CA2732229C - Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand - Google Patents
Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand Download PDFInfo
- Publication number
- CA2732229C CA2732229C CA2732229A CA2732229A CA2732229C CA 2732229 C CA2732229 C CA 2732229C CA 2732229 A CA2732229 A CA 2732229A CA 2732229 A CA2732229 A CA 2732229A CA 2732229 C CA2732229 C CA 2732229C
- Authority
- CA
- Canada
- Prior art keywords
- sirna
- gene
- compound
- expression
- double
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/331—Universal or degenerate base
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US8376308P | 2008-07-25 | 2008-07-25 | |
| US61/083,763 | 2008-07-25 | ||
| PCT/US2009/051648 WO2010011895A1 (en) | 2008-07-25 | 2009-07-24 | Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2732229A1 CA2732229A1 (en) | 2010-01-28 |
| CA2732229C true CA2732229C (en) | 2023-10-17 |
Family
ID=41258560
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2732229A Active CA2732229C (en) | 2008-07-25 | 2009-07-24 | Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US20110223665A1 (enExample) |
| EP (1) | EP2321414B1 (enExample) |
| JP (2) | JP2011528910A (enExample) |
| AU (2) | AU2009273878A1 (enExample) |
| CA (1) | CA2732229C (enExample) |
| WO (1) | WO2010011895A1 (enExample) |
Families Citing this family (95)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8796436B2 (en) | 2003-04-17 | 2014-08-05 | Alnylam Pharmaceuticals, Inc. | Modified iRNA agents |
| US7723509B2 (en) | 2003-04-17 | 2010-05-25 | Alnylam Pharmaceuticals | IRNA agents with biocleavable tethers |
| JP4597976B2 (ja) | 2003-04-17 | 2010-12-15 | アルナイラム ファーマシューティカルズ インコーポレイテッド | 修飾ｉＲＮＡ剤 |
| JP5816556B2 (ja) | 2008-12-03 | 2015-11-18 | アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 治療剤のためのｕｎａオリゴマー構造 |
| US20150025122A1 (en) | 2009-10-12 | 2015-01-22 | Larry J. Smith | Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro |
| WO2011109427A2 (en) | 2010-03-01 | 2011-09-09 | Alnylam Pharmaceuticals, Inc. | Improving the biological activity of sirna through modulation of its thermodynamic profile |
| WO2012007477A1 (en) | 2010-07-12 | 2012-01-19 | Santaris Pharma A/S | Anti hcv oligomers |
| US10501513B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides for the production of oncology-related proteins and peptides |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
| WO2013151663A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| AU2013266238B2 (en) * | 2012-05-23 | 2017-07-27 | The Ohio State University | Lipid-coated albumin nanoparticle compositions and methods of making and method of using the same |
| EP2855679B1 (en) * | 2012-05-31 | 2018-07-18 | QBI Enterprises Ltd. | Therapeutic oligonucleotides comprising pyrazolotriazine nucleotide analogues |
| WO2014066142A1 (en) * | 2012-10-24 | 2014-05-01 | Novamedica Limited Liability Company | Nucleic acid regulation of growth arrest-specific protein 6 (gas6) |
| US9909123B2 (en) | 2013-03-15 | 2018-03-06 | Techulon Inc. | Antisense molecules for treatment of Staphylococcus aureus infection |
| IL316808A (en) | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| EP3234141A4 (en) | 2014-12-18 | 2018-06-20 | Alnylam Pharmaceuticals, Inc. | Reversir tm compounds |
| 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 |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| DK3324980T3 (da) | 2015-07-17 | 2022-02-14 | Alnylam Pharmaceuticals Inc | Multimålrettede enkeltenhedskonjugater |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| CN116064544A (zh) | 2016-01-26 | 2023-05-05 | 日产化学株式会社 | 单链寡核苷酸 |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| US11208653B2 (en) | 2016-04-27 | 2021-12-28 | Whitehead Institute For Biomedical Research | Increasing RNAi efficiency through single nucleotide mismatches |
| US11753638B2 (en) | 2016-08-12 | 2023-09-12 | University Of Massachusetts | Conjugated oligonucleotides |
| TW202313978A (zh) | 2016-11-23 | 2023-04-01 | 美商阿尼拉製藥公司 | 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法 |
| US11572558B2 (en) | 2017-02-06 | 2023-02-07 | Nissan Chemical Corporation | Single-stranded oligonucleotide |
| CA3064590A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | Two-tailed self-delivering sirna and related methods |
| EP3790970A1 (en) | 2018-05-07 | 2021-03-17 | Alnylam Pharmaceuticals Inc. | Extrahepatic delivery |
| 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 |
| JP7470107B2 (ja) | 2018-09-28 | 2024-04-17 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | トランスサイレチン（ＴＴＲ）ｉＲＮＡ組成物及びＴＴＲ関連眼疾患を治療又は予防するためのその使用方法 |
| WO2020150636A1 (en) | 2019-01-18 | 2020-07-23 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| US20220211743A1 (en) | 2019-05-17 | 2022-07-07 | Alnylam Pharmaceuticals, Inc. | Oral delivery of oligonucleotides |
| CA3138627A1 (en) * | 2019-06-21 | 2020-12-24 | Alexander V. KEL'IN | Structurally defined sirna-dual variable domain immunoglobulin conjugates |
| CA3149835A1 (en) | 2019-08-09 | 2021-02-18 | University Of Massachusetts | Chemically modified oligonucleotides targeting snps |
| WO2021030522A1 (en) | 2019-08-13 | 2021-02-18 | Alnylam Pharmaceuticals, Inc. | SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| WO2021067747A1 (en) | 2019-10-04 | 2021-04-08 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for silencing ugt1a1 gene expression |
| CN114728018B (zh) | 2019-11-01 | 2024-07-19 | 阿尔尼拉姆医药品有限公司 | 亨廷顿(HTT)iRNA药剂组合物及其使用方法 |
| WO2021087325A1 (en) | 2019-11-01 | 2021-05-06 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for silencing dnajb1-prkaca fusion gene expression |
| AU2020378414A1 (en) | 2019-11-06 | 2022-05-26 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| EP4055165A1 (en) | 2019-11-06 | 2022-09-14 | Alnylam Pharmaceuticals, Inc. | Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases |
| EP4061945A1 (en) | 2019-11-22 | 2022-09-28 | Alnylam Pharmaceuticals, Inc. | Ataxin3 (atxn3) rnai agent compositions and methods of use thereof |
| TWI897902B (zh) | 2019-12-13 | 2025-09-21 | 美商阿尼拉製藥公司 | 人類染色體9開讀框72 (C9ORF72) iRNA劑組成物及其使用方法 |
| MX2022009763A (es) | 2020-02-10 | 2022-09-09 | Alnylam Pharmaceuticals Inc | Composiciones y metodos para silenciar la expresion del factor de crecimiento endotelial vascular a (vegf-a). |
| EP4114947A1 (en) | 2020-03-05 | 2023-01-11 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases |
| WO2021195307A1 (en) | 2020-03-26 | 2021-09-30 | Alnylam Pharmaceuticals, Inc. | Coronavirus irna compositions and methods of use thereof |
| EP4127171A2 (en) | 2020-03-30 | 2023-02-08 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for silencing dnajc15 gene expression |
| US12534731B2 (en) | 2020-04-01 | 2026-01-27 | Alnylam Pharmaceuticals, Inc. | Alpha-2A adrenergic receptor (ADRA2A) iRNA agent compositions and methods of use thereof |
| CA3179411A1 (en) | 2020-04-06 | 2021-10-14 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for silencing myoc expression |
| WO2021206917A1 (en) | 2020-04-07 | 2021-10-14 | Alnylam Pharmaceuticals, Inc. | ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| IL297130A (en) | 2020-04-07 | 2022-12-01 | Alnylam Pharmaceuticals Inc | Compositions and methods for silencing scn9a expression |
| WO2021206922A1 (en) | 2020-04-07 | 2021-10-14 | Alnylam Pharmaceuticals, Inc. | Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof |
| WO2021222065A1 (en) | 2020-04-27 | 2021-11-04 | Alnylam Pharmaceuticals, Inc. | Apolipoprotein e (apoe) irna agent compositions and methods of use thereof |
| EP4153746A1 (en) | 2020-05-21 | 2023-03-29 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting marc1 gene expression |
| EP4157289A4 (en) | 2020-05-26 | 2024-06-26 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| EP4162050A1 (en) | 2020-06-09 | 2023-04-12 | Alnylam Pharmaceuticals, Inc. | Rnai compositions and methods of use thereof for delivery by inhalation |
| KR102361479B1 (ko) * | 2020-06-18 | 2022-02-11 | 고려대학교 산학협력단 | ｓｉＲＮＡ의 생성 및 기능 증진 활성을 갖는 ＣＭＴＲ１의 신규용도 |
| WO2022011214A1 (en) | 2020-07-10 | 2022-01-13 | Alnylam Pharmaceuticals, Inc. | Circular sirnas |
| EP4217489A1 (en) | 2020-09-24 | 2023-08-02 | Alnylam Pharmaceuticals, Inc. | Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof |
| TW202229552A (zh) | 2020-10-05 | 2022-08-01 | 美商艾拉倫製藥股份有限公司 | Ｇ蛋白－偶合受體７５（ＧＰＲ７５）ｉＲＮＡ組成物及其使用方法 |
| WO2022087329A1 (en) | 2020-10-23 | 2022-04-28 | Alnylam Pharmaceuticals, Inc. | Mucin 5b (muc5b) irna compositions and methods of use thereof |
| WO2022147214A2 (en) | 2020-12-31 | 2022-07-07 | Alnylam Pharmaceuticals, Inc. | Cyclic-disulfide modified phosphate based oligonucleotide prodrugs |
| EP4271695A2 (en) | 2020-12-31 | 2023-11-08 | Alnylam Pharmaceuticals, Inc. | 2'-modified nucleoside based oligonucleotide prodrugs |
| EP4291654A2 (en) | 2021-02-12 | 2023-12-20 | 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 |
| WO2022182864A1 (en) | 2021-02-25 | 2022-09-01 | Alnylam Pharmaceuticals, Inc. | Prion protein (prnp) irna compositions and methods and methods of use thereof |
| CN114958839A (zh) * | 2021-02-25 | 2022-08-30 | 上海市东方医院（同济大学附属东方医院） | 抑制肌肉瘤细胞中FOXO1基因表达的siRNA序列及应用 |
| EP4305169A1 (en) | 2021-03-12 | 2024-01-17 | Alnylam Pharmaceuticals, Inc. | Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof |
| IL307239A (en) | 2021-03-29 | 2023-11-01 | Alnylam Pharmaceuticals Inc | Preparations containing Huntingtin IRNA factor (HTT) and methods of using them |
| CN119792549A (zh) * | 2021-04-23 | 2025-04-11 | 天津全和诚生物技术有限公司 | 缀合物及其制备方法和用途 |
| EP4341401A1 (en) | 2021-05-18 | 2024-03-27 | Alnylam Pharmaceuticals, Inc. | Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof |
| BR112023025224A2 (pt) | 2021-06-04 | 2024-02-27 | Alnylam Pharmaceuticals Inc | Quadro de leitura aberto 72 do cromossomo humano 9 (c9orf72) composições de agente de irna e métodos de uso dos mesmos |
| AR126207A1 (es) | 2021-06-23 | 2023-09-27 | Univ Massachusetts | Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogénicos |
| WO2023283403A2 (en) | 2021-07-09 | 2023-01-12 | Alnylam Pharmaceuticals, Inc. | Bis-rnai compounds for cns delivery |
| CA3226019A1 (en) | 2021-07-20 | 2023-01-26 | Ags Therapeutics Sas | Extracellular vesicles from microalgae, their preparation, and uses |
| KR20240036041A (ko) | 2021-07-21 | 2024-03-19 | 알닐람 파마슈티칼스 인코포레이티드 | 대사 장애-연관 표적 유전자 iRNA 조성물 및 이의 사용 방법 |
| MX2024002440A (es) | 2021-08-31 | 2024-03-08 | Alnylam Pharmaceuticals Inc | Composiciones de acido ribonucleico de interferencia (arni) del efector b similar al factor de fragmentacion de adn subunidad alfa (dffa) que induce la muerte celular (cideb) y metodos de uso de estas. |
| MX2024003519A (es) | 2021-09-24 | 2024-04-01 | Alnylam Pharmaceuticals Inc | Composiciones de agentes de acido ribonucleico de interferencia (arni) de proteina tau asociada a microtubulos (mapt) y sus metodos de uso. |
| IL311864A (en) | 2021-10-15 | 2024-06-01 | Alnylam Pharmaceuticals Inc | IRNA compositions for extrahepatic administration and methods of using them |
| TW202334418A (zh) | 2021-10-29 | 2023-09-01 | 美商艾拉倫製藥股份有限公司 | 杭丁頓（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法 |
| EP4469575A2 (en) | 2022-01-24 | 2024-12-04 | Alnylam Pharmaceuticals, Inc. | Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof |
| WO2023144127A1 (en) | 2022-01-31 | 2023-08-03 | Ags Therapeutics Sas | Extracellular vesicles from microalgae, their biodistribution upon administration, and uses |
| IL315546A (en) | 2022-03-16 | 2024-11-01 | Empirico Inc | GALNAC compositions to improve siRNA bioavailability |
| US12258563B2 (en) | 2022-03-28 | 2025-03-25 | Empirico Inc. | Modified oligonucleotides |
| JP2025516680A (ja) | 2022-05-13 | 2025-05-30 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | 一本鎖ループオリゴヌクレオチド |
| WO2023232976A1 (en) | 2022-06-03 | 2023-12-07 | Ags Therapeutics Sas | Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses |
| EP4547683A2 (en) | 2022-06-30 | 2025-05-07 | Alnylam Pharmaceuticals, Inc. | Cyclic-disulfide modified phosphate based oligonucleotide prodrugs |
| JP2025532985A (ja) | 2022-09-30 | 2025-10-03 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | 修飾二本鎖ｒｎａ剤 |
| EP4608424A1 (en) | 2022-10-24 | 2025-09-03 | AGS Therapeutics SAS | Extracellular vesicles from microalgae, their biodistribution upon intranasal administration, and uses thereof |
| CN121241143A (zh) * | 2023-03-23 | 2025-12-30 | 阿塔兰塔治疗公司 | 用于改善的基因沉默的组合物和方法 |
| CN121816199A (zh) | 2023-04-12 | 2026-04-07 | 阿尔尼拉姆制药公司 | 双链rna药剂的肝外递送 |
| CN121729491A (zh) | 2023-05-12 | 2026-03-24 | 阿尔尼拉姆制药公司 | 单链环寡核苷酸 |
| IL326940A (en) | 2023-09-21 | 2026-05-01 | Alnylam Pharmaceuticals Inc | Activin A Receptor Type 1C (ACVR1C) IRNA Compositions and Methods of Using Them |
| WO2025176847A1 (en) | 2024-02-21 | 2025-08-28 | Ags Therapeutics Sas | Ocular delivery of active agents via microalgae extracellular vesicles |
| WO2025259747A2 (en) | 2024-06-12 | 2025-12-18 | Alnylam Pharmaceuticals, Inc. | Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| FR2645866B1 (fr) * | 1989-04-17 | 1991-07-05 | Centre Nat Rech Scient | Nouvelles lipopolyamines, leur preparation et leur emploi |
| US5283185A (en) * | 1991-08-28 | 1994-02-01 | University Of Tennessee Research Corporation | Method for delivering nucleic acids into cells |
| US5691316A (en) * | 1994-06-01 | 1997-11-25 | Hybridon, Inc. | Cyclodextrin cellular delivery system for oligonucleotides |
| US6509323B1 (en) * | 1998-07-01 | 2003-01-21 | California Institute Of Technology | Linear cyclodextrin copolymers |
| US6066500A (en) * | 1999-06-25 | 2000-05-23 | Isis Pharmaceuticals Inc. | Antisense modulation of Beta catenin expression |
| 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 |
| US6627425B1 (en) * | 2000-06-02 | 2003-09-30 | Millennium Pharmaceuticals, Inc. | Human glucose-6-phosphatase molecules and uses thereof |
| TWI321054B (en) * | 2000-12-19 | 2010-03-01 | California Inst Of Techn | Compositions containing inclusion complexes |
| US7517864B2 (en) * | 2001-05-18 | 2009-04-14 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA) |
| US7888324B2 (en) * | 2001-08-01 | 2011-02-15 | Genzyme Corporation | Antisense modulation of apolipoprotein B expression |
| WO2003046173A1 (en) * | 2001-11-28 | 2003-06-05 | Center For Advanced Science And Technology Incubation, Ltd. | siRNA EXPRESSION SYSTEM AND PROCESS FOR PRODUCING FUNCTIONAL GENE-KNOCKDOWN CELLS AND THE LIKE USING THE SAME |
| US20050233342A1 (en) * | 2003-03-07 | 2005-10-20 | Muthiah Manoharan | Methods of preventing off-target gene silencing |
| US20040198640A1 (en) * | 2003-04-02 | 2004-10-07 | Dharmacon, Inc. | Stabilized polynucleotides for use in RNA interference |
| AU2004252442B2 (en) * | 2003-06-02 | 2010-04-08 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of FNAi |
| EP1666593A4 (en) * | 2003-08-18 | 2009-07-15 | Japan Health Science Found | IMPROVED SIRNA MOLECULE AND METHOD FOR INHIBITING GENE EXPRESSION USED THEREOF |
| US20080249039A1 (en) * | 2004-01-30 | 2008-10-09 | Santaris Pharma A/S | Modified Short Interfering Rna (Modified Sirna) |
| WO2006093526A2 (en) * | 2004-07-21 | 2006-09-08 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a modified or non-natural nucleobase |
| US20080213891A1 (en) * | 2004-07-21 | 2008-09-04 | Alnylam Pharmaceuticals, Inc. | RNAi Agents Comprising Universal Nucleobases |
| CA2574603C (en) * | 2004-08-04 | 2014-11-04 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase |
| US20060089324A1 (en) * | 2004-10-22 | 2006-04-27 | Sailen Barik | RNAi modulation of RSV, PIV and other respiratory viruses and uses thereof |
| EP1951737A4 (en) * | 2005-11-01 | 2009-07-01 | Alnylam Pharmaceuticals Inc | INHIBITION OF INFLUENZA VIRUS REPLICATION BY RNA INTERFERENCE |
| WO2009142822A2 (en) * | 2008-03-26 | 2009-11-26 | Alnylam Pharmaceuticals, Inc. | 2-f modified rna interference agents |
| JP2012147677A (ja) * | 2009-05-14 | 2012-08-09 | Nippon Shinyaku Co Ltd | 二本鎖修飾ｒｎａ |
-
2009
- 2009-07-24 CA CA2732229A patent/CA2732229C/en active Active
- 2009-07-24 EP EP09790796.8A patent/EP2321414B1/en active Active
- 2009-07-24 WO PCT/US2009/051648 patent/WO2010011895A1/en not_active Ceased
- 2009-07-24 US US13/055,894 patent/US20110223665A1/en not_active Abandoned
- 2009-07-24 JP JP2011520219A patent/JP2011528910A/ja active Pending
- 2009-07-24 AU AU2009273878A patent/AU2009273878A1/en not_active Abandoned
-
2013
- 2013-03-20 US US13/847,955 patent/US20130196434A1/en not_active Abandoned
-
2016
- 2016-04-14 AU AU2016202344A patent/AU2016202344B2/en active Active
- 2016-09-28 JP JP2016189465A patent/JP6476155B2/ja active Active
Also Published As
| Publication number | Publication date |
|---|---|
| CA2732229A1 (en) | 2010-01-28 |
| WO2010011895A1 (en) | 2010-01-28 |
| JP2011528910A (ja) | 2011-12-01 |
| EP2321414A1 (en) | 2011-05-18 |
| AU2016202344A1 (en) | 2016-05-05 |
| US20130196434A1 (en) | 2013-08-01 |
| AU2016202344B2 (en) | 2017-11-30 |
| JP6476155B2 (ja) | 2019-02-27 |
| EP2321414B1 (en) | 2018-01-10 |
| US20110223665A1 (en) | 2011-09-15 |
| JP2017002079A (ja) | 2017-01-05 |
| AU2009273878A1 (en) | 2010-01-28 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AU2016202344B2 (en) | Enhancement of siRNA silencing activity using universal bases or mismatches in the sense strand | |
| AU2004233092B2 (en) | Modified iRNA agents | |
| JP2011528910A5 (enExample) | ||
| US20110269814A1 (en) | 2'-f modified rna interference agents | |
| EP3604533A1 (en) | Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components | |
| AU2004232964B2 (en) | Protected monomers | |
| WO2011109427A2 (en) | Improving the biological activity of sirna through modulation of its thermodynamic profile | |
| AU2013205519B2 (en) | Modified irna agents | |
| AU2017206154A1 (en) | Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20140617 |
|
| MPN | Maintenance fee for patent paid |
Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 15TH ANNIV.) - STANDARD Year of fee payment: 15 |
|
| MPN | Maintenance fee for patent paid |
Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 16TH ANNIV.) - STANDARD Year of fee payment: 16 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20250701 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20250701 |