JP2009536827A5 - - Google Patents

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JP2009536827A5
JP2009536827A5 JP2009510173A JP2009510173A JP2009536827A5 JP 2009536827 A5 JP2009536827 A5 JP 2009536827A5 JP 2009510173 A JP2009510173 A JP 2009510173A JP 2009510173 A JP2009510173 A JP 2009510173A JP 2009536827 A5 JP2009536827 A5 JP 2009536827A5
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dsrna
sequence
pcsk9
cell
expression
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JP2009510173A
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JP5570806B2 (ja
JP2009536827A (ja
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Priority claimed from PCT/US2007/068655 external-priority patent/WO2007134161A2/en
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JP2009510173A 2006-05-11 2007-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法 Expired - Fee Related JP5570806B2 (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US79945806P 2006-05-11 2006-05-11
US60/799,458 2006-05-11
US81720306P 2006-06-27 2006-06-27
US60/817,203 2006-06-27
US84008906P 2006-08-25 2006-08-25
US60/840,089 2006-08-25
US82991406P 2006-10-18 2006-10-18
US60/829,914 2006-10-18
US90113407P 2007-02-13 2007-02-13
US60/901,134 2007-02-13
PCT/US2007/068655 WO2007134161A2 (en) 2006-05-11 2007-05-10 Compositions and methods for inhibiting expression of the pcsk9 gene

Related Child Applications (2)

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JP2010108836A Division JP2010246544A (ja) 2006-05-11 2010-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2014018817A Division JP6034316B2 (ja) 2006-05-11 2014-02-03 Pcsk9遺伝子の発現を阻害するための組成物および方法

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JP2009536827A JP2009536827A (ja) 2009-10-22
JP2009536827A5 true JP2009536827A5 (enExample) 2010-07-01
JP5570806B2 JP5570806B2 (ja) 2014-08-13

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JP2009510173A Expired - Fee Related JP5570806B2 (ja) 2006-05-11 2007-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2010108836A Pending JP2010246544A (ja) 2006-05-11 2010-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2014018817A Expired - Fee Related JP6034316B2 (ja) 2006-05-11 2014-02-03 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2015226712A Pending JP2016027830A (ja) 2006-05-11 2015-11-19 Pcsk9遺伝子の発現を阻害するための組成物および方法

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JP2010108836A Pending JP2010246544A (ja) 2006-05-11 2010-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2014018817A Expired - Fee Related JP6034316B2 (ja) 2006-05-11 2014-02-03 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2015226712A Pending JP2016027830A (ja) 2006-05-11 2015-11-19 Pcsk9遺伝子の発現を阻害するための組成物および方法

Country Status (14)

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US (10) US7605251B2 (enExample)
EP (8) EP2194128B1 (enExample)
JP (4) JP5570806B2 (enExample)
KR (2) KR101320916B1 (enExample)
CN (2) CN103614375A (enExample)
AU (2) AU2007249329C1 (enExample)
CA (2) CA2915441A1 (enExample)
EA (2) EA015676B1 (enExample)
ES (2) ES2392478T3 (enExample)
IL (3) IL195181A (enExample)
NZ (2) NZ587616A (enExample)
PL (2) PL2194128T3 (enExample)
SG (1) SG171676A1 (enExample)
WO (1) WO2007134161A2 (enExample)

Families Citing this family (172)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070173473A1 (en) * 2001-05-18 2007-07-26 Sirna Therapeutics, Inc. RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
KR101531400B1 (ko) 2003-06-27 2015-06-26 암젠 프레몬트 인코포레이티드 상피 성장 인자 수용체의 결실 돌연변이체 지향 항체 및 그용도
EP1976567B1 (en) 2005-12-28 2020-05-13 The Scripps Research Institute Natural antisense and non-coding rna transcripts as drug targets
SG171676A1 (en) 2006-05-11 2011-06-29 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of the pcsk9 gene
JP5588175B2 (ja) 2006-11-07 2014-09-10 メルク・シャープ・アンド・ドーム・コーポレーション Pcsk9のアンタゴニスト
DK2094086T3 (da) * 2006-11-08 2013-11-25 Veritas Bio LLC Indgivelse in vivo af dobbeltstrenget rna til en målcelle
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
JP2010510807A (ja) * 2006-11-27 2010-04-08 アイシス ファーマシューティカルズ, インコーポレーテッド 高コレステロール血症を治療するための方法
US7910722B2 (en) 2007-07-05 2011-03-22 Florida State University Research Foundation RNAi therapeutic for treatment of hepatitis C infection
JOP20080381B1 (ar) 2007-08-23 2023-03-28 Amgen Inc بروتينات مرتبطة بمولدات مضادات تتفاعل مع بروبروتين كونفيرتاز سيتيليزين ككسين من النوع 9 (pcsk9)
EP2245039A4 (en) * 2008-01-31 2012-06-06 Alnylam Pharmaceuticals Inc OPTIMIZED METHODS FOR EXPORING DSRNA TO TARGET THE PCSK9 GEN
AR070316A1 (es) 2008-02-07 2010-03-31 Merck & Co Inc Antagonistas de pcsk9 (proproteina subtilisina-kexina tipo 9)
AR070315A1 (es) 2008-02-07 2010-03-31 Merck & Co Inc Anticuerpos 1b20 antagonistas de pcsk9
EP3643782A1 (en) 2008-02-11 2020-04-29 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
WO2009116037A2 (en) * 2008-03-20 2009-09-24 Quark Pharmaceuticals, Inc. NOVEL siRNA COMPOUNDS FOR INHIBITING RTP801
CN108165548B (zh) 2008-09-22 2022-10-14 菲奥医药公司 减小大小的自递送RNAi化合物
US8153606B2 (en) 2008-10-03 2012-04-10 Opko Curna, Llc Treatment of apolipoprotein-A1 related diseases by inhibition of natural antisense transcript to apolipoprotein-A1
CN102317458B (zh) 2008-12-04 2018-01-02 库尔纳公司 通过红细胞生成素(epo)天然反义转录物的抑制对epo相关疾病的治疗
CA2746003C (en) 2008-12-04 2020-03-31 Opko Curna, Llc Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf
EP2370582B1 (en) 2008-12-04 2017-05-10 CuRNA, Inc. Treatment of tumor suppressor gene related diseases by inhibition of natural antisense transcript to the gene
JO3672B1 (ar) 2008-12-15 2020-08-27 Regeneron Pharma أجسام مضادة بشرية عالية التفاعل الكيماوي بالنسبة لإنزيم سبتيليسين كنفرتيز بروبروتين / كيكسين نوع 9 (pcsk9).
WO2010078536A1 (en) * 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
PT2396038E (pt) 2009-02-12 2016-02-19 Curna Inc Tratamento das doenças associadas com o factor neurotrófico derivado do cérebro (bdnf) por inibição do produto antisenso natural da transcrição para bdnf
CA2755409C (en) 2009-03-16 2019-04-30 Joseph Collard Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
JP5904935B2 (ja) 2009-03-17 2016-04-20 クルナ・インコーポレーテッド デルタ様1ホモログ(dlk1)に対する天然アンチセンス転写物の抑制によるdlk1関連疾患の治療
EP2427553A4 (en) 2009-05-06 2012-11-07 Opko Curna Llc TREATMENT OF LIPID TRANSPORT AND METABOLISM-RELATED DISEASES BY INHIBITING THE NATURAL ANTISENSE TRANSCRIPT AGAINST A LIPID TRANSPORT AND METABOLIC TREATMENT
JP6250930B2 (ja) 2009-05-06 2017-12-20 クルナ・インコーポレーテッド トリステトラプロリン(ttp)に対する天然アンチセンス転写物の抑制によるttp関連疾患の治療
CN102459597B (zh) 2009-05-08 2020-06-30 库尔纳公司 通过针对dmd家族的天然反义转录物的抑制治疗肌营养蛋白家族相关疾病
ES2664590T3 (es) 2009-05-18 2018-04-20 Curna, Inc. Tratamiento de enfermedades relacionadas con factores de reprogramación por inhibición del tránscrito antisentido natural a un factor de reprogramación
CN102549158B (zh) 2009-05-22 2017-09-26 库尔纳公司 通过抑制针对转录因子e3(tfe3)的天然反义转录物来治疗tfe3和胰岛素受体底物蛋白2(irs2)相关的疾病
KR101704988B1 (ko) 2009-05-28 2017-02-08 큐알엔에이, 인크. 항바이러스 유전자에 대한 천연 안티센스 전사체의 억제에 의한 항바이러스 유전자 관련된 질환의 치료
WO2010141511A2 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
WO2010147992A1 (en) * 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
EP2442792A4 (en) * 2009-06-15 2015-12-23 Alnylam Pharmaceuticals Inc TESTING GENE PCSK9 LIPID-FORMULATED DSRNA
CN102695797B (zh) 2009-06-16 2018-05-25 库尔纳公司 通过抑制针对胶原基因的天然反义转录物来治疗胶原基因相关的疾病
ES2629339T3 (es) 2009-06-16 2017-08-08 Curna, Inc. Tratamiento de enfermedades relacionadas con la paraoxonasa 1 (pon1) por inhibición de transcrito antisentido natural a pon1
ES2618894T3 (es) 2009-06-24 2017-06-22 Curna, Inc. Tratamiento de enfermedades relacionadas con el receptor del factor de necrosis tumoral 2 (tnfr2) por inhibición del transcrito natural antisentido para tnfr2
KR101807324B1 (ko) 2009-06-26 2017-12-08 큐알엔에이, 인크. 다운 증후군 유전자에 대한 천연 안티센스 전사체의 억제에 의한 다운 증후군 유전자 관련된 질환의 치료
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
WO2011009697A1 (en) 2009-07-21 2011-01-27 Santaris Pharma A/S Antisense oligomers targeting pcsk9
WO2011011700A2 (en) 2009-07-24 2011-01-27 Curna, Inc. Treatment of sirtuin (sirt) related diseases by inhibition of natural antisense transcript to a sirtuin (sirt)
KR101802536B1 (ko) 2009-08-05 2017-11-28 큐알엔에이, 인크. 인슐린 유전자 (ins)에 대한 자연 안티센스 전사체의 저해에 의한 인슐린 유전자 (ins) 관련된 질환의 치료
WO2011019815A2 (en) 2009-08-11 2011-02-17 Curna, Inc. Treatment of adiponectin (adipoq) related diseases by inhibition of natural antisense transcript to an adiponectin (adipoq)
US8791087B2 (en) 2009-08-21 2014-07-29 Curna, Inc. Treatment of ‘C terminus of HSP70-interacting protein’ (CHIP)related diseases by inhibition of natural antisense transcript to CHIP
CN102482671B (zh) 2009-08-25 2017-12-01 库尔纳公司 通过抑制‘含有iq模体的gtp酶激活蛋白’(iqgap)的天然反义转录物而治疗iqgap相关疾病
WO2011028938A1 (en) * 2009-09-02 2011-03-10 Alnylam Pharmaceuticals, Inc. Methods for lowering serum cholestrol in a subject using inhibition of pcsk9
WO2011038031A1 (en) * 2009-09-22 2011-03-31 Alnylam Pharmaceuticals, Inc. Dual targeting sirna agents
CN102791861B (zh) 2009-09-25 2018-08-07 库尔纳公司 通过调节聚丝蛋白(flg)的表达和活性而治疗flg相关疾病
EP3252068B1 (en) 2009-10-12 2025-07-02 Larry J. Smith Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
AR079336A1 (es) * 2009-12-11 2012-01-18 Irm Llc Antagonistas de la pro-proteina convertasa-subtilisina/quexina tipo 9 (pcsk9)
KR101823702B1 (ko) 2009-12-16 2018-01-30 큐알엔에이, 인크. 막 결합 전사 인자 펩티다제, 부위 1(mbtps1)에 대한 천연 안티센스 전사체의 억제에 의한 mbtps1 관련 질환의 치료
US20110152343A1 (en) * 2009-12-22 2011-06-23 Functional Genetics, Inc. Protease inhibitors and broad-spectrum antiviral
KR101793753B1 (ko) 2009-12-23 2017-11-03 큐알엔에이, 인크. 커플링방지 단백질 2(ucp2)에 대한 천연 안티센스 전사체의 저해에 의한 ucp2 관련 질환의 치료
DK2516648T3 (en) 2009-12-23 2018-02-12 Curna Inc TREATMENT OF HEPATOCYTE GROWTH FACTOR (HGF) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT AGAINST HGF
CN102770540B (zh) 2009-12-29 2017-06-23 库尔纳公司 通过抑制肿瘤蛋白63(p63)的天然反义转录物而治疗p63相关疾病
ES2657452T3 (es) 2009-12-29 2018-03-05 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor respiratorio nuclear 1 (NRF1) mediante inhibición de transcrito antisentido natural a NRF1
RU2016115782A (ru) 2009-12-31 2018-11-28 Курна, Инк. Лечение заболеваний, связанных с субстратом рецептора инсулина 2 (irs2), путем ингибирования природного антисмыслового транскрипта к irs2 и транскрипционному фактору е3 (tfe3)
DK2521784T3 (en) 2010-01-04 2018-03-12 Curna Inc TREATMENT OF INTERFERON REGULATORY FACTOR 8- (IRF8) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPT TO IRF8
WO2011085066A2 (en) 2010-01-06 2011-07-14 Curna, Inc. Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene
CA2786535C (en) 2010-01-11 2019-03-26 Curna, Inc. Treatment of sex hormone binding globulin (shbg) related diseases by inhibition of natural antisense transcript to shbg
US8946182B2 (en) 2010-01-25 2015-02-03 Curna, Inc. Treatment of RNASE H1 related diseases by inhibition of natural antisense transcript to RNASE H1
JP5976548B2 (ja) 2010-02-22 2016-08-23 カッパーアールエヌエー,インコーポレイテッド Pycr1に対する天然アンチセンス転写物の阻害によるピロリン−5−カルボン酸レダクターゼ1(pycr1)関連疾患の治療
TWI600759B (zh) 2010-04-02 2017-10-01 可娜公司 藉由抑制群落刺激因子3(csf3)之天然反義轉錄物以治療csf3相關疾病
US9044494B2 (en) 2010-04-09 2015-06-02 Curna, Inc. Treatment of fibroblast growth factor 21 (FGF21) related diseases by inhibition of natural antisense transcript to FGF21
US9089588B2 (en) 2010-05-03 2015-07-28 Curna, Inc. Treatment of sirtuin (SIRT) related diseases by inhibition of natural antisense transcript to a sirtuin (SIRT)
TWI531370B (zh) 2010-05-14 2016-05-01 可娜公司 藉由抑制par4天然反股轉錄本治療par4相關疾病
EP3299464B1 (en) 2010-05-26 2019-10-02 CuRNA, Inc. Treatment of atonal homolog 1 (atoh1) related diseases by inhibition of natural antisense transcript to atoh1
EP2576784B1 (en) 2010-05-26 2017-11-15 CuRNA, Inc. Treatment of methionine sulfoxide reductase a (msra) related diseases by inhibition of natural antisense transcript to msra
JP6023705B2 (ja) 2010-06-23 2016-11-09 カッパーアールエヌエー,インコーポレイテッド ナトリウムチャネル、電位依存性、αサブユニット(SCNA)に対する天然アンチセンス転写物の阻害によるSCNA関連疾患の治療
CN103068982B (zh) 2010-07-14 2017-06-09 库尔纳公司 通过抑制盘状大同系物(dlg)的天然反义转录物而治疗dlg相关疾病
US8993533B2 (en) 2010-10-06 2015-03-31 Curna, Inc. Treatment of sialidase 4 (NEU4) related diseases by inhibition of natural antisense transcript to NEU4
WO2012054723A2 (en) 2010-10-22 2012-04-26 Opko Curna Llc Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
WO2012058693A2 (en) * 2010-10-29 2012-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of pcsk9 genes
WO2012068340A2 (en) 2010-11-18 2012-05-24 Opko Curna Llc Antagonat compositions and methods of use
EP2643463B1 (en) 2010-11-23 2017-09-27 CuRNA, Inc. Treatment of nanog related diseases by inhibition of natural antisense transcript to nanog
HRP20180959T1 (hr) 2011-01-28 2018-07-27 Sanofi Biotechnology Ljudska protutijela za pcsk9 za uporabu u postupcima liječenja određenih skupina subjekata
EP2699271A4 (en) 2011-04-20 2015-10-07 Larry J Smith METHODS AND COMPOSITIONS FOR MODULATING GENE EXPRESSION USING COMPONENTS THAT SELF-ASSEMBLED IN CELLS AND PRODUCE RNAI ACTIVITY
EP2718439B1 (en) 2011-06-09 2017-08-09 CuRNA, Inc. Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn
AR087305A1 (es) 2011-07-28 2014-03-12 Regeneron Pharma Formulaciones estabilizadas que contienen anticuerpos anti-pcsk9, metodo de preparacion y kit
EP2753317B1 (en) 2011-09-06 2020-02-26 CuRNA, Inc. TREATMENT OF DISEASES RELATED TO ALPHA SUBUNITS OF SODIUM CHANNELS, VOLTAGE-GATED (SCNxA) WITH SMALL MOLECULES
RS54639B1 (sr) * 2011-09-13 2016-08-31 Affiris Ag Pcsk9 vakcina
WO2013126803A1 (en) 2012-02-24 2013-08-29 Protiva Biotherapeutics Inc. Trialkyl cationic lipids and methods of use thereof
HK1210211A1 (en) 2012-03-15 2016-04-15 科纳公司 Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
HUE035887T2 (en) 2012-12-05 2018-05-28 Alnylam Pharmaceuticals Inc PCSK9 iRNA preparations and methods for their use
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US10246708B2 (en) 2013-05-06 2019-04-02 Alnylam Pharmaceuticals, Inc. Dosages and methods for delivering lipid formulated nucleic acid molecules
EP2810955A1 (en) 2013-06-07 2014-12-10 Sanofi Methods for inhibiting atherosclerosis by administering an inhibitor of PCSK9
EP2862877A1 (en) 2013-10-18 2015-04-22 Sanofi Methods for inhibiting atherosclerosis by administering an inhibitor of PCSK9
CN111920954A (zh) 2013-06-07 2020-11-13 再生元制药公司 通过施用pcsk9抑制剂抑制动脉粥样硬化的方法
JP6738729B2 (ja) * 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
DK3013959T3 (da) * 2013-06-27 2020-02-17 Roche Innovation Ct Copenhagen As Antisense-oligomerer og konjugater målrettet pcsk9
BR112016013201B1 (pt) 2013-12-12 2023-01-31 The Broad Institute, Inc. Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
SG10201804973TA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
EP3540051B1 (en) * 2013-12-12 2022-08-17 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders.
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
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
US9045548B1 (en) 2014-07-15 2015-06-02 Kymab Limited Precision Medicine by targeting rare human PCSK9 variants for cholesterol treatment
US9017678B1 (en) 2014-07-15 2015-04-28 Kymab Limited Method of treating rheumatoid arthritis using antibody to IL6R
US8883157B1 (en) 2013-12-17 2014-11-11 Kymab Limited Targeting rare human PCSK9 variants for cholesterol treatment
US8992927B1 (en) 2014-07-15 2015-03-31 Kymab Limited Targeting human NAV1.7 variants for treatment of pain
US8986694B1 (en) 2014-07-15 2015-03-24 Kymab Limited Targeting human nav1.7 variants for treatment of pain
US8980273B1 (en) 2014-07-15 2015-03-17 Kymab Limited Method of treating atopic dermatitis or asthma using antibody to IL4RA
US9051378B1 (en) 2014-07-15 2015-06-09 Kymab Limited Targeting rare human PCSK9 variants for cholesterol treatment
US9045545B1 (en) 2014-07-15 2015-06-02 Kymab Limited Precision medicine by targeting PD-L1 variants for treatment of cancer
US8945560B1 (en) 2014-07-15 2015-02-03 Kymab Limited Method of treating rheumatoid arthritis using antibody to IL6R
US9023359B1 (en) 2014-07-15 2015-05-05 Kymab Limited Targeting rare human PCSK9 variants for cholesterol treatment
US9067998B1 (en) 2014-07-15 2015-06-30 Kymab Limited Targeting PD-1 variants for treatment of cancer
US8986691B1 (en) 2014-07-15 2015-03-24 Kymab Limited Method of treating atopic dermatitis or asthma using antibody to IL4RA
US9914769B2 (en) 2014-07-15 2018-03-13 Kymab Limited Precision medicine for cholesterol treatment
US9034332B1 (en) 2014-07-15 2015-05-19 Kymab Limited Precision medicine by targeting rare human PCSK9 variants for cholesterol treatment
KR102357337B1 (ko) * 2013-12-27 2022-01-28 가부시키가이샤 보낙 유전자 발현 제어를 위한 인공 매치형 miRNA 및 그 용도
GB201403775D0 (en) 2014-03-04 2014-04-16 Kymab Ltd Antibodies, uses & methods
US9139648B1 (en) 2014-07-15 2015-09-22 Kymab Limited Precision medicine by targeting human NAV1.9 variants for treatment of pain
US9150660B1 (en) 2014-07-15 2015-10-06 Kymab Limited Precision Medicine by targeting human NAV1.8 variants for treatment of pain
KR20170029613A (ko) 2014-07-16 2017-03-15 사노피 바이오테크놀로지 이형접합성 가족성 고콜레스테롤혈증(heFH) 환자의 치료방법
JOP20200115A1 (ar) * 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3237615B2 (en) 2014-12-24 2023-07-26 The Broad Institute, Inc. Crispr having or associated with destabilization domains
US20180002702A1 (en) 2014-12-26 2018-01-04 Nitto Denko Corporation Methods and compositions for treating malignant tumors associated with kras mutation
US10792299B2 (en) 2014-12-26 2020-10-06 Nitto Denko Corporation Methods and compositions for treating malignant tumors associated with kras mutation
US10264976B2 (en) * 2014-12-26 2019-04-23 The University Of Akron Biocompatible flavonoid compounds for organelle and cell imaging
US11045488B2 (en) 2014-12-26 2021-06-29 Nitto Denko Corporation RNA interference agents for GST-π gene modulation
ES2789049T3 (es) * 2014-12-26 2020-10-23 Nitto Denko Corp Agentes de interferencia de ARN para modulación génica de GST-pi
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
CN109536474A (zh) 2015-06-18 2019-03-29 布罗德研究所有限公司 降低脱靶效应的crispr酶突变
IL314925A (en) 2015-08-18 2024-10-01 Regeneron Pharma Antibodies against PCSK9 for the treatment of patients with hyperlipidemia undergoing lipoprotein-lowering therapy
WO2017035278A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
CN108348541A (zh) 2015-08-25 2018-07-31 阿尔尼拉姆医药品有限公司 用于治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物
US11021707B2 (en) 2015-10-19 2021-06-01 Phio Pharmaceuticals Corp. Reduced size self-delivering nucleic acid compounds targeting long non-coding RNA
BR112018076260A2 (pt) 2016-06-20 2019-03-26 Kymab Limited anticorpo ou fragmento do mesmo que se liga especificamente a hpd-l1, anticorpo biespecífico ou proteína de fusão, uso de um anticorpo ou fragmento, método, composição farmacêutica, método de modulação, método de inibição, método de tratamento, ácido nucleico, vetor, hospedeiro e imunocitocina
HUE067712T2 (hu) * 2016-10-18 2024-11-28 Novartis Ag Eljárások kardiovaszkuláris események megelõzésére 9-es típusú szubtilizin/kexin proprotein-konvertáz (PCSK9) fehérje csökkentésén keresztül
US11779604B2 (en) 2016-11-03 2023-10-10 Kymab Limited Antibodies, combinations comprising antibodies, biomarkers, uses and methods
JOP20190112A1 (ar) 2016-11-14 2019-05-14 Amgen Inc علاجات مدمجة لتصلب الشرايين، شاملة مرض قلبي وعائي تصلبي
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
CN108265052B (zh) * 2016-12-30 2021-05-28 苏州瑞博生物技术股份有限公司 一种小干扰核酸和药物组合物及其用途
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
JOP20190215A1 (ar) * 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
WO2019105418A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
US11414665B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
JP7365052B2 (ja) 2017-12-01 2023-10-19 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
KR102834361B1 (ko) 2017-12-01 2025-07-17 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
WO2019126990A1 (zh) * 2017-12-26 2019-07-04 广州市锐博生物科技有限公司 一种抑制PCSK9基因表达的siRNA分子及其应用
CN109957567B (zh) * 2017-12-26 2022-09-23 阿格纳生物制药有限公司 一种抑制PCSK9基因表达的siRNA分子及其应用
CN109957565B (zh) * 2017-12-26 2023-04-07 广州市锐博生物科技有限公司 一种修饰的siRNA分子及其应用
CA3087106A1 (en) 2017-12-29 2019-07-04 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
CA3097585A1 (en) * 2018-04-18 2019-10-24 Dicerna Pharmaceuticals, Inc. Pcsk9 targeting oligonucleotides for treating hypercholesterolemia and related conditions
JP2021533800A (ja) 2018-08-21 2021-12-09 スーチョウ リボ ライフ サイエンス カンパニー、リミテッドSuzhou Ribo Life Science Co., Ltd. 核酸、当該核酸を含む薬物組成物及び複合体ならびにその使用
CN111655297A (zh) 2018-09-30 2020-09-11 苏州瑞博生物技术有限公司 一种siRNA缀合物及其制备方法和用途
CN111378656B (zh) * 2018-12-28 2022-07-26 苏州瑞博生物技术股份有限公司 一种抑制埃博拉病毒的核酸、含有该核酸的药物组合物及其用途
CN114173762A (zh) * 2019-06-04 2022-03-11 塔夫茨大学信托人 用于mRNA递送的合成脂质
EP4022061A1 (en) * 2019-08-27 2022-07-06 Sanofi Compositions and methods for inhibiting pcsk9
US12378560B2 (en) 2019-10-29 2025-08-05 Northwestern University Sequence multiplicity within spherical nucleic acids
WO2021185765A1 (en) * 2020-03-16 2021-09-23 Argonaute RNA Limited Antagonist of pcsk9
WO2022089486A1 (zh) * 2020-10-28 2022-05-05 江苏柯菲平医药股份有限公司 抑制PCSK9基因表达的siRNA及其修饰物与应用
WO2023017004A1 (en) * 2021-08-09 2023-02-16 Cargene Therapeutics Pte. Ltd. Inhibitory nucleic acids for pcsk9
CN113980966B (zh) * 2021-09-29 2024-09-20 阿格纳生物制药有限公司 一种抑制pcsk9靶基因表达的核酸序列及其应用
WO2024222898A1 (zh) * 2023-04-28 2024-10-31 北京福元医药股份有限公司 用于抑制marc1基因表达的双链核糖核酸及其修饰物、缀合物和用途
WO2025006782A2 (en) * 2023-06-30 2025-01-02 Chroma Medicine, Inc. Guide rna compositions
CN118147134B (zh) * 2023-11-06 2025-02-25 北京悦康科创医药科技股份有限公司 靶向调控PCSK9基因表达的siRNA及其应用
CN117384907B (zh) * 2023-12-11 2024-03-29 上海鼎新基因科技有限公司 抑制PCSK9表达的siRNA分子及其应用

Family Cites Families (133)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
WO1987000201A1 (en) 1985-07-05 1987-01-15 Whitehead Institute For Biomedical Research Epithelial cells expressing foreign genetic material
US4980286A (en) 1985-07-05 1990-12-25 Whitehead Institute For Biomedical Research In vivo introduction and expression of foreign genetic material in epithelial cells
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
EP0633318A1 (en) 1987-09-11 1995-01-11 Whitehead Institute For Biomedical Research Transduced fibroblasts and uses therefor
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
DE3851153T2 (de) 1987-12-11 1995-01-05 Hughes Howard Med Inst Genetische modifizierung von endothelialen zellen.
EP0400047B1 (en) 1988-02-05 1997-04-23 Whitehead Institute For Biomedical Research Modified hepatocytes and uses therefor
WO1989009221A1 (en) 1988-03-25 1989-10-05 University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5223168A (en) 1989-12-12 1993-06-29 Gary Holt Surface cleaner and treatment
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5506351A (en) 1992-07-23 1996-04-09 Isis Pharmaceuticals Process for the preparation of 2'-O-alkyl guanosine and related compounds
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5212295A (en) 1990-01-11 1993-05-18 Isis Pharmaceuticals Monomers for preparation of oligonucleotides having chiral phosphorus linkages
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
KR100211552B1 (ko) 1990-08-03 1999-08-02 디. 꼬쉬 유전자 발현 억제용 화합물 및 방법
US6262241B1 (en) 1990-08-13 2001-07-17 Isis Pharmaceuticals, Inc. Compound for detecting and modulating RNA activity and gene expression
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
JPH06505704A (ja) 1990-09-20 1994-06-30 ギリアド サイエンシズ,インコーポレイテッド 改変ヌクレオシド間結合
JP3418982B2 (ja) 1990-10-31 2003-06-23 ソマティクス セラピー コーポレイション 内皮細胞の遺伝的変性
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
AU2369192A (en) 1991-07-25 1993-02-23 Whitaker Corporation, The Liquid level sensor
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
US5599797A (en) 1991-10-15 1997-02-04 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
ATE239484T1 (de) 1991-10-24 2003-05-15 Isis Pharmaceuticals Inc Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
WO1994022891A1 (en) 1993-03-31 1994-10-13 Sterling Winthrop Inc. Oligonucleotides with amide linkages replacing phosphodiester linkages
US5571902A (en) 1993-07-29 1996-11-05 Isis Pharmaceuticals, Inc. Synthesis of oligonucleotides
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US6054299A (en) 1994-04-29 2000-04-25 Conrad; Charles A. Stem-loop cloning vector and method
US5554746A (en) 1994-05-16 1996-09-10 Isis Pharmaceuticals, Inc. Lactam nucleic acids
DE69634084T2 (de) 1995-06-07 2005-12-08 Inex Pharmaceuticals Corp. Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
EP1068311B2 (en) 1998-04-08 2020-12-09 Commonwealth Scientific and Industrial Research Organisation Methods and means for obtaining modified phenotypes
AR020078A1 (es) 1998-05-26 2002-04-10 Syngenta Participations Ag Metodo para alterar la expresion de un gen objetivo en una celula de planta
AU6298899A (en) 1998-10-09 2000-05-01 Ingene, Inc. Production of ssdna (in vivo)
KR20010099682A (ko) 1998-10-09 2001-11-09 추후보충 단일가닥 dna의 효소 합성
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US6271359B1 (en) 1999-04-14 2001-08-07 Musc Foundation For Research Development Tissue-specific and pathogen-specific toxic agents and ribozymes
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
EP1261620A2 (en) 2000-02-07 2002-12-04 Roche Diagnostics Corporation Cationic amphiphiles for use in nucleic acid transfection
US20070026394A1 (en) 2000-02-11 2007-02-01 Lawrence Blatt 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
RU2322500C2 (ru) 2000-12-01 2008-04-20 Макс-Планк-Гезелльшафт Цур Фердерунг Дер Виссеншафтен Е.Ф. Малые молекулы рнк, опосредующие интерференцию рнк
US20070173473A1 (en) 2001-05-18 2007-07-26 Sirna Therapeutics, Inc. RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
US20080249040A1 (en) 2001-05-18 2008-10-09 Sirna Therapeutics, Inc. RNA interference mediated inhibition of sterol regulatory element binding protein 1 (SREBP1) gene expression using short interfering nucleic acid (siNA)
US20030170891A1 (en) 2001-06-06 2003-09-11 Mcswiggen James A. RNA interference mediated inhibition of epidermal growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20040009216A1 (en) 2002-04-05 2004-01-15 Rodrigueza Wendi V. Compositions and methods for dosing liposomes of certain sizes to treat or prevent disease
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
DK2284266T3 (da) * 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
DE10302421A1 (de) 2003-01-21 2004-07-29 Ribopharma Ag Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit
EP3450559A1 (en) 2003-03-07 2019-03-06 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
EP1608735A4 (en) 2003-04-03 2008-11-05 Alnylam Pharmaceuticals RNAI CONJUGATES
EP1471152A1 (en) * 2003-04-25 2004-10-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Mutations in the human PCSK9 gene associated to hypercholesterolemia
EP2336317B1 (en) * 2003-06-13 2019-09-11 Alnylam Europe AG Double-stranded ribonucleic acid with increased effectiveness in an organism
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
WO2005040379A2 (en) * 2003-10-23 2005-05-06 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF RAS GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
EP1778298A4 (en) 2004-04-01 2010-03-31 Univ Pennsylvania LIPOPROTEIN-NANO PLATFORMS
JP4764426B2 (ja) 2004-06-07 2011-09-07 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 カチオン性脂質および使用方法
AU2005252273B2 (en) 2004-06-07 2011-04-28 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
WO2007086883A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
US7427605B2 (en) 2005-03-31 2008-09-23 Calando Pharmaceuticals, Inc. Inhibitors of ribonucleotide reductase subunit 2 and uses thereof
US7915230B2 (en) 2005-05-17 2011-03-29 Molecular Transfer, Inc. Reagents for transfection of eukaryotic cells
JP5639338B2 (ja) 2005-07-27 2014-12-10 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 リポソームの製造システムおよび製造方法
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
US20070218122A1 (en) 2005-11-18 2007-09-20 Protiva Biotherapeutics, Inc. siRNA silencing of influenza virus gene expression
WO2007115168A2 (en) 2006-03-31 2007-10-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of eg5 gene
SG171676A1 (en) 2006-05-11 2011-06-29 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of the pcsk9 gene
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
AU2007275365A1 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. RNA interference mediated inhibition of Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
JP2010510807A (ja) 2006-11-27 2010-04-08 アイシス ファーマシューティカルズ, インコーポレーテッド 高コレステロール血症を治療するための方法
WO2008109369A2 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tnf gene expression and uses thereof
JOP20080381B1 (ar) 2007-08-23 2023-03-28 Amgen Inc بروتينات مرتبطة بمولدات مضادات تتفاعل مع بروبروتين كونفيرتاز سيتيليزين ككسين من النوع 9 (pcsk9)
CA2710713C (en) 2007-12-27 2017-09-19 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
US20110117125A1 (en) 2008-01-02 2011-05-19 Tekmira Pharmaceuticals Corporation Compositions and methods for the delivery of nucleic acids
EP2245039A4 (en) 2008-01-31 2012-06-06 Alnylam Pharmaceuticals Inc OPTIMIZED METHODS FOR EXPORING DSRNA TO TARGET THE PCSK9 GEN
KR101397407B1 (ko) 2008-03-05 2014-06-19 알닐람 파마슈티칼스 인코포레이티드 Eg5 및 VEGF 유전자의 발현을 억제하기 위한 조성물 및 방법
US20110065644A1 (en) 2008-03-09 2011-03-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
US8058069B2 (en) 2008-04-15 2011-11-15 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
HRP20150796T1 (hr) 2008-10-20 2015-09-11 Alnylam Pharmaceuticals, Inc. Pripravci i postupci za inhibiciju ekspresije transtiretina
CN105152939A (zh) 2008-11-10 2015-12-16 阿尔尼拉姆医药品有限公司 用于递送治疗剂的脂质和组合物
CA2746514C (en) 2008-12-10 2018-11-27 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
US8311190B2 (en) * 2008-12-23 2012-11-13 International Business Machines Corporation Performing human client verification over a voice interface
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
KR20210031549A (ko) 2009-05-05 2021-03-19 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
KR20230098713A (ko) 2009-06-10 2023-07-04 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
EP2442792A4 (en) 2009-06-15 2015-12-23 Alnylam Pharmaceuticals Inc TESTING GENE PCSK9 LIPID-FORMULATED DSRNA
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
WO2011009697A1 (en) 2009-07-21 2011-01-27 Santaris Pharma A/S Antisense oligomers targeting pcsk9

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