CA2687850C - Oligomers for therapeutics - Google Patents

Oligomers for therapeutics Download PDF

Info

Publication number
CA2687850C
CA2687850C CA2687850A CA2687850A CA2687850C CA 2687850 C CA2687850 C CA 2687850C CA 2687850 A CA2687850 A CA 2687850A CA 2687850 A CA2687850 A CA 2687850A CA 2687850 C CA2687850 C CA 2687850C
Authority
CA
Canada
Prior art keywords
monomers
rna
oligomer
monomer
seco
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
CA2687850A
Other languages
English (en)
French (fr)
Other versions
CA2687850A1 (en
Inventor
Jesper Wengel
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Arcturus Therapeutics Inc
Original Assignee
Arcturus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Arcturus Therapeutics Inc filed Critical Arcturus Therapeutics Inc
Publication of CA2687850A1 publication Critical patent/CA2687850A1/en
Application granted granted Critical
Publication of CA2687850C publication Critical patent/CA2687850C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/51Methods 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)
CA2687850A 2007-05-22 2008-05-21 Oligomers for therapeutics Active CA2687850C (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
DKPA200700751 2007-05-22
DKPA200700751 2007-05-22
DKPA200701718 2007-11-30
DKPA200701718 2007-11-30
DKPA200701785 2007-12-14
DKPA200701785 2007-12-14
DKPA200800534 2008-04-11
DKPA200800534 2008-04-11
PCT/US2008/064417 WO2008147824A2 (en) 2007-05-22 2008-05-21 Hydroxymethyl substituted rna oligonucleotides and rna complexes

Publications (2)

Publication Number Publication Date
CA2687850A1 CA2687850A1 (en) 2008-12-04
CA2687850C true CA2687850C (en) 2017-11-21

Family

ID=40075730

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2687850A Active CA2687850C (en) 2007-05-22 2008-05-21 Oligomers for therapeutics

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

Also Published As

Publication number Publication date
AU2008256871B2 (en) 2013-09-19
CA2687850A1 (en) 2008-12-04
WO2008147824A2 (en) 2008-12-04
US20180237780A1 (en) 2018-08-23
EP3045535A1 (en) 2016-07-20
US20100056768A1 (en) 2010-03-04
BRPI0811170A2 (pt) 2014-10-07
KR20150080027A (ko) 2015-07-08
HK1138322A1 (zh) 2010-08-20
JP2010528041A (ja) 2010-08-19
US20130096289A1 (en) 2013-04-18
IL202040A0 (en) 2011-08-01
US8314227B2 (en) 2012-11-20
AU2008256871A1 (en) 2008-12-04
JP6174001B2 (ja) 2017-08-02
US9303260B2 (en) 2016-04-05
US9297009B2 (en) 2016-03-29
BRPI0811170B8 (pt) 2021-05-25
US20150232849A1 (en) 2015-08-20
KR101629017B1 (ko) 2016-06-10
US9944929B2 (en) 2018-04-17
KR101750640B1 (ko) 2017-06-23
IL202040A (en) 2015-07-30
US20160168567A1 (en) 2016-06-16
EP3045535B1 (en) 2018-07-25
WO2008147824A3 (en) 2009-04-23
CN104480112A (zh) 2015-04-01
DK2162538T3 (en) 2016-06-06
US9051570B2 (en) 2015-06-09
JP2015142558A (ja) 2015-08-06
CN101679978B (zh) 2016-05-04
CN101679978A (zh) 2010-03-24
MY153691A (en) 2015-03-13
ZA200907529B (en) 2011-02-23
US10457945B2 (en) 2019-10-29
KR20100023811A (ko) 2010-03-04
MX2009012568A (es) 2009-12-08
ES2573936T3 (es) 2016-06-13
EP2162538B1 (en) 2016-04-20
EP2162538A2 (en) 2010-03-17
CN104480112B (zh) 2018-06-12
JP5726520B2 (ja) 2015-06-03
US20150232851A1 (en) 2015-08-20
BRPI0811170B1 (pt) 2020-12-01
PL2162538T3 (pl) 2016-10-31
NZ580712A (en) 2011-12-22

Similar Documents

Publication Publication Date Title
US10457945B2 (en) UNA oligomers for therapeutics with prolonged stability
AU2009322290B2 (en) Una oligomer structures for therapeutic agents
EP2957568A1 (en) Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
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
EEER Examination request

Effective date: 20130507

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 17TH ANNIV.) - STANDARD

Year of fee payment: 17

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250516

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250516