JP2009513151A5 - - Google Patents

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JP2009513151A5
JP2009513151A5 JP2008538235A JP2008538235A JP2009513151A5 JP 2009513151 A5 JP2009513151 A5 JP 2009513151A5 JP 2008538235 A JP2008538235 A JP 2008538235A JP 2008538235 A JP2008538235 A JP 2008538235A JP 2009513151 A5 JP2009513151 A5 JP 2009513151A5
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nucleotides
modified
sirna
nucleic acid
modified sirna
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JP2008538235A
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JP5336853B2 (ja
JP2009513151A (ja
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Priority claimed from PCT/CA2006/001801 external-priority patent/WO2007051303A1/en
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Publication of JP2009513151A5 publication Critical patent/JP2009513151A5/ja
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JP2008538235A 2005-11-02 2006-11-02 修飾ｓｉＲＮＡ分子およびその使用法 Expired - Fee Related JP5336853B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US73296405P 2005-11-02 2005-11-02
US60/732,964 2005-11-02
US81793306P 2006-06-30 2006-06-30
US60/817,933 2006-06-30
PCT/CA2006/001801 WO2007051303A1 (en) 2005-11-02 2006-11-02 MODIFIED siRNA MOLECULES AND USES THEREOF

Publications (3)

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JP2009513151A JP2009513151A (ja) 2009-04-02
JP2009513151A5 true JP2009513151A5 (https=) 2009-12-17
JP5336853B2 JP5336853B2 (ja) 2013-11-06

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JP2008538235A Expired - Fee Related JP5336853B2 (ja) 2005-11-02 2006-11-02 修飾ｓｉＲＮＡ分子およびその使用法

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US (4) US8101741B2 (https=)
EP (2) EP1948674A4 (https=)
JP (1) JP5336853B2 (https=)
CN (1) CN101346393B (https=)
AU (1) AU2006308765B2 (https=)
CA (1) CA2628300C (https=)
IL (1) IL191192A (https=)
WO (1) WO2007051303A1 (https=)

Families Citing this family (337)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003260370B2 (en) 2002-08-05 2008-05-22 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
AU2005222902B2 (en) 2004-03-12 2010-06-10 Alnylam Pharmaceuticals, Inc. iRNA agents targeting VEGF
CA2587411A1 (en) * 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
US20070054873A1 (en) * 2005-08-26 2007-03-08 Protiva Biotherapeutics, Inc. Glucocorticoid modulation of nucleic acid-mediated immune stimulation
US20120164730A1 (en) * 2005-10-11 2012-06-28 Ben-Gurion University Of The Negev Research And Development Authority Ltd. Compositions for silencing the expression of vdac1 and uses thereof
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2007056861A1 (en) * 2005-11-18 2007-05-24 Protiva Biotherapeutics, Inc. Sirna silencing of influenza virus gene expression
ES2874149T3 (es) 2006-05-11 2021-11-04 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen PCSK9
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
AU2007280690C1 (en) 2006-07-31 2012-08-23 Curevac Gmbh Nucleic acid of formula (I): GIXmGn, or (II): CIXmCn, in particular as an immune-stimulating agent/adjuvant
EP3342415B1 (en) 2006-08-08 2022-01-26 Rheinische Friedrich-Wilhelms-Universität Bonn Structure and use of 5' phosphate oligonucleotides
WO2008019486A1 (en) * 2006-08-16 2008-02-21 Protiva Biotherapeutics, Inc. Nucleic acid modulation of toll-like receptor-mediated immune stimulation
US9371348B2 (en) * 2006-11-27 2016-06-21 The Trustees Of The University Of Pennsylvania Photocleavable oligonucleotide and uses thereof
BRPI0811170B8 (pt) 2007-05-22 2021-05-25 Arcturus Therapeutics Inc oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila
AR066984A1 (es) 2007-06-15 2009-09-23 Novartis Ag Inhibicion de la expresion de la subunidad alfa del canal epitelial de sodio (enac) por medio de arni (arn de interferencia)
US20110046206A1 (en) * 2007-06-22 2011-02-24 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
WO2009030254A1 (en) 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
DK2548962T3 (en) 2007-09-19 2016-04-11 Applied Biosystems Llc Sirna sequence-independent modification formats to reduce off-target phenotype effects in RNAI and stabilized forms thereof
EP2848688A1 (en) 2007-12-10 2015-03-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor VII gene
CN105903029A (zh) * 2007-12-12 2016-08-31 大学健康网络 高密度脂蛋白样肽磷脂支架（“hpps”）纳米颗粒
WO2009082817A1 (en) 2007-12-27 2009-07-09 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
CA2710983A1 (en) * 2007-12-27 2009-10-01 The Ohio State University Research Foundation Lipid nanoparticle compositions and methods of making and using the same
GB0801321D0 (en) * 2008-01-24 2008-03-05 Lab901 Ltd Gel composition
EP2176408B9 (en) * 2008-01-31 2015-11-11 Curevac GmbH NUCLEIC ACIDS COMPRISING FORMULA (NuGiXmGnNv)a AND DERIVATIVES THEREOF AS AN IMMUNOSTIMULATING AGENTS /ADJUVANTS
MX2010008394A (es) * 2008-01-31 2010-11-12 Alnylam Pharmaceuticals Inc Metodos optimizados para administracion de arndc focalizando el gen pcsk9.
SG188121A1 (en) 2008-03-05 2013-03-28 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of eg5 and vegf genes
BRPI0910446A8 (pt) * 2008-03-31 2016-07-26 Nat Inst Of Advanced Ind Scien rna modificado por lipídeo de filamento duplo tendo alto efeito de interferência de rna
AU2009238175C1 (en) * 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
JP2011516094A (ja) 2008-04-15 2011-05-26 プロチバ バイオセラピューティクス インコーポレイティッド 干渉ｒｎａを用いたｃｓｎ５遺伝子発現のサイレンシング方法
FR2931152B1 (fr) * 2008-05-16 2010-07-30 Centre Nat Rech Scient Nouveau systeme de transfert d'acide nucleique
EP2297323A1 (en) * 2008-05-21 2011-03-23 Hartmann, Gunther 5' triphosphate oligonucleotide with blunt end and uses thereof
EP2319519B1 (en) * 2008-08-01 2016-01-27 Kyowa Hakko Kirin Co., Ltd. Composition for inhibiting expression of target gene
US9433684B2 (en) 2008-08-19 2016-09-06 Nektar Therapeutics Conjugates of small-interfering nucleic acids
EP3208337A1 (en) 2008-09-02 2017-08-23 Alnylam Pharmaceuticals, Inc. Compositions for combined inhibition of mutant egfr and il-6 expression
CA2739170A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
WO2010037408A1 (en) 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
EA036772B1 (ru) 2008-10-20 2020-12-18 Элнилэм Фармасьютикалз, Инк. Композиции и способы для ингибирования экспрессии транстиретина
HUE037082T2 (hu) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
SG171879A1 (en) 2008-12-03 2011-07-28 Marina Biotech Inc Usirna complexes
EP2373382B1 (en) 2008-12-10 2017-02-15 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
CA2750561C (en) * 2009-01-26 2017-10-10 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
EP2669290A1 (en) 2009-03-02 2013-12-04 Alnylam Pharmaceuticals Inc. Nucleic Acid Chemical Modifications
US9181525B2 (en) * 2009-03-06 2015-11-10 Mie University Method for enhancing a function of a T cell
AU2010223967B2 (en) * 2009-03-12 2015-07-30 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
JP5889783B2 (ja) 2009-05-05 2016-03-22 テクミラ ファーマシューティカルズ コーポレイションＴｅｋｍｉｒａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｃｏｒｐｏｒａｔｉｏｎ 免疫細胞へオリゴヌクレオチドを送達する方法
KR101224828B1 (ko) 2009-05-14 2013-01-22 (주)바이오니아 ｓｉＲＮＡ 접합체 및 그 제조방법
NZ597504A (en) * 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
US20100324124A1 (en) * 2009-06-17 2010-12-23 Massachusetts Institute Of Technology Compositions and methods relating to DNA-based particles
US20100323018A1 (en) * 2009-06-17 2010-12-23 Massachusetts Institute Of Technology Branched DNA/RNA monomers and uses thereof
GB0910723D0 (en) 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
US8236943B2 (en) * 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
WO2011007795A1 (ja) * 2009-07-14 2011-01-20 協和発酵キリン株式会社 標的遺伝子の発現を抑制する組成物
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
US9029338B2 (en) 2009-08-14 2015-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
US20110053829A1 (en) 2009-09-03 2011-03-03 Curevac Gmbh Disulfide-linked polyethyleneglycol/peptide conjugates for the transfection of nucleic acids
WO2011034798A1 (en) 2009-09-15 2011-03-24 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
US9187746B2 (en) 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
WO2011038160A2 (en) * 2009-09-23 2011-03-31 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
HUE042177T2 (hu) 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
EP3000885B1 (en) 2009-12-18 2018-07-25 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat hsf1-related diseases
JP2011155914A (ja) * 2010-02-01 2011-08-18 Osaka Univ 脂質異常症治療薬剤としての化学修飾ｓｉＲＮＡ
WO2011109294A1 (en) * 2010-03-01 2011-09-09 Dicerna Pharmaceuticals, Inc. Lipid delivery formulations
WO2011112597A1 (en) * 2010-03-08 2011-09-15 Northeastern University Magnetic nanoplatforms for theranostic and multi-modal imaging applications
EP3329924B1 (en) 2010-03-29 2021-05-05 Alnylam Pharmaceuticals, Inc. Dsrna therapy for transthyretin (ttr) related ocular amyloidosis
US8455455B1 (en) 2010-03-31 2013-06-04 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes involved in hemorrhagic fever
CA2792561C (en) 2010-04-06 2021-10-26 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of cd274/pd-l1 gene
CA3098080A1 (en) 2010-04-23 2011-10-27 Arrowhead Research Corporation Organic compositions to treat beta-enac-related diseases
HRP20182039T1 (hr) 2010-04-23 2019-02-08 Cold Spring Harbor Laboratory NOVE STRUKTURNO DIZAJNIRANE MOLEKULE shRNA
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
WO2011141703A1 (en) * 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
CA2801066C (en) 2010-06-02 2021-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
DE102010022937A1 (de) * 2010-06-04 2011-12-08 Universitätsklinikum Jena Zellspezifisch aktivierbare biologisch wirksame Moleküle auf Grundlage von siRNA, Verfahren zu deren Aktivierung sowie Applikationskit zur Verabreichung
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
CA2801523C (en) 2010-07-30 2021-08-03 Curevac Gmbh Complexation of nucleic acids with disulfide-crosslinked cationic components for transfection and immunostimulation
WO2012036215A1 (ja) * 2010-09-17 2012-03-22 Taniguchi Tadatsugu Ｈｍｇｂタンパクによって仲介される免疫応答の活性化の抑制剤及びスクリーニング方法
BR112013005872A2 (pt) 2010-09-22 2019-09-24 Alios Biopharma Inc compostos, composição farmacêutica e respectivos usos
WO2012059612A1 (es) 2010-11-04 2012-05-10 Consejo Superior De Investigaciones Científicas (Csic) Derivados de pequeños arn de interferencia y su uso
WO2012061810A1 (en) 2010-11-05 2012-05-10 Miragen Therapeutics Base modified oligonucleotides
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
EP2648763A4 (en) 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR EXPRESSION INHIBITION OF GENES KLF-1 AND BCL11A
EP2649182A4 (en) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc Compositions and methods for increasing erythropoietin (epo) production
JP5952197B2 (ja) * 2011-01-19 2016-07-13 協和発酵キリン株式会社 標的遺伝子の発現を抑制する組成物
EP2508530A1 (en) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification of triphosphorylated oligonucleotides using capture tags
KR102365961B1 (ko) 2011-03-29 2022-02-23 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
EP3354644A1 (en) 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
PL3586861T3 (pl) 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
CN106442039B (zh) 2011-06-19 2020-08-07 阿博根公司 用于样品采集的装置、溶液和方法
ES2923573T3 (es) 2011-06-21 2022-09-28 Alnylam Pharmaceuticals Inc Composiciones de ARNi de proteína 3 de tipo angiopoyetina (ANGPTL3) y métodos de uso de las mismas
US20140275211A1 (en) 2011-06-21 2014-09-18 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
CN103649103A (zh) 2011-06-21 2014-03-19 阿尔尼拉姆医药品有限公司 用于抑制载脂蛋白c-iii(apoc3)基因表达的组合物与方法
WO2012177949A2 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
EP3366312A1 (en) 2011-06-23 2018-08-29 Alnylam Pharmaceuticals, Inc. Serpina 1 sirnas: compositions of matter and methods of treatment
JP2014526887A (ja) 2011-08-01 2014-10-09 アルナイラム ファーマシューティカルズ， インコーポレイテッド 造血幹細胞移植の成功率を改善する方法
CN103930547A (zh) 2011-09-02 2014-07-16 诺华股份有限公司 用于治疗hsf1相关疾病的有机组合物
US8999950B2 (en) * 2011-10-05 2015-04-07 Protiva Biotherapeutics Inc. Compositions and methods for silencing aldehyde dehydrogenase
JP6294229B2 (ja) 2011-10-18 2018-03-14 ダイセルナ ファーマシューティカルズ， インコーポレイテッドＤｉｃｅｒｎａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アミン陽イオン性脂質およびその使用
US9579338B2 (en) 2011-11-04 2017-02-28 Nitto Denko Corporation Method of producing lipid nanoparticles for drug delivery
CA2853685C (en) * 2011-11-04 2019-09-03 Nitto Denko Corporation Single use system for sterilely producing lipid-nucleic acid particles
HUE048622T2 (hu) 2011-11-18 2020-08-28 Alnylam Pharmaceuticals Inc RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére
EP2780454A2 (en) 2011-11-18 2014-09-24 Alnylam Pharmaceuticals, Inc. Modified rnai agents
EP2794630A4 (en) 2011-12-22 2015-04-01 Alios Biopharma Inc SUBSTITUTED PHOSPHORTHIOAT NUCLEOTIDE ANALOGUE
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
CA2860676A1 (en) 2012-01-09 2013-07-18 Novartis Ag Organic compositions to treat beta-catenin-related diseases
WO2013109057A1 (ko) * 2012-01-18 2013-07-25 (주)바이오니아 자성나노입자-samirna 복합체 및 그 제조방법
WO2013113326A1 (en) 2012-01-31 2013-08-08 Curevac Gmbh Pharmaceutical composition comprising a polymeric carrier cargo complex and at least one protein or peptide antigen
SG11201405157PA (en) 2012-02-24 2014-10-30 Protiva Biotherapeutics Inc Trialkyl cationic lipids and methods of use thereof
WO2013142124A1 (en) 2012-03-21 2013-09-26 Vertex Pharmaceuticals Incorporated Solid forms of a thiophosphoramidate nucleotide prodrug
US9012427B2 (en) 2012-03-22 2015-04-21 Alios Biopharma, Inc. Pharmaceutical combinations comprising a thionucleotide analog
ES2762873T3 (es) 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
HK1206644A1 (en) 2012-03-29 2016-01-15 Shire Human Genetic Therapies, Inc. Lipid-derived neutral nanoparticles
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EP3272868A1 (en) 2012-05-02 2018-01-24 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat kras-related diseases
US9750819B2 (en) 2012-05-23 2017-09-05 Ohio State Innovation Foundation Lipid nanoparticle compositions and methods of making and methods of using the same
US10245229B2 (en) 2012-06-08 2019-04-02 Translate Bio, Inc. Pulmonary delivery of mRNA to non-lung target cells
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, Inc. Nuclease resistant polynucleotides and uses thereof
EP2712870A1 (en) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Novel RIG-I ligands and methods for producing them
US10125369B2 (en) 2012-12-05 2018-11-13 Alnylam Pharmaceuticals, Inc. PCSK9 iRNA compositions and methods of use thereof
DK2931897T3 (en) 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
EP2953647B1 (en) * 2013-02-05 2024-07-17 B.G. Negev Technologies and Applications Ltd. Positively charged polysaccharides for rna transfection
WO2014130922A1 (en) 2013-02-25 2014-08-28 Trustees Of Boston University Compositions and methods for treating fungal infections
SG10201706960TA (en) 2013-02-28 2017-10-30 Arrowhead Res Corp Organic compositions to treat epas1-related diseases
DK2970456T3 (da) 2013-03-14 2021-07-05 Translate Bio Inc Fremgangsmåder og sammensætninger til levering af mrna-kodede antistoffer
WO2014153163A1 (en) 2013-03-14 2014-09-25 Dicerna Pharmaceuticals, Inc. Process for formulating an anionic agent
PL3467108T3 (pl) 2013-03-14 2024-09-30 Translate Bio, Inc. Sposoby oczyszczania informacyjnego rna
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
US10130649B2 (en) 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
US20140288149A1 (en) 2013-03-15 2014-09-25 Graham Lord Mir-142 and antagonists thereof for treating disease
SG11201507141SA (en) * 2013-03-15 2015-10-29 Techulon Inc Antisense molecules for treatment of staphylococcus aureus infection
US9784730B2 (en) 2013-03-21 2017-10-10 University Of Washington Through Its Center For Commercialization Nanoparticle for targeting brain tumors and delivery of O6-benzylguanine
WO2014182661A2 (en) 2013-05-06 2014-11-13 Alnylam Pharmaceuticals, Inc Dosages and methods for delivering lipid formulated nucleic acid molecules
MX2015017312A (es) 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
KR20160019553A (ko) 2013-06-17 2016-02-19 더 브로드 인스티튜트, 인코퍼레이티드 유사분열 후 세포의 질병 및 장애를 표적화하고 모델링하기 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
US9889200B2 (en) * 2013-07-31 2018-02-13 Qbi Enterprises Ltd. Sphingolipid-polyalkylamine-oligonucleotide compounds
BR112016003361A2 (pt) 2013-08-21 2017-11-21 Curevac Ag vacina do vírus sincicial respiratório (rsv)
US10077444B2 (en) 2013-10-02 2018-09-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
TWI768330B (zh) 2013-10-04 2022-06-21 美國西奈山伊坎醫學院 抑制alas1基因表現的組合物及方法
WO2015051135A2 (en) 2013-10-04 2015-04-09 Novartis Ag Organic compositions to treat hepcidin-related diseases
AU2014340149B2 (en) 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
BR112016013207A2 (pt) 2013-12-12 2017-09-26 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para o hbv e distúrbios e doenças virais
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
MX2016007325A (es) 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US9856475B2 (en) 2014-03-25 2018-01-02 Arcturus Therapeutics, Inc. Formulations for treating amyloidosis
JP6771387B2 (ja) 2014-03-25 2020-10-21 アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 遺伝子サイレンシング用トランスサイレチン対立遺伝子選択的ｕｎａオリゴマー
WO2015148580A2 (en) 2014-03-25 2015-10-01 Arcturus Therapeutics, Inc. Una oligomers having reduced off-target effects in gene silencing
CA2936286A1 (en) 2014-04-01 2015-10-08 Curevac Ag Polymeric carrier cargo complex for use as an immunostimulating agent or as an adjuvant
SG10201912038TA (en) * 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
PT3134506T (pt) 2014-04-25 2019-10-31 Translate Bio Inc Métodos de purificação de rna mensageiro
JP6557722B2 (ja) 2014-05-30 2019-08-07 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 核酸の送達のための生分解性脂質
US11491480B2 (en) 2014-06-13 2022-11-08 Children's Medical Center Corporation Products and methods to isolate mitochondria
EP3160959B1 (en) 2014-06-24 2023-08-30 Translate Bio, Inc. Stereochemically enriched compositions for delivery of nucleic acids
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
EP3736334A1 (en) 2014-07-16 2020-11-11 Arrowhead Pharmaceuticals, Inc. Rnai compositions to treat apoc3-related diseases
EP3686279B1 (en) 2014-08-17 2023-01-04 The Broad Institute, Inc. Genome editing using cas9 nickases
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
US9765333B2 (en) 2014-08-21 2017-09-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing marburg virus gene expression
DK3185957T3 (da) 2014-08-29 2022-08-29 Alnylam Pharmaceuticals Inc Patisiran til anvendelse ved behandlingen af transthyretin-medieret amyloidosis
EP3191592A1 (en) 2014-09-11 2017-07-19 Novartis AG Inhibition of prmt5 to treat mtap-deficiency-related diseases
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
EP3227446A1 (en) 2014-12-01 2017-10-11 Novartis AG Compositions and methods for diagnosis and treatment of prostate cancer
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
CA2970795A1 (en) 2014-12-18 2016-06-23 Alnylam Pharmaceuticals, Inc. Reversir compounds
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
US10792299B2 (en) 2014-12-26 2020-10-06 Nitto Denko Corporation Methods and compositions for treating malignant tumors associated with kras mutation
US20180002702A1 (en) 2014-12-26 2018-01-04 Nitto Denko Corporation Methods and compositions for treating malignant tumors associated with kras mutation
US11045488B2 (en) 2014-12-26 2021-06-29 Nitto Denko Corporation RNA interference agents for GST-π gene modulation
US10264976B2 (en) 2014-12-26 2019-04-23 The University Of Akron Biocompatible flavonoid compounds for organelle and cell imaging
ES2789049T3 (es) * 2014-12-26 2020-10-23 Nitto Denko Corp Agentes de interferencia de ARN para modulación génica de GST-pi
US10172924B2 (en) 2015-03-19 2019-01-08 Translate Bio, Inc. MRNA therapy for pompe disease
WO2016161299A1 (en) 2015-04-01 2016-10-06 Arcturus Therapeutics, Inc. Therapeutic una oligomers and uses thereof
PT3277815T (pt) * 2015-04-03 2021-11-11 Beth Israel Deaconess Medical Ct Inc Compostos de oligonucleótidos para o tratamento de pré-eclâmpsia e outros distúrbios angiogénicos
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
EP3302525A2 (en) 2015-06-05 2018-04-11 Novartis AG Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders
EP4159856A1 (en) 2015-06-18 2023-04-05 The Broad Institute, Inc. Novel crispr enzymes and systems
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
TWI813532B (zh) 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的ｃｒｉｓｐｒ酶突變
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2017015671A1 (en) 2015-07-23 2017-01-26 Arcturus Therapeutics, Inc. Compositions for treating amyloidosis
CN108138182B (zh) 2015-07-31 2022-08-19 阿尔尼拉姆医药品有限公司 甲状腺素运载蛋白(TTR)iRNA组合物及其治疗或预防TTR相关疾病的使用方法
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017031370A1 (en) 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US12241053B2 (en) 2015-10-09 2025-03-04 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
CN114686548A (zh) 2015-10-14 2022-07-01 川斯勒佰尔公司 用于增强生产的rna相关酶的修饰
CN109153980B (zh) 2015-10-22 2023-04-14 布罗德研究所有限公司 Vi-b型crispr酶和系统
EP3364981A4 (en) * 2015-10-22 2019-08-07 ModernaTX, Inc. VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS
EP3368687B1 (en) 2015-10-27 2021-09-29 The Broad Institute, Inc. Compositions and methods for targeting cancer-specific sequence variations
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
IL289849B2 (en) 2015-11-10 2024-01-01 Nat Inst Biotechnology Negev Ltd Means and methods for reducing tumorigenicity of cancer stem cells
US11903974B2 (en) 2015-11-30 2024-02-20 Flagship Pioneering Innovations V, Inc. Methods and compositions relating to chondrisomes from cultured cells
AU2016363114B2 (en) * 2015-12-04 2023-04-20 Commonwealth Scientific And Industrial Research Organisation Regulation of cytokine production
ES2952064T3 (es) 2015-12-16 2023-10-26 Walter & Eliza Hall Inst Medical Res Inhibición de la proteína SH2 inducida por citocinas en células NK
EP3181698A1 (en) 2015-12-16 2017-06-21 European Molecular Biology Laboratory (EMBL) Microrna mir-142 as stem cell marker
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
SI4272834T1 (sl) 2016-01-15 2026-03-31 Beth Israel Deaconess Medical Center, Inc. Terapevtska uporaba mitohondrijev in kombiniranih mitohondrijskih sredstev
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
WO2017172941A2 (en) * 2016-03-29 2017-10-05 Berz David Modulation of structural maintenance of chromosome-1 expression
WO2017177169A1 (en) 2016-04-08 2017-10-12 Rana Therapeutics, Inc. Multimeric coding nucleic acid and uses thereof
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
US20200263190A1 (en) 2016-04-19 2020-08-20 The Broad Institute, Inc. Novel crispr enzymes and systems
CN110382692A (zh) 2016-04-19 2019-10-25 博德研究所 新型crispr酶以及系统
US12214299B2 (en) 2016-05-31 2025-02-04 Dna Genotek Inc. Composition, system and method for removal of detergents from aqueous solutions
CA3027312A1 (en) 2016-06-13 2017-12-21 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
CA3028158A1 (en) 2016-06-17 2017-12-21 The Broad Institute, Inc. Type vi crispr orthologs and systems
US12595478B2 (en) 2016-06-29 2026-04-07 The Broad Institute, Inc. Crispr-Cas systems having destabilization domain
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
CN110312799A (zh) 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
WO2018035388A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
US20190262399A1 (en) 2016-09-07 2019-08-29 The Broad Institute, Inc. Compositions and methods for evaluating and modulating immune responses
WO2018047148A1 (en) 2016-09-12 2018-03-15 Novartis Ag Compounds for the inhibition of mirna
WO2018064208A1 (en) 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
US12447213B2 (en) 2016-10-07 2025-10-21 The Broad Institute, Inc. Modulation of novel immune checkpoint targets
CN107951862B (zh) * 2016-10-17 2021-03-12 南京绿叶制药有限公司 一种抑制bcl-2的反义寡聚核酸的脂质纳米粒及其制备方法
MA46584A (fr) 2016-10-21 2019-08-28 Modernatx Inc Vaccin contre le cytomégalovirus humain
US20180245065A1 (en) 2016-11-01 2018-08-30 Novartis Ag Methods and compositions for enhancing gene editing
EP3535274A4 (en) * 2016-11-07 2020-07-29 Nanosur LLC Post-transcriptionally chemically modified double stranded RNA
WO2018089851A2 (en) 2016-11-11 2018-05-17 Modernatx, Inc. Influenza vaccine
JP2019508379A (ja) * 2017-02-16 2019-03-28 日東電工株式会社 悪性腫瘍に対する治療方法及び治療用組成物
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
AU2018234825B2 (en) 2017-03-15 2020-12-17 Massachusetts Institute Of Technology Novel CAS13B orthologues CRISPR enzymes and systems
AU2018251801B2 (en) 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
US12350368B2 (en) 2017-04-14 2025-07-08 The Broad Institute, Inc. Delivery of large payloads
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
AU2018266111B2 (en) 2017-05-08 2024-11-07 Flagship Pioneering Innovations V, Inc. Compositions for facilitating membrane fusion and uses thereof
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
IL271275B1 (en) 2017-06-13 2026-04-01 Flagship Pioneering Innovations V Inc Preparations containing croons and their uses
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
TN2019000308A1 (en) 2017-07-06 2021-05-07 Arrowhead Pharmaceuticals Inc RNAi AGENTS FOR INHIBITING EXPRESSION OF ALPHA-ENaC AND METHODS OF USE
US11530413B2 (en) 2017-07-21 2022-12-20 Novartis Ag Compositions and methods to treat cancer
CN117866959A (zh) * 2017-09-07 2024-04-12 北京泰德制药股份有限公司 靶向ckip-1的双链rna分子及其用途
US10597657B2 (en) 2017-09-11 2020-03-24 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
KR20250046363A (ko) 2017-09-19 2025-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
JP2020535802A (ja) 2017-09-21 2020-12-10 ザ・ブロード・インスティテュート・インコーポレイテッド 標的化核酸編集のための系、方法、及び組成物
EP3692152B1 (en) 2017-10-04 2025-09-03 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
US11547614B2 (en) 2017-10-31 2023-01-10 The Broad Institute, Inc. Methods and compositions for studying cell evolution
WO2019094578A1 (en) 2017-11-09 2019-05-16 Alnylam Pharmaceuticals Inc. Assays and methods for determining expression of the lect2 gene
EP3710039A4 (en) 2017-11-13 2021-08-04 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF CANCER BY TARGETING THE CLEC2D-KLRB1 PATH
EP3727428A1 (en) 2017-12-20 2020-10-28 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
WO2019150309A1 (en) 2018-02-02 2019-08-08 Hammack Scott Modulators of gpr68 and uses thereof for treating and preventing diseases
EP3788138A1 (en) 2018-05-02 2021-03-10 Novartis AG Regulators of human pluripotent stem cells and uses thereof
AU2019293044A1 (en) * 2018-06-29 2021-01-21 John Mansell Compositions and methods for the treatment of anesthesia-induced neurotoxicity
CN113286884A (zh) 2018-08-07 2021-08-20 博德研究所 新型cas12b酶和系统
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
US11987792B2 (en) 2018-08-16 2024-05-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
CA3113648A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
WO2020097493A1 (en) 2018-11-09 2020-05-14 Arbutus Biopharma Corporation Negatively charged peg-lipid conjugates
KR20210105889A (ko) 2018-11-09 2021-08-27 아뷰터스 바이오파마 코포레이션 지질 나노입자 제제
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.
US11166996B2 (en) 2018-12-12 2021-11-09 Flagship Pioneering Innovations V, Inc. Anellovirus compositions and methods of use
MY206794A (en) 2018-12-20 2025-01-08 Humabs Biomed Sa Combination hbv therapy
SG11202105577TA (en) 2019-01-09 2021-06-29 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use
EP3908328A1 (en) 2019-01-10 2021-11-17 BioNTech RNA Pharmaceuticals GmbH Localized administration of rna molecules for therapy
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
AU2020343255A1 (en) 2019-09-03 2022-03-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
MX2022009763A (es) 2020-02-10 2022-09-09 Alnylam Pharmaceuticals Inc Composiciones y metodos para silenciar la expresion del factor de crecimiento endotelial vascular a (vegf-a).
JP7709981B2 (ja) 2020-03-17 2025-07-17 ジェネヴァント サイエンシズ ゲーエムベーハー 肝星細胞への治療薬の脂質ナノ粒子送達のためのカチオン性脂質
EP4127171A2 (en) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
AU2021246024A1 (en) * 2020-03-30 2022-10-27 Alnylam Pharmaceuticals, Inc. Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof
EP4133078A1 (en) 2020-04-06 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
CA3179678A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
CA3182458A1 (en) 2020-06-24 2021-12-30 Laura ROSEN Engineered hepatitis b virus neutralizing antibodies and uses thereof
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
AU2021403156A1 (en) 2020-12-18 2023-07-13 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles
CN112707943B (zh) * 2020-12-31 2022-09-09 北京大学 结合5′-末端缀合物和中性/阳离子混合脂材包载的小干扰rna及其修饰方法
WO2022223515A2 (en) 2021-04-19 2022-10-27 Novo Nordisk A/S Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
AU2022290278A1 (en) 2021-06-11 2024-01-04 LifeEDIT Therapeutics, Inc. Rna polymerase iii promoters and methods of use
AU2022299169A1 (en) 2021-06-23 2024-02-08 Beth Israel Deaconess Medical Center, Inc. Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
CA3226019A1 (en) 2021-07-20 2023-01-26 Ags Therapeutics Sas Extracellular vesicles from microalgae, their preparation, and uses
CA3227852A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
WO2023034837A2 (en) 2021-08-31 2023-03-09 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
CA3250180A1 (en) 2022-01-31 2023-08-03 Genevant Sciences Gmbh IONIZABLE CATIONIC LIPIDS FOR LIPID NANOPARTICLES
CN118900701A (zh) 2022-01-31 2024-11-05 盖纳万科学有限公司 聚(烷基噁唑啉)-脂质缀合物及含有聚(烷基噁唑啉)-脂质缀合物的脂质颗粒
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
CN119562762A (zh) 2022-04-04 2025-03-04 加利福尼亚大学董事会 遗传互补组合物和方法
EP4531819A2 (en) 2022-05-25 2025-04-09 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2023229535A1 (en) 2022-05-27 2023-11-30 Engine Biosciences Pte. Ltd. Bet polypeptide therapeutic agents for generating synthetic lethality in tumors
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
CA3264752A1 (en) 2022-08-12 2024-02-15 Life Edit Therapeutics, Inc. RNA-GUIDED NUCLEASE AND ACTIVE FRAGMENTS, ASSOCIATED VARIANTS AND METHODS OF USE
CN121038801A (zh) 2022-10-24 2025-11-28 Ags治疗简易股份公司 来自微藻的细胞外囊泡、它们在鼻内施用时的生物分布及其用途
WO2024158825A2 (en) 2023-01-23 2024-08-02 Engine Biosciences Pte. Ltd. Methods to modulate tau for treatment of tauopathies
EP4662311A2 (en) 2023-02-09 2025-12-17 Alnylam Pharmaceuticals, Inc. Reversir molecules and methods of use thereof
WO2025022367A2 (en) 2023-07-27 2025-01-30 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2025052278A1 (en) 2023-09-05 2025-03-13 Genevant Sciences Gmbh Pyrrolidine based cationic lipids for lipid nanoparticle delivery of therapeutics to hepatic stellate cells
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025133951A1 (en) 2023-12-21 2025-06-26 Genevant Sciences Gmbh Ionizable lipids suitable for lipid nanoparticles
WO2025171411A1 (en) 2024-02-09 2025-08-14 Herophilus, Inc. Compositions and methods related to modulating macrophage migration inhibitory factor (mif)-cd74 signaling and related treatments for neuroinflammatory conditions
TW202546224A (zh) 2024-02-12 2025-12-01 美商生命編輯治療學公司 新穎的ｒｎａ引導之核酸酶及用於聚合酶編輯之蛋白質
WO2025176847A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Ocular delivery of active agents via microalgae extracellular vesicles
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
WO2026003754A1 (en) 2024-06-25 2026-01-02 Life Edit Therapeutics, Inc. Novel reverse transcriptases and uses thereof
WO2026022656A2 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Lipid particle formulations with permanently charged cationic lipids
WO2026022677A1 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Permanently charged cationic lipids

Family Cites Families (229)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3901654A (en) 1971-06-21 1975-08-26 Biological Developments Receptor assays of biologically active compounds employing biologically specific receptors
US3817827A (en) 1972-03-30 1974-06-18 Scott Paper Co Soft absorbent fibrous webs containing elastomeric bonding material and formed by creping and embossing
US3935074A (en) 1973-12-17 1976-01-27 Syva Company Antibody steric hindrance immunoassay with two antibodies
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4034074A (en) 1974-09-19 1977-07-05 The Board Of Trustees Of Leland Stanford Junior University Universal reagent 2-site immunoradiometric assay using labelled anti (IgG)
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US3984533A (en) 1975-11-13 1976-10-05 General Electric Company Electrophoretic method of detecting antigen-antibody reaction
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
US4098876A (en) 1976-10-26 1978-07-04 Corning Glass Works Reverse sandwich immunoassay
CH624011A5 (https=) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4394448A (en) * 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
EP0032578B1 (de) * 1980-01-16 1984-07-25 Hans Georg Prof. Dr. Weder Verfahren und Dialysiereinrichtung zur Herstellung von Bilayer-Vesikeln und Verwendung der Bilayer-Vesikel
US4598051A (en) * 1980-03-12 1986-07-01 The Regents Of The University Of California Liposome conjugates and diagnostic methods therewith
US4452901A (en) 1980-03-20 1984-06-05 Ciba-Geigy Corporation Electrophoretically transferring electropherograms to nitrocellulose sheets for immuno-assays
US4376110A (en) 1980-08-04 1983-03-08 Hybritech, Incorporated Immunometric assays using monoclonal antibodies
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US4515736A (en) * 1983-05-12 1985-05-07 The Regents Of The University Of California Method for encapsulating materials into liposomes
US5208036A (en) 1985-01-07 1993-05-04 Syntex (U.S.A.) Inc. N-(ω, (ω-1)-dialkyloxy)- and N-(ω, (ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5545412A (en) * 1985-01-07 1996-08-13 Syntex (U.S.A.) Inc. N-[1, (1-1)-dialkyloxy]-and N-[1, (1-1)-dialkenyloxy]-alk-1-yl-n,n,n-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) * 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5320906A (en) * 1986-12-15 1994-06-14 Vestar, Inc. Delivery vehicles with amphiphile-associated active ingredient
US5703055A (en) * 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
FR2645866B1 (fr) * 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
US5013556A (en) * 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5225212A (en) * 1989-10-20 1993-07-06 Liposome Technology, Inc. Microreservoir liposome composition and method
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) * 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US6465188B1 (en) 1990-06-11 2002-10-15 Gilead Sciences, Inc. Nucleic acid ligand complexes
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US6831166B2 (en) 1992-10-23 2004-12-14 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
WO1993012240A1 (en) 1991-12-17 1993-06-24 The Regents Of The University Of California Gene therapy for cystic fibrosis transmembrane conductance regulator activity (cftr)
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
US5858784A (en) * 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
JP2731630B2 (ja) * 1992-02-19 1998-03-25 ベイラー・カレッジ・オブ・メディスン 細胞増殖のオリゴヌクレオチドの変調
ES2105262T3 (es) 1992-05-18 1997-10-16 Minnesota Mining & Mfg Dispositivo de suministro de farmaco a traves de las mucosas.
EP0646178A1 (en) 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
WO1995002698A1 (en) 1993-07-12 1995-01-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
US5426039A (en) 1993-09-08 1995-06-20 Bio-Rad Laboratories, Inc. Direct molecular cloning of primer extended DNA containing an alkane diol
US5674908A (en) 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
FR2714830B1 (fr) 1994-01-10 1996-03-22 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
FR2715847B1 (fr) 1994-02-08 1996-04-12 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
US6075012A (en) 1994-02-11 2000-06-13 Life Technologies, Inc. Reagents for intracellular delivery of macromolecules
US6989434B1 (en) 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
US5639725A (en) 1994-04-26 1997-06-17 Children's Hospital Medical Center Corp. Angiostatin protein
AU696881B2 (en) 1994-06-22 1998-09-24 Megabios Corporation Cationic amphiphiles
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US5908635A (en) 1994-08-05 1999-06-01 The United States Of America As Represented By The Department Of Health And Human Services Method for the liposomal delivery of nucleic acids
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
ATE219660T1 (de) 1994-09-30 2002-07-15 Inex Pharmaceuticals Corp Mittel zum einbringen polyanionischer materialien in zellen
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5627159A (en) 1994-10-27 1997-05-06 Life Technologies, Inc. Enhancement of lipid cationic transfections in the presence of serum
US5580579A (en) 1995-02-15 1996-12-03 Nano Systems L.L.C. Site-specific adhesion within the GI tract using nanoparticles stabilized by high molecular weight, linear poly (ethylene oxide) polymers
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US20030069173A1 (en) 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
US5705385A (en) * 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
EP0830368A1 (en) 1995-06-07 1998-03-25 Genta Incorporated Novel carbamate-based cationic lipids
US5756122A (en) 1995-06-07 1998-05-26 Georgetown University Liposomally encapsulated nucleic acids having high entrapment efficiencies, method of manufacturer and use thereof for transfection of targeted cells
US5958901A (en) 1995-06-07 1999-09-28 Genta Incorporated Phosphonic acid-based cationic lipids
US5811406A (en) 1995-06-07 1998-09-22 Regents Of The University Of California Dry powder formulations of polynucleotide complexes
US5981501A (en) * 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
ATE285477T1 (de) * 1995-06-07 2005-01-15 Inex Pharmaceutical Corp Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US5736392A (en) 1995-06-07 1998-04-07 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
EP0869937A4 (en) 1995-07-21 2004-07-21 Promega Biosciences Inc AMID BASED CATIONIC LIPIDS
US6339173B1 (en) 1996-07-22 2002-01-15 Promega Biosciences, Inc. Amide-based cationic lipids
IL115199A (en) 1995-09-07 2005-05-17 Opperbas Holding Bv Composition comprising a polynucleic acid molecule in a liposome and method using said composition
US20040142895A1 (en) 1995-10-26 2004-07-22 Sirna Therapeutics, Inc. Nucleic acid-based modulation of gene expression in the vascular endothelial growth factor pathway
US20030216335A1 (en) 2001-11-30 2003-11-20 Jennifer Lockridge Method and reagent for the modulation of female reproductive diseases and conditions
US6330349B1 (en) 1995-11-30 2001-12-11 Chromavision Medical Systems, Inc. Automated method for image analysis of residual protein
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6620805B1 (en) 1996-03-14 2003-09-16 Yale University Delivery of nucleic acids by porphyrins
ATE273320T1 (de) 1996-04-11 2004-08-15 Univ British Columbia Fusogene liposomen
US6713069B1 (en) 1996-04-16 2004-03-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and methods for detecting, preventing, and treating African Hemorrhagic Fever
US6756054B1 (en) 1996-05-24 2004-06-29 Ic-Vec Limited Polycationic sterol derivatives as transfection agents
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US20050119470A1 (en) 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
JP4320054B2 (ja) 1996-08-13 2009-08-26 ノバルティス バクシンズ アンド ダイアグノスティックス，インコーポレーテッド ポリヌクレオチド送達のための組成物および方法
US6111085A (en) 1996-09-13 2000-08-29 Isis Pharmaceuticals, Inc. Carbamate-derivatized nucleosides and oligonucleosides
US6174861B1 (en) 1996-10-22 2001-01-16 The Children's Medical Center Corporation Methods of inhibiting angiogenesis via increasing in vivo concentrations of endostatin protein
US5877220A (en) 1997-03-06 1999-03-02 Genta, Incorporated Amide-based oligomeric cationic lipids
US6034135A (en) 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
US6126965A (en) 1997-03-21 2000-10-03 Georgetown University School Of Medicine Liposomes containing oligonucleotides
US20030229040A1 (en) 1997-03-21 2003-12-11 Georgetown University Cationic liposomal delivery system and therapeutic use thereof
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US20030104044A1 (en) * 1997-05-14 2003-06-05 Semple Sean C. Compositions for stimulating cytokine secretion and inducing an immune response
DE69834038D1 (de) 1997-07-01 2006-05-18 Isis Pharmaceutical Inc Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre
US20030073640A1 (en) * 1997-07-23 2003-04-17 Ribozyme Pharmaceuticals, Inc. Novel compositions for the delivery of negatively charged molecules
CA2301166A1 (en) 1997-07-24 1999-02-04 Yuan-Peng Zhang Liposomal compositions for the delivery of nucleic acid catalysts
US6110745A (en) 1997-07-24 2000-08-29 Inex Pharmaceuticals Corp. Preparation of lipid-nucleic acid particles using a solvent extraction and direct hydration method
US20030036516A1 (en) * 1997-09-10 2003-02-20 Sudhir Agrawal Method for using oligonucleotides having modified cpg dinucleotides
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
US6509323B1 (en) 1998-07-01 2003-01-21 California Institute Of Technology Linear cyclodextrin copolymers
US7091192B1 (en) 1998-07-01 2006-08-15 California Institute Of Technology Linear cyclodextrin copolymers
WO2000003683A2 (en) 1998-07-20 2000-01-27 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
DK1129064T3 (da) 1998-11-12 2008-04-28 Invitrogen Corp Transfektionsreagenser
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
ATE511543T1 (de) 1999-02-22 2011-06-15 Univ Georgetown Immunoliposome mit einem targeting- antikörperfragment zur systemischen genverabreichung
WO2000062813A2 (en) 1999-04-20 2000-10-26 The University Of British Columbia Cationic peg-lipids and methods of use
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
US7112337B2 (en) 1999-04-23 2006-09-26 Alza Corporation Liposome composition for delivery of nucleic acid
US7098032B2 (en) 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
CA2378430A1 (en) 1999-07-15 2001-01-25 Inex Pharmaceuticals Corp. Methods and apparatus for preparation of lipid vesicles
US6200599B1 (en) 1999-10-07 2001-03-13 The Regents Of The University Of California Ortho ester lipids
US20050037086A1 (en) 1999-11-19 2005-02-17 Zycos Inc., A Delaware Corporation Continuous-flow method for preparing microparticles
EP1311288A1 (en) * 2000-01-20 2003-05-21 Ottawa Health Research Institute Immunostimulatory nucleic acids for inducing a th2 immune response
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
WO2002081628A2 (en) * 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
US8273866B2 (en) * 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US20050020525A1 (en) * 2002-02-20 2005-01-27 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2005019453A2 (en) 2001-05-18 2005-03-03 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
JP2003528131A (ja) 2000-03-29 2003-09-24 アラダイム コーポレーション カチオン性リポソーム
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
US20040142475A1 (en) 2000-06-02 2004-07-22 Barman Shikha P. Delivery systems for bioactive agents
US20030072794A1 (en) 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
US6680068B2 (en) * 2000-07-06 2004-01-20 The General Hospital Corporation Drug delivery formulations and targeting
DE60144019D1 (de) 2000-07-21 2011-03-24 Nestle Sa Pulverförmige produkte auf eibasis und diesen enthaltende nahrungsmittel
JP2005500806A (ja) 2000-09-15 2005-01-13 コーリー ファーマシューティカル ゲーエムベーハー ＣｐＧに基づく免疫アゴニスト／免疫アンタゴニストの高スループットスクリーニングのためのプロセス
US7427394B2 (en) 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
AU2002214854A1 (en) 2000-10-25 2002-05-06 Inex Pharmaceuticals Corporation Lipid formulations for target delivery
AU2002232387A1 (en) 2000-10-27 2002-05-06 Invitrogen Corporation Method for introducing antisense oligonucleotides into eucaryotic cells
US20040259247A1 (en) * 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
TWI321054B (en) 2000-12-19 2010-03-01 California Inst Of Techn Compositions containing inclusion complexes
GB0106041D0 (en) 2001-03-12 2001-05-02 Cancer Res Ventures Ltd Lipids and liposomes
US7108863B2 (en) 2001-03-26 2006-09-19 Alza Corporation Liposome composition for improved intracellular delivery of a therapeutic agent
WO2002076427A2 (en) 2001-03-26 2002-10-03 Thomas Jefferson University Ph sensitive liposomal drug delivery
US20030026831A1 (en) 2001-04-20 2003-02-06 Aparna Lakkaraju Anionic liposomes for delivery of bioactive agents
US20040142892A1 (en) * 2001-04-30 2004-07-22 The University Of British Columbia Autogene nucleic acids encoding a secretable RNA polymerase
US20030077829A1 (en) * 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
JP2004535388A (ja) 2001-04-30 2004-11-25 ターゲティッド ジェネティクス コーポレイション 脂質含有薬物送達複合体およびそれらの生成方法
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US20050282188A1 (en) * 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20030175950A1 (en) * 2001-05-29 2003-09-18 Mcswiggen James A. RNA interference mediated inhibition of HIV gene expression using short interfering RNA
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20040019001A1 (en) 2002-02-20 2004-01-29 Mcswiggen James A. RNA interference mediated inhibition of protein typrosine phosphatase-1B (PTP-1B) gene expression using short interfering RNA
US20060211642A1 (en) * 2001-05-18 2006-09-21 Sirna Therapeutics, Inc. RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)
US20070042983A1 (en) * 2001-05-18 2007-02-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
DE10127526A1 (de) 2001-05-31 2002-12-12 Novosom Ag Verfahren zur Herstellung und Auflösung von Nano- und Mikrokapseln
US7063860B2 (en) 2001-08-13 2006-06-20 University Of Pittsburgh Application of lipid vehicles and use for drug delivery
US7101995B2 (en) 2001-08-27 2006-09-05 Mirus Bio Corporation Compositions and processes using siRNA, amphipathic compounds and polycations
GB0123401D0 (en) 2001-09-28 2001-11-21 Novartis Forschungsstiftung Methods of inducing gene expression
WO2003031573A2 (en) * 2001-10-05 2003-04-17 Coley Pharmaceutical Gmbh Toll-like receptor 3 signaling agonists and antagonists
DE10152145A1 (de) 2001-10-19 2003-05-22 Novosom Ag Stabilisierung von Liposomen und Emulsionen
GB0125265D0 (en) * 2001-10-20 2001-12-12 Qinetiq Ltd Optical filter
EP1575976A4 (en) 2001-11-02 2006-08-23 Insert Therapeutics Inc METHODS AND COMPOSITIONS FOR THE THERAPEUTIC USE OF RNA INTERFERENCE
DE10157046A1 (de) 2001-11-18 2003-06-12 Novosom Ag Nano- und Mikrokapseln umfassend Reaktivpolymere
AU2002359892A1 (en) 2001-12-31 2003-07-24 Elan Pharmaceuticals, Inc. Efficient nucleic acid encapsulation into medium sized liposomes
CA2472462A1 (en) 2002-01-09 2003-07-24 Elan Pharmaceuticals, Inc. Efficient nucleic acid encapsulation into medium sized liposomes
EP1480657A4 (en) 2002-02-01 2006-07-05 Intradigm Corp POLYMERS FOR ADMINISTERING PEPTIDES AND SMALL MOLECULES IN VIVO / I
US20060009409A1 (en) * 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
DE10207177A1 (de) 2002-02-19 2003-09-04 Novosom Ag Fakultativ kationische Lipide
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
EP1476492A1 (en) 2002-02-22 2004-11-17 Insert Therapeutics Inc. Carbohydrate-modified polymers, compositions and uses related thereto
US7037520B2 (en) 2002-03-22 2006-05-02 Baylor College Of Medicine Reversible masking of liposomal complexes for targeted delivery
WO2003083443A2 (en) 2002-03-29 2003-10-09 University Of Florida Lipid mediated screening of drug candidates for identification of active compounds
US8153141B2 (en) 2002-04-04 2012-04-10 Coley Pharmaceutical Gmbh Immunostimulatory G, U-containing oligoribonucleotides
WO2003097805A2 (en) 2002-05-15 2003-11-27 California Pacific Medical Center Delivery of nucleic acid-like compounds
ES2328031T3 (es) 2002-05-24 2009-11-06 F. Hoffmann-La Roche Ag Composiciones para distribuir acidos nucleicos a celulas.
WO2003103586A2 (en) * 2002-06-05 2003-12-18 Coley Pharmaceutical Group, Inc. Method for treating autoimmune or inflammatory diseases with combinations of inhibitory oligonucleotides and small molecule antagonists of immunostimulatory cpg nucleic acids
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
CA2500224C (en) * 2002-09-25 2015-04-28 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US20040214198A1 (en) 2002-11-15 2004-10-28 University Of Massachusetts Allele-targeted RNA interference
DE602004028587D1 (de) 2003-01-16 2010-09-23 Univ Pennsylvania ZUSAMMENSETZUNGEN UND VERFAHREN ZUR siRNA-HEMMUNG VON ICAM-1
US7682626B2 (en) 2003-02-07 2010-03-23 Roche Madison Inc. Polyvinylethers for delivery of polynucleotides to mammalian cells
DE602004020187D1 (de) 2003-02-20 2009-05-07 Constant Res & Dev Ltd Prozess zur Herstellung einer Dexamethason enthaltenden Formulierung zur oralen Anwendung
US20040167090A1 (en) 2003-02-21 2004-08-26 Monahan Sean D. Covalent modification of RNA for in vitro and in vivo delivery
US7803781B2 (en) * 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
WO2004090105A2 (en) * 2003-04-02 2004-10-21 Dharmacon, Inc. Modified polynucleotides for use in rna interference
US20040198640A1 (en) * 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
AU2004227414A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals iRNA conjugates
CA2521464C (en) 2003-04-09 2013-02-05 Alnylam Pharmaceuticals, Inc. Irna conjugates
DK1620544T3 (en) 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
MXPA05013746A (es) 2003-06-18 2006-06-27 Yissum Res Dev Co Conjugados de esfingoide-polialquilamina para vacunacion.
BRPI0411514A (pt) * 2003-06-20 2006-08-01 Coley Pharm Gmbh antagonistas de receptor toll-like de molécula pequena
ES2559828T3 (es) * 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
US20050136437A1 (en) * 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
NZ592917A (en) * 2003-09-15 2012-12-21 Protiva Biotherapeutics Inc Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates
CA2536139A1 (en) * 2003-09-25 2005-04-07 Coley Pharmaceutical Group, Inc. Nucleic acid-lipophilic conjugates
WO2005045035A2 (en) * 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF NOGO AND NOGO RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20050239733A1 (en) * 2003-10-31 2005-10-27 Coley Pharmaceutical Gmbh Sequence requirements for inhibitory oligonucleotides
US20050100983A1 (en) * 2003-11-06 2005-05-12 Coley Pharmaceutical Gmbh Cell-free methods for identifying compounds that affect toll-like receptor 9 (TLR9) signaling
EP2123759B1 (en) 2004-02-06 2014-01-15 Thermo Fisher Scientific Biosciences Inc. Stabilized RNAS as transfection controls and silencing reagents
WO2005097993A2 (en) 2004-02-19 2005-10-20 Coley Pharmaceutical Group, Inc. Immunostimulatory viral rna oligonucleotides
EP1730281A2 (en) * 2004-04-02 2006-12-13 Coley Pharmaceutical Group, Inc. Immunostimulatory nucleic acids for inducing il-10 responses
US7755603B2 (en) * 2004-05-24 2010-07-13 Alps Electric Co., Ltd. Coordinate input device
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
US7799565B2 (en) * 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
WO2006002538A1 (en) 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory sirna molecules and uses therefor
WO2006007712A1 (en) * 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
US7528118B2 (en) * 2004-09-24 2009-05-05 Alnylam Pharmaceuticals, Inc. RNAi modulation of ApoB and uses thereof
CA2587411A1 (en) 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
EP1819365B1 (en) 2004-12-09 2014-07-02 Alnylam Pharmaceuticals Inc. Compositions and methods for inducing an immune response in a mammal and methods of avoiding an immune response to oligonucleotide agents such as short interfering RNAs
WO2006074546A1 (en) 2005-01-13 2006-07-20 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
WO2006113679A2 (en) * 2005-04-15 2006-10-26 Board Of Regents, The University Of Texas System Delivery of sirna by neutral lipid compositions
SG124419A1 (en) 2005-02-03 2006-08-30 Agency Science Tech & Res Polycations capable of forming complexes with nucleic acids
JP5042863B2 (ja) * 2005-02-14 2012-10-03 サーナ・セラピューティクス・インコーポレイテッド 生物学的に活性な分子をデリバリーするための脂質ナノ粒子系組成物および方法
WO2006102163A2 (en) 2005-03-17 2006-09-28 Invitrogen Corporation Transfection reagents for non-adherent suspension cells
EP3611266B1 (en) * 2005-08-23 2022-11-09 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
EP1764108A1 (en) 2005-09-14 2007-03-21 Gunther Hartmann Compositions comprising immunostimulatory RNA oligonucleotides and methods for producing said RNA oligonucleotides
WO2007048046A2 (en) * 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2007056861A1 (en) * 2005-11-18 2007-05-24 Protiva Biotherapeutics, Inc. Sirna silencing of influenza virus gene expression
CA2652349A1 (en) 2006-05-15 2007-11-22 University Of Kentucky Research Foundation Toll-like receptor (tlr) stimulation for ocular angiogenesis and macular degeneration
US7915399B2 (en) * 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2008019486A1 (en) 2006-08-16 2008-02-21 Protiva Biotherapeutics, Inc. Nucleic acid modulation of toll-like receptor-mediated immune stimulation
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
DE102008028754A1 (de) * 2008-06-17 2009-12-24 Bernd Hansen Vorrichtung zum Herstellen und Befüllen von Behältern
US20110071208A1 (en) 2009-06-05 2011-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated dicer-substrate interfering rna
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
US8236943B2 (en) 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
US20120136073A1 (en) 2010-11-15 2012-05-31 Life Technologies Corporation Amine-Containing Transfection Reagents and methods for making and using same

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