed gene transfer", Gene Therapy, Nature Publishing Group, London, GB, GB , (20080410), vol. 15, no. 17, doi:10.1038/gt.2008.60, ISSN 0969-7128, pages 1193 - 1199, XP055534609 | |
| OPPOSITION | - Akinc A., ET AL, "Development of Lipidoid-siRNA Formulations for Systemic Delivery to the Liver", Molecular Therapy, Elsevier Inc, United States, United States, (20090501), vol. 17, no. 5, doi:10.1038/MT.2009.36, ISSN 1525-0016, pages 872 - 879, XP008105983 | |
| OPPOSITION | - Akin Akinc, William Querbes, Soma De, June Qin, Maria Frank-Kamenetsky, K. Narayanannair Jayaprakash, Muthusamy Jayaraman, Kallanthottathil G Rajeev, William L Cantley, J Robert Dorkin, James S Butler, Liuliang Qin, Timothy Racie, Andrew Sprague, Eugenio Fava, Anja Zeigerer, Michael J Hope, Marino Zerial, Dinah Wy Sah, Kevin Fitzgerald, Mark A Tracy, Muthiah Manoharan, Victor Koteliansky, Antonin De Fougerolles, Martin A Maier, "Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms", Molecular Therapy, No longer published by Elsevier, (20100701), vol. 18, no. 7, doi:10.1038/mt.2010.85, ISSN 15250016, pages 1357 - 1364, XP055016290 | |
| OPPOSITION | - Genc Basha, Tatiana I Novobrantseva, Nicole Rosin, Yuen Yi C Tam, Ismail M Hafez, Matthew K Wong, Tsukasa Sugo, Vera M Ruda, June Qin, Boris Klebanov, Marco Ciufolini, Akin Akinc, Ying K Tam, Michael J Hope, Pieter R Cullis, "Influence of Cationic Lipid Composition on Gene Silencing Properties of Lipid Nanoparticle Formulations of siRNA in Antigen-Presenting Cells", Molecular Therapy, No longer published by Elsevier, (20111201), vol. 19, no. 12, doi:10.1038/mt.2011.190, ISSN 15250016, pages 2186 - 2200, XP055024764 | |
| OPPOSITION | - Martin A Maier, Muthusamy Jayaraman, Shigeo Matsuda, Ju Liu, Scott Barros, William Querbes, Ying K Tam, Steven M Ansell, Varun Kumar, June Qin, Xuemei Zhang, Qianfan Wang, Sue Panesar, Renta Hutabarat, Mary Carioto, Julia Hettinger, Pachamuthu Kandasamy, David Butler, Kallanthottathil G Rajeev, Bo Pang, Klaus Charisse, Kevin Fitzgerald, Barbara L Mui, Xinyao Du, Pieter Cullis, Thomas D Madden, Michael J Hope, Muthiah Manoharan, Akin Akinc, "Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics", Molecular Therapy, Elsevier Inc, United States, United States, (20130801), vol. 21, no. 8, doi:10.1038/mt.2013.124, ISSN 1525-0016, pages 1570 - 1578, XP055237159 | |
| OPPOSITION | - Andreas Thess, Stefanie Grund, Barbara L Mui, Michael J Hope, Patrick Baumhof, Mariola Fotin-Mleczek, Thomas Schlake, "Sequence-engineered mRNA Without Chemical Nucleoside Modifications Enables an Effective Protein Therapy in Large Animals", Molecular Therapy, Elsevier Inc, United States, United States, (20150901), vol. 23, no. 9, doi:10.1038/mt.2015.103, ISSN 1525-0016, pages 1456 - 1464, XP055316897 | |
| OPPOSITION | - Nathan M Belliveau, Jens Huft, Paulo J C Lin, Sam Chen, Alex K K Leung, Timothy J Leaver, Andre W Wild, Justin B Lee, Robert J Taylor, Ying K Tam, Carl L Hansen, and Pieter R Cullis, "Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA.", NUCLEIC ACIDS AND MOLECULAR BIOLOGY., SPRINGER VERLAG, BERLIN., DE, DE , (20120801), vol. 1, no. 8, doi:10.1038/mtna.2012.28, ISSN 0933-1891, pages e37 - e37-9, XP002715253 | |
| OPPOSITION | - Rungta Ravi L, Choi Hyun B, Lin Paulo Jc, Ko Rebecca Wy, Ashby Donovan, Nair Jay, Manoharan Muthiah, Cullis Pieter R, Macvicar Brian A, "Lipid Nanoparticle Delivery of siRNA to Silence Neuronal Gene Expression in the Brain", Molecular Therapy-Nucleic Acids, Cell Press, US, US , vol. 2, no. 11, doi:10.1038/mtna.2013.65, ISSN 2162-2531, page e136, XP093269635 | |
| OPPOSITION | - Tracy S. Zimmermann, Amy C. H. Lee, Akin Akinc, Birgit Bramlage, David Bumcrot, Matthew N. Fedoruk, Jens Harborth, James A. Heyes, Lloyd B. Jeffs, Matthias John, Adam D. Judge, Kieu Lam, Kevin McClintock, Lubomir V. Nechev, Lorne R. Palmer, Timothy Racie, Ingo Röhl, Stephan Seiffert, Sumi Shanmugam, Vandana Sood, Jürgen Soutschek, Ivanka Toudjarska, Amanda J. Wheat, Ed Yaworski, Ian MacLachlan, "RNAi-mediated gene silencing in non-human primates; Including: Supplementary information: Supplementary Methods & Supplementary Figures and Table", NATURE, Springer Nature Publishing AG, LONDON, LONDON , (20060504), vol. 441, no. 7089, doi:10.1038/nature04688, ISSN 1476-4687, pages 111 - 114, 1-7, 1-7, XP002412249 | |
| OPPOSITION | - AKINC AKIN ET AL, "A combinatorial library of lipid-like materials for delivery of RNAi therapeutics.", Nature Biotechnology, Nature Publishing Group US, New York, New York, (20080501), vol. 26, no. 5, doi:10.1038/nbt1402, ISSN 1087-0156, pages 561 - 569, XP002527175 | |
| OPPOSITION | - Jerome Gilleron, William Querbes, Anja Zeigerer, Anna Borodovsky, Giovanni Marsico, Undine Schubert, Kevin Manygoats, Sarah Seifert, Cordula Andree, Martin Stöter, Hila Epstein-Barash, Ligang Zhang, Victor Koteliansky, Kevin Fitzgerald, Eugenio Fava, Marc Bickle, Yannis Kalaidzidis, Akin Akinc, Martin Maier, Marino Zerial, "Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape", Nature Biotechnology, Nature Publishing Group US, New York, New York, (20130701), vol. 31, no. 7, doi:10.1038/nbt.2612, ISSN 1087-0156, pages 638 - 646, XP055450624 | |
| OPPOSITION | - Snoeys J; Lievens J; Wisse E; Jacobs F; Duimel H; Collen D; Frederik P; De Geest B, "Species differences in transgene DNA uptake in hepatocytes after adenoviral transfer correlate with the size of endothelial fenestrae", Gene Therapy, Nature Publishing Group, London, GB, GB , (20070118), vol. 14, no. 7, doi:10.1038/sj.gt.3302899, ISSN 0969-7128, pages 604 - 612, XP037773572 | |
| OPPOSITION | - Kulkarni Jayesh A., "On the role of helper lipids in lipid nanoparticle formulations of si RNA", Nanoscale, doi:10.1039/c9nr09347h, (20191112), pages 21733 - 21739, Nanoscale, URL: https://www.liposomes.ca/publications/2010s/Kulkarni%20et%20al%202019%20-%20On%20the%20role%20of%20helper%20lipids%20in%20lipid%20nanoparticle%20formulations%20of%20siRNA.pdf, (20230707), XP093062170 | |
| OPPOSITION | - FRANK-KAMENETSKY MARIA ET AL, "Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates", Proceedings of the National Academy of Sciences (PNAS), National Academy of Sciences, (20080819), vol. 105, no. 33, doi:10.1073/pnas.0805434105, ISSN 0027-8424, pages 11915 - 11920, XP002534546 | |
| OPPOSITION | - Dong Y, Love K T, Dorkin J R, Sirirungruang S, Zhang Y, Chen D, Bogorad R L, Yin H, Chen Y, Vegas A J, Alabi C A, Sahay G, Olejnik K T, Wang W, Schroeder A, Lytton-Jean A K, Siegwart D J, Akinc A, Barnes C, Barros S A, Carioto M, Fitzgerald K, Hettinger J, Kumar V, Novobrantseva T I, Qin J, Querbes W, Koteliansky V, Langer R, Anderson D G, "Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates.", Proceedings of the National Academy of Sciences (PNAS), National Academy of Sciences, (20140318), vol. 111, no. 11, doi:10.1073/pnas.1322937111, ISSN 0027-8424, pages 3955 - 3960, XP002758392 | |
| OPPOSITION | - Yanez Arteta Marianna, Kjellman Tomas, Bartesaghi Stefano, Wallin Simonetta, Wu Xiaoqiu, Kvist Alexander J., Dabkowska Aleksandra, Székely Noémi, Radulescu Aurel, Bergenholtz Johan, Lindfors Lennart, "Successful reprogramming of cellular protein production through mRNA delivered by functionalized lipid nanoparticles", Proceedings of the National Academy of Sciences (PNAS), National Academy of Sciences, (20180410), vol. 115, no. 15, doi:10.1073/pnas.1720542115, ISSN 0027-8424, pages E3351 - E3360, XP055782146 | |
| OPPOSITION | - P Maguire, C. M., Rosslein, M., Wick, P. & Prina-Mello, A, "Characterisation of particles in solution - a perspective on light scattering and comparative technologies", Science and Technology of Advanced Materials, Elsevier Science, (20180101), vol. 19, no. 1, doi:10.1080/14686996.2018.1517587, ISSN 1468-6996, pages 732 - 745, XP009537405 | |
| OPPOSITION | - Jayesh A. Kulkarni, Pieter R. Cullis, Roy Van Der Meel, "Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility", Nucleic Acid Therapeutics, Mary Ann Liebert, Inc. Publishers, US, US , (20180601), vol. 28, no. 3, doi:10.1089/nat.2018.0721, ISSN 2159-3337, pages 146 - 157, XP055551908 | |
| OPPOSITION | - Harding John D, "Nonhuman Primates and Translational Research: Progress, Opportunities, and Challenges", ILAR JOURNAL, INSTITUTE FOR LABORATORY ANIMAL RESEARCH, US, US , (20171201), vol. 58, no. 2, doi:10.1093/ilar/ilx033, ISSN 1084-2020, pages 141 - 150, XP093269639 | |
| OPPOSITION | - Braet Filip; Wisse Eddie, "Structural and functional aspects of liver sinusoidal endothelial cell fenestrae: a review", COMPARATIVE HEPATOLOGY, BIOMED CENTRAL LTD.,, GB, GB , (20020823), vol. 1, no. 1, doi:10.1186/1476-5926-1-1, ISSN 1476-5926, page 1, XP021008374 | |
| OPPOSITION | - DAVID G. Le COUTEUR; VICTORIA C. COGGER; ROBERT S. McCUSKEY; RAFAEL De CABO; BÂRD SMEDSRØD; KAREN K. SORENSEN; ALESSANDRA WARREN; ROBIN FRASER, "Age‐Related Changes in the Liver Sinusoidal Endothelium", Annals of the New York Academy of Sciences, New York Academy of Sciences., US, US , (20071101), vol. 1114, no. 1, doi:10.1196/annals.1396.003, ISSN 0077-8923, pages 79 - 87, XP071403545 | |
| OPPOSITION | - Jacobs Frank, Wisse Eddie, De Geest Bart, "The Role of Liver Sinusoidal Cells in Hepatocyte-Directed Gene Transfer", The American Journal of Pathology, Elsevier Inc., US, US , (20100101), vol. 176, no. 1, doi:10.2353/ajpath.2010.090136, ISSN 0002-9440, pages 14 - 21, XP093269646 | |
| OPPOSITION | - Yuen Tam, Chen Sam, Cullis Pieter, "Advances in Lipid Nanoparticles for siRNA Delivery", Pharmaceutics, (20130901), vol. 5, no. 3, doi:10.3390/pharmaceutics5030498, pages 498 - 507, XP055246895 | |
| OPPOSITION | - Hermosilla Jesús, Alonso-García Airan, Salmerón-García Antonio, Cabeza-Barrera José, Medina-Castillo Antonio L., Pérez-Robles Raquel, Navas Natalia, "Analysing the In-Use Stability of mRNA-LNP COVID-19 Vaccines Comirnaty™ (Pfizer) and Spikevax™ (Moderna): A Comparative Study of the Particulate", VACCINES, M D P I AG, CH, CH , vol. 11, no. 11, doi:10.3390/vaccines11111635, ISSN 2076-393X, page 1635, XP093269625 | |
| OPPOSITION | - Anroopb Nair, Shery Jacob, "A simple practice guide for dose conversion between animals and human", Journal of Basic and Clinical Pharmacy, India, India , (20160501), vol. 7, no. 2, doi:10.4103/0976-0105.177703, ISSN 0976-0105, page 27, XP055407475 | |
| OPPOSITION | - Andreas M Reichmuth, Matthias A Oberli, Ana Jaklenec, Robert Langer, Daniel Blankschtein, "mRNA vaccine delivery using lipid nanoparticles", Therapeutic Delivery, Future Science Ltd., GB, GB , (20160501), vol. 7, no. 5, doi:10.4155/tde-2016-0006, ISSN 2041-5990, pages 319 - 334, XP055401839 | |
