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
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- 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
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- 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)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (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) | KR101750640B1 (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 (225)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| USRE44779E1 (en) | 1997-03-07 | 2014-02-25 | Santaris Pharma A/S | Bicyclonucleoside and oligonucleotide analogues |
| 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) |
| 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) |
| AU2007310982A1 (en) | 2006-10-18 | 2008-04-24 | Marina Biotech, Inc. | Nicked or gapped nucleic acid molecules and uses thereof |
| BRPI0811170B8 (pt) * | 2007-05-22 | 2021-05-25 | Arcturus Therapeutics Inc | oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila |
| 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 |
| EP2349210B1 (en) * | 2008-10-16 | 2015-03-18 | Marina Biotech, Inc. | Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics |
| 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 |
| WO2011057020A1 (en) | 2009-11-04 | 2011-05-12 | Marina Biotech, Inc. | Activity generating delivery molecules |
| KR101605932B1 (ko) | 2009-12-18 | 2016-03-24 | 노파르티스 아게 | Hsf1-관련 질환을 치료하기 위한 유기 조성물 |
| 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 |
| US10913767B2 (en) | 2010-04-22 | 2021-02-09 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising acyclic and abasic nucleosides and analogs |
| CN107929306B (zh) | 2010-04-23 | 2022-11-15 | 箭头制药公司 | 治疗β-ENaC-相关疾病的有机组合物 |
| WO2011139842A2 (en) * | 2010-04-28 | 2011-11-10 | Marina Biotech, Inc. | Nucleic acid compounds for inhibiting fgfr3 gene expression and uses thereof |
| NZ605420A (en) | 2010-07-06 | 2015-02-27 | 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 |
| WO2012027206A1 (en) | 2010-08-24 | 2012-03-01 | Merck Sharp & Dohme Corp. | SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER |
| EP3766975A1 (en) | 2010-10-29 | 2021-01-20 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of gene expression using short interfering nucleic acid (sina) |
| EP3266873A1 (en) | 2010-11-10 | 2018-01-10 | Nigel L. Webb | Nuclions and 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 |
| CN103930547A (zh) | 2011-09-02 | 2014-07-16 | 诺华股份有限公司 | 用于治疗hsf1相关疾病的有机组合物 |
| ES2886147T3 (es) | 2011-12-22 | 2021-12-16 | Interna Tech B V | MiARN para el tratamiento del cáncer de cabeza y de cuello |
| EP2802658A2 (en) | 2012-01-09 | 2014-11-19 | Novartis AG | Rnai agents to treat beta-catenin related diseases |
| 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 |
| US10501513B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides for the production of oncology-related proteins and peptides |
| EP3736333A1 (en) | 2012-05-02 | 2020-11-11 | Arrowhead Pharmaceuticals, Inc. | Organic compositions to treat kras-related diseases |
| WO2014072357A1 (en) | 2012-11-06 | 2014-05-15 | 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 |
| SG10201706960TA (en) | 2013-02-28 | 2017-10-30 | Arrowhead Res Corp | Organic compositions to treat epas1-related diseases |
| WO2015020960A1 (en) | 2013-08-09 | 2015-02-12 | Novartis Ag | Novel lncrna polynucleotides |
| US20150050738A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Compositions and methods for modulating rna |
| CA2925107A1 (en) | 2013-10-02 | 2015-04-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the lect2 gene |
| EP3052627B1 (en) | 2013-10-04 | 2018-08-22 | Novartis AG | Novel formats for organic compounds for use in rna interference |
| WO2015051135A2 (en) | 2013-10-04 | 2015-04-09 | Novartis Ag | Organic compositions to treat hepcidin-related diseases |
| WO2015051044A2 (en) | 2013-10-04 | 2015-04-09 | Novartis Ag | Novel formats for organic compounds for use in rna interference |
| JP6546161B2 (ja) | 2013-10-04 | 2019-07-17 | ノバルティス アーゲー | B型肝炎ウイルスを治療するための有機化合物 |
| SG11201602631XA (en) | 2013-10-04 | 2016-05-30 | Alnylam Pharmaceuticals Inc | Compositions and methods for inhibiting expression of the alas1 gene |
| KR102298475B1 (ko) | 2013-10-04 | 2021-09-06 | 노파르티스 아게 | RNA 간섭에 사용하기 위한 RNAi 작용제를 위한 3' 말단 캡 |
| CA2931090A1 (en) | 2013-12-12 | 2015-06-18 | Alnylam Pharmaceuticals, Inc. | Complement component irna compositions and methods of use thereof |
| AU2015217301A1 (en) | 2014-02-11 | 2016-08-25 | Alnylam Pharmaceuticals, Inc. | Ketohexokinase (KHK) iRNA compositions and methods of use thereof |
| 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 |
| AU2015236215B2 (en) | 2014-03-25 | 2020-03-19 | Arcturus Therapeutics, Inc. | UNA oligomers having reduced off-target effects in gene silencing |
| WO2015175510A1 (en) | 2014-05-12 | 2015-11-19 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating a serpinc1-associated disorder |
| BR122020023687B1 (pt) | 2014-05-22 | 2023-03-07 | Alnylam Pharmaceuticals, Inc | Agente de ácido ribonucleico (rnai) de fita dupla, seus usos e composição farmacêutica |
| 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 |
| EP3736334A1 (en) | 2014-07-16 | 2020-11-11 | Arrowhead Pharmaceuticals, Inc. | Rnai compositions to treat apoc3-related diseases |
| EP3169309B1 (en) | 2014-07-16 | 2023-05-10 | Novartis AG | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
| EP3191591A1 (en) | 2014-09-12 | 2017-07-19 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting complement component c5 and methods of use thereof |
| KR20170075742A (ko) | 2014-10-02 | 2017-07-03 | 프로티바 바이오쎄라퓨틱스, 인코포레이티드 | B형 간염 바이러스 유전자 발현을 제거하는 조성물 및 방법 |
| 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 |
| AU2015338923B2 (en) | 2014-11-02 | 2021-10-21 | Arcturus Therapeutics, Inc. | Messenger UNA molecules and uses thereof |
| SG11201703638XA (en) | 2014-11-10 | 2017-06-29 | Alnylam Pharmaceuticals Inc | Hepatitis b virus (hbv) irna compositions and methods of use thereof |
| CA2968114A1 (en) | 2014-11-17 | 2016-05-26 | Alnylam Pharmaceuticals, Inc. | Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof |
| WO2016130806A2 (en) | 2015-02-13 | 2016-08-18 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
| WO2016130943A1 (en) | 2015-02-13 | 2016-08-18 | Rana Therapeutics, Inc. | Hybrid oligonucleotides and uses thereof |
| EP3277289A4 (en) * | 2015-04-01 | 2018-12-05 | Arcturus Therapeutics, Inc. | Therapeutic una oligomers and uses thereof |
| MX2017012610A (es) | 2015-04-08 | 2018-03-16 | Alnylam Pharmaceuticals Inc | Composiciones y metodos para inhibir la expresion del gen lect2. |
| US20180245074A1 (en) | 2015-06-04 | 2018-08-30 | Protiva Biotherapeutics, Inc. | Treating hepatitis b virus infection using crispr |
| WO2016201301A1 (en) | 2015-06-12 | 2016-12-15 | 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 |
| US10494632B2 (en) | 2015-07-10 | 2019-12-03 | Alnylam Pharmaceuticals, Inc. | Insulin-like growth factor binding protein, acid labile subunit (IGFALS) 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 |
| HK1248278A1 (zh) | 2015-07-29 | 2018-10-12 | Arbutus Biopharma Corporation | 用於使乙型肝炎病毒基因表达沉默的组合物及方法 |
| CN108368507B (zh) | 2015-09-02 | 2022-03-22 | 阿尔尼拉姆医药品有限公司 | 程序性细胞死亡1配体1(PD-L1)的iRNA组合物及其使用方法 |
| EP3353298B1 (en) | 2015-09-21 | 2023-09-13 | Arcturus Therapeutics, Inc. | Allele selective gene editing and uses thereof |
| SG11201804729RA (en) | 2015-12-07 | 2018-07-30 | Genzyme Corp | Methods and compositions for treating a serpinc1-associated disorder |
| WO2017184689A1 (en) | 2016-04-19 | 2017-10-26 | Alnylam Pharmaceuticals, Inc. | High density lipoprotein binding protein (hdlbp/vigilin) irna compositions and methods of use thereof |
| CA3023764A1 (en) | 2016-06-06 | 2017-12-14 | Arrowhead Pharmaceuticals, Inc. | 5'-cyclo-phosphonate modified nucleotides |
| WO2017214518A1 (en) | 2016-06-10 | 2017-12-14 | Alnylam Pharmaceuticals, Inc. | COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS 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 |
| EP3484908A4 (en) | 2016-07-15 | 2020-04-08 | AM Chemicals Llc | NON-NUCLEOSIDIC SOLID CARRIERS AND PHOSPHORAMIDITE BUILDING BLOCKS FOR OLIGONUCLEOTID SYNTHESIS |
| MA45999A (fr) | 2016-08-22 | 2019-06-26 | Arbutus Biopharma Corp | Anticorps anti-pd-1, ou leurs fragments, pour le traitement de l'hépatite b |
| WO2018075827A1 (en) | 2016-10-19 | 2018-04-26 | Arcturus Therapeutics, Inc. | Trinucleotide mrna cap analogs |
| 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 |
| AU2017376950B2 (en) | 2016-12-16 | 2024-02-22 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing TTR-associated diseases using transthyretin (TTR) iRNA compositions |
| UY37565A (es) | 2017-01-10 | 2018-07-31 | Arrowhead Pharmaceuticals Inc | Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso |
| JP2020530442A (ja) | 2017-07-10 | 2020-10-22 | ジェンザイム・コーポレーション | 血友病を有する対象の出血事象を処置するための方法および組成物 |
| TN2020000039A1 (en) | 2017-09-11 | 2021-10-04 | 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 |
| US20200385719A1 (en) | 2017-11-16 | 2020-12-10 | Alnylam Pharmaceuticals, Inc. | Kisspeptin 1 (kiss1) irna compositions and methods of use thereof |
| WO2019126097A1 (en) | 2017-12-18 | 2019-06-27 | Alnylam Pharmaceuticals, Inc. | High mobility group box-1 (hmgb1) irna compositions and methods of use thereof |
| WO2019155094A1 (en) | 2018-02-12 | 2019-08-15 | Interna Technologies B.V. | Anticancer microrna and lipid formulations thereof |
| WO2019191780A1 (en) | 2018-03-30 | 2019-10-03 | 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 |
| EP3833397A4 (en) | 2018-08-08 | 2023-06-14 | Arcturus Therapeutics, Inc. | COMPOSITIONS AND AGENTS AGAINST NON-ALCOHOLIC STEATOHEPATITIS |
| US20210317461A1 (en) | 2018-08-09 | 2021-10-14 | Verseau Therapeutics, Inc. | Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof |
| MA70658B1 (fr) | 2018-08-13 | 2026-04-30 | Alnylam Pharmaceuticals, Inc. | Compositions d'agent d'arndb du virus de l'hépatite b (vhb) et leurs méthodes d'utilisation |
| US11987792B2 (en) | 2018-08-16 | 2024-05-21 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the LECT2 gene |
| US12281308B2 (en) | 2018-08-29 | 2025-04-22 | University Of Massachusetts | Inhibition of protein kinases to treat Friedreich 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 |
| WO2020117706A1 (en) | 2018-12-03 | 2020-06-11 | Triplet Therapeutics, Inc. | Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity |
| CN113631709A (zh) | 2018-12-20 | 2021-11-09 | 普拉克西斯精密药物股份有限公司 | 用于治疗kcnt1相关病症的组合物和方法 |
| EP3908288A4 (en) | 2019-01-09 | 2023-12-20 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use |
| CN113557023A (zh) | 2019-01-16 | 2021-10-26 | 建新公司 | Serpinc1 iRNA组合物及其使用方法 |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
| EP3980540A4 (en) | 2019-06-06 | 2023-11-01 | Arrowhead Pharmaceuticals, Inc. | METHOD FOR TREATING ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD) |
| CA3142283A1 (en) | 2019-06-06 | 2020-12-10 | Avidity Biosciences, Inc. | Nucleic acid-polypeptide compositions and uses thereof |
| US12006499B2 (en) | 2019-06-06 | 2024-06-11 | Avidity Biosciences, Inc. | Una amidites 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 |
| EP4007811A2 (en) | 2019-08-01 | 2022-06-08 | Alnylam Pharmaceuticals, Inc. | Carboxypeptidase b2 (cpb2) irna compositions and methods of use thereof |
| 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 |
| US20220290152A1 (en) | 2019-09-03 | 2022-09-15 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the lect2 gene |
| JP7667775B2 (ja) * | 2019-09-05 | 2025-04-23 | サノフイ | ヌクレオチド類似体を含有するオリゴヌクレオチド |
| WO2021067747A1 (en) | 2019-10-04 | 2021-04-08 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for silencing ugt1a1 gene expression |
| WO2021076828A1 (en) | 2019-10-18 | 2021-04-22 | Alnylam Pharmaceuticals, Inc. | Solute carrier family member irna compositions and methods of use thereof |
| BR112022007540A2 (pt) | 2019-10-22 | 2022-07-12 | Alnylam Pharmaceuticals Inc | Composições de irna de componente complementar c3 e métodos de uso das mesmas |
| 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 |
| 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 |
| 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 |
| 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). |
| WO2021167841A1 (en) | 2020-02-18 | 2021-08-26 | 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 |
| WO2021178736A1 (en) | 2020-03-06 | 2021-09-10 | Alnylam Pharmaceuticals, Inc. | KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| EP4121534A1 (en) | 2020-03-18 | 2023-01-25 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant |
| 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 |
| EP4142738A1 (en) | 2020-04-30 | 2023-03-08 | Alnylam Pharmaceuticals, Inc. | Complement factor b (cfb) irna compositions and methods of use thereof |
| 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) |
| EP4150088A1 (en) | 2020-05-15 | 2023-03-22 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1) |
| WO2021231691A1 (en) | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi) |
| WO2021231673A1 (en) | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2) |
| WO2021231692A1 (en) | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of otoferlin (otof) |
| 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) |
| WO2021231698A1 (en) | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl) |
| WO2021231679A1 (en) | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2) |
| EP4153746A1 (en) | 2020-05-21 | 2023-03-29 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting marc1 gene expression |
| US11408000B2 (en) | 2020-06-03 | 2022-08-09 | Triplet Therapeutics, Inc. | Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity |
| 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 |
| EP4162050A1 (en) | 2020-06-09 | 2023-04-12 | Alnylam Pharmaceuticals, Inc. | Rnai compositions and methods of use thereof for delivery by inhalation |
| AU2021292296A1 (en) | 2020-06-18 | 2023-01-19 | Alnylam Pharmaceuticals, Inc. | Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof |
| 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 |
| 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 |
| WO2022103999A1 (en) | 2020-11-13 | 2022-05-19 | Alnylam Pharmaceuticals, Inc. | COAGULATION FACTOR V (F5) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| WO2022125490A1 (en) | 2020-12-08 | 2022-06-16 | Alnylam Pharmaceuticals, Inc. | Coagulation factor x (f10) irna compositions and methods of use thereof |
| EP4274896A1 (en) | 2021-01-05 | 2023-11-15 | Alnylam Pharmaceuticals, Inc. | Complement component 9 (c9) irna compositions and methods of use thereof |
| 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 |
| JP2024515423A (ja) | 2021-02-26 | 2024-04-10 | 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 |
| 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 |
| IL307239A (en) | 2021-03-29 | 2023-11-01 | Alnylam Pharmaceuticals Inc | Preparations containing Huntingtin IRNA factor (HTT) and methods of using them |
| WO2022212153A1 (en) | 2021-04-01 | 2022-10-06 | Alnylam Pharmaceuticals, Inc. | Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof |
| BR112023022284A2 (pt) | 2021-04-26 | 2023-12-26 | Alnylam Pharmaceuticals Inc | Composições de irna de protease transmembrana, serina 6 (tmprss6) e métodos de uso da mesma |
| EP4330396A1 (en) | 2021-04-29 | 2024-03-06 | 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 |
| EP4341405A1 (en) | 2021-05-20 | 2024-03-27 | 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 |
| JP2024522996A (ja) | 2021-06-02 | 2024-06-25 | 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 | パタチン様ホスホリパーゼドメイン含有3(PNPLA3)iRNA組成物およびその使用方法 |
| 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 |
| EP4351541A2 (en) | 2021-06-08 | 2024-04-17 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders |
| US20230194709A9 (en) | 2021-06-29 | 2023-06-22 | Seagate Technology Llc | Range information detection using coherent pulse sets with selected waveform characteristics |
| US20250034564A1 (en) | 2021-06-29 | 2025-01-30 | Korro Bio, Inc. | Methods and Compositions for ADAR-Mediated Editing |
| WO2023003805A1 (en) | 2021-07-19 | 2023-01-26 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder |
| JP2024528701A (ja) | 2021-07-23 | 2024-07-30 | 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 | ベータカテニン(CTNNB1)iRNA組成物およびその使用方法 |
| JP2024529437A (ja) | 2021-07-29 | 2024-08-06 | 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 | 3-ヒドロキシ-3-メチルグリタル-COAレダクターゼ(HMGCR)iRNA組成物およびその使用方法 |
| TW202328445A (zh) | 2021-08-03 | 2023-07-16 | 美商艾拉倫製藥股份有限公司 | 甲狀腺素運載蛋白(TTR)iRNA組成物及其使用方法 |
| PE20241132A1 (es) | 2021-08-04 | 2024-05-24 | Alnylam Pharmaceuticals Inc | Composiciones de arni y metodos para silenciar el angiotensinogeno (agt) |
| IL310407A (en) | 2021-08-13 | 2024-03-01 | Alnylam Pharmaceuticals Inc | Factor XII (F12) IRNA compositions and methods of using them |
| 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. |
| JP2024535850A (ja) | 2021-09-17 | 2024-10-02 | 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 | 補体成分(C3)をサイレンシングするためのiRNA組成物および方法 |
| EP4405478A1 (en) | 2021-09-20 | 2024-07-31 | Alnylam Pharmaceuticals, Inc. | Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof |
| 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. |
| CA3233755A1 (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 |
| 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 |
| 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 |
| AU2023283551A1 (en) | 2022-06-10 | 2024-12-19 | Camp4 Therapeutics Corporation | Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory rnas |
| JP2025527531A (ja) | 2022-08-18 | 2025-08-22 | 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 | ユニバーサル非標的sirna組成物およびその使用方法 |
| TW202424193A (zh) | 2022-09-15 | 2024-06-16 | 美商艾拉倫製藥股份有限公司 | 第13型17β-羥基類固醇去氫酶(HSD17B13)iRNA組成物及其使用方法 |
| 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 |
| AU2024217843A1 (en) | 2023-02-09 | 2025-07-31 | Alnylam Pharmaceuticals, Inc. | Reversir molecules and methods of use thereof |
| UY40699A (es) | 2023-04-05 | 2024-08-30 | Ionis Pharmaceuticals Inc | Compuestos y métodos para reducir la expresión de pln |
| WO2024208249A1 (zh) | 2023-04-06 | 2024-10-10 | 上海舶望制药有限公司 | 5'-膦酸酯修饰的核苷类似物及其制备的寡核苷酸 |
| 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 |
| 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 |
| KR20260021635A (ko) | 2023-05-12 | 2026-02-13 | 아다르엑스 파마슈티컬스, 인크. | Nmda 리간드 접합 화합물 및 이의 용도 |
| IL324720A (en) | 2023-05-26 | 2026-01-01 | Adarx Pharmaceuticals Inc | SOD1 modulators and methods of using them |
| CN121712891A (zh) | 2023-06-20 | 2026-03-20 | 阿达尔克斯制药有限公司 | Lrrk2调节组合物及其使用方法 |
| AU2024287308A1 (en) | 2023-07-13 | 2025-12-18 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| KR20260044219A (ko) | 2023-07-21 | 2026-04-01 | 매로우 테라퓨틱스, 인크. | 조혈 세포 표적화 접합체 및 관련 방법 |
| US20250092426A1 (en) | 2023-07-25 | 2025-03-20 | Flagship Pioneering Innovations Vii, Llc | Cas endonucleases and related methods |
| AU2024297923A1 (en) | 2023-07-25 | 2026-01-22 | Flagship Pioneering Innovations Vii, Llc | Cas endonucleases and related methods |
| IL326321A (en) | 2023-08-04 | 2026-04-01 | Alnylam Pharmaceuticals Inc | Methods and compositions for treating CTNNB1-related 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 |
| AU2024348221A1 (en) | 2023-09-27 | 2026-04-30 | 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 |
| WO2025199231A2 (en) | 2024-03-20 | 2025-09-25 | 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 |
| WO2025259747A2 (en) | 2024-06-12 | 2025-12-18 | Alnylam Pharmaceuticals, Inc. | Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof |
| WO2025259743A1 (en) | 2024-06-12 | 2025-12-18 | Alnylam Pharmaceuticals, Inc. | Dual conjugate compounds for extrahepatic delivery |
| WO2026050243A1 (en) | 2024-08-26 | 2026-03-05 | Korro Bio, Inc. | Galnac conjugated oligonucleotides for rna editing |
| WO2026055461A1 (en) | 2024-09-05 | 2026-03-12 | Aperture Therapeutics, Inc. | Antibody oligonucleotide conjugates comprising an antisense polynucleotide agent conjugated to a cd33 antibody and methods of use thereof |
| WO2026057749A1 (en) | 2024-09-11 | 2026-03-19 | Sixfold Bioscience Ltd. | Method and product |
| WO2026062247A1 (en) | 2024-09-20 | 2026-03-26 | Astrazeneca Ab | Liquid-phase oligonucleotide synthesis using 2-(2-nitrophenyl)propyloxycarbonyl (nppoc) as a protecting group |
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 |
| US20080119427A1 (en) * | 1996-06-06 | 2008-05-22 | Isis Pharmaceuticals, Inc. | Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation |
| 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 |
| US20070026394A1 (en) | 2000-02-11 | 2007-02-01 | Lawrence Blatt | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies |
| WO2003070918A2 (en) | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | Rna interference by modified short interfering nucleic acid |
| 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 |
| ATE450621T2 (de) | 2000-03-30 | 2009-12-15 | Whitehead Biomedical Inst | Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind |
| KR100872437B1 (ko) | 2000-12-01 | 2008-12-05 | 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. | Rna 간섭을 매개하는 작은 rna 분자 |
| US8546143B2 (en) | 2001-01-09 | 2013-10-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
| JP2005504020A (ja) | 2001-07-03 | 2005-02-10 | 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 | ヌクレアーゼ耐性キメラオリゴヌクレオチド |
| ES2609014T3 (es) | 2001-07-12 | 2017-04-18 | University Of Massachusetts | Producción in vivo de ARN de interferencia pequeños que median el silenciamiento génico |
| DE60216798T2 (de) * | 2001-10-29 | 2007-10-18 | Mcgill University, Montreal | Azyklische linker enthaltende oligonukleotide und deren verwendungen |
| 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) |
| AU2003248708A1 (en) | 2002-06-17 | 2003-12-31 | Isis Pharmaceuticals, Inc. | Oligomeric compounds that include carbocyclic nucleosides and their use in gene modulation |
| US7923547B2 (en) | 2002-09-05 | 2011-04-12 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| WO2004041889A2 (en) | 2002-11-05 | 2004-05-21 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| 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 |
| AU2004227414A1 (en) | 2003-04-03 | 2004-10-21 | Alnylam Pharmaceuticals | iRNA conjugates |
| 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 |
| ES2712695T3 (es) | 2003-06-02 | 2019-05-14 | Univ Massachusetts | Métodos y composiciones para controlar la eficacia de la silenciación del ARN |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| WO2005014782A2 (en) | 2003-06-13 | 2005-02-17 | Alnylam Europe Ag., | Double-stranded ribonucleic acid with increased effectiveness in an organism |
| US8084599B2 (en) | 2004-03-15 | 2011-12-27 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
| US8790919B2 (en) | 2004-03-15 | 2014-07-29 | 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 |
| CA2562151C (en) | 2004-04-30 | 2016-09-06 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a c5-modified pyrimidine |
| JP2008504840A (ja) | 2004-06-30 | 2008-02-21 | 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 | 非リン酸骨格結合を含むオリゴヌクレオチド |
| US7297786B2 (en) | 2004-07-09 | 2007-11-20 | University Of Iowa Research Foundation | RNA interference in respiratory epitheial cells |
| WO2006093526A2 (en) | 2004-07-21 | 2006-09-08 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a modified or non-natural nucleobase |
| CA2574603C (en) | 2004-08-04 | 2014-11-04 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase |
| EP1931781B1 (en) * | 2005-08-17 | 2016-07-27 | Sirna Therapeutics, Inc. | Chemically modified short interfering nucleic acid molecules that mediate rna interference |
| WO2007051303A1 (en) * | 2005-11-02 | 2007-05-10 | Protiva Biotherapeutics, Inc. | MODIFIED siRNA MOLECULES AND USES THEREOF |
| US7915399B2 (en) | 2006-06-09 | 2011-03-29 | Protiva Biotherapeutics, Inc. | Modified siRNA molecules and uses thereof |
| BRPI0811170B8 (pt) * | 2007-05-22 | 2021-05-25 | Arcturus Therapeutics Inc | oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila |
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