EP0654077A4 - Method and reagent for treatment of animal diseases. - Google Patents

Method and reagent for treatment of animal diseases.

Info

Publication number
EP0654077A4
EP0654077A4 EP93918144A EP93918144A EP0654077A4 EP 0654077 A4 EP0654077 A4 EP 0654077A4 EP 93918144 A EP93918144 A EP 93918144A EP 93918144 A EP93918144 A EP 93918144A EP 0654077 A4 EP0654077 A4 EP 0654077A4
Authority
EP
European Patent Office
Prior art keywords
reagent
treatment
animal diseases
condition
maintenance
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.)
Pending
Application number
EP93918144A
Other languages
German (de)
French (fr)
Other versions
EP0654077A1 (en
Inventor
Sean M Sullivan
Kenneth G Draper
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.)
Sirna Therapeutics Inc
Original Assignee
Ribozyme Pharmaceuticals 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 Ribozyme Pharmaceuticals Inc filed Critical Ribozyme Pharmaceuticals Inc
Priority to EP97101534A priority Critical patent/EP0786522A2/en
Priority to EP02005642A priority patent/EP1251170A3/en
Publication of EP0654077A1 publication Critical patent/EP0654077A1/en
Publication of EP0654077A4 publication Critical patent/EP0654077A4/en
Pending legal-status Critical Current

Links

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/1135—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against oncogenes or tumor suppressor genes
    • 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
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • 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
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6489—Metalloendopeptidases (3.4.24)
    • C12N9/6491—Matrix metalloproteases [MMP's], e.g. interstitial collagenase (3.4.24.7); Stromelysins (3.4.24.17; 3.2.1.22); Matrilysin (3.4.24.23)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/123—Hepatitis delta
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/126—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes involving RNAse P

Abstract

An enzymatic RNA molecule which cleaves mRNA associated with development of maintenance of an inflammatory disease, an arthritic condition, a stenotic condition, or a cardiovascular condition.
EP93918144A 1992-07-17 1993-07-02 Method and reagent for treatment of animal diseases. Pending EP0654077A4 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP97101534A EP0786522A2 (en) 1992-07-17 1993-07-02 Enzymatic RNA molecules for treatment of stenotic conditions
EP02005642A EP1251170A3 (en) 1992-07-17 1993-07-02 Method and reagent for treatment of NF-kappaB dependent animal diseases

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US91676392A 1992-07-17 1992-07-17
US916763 1992-07-17
US98984992A 1992-12-07 1992-12-07
US98984892A 1992-12-07 1992-12-07
US98713292A 1992-12-07 1992-12-07
US987132 1992-12-07
US989849 1992-12-07
US989848 1992-12-07
US889593A 1993-01-19 1993-01-19
US8895 1993-01-19
PCT/US1993/006316 WO1994002595A1 (en) 1992-07-17 1993-07-02 Method and reagent for treatment of animal diseases

Related Child Applications (2)

Application Number Title Priority Date Filing Date
EP02005642A Division EP1251170A3 (en) 1992-07-17 1993-07-02 Method and reagent for treatment of NF-kappaB dependent animal diseases
EP97101534.2 Division-Into 1997-01-31

Publications (2)

Publication Number Publication Date
EP0654077A1 EP0654077A1 (en) 1995-05-24
EP0654077A4 true EP0654077A4 (en) 1996-03-13

Family

ID=27533301

Family Applications (3)

Application Number Title Priority Date Filing Date
EP97101534A Withdrawn EP0786522A2 (en) 1992-07-17 1993-07-02 Enzymatic RNA molecules for treatment of stenotic conditions
EP93918144A Pending EP0654077A4 (en) 1992-07-17 1993-07-02 Method and reagent for treatment of animal diseases.
EP02005642A Withdrawn EP1251170A3 (en) 1992-07-17 1993-07-02 Method and reagent for treatment of NF-kappaB dependent animal diseases

Family Applications Before (1)

Application Number Title Priority Date Filing Date
EP97101534A Withdrawn EP0786522A2 (en) 1992-07-17 1993-07-02 Enzymatic RNA molecules for treatment of stenotic conditions

Family Applications After (1)

Application Number Title Priority Date Filing Date
EP02005642A Withdrawn EP1251170A3 (en) 1992-07-17 1993-07-02 Method and reagent for treatment of NF-kappaB dependent animal diseases

Country Status (6)

Country Link
EP (3) EP0786522A2 (en)
JP (1) JPH07509133A (en)
AU (2) AU4769893A (en)
CA (1) CA2140343A1 (en)
MX (1) MX9304329A (en)
WO (1) WO1994002595A1 (en)

Families Citing this family (505)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6469158B1 (en) * 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5686599A (en) * 1992-05-14 1997-11-11 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) * 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5646042A (en) * 1992-08-26 1997-07-08 Ribozyme Pharmaceuticals, Inc. C-myb targeted ribozymes
US5891684A (en) * 1992-10-15 1999-04-06 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5612215A (en) * 1992-12-07 1997-03-18 Ribozyme Pharmaceuticals, Inc. Stromelysin targeted ribozymes
US5811300A (en) * 1992-12-07 1998-09-22 Ribozyme Pharmaceuticals, Inc. TNF-α ribozymes
US5616488A (en) * 1992-12-07 1997-04-01 Ribozyme Pharmaceuticals, Inc. IL-5 targeted ribozymes
US5837542A (en) 1992-12-07 1998-11-17 Ribozyme Pharmaceuticals, Inc. Intercellular adhesion molecule-1 (ICAM-1) ribozymes
US5658780A (en) 1992-12-07 1997-08-19 Ribozyme Pharmaceuticals, Inc. Rel a targeted ribozymes
JPH09502092A (en) 1993-09-02 1997-03-04 リボザイム・ファーマシューティカルズ・インコーポレイテッド Enzymatic nucleic acid containing non-nucleotide
CA2174339A1 (en) * 1993-10-27 1995-05-04 Lech W. Dudycz 2'-amido and 2'-peptido modified oligonucleotides
EP0728204A1 (en) 1993-11-08 1996-08-28 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
ATE226254T1 (en) * 1993-11-12 2002-11-15 Ribozyme Pharm Inc REAGENT FOR THE TREATMENT OF ARTHRITIC CONDITIONS
US5639647A (en) * 1994-03-29 1997-06-17 Ribozyme Pharmaceuticals, Inc. 2'-deoxy-2'alkylnucleotide containing nucleic acid
US5631359A (en) * 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
CA2183992A1 (en) * 1994-02-23 1995-08-31 Dan T. Stinchcomb Method and reagent for inhibiting the expression of disease related genes
US5693532A (en) * 1994-11-04 1997-12-02 Ribozyme Pharmaceuticals, Inc. Respiratory syncytial virus ribozymes
US5902880A (en) * 1994-08-19 1999-05-11 Ribozyme Pharmaceuticals, Inc. RNA polymerase III-based expression of therapeutic RNAs
US5834440A (en) * 1994-03-07 1998-11-10 Immusol Incorporated Ribozyme therapy for the inhibition of restenosis
US5627053A (en) * 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US6103890A (en) * 1994-05-18 2000-08-15 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids that cleave C-fos
US20030026782A1 (en) * 1995-02-07 2003-02-06 Arthur M. Krieg Immunomodulatory oligonucleotides
US6350934B1 (en) 1994-09-02 2002-02-26 Ribozyme Pharmaceuticals, Inc. Nucleic acid encoding delta-9 desaturase
US5599706A (en) * 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US5716824A (en) * 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
CA2207593A1 (en) * 1994-12-13 1996-06-20 John Gustofson Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
US5672501A (en) * 1994-12-23 1997-09-30 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5705388A (en) * 1994-12-23 1998-01-06 Ribozyme Pharmaceuticals, Inc. CETP Ribozymes
US5663064A (en) * 1995-01-13 1997-09-02 University Of Vermont Ribozymes with RNA protein binding site
EP1484067A3 (en) 1995-02-08 2005-03-02 Takara Bio Inc. Cancer control
US5877021A (en) * 1995-07-07 1999-03-02 Ribozyme Pharmaceuticals, Inc. B7-1 targeted ribozymes
US6346398B1 (en) 1995-10-26 2002-02-12 Ribozyme Pharmaceuticals, Inc. Method and reagent for the treatment of diseases or conditions related to levels of vascular endothelial growth factor receptor
US7034009B2 (en) 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
AU2064297A (en) * 1996-02-29 1997-09-16 Immusol, Inc Hepatitis c virus ribozymes
AU721758B2 (en) * 1996-04-02 2000-07-13 Gene Shears Pty. Limited Asymmetric hammerhead ribozymes
KR20000048820A (en) 1996-10-01 2000-07-25 게론 코포레이션 Telomerase reverse transcriptase
US5807743A (en) * 1996-12-03 1998-09-15 Ribozyme Pharmaceuticals, Inc. Interleukin-2 receptor gamma-chain ribozymes
US7008776B1 (en) 1996-12-06 2006-03-07 Aventis Pharmaceuticals Inc. Compositions and methods for effecting the levels of high density lipoprotein (HDL) cholesterol and apolipoprotein AI very low density lipoprotein (VLDL) cholesterol and low density lipoprotein (LDL) cholesterol
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
US6057156A (en) 1997-01-31 2000-05-02 Robozyme Pharmaceuticals, Inc. Enzymatic nucleic acid treatment of diseases or conditions related to levels of epidermal growth factor receptors
US6159951A (en) 1997-02-13 2000-12-12 Ribozyme Pharmaceuticals Inc. 2'-O-amino-containing nucleoside analogs and polynucleotides
CA2287084A1 (en) * 1997-04-10 1998-10-15 Royal Netherlands Academy Of Arts And Sciences Diagnosis method and reagents
US5942395A (en) * 1997-05-09 1999-08-24 Universite De Montreal Hybrid ribozymes and methods of use
US6280936B1 (en) 1997-06-09 2001-08-28 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6548657B1 (en) 1997-06-09 2003-04-15 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6183959B1 (en) 1997-07-03 2001-02-06 Ribozyme Pharmaceuticals, Inc. Method for target site selection and discovery
US6316612B1 (en) 1997-08-22 2001-11-13 Ribozyme Pharmaceuticals, Inc. Xylofuranosly-containing nucleoside phosphoramidites and polynucleotides
AU750947C (en) * 1997-09-22 2003-05-22 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Nucleic acid catalysts with endonuclease activity
US6656731B1 (en) 1997-09-22 2003-12-02 Max Planck Gesellschaft Zur Forderung Der Wissenschaften E.V. Nucleic acid catalysts with endonuclease activity
AU9509798A (en) * 1997-09-25 1999-04-12 University Of Florida Antisense oligonucleotide compositions targeted to angiotensi n converting enzy me mrna and methods of use
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US6127535A (en) * 1997-11-05 2000-10-03 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into oligonucleotides
DE69835538T2 (en) 1997-12-18 2008-01-03 Monsanto Technology Llc INSECT-RESISTANT TRANSGENIC PLANTS AND METHOD FOR IMPROVING THE ACTIVITY OF DELTA ENDOTOXINS AGAINST INSECTS
US20020147143A1 (en) 1998-03-18 2002-10-10 Corixa Corporation Compositions and methods for the therapy and diagnosis of lung cancer
US6200754B1 (en) 1998-03-19 2001-03-13 Variagenics, Inc. Inhibitors of alternative alleles of genes encoding products that mediate cell response to environmental changes
JP2003525017A (en) 1998-04-20 2003-08-26 リボザイム・ファーマシューティカルズ・インコーポレーテッド Nucleic acid molecules with novel chemical composition that can regulate gene expression
CA2230203A1 (en) 1998-04-29 1999-10-29 Universite De Sherbrooke Delta ribozyme for rna cleavage
US6228642B1 (en) * 1998-10-05 2001-05-08 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of tumor necrosis factor-(α) (TNF-α) expression
US6080580A (en) * 1998-10-05 2000-06-27 Isis Pharmaceuticals Inc. Antisense oligonucleotide modulation of tumor necrosis factor-α (TNF-α) expression
US6995259B1 (en) 1998-10-23 2006-02-07 Sirna Therapeutics, Inc. Method for the chemical synthesis of oligonucleotides
WO2000066780A2 (en) 1999-04-30 2000-11-09 University Of Florida Adeno-associated virus-delivered ribozyme compositions and methods of use
US6831171B2 (en) 2000-02-08 2004-12-14 Yale University Nucleic acid catalysts with endonuclease activity
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
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US20040002068A1 (en) 2000-03-01 2004-01-01 Corixa Corporation Compositions and methods for the detection, diagnosis and therapy of hematological malignancies
US6737271B1 (en) * 2000-03-24 2004-05-18 Biocell Laboratories Compound, composition and method for the treatment of inflammatory and inflammatory-related disorders
US7153839B2 (en) 2000-03-24 2006-12-26 Biocell Laboratories Method of protecting erythricytes, in particular for improvement of blood cytopenia
WO2001074900A2 (en) 2000-03-31 2001-10-11 Aventis Pharmaceuticals Products Inc. Nuclear factor kb inducing factor
GB0018307D0 (en) 2000-07-26 2000-09-13 Aventis Pharm Prod Inc Polypeptides
JP2004513615A (en) 2000-06-28 2004-05-13 コリクサ　コーポレイション Compositions and methods for treatment and diagnosis of lung cancer
US7754435B2 (en) 2000-09-21 2010-07-13 Philadelphia Health And Education Corporation Prostate cancer-related compositions, methods, and kits based on DNA macroarray proteomics platforms
ATE434936T1 (en) 2001-03-14 2009-07-15 Myriad Genetics Inc TSG101-GAG INTERACTION AND THEIR USE
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
WO2002089747A2 (en) 2001-05-09 2002-11-14 Corixa Corporation Compositions and methods for the therapy and diagnosis of prostate cancer
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US7205399B1 (en) 2001-07-06 2007-04-17 Sirna Therapeutics, Inc. Methods and reagents for oligonucleotide synthesis
CA2476755C (en) 2001-12-17 2014-08-26 Corixa Corporation Compositions and methods for the therapy and diagnosis of inflammatory bowel disease
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc Rna interference mediated inhibition of map kinase genes
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)
US20080260744A1 (en) 2002-09-09 2008-10-23 Omeros Corporation G protein coupled receptors and uses thereof
JP2006507841A (en) * 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド Functional and ultrafunctional siRNA
US7829694B2 (en) 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
US7994149B2 (en) 2003-02-03 2011-08-09 Medtronic, Inc. Method for treatment of Huntington's disease through intracranial delivery of sirna
CN102989009B (en) 2003-12-31 2015-06-03 宾夕法尼亚州研究基金会 Methods for predicting and overcoming resistance to chemotherapy in ovarian cancer and for predicting colon cancer occurrence
AU2005222902B2 (en) 2004-03-12 2010-06-10 Alnylam Pharmaceuticals, Inc. iRNA agents targeting VEGF
EP2267458A3 (en) 2004-04-20 2011-04-06 Galapagos N.V. Methods, compositions and compound assays for inhibiting amyloid-beta protein production
MXPA06012162A (en) 2004-04-27 2007-03-30 Galapagos Nv Methods, agents, and compound screening assays for inducing differentiation of undifferentiated mammalian cells into osteoblasts.
US20060040882A1 (en) 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
CA2567749C (en) 2004-05-26 2013-07-23 Anima Cell Metrology Methods for evaluating ribonucleotide sequences
WO2005121778A2 (en) 2004-06-14 2005-12-22 Galapagos N.V. Methods for identification, and compounds useful for the treatment of degenerative & inflammatory diseases
EP2386856B1 (en) 2004-06-21 2013-07-24 Galapagos N.V. Methods and means for treatment of osteoarthritis
ATE501252T1 (en) 2004-06-22 2011-03-15 Univ Illinois METHOD FOR INHIBITING TUMOR CELL GROWTH USING FOXM1 SIRNS
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP4272748A3 (en) 2004-06-28 2024-03-27 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ZA200701232B (en) 2004-07-15 2008-08-27 Amr Technology Inc Aryl-and heteroaryl-substituted tetrahydroisoquinolines and use thereof to block reuptake of norepinephrine, dopamine and serotonin
US7485468B2 (en) 2004-10-15 2009-02-03 Galapagos Bv Molecular targets and compounds, and methods to identify the same, useful in the treatment of joint degenerative and inflammatory diseases
ES2381201T3 (en) 2005-03-31 2012-05-24 Calando Pharmaceuticals, Inc. Inhibitors of the subunit 2 of the ribonucleotide reductase and uses thereof
ES2382814T3 (en) 2005-05-17 2012-06-13 Merck Sharp & Dohme Ltd. Cis-4 - [(4-chlorophenyl) sulfonyl] -4- (2,5-difluorophenyl) cyclohexanopropanoic acid for cancer treatment
WO2007002718A2 (en) 2005-06-27 2007-01-04 Alnylam Pharmaceuticals, Inc. Rnai modulation of hif-1 and theraputic uses thereof
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
EP1904069B1 (en) 2005-07-15 2018-06-13 Albany Molecular Research, Inc. Aryl-and heteroaryl-substituted tetrahydrobenzazepines and use thereof to block reuptake of norepinephrine, dopamine, and serotonin
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US8293900B2 (en) 2005-09-29 2012-10-23 Merck Sharp & Dohme Corp Acylated spiropiperidine derivatives as melanocortin-4 receptor modulators
WO2009051846A2 (en) 2007-10-18 2009-04-23 Cell Signaling Technology, Inc. Translocation and mutant ros kinase in human non-small cell lung carcinoma
US20120208824A1 (en) 2006-01-20 2012-08-16 Cell Signaling Technology, Inc. ROS Kinase in Lung Cancer
JP5554925B2 (en) 2006-01-20 2014-07-23 セル・シグナリング・テクノロジー・インコーポレイテツド Translocation and mutant ROS kinase in human non-small cell lung cancer
GB0603041D0 (en) 2006-02-15 2006-03-29 Angeletti P Ist Richerche Bio Therapeutic compounds
WO2007109097A2 (en) 2006-03-16 2007-09-27 Alnylam Pharmaceuticals, Inc. RNAi MODULATION OF TGF-BETA AND THERAPEUTIC USES THEREOF
NZ571568A (en) 2006-03-31 2010-11-26 Alnylam Pharmaceuticals Inc Double-stranded RNA molecule compositions and methods for inhibiting expression of Eg5 gene
US7741469B2 (en) 2006-04-05 2010-06-22 University Of Iowa Research Foundation Compositions for treating hearing loss and methods of use thereof
EP2010226B1 (en) 2006-04-07 2014-01-15 The Research Foundation of State University of New York Transcobalamin receptor polypeptides, nucleic acids, and modulators thereof, and related methods of use in modulating cell growth and treating cancer and cobalamin deficiency
EP2447360A1 (en) 2006-04-14 2012-05-02 Cell Signaling Technology, Inc. Gene defects and mutant ALK kinase in human solid tumors
US8377448B2 (en) 2006-05-15 2013-02-19 The Board Of Trustees Of The Leland Standford Junior University CD47 related compositions and methods for treating immunological diseases and disorders
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
EP2471815B1 (en) 2006-07-11 2016-03-30 University Of Medicine And Dentistry Of New Jersey Proteins, nucleic acids encoding the same and associated methods of use
EP2049658A2 (en) 2006-07-21 2009-04-22 Silence Therapeutics AG Means for inhibiting the expression of protein kinase 3
EP1900749A1 (en) 2006-09-12 2008-03-19 Institut National De La Sante Et De La Recherche Medicale (Inserm) Nucleic acids for expressing a polynucleotide of interest in mammalian cancer cells
CA2664113C (en) 2006-09-22 2013-05-28 Merck & Co., Inc. Use of platencin and platensimycin as fatty acid synthesis inhibitors to treat obesity, diabetes and cancer
WO2008041933A2 (en) 2006-10-02 2008-04-10 Aprea Ab Rna interference against wrap53 to treat hyperproliferative diseases
KR101161923B1 (en) 2006-10-03 2012-07-03 유니버시티 오브 메디신 앤드 덴티스트리 오브 뉴 저지 Atap peptides, nucleic acids encoding the same and associated methods of use
US20110218176A1 (en) 2006-11-01 2011-09-08 Barbara Brooke Jennings-Spring Compounds, methods, and treatments for abnormal signaling pathways for prenatal and postnatal development
US9375440B2 (en) 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US8481506B2 (en) 2006-12-05 2013-07-09 Rosetta Genomics, Ltd. Nucleic acids involved in viral infection
BRPI0806245B1 (en) 2007-01-10 2022-01-25 Msd Italia S.R.L. Compounds of formula i and their uses
EP2124967A4 (en) 2007-01-26 2011-01-05 Rosetta Genomics Ltd Compositions and methods for treating hematopoietic malignancies
WO2008104974A2 (en) 2007-02-27 2008-09-04 Rosetta Genomics Ltd. Composition and methods for modulating cell proliferation and cell death
US9006206B2 (en) 2007-02-27 2015-04-14 Rosetta Genomics Ltd. Composition and methods for modulating cell proliferation and cell death
JP5596980B2 (en) 2007-02-28 2014-10-01 アメリカ合衆国 Brachyury polypeptides and methods of use
WO2008109377A1 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting vegf family gene expression and uses thereof
CA2682727C (en) 2007-04-02 2016-03-22 Banyu Pharmaceutical Co., Ltd. Indoledione derivative
JP5926487B2 (en) 2007-04-13 2016-05-25 デイナ ファーバー キャンサー インスティチュート，インコーポレイテッド Method for treating cancer resistant to ErbB therapy
HUE040417T2 (en) 2007-05-04 2019-03-28 Marina Biotech Inc Amino acid lipids and uses thereof
EP2162747B1 (en) 2007-06-20 2014-04-30 Galapagos N.V. Molecular targets and methods to identify compounds for treating bone and joint degenerative diseases
EP2170076B1 (en) 2007-06-27 2016-05-18 Merck Sharp & Dohme Corp. 4-carboxybenzylamino derivatives as histone deacetylase inhibitors
AU2008309146B2 (en) 2007-10-09 2014-06-12 Anima Cell Metrology, Inc. Systems and methods for measuring translation activity in viable cells
EP2203569A2 (en) 2007-10-31 2010-07-07 Rosetta Genomics Ltd Diagnosis and prognosis of specific cancers by means of differential detection of micro-rnas / mirnas
WO2009062112A2 (en) 2007-11-09 2009-05-14 The Salk Institute For Biological Studies Use of tam receptor inhibitors as antimicrobials
AU2008333811B2 (en) 2007-12-04 2014-05-01 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
JP5749494B2 (en) 2008-01-02 2015-07-15 テクミラ ファーマシューティカルズ コーポレイション Improved compositions and methods for delivery of nucleic acids
US8598184B2 (en) 2008-03-03 2013-12-03 Tiger Pharmatech Protein kinase inhibitors
US8114983B2 (en) 2008-04-04 2012-02-14 Calando Pharmaceuticals, Inc. Compositions and use of EPAS1 inhibitors
EP2274425A2 (en) 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
WO2009134443A2 (en) 2008-05-02 2009-11-05 The Brigham And Women's Hospital, Inc. Rna-induced translational silencing and cellular apoptosis
EP2291544B1 (en) 2008-05-16 2017-10-25 The Children's Hospital of Philadelphia Genetic alterations on chromosomes 21q, 6q and 15q and methods of use thereof for the diagnosis and treatment of type i diabetes
ATE550024T1 (en) 2008-05-30 2012-04-15 Univ Yale TARGETED OLIGONUCLEOTIDE COMPOSITIONS FOR MODIFYING GENE EXPRESSION
US9156812B2 (en) 2008-06-04 2015-10-13 Bristol-Myers Squibb Company Crystalline form of 6-[(4S)-2-methyl-4-(2-naphthyl)-1,2,3,4-tetrahydroisoquinolin-7-yl]pyridazin-3-amine
EP2297322A1 (en) 2008-06-04 2011-03-23 The Board of Regents of The University of Texas System Modulation of gene expression through endogenous small rna targeting of gene promoters
EP2816113A3 (en) 2008-09-11 2015-03-25 Galapagos N.V. Method for identifying compounds useful for increasing the functional activity and cell surface expression of cf-associated mutant cystic fibrosis transmembrane conductance regulator
AU2009298802A1 (en) 2008-09-23 2010-04-08 Alnylam Pharmaceuticals, Inc. Chemical modifications of monomers and oligonucleotides with cycloaddition
AU2009303345B2 (en) 2008-10-09 2015-08-20 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
CN104382853A (en) 2008-10-16 2015-03-04 玛瑞纳生物技术有限公司 Processes and Compositions for Liposomal and Efficient Delivery of Gene Silencing Therapeutics
JP5864257B2 (en) 2008-10-24 2016-02-17 サレプタ セラピューティクス， インコーポレイテッド Multiple exon skipping compositions for DMD
IT1391866B1 (en) * 2008-10-30 2012-01-27 Mastelli S R L INJECTIONABLE COMPOSITION OF POLYNUCLEOTIDS FOR THE TREATMENT OF OSTEOARTICULAR DISEASES.
HUE037082T2 (en) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Novel lipids and compositions for the delivery of therapeutics
US20120101148A1 (en) 2009-01-29 2012-04-26 Alnylam Pharmaceuticals, Inc. lipid formulation
US9364477B2 (en) 2009-02-12 2016-06-14 Cell Signaling Technology, Inc. Mutant ROS expression in human cancer
WO2010091878A2 (en) 2009-02-13 2010-08-19 Silence Therapeutics Ag Means for inhibiting the expression of opa1
JP2012517815A (en) 2009-02-18 2012-08-09 サイレンス・セラピューティクス・アーゲー Means for inhibiting the expression of ANG2
EP2398479A2 (en) 2009-02-19 2011-12-28 Galapagos N.V. Methods for identifying and compounds useful for the diagnosis and treatment of diseases involving inflammation
US20120035245A1 (en) 2009-02-19 2012-02-09 Richard Antonius Jozef Janssen Methods for identifying and compounds useful for the diagnosis and treatment of diseases involving inflammation
EP2398481A2 (en) 2009-02-19 2011-12-28 Galapagos N.V. Methods for identifying and compounds useful for the diagnosis and treatment of diseases involving inflammation
AU2010221419B2 (en) 2009-03-02 2015-10-01 Alnylam Pharmaceuticals, Inc. Nucleic acid chemical modifications
AU2010224170B2 (en) 2009-03-11 2015-12-24 Promedior, Inc. Treatment and diagnostic methods for hypersensitive disorders
PT2405929T (en) 2009-03-11 2018-07-23 Promedior Inc A sap polypeptide for use in the treatment of autoimmune disorders and graft vs host disease
WO2010107958A1 (en) 2009-03-19 2010-09-23 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF SIGNAL TRANSDUCER AND ACTIVATOR OF TRANSCRIPTION 6 (STAT6) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2010107957A2 (en) 2009-03-19 2010-09-23 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GATA BINDING PROTEIN 3 (GATA3) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
EP2408916A2 (en) 2009-03-19 2012-01-25 Merck Sharp&Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF CONNECTIVE TISSUE GROWTH FACTOR (CTGF) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
AU2010226604A1 (en) 2009-03-19 2011-10-13 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of BTB and CNC homology 1, basic leucine zipper transcription factor 1 (Bach 1) gene expression using short interfering nucleic acid (siNA) sequence listing
JP2012521765A (en) 2009-03-27 2012-09-20 メルク・シャープ・エンド・ドーム・コーポレイション RNA interference-mediated inhibition of intracellular adhesion molecule 1 (ICAM-1) gene expression using small interfering nucleic acids (siNA)
EP2411516A1 (en) 2009-03-27 2012-02-01 Merck Sharp&Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF APOPTOSIS SIGNAL-REGULATING KINASE 1 (ASK1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
JP2012521763A (en) 2009-03-27 2012-09-20 メルク・シャープ・エンド・ドーム・コーポレイション RNA interference-mediated inhibition of signal transduction transcription factor 1 (STAT1) gene expression using small interfering nucleic acids (siNA)
JP2012521762A (en) 2009-03-27 2012-09-20 メルク・シャープ・エンド・ドーム・コーポレイション RNA interference-mediated inhibition of nerve growth factor β chain (NGFβ) gene expression using small interfering nucleic acids (siNA)
JP2012521764A (en) 2009-03-27 2012-09-20 メルク・シャープ・エンド・ドーム・コーポレイション RNA interference-mediated inhibition of thymic stromal lymphocyte formation factor (TSLP) gene expression using small interfering nucleic acids (siNA)
WO2010115825A2 (en) 2009-03-31 2010-10-14 Robert Zimmermann Modulation of adipose triglyceride lipase for prevention and treatment of cachexia, loss of weight and muscle atrophy and methods of screening therefor
WO2010112569A1 (en) 2009-03-31 2010-10-07 Robert Zimmermann Modulation of adipose triglyceride lipase for prevention and treatment of cachexia, loss of weight and muscle atrophy and methods of screening therefor
US8691825B2 (en) 2009-04-01 2014-04-08 Merck Sharp & Dohme Corp. Inhibitors of AKT activity
EP3043179B1 (en) 2009-04-01 2020-05-06 Galapagos N.V. Methods and means for treatment of osteoarthritis
EP2421972A2 (en) 2009-04-24 2012-02-29 The Board of Regents of The University of Texas System Modulation of gene expression using oligomers that target gene regions downstream of 3' untranslated regions
JP5769701B2 (en) 2009-05-05 2015-08-26 テクミラ ファーマシューティカルズ コーポレイションＴｅｋｍｉｒａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｃｏｒｐｏｒａｔｉｏｎ Lipid composition
AU2010245933B2 (en) 2009-05-05 2016-06-16 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
CN102638982B (en) 2009-05-12 2015-07-08 百时美施贵宝公司 Crystalline forms of (s)-7-([1,2,4]triazolo[1,5-a]pyridin-6-yl)-4-(3,4-dichlorohphenyl)-1,2,3,4-tetrahydroisoquinoline and use thereof
CA2760837C (en) 2009-05-12 2018-04-03 Albany Molecular Research, Inc. 7-([1,2,4]triazolo[1,5-.alpha.]pyridin-6-yl)-4-(3,4-dichlorophenyl)-1,2,3,4-tetrahydroisoquinoline and use thereof
KR20120034644A (en) 2009-05-12 2012-04-12 알바니 몰레큘라 리써치, 인크. Aryl, heteroaryl, and heterocycle substituted tetrahydroisoquinolines and use thereof
UA110323C2 (en) 2009-06-04 2015-12-25 Promedior Inc Derivative of serum amyloid p and their receipt and application
DK2440183T3 (en) 2009-06-10 2018-10-01 Arbutus Biopharma Corp Improved lipid formulation
CA2767127A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
US9512164B2 (en) 2009-07-07 2016-12-06 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
WO2011015573A1 (en) 2009-08-03 2011-02-10 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases
WO2011015572A1 (en) 2009-08-03 2011-02-10 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases
EP2480890B1 (en) 2009-09-24 2018-02-28 Anima Biotech Inc. Methods for measuring translation of target proteins in cells
WO2011046771A1 (en) 2009-10-14 2011-04-21 Schering Corporation SUBSTITUTED PIPERIDINES THAT INCREASE p53 ACTIVITY AND THE USES THEREOF
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
CN105838714B (en) 2009-11-12 2020-07-17 西澳大利亚大学 Antisense molecules and methods of treating diseases
EP3199634A1 (en) 2009-11-13 2017-08-02 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating influenza viral infection
US9687550B2 (en) 2009-12-07 2017-06-27 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
TWI465238B (en) 2009-12-09 2014-12-21 Nitto Denko Corp Modulation of hsp47 expression
ES2749426T3 (en) 2009-12-18 2020-03-20 Univ British Columbia Nucleic Acid Administration Methods and Compositions
WO2011094580A2 (en) 2010-01-28 2011-08-04 Alnylam Pharmaceuticals, Inc. Chelated copper for use in the preparation of conjugated oligonucleotides
EP2545173A2 (en) 2010-03-12 2013-01-16 Sarepta Therapeutics, Inc. Antisense modulation of nuclear hormone receptors
WO2011120023A1 (en) 2010-03-26 2011-09-29 Marina Biotech, Inc. Nucleic acid compounds for inhibiting survivin gene expression uses thereof
WO2011123621A2 (en) 2010-04-01 2011-10-06 Alnylam Pharmaceuticals Inc. 2' and 5' modified monomers and oligonucleotides
WO2011133584A2 (en) 2010-04-19 2011-10-27 Marina Biotech, Inc. Nucleic acid compounds for inhibiting hras gene expression and uses thereof
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
WO2011133876A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
US20130260460A1 (en) 2010-04-22 2013-10-03 Isis Pharmaceuticals Inc Conformationally restricted dinucleotide monomers and oligonucleotides
ES2711256T3 (en) 2010-04-23 2019-04-30 Univ Florida Compositions of rAAV-guanylate cyclase and methods to treat congenital amaurosis of Leber 1 (LCA1)
WO2011139842A2 (en) 2010-04-28 2011-11-10 Marina Biotech, Inc. Nucleic acid compounds for inhibiting fgfr3 gene expression and uses thereof
CA3090304A1 (en) 2010-05-13 2011-11-17 Sarepta Therapeutics, Inc. Antisense modulation of interleukins 17 and 23 signaling
CA2800065A1 (en) 2010-05-21 2011-11-24 Peptimed, Inc. Reagents and methods for treating cancer
WO2011153323A2 (en) 2010-06-02 2011-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
WO2011163121A1 (en) 2010-06-21 2011-12-29 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
CA2801928C (en) 2010-06-24 2018-04-10 Quark Pharmaceuticals, Inc. Double stranded rna compounds to rhoa and use thereof
WO2011163330A1 (en) 2010-06-24 2011-12-29 Merck Sharp & Dohme Corp. Novel heterocyclic compounds as erk inhibitors
US20130171241A1 (en) 2010-07-06 2013-07-04 Novartis Ag Liposomes with lipids having an advantageous pka-value for rna delivery
LT3243526T (en) 2010-07-06 2020-02-10 Glaxosmithkline Biologicals S.A. Delivery of rna to trigger multiple immune pathways
DK2591114T3 (en) 2010-07-06 2016-08-29 Glaxosmithkline Biologicals Sa Immunization of large mammals with low doses of RNA
LT2590676T (en) 2010-07-06 2016-10-25 Glaxosmithkline Biologicals Sa Virion-like delivery particles for self-replicating rna molecules
AU2011281121A1 (en) 2010-07-22 2013-02-21 Anima Cell Metrology, Inc. Systems and methods for detection of cellular stress
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
US20130202652A1 (en) 2010-07-30 2013-08-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
CA2805265A1 (en) 2010-08-02 2012-02-09 Merck Sharp & Dohme Corp. Rna interference mediated inhibition of catenin (cadherin-associated protein), beta 1 (ctnnb1) gene expression using short interfering nucleic acid (sina)
US20130210663A1 (en) 2010-08-04 2013-08-15 Cizzle Biotechnology Limited Methods and compounds for the diagnosis and treatment of cancer
US20120101108A1 (en) 2010-08-06 2012-04-26 Cell Signaling Technology, Inc. Anaplastic Lymphoma Kinase In Kidney Cancer
WO2012024170A2 (en) 2010-08-17 2012-02-23 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS B VIRUS (HBV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US8883801B2 (en) 2010-08-23 2014-11-11 Merck Sharp & Dohme Corp. Substituted pyrazolo[1,5-a]pyrimidines as mTOR inhibitors
EP3372684B1 (en) 2010-08-24 2020-10-07 Sirna Therapeutics, Inc. Single-stranded rnai agents containing an internal, non-nucleic acid spacer
EP2609106A4 (en) 2010-08-26 2014-03-19 Merck Sharp & Dohme RNA INTERFERENCE MEDIATED INHIBITION OF PROLYL HYDROXYLASE DOMAIN 2 (PHD2) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
ES2918649T3 (en) 2010-08-31 2022-07-19 Glaxosmithkline Biologicals Sa Pegylated liposomes for delivery of RNA encoding an immunogen
MX341989B (en) * 2010-08-31 2016-09-09 Novartis Ag * Small liposomes for delivery of immunogen-encoding rna.
US8946216B2 (en) 2010-09-01 2015-02-03 Merck Sharp & Dohme Corp. Indazole derivatives useful as ERK inhibitors
EP2615916B1 (en) 2010-09-16 2017-01-04 Merck Sharp & Dohme Corp. Fused pyrazole derivatives as novel erk inhibitors
WO2012047631A2 (en) 2010-09-27 2012-04-12 The Children's Hospital Of Philadelphia Compositions and methods useful for the treatment and diagnosis of inflammatory bowel disease
SG10201508118WA (en) 2010-09-30 2015-11-27 Agency Science Tech & Res Methods and reagents for detection and treatment of esophageal metaplasia
BR112013008700B8 (en) 2010-10-11 2022-10-04 Novartis Ag SELF-REPLICATING RNA MOLECULE, ALPHAVIRUS REPLICON PARTICLE, COMPOSITION, RECOMBINANT DNA MOLECULE, USE OF SELF-REPLICATING RNA MOLECULE
US20140134231A1 (en) 2010-10-11 2014-05-15 Sanford-Burnham Medical Research Institute Mir-211 expression and related pathways in human melanoma
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
AU2011323508B2 (en) 2010-11-01 2017-04-27 Peptimed, Inc. Compositions of a peptide targeting system for treating cancer
EP2649182A4 (en) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc Compositions and methods for increasing erythropoietin (epo) production
WO2012079046A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
WO2012087772A1 (en) 2010-12-21 2012-06-28 Schering Corporation Indazole derivatives useful as erk inhibitors
WO2012093384A1 (en) 2011-01-03 2012-07-12 Rosetta Genomics Ltd. Compositions and methods for treatment of ovarian cancer
AU2012207606B2 (en) 2011-01-11 2017-02-23 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
JP2014510721A (en) 2011-02-15 2014-05-01 イミューン デザイン コーポレイション Methods for enhancing immunogen-specific immune responses with vector vaccines
EP2681314B1 (en) 2011-03-03 2017-11-01 Quark Pharmaceuticals, Inc. Compositions and methods for treating lung disease and injury
US8420617B2 (en) 2011-03-11 2013-04-16 Biocell Laboratories Multiantivirus compound, composition and method for treatment of virus diseases
KR102481317B1 (en) 2011-03-29 2022-12-26 알닐람 파마슈티칼스 인코포레이티드 Compositions and methods for inhibiting expression of tmprss6 gene
NZ616304A (en) 2011-04-08 2016-01-29 Immune Design Corp Immunogenic compositions and methods of using the compositions for inducing humoral and cellular immune responses
JP2014514321A (en) 2011-04-21 2014-06-19 メルク・シャープ・アンド・ドーム・コーポレーション Insulin-like growth factor 1 receptor inhibitor
TWI658830B (en) 2011-06-08 2019-05-11 日東電工股份有限公司 Retinoid-liposomes for enhancing modulation of hsp47 expression
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
MX345095B (en) 2011-06-21 2017-01-17 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (angptl3) irna compostions and methods of use thereof.
WO2012177595A1 (en) 2011-06-21 2012-12-27 Oncofactor Corporation Compositions and methods for the therapy and diagnosis of cancer
CA2839896A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
EP3597750B1 (en) 2011-06-23 2022-05-04 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
WO2013003697A1 (en) 2011-06-30 2013-01-03 Trustees Of Boston University Method for controlling tumor growth, angiogenesis and metastasis using immunoglobulin containing and proline rich receptor-1 (igpr-1)
WO2013006838A1 (en) 2011-07-06 2013-01-10 Novartis Ag Immunogenic combination compositions and uses thereof
EP2557089A2 (en) 2011-07-15 2013-02-13 Fundació Institut d'Investigació Biomèdica de Bellvitge (IDIBELL) Compositions and methods for immunomodulation
AU2012284259A1 (en) 2011-07-15 2014-03-06 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
US20140328811A1 (en) 2011-08-01 2014-11-06 Alnylam Pharmaceuticals, Inc. Method for improving the success rate of hematopoietic stem cell transplants
US9701623B2 (en) 2011-09-27 2017-07-11 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
US9023865B2 (en) 2011-10-27 2015-05-05 Merck Sharp & Dohme Corp. Compounds that are ERK inhibitors
BR112014011417B1 (en) 2011-11-11 2021-10-13 Fred Hutchinson Cancer Research Center ISOLATED POLYPEPTIDE CAPABLE OF ELIGITING AN ANTIGEN-SPECIFIC T-CELL RESPONSE TO HUMAN CYCLIN A1 IMMUNOGENIC COMPOSITION INCLUDING SUCH POLYPEPTIDE, AS WELL AS A METHOD TO PREPARE CELLS PRESENTING SUPER ANTIGEN, ANTIGEN, ANTIGEN-USED ANTIGEN-USED CELLS
HUE038369T2 (en) 2011-12-08 2018-10-29 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna
EP2790736B1 (en) 2011-12-12 2018-01-31 Oncoimmunin, Inc. In vivo delivery of oligonucleotides
CA2858630A1 (en) 2012-01-12 2013-07-18 Quark Pharmaceuticals, Inc. Combination therapy for treating hearing and balance disorders
EP2617434A1 (en) 2012-01-20 2013-07-24 Laboratorios Del. Dr. Esteve, S.A. HIV-1 integrase deficient immunogens and methods for loading dendritic cells with said immunogens
US9352042B2 (en) 2012-02-24 2016-05-31 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
WO2013123996A1 (en) 2012-02-24 2013-08-29 Astrazeneca Uk Limited Novel sirna inhibitors of human icam-1
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
DK2838998T3 (en) 2012-04-18 2018-01-15 Cell Signaling Technology Inc EGFR AND ROS1 IN CANCER
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
US20150299696A1 (en) 2012-05-02 2015-10-22 Sirna Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
KR102213609B1 (en) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 Chiral control
WO2014018375A1 (en) 2012-07-23 2014-01-30 Xenon Pharmaceuticals Inc. Cyp8b1 and uses thereof in therapeutic and diagnostic methods
DK2895608T3 (en) 2012-09-12 2019-01-21 Quark Pharmaceuticals Inc DOUBLE-STRENGTHED OIGONUCLEOTIDE MOLECULES FOR P53 AND PROCEDURES FOR USING IT
BR112015004747A2 (en) 2012-09-12 2017-11-21 Quark Pharmaceuticals Inc p53 double stranded oligonucleotide molecules and methods of using them
WO2014052563A2 (en) 2012-09-28 2014-04-03 Merck Sharp & Dohme Corp. Novel compounds that are erk inhibitors
MX363243B (en) 2012-11-28 2019-03-14 Merck Sharp & Dohme Compositions and methods for treating cancer.
KR102096014B1 (en) 2012-12-05 2020-04-03 알닐람 파마슈티칼스 인코포레이티드 PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
KR20200143739A (en) 2012-12-20 2020-12-24 사렙타 쎄러퓨틱스 인코퍼레이티드 Improved exon skipping compositions for treating muscular dystrophy
RU2690663C2 (en) 2012-12-20 2019-06-05 Мерк Шарп И Доум Корп. Substituted imidazopyridines as hdm2 inhibitors
EP3263559A1 (en) 2013-01-22 2018-01-03 Technische Universität Graz Lipase inhibitors
US9540377B2 (en) 2013-01-30 2017-01-10 Merck Sharp & Dohme Corp. 2,6,7,8 substituted purines as HDM2 inhibitors
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
DK2970964T3 (en) 2013-03-14 2019-04-01 Sarepta Therapeutics Inc EXON SKIPPING COMPOSITIONS FOR TREATMENT OF MUSCLE DYROPHY
EP2972381A2 (en) 2013-03-14 2016-01-20 Galapagos NV Molecular targets and compounds, and methods to identify the same, useful in the treatment of diseases associated with epithelial mesenchymal transition
JP6437467B2 (en) 2013-03-14 2018-12-19 ガラパゴス・ナムローゼ・フェンノートシャップＧａｌａｐａｇｏｓ Ｎ．Ｖ． Molecular targets and compounds useful in the treatment of fibrotic diseases and methods for identifying them
US9952202B2 (en) 2013-03-14 2018-04-24 Galapagos Nv Methods of identifying compounds for the treatment of fibrosis by using S1PR5
EA035882B1 (en) 2013-03-14 2020-08-27 Сарепта Терапьютикс, Инк. Antisense oligonucleotides inducing exon skipping for treating muscular dystrophy
KR102342916B1 (en) 2013-03-14 2021-12-24 알닐람 파마슈티칼스 인코포레이티드 Complement component c5 irna compositions and methods of use thereof
BR112015022998A2 (en) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc improved compositions for treating muscular dystrophy
US9937231B2 (en) 2013-03-27 2018-04-10 The General Hospital Corporation Methods and agents for treating Alzheimer's disease
AR096203A1 (en) 2013-05-06 2015-12-16 Alnylam Pharmaceuticals Inc DOSAGES AND METHODS FOR MANAGING NUCLEIC ACID MOLECULES FORMULATED IN LIPIDS
US20160186263A1 (en) 2013-05-09 2016-06-30 Trustees Of Boston University Using plexin-a4 as a biomarker and therapeutic target for alzheimer's disease
EP3587578A1 (en) 2013-05-22 2020-01-01 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
PT2999785T (en) 2013-05-22 2018-07-09 Alnylam Pharmaceuticals Inc Serpina1 irna compositions and methods of use thereof
WO2014203189A1 (en) 2013-06-18 2014-12-24 Rosetta Genomics Ltd. Nanocarrier system for micrornas and uses thereof
CA3177846A1 (en) 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
WO2015020960A1 (en) 2013-08-09 2015-02-12 Novartis Ag Novel lncrna polynucleotides
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
EP2853595A1 (en) 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules
WO2015050990A1 (en) 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
US10119143B2 (en) 2013-10-04 2018-11-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
PL3054959T3 (en) 2013-10-08 2020-07-13 Promedior Inc. Methods for treating fibrotic cancers
CN105814205B (en) 2013-12-12 2019-11-19 阿尔尼拉姆医药品有限公司 Complement component iRNA composition and its application method
MX2016009753A (en) 2014-01-28 2017-07-07 Buck Inst For Res On Aging Methods and compositions for killing senescent cells and for treating senescence-associated diseases and disorders.
EA201691587A1 (en) 2014-02-11 2017-01-30 Элнилэм Фармасьютикалз, Инк. COMPOSITIONS BASED ON iRNA FOR KETOGEXOKINASE (KHK) AND METHODS OF THEIR APPLICATION
EP3542816A1 (en) 2014-02-14 2019-09-25 Immune Design Corp. Immunotherapy of cancer through combination of local and systemic immune stimulation
TW201607559A (en) 2014-05-12 2016-03-01 阿尼拉製藥公司 Methods and compositions for treating a SERPINC1-associated disorder
KR20220087576A (en) 2014-05-22 2022-06-24 알닐람 파마슈티칼스 인코포레이티드 Angiotensinogen (agt) irna compositions and methods of use thereof
MX2017000630A (en) 2014-07-15 2017-04-27 Immune Design Corp Prime-boost regimens with a tlr4 agonist adjuvant and a lentiviral vector.
EP3186378A1 (en) 2014-08-27 2017-07-05 Peptimed, Inc. Anti-tumor compositions and methods
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
KR102617137B1 (en) 2014-09-15 2023-12-27 칠드런'즈 메디컬 센터 코포레이션 Methods and compositions to increase somatic cell nuclear transfer (scnt) efficiency by removing histone h3-lysine trimethylation
JOP20200115A1 (en) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc Compositions And Methods For Inhibition Of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) Gene Expression
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3212794B1 (en) 2014-10-30 2021-04-07 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (en) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc HEPATITIS B VIRUS (HBV) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP3221451A1 (en) 2014-11-17 2017-09-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
WO2016086227A2 (en) 2014-11-26 2016-06-02 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
WO2016083624A1 (en) 2014-11-28 2016-06-02 Silence Therapeutics Gmbh Means for inhibiting the expression of edn1
JP2018510621A (en) 2015-02-13 2018-04-19 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Patatin-like phospholipase domain-containing 3 (PNPLA3) iRNA compositions and methods of use thereof
US10781445B2 (en) 2015-03-11 2020-09-22 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Decoy oligonucleotides for the treatment of diseases
US10745702B2 (en) 2015-04-08 2020-08-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
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
US11053495B2 (en) 2015-07-17 2021-07-06 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
CA2994724A1 (en) 2015-08-21 2017-03-02 The Children's Hospital Of Philadelphia Compositions and methods for use in combination for the treatment and diagnosis of autoimmune diseases
WO2017040078A1 (en) 2015-09-02 2017-03-09 Alnylam Pharmaceuticals, Inc. PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
US20190054113A1 (en) 2015-09-30 2019-02-21 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
US20190255107A1 (en) 2015-10-09 2019-08-22 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
EP3368689B1 (en) 2015-10-28 2020-06-17 The Broad Institute, Inc. Composition for modulating immune responses by use of immune cell gene signature
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017184586A1 (en) 2016-04-18 2017-10-26 The Trustees Of Columbia University In The City Of New York Therapeutic targets involved in the progression of nonalcoholic steatohepatitis (nash)
MA45295A (en) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc HIGH DENSITY LIPOPROTEIN BINDING PROTEIN (HDLBP / VIGILINE) RNA COMPOSITION AND METHODS FOR USING THEM
KR102329187B1 (en) 2016-04-29 2021-11-22 사렙타 쎄러퓨틱스, 인코퍼레이티드 Oligonucleotide analogues targeting human LMNA
EP3469083A1 (en) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
JP2019525742A (en) 2016-06-30 2019-09-12 サレプタ セラピューティクス， インコーポレイテッド Exon skipping oligomer for muscular dystrophy
EP3269734A1 (en) 2016-07-15 2018-01-17 Fundació Privada Institut d'Investigació Oncològica de Vall-Hebron Methods and compositions for the treatment of cancer
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
TWI788312B (en) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP3330276A1 (en) 2016-11-30 2018-06-06 Universität Bern Novel bicyclic nucleosides and oligomers prepared therefrom
SG10201913552UA (en) 2016-12-16 2020-03-30 Alnylam Pharmaceuticals Inc Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
BR112019012647A2 (en) 2016-12-19 2019-11-19 Sarepta Therapeutics Inc exon jump oligomer conjugates for muscular dystrophy
AU2017382773A1 (en) 2016-12-19 2019-08-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2018118627A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
CA3059446A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
AU2018301477A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof
EA201991450A1 (en) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. OLIGOMER CONJUGATES FOR EXONISM SKIP IN MUSCULAR DYSTROPHY
WO2019067981A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
JP2020536060A (en) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド Combination therapy to treat muscular dystrophy
EP3687519A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
US10947234B2 (en) 2017-11-08 2021-03-16 Merck Sharp & Dohme Corp. PRMT5 inhibitors
US20200385719A1 (en) 2017-11-16 2020-12-10 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
US20200308588A1 (en) 2017-12-18 2020-10-01 Alnylam Pharmaceuticals, Inc. High mobility group box-1 (hmgb1) irna compositions and methods of use thereof
CN112105625A (en) 2018-03-07 2020-12-18 赛诺菲 Nucleotide precursors, nucleotide analogs, and oligomeric compounds containing the same
TW202016304A (en) 2018-05-14 2020-05-01 美商阿尼拉製藥公司 Angiotensinogen (agt) irna compositions and methods of use thereof
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3806868A4 (en) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
TW202020153A (en) 2018-07-27 2020-06-01 美商薩羅塔治療公司 Exon skipping oligomers for muscular dystrophy
WO2020033284A1 (en) 2018-08-07 2020-02-13 Merck Sharp & Dohme Corp. Prmt5 inhibitors
EP3833668A4 (en) 2018-08-07 2022-05-11 Merck Sharp & Dohme Corp. Prmt5 inhibitors
JP2021533767A (en) 2018-08-13 2021-12-09 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Alnylam Pharmaceuticals, Inc. Hepatitis B virus (HBV) dsRNA substance composition and its usage
US20210348162A1 (en) 2018-08-16 2021-11-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
CN112424355A (en) 2018-09-18 2021-02-26 阿尔尼拉姆医药品有限公司 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
WO2020104649A2 (en) 2018-11-23 2020-05-28 Sanofi Novel rna compositions and methods for inhibiting angptl8
KR20210104759A (en) 2018-12-13 2021-08-25 사렙타 쎄러퓨틱스 인코퍼레이티드 Exon skipping oligomer conjugates for muscular dystrophy
CN113631709A (en) 2018-12-20 2021-11-09 普拉克西斯精密药物股份有限公司 Compositions and methods for treating KCNT 1-related disorders
CN113543791A (en) 2018-12-20 2021-10-22 维尔生物科技有限公司 Combination HBV therapy
CA3133155A1 (en) 2019-03-19 2020-09-24 Fundacio Privada Institut D'investigacio Oncologica De Vall Hebron Combination therapy for the treatment for cancer
WO2020198268A1 (en) 2019-03-28 2020-10-01 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy with casimersen
WO2020214763A1 (en) 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
WO2020257489A1 (en) 2019-06-19 2020-12-24 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
JP2022541197A (en) 2019-07-16 2022-09-22 ユニヴェルシテ・コート・ダジュール Methods of assessing susceptibility or resistance of a subject to oncolytic viruses, recombinant viruses, their preparation and use
WO2021022109A1 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. SERPIN FAMILY F MEMBER 2 (SERPINF2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
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
US20230097090A1 (en) 2019-08-14 2023-03-30 Acuitas Therapeutics, Inc. Improved lipid nanoparticles for delivery of nucleic acids
CN115176011A (en) 2019-08-27 2022-10-11 赛诺菲 Compositions and methods for inhibiting PCSK9
CN114616331A (en) 2019-09-03 2022-06-10 阿尔尼拉姆医药品有限公司 Compositions and methods for inhibiting expression of LECT2 gene
CN114728999A (en) 2019-09-05 2022-07-08 赛诺菲 Oligonucleotides containing nucleotide analogs
EP4041894A1 (en) 2019-09-23 2022-08-17 Omega Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING HEPATOCYTE NUCLEAR FACTOR 4-ALPHA (HNF4a) GENE EXPRESSION
AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
WO2021071830A1 (en) 2019-10-07 2021-04-15 University Of Virginia Patent Foundation Modulating lymphatic vessels in neurological disease
EP3808763A1 (en) 2019-10-17 2021-04-21 Fundació Institut d'Investigació Biomèdica de Bellvitge (IDIBELL) Compounds for immunomodulation
EP4045652A1 (en) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
CN115176004A (en) 2019-10-22 2022-10-11 阿尔尼拉姆医药品有限公司 Complement component C3 iRNA compositions and methods of use thereof
AR120341A1 (en) 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSITIONS OF RNAi AGENTS AGAINST HUNTINGTINE (HTT) AND THEIR METHODS OF USE
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
BR112022009216A2 (en) 2019-11-13 2022-08-02 Alnylam Pharmaceuticals Inc METHODS AND COMPOSITIONS TO TREAT AN ANGIOTENSINOGEN-ASSOCIATED DISORDER (AGT)
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
BR112022011417A2 (en) 2019-12-13 2022-08-30 Alnylam Pharmaceuticals Inc COMPOSITIONS OF THE IRNA AGENT OF THE OPEN READING PHASE 72 OF HUMAN CHROMOSOME 9 (C9ORF72) AND METHODS OF USE THEREOF
TW202138559A (en) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2021151079A1 (en) 2020-01-24 2021-07-29 University Of Virginia Patent Foundation Modulating lymphatic vessels in neurological disease
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
KR20220140593A (en) 2020-02-10 2022-10-18 알닐람 파마슈티칼스 인코포레이티드 Compositions and methods for silencing VEGF-A expression
IL295496A (en) 2020-02-18 2022-10-01 Alnylam Pharmaceuticals Inc Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
JP2023516748A (en) 2020-03-06 2023-04-20 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 Compositions and methods for inhibiting expression of transthyretin (TTR)
AU2021232014A1 (en) 2020-03-06 2022-10-06 Alnylam Pharmaceuticals, Inc. Ketohexokinase (KHK) IRNA compositions and methods of use thereof
AU2021234302A1 (en) 2020-03-11 2022-11-10 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
JP2023519274A (en) 2020-03-26 2023-05-10 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 CORONAVIRUS iRNA COMPOSITIONS AND METHODS OF USE THEREOF
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
US20230295622A1 (en) 2020-04-06 2023-09-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
JP2023521094A (en) 2020-04-07 2023-05-23 アルナイラム ファーマシューティカルズ， インコーポレイテッド Compositions and methods for silencing SCN9A expression
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
JP2023523993A (en) 2020-04-27 2023-06-08 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 Apolipoprotein E (ApoE) iRNA agent compositions and methods of use thereof
CN116096381A (en) 2020-04-30 2023-05-09 阿尔尼拉姆医药品有限公司 Complement Factor B (CFB) iRNA compositions and methods of use thereof
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)
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
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)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
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
CA3184289A1 (en) 2020-06-18 2021-12-23 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof
CA3182458A1 (en) 2020-06-24 2021-12-30 Laura ROSEN Engineered hepatitis b virus neutralizing antibodies and uses thereof
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
JP2023546103A (en) 2020-10-16 2023-11-01 サノフイ Novel RNA compositions and methods for inhibiting ANGPTL3
JP2023545502A (en) 2020-10-16 2023-10-30 サノフイ RNA compositions and methods for inhibiting lipoprotein (A)
EP4232455A2 (en) 2020-10-20 2023-08-30 Sanofi Novel ligands for asialoglycoprotein receptor
AU2021365822A1 (en) 2020-10-21 2023-06-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
IL302709A (en) 2020-11-13 2023-07-01 Alnylam Pharmaceuticals Inc COAGULATION FACTOR V (F5) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CA3201452A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
WO2022150260A1 (en) 2021-01-05 2022-07-14 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
AU2022226098A1 (en) 2021-02-26 2023-08-24 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
IL305418A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
AR125230A1 (en) 2021-03-29 2023-06-28 Alnylam Pharmaceuticals Inc COMPOSITIONS OF ANTI-HUNTINGTIN (HTT) RNAi AGENTS AND THEIR METHODS OF USE
EP4314016A1 (en) 2021-03-31 2024-02-07 Entrada Therapeutics, Inc. Cyclic cell penetrating peptides
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
EP4330392A1 (en) 2021-04-26 2024-03-06 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4330395A1 (en) 2021-04-30 2024-03-06 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
WO2022240721A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
AU2022271873A1 (en) 2021-05-10 2024-01-04 Entrada Therapeutics, Inc. Compositions and methods for intracellular therapeutics
EP4337261A2 (en) 2021-05-10 2024-03-20 Entrada Therapeutics, Inc. Compositions and methods for modulating mrna splicing
WO2022245583A1 (en) 2021-05-18 2022-11-24 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
TW202317762A (en) 2021-06-02 2023-05-01 美商艾拉倫製藥股份有限公司 Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
IL308743A (en) 2021-06-04 2024-01-01 Alnylam Pharmaceuticals Inc HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
AR126070A1 (en) 2021-06-08 2023-09-06 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR TREATING OR PREVENTING STARGARDT DISEASE AND/OR DISORDERS ASSOCIATED WITH RETINOL BORDER PROTEIN 4 (RBP4)
WO2022271818A1 (en) 2021-06-23 2022-12-29 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
KR20240026203A (en) 2021-06-30 2024-02-27 알닐람 파마슈티칼스 인코포레이티드 Methods and compositions for treating angiotensinogen (AGT)-related disorders
WO2023283359A2 (en) 2021-07-07 2023-01-12 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
TW202333748A (en) 2021-07-19 2023-09-01 美商艾拉倫製藥股份有限公司 Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder
KR20240036041A (en) 2021-07-21 2024-03-19 알닐람 파마슈티칼스 인코포레이티드 Metabolic Disorder-Associated Target Gene iRNA Composition and Methods of Using Same
IL309905A (en) 2021-07-23 2024-03-01 Alnylam Pharmaceuticals Inc Beta-catenin (ctnnb1) irna compositions and methods of use thereof
WO2023009687A1 (en) 2021-07-29 2023-02-02 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
WO2023014677A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
TW202337474A (en) 2021-08-04 2023-10-01 美商艾拉倫製藥股份有限公司 Irna compositions and methods for silencing angiotensinogen (agt)
AU2022324471A1 (en) 2021-08-05 2024-02-15 Sanegene Bio Usa Inc. 1'-alkyl modified ribose derivatives and methods of use
TW202334413A (en) 2021-08-13 2023-09-01 美商艾拉倫製藥股份有限公司 Factor xii (f12) irna compositions and methods of use thereof
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
WO2023044094A1 (en) 2021-09-20 2023-03-23 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
CA3233113A1 (en) 2021-09-22 2023-03-30 Sanegene Bio Usa Inc. 2'-alkyl or 3'-alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
US20230227480A1 (en) 2021-10-05 2023-07-20 Sanegene Bio Usa Inc. Polyhydroxylated cyclopentane derivatives and methods of use
WO2023064530A1 (en) 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
US20230277600A1 (en) 2021-10-20 2023-09-07 University Of Rochester Treatment Of Age-Related White Matter Loss By Competitive Replacement Of Glial Cells
CA3234835A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
TW202333749A (en) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 Complement factor b (cfb) irna compositions and methods of use thereof
WO2023091704A1 (en) 2021-11-18 2023-05-25 Circularis Biotechnologies, Inc. Compositions and methods for production of circular nucleic acid molecules
WO2023122762A1 (en) 2021-12-22 2023-06-29 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023144792A1 (en) 2022-01-31 2023-08-03 Genevant Sciences Gmbh Poly(alkyloxazoline)-lipid conjugates and lipid particles containing same
WO2023144798A1 (en) 2022-01-31 2023-08-03 Genevant Sciences Gmbh Ionizable cationic lipids for lipid nanoparticles
WO2023164464A1 (en) 2022-02-22 2023-08-31 Sanegene Bio Usa Inc. 5'-modified carbocyclic ribonucleotide derivatives and methods of use
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
WO2023240277A2 (en) 2022-06-10 2023-12-14 Camp4 Therapeutics Corporation Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory rnas
WO2024015796A1 (en) 2022-07-11 2024-01-18 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994010301A1 (en) * 1992-11-03 1994-05-11 Gene Shears Pty. Limited TNF-α RIBOZYMES AND DEGRADATION RESISTANT mRNA DERIVATIVES LINKED TO TNF-α RIBOZYMES

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5168053A (en) * 1989-03-24 1992-12-01 Yale University Cleavage of targeted RNA by RNAase P
US5272263A (en) * 1989-04-28 1993-12-21 Biogen, Inc. DNA sequences encoding vascular cell adhesion molecules (VCAMS)
IE20010427A1 (en) * 1990-04-10 2003-03-05 Canji Inc Gene therapy for cell proliferative diseases
GB9106678D0 (en) * 1991-03-28 1991-05-15 Ferguson Mark W J Wound healing
CA2183992A1 (en) * 1994-02-23 1995-08-31 Dan T. Stinchcomb Method and reagent for inhibiting the expression of disease related genes

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994010301A1 (en) * 1992-11-03 1994-05-11 Gene Shears Pty. Limited TNF-α RIBOZYMES AND DEGRADATION RESISTANT mRNA DERIVATIVES LINKED TO TNF-α RIBOZYMES

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
EDGINGTON, S.M.: "ribozymes: stop making sense", BIOTECHNOLOGY, vol. 10, NEW YORK US, pages 256 - 262, XP001018952, DOI: doi:10.1038/nbt0392-256 *
JOINT MEETING OF THE ASSOCIATION OF AMERICAN PHYSICIANS, THE AMERICAN SOCIETY FOR CLINICAL INVESTIGATION, AND THE AMERICAN FEDERATION FOR CLINICAL RESEARCH, WASHINGTON, DC, USA, APRIL 30-MAY 3, 1993. *
KISICH, K. & ERICKSON, K.: "ANTI-TUMOR NECROSIS FACTOR - ALPHA RIBOZYMES CAN SPECIFICALLY CLEAVE TUMOR NECROSIS FACTOR - ALPHA MESSENGER RNA WITHIN MAMMALIAN CELLS.", J. LEUKOCYTE BIOL. 0 (SUPPL. 2). 1991. 70 *
KISICH, K. ET AL.: "REGULATION OF TUMOR NECROSIS FACTOR - ALPHA GENE EXPRESSION FOLLOWING RIBOZYME TRANSFECTION A GENETICALLY ENGINEERED MODEL FOR THE TREATMENT OF TOXIC SHOCK.", CLIN RES 41 (2). 137A *
See also references of WO9402595A1 *
SIOUD, M. ET AL.: "Preformed ribozyme destroys tumor necrosis factor mRNA in human cells", J. MOL. BIOL. 223 (4), 831-5, 20 February 1992 (1992-02-20) *
TWENTY-EIGHTH NATIONAL MEETING OF THE SOCIETY FOR LEUKOCYTE BIOLOGY AND THE TWENTY-FIRST LEUKOCYTE CULTURE CONFERENCE, ASPEN, COLORADO, USA, 28 September 1991 (1991-09-28) *

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