CA2099712A1 - Carbocyclic nucleosides containing bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates - Google Patents

Carbocyclic nucleosides containing bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates

Info

Publication number
CA2099712A1
CA2099712A1 CA002099712A CA2099712A CA2099712A1 CA 2099712 A1 CA2099712 A1 CA 2099712A1 CA 002099712 A CA002099712 A CA 002099712A CA 2099712 A CA2099712 A CA 2099712A CA 2099712 A1 CA2099712 A1 CA 2099712A1
Authority
CA
Canada
Prior art keywords
formula
oligonucleotide
atoms
amino
formulae
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.)
Abandoned
Application number
CA002099712A
Other languages
English (en)
French (fr)
Inventor
Karl-Heinz Altmann
Rene Imwinkelried
Albert Eschenmoser
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.)
Novartis AG
Original Assignee
Ciba Geigy AG
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 Ciba Geigy AG filed Critical Ciba Geigy AG
Publication of CA2099712A1 publication Critical patent/CA2099712A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
    • C07D239/46Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/47One nitrogen atom and one oxygen or sulfur atom, e.g. cytosine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C215/00Compounds containing amino and hydroxy groups bound to the same carbon skeleton
    • C07C215/42Compounds containing amino and hydroxy groups bound to the same carbon skeleton having amino groups or hydroxy groups bound to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton
    • C07C215/44Compounds containing amino and hydroxy groups bound to the same carbon skeleton having amino groups or hydroxy groups bound to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton bound to carbon atoms of the same ring or condensed ring system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C275/00Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups
    • C07C275/46Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups containing any of the groups, X being a hetero atom, Y being any atom, e.g. acylureas
    • C07C275/48Y being a hydrogen or a carbon atom
    • C07C275/50Y being a hydrogen or an acyclic carbon atom
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
    • C07D239/46Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/48Two nitrogen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
    • C07D239/46Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/50Three nitrogen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
    • C07D239/46Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/52Two oxygen atoms
    • C07D239/54Two oxygen atoms as doubly bound oxygen atoms or as unsubstituted hydroxy radicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D473/00Heterocyclic compounds containing purine ring systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F7/00Compounds containing elements of Groups 4 or 14 of the Periodic Table
    • C07F7/02Silicon compounds
    • C07F7/08Compounds having one or more C—Si linkages
    • C07F7/18Compounds having one or more C—Si linkages as well as one or more C—O—Si linkages
    • C07F7/1804Compounds having Si-O-C linkages
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/645Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having two nitrogen atoms as the only ring hetero atoms
    • C07F9/6509Six-membered rings
    • C07F9/6512Six-membered rings having the nitrogen atoms in positions 1 and 3
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
    • C07F9/65616Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings containing the ring system having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02PCLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00Technologies relating to chemical industry
    • Y02P20/50Improvements relating to the production of bulk chemicals
    • Y02P20/55Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Communicable Diseases (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Saccharide Compounds (AREA)
CA002099712A 1992-07-01 1993-06-29 Carbocyclic nucleosides containing bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates Abandoned CA2099712A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CH2075/92-7 1992-07-01
CH207592 1992-07-01

Publications (1)

Publication Number Publication Date
CA2099712A1 true CA2099712A1 (en) 1994-01-02

Family

ID=4225321

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002099712A Abandoned CA2099712A1 (en) 1992-07-01 1993-06-29 Carbocyclic nucleosides containing bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates

Country Status (5)

Country Link
US (3) US5461152A (cg-RX-API-DMAC10.html)
EP (1) EP0577558A2 (cg-RX-API-DMAC10.html)
JP (1) JPH06100562A (cg-RX-API-DMAC10.html)
CA (1) CA2099712A1 (cg-RX-API-DMAC10.html)
MX (1) MX9303965A (cg-RX-API-DMAC10.html)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6124463A (en) * 1998-07-02 2000-09-26 Dupont Pharmaceuticals Benzimidazoles as corticotropin release factor antagonists
US6143743A (en) * 1997-07-03 2000-11-07 Dupont Pharmaceuticals Company Imidazopyrimidines and imidazopyridines for the treatment of neurological disorders
US6579876B2 (en) 1998-07-02 2003-06-17 Bristol-Myers Squibb Pharma Company Imidazo-pyridines, -pyridazines, and -triazines as corticotropin releasing factor antagonists

Families Citing this family (770)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US8153602B1 (en) 1991-11-19 2012-04-10 Isis Pharmaceuticals, Inc. Composition and methods for the pulmonary delivery of nucleic acids
JP3484197B2 (ja) 1993-09-03 2004-01-06 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 アミン誘導化ヌクレオシドおよびオリゴヌクレオシド
ATE160340T1 (de) * 1993-09-24 1997-12-15 Us Health Konformationell festgelegte nukleosidanaloge
JP3769737B2 (ja) * 1994-03-30 2006-04-26 味の素株式会社 シクロプロパン誘導体及びその製造法
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US20050053976A1 (en) * 1996-06-06 2005-03-10 Baker Brenda F. Chimeric oligomeric compounds and their use in gene modulation
US20040203024A1 (en) * 1996-06-06 2004-10-14 Baker Brenda F. Modified oligonucleotides for use in RNA interference
US7812149B2 (en) * 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US20030044941A1 (en) 1996-06-06 2003-03-06 Crooke Stanley T. Human RNase III and compositions and uses thereof
US20040147022A1 (en) * 1996-06-06 2004-07-29 Baker Brenda F. 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US20040171031A1 (en) * 1996-06-06 2004-09-02 Baker Brenda F. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
WO1998005662A1 (en) * 1996-08-07 1998-02-12 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Conformationally locked nucleoside analogs as antiherpetic agents
US6639062B2 (en) 1997-02-14 2003-10-28 Isis Pharmaceuticals, Inc. Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom
US6716625B1 (en) 1997-04-16 2004-04-06 Claude Selitrennikoff Histidine kinases of Aspergillus and other fungal species, related compositions, and methods of use
US6887906B1 (en) 1997-07-01 2005-05-03 Isispharmaceuticals, Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
US7321828B2 (en) * 1998-04-13 2008-01-22 Isis Pharmaceuticals, Inc. System of components for preparing oligonucleotides
US20040186071A1 (en) * 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
EP1080226A4 (en) 1998-05-21 2004-04-21 Isis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR TOPICAL ADMINISTRATION OF OLIGONUCLEOTIDES
WO1999060012A1 (en) * 1998-05-21 1999-11-25 Isis Pharmaceuticals, Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
US6335439B1 (en) 1998-06-11 2002-01-01 Isis Pharmaceuticals, Inc. Method of preparing phosphoramidites
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
US6225293B1 (en) 1998-09-02 2001-05-01 Isis Pharmaceuticals, Inc. Methods and compounds for tracking the biodistribution of macromolecule-carrier combinations
US6077709A (en) 1998-09-29 2000-06-20 Isis Pharmaceuticals Inc. Antisense modulation of Survivin expression
US6300320B1 (en) 1999-01-05 2001-10-09 Isis Pharmaceuticals, Inc. Modulation of c-jun using inhibitors of protein kinase C
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
US6252843B1 (en) 1999-04-09 2001-06-26 Hewlett Packard Company Automatic clamping of compact discs
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
US6147200A (en) * 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US7332275B2 (en) 1999-10-13 2008-02-19 Sequenom, Inc. Methods for detecting methylated nucleotides
WO2001051490A1 (en) * 2000-01-14 2001-07-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Methanocarba cycloalkyl nucleoside analogues
US20020055479A1 (en) 2000-01-18 2002-05-09 Cowsert Lex M. Antisense modulation of PTP1B expression
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US20030176385A1 (en) * 2000-02-15 2003-09-18 Jingfang Ju Antisense modulation of protein expression
AU2001253408C1 (en) * 2000-04-13 2008-12-04 Mervyn Merrilees Therapeutic compounds and methods for formulating V3, A Versican Isoform
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
US20060166227A1 (en) * 2000-06-20 2006-07-27 Stephen Kingsmore Protein expression profiling
US6323009B1 (en) * 2000-06-28 2001-11-27 Molecular Staging, Inc. Multiply-primed amplification of nucleic acid sequences
US6958214B2 (en) 2000-07-10 2005-10-25 Sequenom, Inc. Polymorphic kinase anchor proteins and nucleic acids encoding the same
US8568766B2 (en) 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
EP1326892A2 (en) 2000-10-12 2003-07-16 University of Rochester Compositions that inhibit proliferation of cancer cells
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US6573051B2 (en) * 2001-03-09 2003-06-03 Molecular Staging, Inc. Open circle probes with intramolecular stem structures
DE60232785D1 (de) 2001-03-14 2009-08-13 Myriad Genetics Inc Tsg101-gag-wechselwirkung und ihre verwendung
KR100628425B1 (ko) 2001-06-20 2006-09-28 제넨테크, 인크. 종양의 진단 및 치료를 위한 방법 및 이를 위한 조성물
US20050107595A1 (en) * 2001-06-20 2005-05-19 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
US7803915B2 (en) 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
CA2790034A1 (en) 2001-06-21 2003-01-03 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. Modulation of C-reactive protein expression
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
US20030096772A1 (en) 2001-07-30 2003-05-22 Crooke Rosanne M. Antisense modulation of acyl CoA cholesterol acyltransferase-2 expression
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
ES2431929T3 (es) 2001-09-18 2013-11-28 Genentech, Inc. Composiciones y procedimientos para el diagnóstico y el tratamiento de tumores
NZ577565A (en) 2001-10-09 2010-10-29 Isis Pharmaceuticals Inc Antisense modulation of insulin-like growth factor binding protein 5 expressions
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
US20030224380A1 (en) * 2001-10-25 2003-12-04 The General Hospital Corporation Genes and polymorphisms on chromosome 10 associated with Alzheimer's disease and other neurodegenerative diseases
US20030170678A1 (en) * 2001-10-25 2003-09-11 Neurogenetics, Inc. Genetic markers for Alzheimer's disease and methods using the same
WO2003054143A2 (en) * 2001-10-25 2003-07-03 Neurogenetics, Inc. Genes and polymorphisms on chromosome 10 associated with alzheimer's disease and other neurodegenerative diseases
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
EP2067472A1 (en) 2002-01-02 2009-06-10 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
US7553619B2 (en) * 2002-02-08 2009-06-30 Qiagen Gmbh Detection method using dissociated rolling circle amplification
US7169916B2 (en) * 2002-04-01 2007-01-30 Isis Pharmaceuticals, Inc. Chloral-free DCA in oligonucleotide synthesis
NZ535925A (en) 2002-04-16 2008-06-30 Genentech Inc An isolated antibody that binds to a particular polypeptide
EP1501863A4 (en) 2002-05-03 2007-01-24 Sequenom Inc KINASE ANCHOR PROTEIN, PEPTIDES AND RELATED METHODS THEREOF
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
US20040092470A1 (en) * 2002-06-18 2004-05-13 Leonard Sherry A. Dry powder oligonucleotide formualtion, preparation and its uses
EP1575992A4 (en) 2002-08-05 2007-02-21 Univ Rochester CHEMICAL PROTEINS FROM PROTEIN TRANSDUCATING DOMAIN / DEAMINASE, RELATED COMPOUNDS AND THEIR USES
CA2498777C (en) 2002-09-13 2015-01-13 Replicor, Inc. Non-sequence complementary antiviral oligonucleotides
WO2004027035A2 (en) 2002-09-20 2004-04-01 Yale University Riboswitches, methods for their use, and compositions for use with riboswitches.
AU2003278957A1 (en) 2002-09-26 2004-04-23 Amgen, Inc. Modulation of forkhead box o1a expression
JP2006515318A (ja) * 2002-10-29 2006-05-25 ファルマシア・コーポレーション 特異的に発現する癌関連遺伝子、それによってコードされるポリペプチドおよびその使用方法
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP2957568B1 (en) * 2002-11-05 2016-12-21 Ionis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
US9150606B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
WO2004044138A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
EP2336318B1 (en) 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
SI2336318T1 (sl) 2002-11-13 2013-07-31 Genzyme Corporation Protismiselna modulacija ekspresije apolipoproteina B
AU2003298650B2 (en) 2002-11-15 2010-03-11 Musc Foundation For Research Development Complement receptor 2 targeted complement modulators
CA2506127C (en) 2002-11-15 2013-07-09 Morphotek, Inc. Methods of generating high-production of antibodies from hybridomas created by in vitro immunization
EP1624753B1 (en) 2002-11-21 2012-01-25 The University of Utah Research Foundation Purinergic modulation of smell
US7144999B2 (en) 2002-11-23 2006-12-05 Isis Pharmaceuticals, Inc. Modulation of hypoxia-inducible factor 1 alpha expression
US20040115643A1 (en) * 2002-12-12 2004-06-17 Lizardi Paul M. Thermodynamic equilibrium extension of primers
US20040121338A1 (en) * 2002-12-19 2004-06-24 Alsmadi Osama A. Real-time detection of rolling circle amplification products
JP4886298B2 (ja) 2002-12-20 2012-02-29 キアゲン ゲゼルシャフト ミット ベシュレンクテル ハフツング 核酸増幅
US9487823B2 (en) * 2002-12-20 2016-11-08 Qiagen Gmbh Nucleic acid amplification
US6977153B2 (en) 2002-12-31 2005-12-20 Qiagen Gmbh Rolling circle amplification of RNA
CA2515484C (en) 2003-02-11 2011-09-20 Antisense Therapeutics Ltd Modulation of insulin like growth factor i receptor expression
US7002006B2 (en) 2003-02-12 2006-02-21 Isis Pharmaceuticals, Inc. Protection of nucleosides
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
US20070141570A1 (en) * 2003-03-07 2007-06-21 Sequenom, Inc. Association of polymorphic kinase anchor proteins with cardiac phenotypes and related methods
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
US8043834B2 (en) 2003-03-31 2011-10-25 Qiagen Gmbh Universal reagents for rolling circle amplification and methods of use
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US7399853B2 (en) 2003-04-28 2008-07-15 Isis Pharmaceuticals Modulation of glucagon receptor expression
EP1660512A4 (en) 2003-06-02 2009-12-23 Isis Pharmaceuticals Inc SYNTHESIS OF OLIGONUCLEOTIDES USING ALTERNATIVE SOLVENTS
CA2524495A1 (en) 2003-06-03 2005-01-13 Eli Lilly And Company Modulation of survivin expression
EP3604537B1 (en) 2003-06-13 2021-12-08 Alnylam Europe AG Double-stranded ribonucleic acid with increased effectiveness in an organism
EP1644475A4 (en) * 2003-06-20 2009-06-03 Isis Pharmaceuticals Inc DOUBLE-STRAND COMPOSITIONS WITH A 3'-ENDO-MODIFIED STRING FOR USE IN GENE MODULATION
US20060241072A1 (en) * 2003-06-20 2006-10-26 Isis Pharmaceuticals, Inc. Oligomeric compounds for use in gene modulation
CA2533701A1 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
US20050053981A1 (en) * 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20070123480A1 (en) * 2003-09-11 2007-05-31 Replicor Inc. Oligonucleotides targeting prion diseases
PE20050469A1 (es) 2003-09-18 2005-09-10 Lilly Co Eli Modulacion de la expresion de eif4e
JP5379347B2 (ja) * 2003-09-18 2013-12-25 アイシス ファーマシューティカルズ, インコーポレーテッド 4’−チオヌクレオシドおよびオリゴマー化合物
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
SI2161283T1 (sl) 2003-11-17 2014-10-30 Genentech, Inc. Sestavki, ki obsegajo protitelesa proti CD79b, konjugirana na sredstvo, ki inhibira rast, ali na citotoksično sredstvo, in postopki za zdravljenje tumorja hematopoetskega izvora
US20050164271A1 (en) 2004-01-20 2005-07-28 Sanjay Bhanot Modulation of glucocorticoid receptor expression
US7468431B2 (en) 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
US8778900B2 (en) 2004-01-22 2014-07-15 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP1 expression
US8569474B2 (en) * 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
US8790919B2 (en) 2004-03-15 2014-07-29 Isis Pharmaceuticals, Inc. Compositions and methods for optimizing cleavage of RNA by RNase H
WO2005097817A2 (en) 2004-04-05 2005-10-20 Alnylam Pharmaceuticals, Inc. Process and reagents for oligonucleotide synthesis and purification
US20050244869A1 (en) * 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
US20050260755A1 (en) * 2004-04-06 2005-11-24 Isis Pharmaceuticals, Inc. Sequential delivery of oligomeric compounds
AU2005323437B2 (en) 2004-04-30 2011-10-06 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a C5-modified pyrimidine
NZ550772A (en) 2004-05-21 2009-10-30 Uab Research Foundation Variable lymphocyte receptors, related polypeptides and nucleic acids, and uses thereof
EP1766052A4 (en) * 2004-06-03 2009-12-16 Isis Pharmaceuticals Inc OLIGOMERIC CHIMERIC COMPOSITIONS WITH BRECH
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
JP2008501694A (ja) * 2004-06-03 2008-01-24 アイシス ファーマシューティカルズ、インク. 遺伝子調節の使用のために個別に修飾された鎖を有する二本鎖組成物
US7427675B2 (en) * 2004-08-23 2008-09-23 Isis Pharmaceuticals, Inc. Compounds and methods for the characterization of oligonucleotides
US7884086B2 (en) * 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
US7759479B1 (en) * 2004-09-13 2010-07-20 Isis Pharmaceuticals, Inc. Compositions and their uses directed to Gemin Genes
WO2006032143A1 (en) 2004-09-23 2006-03-30 Arc Pharmaceuticals, Inc. Pharmaceutical compositions and methods relating to inhibiting fibrous adhesions or inflammatory disease using low sulphate fucans
JP2008516991A (ja) 2004-10-20 2008-05-22 アンチセンス セラピューティクス リミテッド インテグリンα4発現のアンチセンス調節
WO2006063031A2 (en) 2004-12-06 2006-06-15 Haplomics Allelic variants of human factor viii
WO2006091905A1 (en) * 2005-02-25 2006-08-31 Gilead Sciences, Inc. Bicyclo (3.1.0) hexane derivatives as antiviral compounds
RU2007137489A (ru) 2005-03-10 2009-04-20 Дженентек, Инк. (Us) Способы и композиции для модуляции целостности сосудов
US7476733B2 (en) 2005-03-25 2009-01-13 The United States Of America As Represented By The Department Of Health And Human Services Development of a real-time PCR assay for detection of pneumococcal DNA and diagnosis of pneumococccal disease
US8309303B2 (en) 2005-04-01 2012-11-13 Qiagen Gmbh Reverse transcription and amplification of RNA with simultaneous degradation of DNA
WO2007008300A2 (en) 2005-05-31 2007-01-18 ECOLE POLYTECHNIQUE FéDéRALE DE LAUSANNE Triblock copolymers for cytoplasmic delivery of gene-based drugs
WO2006133022A2 (en) 2005-06-03 2006-12-14 The Johns Hopkins University Compositions and methods for decreasing microrna expression for the treatment of neoplasia
WO2006138145A1 (en) 2005-06-14 2006-12-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
MY144014A (en) 2005-08-11 2011-07-29 Synthetic Genomics Inc Method for in vitro recombination
WO2007020081A1 (en) 2005-08-17 2007-02-22 Medexis S.A. Composition and method for determination of ck19 expression
AU2006284855B2 (en) 2005-08-29 2011-10-13 Regulus Therapeutics Inc. Methods for use in modulating miR-122a
EP1931780B1 (en) 2005-08-29 2016-01-06 Regulus Therapeutics Inc. Antisense compounds having enhanced anti-microrna activity
EP1762627A1 (de) 2005-09-09 2007-03-14 Qiagen GmbH Verfahren zur Aktivierung einer Nukleinsäure für eine Polymerase-Reaktion
EP2395076A1 (en) 2005-10-14 2011-12-14 MUSC Foundation For Research Development Targeting PAX2 for the induction of DEFB1-mediated tumor immunity and cancer therapy
US8080534B2 (en) * 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
EP1941059A4 (en) 2005-10-28 2010-11-03 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF THE HUNTINGTIN GENE
CA2626584A1 (en) * 2005-11-04 2007-05-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of nav1.8 gene
ATE510545T1 (de) 2005-11-09 2011-06-15 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur hemmung der expression von faktor v leiden mutationsgen
EP1966377A2 (en) 2005-11-21 2008-09-10 Isis Pharmaceuticals, Inc. Modulation of eif4e-bp2 expression
EP1974052A2 (en) * 2005-12-21 2008-10-01 Yale University Methods and compositions related to the modulation of riboswitches
EP1976567B1 (en) 2005-12-28 2020-05-13 The Scripps Research Institute Natural antisense and non-coding rna transcripts as drug targets
AU2007210038B2 (en) 2006-01-26 2013-01-10 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
DK2314594T3 (da) * 2006-01-27 2014-10-27 Isis Pharmaceuticals Inc 6-modificerede bicykliske nukleinsyreanaloger
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
WO2007090073A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
WO2007115168A2 (en) 2006-03-31 2007-10-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of eg5 gene
EP2013344B1 (en) 2006-05-03 2012-08-29 Baltic Technology Development, Ltd. Antisense agents combining strongly bound base - modified oligonucleotide and artificial nuclease
DE102006020885A1 (de) * 2006-05-05 2007-11-08 Qiagen Gmbh Einführung von Sequenzelementen in Nukleinsäuren
CA2651042A1 (en) 2006-05-05 2007-12-13 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
EP2505647A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of DGAT2
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
ATE528008T1 (de) 2006-05-19 2011-10-15 Alnylam Pharmaceuticals Inc Rnai-modulation von aha und ihre therapeutische verwendung
EP2584051B1 (en) 2006-05-22 2014-07-16 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expressions of IKK-B gene
WO2007137301A2 (en) * 2006-05-23 2007-11-29 Isis Pharmaceuticals, Inc. Modulation of chrebp expression
US8198253B2 (en) 2006-07-19 2012-06-12 Isis Pharmaceuticals, Inc. Compositions and their uses directed to HBXIP
EP2057284A4 (en) * 2006-08-04 2011-06-29 Isis Pharmaceuticals Inc COMPOSITIONS AND METHODS OF MODULATING JNK PROTEINS
WO2008033866A2 (en) * 2006-09-11 2008-03-20 Yale University Methods and compositions for the use of lysine riboswitches
EP2069788B1 (en) 2006-10-05 2017-03-01 Massachusetts Institute of Technology Multifunctional encoded particles for high-throughput analysis
EP3916095A1 (en) 2006-10-18 2021-12-01 Ionis Pharmaceuticals, Inc. Antisense compounds
US8999317B2 (en) 2006-11-01 2015-04-07 University Of Rochester Methods and compositions related to the structure and function of APOBEC3G
EP2453016A1 (en) * 2006-11-27 2012-05-16 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
US7994130B2 (en) * 2006-12-11 2011-08-09 University Of Utah Research Foundation Compositions and methods for treating ocular pathologic angiogenesis and vascular permeability
EP2097448A4 (en) 2006-12-22 2010-07-21 Univ Utah Res Found PROCEDURE FOR THE EVIDENCE OF EYE DISEASES AND SUFFERINGS AND THEIR TREATMENT
KR101488800B1 (ko) 2007-02-09 2015-02-04 노오쓰웨스턴 유니버시티 세포내 타겟 검출용 입자
JP2010520749A (ja) * 2007-02-27 2010-06-17 ノースウェスタン ユニバーシティ ナノ粒子への分子の結合
EP2139319A4 (en) 2007-03-22 2011-02-23 Univ Yale METHOD AND COMPOSITIONS FOR RIBOSWITCHES CONTROLLING THE ALTERNATIVE SPLICE
CA2682161A1 (en) 2007-03-29 2008-10-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a gene from ebola virus
US20110091377A1 (en) 2007-05-11 2011-04-21 The Johns Hopkins University Biomarkers for melanoma
MX2009012773A (es) 2007-05-29 2009-12-16 Univ Yale Ribointerruptores y metodos y composiciones para usarse de y con ribointerruptores.
MX2009012647A (es) * 2007-05-29 2009-12-14 Univ Yale Metodos y composiciones relacionadas con ribointerruptores que controlan la separacion alternativa y proceso de arn.
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
MX2009013046A (es) 2007-05-30 2010-02-17 Univ Northwestern Nanoparticulas con grupo funcional de acido nucleico para aplicaciones terapeuticas.
US7807372B2 (en) * 2007-06-04 2010-10-05 Northwestern University Screening sequence selectivity of oligonucleotide-binding molecules using nanoparticle based colorimetric assay
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
ATE538127T1 (de) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-disubstituierte bicyclische nukleinsäureanaloga
KR101654007B1 (ko) 2007-08-15 2016-09-05 아이오니스 파마수티컬즈, 인코포레이티드 테트라하이드로피란 핵산 유사체
JP2010537638A (ja) 2007-08-28 2010-12-09 ユーエービー リサーチ ファウンデーション 合成アポリポ蛋白質e模倣ポリペプチドおよび使用方法
EP2682400B1 (en) 2007-08-28 2017-09-20 Uab Research Foundation Synthetic apolipoprotein E mimicking polypeptides and methods of use
WO2009039466A1 (en) 2007-09-20 2009-03-26 Vanderbilt University Free solution measurement of molecular interactions by backscattering interferometry
WO2009039442A1 (en) 2007-09-21 2009-03-26 California Institute Of Technology Nfia in glial fate determination, glioma therapy and astrocytoma treatment
EP2217705A2 (en) * 2007-11-05 2010-08-18 Baltic Technology Development, Ltd. Use of oligonucleotides with modified bases in hybridization of nucleic acids
WO2009067243A2 (en) 2007-11-20 2009-05-28 Isis Pharmaceuticals Inc. Modulation of cd40 expression
EP2245159A2 (en) 2007-12-10 2010-11-03 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of factor vii gene
EP3100718B1 (en) 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
WO2009100320A2 (en) 2008-02-07 2009-08-13 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
CN104975020B (zh) 2008-02-11 2020-01-17 菲奥医药公司 经修饰的RNAi多核苷酸及其用途
CA2716793A1 (en) 2008-03-05 2009-09-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of eg5 and vegf genes
US8426378B2 (en) * 2008-03-21 2013-04-23 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucelosides and methods for their use
WO2009124295A2 (en) * 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
US9290534B2 (en) 2008-04-04 2016-03-22 Ionis Pharmaceuticals, Inc. Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside
ES2554168T3 (es) 2008-04-18 2015-12-16 Baxter International Inc. Composición basada en microesferas para prevenir y/o revertir la diabetes autoinmune de nueva aparición
CA2722668A1 (en) * 2008-04-29 2009-11-05 Wyeth Llc Methods for treating inflammation
WO2009149182A1 (en) 2008-06-04 2009-12-10 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small rna targeting of gene promoters
JP5438922B2 (ja) * 2008-06-25 2014-03-12 日東電工株式会社 核酸の製造方法
US20110119781A1 (en) * 2008-07-15 2011-05-19 Birgit Bramlage Compositions and Methods for Inhibiting Expression of TGF-BETA Receptor Genes
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
WO2010014592A1 (en) 2008-07-29 2010-02-04 The Board Of Regents Of The University Of Texas Sytem Selective inhibition of polyglutamine protein expression
WO2010017509A1 (en) * 2008-08-07 2010-02-11 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
CN107083386A (zh) 2008-08-25 2017-08-22 埃克斯雷德制药有限公司 阻止结缔组织生长因子的反义核苷酸及其用途
EP3375451A1 (en) 2008-08-25 2018-09-19 Excaliard Pharmaceuticals, Inc. Method for reducing scarring during wound healing using antisense compounds directed to ctgf
EP3208337A1 (en) 2008-09-02 2017-08-23 Alnylam Pharmaceuticals, Inc. Compositions for combined inhibition of mutant egfr and il-6 expression
EP3336188B1 (en) 2008-09-22 2020-05-06 Phio Pharmaceuticals Corp. Reduced size self-delivering rnai compounds
EP2361256B1 (en) 2008-09-24 2013-04-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
EP2356129B1 (en) 2008-09-24 2013-04-03 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
WO2010036962A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
EP3225621A1 (en) 2008-10-09 2017-10-04 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
CN109797150A (zh) 2008-10-15 2019-05-24 Ionis制药公司 因子11表达的调节
KR102354558B1 (ko) 2008-10-20 2022-01-25 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴의 발현을 억제하기 위한 조성물 및 방법
CA2741294C (en) 2008-10-24 2018-04-24 Isis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
WO2010048585A2 (en) 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
BRPI0921096A8 (pt) * 2008-11-17 2016-05-10 Hoffmann La Roche Composições e métodos para inibir a expressão dos genes de fator vii
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
EP3335705A1 (en) 2008-11-24 2018-06-20 Northwestern University Polyvalent rna-nanoparticle compositions
RU2620970C2 (ru) 2008-12-04 2017-05-30 КьюРНА,Инк., Лечение связанных с эритропоэтином (еро) заболеваний путем ингибирования природного антисмыслового транскрипта к еро
MX358603B (es) 2008-12-04 2018-08-28 Curna Inc Tratamiento de enfermedades relacionadas con un gen supresor de tumor mediante inhibicion del transcrito antisentido natural para el gen.
EP2370580B1 (en) 2008-12-04 2019-09-11 CuRNA, Inc. Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirtuin 1
WO2010068816A1 (en) 2008-12-10 2010-06-17 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
MX2011007350A (es) * 2009-01-08 2011-09-06 Univ Northwestern Inhibicion de produccion de proteina bacteriana por conjugados de nanoparticulas modificadas por oligonucleotidos polivalentes.
KR101546673B1 (ko) * 2009-01-15 2015-08-25 삼성전자주식회사 전자 사진용 토너 및 그의 제조방법
CA2751342C (en) 2009-01-29 2019-05-07 Alnylam Pharmaceuticals, Inc. Lipid formulations comprising cationic lipid and a targeting lipid comprising n-acetyl galactosamine for delivery of nucleic acid
KR20110100316A (ko) 2009-02-03 2011-09-09 에프. 호프만-라 로슈 아게 Ptp1b 유전자의 발현을 억제하기 위한 조성물 및 방법
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
WO2010090969A1 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
WO2010091308A2 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
WO2010093906A2 (en) 2009-02-12 2010-08-19 Curna, Inc. Treatment of glial cell derived neurotrophic factor (gdnf) related diseases by inhibition of natural antisense transcript to gdnf
EP3009150B1 (en) 2009-02-12 2019-11-13 CuRNA, Inc. Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
ES2845644T3 (es) 2009-03-04 2021-07-27 Curna Inc Tratamiento de enfermedades relacionadas con sirtuina1 (SIRT1) mediante inhibición del transcrito del antisentido natural a sirtuina 1
CA2754043A1 (en) 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
WO2010107733A2 (en) 2009-03-16 2010-09-23 Curna, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
ES2627763T3 (es) 2009-03-17 2017-07-31 Curna, Inc. Tratamiento de enfermedades relacionadas con el homólogo tipo delta 1 (dlk1) por inhibición de transcrito antisentido natural a dlk1
JP6145270B2 (ja) 2009-04-15 2017-06-07 ノースウェスタン ユニバーシティ オリゴヌクレオチド機能化ナノ粒子の送達
EP3524275A1 (en) 2009-04-22 2019-08-14 Massachusetts Institute Of Technology Innate immune supression enables repeated delivery of long rna molecules
WO2010124231A2 (en) 2009-04-24 2010-10-28 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
EP2424987B1 (en) 2009-05-01 2017-11-15 CuRNA, Inc. Treatment of hemoglobin (hbf/hbg) related diseases by inhibition of natural antisense transcript to hbf/hbg
AU2010245933B2 (en) 2009-05-05 2016-06-16 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
SG10201402054UA (en) 2009-05-05 2014-09-26 Muthiah Manoharan Lipid compositions
EP2427552B1 (en) 2009-05-06 2016-11-16 CuRNA, Inc. Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp
US9155754B2 (en) 2009-05-06 2015-10-13 Curna, Inc. Treatment of ABCA1 gene related diseases by inhibition of a natural antisense transcript to ABCA1
EP2430161A1 (en) 2009-05-15 2012-03-21 Yale University Gemm riboswitches, structure-based compound design with gemm riboswitches, and methods and compositions for use of and with gemm riboswitches
BRPI1012769A2 (pt) * 2009-05-15 2018-01-30 Hoffmann La Roche composições e métodos para inibir expressão de genes de receptor de glicocorticóide (gcr)
DK2432881T3 (en) 2009-05-18 2018-02-26 Curna Inc TREATMENT OF REPROGRAMMING FACTOR-RELATED DISEASES BY INHIBITING NATURAL ANTISENSE TRANSCRIPTS TO A REPROGRAMMING FACTOR
WO2010135695A2 (en) 2009-05-22 2010-11-25 Curna, Inc. TREATMENT OF TRANSCRIPTION FACTOR E3 (TFE3) and INSULIN RECEPTOR SUBSTRATE 2 (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO TFE3
ES2618576T3 (es) 2009-05-28 2017-06-21 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen antivírico mediante la inhibición de una transcripción antisentido natural a un gen antivírico
PT3431076T (pt) 2009-06-10 2021-10-26 Arbutus Biopharma Corp Formulação lipídica melhorada
KR101801404B1 (ko) 2009-06-16 2017-12-20 큐알엔에이, 인크. 콜라겐 유전자에 대한 천연 안티센스 전사체의 억제에 의한 콜라겐 유전자 관련된 질환의 치료
KR101702689B1 (ko) 2009-06-16 2017-02-06 큐알엔에이, 인크. Pon1에 대한 천연 안티센스 전사체의 억제에 의한 파라옥소나제 1(pon1) 관련된 질환의 치료
CN115227710A (zh) 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
JP6073133B2 (ja) 2009-06-24 2017-02-01 クルナ・インコーポレーテッド 腫瘍壊死因子受容体2(tnfr2)に対する天然アンチセンス転写物の抑制によるtnfr2関連疾患の治療
ES2583691T3 (es) 2009-06-26 2016-09-21 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen del síndrome de Down mediante la inhibición de una transcripción antisentido natural a un gen del síndrome de Down
KR101802536B1 (ko) 2009-08-05 2017-11-28 큐알엔에이, 인크. 인슐린 유전자 (ins)에 대한 자연 안티센스 전사체의 저해에 의한 인슐린 유전자 (ins) 관련된 질환의 치료
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
EP2810643A3 (en) 2009-08-14 2015-03-11 Alnylam Pharmaceuticals Inc. Lipid formulated compositions and mehods for inhibiting expression of a gene from the ebola virus
WO2011022420A1 (en) 2009-08-17 2011-02-24 Yale University Methylation biomarkers and methods of use
CA2771172C (en) 2009-08-25 2021-11-30 Opko Curna, Llc Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap
DK2473522T3 (en) 2009-09-02 2016-11-28 Genentech Inc Smoothened MUTANT AND METHODS OF USING THE SAME
ES2564207T3 (es) 2009-10-22 2016-03-18 F. Hoffmann-La Roche Ag Métodos y composiciones para modular la activación con hepsina de la proteína estimuladora de macrófagos
WO2011056687A2 (en) 2009-10-27 2011-05-12 Swift Biosciences, Inc. Polynucleotide primers and probes
CN102666879B (zh) 2009-10-30 2016-02-24 西北大学 模板化的纳米缀合物
WO2011053994A1 (en) 2009-11-02 2011-05-05 Alnylam Pharmaceuticals, Inc. Modulation of ldl receptor gene expression with double-stranded rnas targeting the ldl receptor gene promoter
WO2011056215A1 (en) 2009-11-03 2011-05-12 Landers James P Versatile, visible method for detecting polymeric analytes
WO2011054939A2 (en) 2009-11-09 2011-05-12 F. Hoffmann-La Roche Ag Compositions and methods for inhibiting expression of kif10 genes
WO2011063403A1 (en) 2009-11-23 2011-05-26 Swift Biosciences, Inc. Devices to extend single stranded target molecules
CN102741294A (zh) 2009-11-30 2012-10-17 霍夫曼-拉罗奇有限公司 治疗和诊断表达slc34a2(tat211=seqid2)的肿瘤的抗体
EP2507241A1 (en) 2009-12-02 2012-10-10 The U.S.A. As Represented By The Secretary, Department Of Health And Human Services Methanocarba adenosine derivatives and dendrimer conjugates thereof
NO2513310T3 (cg-RX-API-DMAC10.html) 2009-12-16 2018-03-31
US20110152349A1 (en) 2009-12-18 2011-06-23 Anke Geick Compositions and methods for inhibiting expression of il-18 genes
JP5934106B2 (ja) 2009-12-23 2016-06-15 カッパーアールエヌエー,インコーポレイテッド 肝細胞増殖因子(hgf)に対する天然アンチセンス転写物の阻害によるhgf関連性疾患の治療
EP2515947B1 (en) 2009-12-23 2021-10-06 CuRNA, Inc. Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
JP5993744B2 (ja) 2009-12-29 2016-09-14 カッパーアールエヌエー,インコーポレイテッド 核呼吸因子1(nrf1)に対する天然アンチセンス転写物の阻害による核呼吸因子1関連疾患の治療
EP2519634B1 (en) 2009-12-29 2016-06-01 CuRNA, Inc. TREATMENT OF TUMOR PROTEIN 63 (p63) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO p63
NO2521784T3 (cg-RX-API-DMAC10.html) 2010-01-04 2018-05-05
CA2786056C (en) 2010-01-06 2023-03-14 Curna, Inc. Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene
EP2524039B1 (en) 2010-01-11 2017-11-29 CuRNA, Inc. Treatment of sex hormone binding globulin (shbg) related diseases by inhibition of natural antisense transcript to shbg
US8779118B2 (en) 2010-01-11 2014-07-15 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
US20120321647A1 (en) 2010-01-12 2012-12-20 Yale University Structured rna motifs and compounds and methods for their use
US20110177107A1 (en) * 2010-01-14 2011-07-21 Haplomics, Inc. Predicting and reducing alloimmunogenicity of protein therapeutics
WO2011091390A2 (en) 2010-01-25 2011-07-28 Opko Curna, Llc Treatment of rnase h1 related diseases by inhibition of natural antisense transcript to rnase h1
WO2011097388A1 (en) 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
US20130028889A1 (en) 2010-02-04 2013-01-31 Ico Therapeutics Inc. Dosing regimens for treating and preventing ocular disorders using c-raf antisense
TW201129365A (en) 2010-02-05 2011-09-01 Hoffmann La Roche Compositions and methods for inhibiting expression of IKK2 genes
WO2011103528A2 (en) 2010-02-22 2011-08-25 Opko Curna Llc Treatment of pyrroline-5-carboxylate reductase 1 (pycr1) related diseases by inhibition of natural antisense transcript to pycr1
EP2538981B1 (en) 2010-02-23 2017-12-20 F. Hoffmann-La Roche AG Compositions and methods for the diagnosis and treatment of tumor
WO2011105900A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-alpha (c8-alpha) and uses thereof
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011105902A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-beta (c8-beta) and uses thereof
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
US20130101512A1 (en) 2010-03-12 2013-04-25 Chad A. Mirkin Crosslinked polynucleotide structure
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2550000A4 (en) 2010-03-24 2014-03-26 Advirna Inc RNAI COMPOUNDS OF REDUCED SIZE ADMINISTERING
US9095504B2 (en) 2010-03-24 2015-08-04 Rxi Pharmaceuticals Corporation RNA interference in ocular indications
KR101852210B1 (ko) 2010-03-24 2018-04-25 알엑스아이 파마슈티칼스 코포레이션 진피 및 섬유증성 적응증에서의 rna 간섭
US20130281510A1 (en) 2010-03-29 2013-10-24 Kumamoto University siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis
AU2011237630B2 (en) 2010-04-06 2016-01-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of CD274/PD-L1 gene
RU2610661C2 (ru) 2010-04-09 2017-02-14 Курна, Инк. Лечение заболеваний, связанных с фактором роста фибробластов 21 (fgf21), путем ингибирования природного антисмыслового транскрипта к fgf21
WO2011133695A2 (en) 2010-04-20 2011-10-27 Swift Biosciences, Inc. Materials and methods for nucleic acid fractionation by solid phase entrapment and enzyme-mediated detachment
EP2625186B1 (en) 2010-04-28 2016-07-27 Ionis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
JP6005628B2 (ja) 2010-04-28 2016-10-12 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. 修飾ヌクレオシド、その類似体、およびこれらから調製されるオリゴマー化合物
SI2563920T1 (sl) 2010-04-29 2017-05-31 Ionis Pharmaceuticals, Inc. Modulacija izražanja transtiretina
JP2013533732A (ja) 2010-05-03 2013-08-29 ジェネンテック, インコーポレイテッド 腫瘍の診断と治療のための組成物と方法
KR101915115B1 (ko) 2010-05-03 2018-11-05 큐알엔에이, 인크. 시르투인 (sirt)에 대한 자연 안티센스 전사체의 저해에 의한 시르투인 (sirt) 관련된 질환의 치료
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制par4天然反股轉錄本治療par4相關疾病
DK2576783T3 (en) 2010-05-26 2018-03-12 Curna Inc TREATMENT OF ATONAL HOMOLOGY 1- (ATOH1) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPTS AT ATOH1
WO2011150226A1 (en) 2010-05-26 2011-12-01 Landers James P Method for detecting nucleic acids based on aggregate formation
AU2011261434B2 (en) 2010-06-02 2015-11-26 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
KR101932628B1 (ko) 2010-06-07 2018-12-27 파이어플라이 바이오웍스, 인코포레이티드 후­혼성 표지화 및 범용 코드화에 의한 핵산 검출 및 정량화
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011156713A1 (en) 2010-06-11 2011-12-15 Vanderbilt University Multiplexed interferometric detection system and method
US9168297B2 (en) 2010-06-23 2015-10-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Regulation of skin pigmentation by neuregulin-1 (NRG-1)
CN103068982B (zh) 2010-07-14 2017-06-09 库尔纳公司 通过抑制盘状大同系物(dlg)的天然反义转录物而治疗dlg相关疾病
EP3633038A3 (en) 2010-07-19 2020-07-29 Ionis Pharmaceuticals, Inc. Modulation of dystrophia myotonica-protein kinase (dmpk) expression
US20130143955A1 (en) 2010-08-09 2013-06-06 Yale University Cyclic di-GMP-II Riboswitches, Motifs, and Compounds, and Methods for Their Use
ES2588981T3 (es) 2010-10-05 2016-11-08 Genentech, Inc. Smoothened mutante y métodos de uso de la misma
JP5986998B2 (ja) 2010-10-06 2016-09-06 カッパーアールエヌエー,インコーポレイテッド シアリダーゼ4(neu4)への天然アンチセンス転写物の阻害によるneu4関連疾患の治療
WO2012052258A1 (en) 2010-10-18 2012-04-26 Arrowhead Research Corporation Compositions and methods for inhibiting expression of rrm2 genes
WO2012054723A2 (en) 2010-10-22 2012-04-26 Opko Curna Llc Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
KR101913232B1 (ko) 2010-10-27 2018-10-30 큐알엔에이, 인크. 인터페론-관련된 발달성 조절물질 1 (ifrd1)에 대한 자연 안티센스 전사체의 저해에 의한 ifrd1 관련된 질환의 치료
WO2012064824A1 (en) 2010-11-09 2012-05-18 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
WO2012068405A2 (en) 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
ES2657590T3 (es) 2010-11-23 2018-03-06 Curna, Inc. Tratamiento de enfermedades relacionadas con nanog mediante inhibición del transcrito antisentido natural a nanog
US9150926B2 (en) 2010-12-06 2015-10-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Diagnosis and treatment of adrenocortical tumors using human microRNA-483
EP2648763A4 (en) 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR EXPRESSION INHIBITION OF GENES KLF-1 AND BCL11A
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
HUE044178T4 (hu) 2011-02-02 2019-10-28 Excaliard Pharmaceuticals Inc Kötõszöveti növekedési faktort (CTGF) célzó antiszensz vegyületek keloidok vagy hipertrófiás hegek kezelési eljárásban történõ alkalmazásra
US20130338178A1 (en) 2011-02-02 2013-12-19 The Trustees Of Princeton University Sirtuin modulators as inhibitors of cytomegalovirus
WO2012106591A1 (en) 2011-02-03 2012-08-09 Mirna Therapeutics, Inc. Synthetic mimics of mir-34
MX2013008944A (es) 2011-02-03 2014-01-08 Mirna Therapeutics Inc Mimeticos sinteticos de mir-124.
US10017764B2 (en) 2011-02-08 2018-07-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US9562853B2 (en) 2011-02-22 2017-02-07 Vanderbilt University Nonaqueous backscattering interferometric methods
CN103813810B (zh) 2011-03-29 2021-08-03 阿尔尼拉姆医药品有限公司 用于抑制tmprss6基因表达的组合物和方法
EP3460064B8 (en) 2011-04-03 2024-03-20 The General Hospital Corporation d/b/a Massachusetts General Hospital Efficient protein expression in vivo using modified rna (mod-rna)
US20140186844A1 (en) 2011-04-26 2014-07-03 Swift Biosciences, Inc. Polynucleotide primers and probes
WO2012151289A2 (en) 2011-05-02 2012-11-08 University Of Virginia Patent Foundation Method and system to detect aggregate formation on a substrate
WO2012151268A1 (en) 2011-05-02 2012-11-08 University Of Virginia Patent Foundation Method and system for high throughput optical and label free detection of analytes
EP2718439B1 (en) 2011-06-09 2017-08-09 CuRNA, Inc. Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn
WO2012170347A1 (en) 2011-06-09 2012-12-13 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2717923B1 (en) 2011-06-10 2017-09-27 Ionis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
WO2012177949A2 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
CN103890000B (zh) 2011-06-21 2017-09-01 阿尔尼拉姆医药品有限公司 血管生成素样3(ANGPTL3)iRNA组合物及其使用方法
EP3693464A3 (en) 2011-06-21 2020-12-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
EP3597750B1 (en) 2011-06-23 2022-05-04 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
US9322021B2 (en) 2011-06-29 2016-04-26 Ionis Pharmaceuticals, Inc. Methods for modulating kallikrein (KLKB1) expression
TW202424196A (zh) 2011-06-30 2024-06-16 美商艾羅海德製藥公司 用於抑制b型肝炎病毒基因表現之組合物及方法
JP2014526887A (ja) 2011-08-01 2014-10-09 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 造血幹細胞移植の成功率を改善する方法
EP3922722B1 (en) 2011-08-11 2023-06-28 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US9889209B2 (en) 2011-09-14 2018-02-13 Northwestern University Nanoconjugates able to cross the blood-brain barrier
WO2013040548A2 (en) 2011-09-17 2013-03-21 Yale University Fluoride-responsive riboswitchs, fluoride transporters, and methods of use
BR112014008862A2 (pt) 2011-10-14 2018-08-07 Genentech Inc anticorpo isolado que se liga à htra1, ácido nucleico isolado, célula hospedeira, imunoconjugado, formulação farmacêutica, métodos e usos
WO2013059740A1 (en) 2011-10-21 2013-04-25 Foundation Medicine, Inc. Novel alk and ntrk1 fusion molecules and uses thereof
HRP20220717T1 (hr) 2011-12-16 2022-07-22 Modernatx, Inc. Modificirani pripravci mrna
EP2794880B1 (en) 2011-12-22 2018-05-02 Ionis Pharmaceuticals, Inc. Methods for modulating metastasis-associated-in-lung-adenocarcinoma-transcript-1(malat-1) expression
CA2860731C (en) 2012-01-10 2023-02-28 M. Mahmood Hussain Method of treating hyperlipidemia and atherosclerosis with mir-30c
US20140378533A1 (en) 2012-02-08 2014-12-25 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
CN104395480B (zh) 2012-03-13 2018-01-30 斯威夫特生物科学公司 用于通过核酸聚合酶对衬底多核苷酸进行大小受控的同聚物加尾的方法和组合物
CN110438125A (zh) 2012-03-15 2019-11-12 科纳公司 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
CA2907072A1 (en) 2012-03-16 2013-09-19 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of dmpk
WO2013142514A1 (en) 2012-03-19 2013-09-26 Isis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
JP2015516143A (ja) 2012-04-02 2015-06-08 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. ヒト疾患に関連するタンパク質の産生のための修飾ポリヌクレオチド
WO2013151666A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of biologics and proteins associated with human disease
WO2013154798A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP2839006B1 (en) 2012-04-20 2018-01-03 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
US9273949B2 (en) 2012-05-11 2016-03-01 Vanderbilt University Backscattering interferometric methods
US9518261B2 (en) 2012-05-22 2016-12-13 Ionis Pharmaceuticals, Inc. Modulation of enhancer RNA mediated gene expression
PT3461895T (pt) 2012-06-25 2020-09-08 Baylor College Medicine Modulação da expressão de ube3a-ats
US20140038182A1 (en) 2012-07-17 2014-02-06 Dna Logix, Inc. Cooperative primers, probes, and applications thereof
WO2014018930A1 (en) 2012-07-27 2014-01-30 Isis Pharmaceuticals. Inc. Modulation of renin-angiotensin system (ras) related diseases by angiotensinogen
WO2014028739A1 (en) 2012-08-15 2014-02-20 Isis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified capping protocols
WO2014045126A2 (en) 2012-09-18 2014-03-27 Uti Limited Partnership Treatment of pain by inhibition of usp5 de-ubiquitinase
AR092982A1 (es) 2012-10-11 2015-05-13 Isis Pharmaceuticals Inc Modulacion de la expresion de receptores androgenicos
WO2014059353A2 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
WO2014059364A1 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Methods of treating kennedy's disease
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
CA2887702C (en) 2012-10-26 2023-08-01 Geron Corporation C-myc antisense oligonucleotides and methods for using the same to treat cell-proliferative disorders
HK1214830A1 (zh) 2012-11-05 2016-08-05 Foundation Medicine, Inc. 新型ntrk1融合分子及其应用
CA2890346A1 (en) 2012-11-05 2014-05-08 Foundation Medicine, Inc. Novel fusion molecules and uses thereof
SMT202200337T1 (it) 2012-11-26 2022-09-14 Modernatx Inc Rna modificato al livello del terminale
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
AU2013374345A1 (en) 2013-01-17 2015-08-06 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
EP3939614A1 (en) 2013-01-18 2022-01-19 Foundation Medicine, Inc. Methods of treating cholangiocarcinoma
DK2951191T3 (en) 2013-01-31 2019-01-14 Ionis Pharmaceuticals Inc PROCEDURE FOR MANUFACTURING OLIGOMERIC COMPOUNDS USING MODIFIED CLUTCH PROTOCOLS
CN108743943A (zh) 2013-02-14 2018-11-06 Ionis制药公司 对脂蛋白脂肪酶缺乏群体中的载脂蛋白c-iii(apociii)表达的调节
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
EP2961853B1 (en) 2013-02-28 2018-09-19 The Board of Regents of The University of Texas System Methods for classifying a cancer as susceptible to tmepai-directed therapies and treating such cancers
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
PT2970974T (pt) 2013-03-14 2017-11-29 Alnylam Pharmaceuticals Inc Composições de irna de componente do complemento c5 e métodos para a sua utilização
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
JP2016522679A (ja) 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ CRISPR/Cas系を用いたゲノム編集の治療的使用
CN104098553B (zh) * 2013-04-10 2017-11-28 江苏恒瑞医药股份有限公司 替格瑞洛的中间体及其制备方法和替格瑞洛的制备方法
WO2014172698A1 (en) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
SG10201906382QA (en) 2013-05-01 2019-08-27 Ionis Pharmaceuticals Inc Compositions and methods for modulating hbv and ttr expression
EP2999785B1 (en) 2013-05-22 2018-04-04 Alnylam Pharmaceuticals, Inc. Serpina1 irna compositions and methods of use thereof
KR102365809B1 (ko) 2013-05-22 2022-02-23 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 조성물 및 이의 사용 방법
EP3004396B1 (en) 2013-06-06 2019-10-16 The General Hospital Corporation Compositions for the treatment of cancer
DK3007704T3 (da) 2013-06-13 2021-03-29 Antisense Therapeutics Ltd Kombinationsterapi til akromegali
EP3011026B1 (en) 2013-06-21 2019-12-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile
ES2787600T3 (es) 2013-07-02 2020-10-16 Ionis Pharmaceuticals Inc Moduladores del receptor de la hormona del crecimiento
EP3971287A1 (en) 2013-07-11 2022-03-23 ModernaTX, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
HRP20200250T1 (hr) 2013-08-08 2020-05-29 The Scripps Research Institute Metoda site-specifično enzimsko obilježavanje nukleinskih kiselina in vitro ugradnjom neprirodnih nukleotida
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
EP3715457A3 (en) 2013-08-28 2020-12-16 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (pkk) expression
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
US20160194625A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Chimeric polynucleotides
PE20160500A1 (es) 2013-09-13 2016-05-21 Ionis Pharmaceuticals Inc Moduladores del factor del complemento b
CN109536493A (zh) 2013-10-02 2019-03-29 阿尔尼拉姆医药品有限公司 用于抑制lect2基因表达的组合物和方法
WO2015051214A1 (en) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
US10227588B2 (en) 2013-10-04 2019-03-12 Novartis Ag 3′end caps for RNAi agents for use in RNA interference
NZ718995A (en) 2013-10-04 2022-07-01 Icahn School Med Mount Sinai Compositions and methods for inhibiting expression of the alas1 gene
EP3052107B1 (en) 2013-10-04 2018-05-02 Novartis AG Organic compounds to treat hepatitis b virus
EP3052627B1 (en) 2013-10-04 2018-08-22 Novartis AG Novel formats for organic compounds for use in rna interference
EP3055426B1 (en) 2013-10-09 2019-06-19 The United States of America as represented by The Secretary Department of Health and Human Services Detection of hepatitis delta virus (hdv) for the diagnosis and treatment of sjögren's syndrome and lymphoma
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
EP3060680B1 (en) 2013-10-21 2019-02-27 The General Hospital Corporation Methods relating to circulating tumor cell clusters and the treatment of cancer
WO2015061246A1 (en) 2013-10-21 2015-04-30 Isis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
US10301622B2 (en) 2013-11-04 2019-05-28 Northwestern University Quantification and spatio-temporal tracking of a target using a spherical nucleic acid (SNA)
CN113151180A (zh) 2013-12-02 2021-07-23 菲奥医药公司 癌症的免疫治疗
JP6527516B2 (ja) 2013-12-03 2019-06-05 ノースウェスタン ユニバーシティ リポソーム粒子、前述のものを作製する方法及びその使用
CA2844640A1 (en) 2013-12-06 2015-06-06 The University Of British Columbia Method for treatment of castration-resistant prostate cancer
WO2015085183A2 (en) 2013-12-06 2015-06-11 Swift Biosciences, Inc. Cleavable competitor polynucleotides
EA201691215A1 (ru) 2013-12-12 2016-11-30 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ iRNA К КОМПОНЕНТУ КОМПЛЕМЕНТА И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
AU2014364520B2 (en) 2013-12-20 2020-01-02 The General Hospital Corporation Methods and assays relating to circulating tumor cells
CA2931510A1 (en) 2013-12-24 2015-07-02 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
DK3102197T3 (en) 2014-02-04 2018-11-19 Genentech Inc Smoothened mutant and methods for its use
EP3960860A3 (en) 2014-02-11 2022-06-08 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
EP3119789B1 (en) 2014-03-17 2020-04-22 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
RS62526B1 (sr) 2014-03-19 2021-11-30 Ionis Pharmaceuticals Inc Kompozicije za modulaciju ekspresije ataksina 2
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
DK3757214T3 (da) 2014-04-01 2022-07-04 Biogen Ma Inc Sammensætninger til modulering af sod-1-ekspression
EP3943607A1 (en) 2014-04-09 2022-01-26 The Scripps Research Institute Import of unnatural or modified nucleoside triphosphates into cells via nucleic acid triphosphate transporters
MA39835A (fr) 2014-04-17 2017-02-22 Biogen Ma Inc Compositions et procédés de modulation de l'épissage du smn2 chez un patient
WO2015164693A1 (en) 2014-04-24 2015-10-29 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
WO2015168514A1 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
SG10201809953QA (en) 2014-05-01 2018-12-28 Ionis Pharmaceuticals Inc Compositions and methods for modulating complement factor b expression
CN110903337A (zh) 2014-05-01 2020-03-24 Ionis制药公司 用于调节生长激素受体表达的组合物和方法
JP2017521045A (ja) 2014-05-01 2017-08-03 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. アンジオポエチン様因子3発現を調節するための組成物及び方法
WO2015168532A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating pkk expression
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療serpinc1相關疾患之方法和組成物
SG11201609376SA (en) 2014-05-22 2016-12-29 Alnylam Pharmaceuticals Inc Angiotensinogen (agt) irna compositions and methods of use thereof
KR20170031668A (ko) 2014-06-02 2017-03-21 칠드런'즈 메디컬 센터 코포레이션 면역 조절을 위한 방법 및 조성물
WO2015187966A1 (en) 2014-06-04 2015-12-10 Aurasense Therapeutics, Llc Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
CN106661580B (zh) 2014-06-10 2022-02-15 鹿特丹伊拉斯谟大学医疗中心 用于治疗庞帕病的反义寡核苷酸
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
JP2017524357A (ja) 2014-07-16 2017-08-31 モデルナティエックス インコーポレイテッドModernaTX,Inc. キメラポリヌクレオチド
US9951327B1 (en) 2014-07-17 2018-04-24 Integrated Dna Technologies, Inc. Efficient and rapid method for assembling and cloning double-stranded DNA fragments
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
EP3189069B1 (en) 2014-07-31 2024-10-23 UAB Research Foundation Apoe mimetic peptides and higher potency to clear plasma cholesterol
CN113069551A (zh) 2014-08-19 2021-07-06 西北大学 蛋白质/寡核苷酸核-壳纳米颗粒治疗剂
CN107106704A (zh) 2014-08-29 2017-08-29 儿童医疗中心有限公司 用于治疗癌症的方法和组合物
WO2016033424A1 (en) 2014-08-29 2016-03-03 Genzyme Corporation Methods for the prevention and treatment of major adverse cardiovascular events using compounds that modulate apolipoprotein b
PL3185957T3 (pl) 2014-08-29 2022-11-14 Alnylam Pharmaceuticals, Inc. Patisiran do zastosowania w leczeniu amyloidozy związanej z transtyretyną
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
WO2016040589A1 (en) 2014-09-12 2016-03-17 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
US10533172B2 (en) 2014-09-18 2020-01-14 The University Of British Columbia Allele-specific therapy for huntington disease haplotypes
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
WO2016061487A1 (en) 2014-10-17 2016-04-21 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 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
EP3218486A1 (en) 2014-11-10 2017-09-20 Glaxosmithkline Intellectual Property (No. 2) Limited Combination long acting compositions and methods for hepatitis c
EP3218485A1 (en) 2014-11-10 2017-09-20 Glaxosmithkline Intellectual Property (No. 2) Limited Long acting pharmaceutical compositions for hepatitis c
CA2968114A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
CA2968531A1 (en) 2014-11-21 2016-05-26 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
US10400243B2 (en) 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
EP3234141A4 (en) 2014-12-18 2018-06-20 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
JP2018506715A (ja) 2015-01-23 2018-03-08 ヴァンダービルト ユニバーシティー 堅固なインターフェロメーター及びその使用方法
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
EP3262181B1 (en) 2015-02-23 2024-04-10 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
CA2977965C (en) 2015-02-26 2021-12-21 Ionis Pharmaceuticals, Inc. Allele specific modulators of p23h rhodopsin
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
WO2016160721A1 (en) 2015-03-27 2016-10-06 President And Fellows Of Harvard College Modified t cells and methods of making and using the same
EP3277817A4 (en) 2015-04-03 2018-12-05 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating tmprss6 expression
MX2017012610A (es) 2015-04-08 2018-03-16 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen lect2.
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
MX385674B (es) 2015-04-16 2025-03-18 Ionis Pharmaceuticals Inc Composiciones para modular la expresion de c9orf72.
EP3307316A1 (en) 2015-06-12 2018-04-18 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
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
JP6983752B2 (ja) 2015-07-06 2021-12-17 フィオ ファーマシューティカルズ コーポレーションPhio Pharmaceuticals Corp. スーパーオキシドディスムターゼ1(sod1)を標的とする核酸分子
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
EP3919619A1 (en) 2015-07-17 2021-12-08 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
EP3332009A1 (en) 2015-08-04 2018-06-13 Yeda Research and Development Co., Ltd. Methods of screening for riboswitches and attenuators
WO2017027350A2 (en) 2015-08-07 2017-02-16 Arrowhead Pharmaceuticals, Inc. Rnai therapy for hepatitis b virus infection
KR20250145703A (ko) 2015-09-02 2025-10-13 알닐람 파마슈티칼스 인코포레이티드 프로그램된 세포사 1 리간드 1 (PD-L1) iRNA 조성물 및 그의 사용 방법
US10533175B2 (en) 2015-09-25 2020-01-14 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating Ataxin 3 expression
MY200263A (en) 2015-10-08 2023-12-18 Ionis Pharmaceuticals Inc Compounds and methods for modulating angiotensinogen expression
US11021707B2 (en) 2015-10-19 2021-06-01 Phio Pharmaceuticals Corp. Reduced size self-delivering nucleic acid compounds targeting long non-coding RNA
HUE054093T2 (hu) 2015-10-30 2021-08-30 Hoffmann La Roche Anti-HtrA1 antitestek és azok alkalmazási eljárásai
ES2938883T3 (es) 2015-11-05 2023-04-17 Los Angeles Childrens Hospital Oligo antisentido para su uso en el tratamiento de la leucemia mieloide aguda
PE20241347A1 (es) 2015-11-06 2024-07-03 Ionis Pharmaceuticals Inc MODULAR LA EXPRESION DE APOLIPOPROTEINA (a)
EP3389670A4 (en) 2015-12-04 2020-01-08 Ionis Pharmaceuticals, Inc. METHODS OF TREATING BREAST CANCER
CA3007014A1 (en) 2015-12-07 2017-06-15 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating a serpinc1-associated disorder
US10993995B2 (en) 2015-12-07 2021-05-04 Erasmus University Medical Center Rotterdam Enzymatic replacement therapy and antisense therapy for pompe disease
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
CA3006015A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
AU2017205462A1 (en) 2016-01-05 2018-06-07 Ionis Pharmaceuticals, Inc. Methods for reducing LRRK2 expression
WO2017132483A1 (en) 2016-01-29 2017-08-03 Vanderbilt University Free-solution response function interferometry
JP2019509721A (ja) 2016-02-04 2019-04-11 キュリス,インコーポレイテッド 突然変異体スムースンド及びその使用方法
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
EP3429690A4 (en) 2016-03-16 2019-10-23 Ionis Pharmaceuticals, Inc. METHOD FOR MODULATING KEAP1
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
JP2019518028A (ja) 2016-06-10 2019-06-27 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. 補体成分C5iRNA組成物及び発作性夜間血色素尿症(PNH)を処置するためのその使用方法
US11236339B2 (en) 2016-06-17 2022-02-01 Ionis Pharmaceuticals, Inc. Modulation of GYS1 expression
EP3475295B1 (en) 2016-06-24 2022-08-10 The Scripps Research Institute Novel nucleoside triphosphate transporter and uses thereof
KR20230088522A (ko) 2016-07-21 2023-06-19 맥스시티 인코포레이티드 게놈 dna를 변경하기 위한 방법 및 조성물
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
NL2017295B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
US11364304B2 (en) 2016-08-25 2022-06-21 Northwestern University Crosslinked micellar spherical nucleic acids
WO2018055577A1 (en) 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
US11033570B2 (en) 2016-12-02 2021-06-15 Cold Spring Harbor Laboratory Modulation of Lnc05 expression
SG10201913552UA (en) 2016-12-16 2020-03-30 Alnylam Pharmaceuticals Inc Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
CN119082104A (zh) 2017-01-10 2024-12-06 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
IL285053B (en) 2017-01-23 2022-07-01 Regeneron Pharma Variants of hsd17b13 and their uses
WO2018165564A1 (en) 2017-03-09 2018-09-13 Ionis Pharmaceuticals, Inc. Morpholino modified oligomeric compounds
WO2018183969A1 (en) 2017-03-30 2018-10-04 California Institute Of Technology Barcoded rapid assay platform for efficient analysis of candidate molecules and methods of making and using the platform
TWI801377B (zh) 2017-04-18 2023-05-11 美商阿尼拉製藥公司 治療具有b型肝炎病毒(hbv)感染之個體之方法
KR102778110B1 (ko) 2017-04-20 2025-03-10 신테나 아게 트리시클로-dna 뉴클레오시드를 포함하는 변형 올리고머 화합물 및 그의 용도
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
US20200163987A1 (en) 2017-07-10 2020-05-28 Genzyme Corporation Methods and compositions for treating a bleeding event in a subject having hemophilia
AR112756A1 (es) 2017-07-11 2019-12-11 Synthorx Inc Incorporación de nucleótidos no naturales, y su método
MA49716A (fr) 2017-07-11 2021-04-07 Scripps Research Inst Incorporation de nucléotides non naturels et procédés d'utilisationin vivo
EP3652317A1 (en) 2017-07-13 2020-05-20 Alnylam Pharmaceuticals, Inc. Lactate dehydrogenase a (ldha) irna compositions and methods of use thereof
US11690920B2 (en) 2017-07-13 2023-07-04 Northwestern University General and direct method for preparing oligonucleotide-functionalized metal-organic framework nanoparticles
KR20250130723A (ko) 2017-08-03 2025-09-02 신톡스, 인크. 증식성 질환 및 감염성 질환의 치료를 위한 사이토카인 접합체
EP3668984A4 (en) 2017-08-18 2021-09-08 Ionis Pharmaceuticals, Inc. MODULATION OF THE NOTCH SIGNALING PATH FOR THE TREATMENT OF RESPIRATORY DISORDERS
US10517889B2 (en) 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
WO2019055460A1 (en) 2017-09-13 2019-03-21 The Children's Medical Center Corporation COMPOSITIONS AND METHODS FOR THE TREATMENT OF TRANSPOSON-ASSOCIATED DISEASES
EP3684931A1 (en) 2017-09-19 2020-07-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
SG11202002940QA (en) 2017-11-01 2020-04-29 Alnylam Pharmaceuticals Inc Complement component c3 irna compositions and methods of use thereof
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
EP3710587A1 (en) 2017-11-16 2020-09-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
EP3714054A1 (en) 2017-11-20 2020-09-30 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
US12276654B2 (en) 2017-11-22 2025-04-15 The University Of Chicago Chemical probe-dependent evaluation of protein activity and uses thereof
BR112020012088A2 (pt) 2017-12-18 2020-11-17 Alnylam Pharmaceuticals, Inc. composições de irna do grupo de elevada mobilidade box-1 (hmgb1) e métodos de uso dos mesmos
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
KR20250035031A (ko) 2017-12-29 2025-03-11 더 스크립스 리서치 인스티튜트 비천연 염기쌍 조성물 및 사용 방법
SG11202006711UA (en) 2018-01-15 2020-08-28 Ionis Pharmaceuticals Inc Modulators of dnm2 expression
US20190233816A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
BR112020017016A2 (pt) 2018-02-26 2020-12-29 Synthorx, Inc. Conjugados da il-15 e usos dos mesmos
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
EP3768694A4 (en) 2018-03-22 2021-12-29 Ionis Pharmaceuticals, Inc. Methods for modulating fmr1 expression
KR20200136977A (ko) 2018-03-28 2020-12-08 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 엑소좀으로부터 단리된 dna에서의 후성유전학적 변화의 식별
CN112272516B (zh) 2018-04-06 2023-05-30 儿童医疗中心有限公司 用于体细胞重新编程和调整印记的组合物和方法
PE20201349A1 (es) 2018-04-11 2020-11-30 Ionis Pharmaceuticals Inc Moduladores de la expresion de ezh2
BR112020022546A8 (pt) 2018-05-07 2022-05-17 Alnylam Pharmaceuticals Inc Entrega extra-hepática
MX2020011911A (es) 2018-05-09 2021-01-29 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir de la expresion de atxn3.
PE20210393A1 (es) 2018-05-09 2021-03-02 Ionis Pharmaceuticals Inc Compuestos y metodos para la reduccion de la expresion de fxi
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
AU2019287635A1 (en) 2018-06-14 2020-12-17 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
US12496311B2 (en) 2018-07-17 2025-12-16 Aronora, Inc. Methods for safely reducing thrombopoietin
SG11202100077PA (en) 2018-07-25 2021-02-25 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn2 expression
JP7625512B2 (ja) 2018-08-13 2025-02-03 アルナイラム ファーマシューティカルズ, インコーポレイテッド B型肝炎ウイルス(HBV)dsRNA物質組成物およびその使用方法
TW202020157A (zh) 2018-08-16 2020-06-01 美商艾爾妮蘭製藥公司 用於抑制lect2基因表現之組合物及方法
AU2019339509A1 (en) 2018-09-14 2021-05-13 Northwestern University Programming protein polymerization with DNA
JP2022500003A (ja) 2018-09-18 2022-01-04 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. ケトヘキソキナーゼ(KHK)iRNA組成物およびその使用方法
CA3114004A1 (en) 2018-09-25 2020-04-02 Co-Diagnostics, Inc. Allele-specific design of cooperative primers for improved nucleic acid variant genotyping
JP7470107B2 (ja) 2018-09-28 2024-04-17 アルナイラム ファーマシューティカルズ, インコーポレイテッド トランスサイレチン(TTR)iRNA組成物及びTTR関連眼疾患を治療又は予防するためのその使用方法
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
KR20210091170A (ko) 2018-11-08 2021-07-21 신톡스, 인크. 인터류킨 10 접합체 및 이의 용도
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
KR20210092761A (ko) 2018-11-15 2021-07-26 아이오니스 파마수티컬즈, 인코포레이티드 Irf5 발현 조절인자
AU2019384181B2 (en) 2018-11-21 2025-04-10 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing prion expression
WO2020117706A1 (en) 2018-12-03 2020-06-11 Triplet Therapeutics, Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity
WO2020118259A1 (en) 2018-12-06 2020-06-11 Northwestern University Protein crystal engineering through dna hybridization interactions
CA3122402A1 (en) 2018-12-20 2020-06-25 Vir Biotechnology, Inc. Combination hbv therapy
JP2022515744A (ja) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ, インコーポレイテッド Kcnt1関連障害の治療のための組成物及び方法
MX2021008628A (es) 2019-01-16 2021-11-17 Genzyme Corp Composiciones de arni para serpinc1 y metodos de uso de las mismas.
AU2020216444A1 (en) 2019-01-31 2021-07-29 Ionis Pharmaceuticals, Inc. Modulators of YAP1 expression
TWI865486B (zh) 2019-02-06 2024-12-11 美商欣爍克斯公司 Il-2結合物及其使用方法
CN113544271B (zh) 2019-02-27 2025-07-11 Ionis制药公司 Malat1表达的调节剂
EP3941483A4 (en) 2019-03-21 2023-10-18 Arnay Sciences, Llc ANTISEN OLIGONUCLEOTIDES FOR ALLEL SPECIFICITY
LT3947684T (lt) 2019-03-29 2025-05-26 Ionis Pharmaceuticals, Inc. Ube3a-ats moduliavimui skirti junginiai ir būdai
JP7667074B2 (ja) 2019-03-29 2025-04-22 田辺三菱製薬株式会社 Dux4の発現を調節するための化合物、方法及び医薬組成物
JP2022532211A (ja) 2019-05-13 2022-07-13 ヴィア・バイオテクノロジー・インコーポレイテッド B型肝炎ウイルス(hbv)感染を処置するための組成物および方法
US20220211743A1 (en) 2019-05-17 2022-07-07 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
US11879145B2 (en) 2019-06-14 2024-01-23 The Scripps Research Institute Reagents and methods for replication, transcription, and translation in semi-synthetic organisms
AU2020297008A1 (en) 2019-06-18 2022-02-17 Janssen Sciences Ireland Unlimited Company Combination of hepatitis B virus (HBV) vaccines and HBV-targeting RNAi
JP2022536945A (ja) 2019-06-18 2022-08-22 ヤンセン・サイエンシズ・アイルランド・アンリミテッド・カンパニー B型肝炎ウイルス(HBV)ワクチンおよびHBVを標的化するRNAiの組合せ
PL3956450T3 (pl) 2019-07-26 2025-12-22 Ionis Pharmaceuticals, Inc. Związki i sposoby modulowania gfap
WO2021022109A1 (en) 2019-08-01 2021-02-04 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
KR20220062517A (ko) 2019-08-15 2022-05-17 아이오니스 파마수티컬즈, 인코포레이티드 결합 변형된 올리고머 화합물 및 이의 용도
CA3150163A1 (en) 2019-08-15 2021-02-18 Synthorx, Inc. Immuno oncology combination therapies with il-2 conjugates
MX2022002053A (es) 2019-08-23 2022-03-17 Synthorx Inc Conjugados de il-15 y sus usos.
CN114616331B (zh) 2019-09-03 2024-10-22 阿尔尼拉姆医药品有限公司 抑制lect2基因表达的组合物和方法
JP7758662B2 (ja) 2019-09-10 2025-10-22 シンソークス, インコーポレイテッド Il-2コンジュゲートおよび自己免疫疾患を治療するための使用方法
US12319711B2 (en) 2019-09-20 2025-06-03 Northwestern University Spherical nucleic acids with tailored and active protein coronae
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
EP4045652A1 (en) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
IL292360A (en) 2019-10-22 2022-06-01 Alnylam Pharmaceuticals Inc IRNA components c3 supplementary component and methods of using them
US12378560B2 (en) 2019-10-29 2025-08-05 Northwestern University Sequence multiplicity within spherical nucleic acids
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
AU2020376840A1 (en) 2019-11-01 2022-04-07 Alnylam Pharmaceuticals, Inc. Huntingtin (HTT) iRNA agent compositions and methods of use thereof
JP2022554272A (ja) 2019-11-04 2022-12-28 シンソークス, インコーポレイテッド インターロイキン10コンジュゲートおよびその使用
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
CA3160329A1 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
WO2021102373A1 (en) 2019-11-22 2021-05-27 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
US12503695B2 (en) 2020-01-27 2025-12-23 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services RAB13 and NET1 antisense oligonucleotides to treat metastatic cancer
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)
JP2023514190A (ja) 2020-02-10 2023-04-05 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 Vegf-a発現をサイレンシングするための組成物および方法
AU2021224778A1 (en) 2020-02-18 2022-09-29 Alnylam Pharmaceuticals, Inc. Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
CA3173034A1 (en) 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
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
AU2021232014A1 (en) 2020-03-06 2022-10-06 Alnylam Pharmaceuticals, Inc. Ketohexokinase (KHK) IRNA compositions and methods of use thereof
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 (ja) 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 コロナウイルスiRNA組成物およびその使用方法
WO2021202443A2 (en) 2020-03-30 2021-10-07 Alnylam Pharmaceucticals, 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
AR121769A1 (es) 2020-04-06 2022-07-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para el silenciamiento de la expresión de myoc
BR112022020227A2 (pt) 2020-04-07 2022-11-22 Alnylam Pharmaceuticals Inc Composições e métodos para silenciamento da expressão de scn9a
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 (ja) 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 アポリポタンパク質E(ApoE)iRNA剤組成物およびその使用方法
AU2021265813A1 (en) 2020-04-30 2022-11-10 Alnylam Pharmaceuticals, Inc. Complement factor B (CFB) iRNA compositions and methods of use thereof
IL297435A (en) 2020-05-01 2022-12-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating atxn1
EP4151237A4 (en) 2020-05-12 2024-08-14 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for regulating expression of ataxin 3
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)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
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)
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)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
WO2021231685A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021237097A1 (en) 2020-05-21 2021-11-25 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
JP2023530234A (ja) 2020-06-05 2023-07-14 ザ・ブロード・インスティテュート・インコーポレイテッド 新生物を治療するための組成物および方法
WO2021252557A1 (en) 2020-06-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
CN116075592A (zh) 2020-06-09 2023-05-05 阿尔尼拉姆医药品有限公司 用于沉默gpam(线粒体甘油-3-磷酸酰基转移酶1)表达的sirna组合物和方法
IL299063A (en) 2020-06-18 2023-02-01 Alnylam Pharmaceuticals Inc Xanthine dehydrogenase (xdh) RNA compositions and methods of using them
CA3182458A1 (en) 2020-06-24 2021-12-30 Laura ROSEN Engineered hepatitis b virus neutralizing antibodies and uses thereof
CN116209465A (zh) 2020-06-25 2023-06-02 新索思股份有限公司 用il-2缀合物与抗egfr抗体的免疫肿瘤学组合疗法
EP4172338A4 (en) 2020-06-29 2025-06-11 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING PLP1
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
MX2023001222A (es) 2020-07-28 2023-04-26 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de app.
WO2022032060A2 (en) 2020-08-07 2022-02-10 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating scn2a
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
EP3978608A1 (en) 2020-10-05 2022-04-06 SQY Therapeutics Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51
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
AU2021356610A1 (en) 2020-10-09 2023-06-15 Synthorx, Inc. Immuno oncology therapies with il-2 conjugates
JP2023546009A (ja) 2020-10-09 2023-11-01 シンソークス, インコーポレイテッド Il-2コンジュゲートおよびペムブロリズマブを用いる免疫腫瘍学併用療法
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
AU2021364920A1 (en) 2020-10-23 2023-06-22 Synthorx, Inc. Reverse transcription of polynucleotides comprising unnatural nucleotides
EP4244355A1 (en) 2020-11-13 2023-09-20 Alnylam Pharmaceuticals, Inc. Coagulation factor v (f5) irna compositions and methods of use thereof
LT4136092T (lt) 2020-11-18 2024-09-25 Ionis Pharmaceuticals, Inc. Junginiai ir būdai, skirti angiotenzinogeno raiškos moduliavimui
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
CN112480197B (zh) * 2020-12-02 2022-03-08 新乡拓新药业股份有限公司 一种合成胞嘧啶核苷的方法
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
CA3205040A1 (en) 2020-12-18 2022-06-23 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor xii
WO2022140702A1 (en) 2020-12-23 2022-06-30 Flagship Pioneering, Inc. Compositions of modified trems and uses thereof
AU2021411579A1 (en) 2020-12-31 2023-07-13 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2022147223A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
WO2022174101A1 (en) 2021-02-12 2022-08-18 Synthorx, Inc. Skin cancer combination therapy with il-2 conjugates and cemiplimab
EP4291243A1 (en) 2021-02-12 2023-12-20 Synthorx, Inc. Lung cancer combination therapy with il-2 conjugates and an anti-pd-1 antibody or antigen-binding fragment thereof
MX2023009324A (es) 2021-02-12 2023-10-13 Alnylam Pharmaceuticals Inc Composiciones de arni de superóxido dismutasa 1 (sod1), y métodos de uso de este para el tratamiento o prevención de enfermedades neurodegenerativas relacionadas con superóxido dismutasa 1 (sod1).
EP4298220A1 (en) 2021-02-25 2024-01-03 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods of use thereof
CA3211059A1 (en) 2021-02-26 2022-09-01 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
IL305414A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
EP4304640A1 (en) 2021-03-12 2024-01-17 Northwestern University Antiviral vaccines using spherical nucleic acids
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
KR20230162024A (ko) 2021-03-29 2023-11-28 알닐람 파마슈티칼스 인코포레이티드 헌팅틴(HTT) iRNA 제제 조성물 및 이의 사용 방법
WO2022213118A1 (en) 2021-03-31 2022-10-06 Entrada Therapeutics, Inc. Cyclic cell penetrating peptides
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
WO2022231999A1 (en) 2021-04-26 2022-11-03 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
JP2024519293A (ja) 2021-04-29 2024-05-10 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 シグナル伝達兼転写活性化因子6(STAT6)iRNA組成物およびその使用方法
EP4334448A1 (en) 2021-05-03 2024-03-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
EP4337263A1 (en) 2021-05-10 2024-03-20 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
CA3218805A1 (en) 2021-05-10 2022-11-17 Ziqing QIAN Compositions and methods for intracellular therapeutics
EP4337261A2 (en) 2021-05-10 2024-03-20 Entrada Therapeutics, Inc. Compositions and methods for modulating mrna splicing
JP2024522068A (ja) 2021-05-18 2024-06-11 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 ナトリウム-グルコース共輸送体2(sglt2)irna組成物およびその使用方法
WO2022246023A1 (en) 2021-05-20 2022-11-24 Korro Bio, Inc. Methods and compositions for adar-mediated editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
TW202317762A (zh) 2021-06-02 2023-05-01 美商艾拉倫製藥股份有限公司 含有類PATATIN磷脂酶結構域3(PNPLA3)的iRNA組成物及其使用方法
TW202313679A (zh) 2021-06-03 2023-04-01 美商欣爍克斯公司 包含il-2接合物及pd-1拮抗劑之頭頸癌組合療法
KR20240017911A (ko) 2021-06-04 2024-02-08 알닐람 파마슈티칼스 인코포레이티드 인간 염색체 9 개방 해독 프레임 72(C9orf72) iRNA 제제 조성물 및 이의 사용 방법
JP2024523000A (ja) 2021-06-08 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 シュタルガルト病及び/又は網膜結合タンパク質4(rbp4)関連障害を治療又は予防するための組成物及び方法
WO2022266414A1 (en) 2021-06-18 2022-12-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
WO2022271818A1 (en) 2021-06-23 2022-12-29 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
EP4363574A1 (en) 2021-06-29 2024-05-08 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
JP2024527584A (ja) 2021-07-09 2024-07-25 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 CNS送達のためのBis-RNAi化合物
TW202333748A (zh) 2021-07-19 2023-09-01 美商艾拉倫製藥股份有限公司 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物
CN117716032A (zh) 2021-07-21 2024-03-15 阿尔尼拉姆医药品有限公司 代谢病症相关靶基因iRNA组合物和其使用方法
EP4373933A1 (en) 2021-07-23 2024-05-29 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
IL310244A (en) 2021-08-03 2024-03-01 Alnylam Pharmaceuticals Inc Transthyretin (TTR) IRNA compositions and methods of using them
MX2024001445A (es) 2021-08-04 2024-02-27 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) y metodos para silenciar el angiotensinogeno (agt).
TW202334413A (zh) 2021-08-13 2023-09-01 美商艾拉倫製藥股份有限公司 第十二因子(F12)iRNA組成物及其使用方法
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.
EP4395829A1 (en) 2021-09-01 2024-07-10 Entrada Therapeutics, Inc. Compounds and methods for skipping exon 44 in duchenne muscular dystrophy
EP4401742A2 (en) 2021-09-17 2024-07-24 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
KR20240083183A (ko) 2021-09-20 2024-06-11 알닐람 파마슈티칼스 인코포레이티드 인히빈 서브유닛 베타 e (inhbe) 조절제 조성물 및 이의 이용 방법
EP4405484A2 (en) 2021-09-24 2024-07-31 Alnylam Pharmaceuticals, Inc. Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof
KR20240082351A (ko) 2021-10-01 2024-06-10 아다르엑스 파마슈티컬스, 인크. 프리칼리크레인 조절 조성물 및 이의 사용 방법
CA3234887A1 (en) 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
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
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
WO2023076451A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
AU2022391744A1 (en) 2021-11-18 2024-06-27 Cornell University Microrna-dependent mrna switches for tissue-specific mrna-based therapies
EP4452327A1 (en) 2021-12-20 2024-10-30 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023220744A2 (en) 2022-05-13 2023-11-16 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
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
CN120239607A (zh) 2022-09-23 2025-07-01 Ionis制药公司 用于降低mecp2表达的化合物和方法
JP2025532985A (ja) 2022-09-30 2025-10-03 アルナイラム ファーマシューティカルズ, インコーポレイテッド 修飾二本鎖rna剤
WO2024136899A1 (en) 2022-12-21 2024-06-27 Synthorx, Inc. Cancer therapy with il-2 conjugates and chimeric antigen receptor therapies
IL322261A (en) 2023-02-09 2025-09-01 Alnylam Pharmaceuticals Inc Reverser molecules and methods of using them
EP4665865A1 (en) 2023-02-17 2025-12-24 Anjarium Biosciences AG Methods of making dna molecules and compositions and uses thereof
WO2024196937A1 (en) 2023-03-20 2024-09-26 Synthorx, Inc. Cancer therapy with il-2 peg conjugates
AU2024254919A1 (en) 2023-04-12 2025-10-30 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
WO2024220930A2 (en) 2023-04-20 2024-10-24 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024226499A1 (en) 2023-04-24 2024-10-31 The Broad Institute, Inc. Compositions and methods for modifying fertility
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
TW202502385A (zh) 2023-05-12 2025-01-16 美商雅迪克斯製藥公司 Nmda配位體結合之化合物及其用途
AU2024279767A1 (en) 2023-05-26 2025-12-04 Adarx Pharmaceuticals, Inc. Sod1-modulating compositions and methods of use thereof
WO2024263694A1 (en) 2023-06-20 2024-12-26 Adarx Pharmaceuticals, Inc. Lrrk2-modulating compositions and methods of use thereof
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025024334A1 (en) 2023-07-21 2025-01-30 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods
AU2024322388A1 (en) 2023-08-04 2026-01-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025072713A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Polymyxins for delivery of agents to the kidney
WO2025072699A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Aminoglycosides for delivery of agents to the kidney
WO2025072672A2 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
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
AU2024369609A1 (en) 2023-10-31 2025-12-11 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
WO2025178854A2 (en) 2024-02-19 2025-08-28 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting cideb and related methods
US20250313840A1 (en) 2024-03-20 2025-10-09 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
WO2025207517A2 (en) 2024-03-25 2025-10-02 Synthorx, Inc. Synthetic trna synthetases and cells comprising synthetic molecules for production of polypeptides
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
WO2025259743A1 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dual conjugate compounds for extrahepatic delivery
WO2025259747A2 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof
WO2026006436A1 (en) 2024-06-25 2026-01-02 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of tar dna binding protein 43 kda (tdp43)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4806463A (en) * 1986-05-23 1989-02-21 Worcester Foundation For Experimental Biology Inhibition of HTLV-III by exogenous oligonucleotides
EP0266099A3 (en) * 1986-10-28 1990-09-19 The Johns Hopkins University Oligonucleoside alkyl or arylphosphonate derivatives capable of crosslinking with or cleaving nucleic acids
EP0267878A1 (en) * 1986-11-14 1988-05-18 Ciba-Geigy Ag N9-cyclopentyl-substituted adenine derivatives
IN166499B (cg-RX-API-DMAC10.html) * 1987-02-12 1990-05-19 Takeda Chemical Industries Ltd
ATE132190T1 (de) * 1988-02-26 1996-01-15 Worcester Found Ex Biology Hemmung von htlv-iii durch exogene oligonukleotide
DK0942000T3 (da) * 1989-10-24 2004-11-01 Isis Pharmaceuticals Inc 2'-modificerede oligonukleotider

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6143743A (en) * 1997-07-03 2000-11-07 Dupont Pharmaceuticals Company Imidazopyrimidines and imidazopyridines for the treatment of neurological disorders
US6362180B1 (en) 1997-07-03 2002-03-26 Bristol-Myers Squibb Pharma Company Imidazopyridines for the treatment of neurological disorders
US6642230B2 (en) 1997-07-03 2003-11-04 Bristol-Myers Squibb Pharma Company Imidazopyrimidines and imidazopyridines for the treatment of neurological disorders
US6124463A (en) * 1998-07-02 2000-09-26 Dupont Pharmaceuticals Benzimidazoles as corticotropin release factor antagonists
US6579876B2 (en) 1998-07-02 2003-06-17 Bristol-Myers Squibb Pharma Company Imidazo-pyridines, -pyridazines, and -triazines as corticotropin releasing factor antagonists

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US5700920A (en) 1997-12-23
EP0577558A3 (cg-RX-API-DMAC10.html) 1994-04-20
JPH06100562A (ja) 1994-04-12

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