AU2009213147A1 - Modified RNAi polynucleotides and uses thereof - Google Patents

Modified RNAi polynucleotides and uses thereof Download PDF

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Publication number
AU2009213147A1
AU2009213147A1 AU2009213147A AU2009213147A AU2009213147A1 AU 2009213147 A1 AU2009213147 A1 AU 2009213147A1 AU 2009213147 A AU2009213147 A AU 2009213147A AU 2009213147 A AU2009213147 A AU 2009213147A AU 2009213147 A1 AU2009213147 A1 AU 2009213147A1
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Australia
Prior art keywords
dsrna
antisense
sense
nucleotides
strand
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Abandoned
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AU2009213147A
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English (en)
Inventor
Joanne Kamens
Anastasia Khvorova
Pamela A. Pavco
William Salomon
Tod M. Woolf
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Phio Pharmaceuticals Corp
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RXi Pharmaceuticals Corp
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Publication of AU2009213147A1 publication Critical patent/AU2009213147A1/en
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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AU2009213147A 2008-02-11 2009-02-11 Modified RNAi polynucleotides and uses thereof Abandoned AU2009213147A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US6533508P 2008-02-11 2008-02-11
US61/065,335 2008-02-11
PCT/US2009/000852 WO2009102427A2 (en) 2008-02-11 2009-02-11 Modified rnai polynucleotides and uses thereof

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AU2009213147A1 true AU2009213147A1 (en) 2009-08-20

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AU2009213147A Abandoned AU2009213147A1 (en) 2008-02-11 2009-02-11 Modified RNAi polynucleotides and uses thereof

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US (2) US10131904B2 (https=)
EP (2) EP2247729B1 (https=)
JP (3) JP5697993B2 (https=)
CN (2) CN104975020B (https=)
AU (1) AU2009213147A1 (https=)
CA (1) CA2715289C (https=)
IL (1) IL207548A0 (https=)
WO (1) WO2009102427A2 (https=)

Families Citing this family (130)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10131904B2 (en) 2008-02-11 2018-11-20 Rxi Pharmaceuticals Corporation Modified RNAi polynucleotides and uses thereof
TWI455944B (zh) 2008-07-01 2014-10-11 Daiichi Sankyo Co Ltd 雙股多核苷酸
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
CA2746527A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Rna interference in skin indications
WO2010059226A2 (en) * 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
US9493774B2 (en) 2009-01-05 2016-11-15 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
US8871730B2 (en) 2009-07-13 2014-10-28 Somagenics Inc. Chemical modification of short small hairpin RNAs for inhibition of gene expression
WO2011109248A1 (en) 2010-03-02 2011-09-09 Hapten Pharmaceuticals, Llc Effective sensitizing dose of a gelled immunomodulating topical composition
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
BR112012024049A2 (pt) 2010-03-24 2017-03-01 Rxi Pharmaceuticals Corp interferência de rna em indicações dérmicas e fibróticas
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
CN106074591B (zh) 2010-03-24 2020-01-14 菲奥医药公司 眼部症候中的rna干扰
JO3756B1 (ar) 2010-11-23 2021-01-31 Regeneron Pharma اجسام مضادة بشرية لمستقبلات الجلوكاجون
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
AU2013315007A1 (en) 2012-09-17 2015-04-09 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
EP2927320B1 (en) 2012-11-29 2019-09-04 Gifu University Rna interference agent, method for producing same, and use thereof
LT3702466T (lt) * 2013-08-27 2023-02-27 Research Institute At Nationwide Children's Hospital Produktai ir būdai, skirti šoninės amiotrofinės sklerozės gydymui
WO2015069647A1 (en) * 2013-11-05 2015-05-14 The Research Institute At Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS
WO2015084897A2 (en) 2013-12-02 2015-06-11 Mirimmune, Llc Immunotherapy of cancer
EP3077050B1 (en) 2013-12-04 2020-10-21 Phio Pharmaceuticals Corp. Methods for treatment of wound healing utilizing chemically modified oligonucleotides
EP4410805A3 (en) 2014-03-18 2024-11-27 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
RS60707B1 (sr) * 2014-04-01 2020-09-30 Biogen Ma Inc Kompozicije za modulaciju ekspresije sod-1
EP3733856A1 (en) 2014-04-18 2020-11-04 University of Massachusetts Exosomal loading using hydrophobically modified oligonucleotides
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
WO2015168605A1 (en) 2014-05-01 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treatment of disorders in the front of the eye utilizing nucleic acid molecules
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
KR20170062466A (ko) 2014-09-16 2017-06-07 리제너론 파마슈티칼스 인코포레이티드 항-글루카곤 항체 및 그것의 사용
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
CA3193811A1 (en) * 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP4372091A3 (en) 2014-12-12 2024-07-31 Tod M. Woolf Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
CN108135923B (zh) * 2015-07-06 2021-03-02 菲奥医药公司 靶向超氧化物歧化酶1（sod1）的核酸分子
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
WO2017173304A1 (en) 2016-04-01 2017-10-05 Avidity Biosciences Llc Kras nucleic acids and uses thereof
WO2017173297A1 (en) 2016-04-01 2017-10-05 Avidity Biosciences Llc Beta-catenin nucleic acids and uses thereof
MA45349A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques egfr et leurs utilisations
JP6872560B2 (ja) 2016-05-06 2021-05-19 エム． ウルフ、トッド プログラム可能ヌクレアーゼのあるゲノム編集及びプログラム可能ヌクレアーゼのないゲノム編集のための改善された方法
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
US10876116B2 (en) * 2016-07-15 2020-12-29 The Board Of Regents Of The University Of Oklahoma Anti-ARID3a treatments for inflammatory disorders
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
CN106244590B (zh) * 2016-08-18 2019-10-01 广州市锐博生物科技有限公司 经修饰的siRNA分子、RNAi分子混合物及其应用
US20190248888A1 (en) 2016-10-20 2019-08-15 Regeneron Pharmaceuticals, Inc. Methods of lowering blood glucose levels
EP3535274A4 (en) * 2016-11-07 2020-07-29 Nanosur LLC Post-transcriptionally chemically modified double stranded RNA
JP7155239B2 (ja) 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
WO2018195355A1 (en) 2017-04-19 2018-10-25 Rxi Pharmaceuticals Corporation Topical delivery of nucleic acid compounds
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2018208972A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
WO2018226788A1 (en) * 2017-06-07 2018-12-13 University Of Massachusetts Anti-adam33 oligonucleotides and related methods
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
WO2019014656A1 (en) 2017-07-14 2019-01-17 Han Si Ping METALLIC OLIGONUCLEOTIDE JONCTIONS FOR THE ADMINISTRATION OF THERAPEUTIC AGENTS
EP3665281A4 (en) 2017-08-10 2021-05-05 City of Hope CONDITIONAL SIRNA AND ITS USE IN THE TREATMENT OF CARDIAC HYERTROPHY
CA3075643A1 (en) 2017-09-22 2019-03-28 University Of Massachusetts Sod1 dual expression vectors and uses thereof
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP4501408A3 (en) 2017-11-17 2025-04-09 Iovance Biotherapeutics, Inc. Til expansion from fine needle aspirates and small biopsies
EP3714041A1 (en) 2017-11-22 2020-09-30 Iovance Biotherapeutics, Inc. Expansion of peripheral blood lymphocytes (pbls) from peripheral blood
WO2019105437A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
WO2019105435A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CA3083968C (en) * 2017-12-01 2024-04-23 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
CN110945130B (zh) 2017-12-01 2024-04-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
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
JP7506601B2 (ja) 2017-12-06 2024-06-26 アビディティー バイオサイエンシーズ，インク． 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
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
EP3732185B1 (en) 2017-12-29 2025-02-26 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
SG11202006541UA (en) 2018-01-08 2020-08-28 Iovance Biotherapeutics Inc Processes for generating til products enriched for tumor antigen-specific t-cells
US11713446B2 (en) 2018-01-08 2023-08-01 Iovance Biotherapeutics, Inc. Processes for generating TIL products enriched for tumor antigen-specific T-cells
WO2019136459A1 (en) 2018-01-08 2019-07-11 Iovance Biotherapeutics, Inc. Processes for generating til products enriched for tumor antigen-specific t-cells
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
CN113166750B (zh) 2018-08-10 2025-02-18 希望之城 可编程的条件性sirna及其用途
US11918600B2 (en) 2018-08-21 2024-03-05 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof
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
EP3862024A4 (en) 2018-09-30 2022-08-17 Suzhou Ribo Life Science Co., Ltd. SIRNA CONJUGATE, METHOD FOR ITS PRODUCTION AND ITS USE
US20230039976A1 (en) 2018-11-05 2023-02-09 Iovance Biotherapeutics, Inc. Selection of improved tumor reactive t-cells
CA3118493A1 (en) 2018-11-05 2020-05-14 Iovance Biotherapeutics, Inc. Expansion of tils utilizing akt pathway inhibitors
TW202039830A (zh) 2018-11-05 2020-11-01 美商艾歐凡斯生物治療公司 用於製造腫瘤浸潤性淋巴細胞之方法及其在免疫療法中之用途
TW202039831A (zh) 2018-11-05 2020-11-01 美商艾歐凡斯生物治療公司 對抗ｐｄ－１抗體呈現難治性之非小細胞肺癌（ｎｓｃｌｃ）病患之治療
JP7710372B2 (ja) 2018-12-19 2025-07-18 アイオバンス バイオセラピューティクス，インコーポレイテッド 操作されたサイトカイン受容体対を使用して腫瘍浸潤リンパ球を拡大培養する方法及びその使用
US12496347B2 (en) 2018-12-28 2025-12-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
WO2020149644A1 (ko) * 2019-01-15 2020-07-23 (주)바이오니아 Dkk1 유전자를 표적으로 하는 이중나선 올리고뉴클레오티드, 이를 포함하는 구조체 및 이를 포함하는 탈모 예방 또는 발모용 조성물
CN113614232A (zh) * 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
US20220133795A1 (en) 2019-03-01 2022-05-05 Iovance Biotherapeutics, Inc. Expansion of Tumor Infiltrating Lymphocytes From Liquid Tumors and Therapeutic Uses Thereof
US20220249559A1 (en) 2019-05-13 2022-08-11 Iovance Biotherapeutics, Inc. Methods and compositions for selecting tumor infiltrating lymphocytes and uses of the same in immunotherapy
JP2022532652A (ja) * 2019-05-17 2022-07-15 アルニラム ファーマスーティカルズ インコーポレイテッド オリゴヌクレオチドの経口送達
AU2020280438B2 (en) 2019-05-22 2025-03-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
JP7610268B2 (ja) 2019-05-22 2025-01-08 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、薬物組成物及び複合体ならびに調製方法と使用
WO2020233680A1 (zh) 2019-05-22 2020-11-26 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
EP3978609A4 (en) 2019-05-24 2024-02-07 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
JP2022545118A (ja) * 2019-08-23 2022-10-25 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
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
CN115135765A (zh) 2019-11-08 2022-09-30 菲奥医药公司 用于免疫治疗的靶向含布罗莫结构域之蛋白4(brd4)的经化学修饰的寡核苷酸
KR102757180B1 (ko) 2019-11-22 2025-01-21 (주)바이오니아 Ctgf 유전자 특이적 이중가닥 올리고뉴클레오티드 및 이를 포함하는 섬유증 관련 질환 및 호흡기 관련 질환 예방 및 치료용 조성물
US12516291B2 (en) 2019-12-11 2026-01-06 Iovance Biotherapeutics, Inc. Processes for the production of tumor infiltrating lymphocytes (TILs) and methods of using the same
US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
EP4146794A1 (en) 2020-05-04 2023-03-15 Iovance Biotherapeutics, Inc. Processes for production of tumor infiltrating lymphocytes and uses of the same in immunotherapy
TW202208616A (zh) 2020-05-04 2022-03-01 美商艾歐凡斯生物治療公司 改良之腫瘤反應性ｔ細胞的選擇
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
TW202214858A (zh) * 2020-08-04 2022-04-16 大陸商上海拓界生物醫藥科技有限公司 脫靶活性降低的修飾siRNA
US20230372397A1 (en) 2020-10-06 2023-11-23 Iovance Biotherapeutics, Inc. Treatment of nsclc patients with tumor infiltrating lymphocyte therapies
WO2022076606A1 (en) 2020-10-06 2022-04-14 Iovance Biotherapeutics, Inc. Treatment of nsclc patients with tumor infiltrating lymphocyte therapies
TW202241468A (zh) 2020-12-11 2022-11-01 美商艾歐凡斯生物治療公司 用腫瘤浸潤性淋巴球療法與ｂｒａｆ抑制劑及／或ｍｅｋ抑制劑組合治療癌症患者
CA3202483A1 (en) 2020-12-17 2022-06-23 Maria Fardis Treatment with tumor infiltrating lymphocyte therapies in combination with ctla-4 and pd-1 inhibitors
US20240123067A1 (en) 2020-12-17 2024-04-18 Iovance Biotherapeutics, Inc. Treatment of cancers with tumor infiltrating lymphocyte therapies
AU2022212580A1 (en) * 2021-01-30 2023-07-27 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
US20240318174A1 (en) * 2021-02-08 2024-09-26 Ractigen Therapeutics Multi-Valent Oligonucleotide Agent and Methods of Use Thereof
WO2022174000A2 (en) * 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
AU2022246174A1 (en) 2021-03-25 2023-09-14 Iovance Biotherapeutics, Inc. Methods and compositions for t-cell coculture potency assays and use with cell therapy products
EP4326287A2 (en) 2021-04-19 2024-02-28 Iovance Biotherapeutics, Inc. Chimeric costimulatory receptors, chemokine receptors, and the use of same in cellular immunotherapies
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
US20250297258A1 (en) * 2021-07-07 2025-09-25 Ractigen Therapeutics Oligonucleotide-based delivery vehicle for oligonucleotides agents and methods of use thereof
US20240342285A1 (en) 2021-07-28 2024-10-17 Iovance Biotherapeutics, Inc. Treatment of cancer patients with tumor infiltrating lymphocyte therapies in combination with kras inhibitors
CN115725574A (zh) * 2021-08-31 2023-03-03 上海交通大学医学院附属仁济医院 一种含三氟甲基碱基类似物的小干扰RNA（siRNA）
WO2023077015A2 (en) 2021-10-27 2023-05-04 Iovance Biotherapeutics, Inc. Systems and methods for coordinating manufacturing of cells for patient-specific immunotherapy
EP4430191A2 (en) * 2021-11-08 2024-09-18 University Of Massachusetts Oligonucleotides for sod1 modulation
EP4430167A1 (en) 2021-11-10 2024-09-18 Iovance Biotherapeutics, Inc. Methods of expansion treatment utilizing cd8 tumor infiltrating lymphocytes
JP2024543963A (ja) * 2021-12-03 2024-11-26 マイクロバイオ （シャンハイ） カンパニー リミテッド 高い安定性及び遺伝子サイレンシング活性を有する低分子干渉ｒｎａ分子のための修飾パターン
CA3248034A1 (en) 2022-04-15 2023-10-19 Iovance Biotherapeutics, Inc. METHODS FOR EXPANSION OF TIL CELLS BY MEANS OF SPECIFIC CYTOKINE COMBINATIONS AND/OR AKT INHIBITOR TREATMENT
CN119161484A (zh) 2022-04-25 2024-12-20 武汉大学 抗asgr1单克隆抗体及其应用
EP4540390A2 (en) 2022-06-15 2025-04-23 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of superoxide dismutase 1 (sod1), compositions thereof, and methods of use
WO2023246935A1 (zh) * 2022-06-23 2023-12-28 安沛治疗有限公司 包含喹啉修饰的靶特异性核酸分子
EP4565683A1 (en) 2022-08-01 2025-06-11 Iovance Biotherapeutics, Inc. Chimeric costimulatory receptors, chemokine receptors, and the use of same in cellular immunotherapies
WO2024098024A1 (en) 2022-11-04 2024-05-10 Iovance Biotherapeutics, Inc. Expansion of tumor infiltrating lymphocytes from liquid tumors and therapeutic uses thereof
WO2025101484A1 (en) 2023-11-06 2025-05-15 Iovance Biotherapeutics, Inc. Treatment of endometrial cancers with tumor infiltrating lymphocyte therapies
WO2026080661A1 (en) * 2024-10-10 2026-04-16 Eli Lilly And Company Sod1 rna interference agents

Family Cites Families (252)

* 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
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4201860A (en) 1978-05-09 1980-05-06 Bristol-Myers Company Purine derivatives
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4426330A (en) 1981-07-20 1984-01-17 Lipid Specialties, Inc. Synthetic phospholipid compounds
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US5643889A (en) 1984-07-11 1997-07-01 Temple University-Of The Commonwealth System Of Pennsylvania Cholesterol conjugates of 2'5'-oligoadenylate derivatives and antiviral uses thereof
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
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
DE3788914T2 (de) 1986-09-08 1994-08-25 Ajinomoto Kk Verbindungen zur Spaltung von RNS an eine spezifische Position, Oligomere, verwendet bei der Herstellung dieser Verbindungen und Ausgangsprodukte für die Synthese dieser Oligomere.
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
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
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
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
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5750666A (en) 1988-05-26 1998-05-12 Competitve Technologies, Inc. Polynucleotide phosphorodithioate compounds
ZA902710B (en) 1989-05-22 1991-12-24 Univ Georgia Res Found Enzyme luminescence assay
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5495009A (en) 1989-10-24 1996-02-27 Gilead Sciences, Inc. Oligonucleotide analogs containing thioformacetal linkages
ATE269870T1 (de) 1989-10-24 2004-07-15 Isis Pharmaceuticals Inc 2'-modifizierte oligonukleotide
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US6395492B1 (en) 1990-01-11 2002-05-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US6153737A (en) 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US6399754B1 (en) 1991-12-24 2002-06-04 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5514786A (en) 1990-01-11 1996-05-07 Isis Pharmaceuticals, Inc. Compositions for inhibiting RNA activity
US6358931B1 (en) 1990-01-11 2002-03-19 Isis Pharmaceuticals, Inc. Compositions and methods for modulating RNA
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5658731A (en) 1990-04-09 1997-08-19 Europaisches Laboratorium Fur Molekularbiologie 2'-O-alkylnucleotides as well as polymers which contain such nucleotides
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5151510A (en) 1990-04-20 1992-09-29 Applied Biosystems, Inc. Method of synethesizing sulfurized oligonucleotide analogs
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
BR9106702A (pt) 1990-07-27 1993-06-08 Isis Pharmaceuticals Inc Analogo de oligonucleotideos e processos para modular a producao de uma proteina por um organismo e para tratar um organismo
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
JPH0813274B2 (ja) 1990-08-13 1996-02-14 アイシス・ファーマシューティカルス・インコーポレーテッド 遺伝子発現を検出及び変調する糖修飾されたオリゴヌクレオチド
EP0547142A1 (en) 1990-08-28 1993-06-23 Epoch Pharmaceuticals, Inc. Solid support synthesis of 3'-tailed oligonucleotides via a linking molecule
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
CA2092002A1 (en) 1990-09-20 1992-03-21 Mark Matteucci Modified internucleoside linkages
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
WO1994008003A1 (en) 1991-06-14 1994-04-14 Isis Pharmaceuticals, Inc. ANTISENSE OLIGONUCLEOTIDE INHIBITION OF THE ras GENE
US5525719A (en) 1991-08-30 1996-06-11 Chemgenes Corporation N-protected-2'-O-methyl-and N-protected-3'-O-methyl-ribonucleosides and their phosphoramidite derivatives
US5214135A (en) 1991-08-30 1993-05-25 Chemgenes Corporation N-protected-2'-O-methyl-ribonucleosides and N-protected 2'-O-methyl-3'-cyanoethyl-N-,N-diisopropyl phosphoramidite ribonucleosides
US5599797A (en) 1991-10-15 1997-02-04 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5607923A (en) 1991-10-15 1997-03-04 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating cytomegalovirus having phosphorothioate linkages of high chiral purity
US5661134A (en) 1991-10-15 1997-08-26 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating Ha-ras or Ki-ras having phosphorothioate linkages of high chiral purity
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5856455A (en) 1991-12-24 1999-01-05 Isis Pharmaceuticals, Inc. Gapped 2'-modified oligonucleotides
FI115214B (fi) 1992-01-22 2005-03-31 Hoechst Ag Menetelmä oligonukleotidianalogien valmistamiseksi ja niiden käyttö
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5817781A (en) 1992-06-01 1998-10-06 Gilead Sciences, Inc. Modified internucleoside linkages (II)
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
US6346614B1 (en) 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
JP3351476B2 (ja) 1993-01-22 2002-11-25 三菱化学株式会社 リン脂質誘導体及びそれを含有するリポソーム
AU6527094A (en) 1993-03-31 1994-10-24 Hybridon, Inc. Modified oligonucleotides having improved anti-influenza activity
CA2159629A1 (en) 1993-03-31 1994-10-13 Sanofi Oligonucleotides with amide linkages replacing phosphodiester linkages
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
ES2128535T3 (es) 1993-05-12 1999-05-16 Novartis Ag Nucleosidos y oligonucleotidos con grupos 2'-eter.
US6015886A (en) 1993-05-24 2000-01-18 Chemgenes Corporation Oligonucleotide phosphate esters
US5428149A (en) 1993-06-14 1995-06-27 Washington State University Research Foundation Method for palladium catalyzed carbon-carbon coulping and products
US5580972A (en) 1993-06-14 1996-12-03 Nexstar Pharmaceuticals, Inc. Purine nucleoside modifications by palladium catalyzed methods
US5534259A (en) 1993-07-08 1996-07-09 Liposome Technology, Inc. Polymer compound and coated particle composition
US5532130A (en) 1993-07-20 1996-07-02 Dyad Pharmaceutical Corporation Methods and compositions for sequence-specific hybridization of RNA by 2'-5' oligonucleotides
US5417978A (en) 1993-07-29 1995-05-23 Board Of Regents, The University Of Texas System Liposomal antisense methyl phosphonate oligonucleotides and methods for their preparation and use
US5614621A (en) 1993-07-29 1997-03-25 Isis Pharmaceuticals, Inc. Process for preparing oligonucleotides using silyl-containing diamino phosphorous reagents
WO1995006731A2 (en) 1993-09-02 1995-03-09 Ribozyme Pharmaceuticals, Inc. Non-nucleotide containing enzymatic nucleic acid
AU679566B2 (en) 1993-09-03 1997-07-03 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
EP0725788B1 (en) 1993-10-27 1998-12-16 Ribozyme Pharmaceuticals, Inc. 2'-amido and 2'-peptido modified oligonucleotides
AU687492B2 (en) 1993-11-16 1998-02-26 Genta Incorporated Synthetic oligomers having phosphonate internucleosidyl linkages of undefined chirality mixed with non-phosphonate internucleosidyl linkages
DE69500618D1 (de) 1994-02-18 1997-10-02 Hybridon Inc Oligonukleotide mit wirkung gegen das respiratorische synzytial virus
US5646126A (en) 1994-02-28 1997-07-08 Epoch Pharmaceuticals Sterol modified oligonucleotide duplexes having anticancer activity
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5651981A (en) 1994-03-29 1997-07-29 Northwestern University Cationic phospholipids for transfection
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5777153A (en) 1994-07-08 1998-07-07 Gilead Sciences, Inc. Cationic lipids
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5591721A (en) 1994-10-25 1997-01-07 Hybridon, Inc. Method of down-regulating gene expression
US5681940A (en) 1994-11-02 1997-10-28 Icn Pharmaceuticals Sugar modified nucleosides and oligonucleotides
US5767099A (en) 1994-12-09 1998-06-16 Genzyme Corporation Cationic amphiphiles containing amino acid or dervatized amino acid groups for intracellular delivery of therapeutic molecules
US5948767A (en) 1994-12-09 1999-09-07 Genzyme Corporation Cationic amphiphile/DNA complexes
US5830430A (en) 1995-02-21 1998-11-03 Imarx Pharmaceutical Corp. Cationic lipids and the use thereof
US6420549B1 (en) 1995-06-06 2002-07-16 Isis Pharmaceuticals, Inc. Oligonucleotide analogs having modified dimers
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
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5851548A (en) 1995-06-07 1998-12-22 Gen-Probe Incorporated Liposomes containing cationic lipids and vitamin D
US5734041A (en) 1995-10-20 1998-03-31 Mcgill University Preparation of chiral phosphorothioate oligomers
US5705621A (en) 1995-11-17 1998-01-06 Isis Pharmaceuticals, Inc. Oligomeric phosphite, phosphodiester, Phosphorothioate and phosphorodithioate compounds and intermediates for preparing same
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
US6331617B1 (en) 1996-03-21 2001-12-18 University Of Iowa Research Foundation Positively charged oligonucleotides as regulators of gene expression
US6444806B1 (en) 1996-04-30 2002-09-03 Hisamitsu Pharmaceutical Co., Inc. Conjugates and methods of forming conjugates of oligonucleotides and carbohydrates
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US5789416B1 (en) 1996-08-27 1999-10-05 Cv Therapeutics Inc N6 mono heterocyclic substituted adenosine derivatives
US6111085A (en) 1996-09-13 2000-08-29 Isis Pharmaceuticals, Inc. Carbamate-derivatized nucleosides and oligonucleosides
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6028183A (en) 1997-11-07 2000-02-22 Gilead Sciences, Inc. Pyrimidine derivatives and oligonucleotides containing same
AU1507199A (en) 1997-12-15 1999-07-05 Yamanouchi Pharmaceutical Co., Ltd. Novel pyrimidine-5-carboxamide derivatives
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6020475A (en) 1998-02-10 2000-02-01 Isis Pharmeuticals, Inc. Process for the synthesis of oligomeric compounds
AU3751299A (en) 1998-04-20 1999-11-08 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
US20040241845A1 (en) 1998-05-22 2004-12-02 Luc Desgroseillers Mammalian staufen and use thereof
US6277967B1 (en) 1998-07-14 2001-08-21 Isis Pharmaceuticals, Inc. Carbohydrate or 2′-modified oligonucleotides having alternating internucleoside linkages
US6242589B1 (en) 1998-07-14 2001-06-05 Isis Pharmaceuticals, Inc. Phosphorothioate oligonucleotides having modified internucleoside linkages
US6271358B1 (en) 1998-07-27 2001-08-07 Isis Pharmaceuticals, Inc. RNA targeted 2′-modified oligonucleotides that are conformationally preorganized
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
US20040009938A1 (en) 1998-08-07 2004-01-15 Muthiah Manoharan Methods of enhancing renal uptake of oligonucleotides
US6020483A (en) 1998-09-25 2000-02-01 Nexstar Pharmaceuticals, Inc. Nucleoside modifications by palladium catalyzed methods
US6328765B1 (en) 1998-12-03 2001-12-11 Gore Enterprise Holdings, Inc. Methods and articles for regenerating living tissue
US6455586B1 (en) 1998-12-15 2002-09-24 Leonard L. Kaplan Topical immunomodulating compositions for treatment of aids, Hepatitis B & C, other infectious diseases, and cancer
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US6465628B1 (en) 1999-02-04 2002-10-15 Isis Pharmaceuticals, Inc. Process for the synthesis of oligomeric compounds
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
US6207819B1 (en) 1999-02-12 2001-03-27 Isis Pharmaceuticals, Inc. Compounds, processes and intermediates for synthesis of mixed backbone oligomeric compounds
US6121437A (en) 1999-03-16 2000-09-19 Isis Pharmaceuticals, Inc. Phosphate and thiophosphate protecting groups
US6033910A (en) 1999-07-19 2000-03-07 Isis Pharmaceuticals Inc. Antisense inhibition of MAP kinase kinase 6 expression
GB9925459D0 (en) 1999-10-27 1999-12-29 Plant Bioscience Ltd Gene silencing
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US20050032733A1 (en) 2001-05-18 2005-02-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
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
EP1272630A2 (en) 2000-03-16 2003-01-08 Genetica, Inc. Methods and compositions for rna interference
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
DK1303495T3 (da) 2000-07-24 2010-09-20 Krenitsky Pharmaceuticals Inc Substituerede 5-alkylnylpyrimidiner med neurotrofisk aktivitet
US20020132788A1 (en) 2000-11-06 2002-09-19 David Lewis Inhibition of gene expression by delivery of small interfering RNA to post-embryonic animal cells in vivo
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
FR2818642B1 (fr) 2000-12-26 2005-07-15 Hoechst Marion Roussel Inc Nouveaux derives de la purine, leur procede de preparation, leur application a titre de medicaments, compositions pharmaceutiques et nouvelle utilistion
JP2004532616A (ja) 2000-12-28 2004-10-28 ジョンソン・アンド・ジョンソン・リサーチ・ピー・ティー・ワイ・リミテッド 二本鎖ｒｎａ仲介遺伝子抑制
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
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)
US20070032441A1 (en) 2001-05-18 2007-02-08 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina)
US20050239731A1 (en) 2001-05-18 2005-10-27 Sirna Therapeutics, Inc. RNA interference mediated inhibition of MAP kinase gene expression using short interfering nucleic acid (siNA)
CA2790034A1 (en) 2001-06-21 2003-01-03 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US20050019915A1 (en) 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
DE10133858A1 (de) 2001-07-12 2003-02-06 Aventis Pharma Gmbh Synthetische doppelsträngige Oligonucleotide zur gezielten Hemmung der Genexpression
AU2002329667A1 (en) 2001-07-30 2003-02-17 Uta Griesenbach Specific inhibition of gene expression by small double stranded rnas
AU2002343475A1 (en) 2001-10-03 2003-04-14 Selective Genetics, Inc. Traversal of nucleic acid molecules through a fluid space and expression in repair cells
EP1575976A4 (en) 2001-11-02 2006-08-23 Insert Therapeutics Inc METHODS AND COMPOSITIONS FOR THE THERAPEUTIC USE OF RNA INTERFERENCE
US20040087526A1 (en) 2001-11-12 2004-05-06 Shi-Lung Lin Therapeutically useful compositions of DNA-RNA hybrid duplex constructs
EP2213737B1 (en) 2002-02-01 2012-11-07 Life Technologies Corporation Double-stranded oligonucleotides
US20030166282A1 (en) 2002-02-01 2003-09-04 David Brown High potency siRNAS for reducing the expression of target genes
DE60325016D1 (de) 2002-02-01 2009-01-15 Univ Mcgill Oligonukleotide mit alternierenden segmenten und deren verwendungen
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
AU2003216255A1 (en) 2002-02-20 2003-09-09 Ribozyme Pharmaceuticals, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF MDR P-GLYCOPROTEIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20040014957A1 (en) 2002-05-24 2004-01-22 Anne Eldrup Oligonucleotides having modified nucleoside units
AU2003260370B2 (en) 2002-08-05 2008-05-22 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules
US20040032733A1 (en) * 2002-08-08 2004-02-19 Gabriel Frederick V. Candle carrier for use with candle accessories burning tea light candles
AU2003273336A1 (en) 2002-09-18 2004-04-08 Isis Pharmaceuticals, Inc. Efficient reduction of target rna's by single- and double-stranded oligomeric compounds
CA2500224C (en) 2002-09-25 2015-04-28 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
AU2003284323A1 (en) 2002-10-18 2004-05-04 Alnylam Pharmaceuticals Inc Double-stranded rna structures and constructs, and methods for generating and using the same
ES2334125T3 (es) * 2002-11-04 2010-03-05 University Of Massachusetts Interferencia de arn especifico de alelos.
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP2305812A3 (en) 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
ES2343318T3 (es) 2002-11-26 2010-07-28 University Of Massachusetts Administracion de arnsis.
WO2004065600A2 (en) 2003-01-17 2004-08-05 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna interference by palindromic or modified rna molecules
DE10302421A1 (de) 2003-01-21 2004-07-29 Ribopharma Ag Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit
EP1589931A2 (en) 2003-02-05 2005-11-02 University of Massachusetts RNAi TARGETING OF VIRUSES
US20040167090A1 (en) 2003-02-21 2004-08-26 Monahan Sean D. Covalent modification of RNA for in vitro and in vivo delivery
WO2004080406A2 (en) 2003-03-07 2004-09-23 Alnylam Pharmaceuticals Therapeutic compositions
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
DK1620544T3 (en) 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
US20060178327A1 (en) 2003-05-30 2006-08-10 Yeung Wah Hin A Inhibition of gene expression by delivery of specially selected double stranded or other forms of small interfering RNA precursors enabling the formation and function of small interfering RNA in vivo and in vitro
US7750144B2 (en) * 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
ES2357116T5 (es) 2003-06-02 2021-09-03 Univ Massachusetts Métodos y composiciones para mejorar la eficacia y especificad de FNAi
AU2004263830B2 (en) 2003-06-13 2008-12-18 Alnylam Pharmaceuticals, Inc. Double-stranded ribonucleic acid with increased effectiveness in an organism
US20050106594A1 (en) 2003-08-22 2005-05-19 Andrew Ellington In vitro selection of aptamer beacons
EP2123759B1 (en) 2004-02-06 2014-01-15 Thermo Fisher Scientific Biosciences Inc. Stabilized RNAS as transfection controls and silencing reagents
WO2005079533A2 (en) 2004-02-17 2005-09-01 University Of Massachusetts Methods and compositions for mediating gene silencing
EP1731615A1 (en) 2004-02-20 2006-12-13 National Institute of Advanced Industrial Science and Technology Cytoplasmic localization dna and rna
KR101147147B1 (ko) * 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
US7498316B2 (en) 2004-04-06 2009-03-03 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using RNA interference
US20060014289A1 (en) 2004-04-20 2006-01-19 Nastech Pharmaceutical Company Inc. Methods and compositions for enhancing delivery of double-stranded RNA or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells
AU2005252663B2 (en) 2004-06-03 2011-07-07 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
US20060211766A1 (en) 2004-09-30 2006-09-21 Kaplan Leonard L Gelled immunomodulating topical compositions and a method of treating warts and other human papilloma virus skin infections
ES2441791T3 (es) 2004-10-01 2014-02-06 Novartis Vaccines And Diagnostics, Inc. Moléculas de ARN de interferencia pequeñas modificadas y métodos de uso
US7923207B2 (en) 2004-11-22 2011-04-12 Dharmacon, Inc. Apparatus and system having dry gene silencing pools
US20100069620A1 (en) 2004-12-02 2010-03-18 Rxi Pharmaceuticals Corp. Novel compositions of chemically modified small interfering rna
AU2005311684A1 (en) 2004-12-02 2006-06-08 University Of Massachusetts Glucose-transport related genes, polypeptides, and methods of use thereof
US7910561B2 (en) 2004-12-15 2011-03-22 Merck Sharp & Dohme Corp. Inhibitors of Akt activity
US20060229268A1 (en) 2004-12-16 2006-10-12 Alsgen, Llc Small interference RNA (siRNA) molecules for modulating superoxide dismutase (SOD)
EP1891217A2 (en) * 2005-05-27 2008-02-27 Sirna Therapeutics, Inc. Rna interference mediated inhibition of stromal cell-derived factor-1 (sdf-1) gene expression using short interfering nucleic acid (sina)
WO2007030167A1 (en) * 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
WO2007056604A2 (en) * 2005-11-09 2007-05-18 Irm Llc Methods and compositions for modulating cell motility and inhibiting tumor metastasis
CA2633063A1 (en) 2005-12-16 2007-06-21 Diatos Cell penetrating peptide conjugates for delivering of nucleic acids intocells
US7825099B2 (en) 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
US20070231392A1 (en) 2006-01-23 2007-10-04 Ernst Wagner CHEMICALLY MODIFIED POLYCATION POLYMER FOR siRNA DELIVERY
WO2007089607A2 (en) 2006-01-26 2007-08-09 University Of Massachusetts Rna silencing agents for use in therapy and nanotransporters for efficient delivery of same
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
ES2874149T3 (es) 2006-05-11 2021-11-04 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen PCSK9
US7572618B2 (en) 2006-06-30 2009-08-11 Bristol-Myers Squibb Company Polynucleotides encoding novel PCSK9 variants
WO2008036841A2 (en) 2006-09-22 2008-03-27 Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by rna interference
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
WO2008109353A1 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting map2k gene expression and uses thereof
US20080311040A1 (en) 2007-03-06 2008-12-18 Flagship Ventures METHODS AND COMPOSITIONS FOR IMPROVED THERAPEUTIC EFFECTS WITH siRNA
WO2009045457A2 (en) 2007-10-02 2009-04-09 Rxi Pharmaceuticals Corp. Tripartite rnai constructs
AU2008306455C1 (en) 2007-10-03 2014-04-17 Quark Pharmaceuticals, Inc. Novel siRNA structures
MX2010008394A (es) 2008-01-31 2010-11-12 Alnylam Pharmaceuticals Inc Metodos optimizados para administracion de arndc focalizando el gen pcsk9.
US10131904B2 (en) 2008-02-11 2018-11-20 Rxi Pharmaceuticals Corporation Modified RNAi polynucleotides and uses thereof
WO2009114475A2 (en) 2008-03-09 2009-09-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
US20110218334A1 (en) 2008-07-11 2011-09-08 Alnylam Pharmaceuticals, Inc. PHOSPHOROTHIOATE OLIGONUCLEOTIDES AND NON-NUCLEOSIDIC PHOSPHOROTHIOATES AS DELIVERY AGENTS FOR iRNA AGENTS
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
EP2318528A1 (en) 2008-07-24 2011-05-11 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
NZ601660A (en) 2008-08-25 2014-05-30 Excaliard Pharmaceuticals Inc Antisense oligonucleotides directed against connective tissue growth factor and uses thereof
WO2010027830A2 (en) 2008-08-25 2010-03-11 Excaliard Pharmaceuticals, Inc. Methods for modulation of connective tissue growth factor expression by administering antisense oligonucleotides through a delivery system so as to reduce scarring from wound healing and threat fibrotic diseases
CA2746527A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Rna interference in skin indications
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
US9493774B2 (en) 2009-01-05 2016-11-15 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
WO2011109248A1 (en) 2010-03-02 2011-09-09 Hapten Pharmaceuticals, Llc Effective sensitizing dose of a gelled immunomodulating topical composition
WO2011109698A1 (en) 2010-03-04 2011-09-09 Rxi Pharmaceuticals Corporation Formulations and methods for targeted delivery to phagocyte cells
BR112012024049A2 (pt) 2010-03-24 2017-03-01 Rxi Pharmaceuticals Corp interferência de rna em indicações dérmicas e fibróticas
CN106074591B (zh) 2010-03-24 2020-01-14 菲奥医药公司 眼部症候中的rna干扰
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
US20140072613A1 (en) 2012-09-10 2014-03-13 Cynthia Lander Compositions and Methods for Treating Cutaneous Scarring
WO2014191493A1 (en) 2013-05-28 2014-12-04 Vib Vzw Single domain antibodies against sod1 and their use in medicine
LT3702466T (lt) 2013-08-27 2023-02-27 Research Institute At Nationwide Children's Hospital Produktai ir būdai, skirti šoninės amiotrofinės sklerozės gydymui
WO2015084897A2 (en) 2013-12-02 2015-06-11 Mirimmune, Llc Immunotherapy of cancer
EP3077050B1 (en) 2013-12-04 2020-10-21 Phio Pharmaceuticals Corp. Methods for treatment of wound healing utilizing chemically modified oligonucleotides
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
WO2015168605A1 (en) 2014-05-01 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treatment of disorders in the front of the eye utilizing nucleic acid molecules
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
EP3226848A4 (en) 2014-12-03 2019-04-03 Phio Pharmaceuticals Corp. METHOD FOR TREATING ALOPECIA AREATA USING GENE MODULATION APPARATUS
SMT202000454T1 (it) 2015-04-03 2020-11-10 Univ Massachusetts Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina
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
CN108135923B (zh) 2015-07-06 2021-03-02 菲奥医药公司 靶向超氧化物歧化酶1（sod1）的核酸分子
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
EP3347000A4 (en) 2015-09-11 2019-03-27 Phio Pharmaceuticals Corp. METHODS OF TREATING DISORDERS AND SKIN DISORDERS USING HAPTENES
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna

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