NZ580712A - Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomers - Google Patents
Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomersInfo
- Publication number
- NZ580712A NZ580712A NZ580712A NZ58071208A NZ580712A NZ 580712 A NZ580712 A NZ 580712A NZ 580712 A NZ580712 A NZ 580712A NZ 58071208 A NZ58071208 A NZ 58071208A NZ 580712 A NZ580712 A NZ 580712A
- Authority
- NZ
- New Zealand
- Prior art keywords
- rna
- monomers
- oligonucleotide
- monomer
- hydroxymethyl
- Prior art date
Links
Classifications
-
- 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
- C12N15/113—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- 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
-
- 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
-
- 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
-
- 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
- C12N2310/31—Chemical structure of the backbone
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- 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
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Public Health (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DKPA200700751 | 2007-05-22 | ||
| DKPA200701718 | 2007-11-30 | ||
| DKPA200701785 | 2007-12-14 | ||
| DKPA200800534 | 2008-04-11 | ||
| PCT/US2008/064417 WO2008147824A2 (en) | 2007-05-22 | 2008-05-21 | Hydroxymethyl substituted rna oligonucleotides and rna complexes |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NZ580712A true NZ580712A (en) | 2011-12-22 |
Family
ID=40075730
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ580712A NZ580712A (en) | 2007-05-22 | 2008-05-21 | Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomers |
Country Status (17)
| Country | Link |
|---|---|
| US (6) | US8314227B2 (enExample) |
| EP (2) | EP2162538B1 (enExample) |
| JP (2) | JP5726520B2 (enExample) |
| KR (2) | KR101629017B1 (enExample) |
| CN (2) | CN104480112B (enExample) |
| AU (1) | AU2008256871B2 (enExample) |
| BR (1) | BRPI0811170B8 (enExample) |
| CA (1) | CA2687850C (enExample) |
| DK (1) | DK2162538T3 (enExample) |
| ES (1) | ES2573936T3 (enExample) |
| IL (1) | IL202040A (enExample) |
| MX (1) | MX2009012568A (enExample) |
| MY (1) | MY153691A (enExample) |
| NZ (1) | NZ580712A (enExample) |
| PL (1) | PL2162538T3 (enExample) |
| WO (1) | WO2008147824A2 (enExample) |
| ZA (1) | ZA200907529B (enExample) |
Families Citing this family (218)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| USRE44779E1 (en) | 1997-03-07 | 2014-02-25 | Santaris Pharma A/S | Bicyclonucleoside and oligonucleotide analogues |
| US9657294B2 (en) | 2002-02-20 | 2017-05-23 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| US9181551B2 (en) | 2002-02-20 | 2015-11-10 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| WO2008049078A1 (en) | 2006-10-18 | 2008-04-24 | Nastech Pharmaceutical Company Inc. | Nicked or gapped nucleic acid molecules and uses thereof |
| CN104480112B (zh) * | 2007-05-22 | 2018-06-12 | 阿克丘勒斯治疗公司 | 用于治疗的una寡聚体 |
| US20100015708A1 (en) * | 2008-06-18 | 2010-01-21 | Mdrna, Inc. | Ribonucleic acids with non-standard bases and uses thereof |
| EP2315831A2 (en) * | 2008-08-05 | 2011-05-04 | Marina Biotech, Inc. | Nucleic acid compounds for inhibiting plk1 gene expression and uses thereof |
| AU2009305639B2 (en) | 2008-10-16 | 2016-06-23 | Marina Biotech, Inc. | Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics |
| CA2744093A1 (en) * | 2008-12-03 | 2010-06-10 | Marina Biotech, Inc. | Una oligomer structures for therapeutic agents |
| KR20120128598A (ko) | 2009-11-04 | 2012-11-27 | 마리나 바이오테크, 인크. | 활성 생성 전달 분자들 |
| BR112012014760A2 (pt) | 2009-12-18 | 2016-06-14 | Novartis Ag | "composições orgânicas para tratar doenças relacionadas a hsf1, bem como seu uso" |
| EP3214174B1 (en) | 2010-03-04 | 2019-10-16 | InteRNA Technologies B.V. | A mirna molecule defined by its source and its diagnostic and therapeutic uses in diseases or conditions associated with emt |
| WO2011133584A2 (en) * | 2010-04-19 | 2011-10-27 | Marina Biotech, Inc. | Nucleic acid compounds for inhibiting hras gene expression and uses thereof |
| WO2011133876A2 (en) * | 2010-04-22 | 2011-10-27 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising acyclic and abasic nucleosides and analogs |
| US8344127B2 (en) | 2010-04-23 | 2013-01-01 | Novartis Ag | Organic compositions to treat beta-ENaC-related diseases |
| WO2011139843A2 (en) * | 2010-04-28 | 2011-11-10 | Marina Biotech, Inc. | Multi-sirna compositions for reducing gene expression |
| AU2011274619B2 (en) | 2010-07-06 | 2016-11-10 | Interna Technologies Bv | miRNA and its diagnostic and therapeutic uses in diseases or conditions associated with melanoma, or in diseases or conditions associated with activated BRAF pathway |
| US9243246B2 (en) | 2010-08-24 | 2016-01-26 | Sirna Therapeutics, Inc. | Single-stranded RNAi agents containing an internal, non-nucleic acid spacer |
| DK2632472T3 (en) | 2010-10-29 | 2018-03-19 | Sirna Therapeutics Inc | RNA INTERFERENCE-MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERRING NUCLEIC ACIDS (SINA) |
| JP6157354B2 (ja) | 2010-11-10 | 2017-07-05 | ナイジェル, エル. ウェブ, | ヌクリオン(Nuclions)及びリボキャプシド(ribocapsids) |
| EP2474617A1 (en) | 2011-01-11 | 2012-07-11 | InteRNA Technologies BV | Mir for treating neo-angiogenesis |
| CN102719434A (zh) * | 2011-03-31 | 2012-10-10 | 百奥迈科生物技术有限公司 | 抑制rna干扰脱靶效应的特异性修饰 |
| EA201490553A1 (ru) | 2011-09-02 | 2014-08-29 | Новартис Аг | Органические композиции для лечения связанных с hsf1 заболеваний |
| EP2794881B1 (en) | 2011-12-22 | 2018-06-27 | InteRNA Technologies B.V. | Mirna for treating head and neck cancer |
| HK1205758A1 (en) | 2012-01-09 | 2015-12-24 | Novartis Ag | Rnai agents to treat beta-catenin related diseases |
| US10501513B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides for the production of oncology-related proteins and peptides |
| JP6189415B2 (ja) | 2012-04-02 | 2017-08-30 | モデルナティエックス インコーポレイテッドModernaTX,Inc. | 細胞質および細胞骨格タンパク質の産生のための修飾ポリヌクレオチド |
| US10501512B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides |
| US20150238515A1 (en) | 2012-05-02 | 2015-08-27 | Arrowhead Research Corporation | Organic compositions to treat kras-related diseases |
| EP2917348A1 (en) | 2012-11-06 | 2015-09-16 | InteRNA Technologies B.V. | Combination for use in treating diseases or conditions associated with melanoma, or treating diseases or conditions associated with activated b-raf pathway |
| SG11201506805QA (en) | 2013-02-28 | 2015-09-29 | Arrowhead Res Corp | Organic compositions to treat epas1-related diseases |
| WO2015020960A1 (en) | 2013-08-09 | 2015-02-12 | Novartis Ag | Novel lncrna polynucleotides |
| JP2016528897A (ja) * | 2013-08-16 | 2016-09-23 | ラナ セラピューティクス インコーポレイテッド | Rnaを調節するための組成物および方法 |
| CA2925107A1 (en) | 2013-10-02 | 2015-04-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the lect2 gene |
| US10227588B2 (en) | 2013-10-04 | 2019-03-12 | Novartis Ag | 3′end caps for RNAi agents for use in RNA interference |
| WO2015051044A2 (en) | 2013-10-04 | 2015-04-09 | Novartis Ag | Novel formats for organic compounds for use in rna interference |
| ES2804510T3 (es) | 2013-10-04 | 2021-02-08 | Alnylam Pharmaceuticals Inc | Composiciones y métodos para inhibir la expresión del gen ALAS1 |
| EP3052627B1 (en) | 2013-10-04 | 2018-08-22 | Novartis AG | Novel formats for organic compounds for use in rna interference |
| CN105792832B (zh) | 2013-10-04 | 2021-03-23 | 诺华股份有限公司 | 用于治疗乙肝病毒的有机化合物 |
| WO2015051135A2 (en) | 2013-10-04 | 2015-04-09 | Novartis Ag | Organic compositions to treat hepcidin-related diseases |
| IL314045A (en) | 2013-12-12 | 2024-09-01 | Alnylam Pharmaceuticals Inc | Complement component irna compositions and methods of use thereof |
| JP6594902B2 (ja) | 2014-02-11 | 2019-10-23 | 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 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
| US9856475B2 (en) | 2014-03-25 | 2018-01-02 | Arcturus Therapeutics, Inc. | Formulations for treating amyloidosis |
| JP6771387B2 (ja) | 2014-03-25 | 2020-10-21 | アークトゥラス・セラピューティクス・インコーポレイテッドArcturus Therapeutics,Inc. | 遺伝子サイレンシング用トランスサイレチン対立遺伝子選択的unaオリゴマー |
| CN110846316B (zh) | 2014-03-25 | 2023-10-31 | 阿克丘勒斯治疗公司 | 在基因沉默中具有降低的脱靶效应的una寡聚物 |
| TW201607559A (zh) | 2014-05-12 | 2016-03-01 | 阿尼拉製藥公司 | 治療serpinc1相關疾患之方法和組成物 |
| SG10202104570TA (en) | 2014-05-22 | 2021-06-29 | Alnylam Pharmaceuticals Inc | Angiotensinogen (agt) irna compositions and methods of use thereof |
| TW201620526A (zh) | 2014-06-17 | 2016-06-16 | 愛羅海德研究公司 | 用於抑制α-1抗胰蛋白酶基因表現之組合物及方法 |
| JP6778175B2 (ja) | 2014-07-16 | 2020-10-28 | ノバルティス アーゲー | 脂質ナノ粒子ホスト中に核酸を封入する方法 |
| WO2016011123A1 (en) | 2014-07-16 | 2016-01-21 | Arrowhead Research Corporation | Organic compositions to treat apoc3-related diseases |
| EP3191591A1 (en) | 2014-09-12 | 2017-07-19 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting complement component c5 and methods of use thereof |
| CA2963271A1 (en) | 2014-10-02 | 2016-04-07 | Protiva Biotherapeutics, Inc. | Compositions and methods for silencing hepatitis b virus gene expression |
| EP3207138B1 (en) | 2014-10-17 | 2020-07-15 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof |
| EP3904519A1 (en) | 2014-10-30 | 2021-11-03 | Genzyme Corporation | Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof |
| US20160130567A1 (en) | 2014-11-02 | 2016-05-12 | Arcturus Therapeutics, Inc. | Messenger una molecules and uses thereof |
| JOP20200092A1 (ar) | 2014-11-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها |
| HK1244843A1 (zh) | 2014-11-17 | 2018-08-17 | Alnylam Pharmaceuticals, Inc. | 载脂蛋白c3(apoc3)irna组合物及其使用方法 |
| EP3256587A2 (en) | 2015-02-13 | 2017-12-20 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
| US10758558B2 (en) | 2015-02-13 | 2020-09-01 | Translate Bio Ma, Inc. | Hybrid oligonucleotides and uses thereof |
| EP3277289A4 (en) * | 2015-04-01 | 2018-12-05 | Arcturus Therapeutics, Inc. | Therapeutic una oligomers and uses thereof |
| US10745702B2 (en) | 2015-04-08 | 2020-08-18 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the LECT2 gene |
| WO2016197132A1 (en) | 2015-06-04 | 2016-12-08 | Protiva Biotherapeutics Inc. | Treating hepatitis b virus infection using crispr |
| EP4365291A3 (en) | 2015-06-12 | 2024-08-14 | Alnylam Pharmaceuticals, Inc. | Complement component c5 irna compositions and methods of use thereof |
| EP3310918B1 (en) | 2015-06-18 | 2020-08-05 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof |
| WO2016209862A1 (en) | 2015-06-23 | 2016-12-29 | Alnylam Pharmaceuticals, Inc. | Glucokinase (gck) irna compositions and methods of use thereof |
| WO2017011286A1 (en) | 2015-07-10 | 2017-01-19 | Alnylam Pharmaceuticals, Inc. | Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof |
| CA2996722A1 (en) | 2015-07-17 | 2017-01-26 | Arcturus Therapeutics, Inc. | Compositions and agents against hepatitis b virus and uses thereof |
| WO2017015671A1 (en) | 2015-07-23 | 2017-01-26 | Arcturus Therapeutics, Inc. | Compositions for treating amyloidosis |
| CN108350455A (zh) | 2015-07-29 | 2018-07-31 | 阿布特斯生物制药公司 | 用于使b型肝炎病毒基因表达沉默的组合物和方法 |
| KR102866196B1 (ko) | 2015-09-02 | 2025-09-29 | 알닐람 파마슈티칼스 인코포레이티드 | 프로그램된 세포사 1 리간드 1 (PD-L1) iRNA 조성물 및 그의 사용 방법 |
| JP6799058B2 (ja) | 2015-09-21 | 2020-12-09 | 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. | アレル選択的な遺伝子編集およびその使用 |
| IL259795B2 (en) | 2015-12-07 | 2024-04-01 | Genzyme Corp | Methods and compositions for treating a serpinc1-associated disorder |
| MA45295A (fr) | 2016-04-19 | 2019-02-27 | Alnylam Pharmaceuticals Inc | Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser |
| CA3023764A1 (en) | 2016-06-06 | 2017-12-14 | Arrowhead Pharmaceuticals, Inc. | 5'-cyclo-phosphonate modified nucleotides |
| US20190256845A1 (en) | 2016-06-10 | 2019-08-22 | Alnylam Pharmaceuticals, Inc. | COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) |
| AU2017286980B2 (en) | 2016-06-30 | 2023-10-26 | Arbutus Biopharma Corporation | Compositions and methods for delivering messenger RNA |
| WO2018013999A1 (en) | 2016-07-15 | 2018-01-18 | Am Chemicals Llc | Non-nucleosidic solid supports and phosphoramidite building blocks for oligonucleotide synthesis |
| EP3500294A4 (en) | 2016-08-22 | 2020-07-29 | Arbutus Biopharma Corporation | ANTI-PD-1 ANTIBODIES, OR THEIR FRAGMENTS, FOR THE TREATMENT OF HEPATITIS B |
| US10487105B2 (en) | 2016-10-19 | 2019-11-26 | Arcturus Therapeutics, Inc. | Trinucleotide MRNA cap analogs |
| TWI788312B (zh) | 2016-11-23 | 2023-01-01 | 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 |
| EP3555292A1 (en) | 2016-12-16 | 2019-10-23 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions |
| US10450565B2 (en) | 2017-01-10 | 2019-10-22 | Arrowhead Pharmaceuticals, Inc. | Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use |
| SG11201912243PA (en) | 2017-07-10 | 2020-01-30 | Genzyme Corp | Methods and compositions for treating a bleeding event in a subject having hemophilia |
| SG11201912178VA (en) | 2017-09-11 | 2020-01-30 | Arrowhead Pharmaceuticals Inc | Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3) |
| US11110180B2 (en) | 2017-10-04 | 2021-09-07 | Avidity Biosciences Inc. | Nucleic acid-polypeptide compositions and uses thereof |
| EP3704250A1 (en) | 2017-11-03 | 2020-09-09 | InteRNA Technologies B.V. | Mirna molecule, equivalent, antagomir, or source thereof for treating and/or diagnosing a condition and/or a disease associated with neuronal deficiency or for neuronal (re)generation |
| EP3710587A1 (en) | 2017-11-16 | 2020-09-23 | Alnylam Pharmaceuticals, Inc. | Kisspeptin 1 (kiss1) irna compositions and methods of use thereof |
| EA202091520A1 (ru) | 2017-12-18 | 2020-10-05 | Элнилэм Фармасьютикалз, Инк. | Композиции на основе irna против бокса-1 высокомобильной группы (hmgb1) и способы их применения |
| CN111936150A (zh) | 2018-02-12 | 2020-11-13 | 因特尔纳技术有限公司 | 抗癌微小rna及其脂质制剂 |
| US12083224B2 (en) | 2018-03-30 | 2024-09-10 | Arcturus Therapeutics, Inc. | Lipid particles for nucleic acid delivery |
| TWI851574B (zh) | 2018-05-14 | 2024-08-11 | 美商阿尼拉製藥公司 | 血管收縮素原(AGT)iRNA組成物及其使用方法 |
| WO2020033748A1 (en) | 2018-08-08 | 2020-02-13 | Arcturus Therapeutics, Inc. | Compositions and agents against nonalcoholic steatohepatitis |
| JP2021534101A (ja) | 2018-08-09 | 2021-12-09 | ヴェルソー セラピューティクス, インコーポレイテッド | Ccr2及びcsf1rを標的とするためのオリゴヌクレオチド組成物ならびにその使用 |
| EP3837366A1 (en) | 2018-08-13 | 2021-06-23 | Alnylam Pharmaceuticals, Inc. | Hepatitis b virus (hbv) dsrna agent compositions and methods of use thereof |
| TW202020157A (zh) | 2018-08-16 | 2020-06-01 | 美商艾爾妮蘭製藥公司 | 用於抑制lect2基因表現之組合物及方法 |
| EP3843845A4 (en) | 2018-08-29 | 2022-05-11 | University Of Massachusetts | INHIBITION OF PROTEIN KINASE FOR THE TREATMENT OF FRIEDREICH'S ATAXIA |
| WO2020060986A1 (en) | 2018-09-18 | 2020-03-26 | Alnylam Pharmaceuticals, Inc. | Ketohexokinase (khk) irna compositions and methods of use thereof |
| US10913951B2 (en) | 2018-10-31 | 2021-02-09 | University of Pittsburgh—of the Commonwealth System of Higher Education | Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure |
| EP3894559A4 (en) | 2018-12-03 | 2023-04-05 | Triplet Therapeutics, Inc. | METHODS OF TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS RELATED TO MLH3 ACTIVITY |
| WO2020132521A1 (en) | 2018-12-20 | 2020-06-25 | Praxis Precision Medicines, Inc. | Compositions and methods for the treatment of kcnt1 related disorders |
| CA3120580A1 (en) | 2019-01-09 | 2020-07-16 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use |
| AU2020209186A1 (en) | 2019-01-16 | 2021-09-09 | Genzyme Corporation | Serpinc1 iRNA compositions and methods of use thereof |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
| WO2020247818A1 (en) * | 2019-06-06 | 2020-12-10 | Avidity Biosciences, Inc. | Una amidites and uses thereof |
| WO2020247782A1 (en) | 2019-06-06 | 2020-12-10 | Avidity Biosciences, Inc. | Nucleic acid-polypeptide compositions and uses thereof |
| EP4007812A1 (en) | 2019-08-01 | 2022-06-08 | Alnylam Pharmaceuticals, Inc. | Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof |
| WO2021022108A2 (en) | 2019-08-01 | 2021-02-04 | Alnylam Pharmaceuticals, Inc. | CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| EP4013870A1 (en) | 2019-08-13 | 2022-06-22 | Alnylam Pharmaceuticals, Inc. | Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof |
| AU2020343255A1 (en) | 2019-09-03 | 2022-03-24 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the LECT2 gene |
| WO2021044004A1 (en) * | 2019-09-05 | 2021-03-11 | Sanofi | Oligonucleotides containing nucleotide analogs |
| EP4038189A1 (en) | 2019-10-04 | 2022-08-10 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for silencing ugt1a1 gene expression |
| US12486512B2 (en) | 2019-10-18 | 2025-12-02 | Alnylam Pharmaceuticals, Inc. | Solute carrier family member iRNA compositions and methods of use thereof |
| MX2022004726A (es) | 2019-10-22 | 2022-05-13 | Alnylam Pharmaceuticals Inc | Composiciones de acido ribonucleico de interferencia (arni) de componente c3 de complemento y metodos de uso de las mismas. |
| MX2022004388A (es) | 2019-11-01 | 2022-05-06 | Alnylam Pharmaceuticals Inc | Composiciones de agentes de arni de huntingtina (htt) y metodos de uso de las mismas. |
| US20230040920A1 (en) | 2019-11-01 | 2023-02-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for silencing dnajb1-prkaca fusion gene expression |
| US20230056569A1 (en) | 2019-11-22 | 2023-02-23 | Alnylam Pharmaceuticals, Inc. | Ataxin3 (atxn3) rnai agent compositions and methods of use thereof |
| MX2022006433A (es) | 2019-12-13 | 2022-06-23 | Alnylam Pharmaceuticals Inc | Composiciones de agentes de acido ribonucleico de interferencia (arni) del marco de lectura abierto 72 del cromosoma 9 humano (c9orf72) y metodos de uso de los mismos. |
| WO2021126734A1 (en) | 2019-12-16 | 2021-06-24 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
| AU2021220765A1 (en) | 2020-02-10 | 2022-09-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for silencing VEGF-A expression |
| AU2021224778A1 (en) | 2020-02-18 | 2022-09-29 | Alnylam Pharmaceuticals, Inc. | Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof |
| 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 |
| JP2023516095A (ja) | 2020-03-06 | 2023-04-17 | 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 | ケトヘキソキナーゼ(KHK)iRNA組成物およびその使用方法 |
| WO2021188611A1 (en) | 2020-03-18 | 2021-09-23 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant |
| EP4127168A1 (en) | 2020-03-26 | 2023-02-08 | Alnylam Pharmaceuticals, Inc. | Coronavirus irna compositions and methods of use thereof |
| US20230190785A1 (en) | 2020-03-30 | 2023-06-22 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for silencing dnajc15 gene expression |
| WO2021207167A1 (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 |
| CA3179678A1 (en) | 2020-04-07 | 2021-10-14 | 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 |
| IL297680A (en) | 2020-04-30 | 2022-12-01 | Alnylam Pharmaceuticals Inc | Complement factor b (cfb) irna compositions and methods of use thereof |
| EP4150087A1 (en) | 2020-05-15 | 2023-03-22 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2) |
| EP4150086A1 (en) | 2020-05-15 | 2023-03-22 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2) |
| EP4150077A1 (en) | 2020-05-15 | 2023-03-22 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1) |
| EP4150090A1 (en) | 2020-05-15 | 2023-03-22 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of otoferlin (otof) |
| WO2021231675A1 (en) | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1) |
| WO2021231698A1 (en) | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl) |
| WO2021231680A1 (en) | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2) |
| WO2021231691A1 (en) | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi) |
| US20230183707A1 (en) | 2020-05-21 | 2023-06-15 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting marc1 gene expression |
| AR122534A1 (es) | 2020-06-03 | 2022-09-21 | Triplet Therapeutics Inc | Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3 |
| EP4162050A1 (en) | 2020-06-09 | 2023-04-12 | Alnylam Pharmaceuticals, Inc. | Rnai compositions and methods of use thereof for delivery by inhalation |
| US20230203496A1 (en) | 2020-06-09 | 2023-06-29 | Alnylam Pharmaceuticals, Inc. | Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression |
| TW202214856A (zh) | 2020-06-18 | 2022-04-16 | 美商阿尼拉製藥公司 | 黃嘌呤脫氫酶(XDH)iRNA組成物及其使用之方法 |
| TW202227102A (zh) | 2020-09-22 | 2022-07-16 | 瑞典商阿斯特捷利康公司 | 治療脂肪肝病之方法 |
| EP4217489A1 (en) | 2020-09-24 | 2023-08-02 | Alnylam Pharmaceuticals, Inc. | Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof |
| WO2022076291A1 (en) | 2020-10-05 | 2022-04-14 | Alnylam Pharmaceuticals, Inc. | G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof |
| WO2022087329A1 (en) | 2020-10-23 | 2022-04-28 | Alnylam Pharmaceuticals, Inc. | Mucin 5b (muc5b) irna compositions and methods of use thereof |
| IL302709A (en) | 2020-11-13 | 2023-07-01 | Alnylam Pharmaceuticals Inc | Coagulation factor iRNA compositions (F5) and methods of using them |
| EP4259795A1 (en) | 2020-12-08 | 2023-10-18 | Alnylam Pharmaceuticals, Inc. | Coagulation factor x (f10) irna compositions and methods of use thereof |
| WO2022150260A1 (en) | 2021-01-05 | 2022-07-14 | Alnylam Pharmaceuticals, Inc. | COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| CA3210763A1 (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 |
| WO2022182864A1 (en) | 2021-02-25 | 2022-09-01 | Alnylam Pharmaceuticals, Inc. | Prion protein (prnp) irna compositions and methods and methods of use thereof |
| IL305153A (en) | 2021-02-26 | 2023-10-01 | Alnylam Pharmaceuticals Inc | Ketohexokinase (KHK) iRNA compositions and methods of using them |
| AU2022231003A1 (en) | 2021-03-04 | 2023-09-14 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
| EP4305169A1 (en) | 2021-03-12 | 2024-01-17 | Alnylam Pharmaceuticals, Inc. | Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof |
| KR20230162024A (ko) | 2021-03-29 | 2023-11-28 | 알닐람 파마슈티칼스 인코포레이티드 | 헌팅틴(HTT) iRNA 제제 조성물 및 이의 사용 방법 |
| EP4314293A1 (en) | 2021-04-01 | 2024-02-07 | Alnylam Pharmaceuticals, Inc. | Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof |
| IL307926A (en) | 2021-04-26 | 2023-12-01 | Alnylam Pharmaceuticals Inc | Transmembrane assemblies, serine 6 ((TMPRSS6 IRNA) and methods of using them |
| WO2022232343A1 (en) | 2021-04-29 | 2022-11-03 | Alnylam Pharmaceuticals, Inc. | Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof |
| EP4341401A1 (en) | 2021-05-18 | 2024-03-27 | Alnylam Pharmaceuticals, Inc. | Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof |
| WO2022246023A1 (en) | 2021-05-20 | 2022-11-24 | Korro Bio, Inc. | Methods and compositions for adar-mediated editing |
| WO2022256283A2 (en) | 2021-06-01 | 2022-12-08 | Korro Bio, Inc. | Methods for restoring protein function using adar |
| EP4347823A1 (en) | 2021-06-02 | 2024-04-10 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) 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 |
| WO2022260939A2 (en) | 2021-06-08 | 2022-12-15 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders |
| EP4363574A1 (en) | 2021-06-29 | 2024-05-08 | Korro Bio, Inc. | Methods and compositions for adar-mediated editing |
| US20230194709A9 (en) | 2021-06-29 | 2023-06-22 | Seagate Technology Llc | Range information detection using coherent pulse sets with selected waveform characteristics |
| TW202333748A (zh) | 2021-07-19 | 2023-09-01 | 美商艾拉倫製藥股份有限公司 | 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物 |
| IL309905A (en) | 2021-07-23 | 2024-03-01 | Alnylam Pharmaceuticals Inc | IRNA compositions in β-catenin (CTNNB1) and methods of using them |
| EP4377458A1 (en) | 2021-07-29 | 2024-06-05 | Alnylam Pharmaceuticals, Inc. | 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof |
| JP2024530018A (ja) | 2021-08-03 | 2024-08-14 | 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組成物およびその使用方法 |
| EP4381071A1 (en) | 2021-08-04 | 2024-06-12 | Alnylam Pharmaceuticals, Inc. | Irna compositions and methods for silencing angiotensinogen (agt) |
| EP4384617A1 (en) | 2021-08-13 | 2024-06-19 | Alnylam Pharmaceuticals, Inc. | Factor xii (f12) irna compositions and methods of use thereof |
| 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. |
| EP4401742A2 (en) | 2021-09-17 | 2024-07-24 | Alnylam Pharmaceuticals, Inc. | Irna compositions and methods for silencing complement component 3 (c3) |
| CN118159654A (zh) | 2021-09-20 | 2024-06-07 | 阿尔尼拉姆医药品有限公司 | 抑制素亚基βE(INHBE)调节剂组合物及其使用方法 |
| KR20240067943A (ko) | 2021-09-24 | 2024-05-17 | 알닐람 파마슈티칼스 인코포레이티드 | 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법 |
| WO2023056440A1 (en) | 2021-10-01 | 2023-04-06 | Adarx Pharmaceuticals, Inc. | Prekallikrein-modulating compositions and methods of use thereof |
| AU2022370009A1 (en) | 2021-10-22 | 2024-05-16 | Korro Bio, Inc. | Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing |
| CA3234636A1 (en) | 2021-10-29 | 2023-05-04 | Alnylam Pharmaceuticals, Inc. | Complement factor b (cfb) irna compositions and methods of use thereof |
| EP4423272A2 (en) | 2021-10-29 | 2024-09-04 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| KR20240126870A (ko) | 2021-12-22 | 2024-08-21 | 캠프4 테라퓨틱스 코포레이션 | 조절 rna들을 표적으로 하는 안티센스 올리고뉴클레오티드를 사용한 유전자 전사의 조절 |
| EP4469575A2 (en) | 2022-01-24 | 2024-12-04 | Alnylam Pharmaceuticals, Inc. | Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof |
| KR20250022763A (ko) | 2022-06-10 | 2025-02-17 | 캠프4 테라퓨틱스 코포레이션 | 조절 rna를 표적으로 하는 안티센스 올리고뉴클레오타이드를 사용하여 프로그래뉼린 발현을 조절하는 방법 |
| CN120077130A (zh) | 2022-08-18 | 2025-05-30 | 阿尔尼拉姆医药品有限公司 | 通用非靶向sirna组合物及其使用方法 |
| EP4569113A1 (en) | 2022-09-15 | 2025-06-18 | Regeneron Pharmaceuticals, Inc. | 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof |
| JP2025540101A (ja) | 2022-12-01 | 2025-12-11 | キャンプ4 セラピューティクス コーポレイション | 調節rnaを標的とするアンチセンスオリゴヌクレオチドを用いたsyngap1遺伝子転写の調節 |
| IT202200026595A1 (it) | 2022-12-22 | 2024-06-22 | Fond Telethon Ets | Nuovi inibitori di regolatori epigenetici |
| WO2024168010A2 (en) | 2023-02-09 | 2024-08-15 | Alnylam Pharmaceuticals, Inc. | Reversir molecules and methods of use thereof |
| AU2024244071A1 (en) | 2023-04-06 | 2025-10-30 | Shanghai Argo Biopharmaceutical Co., Ltd. | Nucleoside analog for 5'-phosphonate modification and oligonucleotide prepared therefrom |
| CN121285630A (zh) | 2023-04-20 | 2026-01-06 | 阿达尔克斯制药有限公司 | Mapt调节组合物及其使用方法 |
| WO2024220746A2 (en) | 2023-04-21 | 2024-10-24 | Flagship Pioneering Innovations Vii, Llc | Rnai agents targeting fatty acid synthase and related methods |
| AU2024273075A1 (en) | 2023-05-12 | 2025-11-20 | Adarx Pharmaceuticals, Inc. | Nmda ligand conjugated compounds and uses thereof |
| TW202508606A (zh) * | 2023-05-12 | 2025-03-01 | 大陸商北京炫景瑞醫藥科技有限公司 | 修飾的核苷類似物及其用途與含核苷酸類似物的雙股寡核苷酸及其用途 |
| WO2024249328A2 (en) | 2023-05-26 | 2024-12-05 | Adarx Pharmaceuticals, Inc. | Sod1-modulating compositions and methods of use thereof |
| WO2024263694A1 (en) | 2023-06-20 | 2024-12-26 | Adarx Pharmaceuticals, Inc. | Lrrk2-modulating compositions and methods of use thereof |
| WO2025015335A1 (en) | 2023-07-13 | 2025-01-16 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| WO2025024334A1 (en) | 2023-07-21 | 2025-01-30 | Marrow Therapeutics, Inc. | Hematopoietic cell targeting conjugates and related methods |
| WO2025024493A1 (en) | 2023-07-25 | 2025-01-30 | Flagship Pioneering Innovations Vii, Llc | Cas endonucleases and related methods |
| US20250092426A1 (en) | 2023-07-25 | 2025-03-20 | Flagship Pioneering Innovations Vii, Llc | Cas endonucleases and related methods |
| WO2025034422A1 (en) | 2023-08-04 | 2025-02-13 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating ctnnb1-associated disorders |
| WO2025072331A1 (en) | 2023-09-26 | 2025-04-03 | Flagship Pioneering Innovations Vii, Llc | Cas nucleases and related methods |
| GB202314724D0 (en) | 2023-09-26 | 2023-11-08 | Astrazeneca Ab | compounds and methods for reducing psd3 expression |
| WO2025072699A1 (en) | 2023-09-27 | 2025-04-03 | Judo Bio, Inc. | Aminoglycosides for delivery of agents to the kidney |
| WO2025072672A2 (en) | 2023-09-27 | 2025-04-03 | Judo Bio, Inc. | Slc6a19-targeting modulatory nucleic acid agents |
| WO2025072713A1 (en) | 2023-09-27 | 2025-04-03 | Judo Bio, Inc. | Polymyxins for delivery of agents to the kidney |
| WO2025076031A2 (en) | 2023-10-03 | 2025-04-10 | Alnylam Pharmaceuticals, Inc. | Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof |
| WO2025096809A1 (en) | 2023-10-31 | 2025-05-08 | Korro Bio, Inc. | Oligonucleotides comprising phosphoramidate internucleotide linkages |
| WO2025117877A2 (en) | 2023-12-01 | 2025-06-05 | Flagship Pioneering Innovations Vii, Llc | Cas nucleases and related methods |
| WO2025128799A1 (en) | 2023-12-12 | 2025-06-19 | Korro Bio, Inc. | Double-stranded rna-editing oligonucleotides and uses thereof |
| WO2025140551A1 (en) | 2023-12-29 | 2025-07-03 | Neuro3 Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATING NaV1.7 (SCN9A) -ASSOCIATED DISORDERS |
| WO2025178854A2 (en) | 2024-02-19 | 2025-08-28 | Flagship Pioneering Innovations Vii, Llc | Rnai agents targeting cideb and related methods |
| US20250313840A1 (en) | 2024-03-20 | 2025-10-09 | Vertex Pharmaceuticals Incorporated | Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof |
| WO2025217275A2 (en) | 2024-04-10 | 2025-10-16 | Flagship Pioneering Innovations Vii, Llc | Immune cell targeted compositions and related methods |
| WO2025255388A1 (en) | 2024-06-05 | 2025-12-11 | Camp4 Therapeutics Corporation | Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas |
| WO2025259743A1 (en) | 2024-06-12 | 2025-12-18 | Alnylam Pharmaceuticals, Inc. | Dual conjugate compounds for extrahepatic delivery |
| 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 (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4199574A (en) | 1974-09-02 | 1980-04-22 | Burroughs Wellcome Co. | Methods and compositions for treating viral infections and guanine acyclic nucleosides |
| US4968686A (en) | 1988-04-08 | 1990-11-06 | The Regents Of The University Of Michigan | Acyclic pyrrolo [2,3-d]pyrimidine analogs as antiviral agents |
| US5652355A (en) * | 1992-07-23 | 1997-07-29 | Worcester Foundation For Experimental Biology | Hybrid oligonucleotide phosphorothioates |
| US5786359A (en) | 1994-05-27 | 1998-07-28 | The Scripps Research Institute | N9 alkyl or aralkyl derivatives of 7, 8-disubstituted guanines |
| US6608035B1 (en) | 1994-10-25 | 2003-08-19 | Hybridon, Inc. | Method of down-regulating gene expression |
| GB9505025D0 (en) | 1995-03-13 | 1995-05-03 | Medical Res Council | Chemical compounds |
| US5898031A (en) | 1996-06-06 | 1999-04-27 | Isis Pharmaceuticals, Inc. | Oligoribonucleotides for cleaving RNA |
| US6069132A (en) | 1996-08-14 | 2000-05-30 | Revanker; Ganapathi R. | Phosphazole compounds |
| US6506559B1 (en) | 1997-12-23 | 2003-01-14 | Carnegie Institute Of Washington | Genetic inhibition by double-stranded RNA |
| DE19956568A1 (de) | 1999-01-30 | 2000-08-17 | Roland Kreutzer | Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens |
| US7098192B2 (en) | 1999-04-08 | 2006-08-29 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of STAT3 expression |
| US6037176A (en) | 1999-06-25 | 2000-03-14 | Isis Pharmaceuticals Inc. | Antisense inhibition of integrin beta 3 expression |
| GB9925459D0 (en) | 1999-10-27 | 1999-12-29 | Plant Bioscience Ltd | Gene silencing |
| US7829693B2 (en) | 1999-11-24 | 2010-11-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
| WO2003070918A2 (en) | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | Rna interference by modified short interfering nucleic acid |
| 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 |
| US8202979B2 (en) | 2002-02-20 | 2012-06-19 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid |
| KR101215789B1 (ko) | 2000-03-30 | 2012-12-26 | 화이트헤드 인스티튜트 포 바이오메디칼 리서치 | Rna 간섭의 rna 서열 특이적인 매개체 |
| SI1407044T2 (en) | 2000-12-01 | 2018-03-30 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | Rna interference mediating small rna molecules |
| US8546143B2 (en) | 2001-01-09 | 2013-10-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
| CA2452458A1 (en) | 2001-07-03 | 2003-01-16 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
| ES2566561T3 (es) | 2001-07-12 | 2016-04-13 | University Of Massachusetts | Producción in vivo de ARN pequeños de interferencia que median el silenciamiento génico |
| EP1440079B1 (en) * | 2001-10-29 | 2006-12-13 | McGILL UNIVERSITY | Acyclic linker-containing oligonucleotides and uses thereof |
| US7388002B2 (en) | 2001-11-14 | 2008-06-17 | Biocryst Pharmaceuticals, Inc. | Nucleosides, preparation thereof and use as inhibitors of RNA viral polymerases |
| US7897753B2 (en) * | 2002-02-20 | 2011-03-01 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of XIAP gene expression using short interfering nucleic acid (siNA) |
| WO2003106477A1 (en) | 2002-06-01 | 2003-12-24 | Isis Pharmaceuticals, Inc. | Oligomeric compounds that include carbocyclic nucleosides and their use in gene modulation |
| US7956176B2 (en) | 2002-09-05 | 2011-06-07 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| CA2504694C (en) | 2002-11-05 | 2013-10-01 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| ATE479752T1 (de) | 2003-03-07 | 2010-09-15 | Alnylam Pharmaceuticals Inc | Therapeutische zusammensetzungen |
| JP4605799B2 (ja) | 2003-04-02 | 2011-01-05 | ダーマコン, インコーポレイテッド | Rna干渉において使用するための修飾ポリヌクレオチド |
| AU2004227414A1 (en) | 2003-04-03 | 2004-10-21 | Alnylam Pharmaceuticals | iRNA conjugates |
| WO2004094595A2 (en) | 2003-04-17 | 2004-11-04 | Alnylam Pharmaceuticals Inc. | MODIFIED iRNA AGENTS |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| PL1633767T3 (pl) | 2003-06-02 | 2019-07-31 | University Of Massachusetts | Sposoby i kompozycje do kontrolowania wydajności wyciszania rna |
| GB2417727B (en) | 2003-06-13 | 2008-01-16 | Alnylam Europe Ag | Double-stranded ribonucleic acid with increased effectiveness in an organism |
| EP2514758B2 (en) * | 2004-03-15 | 2021-06-23 | City of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
| EP2700720A3 (en) | 2004-03-15 | 2015-01-28 | Isis Pharmaceuticals, Inc. | Compositions and methods for optimizing cleavage of RNA by RNASE H |
| KR101147147B1 (ko) | 2004-04-01 | 2012-05-25 | 머크 샤프 앤드 돔 코포레이션 | Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드 |
| WO2006073458A2 (en) | 2004-04-30 | 2006-07-13 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a c5-modified pyrimidine |
| EP1765416A4 (en) * | 2004-06-03 | 2010-03-24 | Isis Pharmaceuticals Inc | DOUBLE-STRONG COMPOSITIONS WITH DIFFERENTLY MODIFIED STRANDS FOR USE IN GENE MODULATION |
| EP1789553B1 (en) | 2004-06-30 | 2014-03-26 | Alnylam Pharmaceuticals Inc. | Oligonucleotides comprising a non-phosphate backbone linkage |
| US7297786B2 (en) | 2004-07-09 | 2007-11-20 | University Of Iowa Research Foundation | RNA interference in respiratory epitheial cells |
| CA2574088C (en) | 2004-07-21 | 2013-09-17 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a modified or non-natural nucleobase |
| AU2005330637B2 (en) | 2004-08-04 | 2012-09-20 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase |
| AU2006279454B2 (en) * | 2005-08-17 | 2011-12-15 | Sirna Therapeutics, Inc. | Chemically modified short interfering nucleic acid molecules that mediate rna interference |
| CN101346393B (zh) * | 2005-11-02 | 2015-07-22 | 普洛体维生物治疗公司 | 修饰的siRNA分子及其应用 |
| US7915399B2 (en) | 2006-06-09 | 2011-03-29 | Protiva Biotherapeutics, Inc. | Modified siRNA molecules and uses thereof |
| CN104480112B (zh) * | 2007-05-22 | 2018-06-12 | 阿克丘勒斯治疗公司 | 用于治疗的una寡聚体 |
-
2008
- 2008-05-21 CN CN201410554369.7A patent/CN104480112B/zh active Active
- 2008-05-21 MX MX2009012568A patent/MX2009012568A/es active IP Right Grant
- 2008-05-21 JP JP2010509534A patent/JP5726520B2/ja active Active
- 2008-05-21 US US12/515,403 patent/US8314227B2/en active Active
- 2008-05-21 PL PL08780679.0T patent/PL2162538T3/pl unknown
- 2008-05-21 KR KR1020097024222A patent/KR101629017B1/ko active Active
- 2008-05-21 CA CA2687850A patent/CA2687850C/en active Active
- 2008-05-21 ES ES08780679.0T patent/ES2573936T3/es active Active
- 2008-05-21 CN CN200880016977.5A patent/CN101679978B/zh active Active
- 2008-05-21 BR BRPI0811170A patent/BRPI0811170B8/pt active IP Right Grant
- 2008-05-21 NZ NZ580712A patent/NZ580712A/en unknown
- 2008-05-21 AU AU2008256871A patent/AU2008256871B2/en active Active
- 2008-05-21 MY MYPI20094688A patent/MY153691A/en unknown
- 2008-05-21 EP EP08780679.0A patent/EP2162538B1/en active Active
- 2008-05-21 EP EP16156958.7A patent/EP3045535B1/en active Active
- 2008-05-21 DK DK08780679.0T patent/DK2162538T3/en active
- 2008-05-21 KR KR1020157016899A patent/KR101750640B1/ko active Active
- 2008-05-21 WO PCT/US2008/064417 patent/WO2008147824A2/en not_active Ceased
-
2009
- 2009-10-27 ZA ZA2009/07529A patent/ZA200907529B/en unknown
- 2009-11-10 IL IL202040A patent/IL202040A/en active IP Right Grant
-
2012
- 2012-10-16 US US13/652,965 patent/US9051570B2/en active Active
-
2014
- 2014-12-03 JP JP2014245255A patent/JP6174001B2/ja active Active
-
2015
- 2015-05-04 US US14/702,991 patent/US9303260B2/en active Active
- 2015-05-04 US US14/703,016 patent/US9297009B2/en active Active
-
2016
- 2016-02-22 US US15/050,065 patent/US9944929B2/en active Active
-
2018
- 2018-04-13 US US15/952,680 patent/US10457945B2/en active Active
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US10457945B2 (en) | UNA oligomers for therapeutics with prolonged stability | |
| AU2009322290B2 (en) | Una oligomer structures for therapeutic agents | |
| AU2013273719B2 (en) | UNA amidites for therapeutic oligomers | |
| HK1138322B (en) | Hydroxymethyl substituted rna oligonucleotides and rna complexes | |
| HK1158680A (en) | Usirna complexes |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PSEA | Patent sealed | ||
| RENW | Renewal (renewal fees accepted) | ||
| ASS | Change of ownership |
Owner name: ARCTURUS THERAPEUTICS, INC., US Effective date: 20131031 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 21 MAY 2019 BY CPA GLOBAL Effective date: 20181018 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 21 MAY 2020 BY COMPUTER PACKAGES INC Effective date: 20190501 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 21 MAY 2021 BY COMPUTER PACKAGES INC Effective date: 20200501 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 21 MAY 2022 BY COMPUTER PACKAGES INC Effective date: 20210430 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 21 MAY 2023 BY COMPUTER PACKAGES INC Effective date: 20220430 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 21 MAY 2024 BY COMPUTER PACKAGES INC Effective date: 20230430 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 21 MAY 2025 BY COMPUTER PACKAGES INC Effective date: 20240430 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 21 MAY 2026 BY COMPUTER PACKAGES INC Effective date: 20250430 |