MX9303965A - Nucleosidos carbociclicos que contienen anillos biciclicos, oligonucleotidos a partir de los mismos, procedimiento para su preparacion, su uso y sus intermediarios. - Google Patents

Nucleosidos carbociclicos que contienen anillos biciclicos, oligonucleotidos a partir de los mismos, procedimiento para su preparacion, su uso y sus intermediarios.

Info

Publication number
MX9303965A
MX9303965A MX9303965A MX9303965A MX9303965A MX 9303965 A MX9303965 A MX 9303965A MX 9303965 A MX9303965 A MX 9303965A MX 9303965 A MX9303965 A MX 9303965A MX 9303965 A MX9303965 A MX 9303965A
Authority
MX
Mexico
Prior art keywords
preparation
oligonucleotides
carbociclic
intermediaries
procedure
Prior art date
Application number
MX9303965A
Other languages
English (en)
Spanish (es)
Inventor
Karl-Heinz Altmann
Rene Imwinkelried
Albert Eschenmoser
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 MX9303965A publication Critical patent/MX9303965A/es

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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)
  • Saccharide Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
MX9303965A 1992-07-01 1993-06-30 Nucleosidos carbociclicos que contienen anillos biciclicos, oligonucleotidos a partir de los mismos, procedimiento para su preparacion, su uso y sus intermediarios. MX9303965A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CH207592 1992-07-01

Publications (1)

Publication Number Publication Date
MX9303965A true MX9303965A (es) 1994-04-29

Family

ID=4225321

Family Applications (1)

Application Number Title Priority Date Filing Date
MX9303965A MX9303965A (es) 1992-07-01 1993-06-30 Nucleosidos carbociclicos que contienen anillos biciclicos, oligonucleotidos a partir de los mismos, procedimiento para su preparacion, su uso y sus intermediarios.

Country Status (5)

Country Link
US (3) US5461152A (OSRAM)
EP (1) EP0577558A2 (OSRAM)
JP (1) JPH06100562A (OSRAM)
CA (1) CA2099712A1 (OSRAM)
MX (1) MX9303965A (OSRAM)

Families Citing this family (772)

* 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
AU679566B2 (en) 1993-09-03 1997-07-03 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
AU677441B2 (en) * 1993-09-24 1997-04-24 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Conformationally locked nucleoside analogues
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
US20030044941A1 (en) 1996-06-06 2003-03-06 Crooke Stanley T. Human RNase III and compositions and uses thereof
WO2005121368A1 (en) * 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Chimeric gapped oligomeric compositions
US20040171031A1 (en) * 1996-06-06 2004-09-02 Baker Brenda F. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US20050053976A1 (en) * 1996-06-06 2005-03-10 Baker Brenda F. Chimeric oligomeric compounds and their use in gene modulation
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
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US7812149B2 (en) * 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US20040203024A1 (en) * 1996-06-06 2004-10-14 Baker Brenda F. Modified oligonucleotides for use in RNA interference
AU6766496A (en) * 1996-08-07 1998-02-25 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The 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
JP2002510319A (ja) 1997-07-01 2002-04-02 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 オリゴヌクレオチドの消化管を介したデリバリーのための組成物及び方法
SK179899A3 (en) * 1997-07-03 2001-12-03 Du Pont Pharm Co Imidazopyrimidines and imidazopyridines for the treatment of neurological disorders
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
US6841539B1 (en) 1998-05-21 2005-01-11 Isis Pharmaceuticals, Inc. Compositions and methods for topical delivery of oligonucleotides
EP1080103A4 (en) * 1998-05-21 2003-07-02 Isis Pharmaceuticals Inc PREPARATIONS AND METHOD FOR THE NON-PARENTERAL ADMINISTRATION OF OLIGONUCLEOTIDES
US6335439B1 (en) 1998-06-11 2002-01-01 Isis Pharmaceuticals, Inc. Method of preparing phosphoramidites
US6365589B1 (en) 1998-07-02 2002-04-02 Bristol-Myers Squibb Pharma Company Imidazo-pyridines, -pyridazines, and -triazines as corticotropin releasing factor antagonists
US6124463A (en) * 1998-07-02 2000-09-26 Dupont Pharmaceuticals Benzimidazoles as corticotropin release factor antagonists
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
US7668658B2 (en) * 1999-10-13 2010-02-23 Sequenom, Inc. Methods for generating databases and databases for identifying polymorphic genetic markers
CA2397366C (en) 2000-01-14 2011-03-15 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
AU5340801A (en) * 2000-04-13 2001-10-30 Thomas N Wight Therapeutic compounds and methods
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
WO2002030465A2 (en) 2000-10-12 2002-04-18 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
US7803915B2 (en) * 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
EP2000545B1 (en) 2001-06-20 2011-08-31 Genentech, Inc. Compositions and methods for the diagnosis and treatment of lung tumor
US20050107595A1 (en) * 2001-06-20 2005-05-19 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
EP2270024B1 (en) 2001-06-21 2018-10-24 Ionis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. 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
EP2153843B1 (en) 2001-09-18 2012-08-15 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
NZ566396A (en) 2001-10-09 2009-07-31 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
AU2002364945A1 (en) * 2001-10-25 2003-07-09 Neurogenetics, Inc. 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
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
US20030228305A1 (en) 2002-01-02 2003-12-11 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
MXPA04010092A (es) 2002-04-16 2004-12-13 Genentech Inc Composiciones y metodos para el diagnostico y tratamiento de tumores.
US7432342B2 (en) 2002-05-03 2008-10-07 Sequenom, Inc. Kinase anchor protein muteins, peptides thereof and related documents
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
WO2003105780A2 (en) * 2002-06-18 2003-12-24 Epigenesis Pharmaceuticals, Inc. A dry powder oligonucleotide formulation, preparation and its uses
US20050287648A1 (en) 2002-08-05 2005-12-29 University Of Rochester Protein Transducing Domain/Deaminase Chimeric Proteins, Related Compounds, and Uses Thereof
EP1537208A1 (en) 2002-09-13 2005-06-08 Replicor, Inc. Non-sequence complementary antiviral oligonucleotides
EP2322535A3 (en) 2002-09-20 2011-09-28 Yale University Riboswitches, methods for their use, and compositions for use with riboswitches
EP2272958A1 (en) 2002-09-26 2011-01-12 ISIS Pharmaceuticals, Inc. Modulation of forkhead box O1A expression
EP1560597A4 (en) * 2002-10-29 2007-06-27 Pharmacia Corp DIFFERENTIALLY EXPRESSED CANCER GENES, POLYPEPTIDES CODED THEREIN, AND METHOD OF USE THEREOF
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP1560839A4 (en) 2002-11-05 2008-04-23 Isis Pharmaceuticals Inc CHIMERIC OLIGOMER COMPOUNDS AND THEIR 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
AU2003291755A1 (en) * 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
CA2505801A1 (en) 2002-11-13 2004-05-27 Rosanne Crooke Antisense modulation of apolipoprotein b expression
SI1569695T1 (sl) 2002-11-13 2013-08-30 Genzyme Corporation Protismiselna modulacija ekspresije apolipoproteina B
CA2506127C (en) 2002-11-15 2013-07-09 Morphotek, Inc. Methods of generating high-production of antibodies from hybridomas created by in vitro immunization
EP1569685B8 (en) 2002-11-15 2012-12-05 MUSC Foundation For Research Development Complement receptor 2 targeted complement modulators
AU2003294462C1 (en) 2002-11-21 2011-06-30 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
US9487823B2 (en) * 2002-12-20 2016-11-08 Qiagen Gmbh Nucleic acid amplification
EP2261371B1 (en) 2002-12-20 2015-07-22 QIAGEN GmbH Nucleic acid amplification
US6977153B2 (en) 2002-12-31 2005-12-20 Qiagen Gmbh Rolling circle amplification of RNA
DK1597366T3 (da) 2003-02-11 2013-02-25 Antisense Therapeutics Ltd Modulering af ekspression af insulin-lignende vækstfaktor receptor I
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
WO2004108081A2 (en) 2003-06-02 2004-12-16 Isis Pharmaceuticals, Inc. Oligonucleotide synthesis with alternative solvents
WO2005002507A2 (en) 2003-06-03 2005-01-13 Isis Pharmaceuticals, Inc. Modulation of survivin expression
GB2417727B (en) 2003-06-13 2008-01-16 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
EP1636342A4 (en) * 2003-06-20 2008-10-08 Isis Pharmaceuticals Inc OLIGOMER COMPOUNDS FOR USE IN THE MODULATION OF GENES
EP2530157B1 (en) 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of miRNAs
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
SG146682A1 (en) 2003-09-18 2008-10-30 Isis Pharmaceuticals Inc Modulation of eif4e expression
AU2004274021B2 (en) 2003-09-18 2009-08-13 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
ES2472690T3 (es) 2003-11-17 2014-07-02 Genentech, Inc. Anticuerpo contra CD22 para el tratamiento de tumores de origen hematopoy�tico
US20050164271A1 (en) 2004-01-20 2005-07-28 Sanjay Bhanot Modulation of glucocorticoid receptor expression
US8778900B2 (en) 2004-01-22 2014-07-15 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP1 expression
US7468431B2 (en) 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 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
EP2700720A3 (en) 2004-03-15 2015-01-28 Isis Pharmaceuticals, Inc. Compositions and methods for optimizing cleavage of RNA by RNASE H
US20050244869A1 (en) * 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
US20050267300A1 (en) 2004-04-05 2005-12-01 Muthiah Manoharan Processes and reagents for oligonucleotide synthesis and purification
US20050260755A1 (en) * 2004-04-06 2005-11-24 Isis Pharmaceuticals, Inc. Sequential delivery of oligomeric compounds
WO2006073458A2 (en) 2004-04-30 2006-07-13 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a c5-modified pyrimidine
JP2008500064A (ja) 2004-05-21 2008-01-10 ザ ユーエービー リサーチ ファウンデーション 可変性リンパ球受容体、その関連ポリペプチドおよび核酸、ならびにその使用
EP1765416A4 (en) * 2004-06-03 2010-03-24 Isis Pharmaceuticals Inc DOUBLE-STRONG COMPOSITIONS WITH DIFFERENTLY MODIFIED STRANDS FOR USE IN GENE MODULATION
US8394947B2 (en) * 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
WO2006023880A2 (en) 2004-08-23 2006-03-02 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
CA2989941A1 (en) * 2004-09-23 2006-03-30 Arc Pharmaceuticals, Inc. Pharmaceutical compostions and methods relating to inhibiting fibrous adhesions or inflammatory disease using low sulphate fucans
EP1809302B1 (en) 2004-10-20 2012-08-22 Antisense Therapeutics Ltd Antisens modulation of integrin alpha-4 expression
US20100256062A1 (en) * 2004-12-06 2010-10-07 Howard Tommy E 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
EP1891141B1 (en) 2005-05-31 2016-11-16 Ecole Polytechnique Fédérale de Lausanne (EPFL) Triblock copolymers for cytoplasmic delivery of gene-based drugs
US20090209621A1 (en) 2005-06-03 2009-08-20 The Johns Hopkins University Compositions and methods for decreasing microrna expression for the treatment of neoplasia
US8252756B2 (en) 2005-06-14 2012-08-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
US7776532B2 (en) 2005-08-11 2010-08-17 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
WO2007027894A2 (en) 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Antisense compounds having enhanced anti-microrna activity
CA2620856C (en) 2005-08-29 2017-11-28 Isis Pharmaceuticals, Inc. Methods for use in modulating mir-122a
EP1762627A1 (de) 2005-09-09 2007-03-14 Qiagen GmbH Verfahren zur Aktivierung einer Nukleinsäure für eine Polymerase-Reaktion
EP2392646A1 (en) 2005-10-14 2011-12-07 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
CN101365801B (zh) * 2005-10-28 2013-03-27 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
WO2007056326A2 (en) * 2005-11-04 2007-05-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of nav1.8 gene
EP1945270B1 (en) 2005-11-09 2011-05-25 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of factor v leiden mutant gene
AU2006318194B2 (en) 2005-11-21 2012-08-09 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
US8288354B2 (en) 2005-12-28 2012-10-16 The Scripps Research Institute Natural antisense and non-coding RNA transcripts as drug targets
DK2161038T3 (da) 2006-01-26 2014-03-03 Isis Pharmaceuticals Inc Sammensætninger og deres anvendelser rettet mod Huntington
JP5213723B2 (ja) 2006-01-27 2013-06-19 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 マイクロrnaの調節に使用するためのオリゴマー化合物及び組成物
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
US7399845B2 (en) 2006-01-27 2008-07-15 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
CN101448849B (zh) 2006-03-31 2013-08-21 阿尔尼拉姆医药品有限公司 抑制Eg5基因表达的组合物和方法
CA2651031A1 (en) * 2006-05-03 2007-11-08 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
JP5731115B2 (ja) 2006-05-05 2015-06-10 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 遺伝子発現を調節するための化合物および方法
EP2505649A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of GCGR
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
BRPI0712034A2 (pt) 2006-05-19 2012-01-10 Alnylam Pharmaceuticals Inc modulação de rnai de aha e usos terapêuticos do mesmo
CA2653451C (en) 2006-05-22 2015-12-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression 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
CA2660052A1 (en) * 2006-08-04 2008-02-07 Isis Pharmaceuticals, Inc. Compositions and methods for the modulation of jnk proteins
WO2008033866A2 (en) * 2006-09-11 2008-03-20 Yale University Methods and compositions for the use of lysine riboswitches
AU2007324117B2 (en) 2006-10-05 2014-03-06 Massachussetts Institute Of Technology Multifunctional encoded particles for high-throughput analysis
ES2620472T5 (es) 2006-10-18 2020-07-09 Ionis Pharmaceuticals Inc Compuestos antisentido
WO2008136852A2 (en) 2006-11-01 2008-11-13 University Of Rochester Methods and compositions related to the structure and function of apobec3g
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
EP2453016A1 (en) * 2006-11-27 2012-05-16 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
WO2008094370A2 (en) 2006-12-22 2008-08-07 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
AU2008212820B2 (en) 2007-02-09 2014-01-30 Northwestern University Particles for detecting intracellular targets
WO2008127789A2 (en) * 2007-02-27 2008-10-23 Northwestern University Molecule attachment to nanoparticles
CA2681634A1 (en) 2007-03-22 2008-09-25 Yale University Methods and compositions related to riboswitches that control alternative splicing
AP2014007971A0 (en) 2007-03-29 2014-09-30 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expressionof a gene from the ebola
AU2008251381B2 (en) 2007-05-11 2014-10-30 The Johns Hopkins University Biomarkers for melanoma
AU2008260089A1 (en) * 2007-05-29 2008-12-11 Yale University Methods and compositions related to riboswitches that control alternative splicing and RNA processing
JP2010528617A (ja) 2007-05-29 2010-08-26 イェール ユニバーシティー リボスイッチならびにリボスイッチを使用するためのおよびリボスイッチと共てに使用するための方法および組成物
DK2160464T3 (da) 2007-05-30 2014-08-04 Univ Northwestern Nukleinsyrefunktionaliserede nanopartikler til terapeutiske anvendelser
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
US7807372B2 (en) * 2007-06-04 2010-10-05 Northwestern University Screening sequence selectivity of oligonucleotide-binding molecules using nanoparticle based colorimetric assay
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
ATE538127T1 (de) * 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-disubstituierte bicyclische nukleinsäureanaloga
JP5572090B2 (ja) 2007-08-15 2014-08-13 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 テトラヒドロピラン核酸類似体
CA2704729C (en) 2007-08-28 2020-06-23 Uab Research Foundation Synthetic apolipoprotein e mimicking polypeptides and methods of use
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
US8445217B2 (en) 2007-09-20 2013-05-21 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
US20110152346A1 (en) * 2007-11-05 2011-06-23 Baltic Technology Development Ltd. Use of Oligonucleotides with Modified Bases in Hybridization of Nucleic Acids
USRE47320E1 (en) 2007-11-20 2019-03-26 Ionis Pharmaceuticals, Inc. Modulation of CD40 expression
JP5530933B2 (ja) 2007-12-10 2014-06-25 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 第vii因子遺伝子発現阻害のための組成物及び方法
EP2224912B1 (en) 2008-01-02 2016-05-11 TEKMIRA Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
US8530640B2 (en) * 2008-02-07 2013-09-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
CA2715289C (en) 2008-02-11 2019-12-24 Rxi Pharmaceuticals Corporation Modified rnai polynucleotides and uses thereof
MX2010009611A (es) 2008-03-05 2010-12-15 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de genes eg5 y vegf.
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
EP2285819B1 (en) 2008-04-04 2013-10-16 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
CA2720675C (en) 2008-04-18 2019-08-27 Baxter International Inc. Microsphere-based composition for preventing and/or reversing new-onset autoimmune diabetes
EP2280995A2 (en) * 2008-04-29 2011-02-09 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
EP2313505A2 (en) * 2008-07-15 2011-04-27 F. Hoffmann-La Roche AG Compositions and methods for inhibiting expression of tgf-beta receptor genes
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
EP2317847B1 (en) 2008-07-29 2019-04-17 The Board of Regents of The University of Texas System Selective inhibition of polyglutamine protein expression
US20110237646A1 (en) * 2008-08-07 2011-09-29 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
SG196769A1 (en) 2008-08-25 2014-02-13 Excaliard Pharmaceuticals Inc Antisense oligonucleotides directed against connective tissue growth factor and uses thereof
EP2334319B1 (en) 2008-08-25 2017-12-06 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
CN102405286A (zh) 2008-09-22 2012-04-04 阿克赛医药公司 减小大小的自递送RNAi化合物
DK2356129T3 (da) * 2008-09-24 2013-05-13 Isis Pharmaceuticals Inc Substituerede alpha-L-bicykliske nukleosider
DK2361256T3 (da) 2008-09-24 2013-07-01 Isis Pharmaceuticals Inc Cyclohexenyl-nukleinsyreanaloger
EP3109321B1 (en) 2008-09-25 2019-05-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
EP3335715A3 (en) 2008-10-15 2018-08-08 Ionis Pharmaceuticals, Inc. Modulation of factor 11 expression
CA2739895C (en) 2008-10-20 2018-09-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
JP5763539B2 (ja) 2008-10-24 2015-08-12 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 5’及び2’ビス置換ヌクレオシド及びそれから製造されるオリゴマー化合物
US8987435B2 (en) 2008-10-24 2015-03-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
PE20120115A1 (es) * 2008-11-17 2012-02-20 Hoffmann La Roche Composiciones y metodos para inhibir la expresion de genes del factor vii
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
EP2365803B1 (en) * 2008-11-24 2017-11-01 Northwestern University Polyvalent rna-nanoparticle compositions
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
EP2370582B1 (en) 2008-12-04 2017-05-10 CuRNA, Inc. Treatment of tumor suppressor gene related diseases by inhibition of natural antisense transcript to the gene
MX2011005910A (es) 2008-12-04 2011-06-17 Opko Curna Llc Tratamiento de enfermedades relacionadas con eritropoyetina (epo) mediante inhibicion del transcrito antisentido natural a eritropoyetina.
NZ593618A (en) 2008-12-10 2013-02-22 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
CN102307470B (zh) * 2009-01-08 2015-05-20 西北大学 通过多价寡核苷酸修饰的纳米颗粒缀合物抑制细菌蛋白的产生
KR101546673B1 (ko) * 2009-01-15 2015-08-25 삼성전자주식회사 전자 사진용 토너 및 그의 제조방법
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
MX2011007776A (es) * 2009-02-03 2011-08-12 Hoffmann La Roche Compsiciones y metodos para inhibir la expresion de genes ptp1b.
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
EP2393825A2 (en) 2009-02-06 2011-12-14 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US8536320B2 (en) 2009-02-06 2013-09-17 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
CA2752239C (en) 2009-02-12 2021-03-30 Opko Curna, Llc 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
US20120041051A1 (en) 2009-02-26 2012-02-16 Kevin Fitzgerald Compositions And Methods For Inhibiting Expression Of MIG-12 Gene
EP2963116B1 (en) 2009-03-04 2020-11-11 CuRNA, Inc. Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirt 1
US20100267806A1 (en) 2009-03-12 2010-10-21 David Bumcrot LIPID FORMULATED COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF Eg5 AND VEGF GENES
CN102482677B (zh) 2009-03-16 2017-10-17 库尔纳公司 通过抑制nrf2的天然反义转录物治疗核因子(红细胞衍生2)‑样2(nrf2)相关疾病
EP2408920B1 (en) 2009-03-17 2017-03-08 CuRNA, Inc. Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
KR102036676B1 (ko) 2009-04-15 2019-10-24 노오쓰웨스턴 유니버시티 올리고뉴클레오티드 관능화된 나노입자의 전달
EP3248618A1 (en) 2009-04-22 2017-11-29 Massachusetts Institute Of Technology Innate immune suppression 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
US8318690B2 (en) 2009-05-01 2012-11-27 Curna, Inc. Treatment of hemoglobin (HBF/HBG) related diseases by inhibition of natural antisense transcript to HBF/HBG
KR102229618B1 (ko) 2009-05-05 2021-03-18 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
JP5889783B2 (ja) 2009-05-05 2016-03-22 テクミラ ファーマシューティカルズ コーポレイションTekmira Pharmaceuticals Corporation 免疫細胞へオリゴヌクレオチドを送達する方法
KR101835889B1 (ko) 2009-05-06 2018-03-08 큐알엔에이, 인크. 지질 수송 및 대사 유전자에 대한 천연 안티센스 전사체의 억제에 의해 지질 수송 및 대사 유전자 관련된 질환의 치료
EP2427552B1 (en) 2009-05-06 2016-11-16 CuRNA, Inc. Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp
CA2759838A1 (en) * 2009-05-15 2010-11-18 F. Hoffmann-La Roche Ag Compositions and methods for inhibiting expression of glucocorticoid receptor (gcr) genes
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
CA2762369C (en) 2009-05-18 2021-12-28 Joseph Collard Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor
US8895527B2 (en) 2009-05-22 2014-11-25 Curna, Inc. Treatment of transcription factor E3 (TFE3) and insulin receptor substrate 2(IRS2) related diseases by inhibition of natural antisense transcript to TFE3
US8791085B2 (en) 2009-05-28 2014-07-29 Curna, Inc. Treatment of antiviral gene related diseases by inhibition of natural antisense transcript to an antiviral gene
NZ622843A (en) 2009-06-10 2015-10-30 Tekmira Pharmaceuticals Corp Improved lipid formulation
US20120171170A1 (en) 2009-06-16 2012-07-05 Opko Curna, Llc Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
KR101702689B1 (ko) 2009-06-16 2017-02-06 큐알엔에이, 인크. Pon1에 대한 천연 안티센스 전사체의 억제에 의한 파라옥소나제 1(pon1) 관련된 질환의 치료
LT3305302T (lt) 2009-06-17 2018-12-10 Biogen Ma Inc. Junginiai ir būdai smn2 splaisingo moduliavimui subjekte
WO2010151671A2 (en) 2009-06-24 2010-12-29 Curna, Inc. Treatment of tumor necrosis factor receptor 2 (tnfr2) related diseases by inhibition of natural antisense transcript to tnfr2
WO2010151674A2 (en) 2009-06-26 2010-12-29 Curna, Inc. Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
EP2462229B1 (en) 2009-08-05 2016-05-11 CuRNA, Inc. Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US9029338B2 (en) 2009-08-14 2015-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
US20120157324A1 (en) 2009-08-17 2012-06-21 Yale University Methylation biomarkers and methods of use
KR101892760B1 (ko) 2009-08-25 2018-08-28 큐알엔에이, 인크. IQGAP에 대한 천연 안티센스 전사체의 억제에 의한 GTPase 활성화 단백질을 함유하는 IQ 모티프(IQGAP)와 관련된 질환의 치료
WO2011028950A1 (en) 2009-09-02 2011-03-10 Genentech, Inc. Mutant smoothened and methods of using the same
CN102711826B (zh) 2009-10-22 2017-03-29 霍夫曼-拉罗奇有限公司 用于调控巨噬细胞刺激性蛋白的hepsin活化的方法和组合物
EP2494066B1 (en) 2009-10-27 2017-04-05 Swift Biosciences, Inc. Bimolecular primers
US9376690B2 (en) 2009-10-30 2016-06-28 Northwestern University Templated nanoconjugates
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
US20130084565A1 (en) 2009-11-03 2013-04-04 University Of Virginia Patent Foundation Versatile, visible method for detecting polymeric analytes
AR078921A1 (es) 2009-11-09 2011-12-14 Hoffmann La Roche Composiciones y metodos para inhibir la expresion de genes de la superfamilia de quinesinas, kif10
EP2504450B1 (en) 2009-11-23 2016-07-06 Swift Biosciences, Inc. Devices to extend single stranded target molecules
SG10201408599QA (en) 2009-11-30 2015-02-27 Genentech Inc Antibodies for treating and diagnosing tumors expressing slc34a2 (tat211 = seqid2 )
WO2011068978A1 (en) 2009-12-02 2011-06-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Methanocarba adenosine derivatives and dendrimer conjugates thereof
US9173895B2 (en) 2009-12-16 2015-11-03 Curna, Inc. Treatment of membrane bound transcription factor peptidase, site 1 (MBTPS1) related diseases by inhibition of natural antisense transcript to MBTPS1
US20110152349A1 (en) 2009-12-18 2011-06-23 Anke Geick Compositions and methods for inhibiting expression of il-18 genes
KR101793753B1 (ko) 2009-12-23 2017-11-03 큐알엔에이, 인크. 커플링방지 단백질 2(ucp2)에 대한 천연 안티센스 전사체의 저해에 의한 ucp2 관련 질환의 치료
KR101891352B1 (ko) 2009-12-23 2018-08-24 큐알엔에이, 인크. 간세포 성장 인자(hgf)에 대한 천연 안티센스 전사체의 억제에 의한 hgf 관련 질환의 치료
RU2615450C2 (ru) 2009-12-29 2017-04-04 Курна, Инк. Лечение заболеваний, связанных с ядерным респираторным фактором 1(nrf1), путем ингибирования природного антисмыслового транскрипта к nrf1
WO2011090741A2 (en) 2009-12-29 2011-07-28 Opko Curna, Llc TREATMENT OF TUMOR PROTEIN 63 (p63) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO p63
US8946181B2 (en) 2010-01-04 2015-02-03 Curna, Inc. Treatment of interferon regulatory factor 8 (IRF8) related diseases by inhibition of natural antisense transcript to IRF8
KR101853509B1 (ko) 2010-01-06 2018-04-30 큐알엔에이, 인크. 췌장 발달 유전자에 대한 천연 안티센스 전사체의 억제에 의한 췌장 발달 유전자와 관련된 질환의 치료
RU2611191C2 (ru) 2010-01-11 2017-02-21 Курна, Инк. Лечение заболеваний, связанных со связывающим половые гормоны глобулином (гспг), путем ингибирования природного антисмыслового транскрипта к гспг
WO2011085102A1 (en) 2010-01-11 2011-07-14 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2524042A2 (en) 2010-01-12 2012-11-21 Yale University Structured rna motifs and compounds and methods for their use
WO2011088391A2 (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
US9574191B2 (en) 2010-02-03 2017-02-21 The Board Of Regents Of The University Of Texas System Selective inhibition of polyglutamine protein expression
WO2011097407A1 (en) 2010-02-04 2011-08-11 Ico Therapeutics Inc. Dosing regimens for treating and preventing ocular disorders using c-raf antisense
US20110196016A1 (en) * 2010-02-05 2011-08-11 Anke Geick 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
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
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
BR112012020373A8 (pt) 2010-02-23 2018-01-02 Genentech Inc anticorpo isolado, célula, ácido nucleico isolado, método de identificação, de inibição da proliferação de uma célula, de tratamento terapêutico, de determinação da presença de uma proteína tat419, de diagnóstico da presença de câncer e de distribuição de agente citotóxico
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
US9193752B2 (en) 2010-03-17 2015-11-24 Isis Pharmaceuticals, Inc. 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
KR101852210B1 (ko) 2010-03-24 2018-04-25 알엑스아이 파마슈티칼스 코포레이션 진피 및 섬유증성 적응증에서의 rna 간섭
EP2550001B1 (en) 2010-03-24 2019-05-22 Phio Pharmaceuticals Corp. Rna interference in ocular indications
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
ES2893199T3 (es) 2010-03-29 2022-02-08 Alnylam Pharmaceuticals Inc Terapia de ARNbc para amiloidosis ocular relacionada con transtiretina (TTR)
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
US20110269194A1 (en) 2010-04-20 2011-11-03 Swift Biosciences, Inc. Materials and methods for nucleic acid fractionation by solid phase entrapment and enzyme-mediated detachment
KR101869570B1 (ko) 2010-04-28 2018-06-20 아이오니스 파마수티컬즈, 인코포레이티드 변형된 뉴클레오사이드 및 그로부터 제조된 올리고머 화합물
EP3091027B1 (en) 2010-04-28 2018-01-17 Ionis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
RS56011B1 (sr) 2010-04-29 2017-09-29 Ionis Pharmaceuticals Inc Modulacija ekspresije transtiretina
CN107090045A (zh) 2010-05-03 2017-08-25 霍夫曼-拉罗奇有限公司 用于肿瘤诊断和治疗的组合物和方法
CN107988228B (zh) 2010-05-03 2022-01-25 库尔纳公司 通过抑制沉默调节蛋白(sirt)的天然反义转录物而治疗沉默调节蛋白(sirt)相关疾病
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
WO2011150226A1 (en) 2010-05-26 2011-12-01 Landers James P Method for detecting nucleic acids based on aggregate formation
KR20180053419A (ko) 2010-05-26 2018-05-21 큐알엔에이, 인크. 무조(無調)의 동소체 1 (atoh1)에 대한 자연 안티센스 전사체의 저해에 의한 atoh1 관련된 질환의 치료
EP3456827A3 (en) 2010-06-02 2019-05-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US9476101B2 (en) 2010-06-07 2016-10-25 Firefly Bioworks, Inc. Scanning multifunctional particles
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
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)
EP2593547B1 (en) 2010-07-14 2017-11-15 CuRNA, Inc. Treatment of discs large homolog (dlg) related diseases by inhibition of natural antisense transcript to dlg
AU2011282243B2 (en) 2010-07-19 2016-09-22 Ionis Pharmaceuticals, Inc. Modulation of nuclear-retained RNA
US20130143955A1 (en) 2010-08-09 2013-06-06 Yale University Cyclic di-GMP-II Riboswitches, Motifs, and Compounds, and Methods for Their Use
DK2625197T3 (en) 2010-10-05 2016-10-03 Genentech Inc Smoothened MUTANT AND METHODS OF USING THE SAME
ES2640755T3 (es) 2010-10-06 2017-11-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la Sialidasa 4 (neu4) mediante inhibición del transcrito antisentido natural al gen neu4
EP2630240A1 (en) 2010-10-18 2013-08-28 Arrowhead Research Corporation Compositions and methods for inhibiting expression of rrm2 genes
RU2597972C2 (ru) 2010-10-22 2016-09-20 Курна Инк. Лечение заболеваний, связанных с геном альфа-l-идуронидазы (idua), путем ингибирования природного антисмыслового транскрипта гена idua
CA2816056A1 (en) 2010-10-27 2012-05-03 Curna, Inc. Treatment of interferon-related developmental regulator 1 (ifrd1) related diseases by inhibition of natural antisense transcript to ifrd1
US9339513B2 (en) 2010-11-09 2016-05-17 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
JP6126009B2 (ja) 2010-11-17 2017-05-10 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. α−シヌクレイン発現の調節
RU2608493C2 (ru) 2010-11-23 2017-01-18 Курна, Инк. Лечение заболеваний, связанных с nanog, путем ингибирования природного антисмыслового транскрипта 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
EP2649182A4 (en) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc Compositions and methods for increasing erythropoietin (epo) production
US9127275B2 (en) 2010-12-10 2015-09-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
WO2012106509A1 (en) 2011-02-02 2012-08-09 The Trustees Of Princeton University Sirtuin modulators as virus production modulators
CN103635197A (zh) 2011-02-02 2014-03-12 埃克斯利尔德生物制药公司 使用靶向结缔组织生长因子(ctgf)的反义化合物治疗疤痕疙瘩或肥大性疤痕的方法
MX342927B (es) 2011-02-03 2016-10-19 Mirna Therapeutics Inc Mimeticos sinteticos de mir-34.
WO2012106586A1 (en) 2011-02-03 2012-08-09 Mirna Therapeutics, Inc. Synthetic mimics of mir-124
EP3467109A1 (en) 2011-02-08 2019-04-10 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
KR102481317B1 (ko) 2011-03-29 2022-12-26 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
WO2012138453A1 (en) 2011-04-03 2012-10-11 The General Hospital Corporation Efficient protein expression in vivo using modified rna (mod-rna)
WO2012149154A1 (en) 2011-04-26 2012-11-01 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
CN103620036B (zh) 2011-06-09 2016-12-21 库尔纳公司 通过抑制共济蛋白(fxn)的天然反义转录物而治疗fxn 相关疾病
WO2012170347A1 (en) 2011-06-09 2012-12-13 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2012170945A2 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
MX345095B (es) 2011-06-21 2017-01-17 Alnylam Pharmaceuticals Inc Composiciones de arni similares a angiopoyetina 3 (angptl3) y metodos para su uso.
WO2012177949A2 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
CN107201364A (zh) 2011-06-21 2017-09-26 阿尔尼拉姆医药品有限公司 用于抑制载脂蛋白c‑iii(apoc3)基因表达的组合物与方法
EP3597750B1 (en) 2011-06-23 2022-05-04 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
AU2012275096B2 (en) 2011-06-29 2016-02-04 Ionis Pharmaceuticals, Inc. Methods for modulating kallikrein (KLKB1) expression
TWI695066B (zh) 2011-06-30 2020-06-01 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
US20140328811A1 (en) 2011-08-01 2014-11-06 Alnylam Pharmaceuticals, Inc. Method for improving the success rate of hematopoietic stem cell transplants
US9752142B2 (en) 2011-08-11 2017-09-05 Ionis Pharmaceuticals, Inc. Gapped oligomeric compounds comprising 5′-modified deoxyribonucleosides in the gap and uses thereof
JP2014526517A (ja) 2011-09-14 2014-10-06 ノースウェスタン ユニバーシティ 血液脳関門を通過することができるナノ抱合体
US9580713B2 (en) 2011-09-17 2017-02-28 Yale University Fluoride-responsive riboswitches, fluoride transporters, and methods of use
CN108373506A (zh) 2011-10-14 2018-08-07 霍夫曼-拉罗奇有限公司 抗HtrA1抗体及使用方法
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
DK2794880T3 (en) 2011-12-22 2018-07-23 Ionis Pharmaceuticals Inc METHOD OF MODULATING METASTASE-ASSOCIATED-IN-LONG-ADENOCARCINOM TRANSCRIPT-1- (MALATE-1) EXPRESSION
CA2860731C (en) 2012-01-10 2023-02-28 M. Mahmood Hussain Method of treating hyperlipidemia and atherosclerosis with mir-30c
EP2812342B1 (en) 2012-02-08 2017-11-15 Ionis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
US9896709B2 (en) 2012-03-13 2018-02-20 Swift Biosciences, Inc. Methods and compositions for size-controlled homopolymer tailing of substrate polynucleotides by a nucleic acid polymerase
CN104583405A (zh) 2012-03-15 2015-04-29 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
CN108949772A (zh) 2012-04-02 2018-12-07 现代泰克斯公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
JP6189415B2 (ja) 2012-04-02 2017-08-30 モデルナティエックス インコーポレイテッドModernaTX,Inc. 細胞質および細胞骨格タンパク質の産生のための修飾ポリヌクレオチド
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
WO2013154798A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US9914922B2 (en) 2012-04-20 2018-03-13 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
EP2852606B1 (en) 2012-05-22 2019-08-07 Ionis Pharmaceuticals, Inc. Modulation of enhancer rna mediated gene expression
EP4458361A3 (en) 2012-06-25 2025-01-22 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
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
CN104736551B (zh) 2012-08-15 2017-07-28 Ionis制药公司 使用改进的封端方案制备寡聚化合物的方法
EP2897633B1 (en) 2012-09-18 2020-01-01 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
US9695418B2 (en) 2012-10-11 2017-07-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
EP4052709A1 (en) 2012-10-11 2022-09-07 Ionis 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
EP2912179A4 (en) 2012-10-26 2016-10-12 Geron Corp C-MYC ANTISENSE OLIGONUCLEOTIDES AND METHOD FOR USE THEREOF FOR TREATING DISORDERS AFFECTING CELL GROWTH
EP4223770A3 (en) 2012-11-05 2023-10-18 Foundation Medicine, Inc. Novel fusion molecules and uses thereof
EP2914621B1 (en) 2012-11-05 2023-06-07 Foundation Medicine, Inc. Novel ntrk1 fusion molecules and uses thereof
EP2922554B1 (en) 2012-11-26 2022-02-23 ModernaTX, Inc. Terminally modified rna
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
JP2016504050A (ja) 2013-01-17 2016-02-12 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. 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド
WO2014113729A2 (en) 2013-01-18 2014-07-24 Foundation Mecicine, Inc. Methods of treating cholangiocarcinoma
US9701708B2 (en) 2013-01-31 2017-07-11 Ionis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified coupling protocols
AU2014216137B2 (en) 2013-02-14 2018-05-10 Ionis Pharmaceuticals, Inc. Modulation of Apolipoprotein C-III (ApoCIII) expression in lipoprotein lipase deficient (LPLD) populations
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
US10398661B2 (en) 2013-02-28 2019-09-03 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
US20160024181A1 (en) 2013-03-13 2016-01-28 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
CN105324485B (zh) 2013-03-14 2021-11-23 阿尔尼拉姆医药品有限公司 补体组分C5 iRNA组合物及其使用方法
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014165825A2 (en) 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
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
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
IL296543B2 (en) 2013-05-01 2025-02-01 Ionis Pharmaceuticals Inc Compositions and methods for modulating HBV and TTR expression
MY181888A (en) 2013-05-22 2021-01-12 Alnylam Pharmaceuticals Inc Tmprss6 irna compositions and methods of use thereof
KR102295648B1 (ko) 2013-05-22 2021-08-31 알닐람 파마슈티칼스 인코포레이티드 SERPINA1 iRNA 조성물 및 이의 사용 방법
US20160113911A1 (en) 2013-06-06 2016-04-28 The General Hospital Corporation Methods and compositions for the treatment of cancer
EP3007704B1 (en) 2013-06-13 2021-01-06 Antisense Therapeutics Ltd Combination therapy for acromegaly
EP3656386A1 (en) 2013-06-21 2020-05-27 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile
KR102236784B1 (ko) 2013-07-02 2021-04-05 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체의 조절인자
SI3019619T1 (sl) 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
HUE048710T2 (hu) 2013-08-08 2020-08-28 Scripps Research Inst Eljárás nukleinsavak in vitro helyspecifikus enzimes jelölésére nem-természetes nukleotidok beépítésével
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
MX2016002587A (es) 2013-08-28 2017-02-20 Ionis Pharmaceuticals Inc Modulacion de la expresion de precalicreina (pkk).
EP3041938A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Circular polynucleotides
JP2016530294A (ja) 2013-09-03 2016-09-29 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. キメラポリヌクレオチド
CN105744959B (zh) 2013-09-13 2020-12-01 Ionis制药公司 补体因子b的调节剂
CA2925107A1 (en) 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
CN105792832B (zh) 2013-10-04 2021-03-23 诺华股份有限公司 用于治疗乙肝病毒的有机化合物
US10227588B2 (en) 2013-10-04 2019-03-12 Novartis Ag 3′end caps for RNAi agents for use in RNA interference
ES2804510T3 (es) 2013-10-04 2021-02-08 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen ALAS1
EP3052627B1 (en) 2013-10-04 2018-08-22 Novartis AG Novel formats for organic compounds for use in rna interference
WO2015054451A1 (en) 2013-10-09 2015-04-16 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
WO2015061246A1 (en) 2013-10-21 2015-04-30 Isis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
JP2016536303A (ja) 2013-10-21 2016-11-24 ザ ジェネラル ホスピタル コーポレイション 末梢循環腫瘍細胞クラスターおよび癌処置に関する方法
WO2015066708A1 (en) 2013-11-04 2015-05-07 Northwestern University Quantification and spatio-temporal tracking of a target using a spherical nucleic acid (sna)
WO2015084897A2 (en) 2013-12-02 2015-06-11 Mirimmune, Llc Immunotherapy of cancer
CN112107693B (zh) 2013-12-03 2023-05-26 西北大学 脂质体颗粒、制备所述脂质体颗粒的方法以及其用途
WO2015085183A2 (en) 2013-12-06 2015-06-11 Swift Biosciences, Inc. Cleavable competitor polynucleotides
CA2844640A1 (en) 2013-12-06 2015-06-06 The University Of British Columbia Method for treatment of castration-resistant prostate cancer
IL314045A (en) 2013-12-12 2024-09-01 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
AU2014364520B2 (en) 2013-12-20 2020-01-02 The General Hospital Corporation Methods and assays relating to circulating tumor cells
AU2014369900B2 (en) 2013-12-24 2021-05-20 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
WO2015120075A2 (en) 2014-02-04 2015-08-13 Genentech, Inc. Mutant smoothened and methods of using the same
JP6594902B2 (ja) 2014-02-11 2019-10-23 アルナイラム ファーマシューティカルズ, インコーポレイテッド ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法
EP3119789B1 (en) * 2014-03-17 2020-04-22 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
CA2942340A1 (en) 2014-03-19 2015-09-24 Ionis Pharmaceuticals, Inc. Compositions for modulating ataxin 2 expression
EP4137573A3 (en) 2014-04-01 2023-05-03 Biogen MA Inc. Compositions for modulating sod-1 expression
TWI638047B (zh) 2014-04-09 2018-10-11 史基普研究協會 藉由核酸三磷酸酯轉運子將非天然或經修飾的核苷三磷酸酯輸入至細胞中
EP3797780B1 (en) 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
US10221416B2 (en) 2014-04-24 2019-03-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
CA2947270A1 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic acids targeting mdm2 or mycn
UA121656C2 (uk) 2014-05-01 2020-07-10 Айоніс Фармасьютикалз, Інк. Олігомерна сполука та спосіб лікування захворювання, пов'язаного з порушенням регуляції альтернативного шляху комплементу
KR20200102553A (ko) 2014-05-01 2020-08-31 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
EP4534092A3 (en) 2014-05-01 2025-07-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating pkk expression
BR122020024443B1 (pt) 2014-05-01 2022-02-22 Ionis Pharmaceuticals, Inc Composto e composição farmacêutica para modulação da expressão de angptl3
EP3811977B1 (en) 2014-05-01 2025-12-03 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療serpinc1相關疾患之方法和組成物
SG10202104570TA (en) 2014-05-22 2021-06-29 Alnylam Pharmaceuticals Inc Angiotensinogen (agt) irna compositions and methods of use thereof
JP2017518307A (ja) 2014-06-02 2017-07-06 チルドレンズ メディカル センター コーポレーション 免疫調節のための方法および組成物
CA2953216C (en) 2014-06-04 2020-12-22 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
EP3155100B1 (en) 2014-06-10 2021-12-22 Erasmus University Medical Center Rotterdam Antisense oligonucleotides useful in treatment of pompe disease
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
CA2955250A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
US9951327B1 (en) 2014-07-17 2018-04-24 Integrated Dna Technologies, Inc. Efficient and rapid method for assembling and cloning double-stranded DNA fragments
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
BR112017001860A2 (pt) 2014-07-31 2018-02-27 Uab Research Foundation peptídeo sintético, composição farmacêutica, métodos, regime de dosagem, e, anticorpo monoclonal
WO2016028940A1 (en) 2014-08-19 2016-02-25 Northwestern University Protein/oligonucleotide core-shell nanoparticle therapeutics
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
CA2959386C (en) 2014-08-29 2024-06-04 Lee Adam Wheeler Methods and compositions for the treatment of cancer
JP6836987B2 (ja) 2014-09-05 2021-03-03 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. Tyrまたはmmp1を標的とする核酸を用いて老化および皮膚障害を処置するための方法
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
JP2017529082A (ja) 2014-09-18 2017-10-05 ザ・ユニバーシティ・オブ・ブリティッシュ・コロンビア ハンチントン病ハプロタイプのための対立遺伝子特異的な療法
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3904519A1 (en) 2014-10-30 2021-11-03 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
KR20170083108A (ko) 2014-11-10 2017-07-17 글락소스미스클라인 인털렉츄얼 프로퍼티 (넘버 2) 리미티드 C형 간염에 대한 장기 작용 약학적 조성물
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
SG11201703646SA (en) 2014-11-10 2017-06-29 Glaxosmithkline Intellectual Property (No 2) Ltd Combination long acting compositions and methods for hepatitis c
HK1244843A1 (zh) 2014-11-17 2018-08-17 Alnylam Pharmaceuticals, Inc. 载脂蛋白c3(apoc3)irna组合物及其使用方法
AU2015349680A1 (en) 2014-11-21 2017-06-08 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
WO2016115490A1 (en) 2015-01-16 2016-07-21 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
EP3256587A2 (en) 2015-02-13 2017-12-20 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
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
MX2017012407A (es) 2015-03-27 2018-03-07 Harvard College Celulas t modificadas y métodos para hacer y usar las mismas.
MX2017012426A (es) 2015-04-03 2018-01-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tmprss6.
US10745702B2 (en) 2015-04-08 2020-08-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
WO2016168592A2 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
EP4365291A3 (en) 2015-06-12 2024-08-14 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3310918B1 (en) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
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
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
EP3919619A1 (en) 2015-07-17 2021-12-08 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
US20180237774A1 (en) 2015-08-04 2018-08-23 Yeda Research And Development Co. Ltd. Methods of screening for riboswitches and attenuators
CN108271387B (zh) 2015-08-07 2023-06-27 箭头药业股份有限公司 乙型肝炎病毒感染的RNAi疗法
KR102866196B1 (ko) 2015-09-02 2025-09-29 알닐람 파마슈티칼스 인코포레이티드 프로그램된 세포사 1 리간드 1 (PD-L1) iRNA 조성물 및 그의 사용 방법
US10533175B2 (en) 2015-09-25 2020-01-14 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating Ataxin 3 expression
KR20240033305A (ko) 2015-10-08 2024-03-12 아이오니스 파마수티컬즈, 인코포레이티드 앤지오텐시노겐 발현 조정을 위한 화합물 및 방법
CA3002744A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
BR112018007703A2 (pt) 2015-10-30 2018-11-06 Genentech Inc anticorpos, ácido nucleico isolado, métodos de produção de um anticorpo e de tratamento de um distúrbio, composição farmacêutica, terapia de combinação e uso do anticorpo
AU2016349954B2 (en) 2015-11-05 2022-08-25 Antisense Therapeutics Ltd Mobilizing leukemia cells
JP7486920B2 (ja) 2015-11-06 2024-05-20 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 アポリポタンパク質(a)発現の調節
WO2017096395A1 (en) 2015-12-04 2017-06-08 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
IL259795B2 (en) 2015-12-07 2024-04-01 Genzyme Corp Methods and compositions for treating a serpinc1-associated disorder
WO2017099579A1 (en) 2015-12-07 2017-06-15 Erasmus University Medical Center Rotterdam Enzymatic replacement therapy and antisense therapy for pompe disease
US11761007B2 (en) 2015-12-18 2023-09-19 The Scripps Research Institute Production of unnatural nucleotides using a CRISPR/Cas9 system
EP3397288A4 (en) 2015-12-31 2019-09-11 Ionis Pharmaceuticals, Inc. PROCESS FOR REDUCING ATAXIN-2 EXPRESSION
EP3400300A4 (en) 2016-01-05 2019-08-07 Ionis Pharmaceuticals, Inc. PROCESS FOR REDUCING LRRK2 EXPRESSION
WO2017132483A1 (en) 2016-01-29 2017-08-03 Vanderbilt University Free-solution response function interferometry
EP3411396A1 (en) 2016-02-04 2018-12-12 Curis, Inc. Mutant smoothened and methods of using the same
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
US20190256845A1 (en) 2016-06-10 2019-08-22 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
EP3471781A4 (en) 2016-06-17 2020-05-06 Ionis Pharmaceuticals, Inc. MODULATION OF GYS1 EXPRESSION
HUE060123T2 (hu) 2016-06-24 2023-01-28 Scripps Research Inst Új nukleozid-trifoszfát-transzporter és alkalmazásai
KR20190031306A (ko) 2016-07-21 2019-03-25 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
NL2017295B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
WO2018039629A2 (en) 2016-08-25 2018-03-01 Northwestern University Micellar spherical nucleic acids from thermoresponsive, traceless templates
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
EP3555292A1 (en) 2016-12-16 2019-10-23 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
US10450565B2 (en) 2017-01-10 2019-10-22 Arrowhead Pharmaceuticals, Inc. Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
WO2018136702A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
US11180756B2 (en) 2017-03-09 2021-11-23 Ionis Pharmaceuticals 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
KR102720270B1 (ko) 2017-04-18 2024-10-23 알닐람 파마슈티칼스 인코포레이티드 B형 간염 바이러스 (hbv)에 감염된 대상체의 치료 방법
CN110945005A (zh) 2017-04-20 2020-03-31 新特纳股份公司 包含三环dna核苷的修饰的寡聚化合物及其用途
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
SG11201912243PA (en) 2017-07-10 2020-01-30 Genzyme Corp Methods and compositions for treating a bleeding event in a subject having hemophilia
AU2018300069C1 (en) 2017-07-11 2025-11-20 Synthorx, Inc. Incorporation of unnatural nucleotides and methods thereof
EP3651774A4 (en) 2017-07-11 2021-07-07 The Scripps Research Institute INTEGRATION OF INNATURAL NUCLEOTIDES AND APPLICATION METHODS IN VIVO
MX2020000387A (es) 2017-07-13 2020-08-17 Univ Northwestern Método general y directo para preparar nanopartículas de estructura organometálica funcionalizadas con oligonucleotidos.
JP7277432B2 (ja) 2017-07-13 2023-05-19 アルナイラム ファーマシューティカルズ, インコーポレイテッド 乳酸脱水素酵素A(LDHA)iRNA組成物及びその使用方法
MX2020001332A (es) 2017-08-03 2020-09-09 Synthorx Inc Conjugados de citoquina para el tratamiento de enfermedades proliferativas e infecciosas.
WO2019036613A1 (en) 2017-08-18 2019-02-21 Ionis Pharmaceuticals, Inc. MODULATION OF THE NOTCH SIGNALING PATHWAY FOR THE TREATMENT OF RESPIRATORY DISORDERS
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
US11999953B2 (en) 2017-09-13 2024-06-04 The Children's Medical Center Corporation Compositions and methods for treating transposon associated diseases
WO2019060442A1 (en) 2017-09-19 2019-03-28 Alnylam Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF TRANSTHYRETIN MEDIATED AMYLOSIS (TTR)
WO2019089922A1 (en) 2017-11-01 2019-05-09 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
EA202091520A1 (ru) 2017-12-18 2020-10-05 Элнилэм Фармасьютикалз, Инк. Композиции на основе irna против бокса-1 высокомобильной группы (hmgb1) и способы их применения
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
IL319501A (en) 2017-12-29 2025-05-01 Scripps Research Inst Unnatural nucleotide compounds and methods of using them
MX2020007369A (es) 2018-01-15 2020-10-28 Ionis Pharmaceuticals Inc Moduladores de la expresion de dnm2.
US20190233816A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
MY203509A (en) 2018-02-26 2024-06-30 Synthorx Inc Il-15 conjugates and uses thereof
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
US11661601B2 (en) 2018-03-22 2023-05-30 Ionis Pharmaceuticals, Inc. Methods for modulating FMR1 expression
CA3095134A1 (en) 2018-03-28 2019-10-03 Board Of Regents, The University Of Texas System Identification of epigenetic alterations in dna isolated from exosomes
AU2019247490A1 (en) 2018-04-06 2020-10-22 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
BR112020018698A2 (pt) 2018-04-11 2021-01-05 Ionis Pharmaceuticals, Inc. Moduladores de expressão de ezh2
US12397013B2 (en) 2018-05-07 2025-08-26 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
JOP20200280A1 (ar) 2018-05-09 2020-11-05 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير الوراثي عن fxi
US11434488B2 (en) 2018-05-09 2022-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ATXN3 expression
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
EP3823725A4 (en) 2018-07-17 2023-05-10 Aronora, Inc. METHODS FOR SAFELY REDUCING THROMBOPOIETIN
EA202190299A1 (ru) 2018-07-25 2021-06-11 Ионис Фармасьютикалз, Инк. Соединения и способы для снижения экспрессии atxn2
EP3837366A1 (en) 2018-08-13 2021-06-23 Alnylam Pharmaceuticals, Inc. Hepatitis b virus (hbv) dsrna agent compositions and methods of use thereof
TW202020157A (zh) 2018-08-16 2020-06-01 美商艾爾妮蘭製藥公司 用於抑制lect2基因表現之組合物及方法
JP7535310B2 (ja) 2018-09-14 2024-08-16 ノースウェスタン ユニバーシティ Dnaとのタンパク質重合のプログラミング
WO2020060986A1 (en) 2018-09-18 2020-03-26 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
US12258620B2 (en) 2018-09-25 2025-03-25 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
BR112021005401A2 (pt) 2018-11-08 2021-06-29 Synthorx, Inc. conjugados de interleucina 10 e usos dos mesmos
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
EP3884053A4 (en) 2018-11-21 2023-02-01 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING PRION EXPRESSION
EP3894559A4 (en) 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. METHODS OF TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS RELATED TO MLH3 ACTIVITY
WO2020118259A1 (en) 2018-12-06 2020-06-11 Northwestern University Protein crystal engineering through dna hybridization interactions
DK3897672T5 (da) 2018-12-20 2024-08-05 Vir Biotechnology Inc Hbv kombinationsbehandling
WO2020132521A1 (en) 2018-12-20 2020-06-25 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
AU2020209186A1 (en) 2019-01-16 2021-09-09 Genzyme Corporation Serpinc1 iRNA compositions and methods of use thereof
CN113395971A (zh) 2019-01-31 2021-09-14 Ionis制药公司 Yap1表达的调节剂
BR112021014415A2 (pt) 2019-02-06 2021-09-21 Synthorx, Inc. Conjugados de il-2 e métodos de uso dos mesmos
WO2020176771A1 (en) 2019-02-27 2020-09-03 Ionis Pharmaceuticals, Inc. Modulators of malat1 expression
EP3941483A4 (en) 2019-03-21 2023-10-18 Arnay Sciences, Llc ANTISEN OLIGONUCLEOTIDES FOR ALLEL SPECIFICITY
CA3198199A1 (en) 2019-03-29 2020-10-08 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for modulating expression of dux4
MX2021011916A (es) 2019-03-29 2021-10-26 Ionis Pharmaceuticals Inc Compuestos y metodos para modular ube3a-ats.
SG11202112240VA (en) 2019-05-13 2021-12-30 Vir Biotechnology Inc Compositions and methods for treating hepatitis b virus (hbv) infection
CA3138915A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
IL288941B2 (en) 2019-06-14 2025-10-01 Scripps Research Inst Reagents and methods for replication, transcription, and translation in semisynthetic organisms
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
US20220305107A1 (en) 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company COMBINATION OF HEPATITIS B VIRUS (HBV) VACCINES AND HBV-TARGETING RNAi
EP4660307A2 (en) 2019-07-26 2025-12-10 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
TWI873169B (zh) 2019-08-15 2025-02-21 美商欣爍克斯公司 使用il-2接合物之免疫腫瘤學組合療法
EP4013767A4 (en) 2019-08-15 2023-10-25 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds and uses thereof
EP4017540A1 (en) 2019-08-23 2022-06-29 Synthorx, Inc. Il-15 conjugates and uses thereof
AU2020343255A1 (en) 2019-09-03 2022-03-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
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
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
US12486512B2 (en) 2019-10-18 2025-12-02 Alnylam Pharmaceuticals, Inc. Solute carrier family member iRNA compositions and methods of use thereof
MX2022004726A (es) 2019-10-22 2022-05-13 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de componente c3 de complemento y metodos de uso de las mismas.
US12378560B2 (en) 2019-10-29 2025-08-05 Northwestern University Sequence multiplicity within spherical nucleic acids
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
MX2022004388A (es) 2019-11-01 2022-05-06 Alnylam Pharmaceuticals Inc Composiciones de agentes de arni de huntingtina (htt) y metodos de uso de las mismas.
IL291786A (en) 2019-11-04 2022-06-01 Synthorx Inc Interleukin 10 conjugates and their use
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
KR20220110749A (ko) 2019-11-06 2022-08-09 알닐람 파마슈티칼스 인코포레이티드 간외 전달
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
MX2022006433A (es) 2019-12-13 2022-06-23 Alnylam Pharmaceuticals Inc Composiciones de agentes de acido ribonucleico de interferencia (arni) del marco de lectura abierto 72 del cromosoma 9 humano (c9orf72) y metodos de uso de los mismos.
WO2021126734A1 (en) 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2021154705A1 (en) 2020-01-27 2021-08-05 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)
AU2021220765A1 (en) 2020-02-10 2022-09-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing VEGF-A expression
AU2021224778A1 (en) 2020-02-18 2022-09-29 Alnylam Pharmaceuticals, Inc. Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
TW202517787A (zh) 2020-02-28 2025-05-01 美商Ionis製藥公司 用於調節smn2之化合物及方法
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
JP2023516095A (ja) 2020-03-06 2023-04-17 アルナイラム ファーマシューティカルズ, インコーポレイテッド ケトヘキソキナーゼ(KHK)iRNA組成物およびその使用方法
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
EP4127168A1 (en) 2020-03-26 2023-02-08 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
WO2021207167A1 (en) 2020-04-06 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
CA3179678A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
WO2021206917A1 (en) 2020-04-07 2021-10-14 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
WO2021222065A1 (en) 2020-04-27 2021-11-04 Alnylam Pharmaceuticals, Inc. Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
IL297680A (en) 2020-04-30 2022-12-01 Alnylam Pharmaceuticals Inc Complement factor b (cfb) irna compositions and methods of use thereof
BR112022021333A2 (pt) 2020-05-01 2022-12-13 Ionis Pharmaceuticals Inc Compostos e métodos para modular atxn1
WO2021230286A1 (ja) 2020-05-12 2021-11-18 田辺三菱製薬株式会社 Ataxin 3発現を調節するための化合物、方法及び医薬組成物
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150087A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
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)
US20230183707A1 (en) 2020-05-21 2023-06-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
WO2021248052A1 (en) 2020-06-05 2021-12-09 The Broad Institute, Inc. Compositions and methods for treating neoplasia
US20230203496A1 (en) 2020-06-09 2023-06-29 Alnylam Pharmaceuticals, Inc. Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
TW202214856A (zh) 2020-06-18 2022-04-16 美商阿尼拉製藥公司 黃嘌呤脫氫酶(XDH)iRNA組成物及其使用之方法
TW202208422A (zh) 2020-06-24 2022-03-01 美商維爾生物科技股份有限公司 經工程化之b型肝炎病毒中和抗體及其用途
KR20230027235A (ko) 2020-06-25 2023-02-27 신톡스, 인크. Il-2 콘쥬게이트 및 항-egfr 항체를 사용한 면역 종양학 병용 요법
CN116096899A (zh) 2020-06-29 2023-05-09 Ionis制药公司 调节plp1的化合物和方法
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
UY39344A (es) 2020-07-28 2022-02-25 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de app
CN116322707A (zh) 2020-08-07 2023-06-23 艾欧尼斯制药公司 用于调节scn2a的化合物和方法
EP4217489A1 (en) 2020-09-24 2023-08-02 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
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
TW202228786A (zh) 2020-10-09 2022-08-01 美商欣爍克斯公司 Il-2接合物的免疫腫瘤學療法
WO2022076853A1 (en) 2020-10-09 2022-04-14 Synthorx, Inc. Immuno oncology combination therapy with il-2 conjugates and pembrolizumab
WO2022087041A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
JP2023547615A (ja) 2020-10-23 2023-11-13 ザ スクリプス リサーチ インスティテュート 非天然ヌクレオチドを含むポリヌクレオチドの逆転写
IL302709A (en) 2020-11-13 2023-07-01 Alnylam Pharmaceuticals Inc Coagulation factor iRNA compositions (F5) and methods of using them
EP4488371A3 (en) 2020-11-18 2025-04-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
AU2021393417A1 (en) 2020-12-01 2023-06-29 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
WO2022133278A2 (en) 2020-12-18 2022-06-23 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor xii
IL303886A (en) 2020-12-23 2023-08-01 Flagship Pioneering Inc Compositions of modified trems and uses thereof
US20240343746A1 (en) 2020-12-31 2024-10-17 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
EP4271695A2 (en) 2020-12-31 2023-11-08 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
WO2022150260A1 (en) 2021-01-05 2022-07-14 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
TW202245843A (zh) 2021-02-12 2022-12-01 美商欣爍克斯公司 Il-2接合物及西米普利單抗(cemiplimab)之皮膚癌組合療法
CA3210763A1 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
TW202302148A (zh) 2021-02-12 2023-01-16 美商欣爍克斯公司 使用il-2接合物和抗pd-1抗體或其抗原結合片段的肺癌組合療法
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
IL305153A (en) 2021-02-26 2023-10-01 Alnylam Pharmaceuticals Inc Ketohexokinase (KHK) iRNA compositions and methods of using them
AU2022231003A1 (en) 2021-03-04 2023-09-14 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
EP4304640A1 (en) 2021-03-12 2024-01-17 Northwestern University Antiviral vaccines using spherical nucleic acids
KR20230162024A (ko) 2021-03-29 2023-11-28 알닐람 파마슈티칼스 인코포레이티드 헌팅틴(HTT) iRNA 제제 조성물 및 이의 사용 방법
EP4314016A1 (en) 2021-03-31 2024-02-07 Entrada Therapeutics, Inc. Cyclic cell penetrating peptides
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
IL307926A (en) 2021-04-26 2023-12-01 Alnylam Pharmaceuticals Inc Transmembrane assemblies, serine 6 ((TMPRSS6 IRNA) and methods of using them
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4334448A1 (en) 2021-05-03 2024-03-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
JP2024518476A (ja) 2021-05-10 2024-05-01 エントラーダ セラピューティクス,インコーポレイティド mRNAスプライシングを調節するための組成物及び方法
US20250051393A1 (en) 2021-05-10 2025-02-13 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
JP2024518068A (ja) 2021-05-10 2024-04-24 エントラーダ セラピューティクス,インコーポレイティド 細胞内治療のための組成物及び方法
EP4341401A1 (en) 2021-05-18 2024-03-27 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
WO2022246023A1 (en) 2021-05-20 2022-11-24 Korro Bio, Inc. Methods and compositions for adar-mediated editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
EP4346903A1 (en) 2021-06-03 2024-04-10 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
BR112023025224A2 (pt) 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Quadro de leitura aberto 72 do cromossomo humano 9 (c9orf72) composições de agente de irna e métodos de uso dos mesmos
WO2022260939A2 (en) 2021-06-08 2022-12-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
US11753644B2 (en) 2021-06-18 2023-09-12 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
EP4367237A2 (en) 2021-07-09 2024-05-15 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
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
KR20240036041A (ko) 2021-07-21 2024-03-19 알닐람 파마슈티칼스 인코포레이티드 대사 장애-연관 표적 유전자 iRNA 조성물 및 이의 사용 방법
IL309905A (en) 2021-07-23 2024-03-01 Alnylam Pharmaceuticals Inc IRNA compositions in β-catenin (CTNNB1) and methods of using them
EP4377458A1 (en) 2021-07-29 2024-06-05 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
JP2024530018A (ja) 2021-08-03 2024-08-14 アルナイラム ファーマシューティカルズ, インコーポレイテッド トランスサイレチン(TTR)iRNA組成物およびその使用方法
EP4381071A1 (en) 2021-08-04 2024-06-12 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing angiotensinogen (agt)
EP4384617A1 (en) 2021-08-13 2024-06-19 Alnylam Pharmaceuticals, Inc. Factor xii (f12) irna compositions and methods of use thereof
MX2024002440A (es) 2021-08-31 2024-03-08 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) del efector b similar al factor de fragmentacion de adn subunidad alfa (dffa) que induce la muerte celular (cideb) y metodos de uso de estas.
WO2023034817A1 (en) 2021-09-01 2023-03-09 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)
CN118159654A (zh) 2021-09-20 2024-06-07 阿尔尼拉姆医药品有限公司 抑制素亚基βE(INHBE)调节剂组合物及其使用方法
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
WO2023056440A1 (en) 2021-10-01 2023-04-06 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
CA3234887A1 (en) 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
AU2022370009A1 (en) 2021-10-22 2024-05-16 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
CA3234636A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
EP4423272A2 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
AU2022391744A1 (en) 2021-11-18 2024-06-27 Cornell University Microrna-dependent mrna switches for tissue-specific mrna-based therapies
WO2023122573A1 (en) 2021-12-20 2023-06-29 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
JP2025516680A (ja) 2022-05-13 2025-05-30 アルナイラム ファーマシューティカルズ, インコーポレイテッド 一本鎖ループオリゴヌクレオチド
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
CN120077130A (zh) 2022-08-18 2025-05-30 阿尔尼拉姆医药品有限公司 通用非靶向sirna组合物及其使用方法
EP4569113A1 (en) 2022-09-15 2025-06-18 Regeneron Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2024064858A2 (en) 2022-09-23 2024-03-28 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing mecp2 expression
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
WO2024168010A2 (en) 2023-02-09 2024-08-15 Alnylam Pharmaceuticals, Inc. Reversir molecules and methods of use thereof
WO2024170778A1 (en) 2023-02-17 2024-08-22 Anjarium Biosciences Ag Methods of making dna molecules and compositions and uses thereof
TW202444349A (zh) 2023-03-20 2024-11-16 美商欣爍克斯公司 使用il-2綴合物之癌症療法
WO2024216155A1 (en) 2023-04-12 2024-10-17 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
CN121285630A (zh) 2023-04-20 2026-01-06 阿达尔克斯制药有限公司 Mapt调节组合物及其使用方法
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024226499A1 (en) 2023-04-24 2024-10-31 The Broad Institute, Inc. Compositions and methods for modifying fertility
AU2024273075A1 (en) 2023-05-12 2025-11-20 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2024249328A2 (en) 2023-05-26 2024-12-05 Adarx Pharmaceuticals, Inc. Sod1-modulating compositions and methods of use thereof
WO2024263694A1 (en) 2023-06-20 2024-12-26 Adarx Pharmaceuticals, Inc. Lrrk2-modulating compositions and methods of use thereof
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025024334A1 (en) 2023-07-21 2025-01-30 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025072672A2 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
WO2025072713A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Polymyxins for delivery of agents to the kidney
WO2025072699A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Aminoglycosides for delivery of agents to the kidney
WO2025076031A2 (en) 2023-10-03 2025-04-10 Alnylam Pharmaceuticals, Inc. Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
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
IL84414A0 (en) * 1986-11-14 1988-04-29 Ciba Geigy Ag N9-cyclopentyl-substituted adenine derivatives
IN166499B (OSRAM) * 1987-02-12 1990-05-19 Takeda Chemical Industries Ltd
JPH04501052A (ja) * 1988-02-26 1992-02-27 ザ・ウスター・フアウンデーシヨン・フオー・バイオメデイカル・リサーチ 外来性オリゴヌクレオチドによるhtlv―3の抑制
DE69034150T2 (de) * 1989-10-24 2005-08-25 Isis Pharmaceuticals, Inc., Carlsbad 2'-Modifizierte Oligonukleotide

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US5610300A (en) 1997-03-11
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US5700920A (en) 1997-12-23
US5461152A (en) 1995-10-24
JPH06100562A (ja) 1994-04-12
CA2099712A1 (en) 1994-01-02

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