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

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D239/00—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24—Heterocyclic 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
    • C07D239/28—Heterocyclic 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
    • C07D239/46—Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/47—One nitrogen atom and one oxygen or sulfur atom, e.g. cytosine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C215/00—Compounds containing amino and hydroxy groups bound to the same carbon skeleton
    • C07C215/42—Compounds 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
    • C07C215/44—Compounds 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C275/00—Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups
    • C07C275/46—Derivatives 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
    • C07C275/48—Y being a hydrogen or a carbon atom
    • C07C275/50—Y being a hydrogen or an acyclic carbon atom
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D239/00—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24—Heterocyclic 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
    • C07D239/28—Heterocyclic 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
    • C07D239/46—Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/48—Two nitrogen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D239/00—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24—Heterocyclic 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
    • C07D239/28—Heterocyclic 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
    • C07D239/46—Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/50—Three nitrogen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D239/00—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24—Heterocyclic 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
    • C07D239/28—Heterocyclic 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
    • C07D239/46—Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/52—Two oxygen atoms
    • C07D239/54—Two oxygen atoms as doubly bound oxygen atoms or as unsubstituted hydroxy radicals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D473/00—Heterocyclic compounds containing purine ring systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F7/00—Compounds containing elements of Groups 4 or 14 of the Periodic Table
    • C07F7/02—Silicon compounds
    • C07F7/08—Compounds having one or more C—Si linkages
    • C07F7/18—Compounds having one or more C—Si linkages as well as one or more C—O—Si linkages
    • C07F7/1804—Compounds having Si-O-C linkages
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/645—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having two nitrogen atoms as the only ring hetero atoms
    • C07F9/6509—Six-membered rings
    • C07F9/6512—Six-membered rings having the nitrogen atoms in positions 1 and 3
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561—Heterocyclic 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
    • C07F9/65616—Heterocyclic 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
    • Y—GENERAL 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
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02P—CLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00—Technologies relating to chemical industry
    • Y02P20/50—Improvements relating to the production of bulk chemicals
    • Y02P20/55—Design 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 (enExample)
EP (1) EP0577558A2 (enExample)
JP (1) JPH06100562A (enExample)
CA (1) CA2099712A1 (enExample)
MX (1) MX9303965A (enExample)

Families Citing this family (790)

* 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
CA2170869C (en) 1993-09-03 1999-09-14 Phillip Dan Cook Amine-derivatized nucleosides and oligonucleosides
DE69406943T2 (de) * 1993-09-24 1998-05-07 Us Health Konformationell festgelegte nukleosidanaloge
JP3769737B2 (ja) * 1994-03-30 2006-04-26 味の素株式会社 シクロプロパン誘導体及びその製造法
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US20040147022A1 (en) * 1996-06-06 2004-07-29 Baker Brenda F. 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations
US20050053976A1 (en) * 1996-06-06 2005-03-10 Baker Brenda F. Chimeric oligomeric compounds and their use in gene modulation
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
US20030044941A1 (en) 1996-06-06 2003-03-06 Crooke Stanley T. Human RNase III and compositions and uses thereof
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US20040171031A1 (en) * 1996-06-06 2004-09-02 Baker Brenda F. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US20040203024A1 (en) * 1996-06-06 2004-10-14 Baker Brenda F. Modified oligonucleotides for use in RNA interference
US20080119427A1 (en) * 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
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
WO1999001579A1 (en) 1997-07-01 1999-01-14 Isis Pharmaceuticals, Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
JP2002507996A (ja) 1997-07-03 2002-03-12 デュポン ファーマシューティカルズ カンパニー 神経学的障害を治療するためのイミダゾピリミジン類およびイミダゾピリジン類
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
AU753270B2 (en) * 1998-05-21 2002-10-10 Isis Pharmaceuticals, Inc. Compositions and methods for topical delivery of oligonucleotides
CA2329130A1 (en) * 1998-05-21 1999-11-25 Isis Pharmaceuticals Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
US6335439B1 (en) 1998-06-11 2002-01-01 Isis Pharmaceuticals, Inc. Method of preparing phosphoramidites
US6124463A (en) * 1998-07-02 2000-09-26 Dupont Pharmaceuticals Benzimidazoles as corticotropin release factor antagonists
US6365589B1 (en) 1998-07-02 2002-04-02 Bristol-Myers Squibb Pharma Company Imidazo-pyridines, -pyridazines, and -triazines as corticotropin releasing 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
US7332275B2 (en) * 1999-10-13 2008-02-19 Sequenom, Inc. Methods for detecting methylated nucleotides
ATE336492T1 (de) 2000-01-14 2006-09-15 Us Gov Health & Human Serv Methonocarbacycloalkylanaloga von nucleosiden
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
WO2001079284A2 (en) * 2000-04-13 2001-10-25 Wight Thomas N Therapeutic compounds and methods for formulating v3, a versican isoform
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
US20060166227A1 (en) * 2000-06-20 2006-07-27 Stephen Kingsmore Protein expression profiling
US6323009B1 (en) * 2000-06-28 2001-11-27 Molecular Staging, Inc. Multiply-primed amplification of nucleic acid sequences
US6958214B2 (en) 2000-07-10 2005-10-25 Sequenom, Inc. Polymorphic kinase anchor proteins and nucleic acids encoding the same
US8568766B2 (en) 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
JP5305553B2 (ja) 2000-10-12 2013-10-02 ユニバーシティー オブ ロチェスター 癌細胞の増殖を阻害する組成物
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
AU2002257067A1 (en) 2001-03-14 2002-09-24 Myriad Genetics, Inc Tsg101-gag interaction and use thereof
US7803915B2 (en) * 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
US20050107595A1 (en) * 2001-06-20 2005-05-19 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
EP1409549A4 (en) 2001-06-20 2007-10-24 Genentech Inc COMPOSITIONS AND METHODS FOR THE DIAGNOSIS AND TREATMENT OF TUMORS
CA2451643C (en) 2001-06-21 2012-11-13 Isis 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
NZ573739A (en) 2001-09-18 2010-07-30 Genentech Inc Compositions and methods for the diagnosis and treatment of tumor, particularly colorectal tumor - TAT201
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
NZ577565A (en) 2001-10-09 2010-10-29 Isis Pharmaceuticals Inc Antisense modulation of insulin-like growth factor binding protein 5 expressions
US20030228319A1 (en) 2002-04-16 2003-12-11 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
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
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
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
EP1575571A4 (en) 2002-01-02 2008-06-25 Genentech Inc COMPOSITIONS AND METHODS FOR DIAGNOSIS AND TREATMENT OF A 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
CA2484676A1 (en) * 2002-05-03 2003-11-13 Sequenom, Inc. Kinase anchor protein muteins, peptides thereof, and related methods
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
AU2003276131A1 (en) * 2002-06-18 2003-12-31 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
EA008940B1 (ru) 2002-09-13 2007-10-26 Репликор, Инк. Антивирусные олигонуклеотиды, не связанные с комплементарностью последовательностей
CA2499770A1 (en) 2002-09-20 2004-04-01 Yale University Riboswitches, methods for their use, and compositions for use with riboswitches.
AU2003278957A1 (en) 2002-09-26 2004-04-23 Amgen, Inc. Modulation of forkhead box o1a expression
EP1560597A4 (en) * 2002-10-29 2007-06-27 Pharmacia Corp DIFFERENTIALLY EXPRESSED CANCER GENES, POLYPEPTIDES CODED THEREIN, AND METHOD OF USE THEREOF
WO2004044138A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Chimeric oligomeric 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
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
AU2003294281B2 (en) 2002-11-13 2010-05-20 Kastle Therapeutics, Llc Antisense modulation of apolipoprotein B expression
SI1569695T1 (sl) 2002-11-13 2013-08-30 Genzyme Corporation Protismiselna modulacija ekspresije apolipoproteina B
WO2004046330A2 (en) 2002-11-15 2004-06-03 Morphotek, Inc. Methods of generating high-production of antibodies from hybridomas created by in vitro immunization
CN100594037C (zh) 2002-11-15 2010-03-17 Musc研究发展基金会 通过补体受体2定向的补体调节剂
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
CA2510587A1 (en) 2002-12-20 2004-07-15 Qiagen Gmbh Nucleic acid amplification
US9487823B2 (en) * 2002-12-20 2016-11-08 Qiagen Gmbh Nucleic acid amplification
US6977153B2 (en) 2002-12-31 2005-12-20 Qiagen Gmbh Rolling circle amplification of RNA
EP1597366B1 (en) 2003-02-11 2012-11-21 Antisense Therapeutics Ltd Modulation of insulin like growth factor i receptor expression
US7002006B2 (en) * 2003-02-12 2006-02-21 Isis Pharmaceuticals, Inc. Protection of nucleosides
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
US20070141570A1 (en) * 2003-03-07 2007-06-21 Sequenom, Inc. Association of polymorphic kinase anchor proteins with cardiac phenotypes and related methods
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
US8043834B2 (en) 2003-03-31 2011-10-25 Qiagen Gmbh Universal reagents for rolling circle amplification and methods of use
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US7399853B2 (en) 2003-04-28 2008-07-15 Isis Pharmaceuticals Modulation of glucagon receptor expression
EP1660512A4 (en) 2003-06-02 2009-12-23 Isis Pharmaceuticals Inc SYNTHESIS OF OLIGONUCLEOTIDES USING ALTERNATIVE SOLVENTS
JP4579911B2 (ja) 2003-06-03 2010-11-10 アイシス・ファーマシューティカルズ・インコーポレイテッド スルビビン発現の調節
ES2905724T3 (es) 2003-06-13 2022-04-11 Alnylam Europe Ag Acido ribonucleico bicatenario con elevada eficacia en un organismo
WO2005044976A2 (en) * 2003-06-20 2005-05-19 Isis Pharmaceuticals, Inc. Oligomeric compounds for use in gene modulation
US7790691B2 (en) * 2003-06-20 2010-09-07 Isis Pharmaceuticals, Inc. Double stranded compositions comprising a 3′-endo modified strand for use in gene modulation
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
US20050053981A1 (en) * 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20070123480A1 (en) * 2003-09-11 2007-05-31 Replicor Inc. Oligonucleotides targeting prion diseases
WO2005027962A1 (en) * 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. 4’-thionucleosides and oligomeric compounds
EP2182063A3 (en) 2003-09-18 2012-07-18 Isis Pharmaceuticals, Inc. Modulation of EIF4E expression
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
NZ576411A (en) 2003-11-17 2010-04-30 Genentech Inc Compositions and methods for the treatment of tumor of hematopoietic origin
WO2005071080A2 (en) 2004-01-20 2005-08-04 Isis Pharmaceuticals, Inc. Modulation of glucocorticoid receptor expression
US7468431B2 (en) * 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
US8778900B2 (en) * 2004-01-22 2014-07-15 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP1 expression
US8569474B2 (en) * 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
WO2005089268A2 (en) 2004-03-15 2005-09-29 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
EP2540734B1 (en) 2004-04-05 2016-03-30 Alnylam Pharmaceuticals, Inc. Process and reagents for oligonucleotide synthesis and purification
US20050260755A1 (en) * 2004-04-06 2005-11-24 Isis Pharmaceuticals, Inc. Sequential delivery of oligomeric compounds
AU2005323437B2 (en) 2004-04-30 2011-10-06 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a C5-modified pyrimidine
US8039588B2 (en) 2004-05-21 2011-10-18 The Uab Research Foundation Variable lymphocyte receptors
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
EP1766052A4 (en) * 2004-06-03 2009-12-16 Isis Pharmaceuticals Inc OLIGOMERIC CHIMERIC COMPOSITIONS WITH BRECH
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
WO2006032143A1 (en) 2004-09-23 2006-03-30 Arc Pharmaceuticals, Inc. Pharmaceutical compositions and methods relating to inhibiting fibrous adhesions or inflammatory disease using low sulphate fucans
JP2008516991A (ja) 2004-10-20 2008-05-22 アンチセンス セラピューティクス リミテッド インテグリンα4発現のアンチセンス調節
WO2006063031A2 (en) 2004-12-06 2006-06-15 Haplomics Allelic variants of human factor viii
WO2006091905A1 (en) * 2005-02-25 2006-08-31 Gilead Sciences, Inc. Bicyclo (3.1.0) hexane derivatives as antiviral compounds
MX2007010922A (es) 2005-03-10 2007-10-12 Genentech Inc Metodos y composiciones para modular la integridad vascular.
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
EP1863908B1 (de) 2005-04-01 2010-11-17 Qiagen GmbH Reverse transkription und amplifikation von rna bei simultaner degradierung von dna
WO2007008300A2 (en) 2005-05-31 2007-01-18 ECOLE POLYTECHNIQUE FéDéRALE DE LAUSANNE 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
WO2006138145A1 (en) 2005-06-14 2006-12-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
WO2007032837A2 (en) 2005-08-11 2007-03-22 The J. Craig Venter Institute Method for in vitro recombination
KR20080036646A (ko) 2005-08-17 2008-04-28 메덱시스 에스.에이. Ck19 발현 측정을 위한 조성물 및 방법
WO2007027894A2 (en) 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Antisense compounds having enhanced anti-microrna activity
ATE494372T1 (de) 2005-08-29 2011-01-15 Regulus Therapeutics Inc Verfahren für mir-122a-modulation
EP1762627A1 (de) 2005-09-09 2007-03-14 Qiagen GmbH Verfahren zur Aktivierung einer Nukleinsäure für eine Polymerase-Reaktion
US8080534B2 (en) 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
EP2395076A1 (en) 2005-10-14 2011-12-14 MUSC Foundation For Research Development Targeting PAX2 for the induction of DEFB1-mediated tumor immunity and cancer therapy
EP2325315B1 (en) 2005-10-28 2014-05-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of huntingtin gene
AU2006311725B2 (en) * 2005-11-04 2011-11-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of NAV1.8 gene
CA2626690A1 (en) 2005-11-09 2007-05-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor v leiden mutant gene
CA2630602A1 (en) 2005-11-21 2007-05-31 Isis Pharmaceuticals, Inc. Modulation of eif4e-bp2 expression
JP2009524411A (ja) 2005-12-21 2009-07-02 イェール ユニバーシティー リボスイッチの調節に関連した方法および組成物
CN103301475B (zh) 2005-12-28 2016-08-03 斯克里普斯研究所 药物组合物和表达载体以及调节基因表达的方法和核酸分子的应用
EP2422819B1 (en) 2006-01-26 2017-03-01 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
DE602007009487D1 (de) * 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
EP2388328A1 (en) 2006-01-27 2011-11-23 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
JP5704741B2 (ja) 2006-03-31 2015-04-22 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. Eg5遺伝子発現の抑制のための組成物および方法
CN101437943A (zh) 2006-05-03 2009-05-20 波罗的科技发展有限公司 牢固结合的碱基-修饰的寡核苷酸和人工核酸酶组合的反义药剂
EP2505646A1 (en) * 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of CRP
DE102006020885A1 (de) * 2006-05-05 2007-11-08 Qiagen Gmbh Einführung von Sequenzelementen in Nukleinsäuren
EP2505647A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of DGAT2
US7666854B2 (en) * 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
CA2651453C (en) * 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
EP2392583A1 (en) 2006-05-19 2011-12-07 Alnylam Europe AG. RNAi modulation of Aha and therapeutic uses thereof
US7888498B2 (en) 2006-05-22 2011-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of IKK-B gene
EP2023938A4 (en) * 2006-05-23 2010-11-10 Isis Pharmaceuticals Inc MODULATION OF CHREBP EXPRESSION
WO2008011473A2 (en) 2006-07-19 2008-01-24 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hbxip
WO2008017081A1 (en) * 2006-08-04 2008-02-07 Isis Pharmaceuticals, Inc. Compositions and methods for the modulation of jnk proteins
EP2061799A4 (en) * 2006-09-11 2010-12-22 Univ Yale METHOD AND COMPOSITIONS FOR USE OF LYSIN RIBOSWITCHES
KR101556798B1 (ko) 2006-10-05 2015-10-01 메사츄세츠 인스티튜트 어브 테크놀로지 고성능 분석을 위한 다중기능성 인코딩된 입자
EP2410053B2 (en) 2006-10-18 2020-07-15 Ionis Pharmaceuticals, Inc. Antisense compounds
WO2008136852A2 (en) 2006-11-01 2008-11-13 University Of Rochester Methods and compositions related to the structure and function of apobec3g
EA200900741A1 (ru) * 2006-11-27 2010-04-30 Айзис Фармасьютикалз, Инк. Способы лечения гиперхолестеринемии
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
JP2010512327A (ja) 2006-12-11 2010-04-22 ユニヴァーシティー オブ ユタ リサーチ ファウンデーション 病的血管形成および脈管透過性の処置用の組成物および方法
US20100129358A1 (en) 2006-12-22 2010-05-27 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
CA2679586A1 (en) * 2007-02-27 2008-10-23 Northwestern University Molecule attachment to nanoparticles
KR20100015757A (ko) 2007-03-22 2010-02-12 예일 유니버시티 택일적 스플라이싱을 조절하는 리보스위치와 관련된 방법 및 조성물
JP5350360B2 (ja) 2007-03-29 2013-11-27 アルナイラム ファーマシューティカルズ, インコーポレイテッド エボラ由来の遺伝子の発現を阻害するための組成物および方法
US20110091377A1 (en) 2007-05-11 2011-04-21 The Johns Hopkins University Biomarkers for melanoma
CN101688251A (zh) * 2007-05-29 2010-03-31 耶鲁大学 与控制可变剪接和rna加工的核糖开关有关的方法和组合物
EP2426219A1 (en) 2007-05-29 2012-03-07 Yale University Riboswitches and methods and compositions for use of and with riboswitches
EP2826863B1 (en) 2007-05-30 2017-08-23 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
US7807372B2 (en) * 2007-06-04 2010-10-05 Northwestern University Screening sequence selectivity of oligonucleotide-binding molecules using nanoparticle based colorimetric assay
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
AU2008272918B2 (en) * 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
EP2188298B1 (en) 2007-08-15 2013-09-18 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
JP2010538005A (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
AU2008296487A1 (en) 2007-08-28 2009-03-12 The Uab Research Foundation Synthetic apolipoprotein E mimicking polypeptides and methods of use
WO2009039466A1 (en) 2007-09-20 2009-03-26 Vanderbilt University Free solution measurement of molecular interactions by backscattering interferometry
WO2009039442A1 (en) * 2007-09-21 2009-03-26 California Institute Of Technology Nfia in glial fate determination, glioma therapy and astrocytoma treatment
MX2010004984A (es) * 2007-11-05 2010-07-29 Baltic Technology Dev Ltd Uso de oligonucleotidos con bases modificadas en hibridacion de acidos nucleicos.
WO2009067243A2 (en) * 2007-11-20 2009-05-28 Isis Pharmaceuticals Inc. Modulation of cd40 expression
WO2009076400A2 (en) 2007-12-10 2009-06-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor vii gene
EP3100718B1 (en) 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
EP2265627A2 (en) 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
EP3643782A1 (en) 2008-02-11 2020-04-29 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
CN105267233B (zh) 2008-03-05 2019-07-26 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因表达的组合物和方法
US8426378B2 (en) * 2008-03-21 2013-04-23 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucelosides and methods for their use
US8846639B2 (en) 2008-04-04 2014-09-30 Isis Pharmaceutical, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
US9290534B2 (en) 2008-04-04 2016-03-22 Ionis Pharmaceuticals, Inc. Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside
AU2009237577B2 (en) 2008-04-18 2014-06-05 Baxter Healthcare Sa Microsphere-based composition for preventing and/or reversing new-onset autoimmune diabetes
US20090274696A1 (en) * 2008-04-29 2009-11-05 Wyeth Methods for treating inflammation
CA2726052A1 (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
KR20110017005A (ko) * 2008-07-15 2011-02-18 에프. 호프만-라 로슈 아게 Tgf-베타 수용체 유전자 발현 억제용 조성물 및 방법
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
AU2009276763B2 (en) 2008-07-29 2015-07-16 The Board Of Regents Of The University Of Texas Sytem Selective inhibition of polyglutamine protein expression
EP2323667A4 (en) * 2008-08-07 2012-07-25 Isis Pharmaceuticals Inc MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES
CN107083386A (zh) 2008-08-25 2017-08-22 埃克斯雷德制药有限公司 阻止结缔组织生长因子的反义核苷酸及其用途
EP2334319B1 (en) 2008-08-25 2017-12-06 Excaliard Pharmaceuticals, Inc. Method for reducing scarring during wound healing using antisense compounds directed to ctgf
EP2690175B1 (en) 2008-09-02 2016-12-28 Alnylam Pharmaceuticals, Inc. Compositions and methods for combined inhibition of mutant EGFR gene and IL-6 expression
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
WO2010036696A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
US8546554B2 (en) 2008-09-25 2013-10-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Serum Amyloid A gene
AU2009303345B2 (en) 2008-10-09 2015-08-20 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
CA2740785C (en) 2008-10-15 2017-06-20 Isis Pharmaceuticals, Inc. Modulation of factor 11 expression
EP2937418B1 (en) 2008-10-20 2017-10-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
EP2358397B1 (en) 2008-10-24 2020-01-01 Ionis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
EP2447274B1 (en) 2008-10-24 2017-10-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds and methods
AU2009315665A1 (en) * 2008-11-17 2010-05-20 Arrowhead Research Corporation Compositions and methods for inhibiting expression of factor VII genes
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
AU2009316286B2 (en) 2008-11-24 2016-05-26 Northwestern University Polyvalent RNA-nanoparticle compositions
JP6099868B2 (ja) 2008-12-04 2017-03-22 クルナ・インコーポレーテッド サーチュイン1(sirt1)に対する天然アンチセンス転写物の抑制によるサーチュイン1関連疾患の治療
KR20110091796A (ko) 2008-12-04 2011-08-12 오피케이오 큐알엔에이, 엘엘씨 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료
CA2745329C (en) 2008-12-04 2022-07-12 Opko Curna, Llc Treatment of erythropoietin (epo) related diseases by inhibition of natural antisense transcript to epo
EP2373382B1 (en) 2008-12-10 2017-02-15 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
AU2010203474B2 (en) * 2009-01-08 2015-11-19 Northwestern University Inhibition of bacterial protein production by polyvalent oligonucleotide modified nanoparticle conjugates
KR101546673B1 (ko) * 2009-01-15 2015-08-25 삼성전자주식회사 전자 사진용 토너 및 그의 제조방법
EP2391343B1 (en) 2009-01-29 2017-03-01 Arbutus Biopharma Corporation Improved lipid formulation for the delivery of nucleic acids
CN102307996A (zh) 2009-02-03 2012-01-04 弗·哈夫曼-拉罗切有限公司 用于抑制ptp1b基因表达的组合物和方法
WO2010090762A1 (en) 2009-02-04 2010-08-12 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
HUE026280T2 (en) 2009-02-12 2016-06-28 Curna Inc Treatment of brain-derived neurotrophic factor (BDNF) -related diseases by inhibition of natural antisense transcripts associated with BDNF \ t
JP6066035B2 (ja) 2009-02-12 2017-01-25 クルナ・インコーポレーテッド グリア細胞由来神経栄養因子(gdnf)関連疾病の、gdnfに対する天然アンチセンス転写物の抑制による治療
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. 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
CA2754043A1 (en) 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
MX2011009751A (es) 2009-03-16 2011-09-29 Opko Curna Llc Tratamiento de enfermedades relacionadas con factor nuclear (eritroide derivado 2) tipo 2 (nrf2) por inhibicion del transcrito antisentido natural a nrf2.
US9708604B2 (en) 2009-03-17 2017-07-18 Curna, Inc. Treatment of delta-like 1 homolog (DLK1) related diseases by inhibition of natural antisense transcript to DLK1
KR20170072367A (ko) 2009-04-15 2017-06-26 노오쓰웨스턴 유니버시티 올리고뉴클레오티드 관능화된 나노입자의 전달
EP3524275A1 (en) 2009-04-22 2019-08-14 Massachusetts Institute Of Technology Innate immune supression enables repeated delivery of long rna molecules
WO2010124231A2 (en) 2009-04-24 2010-10-28 The Board Of Regents Of The University Of Texas System Modulation of gene expression using oligomers that target gene regions downstream of 3' untranslated regions
WO2010127195A2 (en) 2009-05-01 2010-11-04 Curna, Inc. Antisense oligonucleotides of hemoglobins
CA2760706C (en) 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
CA3042927C (en) 2009-05-05 2022-05-17 Arbutus Biopharma Corporation Lipid compositions for the delivery of therapeutic agents
CN102459596B (zh) 2009-05-06 2016-09-07 库尔纳公司 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病
ES2609655T3 (es) 2009-05-06 2017-04-21 Curna, Inc. Tratamiento de enfermedades relacionadas con tristetraprolina (TTP) mediante inhibición de transcrito antisentido natural para TTP
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
JP2012526533A (ja) * 2009-05-15 2012-11-01 エフ.ホフマン−ラ ロシュ アーゲー グルココルチコイドレセプター(gcr)遺伝子の発現を阻害するための組成物及び方法
DK2432881T3 (en) 2009-05-18 2018-02-26 Curna Inc TREATMENT OF REPROGRAMMING FACTOR-RELATED DISEASES BY INHIBITING NATURAL ANTISENSE TRANSCRIPTS TO A REPROGRAMMING FACTOR
KR101703695B1 (ko) 2009-05-22 2017-02-08 큐알엔에이, 인크. 전사 인자 e3(tfe3)에 대한 천연 안티센스 전사체의 억제에 의해 tfe3 및 인슐린 수용체 기질 2(irs2)의 치료
KR101704988B1 (ko) 2009-05-28 2017-02-08 큐알엔에이, 인크. 항바이러스 유전자에 대한 천연 안티센스 전사체의 억제에 의한 항바이러스 유전자 관련된 질환의 치료
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2010148050A2 (en) 2009-06-16 2010-12-23 Curna, Inc. Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
ES2629339T3 (es) 2009-06-16 2017-08-08 Curna, Inc. Tratamiento de enfermedades relacionadas con la paraoxonasa 1 (pon1) por inhibición de transcrito antisentido natural a pon1
KR20220084437A (ko) 2009-06-17 2022-06-21 바이오젠 엠에이 인코포레이티드 대상에게서 smn2 스플라이싱을 조정하기 위한 조성물 및 방법
ES2618894T3 (es) 2009-06-24 2017-06-22 Curna, Inc. Tratamiento de enfermedades relacionadas con el receptor del factor de necrosis tumoral 2 (tnfr2) por inhibición del transcrito natural antisentido para tnfr2
CN102482672B (zh) 2009-06-26 2016-11-09 库尔纳公司 通过抑制唐氏综合征基因的天然反义转录物治疗唐氏综合征基因相关疾病
CA2769665A1 (en) 2009-08-05 2011-02-10 Opko Curna, Llc Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
EP2462153B1 (en) 2009-08-06 2015-07-29 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
CN102482671B (zh) 2009-08-25 2017-12-01 库尔纳公司 通过抑制‘含有iq模体的gtp酶激活蛋白’(iqgap)的天然反义转录物而治疗iqgap相关疾病
JP5887270B2 (ja) 2009-09-02 2016-03-16 ジェネンテック, インコーポレイテッド 突然変異体smoothenedおよびその使用方法
RU2539772C2 (ru) 2009-10-22 2015-01-27 Дженентек, Инк. Способы и композиции для модуляции гепсином стимулирующего макрофаги белка
WO2011056687A2 (en) 2009-10-27 2011-05-12 Swift Biosciences, Inc. Polynucleotide primers and probes
CN102666879B (zh) 2009-10-30 2016-02-24 西北大学 模板化的纳米缀合物
US20110110860A1 (en) 2009-11-02 2011-05-12 The Board Of Regents Of The University Of Texas System Modulation of ldl receptor gene expression with double-stranded rnas targeting the ldl receptor gene promoter
EP2496716A1 (en) 2009-11-03 2012-09-12 University Of Virginia Patent Foundation Versatile, visible method for detecting polymeric analytes
US20110112176A1 (en) 2009-11-09 2011-05-12 John Frederick Boylan Compositions and methods for inhibiting expression of kif10 genes
JP2013511285A (ja) 2009-11-23 2013-04-04 スイフト・バイオサイエンシズ・インコーポレイテツド 一本鎖標的分子を伸長させるデバイス
TWI507524B (zh) 2009-11-30 2015-11-11 建南德克公司 診斷及治療腫瘤之組合物及方法
EP2507241A1 (en) 2009-12-02 2012-10-10 The U.S.A. As Represented By The Secretary, Department Of Health And Human Services Methanocarba adenosine derivatives and dendrimer conjugates thereof
CA2782366A1 (en) 2009-12-16 2011-07-14 Opko Curna, Llc 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
US8940708B2 (en) 2009-12-23 2015-01-27 Curna, Inc. Treatment of hepatocyte growth factor (HGF) related diseases by inhibition of natural antisense transcript to HGF
EP2515947B1 (en) 2009-12-23 2021-10-06 CuRNA, Inc. Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
ES2585829T3 (es) 2009-12-29 2016-10-10 Curna, Inc. Tratamiento de las enfermedades relacionadas con la proteína tumoral 63 (p63) por inhibición de transcripción antisentido natural a p63
US8921334B2 (en) 2009-12-29 2014-12-30 Curna, Inc. Treatment of nuclear respiratory factor 1 (NRF1) related diseases by inhibition of natural antisense transcript to NRF1
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
RU2612161C2 (ru) 2010-01-06 2017-03-02 Курна, Инк. Лечение заболеваний, связанных с геном развития поджелудочной железы, путем ингибирования природного антисмыслового транскрипта к гену развития поджелудочной железы
NO2524039T3 (enExample) 2010-01-11 2018-04-28
US8779118B2 (en) 2010-01-11 2014-07-15 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
US20120321647A1 (en) 2010-01-12 2012-12-20 Yale University Structured rna motifs and compounds and methods for their use
US20110177107A1 (en) * 2010-01-14 2011-07-21 Haplomics, Inc. Predicting and reducing alloimmunogenicity of protein therapeutics
ES2671877T3 (es) 2010-01-25 2018-06-11 Curna, Inc. Tratamiento de enfermedades relacionadas con la RNASA (H1) mediante inhibición del transcrito antisentido natural a RNASA H1
WO2011097388A1 (en) 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
US20130028889A1 (en) 2010-02-04 2013-01-31 Ico Therapeutics Inc. Dosing regimens for treating and preventing ocular disorders using c-raf antisense
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
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
AU2011221226A1 (en) 2010-02-23 2012-08-16 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
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
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
US20130101512A1 (en) 2010-03-12 2013-04-25 Chad A. Mirkin Crosslinked polynucleotide structure
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011119852A1 (en) 2010-03-24 2011-09-29 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
CA2794189C (en) 2010-03-24 2022-01-11 Rxi Pharmaceuticals Corporation Rna interference in dermal and fibrotic indications
US9095504B2 (en) 2010-03-24 2015-08-04 Rxi Pharmaceuticals Corporation RNA interference in ocular indications
US20130281510A1 (en) 2010-03-29 2013-10-24 Kumamoto University siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis
EP3578657B1 (en) 2010-04-06 2024-03-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of cd274/pd-l1 gene
WO2011127337A2 (en) 2010-04-09 2011-10-13 Opko Curna Llc Treatment of fibroblast growth factor 21 (fgf21) related diseases by inhibition of natural antisense transcript to fgf21
WO2011133695A2 (en) 2010-04-20 2011-10-27 Swift Biosciences, Inc. Materials and methods for nucleic acid fractionation by solid phase entrapment and enzyme-mediated detachment
WO2011139702A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
WO2011139699A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
ES2625689T3 (es) 2010-04-29 2017-07-20 Ionis Pharmaceuticals, Inc. Modulación de la expresión de transtiretina
CN102958941A (zh) 2010-05-03 2013-03-06 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
RU2693462C2 (ru) 2010-05-03 2019-07-03 Курна, Инк. Лечение заболеваний, связанных с сиртуином (sirt), путем ингибирования природного антисмыслового транскрипта к сиртуину (sirt)
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
WO2011150005A2 (en) 2010-05-26 2011-12-01 Opko Curna Llc Treatment of atonal homolog 1 (atoh1) related diseases by inhibition of natural antisense transcript to atoh1
WO2011150226A1 (en) 2010-05-26 2011-12-01 Landers James P Method for detecting nucleic acids based on aggregate formation
EP3456827A3 (en) 2010-06-02 2019-05-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
US9476101B2 (en) 2010-06-07 2016-10-25 Firefly Bioworks, Inc. Scanning multifunctional particles
US8846637B2 (en) 2010-06-08 2014-09-30 Isis Pharmaceuticals, Inc. Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011156713A1 (en) 2010-06-11 2011-12-15 Vanderbilt University Multiplexed interferometric detection system and method
US9168297B2 (en) 2010-06-23 2015-10-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Regulation of skin pigmentation by neuregulin-1 (NRG-1)
CN103068982B (zh) 2010-07-14 2017-06-09 库尔纳公司 通过抑制盘状大同系物(dlg)的天然反义转录物而治疗dlg相关疾病
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
WO2012021554A1 (en) 2010-08-09 2012-02-16 Yale University Cyclic di-gmp-ii riboswitches, motifs, and compounds, and methods for their use
US8481680B2 (en) 2010-10-05 2013-07-09 Genentech, Inc. Mutant smoothened and methods of using the same
US8993533B2 (en) 2010-10-06 2015-03-31 Curna, Inc. Treatment of sialidase 4 (NEU4) related diseases by inhibition of natural antisense transcript to NEU4
EP3434772A3 (en) 2010-10-18 2019-03-20 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of rrm2 genes
CN103180445B (zh) 2010-10-22 2018-02-16 库尔纳公司 通过抑制α‑L‑艾杜糖醛酸酶(IDUA)的天然反义转录物而治疗IDUA相关疾病
EP2633052B1 (en) 2010-10-27 2018-04-11 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
EP3521451A1 (en) 2010-11-17 2019-08-07 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
US8987225B2 (en) 2010-11-23 2015-03-24 Curna, Inc. Treatment of NANOG related diseases by inhibition of natural antisense transcript to 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
WO2012079046A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
DK2670411T3 (da) 2011-02-02 2019-05-13 Excaliard Pharmaceuticals Inc Antisense-forbindelser rettet mod bindevævsvækstfaktor (CTGF) til anvendelse i en fremgangsmåde til behandling af keloider eller hypertrofiske ar
CN103391777A (zh) 2011-02-02 2013-11-13 普林斯顿大学理事会 作为病毒产生调节剂的去乙酰化酶调节剂
AU2012212023B9 (en) 2011-02-03 2017-01-12 Mirna Therapeutics, Inc. Synthetic mimics of miR-34
US9222085B2 (en) 2011-02-03 2015-12-29 Mirna Therapeutics, Inc. Synthetic mimics of MIR-124
US10017764B2 (en) 2011-02-08 2018-07-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US9562853B2 (en) 2011-02-22 2017-02-07 Vanderbilt University Nonaqueous backscattering interferometric methods
KR102104401B1 (ko) 2011-03-29 2020-04-27 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
US10086043B2 (en) 2011-04-03 2018-10-02 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
WO2012170771A1 (en) 2011-06-09 2012-12-13 Curna, Inc. Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn
WO2012170347A1 (en) 2011-06-09 2012-12-13 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2012170945A2 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
EP2723351B1 (en) 2011-06-21 2018-02-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
CA2839573A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
EP2723756B1 (en) 2011-06-21 2020-03-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
US20140235693A1 (en) 2011-06-23 2014-08-21 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
PT2726613T (pt) 2011-06-30 2018-10-26 Arrowhead Pharmaceuticals Inc Composições e métodos para inibição da expressão de genes do vírus da hepatite b
JP2014526887A (ja) 2011-08-01 2014-10-09 アルナイラム ファーマシューティカルズ, インコーポレイテッド 造血幹細胞移植の成功率を改善する方法
US20140303235A1 (en) 2011-08-11 2014-10-09 Isis Pharmaceuticals, Inc. Linkage modified gapped oligomeric compounds and uses thereof
CN104023749B (zh) 2011-09-14 2019-12-17 西北大学 能穿过血脑屏障的纳米缀合物
WO2013040548A2 (en) 2011-09-17 2013-03-21 Yale University Fluoride-responsive riboswitchs, fluoride transporters, and methods of use
PE20141562A1 (es) 2011-10-14 2014-11-12 Genentech Inc Anticuerpos anti-htra1 y metodos de uso
WO2013059740A1 (en) 2011-10-21 2013-04-25 Foundation Medicine, Inc. Novel alk and ntrk1 fusion molecules and uses thereof
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
ES2673960T3 (es) 2011-12-22 2018-06-26 Ionis Pharmaceuticals, Inc. Métodos para modular la expresión de un transcrito-1 de adenocarcinoma en pulmón asociado a metástasis (MALAT-1)
WO2013106358A1 (en) 2012-01-10 2013-07-18 Hussain M Mahmood Method of treating hyperlipidemia and atherosclerosis with mir-30c
EP3330278A1 (en) 2012-02-08 2018-06-06 Ionis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
LT2825672T (lt) 2012-03-13 2019-06-10 Swift Biosciences, Inc. Būdai ir kompozicijos, skirti valdomam pagal dydį substrato polinukleotidų homopolimeriniam uodegų formavimui su nukleorūgščių polimeraze
ES2694592T3 (es) 2012-03-15 2018-12-21 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado del cerebro (BDNF) por inhibición del transcrito antisentido natural de BDNF
US9610362B2 (en) 2012-03-16 2017-04-04 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of DMPK
US9340784B2 (en) 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
CN104411338A (zh) 2012-04-02 2015-03-11 现代治疗公司 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
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP3336189A1 (en) 2012-04-20 2018-06-20 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
WO2013177248A2 (en) 2012-05-22 2013-11-28 Isis Pharmaceuticals, Inc. Modulation of enhancer rna mediated gene expression
ES2809199T3 (es) 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
US20140038182A1 (en) 2012-07-17 2014-02-06 Dna Logix, Inc. Cooperative primers, probes, and applications thereof
EP3693460A1 (en) 2012-07-27 2020-08-12 Ionis Pharmaceuticals, Inc. Modulation of renin-angiotensin system (ras) related diseases by angiotensinogen
WO2014028739A1 (en) 2012-08-15 2014-02-20 Isis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified capping protocols
WO2014045126A2 (en) 2012-09-18 2014-03-27 Uti Limited Partnership Treatment of pain by inhibition of usp5 de-ubiquitinase
WO2014059364A1 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Methods of treating kennedy's disease
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
US9695418B2 (en) 2012-10-11 2017-07-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
HK1214293A1 (zh) 2012-10-26 2016-07-22 Geron Corporation C-myc反义寡核苷酸和利用其治疗细胞增殖性疾病的方法
EP2914621B1 (en) 2012-11-05 2023-06-07 Foundation Medicine, Inc. Novel ntrk1 fusion molecules and uses thereof
EP4223770A3 (en) 2012-11-05 2023-10-18 Foundation Medicine, Inc. Novel fusion molecules and uses thereof
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
EP3434774A1 (en) 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
CA2898326C (en) 2013-01-18 2022-05-17 Foundation Medicine, Inc. Methods of treating cholangiocarcinoma
DK2951191T3 (en) 2013-01-31 2019-01-14 Ionis Pharmaceuticals Inc PROCEDURE FOR MANUFACTURING OLIGOMERIC COMPOUNDS USING MODIFIED CLUTCH PROTOCOLS
KR20200123263A (ko) 2013-02-14 2020-10-28 아이오니스 파마수티컬즈, 인코포레이티드 지질단백질 리파제 결핍 (lpld) 모집단에서 아포지질단백질 c-iii (apociii) 발현의 조절
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
EP2961853B1 (en) 2013-02-28 2018-09-19 The Board of Regents of The University of Texas System Methods for classifying a cancer as susceptible to tmepai-directed therapies and treating such cancers
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
PT2970974T (pt) 2013-03-14 2017-11-29 Alnylam Pharmaceuticals Inc Composições de irna de componente do complemento c5 e métodos para a sua utilização
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP4286517A3 (en) 2013-04-04 2024-03-13 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
WO2014172698A1 (en) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
TWI664187B (zh) 2013-05-22 2019-07-01 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
AU2014268529C1 (en) 2013-05-22 2020-10-01 Alnylam Pharmaceuticals, Inc. TMPRSS6 iRNA compositions and methods of use thereof
US20160113911A1 (en) 2013-06-06 2016-04-28 The General Hospital Corporation Methods and compositions for the treatment of cancer
DK3007704T3 (da) 2013-06-13 2021-03-29 Antisense Therapeutics Ltd Kombinationsterapi til akromegali
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
JP6487913B2 (ja) 2013-07-02 2019-03-20 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Ionis Pharmaceuticals,Inc. 成長ホルモン受容体のモジュレータ
PL3019619T3 (pl) 2013-07-11 2022-01-10 Modernatx, Inc. Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania
AU2014306271A1 (en) 2013-08-08 2016-03-24 The Scripps Research Institute A method for the site-specific enzymatic labelling of nucleic acids in vitro by incorporation of unnatural nucleotides
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
US9670492B2 (en) 2013-08-28 2017-06-06 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (PKK) expression
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
PL3043827T3 (pl) 2013-09-13 2020-03-31 Ionis Pharmaceuticals, Inc. Modulatory czynnika b dopełniacza
MX385871B (es) 2013-10-02 2025-03-18 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen lect2.
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
WO2015051318A1 (en) 2013-10-04 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
WO2015050871A2 (en) 2013-10-04 2015-04-09 Novartis Ag Organic compounds to treat hepatitis b virus
EP3722277A3 (en) 2013-10-04 2021-01-20 Novartis AG 3'end caps for rna-interferring agents for use in rna
WO2015051366A2 (en) 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
EP3055426B1 (en) 2013-10-09 2019-06-19 The United States of America as represented by The Secretary Department of Health and Human Services Detection of hepatitis delta virus (hdv) for the diagnosis and treatment of sjögren's syndrome and lymphoma
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
US9758546B2 (en) 2013-10-21 2017-09-12 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
EP3502270B1 (en) 2013-10-21 2020-03-18 The General Hospital Corporation Methods relating to circulating tumor cell clusters and the treatment of cancer
US10301622B2 (en) 2013-11-04 2019-05-28 Northwestern University Quantification and spatio-temporal tracking of a target using a spherical nucleic acid (SNA)
JP6772062B2 (ja) 2013-12-02 2020-10-21 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. 癌の免疫療法
WO2015126502A2 (en) 2013-12-03 2015-08-27 Northwestern University Liposomal particles, methods of making same and uses thereof
US10385388B2 (en) 2013-12-06 2019-08-20 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
WO2015089368A2 (en) 2013-12-12 2015-06-18 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
PL3087183T3 (pl) 2013-12-24 2021-01-11 Ionis Pharmaceuticals, Inc. Modulacja ekspresji białka angiopoetynopodobnego 3
US20170044232A1 (en) 2014-02-04 2017-02-16 Genentech, Inc. Mutant smoothened and methods of using the same
EP3105331B1 (en) 2014-02-11 2021-06-23 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
EP3119789B1 (en) * 2014-03-17 2020-04-22 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
SI3119888T1 (sl) 2014-03-19 2021-11-30 Ionis Pharmaceuticals, Inc. Sestavki za moduliranje izražanja ataksina 2
HUE050704T2 (hu) 2014-04-01 2020-12-28 Biogen Ma Inc Összetételek a SOD-1 expressziójának modulálására
EP3129493B1 (en) 2014-04-09 2021-07-07 The Scripps Research Institute Import of unnatural or modified nucleoside triphosphates into cells via nucleic acid triphosphate transporters
US20170044538A1 (en) 2014-04-17 2017-02-16 Biogen Ma Inc. Compositions and Methods for Modulation of SMN2 Splicing in a Subject
WO2015164693A1 (en) 2014-04-24 2015-10-29 Isis 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
WO2015168589A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
WO2015168618A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
CR20160554A (es) 2014-05-01 2017-03-07 Ionis Pharmaceuticals Inc Composiciones y métodos para modular la expresión del factor b del complemento
EP3137115B1 (en) 2014-05-01 2020-10-14 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
HUE052709T2 (hu) 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療serpinc1相關疾患之方法和組成物
SG11201609376SA (en) 2014-05-22 2016-12-29 Alnylam Pharmaceuticals Inc Angiotensinogen (agt) irna compositions and methods of use thereof
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
AU2015269412B2 (en) 2014-06-04 2020-03-12 Exicure Operating Company Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
EP4039807A1 (en) 2014-06-10 2022-08-10 Erasmus University Rotterdam Medical Center 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
WO2016011226A1 (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
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
JP7081923B2 (ja) 2014-07-31 2022-06-07 ユーエイビー リサーチ ファンデーション アポe模倣ペプチド及び血漿コレステロールを取り除くためのより高い効果
EP3183007B1 (en) 2014-08-19 2020-06-17 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
KR102410072B1 (ko) 2014-08-29 2022-06-20 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증의 치료 방법
US10385343B2 (en) 2014-08-29 2019-08-20 Children's Medical Center Corporation Methods and compositions for the treatment of cancer
EP3188799B1 (en) 2014-09-05 2022-07-06 Phio Pharmaceuticals Corp. Methods for treating aging and skin disorders using nucleic acids targeting tyr or mmp1
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
WO2016040589A1 (en) 2014-09-12 2016-03-17 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
US10533172B2 (en) 2014-09-18 2020-01-14 The University Of British Columbia Allele-specific therapy for huntington disease haplotypes
TWI864340B (zh) 2014-10-10 2024-12-01 美商艾爾妮蘭製藥公司 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法
WO2016061487A1 (en) 2014-10-17 2016-04-21 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
TWI769565B (zh) 2014-11-10 2022-07-01 美商阿尼拉製藥公司 B型肝炎病毒(HBV)iRNA組成物及其用途方法
US10308937B2 (en) 2014-11-10 2019-06-04 Glaxosmithkline Intellectual Property (No. 2) Limited Combination long acting compositions and methods for Hepatitis C
MA40844A (fr) 2014-11-10 2017-09-20 Glaxosmithkline Ip No 2 Ltd Compositions pharmaceutiques à action prolongée pour l'hépatite c
WO2016081444A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
WO2016081911A2 (en) 2014-11-21 2016-05-26 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
US20170369872A1 (en) 2014-12-18 2017-12-28 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
WO2016118812A1 (en) 2015-01-23 2016-07-28 Vanderbilt University A robust interferometer and methods of using same
JP2018510621A (ja) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. パタチン様ホスホリパーゼドメイン含有3(PNPLA3)iRNA組成物およびその使用方法
US10450342B2 (en) 2015-02-23 2019-10-22 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
MX391217B (es) 2015-02-26 2025-03-21 Ionis Pharmaceuticals Inc Moduladores de rodopsina p23h con especificidad de alelo.
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
BR112017020750A2 (en) 2015-03-27 2018-06-26 President And Fellows Of Harvard College modified t-cells and methods of producing and using them
CN107429250B (zh) 2015-04-03 2022-03-01 Ionis制药公司 用于调节tmprss6表达的化合物和方法
WO2016164746A1 (en) 2015-04-08 2016-10-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016168592A2 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
EP3307316A1 (en) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde 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
US11001845B2 (en) 2015-07-06 2021-05-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (SOD1)
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
CA3205381A1 (en) 2015-07-17 2017-01-26 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
WO2017021961A1 (en) 2015-08-04 2017-02-09 Yeda Research And Development Co. Ltd. Methods of screening for riboswitches and attenuators
CN115957337A (zh) 2015-08-07 2023-04-14 箭头药业股份有限公司 乙型肝炎病毒感染的RNAi疗法
US10889813B2 (en) 2015-09-02 2021-01-12 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof
WO2017053781A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
MX392014B (es) 2015-10-08 2025-03-21 Ionis Pharmaceuticals Inc Composiciones y métodos para modular la expresión de angiotensinógeno.
US11021707B2 (en) 2015-10-19 2021-06-01 Phio Pharmaceuticals Corp. 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
EP3370734B1 (en) 2015-11-05 2023-01-04 Children's Hospital Los Angeles Antisense oligo for use in treating acute myeloid leukemia
AU2016349625B2 (en) 2015-11-06 2022-07-07 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
EP3389670A4 (en) 2015-12-04 2020-01-08 Ionis Pharmaceuticals, Inc. METHODS OF TREATING BREAST CANCER
US10993995B2 (en) 2015-12-07 2021-05-04 Erasmus University Medical Center Rotterdam Enzymatic replacement therapy and antisense therapy for pompe disease
IL308717A (en) 2015-12-07 2024-01-01 Genzyme Corp Methods and compositions for treating a serpinc1-associated disorder
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
WO2017117496A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
EP3400300A4 (en) 2016-01-05 2019-08-07 Ionis Pharmaceuticals, Inc. PROCESS FOR REDUCING LRRK2 EXPRESSION
EP3408649B1 (en) 2016-01-29 2023-06-14 Vanderbilt University Free-solution response function interferometry
AU2017213826A1 (en) 2016-02-04 2018-08-23 Curis, Inc. Mutant smoothened and methods of using the same
CA3013799A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Methods of modulating keap1
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
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
EP3469083A1 (en) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
AU2017286811A1 (en) 2016-06-17 2018-11-22 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
EP3475295B1 (en) 2016-06-24 2022-08-10 The Scripps Research Institute Novel nucleoside triphosphate transporter and uses thereof
US20190247436A1 (en) 2016-07-21 2019-08-15 Maxcyte, Inc. Methods and compositions for modifying genomic dna
JOP20190015A1 (ar) 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
NL2017295B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
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
TW202313978A (zh) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
US11033570B2 (en) 2016-12-02 2021-06-15 Cold Spring Harbor Laboratory Modulation of Lnc05 expression
TWI865997B (zh) 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白(TTR)iRNA組成物於治療或預防TTR相關疾病之方法
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
CA3049980A1 (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
WO2018195165A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
JP7167059B2 (ja) 2017-04-19 2022-11-08 フィオ ファーマシューティカルズ コーポレーション 核酸化合物の局所送達
CA3059321A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
US11060094B2 (en) 2017-05-19 2021-07-13 Band Therapeutics, Llc Compositions and methods for the treatment of complications and disorders relating to Von Willebrand Factor
JP2020530442A (ja) 2017-07-10 2020-10-22 ジェンザイム・コーポレーション 血友病を有する対象の出血事象を処置するための方法および組成物
WO2019014262A1 (en) 2017-07-11 2019-01-17 The Scripps Research Institute INCORPORATION OF NON-NATURAL NUCLEOTIDES AND METHODS OF IN VIVO USE THEREOF
TWI821192B (zh) 2017-07-11 2023-11-11 美商新索思股份有限公司 非天然核苷酸之導入及其方法
AU2018301829B2 (en) 2017-07-13 2024-08-01 Alnylam Pharmaceuticals, Inc. Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase) gene expression
JP2020526558A (ja) 2017-07-13 2020-08-31 ノースウェスタン ユニバーシティ オリゴヌクレオチド官能化金属有機構造体ナノ粒子を調製するための一般的かつ直接的な方法
CA3069868A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (ldha) irna compositions and methods of use thereof
IL321812A (en) 2017-08-03 2025-08-01 Synthorx Inc Cytokine complexes for the treatment of autoimmune diseases
CA3071033A1 (en) 2017-08-18 2019-02-21 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
US10517889B2 (en) 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
WO2019055460A1 (en) 2017-09-13 2019-03-21 The Children's Medical Center Corporation COMPOSITIONS AND METHODS FOR THE TREATMENT OF TRANSPOSON-ASSOCIATED DISEASES
EP3684931A1 (en) 2017-09-19 2020-07-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
CA3078971A1 (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
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
EP3714053B1 (en) 2017-11-22 2025-12-31 The University of Chicago CHEMICALLY DEPENDENT PROBE EVALUATION OF PROTEIN ACTIVITY AND ITS USES
WO2019126097A1 (en) 2017-12-18 2019-06-27 Alnylam Pharmaceuticals, Inc. High mobility group box-1 (hmgb1) irna compositions and methods of use thereof
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
JP7429642B2 (ja) 2017-12-29 2024-02-08 ザ スクリプス リサーチ インスティテュート 非天然塩基対組成物および使用の方法
CA3088522A1 (en) 2018-01-15 2019-07-18 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
US20190233816A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
AU2019224159B2 (en) 2018-02-26 2025-05-29 Sanofi 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
WO2019183440A1 (en) 2018-03-22 2019-09-26 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
EP3772928B1 (en) 2018-04-06 2025-02-26 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
WO2019200172A1 (en) 2018-04-11 2019-10-17 Ionis Pharmaceuticals, Inc. Modulators of ezh2 expression
CA3098623A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
JP2021522800A (ja) 2018-05-09 2021-09-02 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. Atxn3発現を低減するための化合物及び方法
IL277970B2 (en) 2018-05-09 2025-03-01 Ionis Pharmaceuticals Inc Oligomeric compounds comprising modified oligonucleotides and conjugated groups for use in the treatment of end-stage renal disease, chronic kidney disease and coronary artery disease
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
CA3103429A1 (en) 2018-06-14 2019-12-19 Don W. Cleveland 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
WO2020018558A1 (en) 2018-07-17 2020-01-23 Aronora, Inc. Methods for safely reducing thrombopoietin
AU2019310097A1 (en) 2018-07-25 2021-02-04 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ATXN2 expression
WO2020036862A1 (en) 2018-08-13 2020-02-20 Alnylam Pharmaceuticals, Inc. HEPATITIS B VIRUS (HBV) dsRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
AR115960A1 (es) 2018-08-16 2021-03-17 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen lect2
CN112912422A (zh) 2018-09-14 2021-06-04 西北大学 用dna编程蛋白质聚合
JP2022500003A (ja) 2018-09-18 2022-01-04 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. ケトヘキソキナーゼ(KHK)iRNA組成物およびその使用方法
WO2020068983A1 (en) 2018-09-25 2020-04-02 Co-Diagnostics, Inc. Allele-specific design of cooperative primers for improved nucleic acid variant genotyping
CA3114396A1 (en) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
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
TW202031284A (zh) 2018-11-08 2020-09-01 美商新索思股份有限公司 介白素10結合物及其用途
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
MX2021005758A (es) 2018-11-15 2021-09-21 Ionis Pharmaceuticals Inc Moduladores de la expresión de irf5.
US12281305B2 (en) 2018-11-21 2025-04-22 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
JP2022515744A (ja) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ, インコーポレイテッド Kcnt1関連障害の治療のための組成物及び方法
PT4285929T (pt) 2018-12-20 2026-01-02 Humabs Biomed Sa Terapêutica de combinação contra o vhb
CA3126933A1 (en) 2019-01-16 2020-07-23 Genzyme Corporation Serpinc1 irna compositions and methods of use thereof
TW202214854A (zh) 2019-01-31 2022-04-16 美商Ionis製藥公司 Yap1表現之調節劑
MA54952A (fr) 2019-02-06 2022-05-11 Synthorx Inc Conjugués d'il-2 et méthodes d'utilisation de ceux-ci
WO2020176771A1 (en) 2019-02-27 2020-09-03 Ionis Pharmaceuticals, Inc. Modulators of malat1 expression
US12702719B2 (en) 2019-03-06 2026-08-11 Northwestern University Hairpin-like oligonucleotide-conjugated spherical nucleic acid
WO2020191177A1 (en) 2019-03-21 2020-09-24 Sudhir Agrawal Antisense oligonucleotides for allele specificity
TW202536179A (zh) 2019-03-29 2025-09-16 美商Ionis製藥公司 用於調節ube3a-ats之化合物及方法
TW202104595A (zh) 2019-03-29 2021-02-01 日商田邊三菱製藥股份有限公司 用來調節dux4的表現之化合物、方法及醫藥組成物
WO2020232024A1 (en) 2019-05-13 2020-11-19 Vir Biotechnology, Inc. Compositions and methods for treating hepatitis b virus (hbv) infection
WO2020236600A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
WO2020243021A2 (en) 2019-05-24 2020-12-03 Foundation Medicine, Inc. Ntrk fusion molecules and uses thereof
AU2020286462B2 (en) 2019-06-06 2026-07-02 Arrowhead Pharmaceuticals, Inc. Methods for the treatment of alpha-1 antitrypsin deficiency (AATD)
WO2020252262A1 (en) 2019-06-14 2020-12-17 The Scripps Research Institute Reagents and methods for replication, transcription, and translation in semi-synthetic 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
WO2020255008A1 (en) 2019-06-18 2020-12-24 Janssen Sciences Ireland Unlimited Company COMBINATION OF HEPATITIS B VIRUS (HBV) VACCINES AND HBV-TARGETING RNAi
PL3956450T3 (pl) 2019-07-26 2025-12-22 Ionis Pharmaceuticals, Inc. Związki i sposoby modulowania gfap
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
KR20220062517A (ko) 2019-08-15 2022-05-17 아이오니스 파마수티컬즈, 인코포레이티드 결합 변형된 올리고머 화합물 및 이의 용도
EP4013454A1 (en) 2019-08-15 2022-06-22 Synthorx, Inc. Immuno oncology combination therapies with il-2 conjugates
CA3148135A1 (en) 2019-08-23 2021-03-04 Carolina E. CAFFARO Il-15 conjugates and uses thereof
US20220290152A1 (en) 2019-09-03 2022-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
WO2021050554A1 (en) 2019-09-10 2021-03-18 Synthorx, Inc. Il-2 conjugates and methods of use to treat autoimmune diseases
US12319711B2 (en) 2019-09-20 2025-06-03 Northwestern University Spherical nucleic acids with tailored and active protein coronae
US12503699B2 (en) 2019-10-04 2025-12-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing UGT1a1 gene expression
EP4045652A1 (en) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
CA3158320A1 (en) 2019-10-22 2021-04-29 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
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
WO2021087036A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
JP2022554272A (ja) 2019-11-04 2022-12-28 シンソークス, インコーポレイテッド インターロイキン10コンジュゲートおよびその使用
WO2021092371A2 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
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
EP4061945A1 (en) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
IL293824A (en) 2019-12-13 2022-08-01 Alnylam Pharmaceuticals Inc Compositions of RNA material (c9orf72) human chromosome 9 open reading frame 72 and methods of using them
CN115176007A (zh) 2019-12-16 2022-10-11 阿尔尼拉姆医药品有限公司 含类PATATIN磷脂酶结构域3(PNPLA3)iRNA组合物及其使用方法
EP4058032A4 (en) 2019-12-19 2024-01-10 Entrada Therapeutics, Inc. Compositions for delivery of antisense compounds
US12503695B2 (en) 2020-01-27 2025-12-23 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services RAB13 and NET1 antisense oligonucleotides to treat metastatic cancer
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
PH12022552080A1 (en) 2020-02-10 2024-02-12 Alnylam Pharmaceuticals Inc Compositions and methods for silencing vegf-a expression
CN115397989A (zh) 2020-02-18 2022-11-25 阿尔尼拉姆医药品有限公司 载脂蛋白C3(APOC3)iRNA组合物及其使用方法
AU2021225957A1 (en) 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
MX2022011009A (es) 2020-03-06 2022-10-07 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de ketohexocinasa (khk) y metodos de uso de las mismas.
EP4121534A1 (en) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
EP4127134A4 (en) 2020-04-01 2024-07-31 Alnylam Pharmaceuticals, Inc. COMPOSITIONS OF ALPHA-2A ADRENERGIC RECEPTOR (ADRA2A) ARNI AGENTS AND METHODS OF USE THEREOF
IL297121A (en) 2020-04-06 2022-12-01 Alnylam Pharmaceuticals Inc Compositions and methods for silencing myoc expression
AU2021251754A1 (en) 2020-04-07 2022-11-03 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
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
JP7844347B2 (ja) 2020-04-27 2026-04-13 アルナイラム ファーマシューティカルズ, インコーポレイテッド アポリポタンパク質E(ApoE)iRNA剤組成物およびその使用方法
MX2022013606A (es) 2020-04-30 2023-01-16 Alnylam Pharmaceuticals Inc Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas.
TWI901676B (zh) 2020-05-01 2025-10-21 美商Ionis製藥公司 調節atxn1之化合物及方法
JP7776420B2 (ja) 2020-05-12 2025-11-26 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
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
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)
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
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)
EP4150088A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
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)
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
JP2023530234A (ja) 2020-06-05 2023-07-14 ザ・ブロード・インスティテュート・インコーポレイテッド 新生物を治療するための組成物および方法
CN116075592A (zh) 2020-06-09 2023-05-05 阿尔尼拉姆医药品有限公司 用于沉默gpam(线粒体甘油-3-磷酸酰基转移酶1)表达的sirna组合物和方法
WO2021252557A1 (en) 2020-06-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
AU2021292296A1 (en) 2020-06-18 2023-01-19 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof
BR112022026316A2 (pt) 2020-06-24 2023-03-07 Vir Biotechnology Inc Anticorpos neutralizantes do vírus da hepatite b engenheirados e usos dos mesmos
WO2021263026A1 (en) 2020-06-25 2021-12-30 Synthorx, Inc. Immuno oncology combination therapy with il-2 conjugates and anti-egfr antibodies
TW202216996A (zh) 2020-06-29 2022-05-01 美商Ionis製藥公司 調節plp1之化合物及方法
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
MX2023001222A (es) 2020-07-28 2023-04-26 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de app.
IL300258A (en) 2020-08-07 2023-03-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating SCN2A
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
EP4225917A1 (en) 2020-10-05 2023-08-16 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) 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
IL301612A (en) 2020-10-09 2023-05-01 Synthorx Inc Immuno oncology therapies with il-2 conjugates
EP4225376A1 (en) 2020-10-09 2023-08-16 Synthorx, Inc. Immuno oncology combination therapy with il-2 conjugates and pembrolizumab
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
CN116761885A (zh) 2020-10-23 2023-09-15 斯克利普斯研究所 包含非天然核苷酸的多核苷酸的逆转录
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
CA3200595A1 (en) 2020-11-13 2022-05-19 Alnylam Pharmaceuticals, Inc. Coagulation factor v (f5) irna compositions and methods of use thereof
KR20230108728A (ko) 2020-11-18 2023-07-18 아이오니스 파마수티컬즈, 인코포레이티드 앤지오텐시노겐 발현을 조절하기 위한 화합물 및 방법
WO2022119873A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
CN112480197B (zh) * 2020-12-02 2022-03-08 新乡拓新药业股份有限公司 一种合成胞嘧啶核苷的方法
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
AR124408A1 (es) 2020-12-18 2023-03-22 Ionis Pharmaceuticals Inc Compuestos y métodos para modular el factor xii
KR20230135585A (ko) 2020-12-23 2023-09-25 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변형된 trem의 조성물 및 이의 용도
US20240336914A1 (en) 2020-12-31 2024-10-10 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
US20240343746A1 (en) 2020-12-31 2024-10-17 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
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
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
WO2022174101A1 (en) 2021-02-12 2022-08-18 Synthorx, Inc. Skin cancer combination therapy with il-2 conjugates and cemiplimab
WO2022183006A1 (en) 2021-02-25 2022-09-01 Mayo Foundation For Medical Education And Research Compounds for programmable protein degradation and methods of use for the disease treatment
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
CA3211059A1 (en) 2021-02-26 2022-09-01 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
BR112023017737A2 (pt) 2021-03-04 2023-10-03 Alnylam Pharmaceuticals Inc Composições de irna de angiopoietina-semelhantes 3 (angptl3) e métodos de uso das mesmas
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
BR112023019981A2 (pt) 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas
KR20240009393A (ko) 2021-03-31 2024-01-22 엔트라다 테라퓨틱스, 인크. 사이클릭 세포 침투 펩티드
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
CA3216106A1 (en) 2021-04-26 2022-11-03 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
JP2024518374A (ja) 2021-05-03 2024-05-01 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)媒介性アミロイドーシスを治療するための組成物および方法
IL308353A (en) 2021-05-10 2024-01-01 Entrada Therapeutics Inc Compositions and methods for modulating tissue distribution of intracellular therapies
US20250051393A1 (en) 2021-05-10 2025-02-13 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
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
JP2024522068A (ja) 2021-05-18 2024-06-11 アルナイラム ファーマシューティカルズ, インコーポレイテッド ナトリウム-グルコース共輸送体2(sglt2)irna組成物およびその使用方法
US20240263177A1 (en) 2021-05-20 2024-08-08 Korro Bio, Inc. Methods and Compositions for Adar-Mediated Editing
MX2023014008A (es) 2021-05-27 2023-12-11 Alnylam Pharmaceuticals Inc Composiciones y metodos para silenciar la expresion de anhidrasa carbonica 2.
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
WO2022256395A1 (en) 2021-06-02 2022-12-08 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2022256534A1 (en) 2021-06-03 2022-12-08 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
MX2023014168A (es) 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Marco de lectura abierto 72 del cromosoma humano 9 (c9orf72) composiciones de agente de arni y metodos de uso de este.
JP2024523000A (ja) 2021-06-08 2024-06-25 アルナイラム ファーマシューティカルズ, インコーポレイテッド シュタルガルト病及び/又は網膜結合タンパク質4(rbp4)関連障害を治療又は予防するための組成物及び方法
US11753644B2 (en) 2021-06-18 2023-09-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing IFNAR1 expression
AU2022298774A1 (en) 2021-06-23 2023-12-14 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
JP2024528659A (ja) 2021-07-19 2024-07-30 アルナイラム ファーマシューティカルズ, インコーポレイテッド 非原発性高シュウ酸尿症疾患または障害を有するまたは発症するリスクがある対象を治療するための方法および組成物
IL309897A (en) 2021-07-21 2024-03-01 Alnylam Pharmaceuticals Inc Target gene IRNA compositions associated with a metabolic disorder and methods of using them
TW202325312A (zh) 2021-07-23 2023-07-01 美商艾拉倫製藥股份有限公司 β-鏈蛋白(CTNNB1)iRNA組成物及其使用方法
JP2024529437A (ja) 2021-07-29 2024-08-06 アルナイラム ファーマシューティカルズ, インコーポレイテッド 3-ヒドロキシ-3-メチルグリタル-COAレダクターゼ(HMGCR)iRNA組成物およびその使用方法
AR126675A1 (es) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA LA TRANSTIRRETINA (TTR) Y SUS MÉTODOS DE USO
CA3228255A1 (en) 2021-08-04 2023-02-09 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing angiotensinogen (agt)
CN118076737A (zh) 2021-08-13 2024-05-24 阿尔尼拉姆医药品有限公司 因子XII(F12)iRNA组合物及其使用方法
TW202325311A (zh) 2021-08-31 2023-07-01 美商艾拉倫製藥公司 誘導細胞死亡之DFFA樣效應子B (CIDEB) iRNA組合物及其使用方法
EP4395829A1 (en) 2021-09-01 2024-07-10 Entrada Therapeutics, Inc. Compounds and methods for skipping exon 44 in duchenne muscular dystrophy
JP2024535850A (ja) 2021-09-17 2024-10-02 アルナイラム ファーマシューティカルズ, インコーポレイテッド 補体成分(C3)をサイレンシングするためのiRNA組成物および方法
JP2024535888A (ja) 2021-09-20 2024-10-02 アルナイラム ファーマシューティカルズ, インコーポレイテッド インヒビンサブユニットベータe(inhbe)モジュレーター組成物およびその使用方法
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
AU2022357561A1 (en) 2021-10-01 2024-04-18 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
US20240401045A1 (en) 2021-10-01 2024-12-05 Adarx Pharmaceuticals, Inc. Angiotensinogen-modulating compositions and methods of use thereof
EP4416284A1 (en) 2021-10-15 2024-08-21 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
AR127477A1 (es) 2021-10-29 2024-01-31 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA EL FACTOR B DEL COMPLEMENTO (CFB) Y MÉTODOS DE USO DE LAS MISMAS
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
EP4452327A1 (en) 2021-12-20 2024-10-30 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
EP4490187A4 (en) 2022-03-08 2026-02-25 Immunedge Inc EPO RECEPTOR AGONISTS AND ANTAGONISTS
EP4522742A2 (en) 2022-05-13 2025-03-19 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
EP4547683A2 (en) 2022-06-30 2025-05-07 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
CN119907856A (zh) 2022-09-15 2025-04-29 阿尔尼拉姆医药品有限公司 第13型17β-羟基类固醇脱氢酶(HSD17B13)iRNA组合物及其使用方法
JP2025532127A (ja) 2022-09-23 2025-09-29 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 Mecp2発現を低減する化合物及び方法
JP2025532985A (ja) 2022-09-30 2025-10-03 アルナイラム ファーマシューティカルズ, インコーポレイテッド 修飾二本鎖rna剤
WO2024136899A1 (en) 2022-12-21 2024-06-27 Synthorx, Inc. Cancer therapy with il-2 conjugates and chimeric antigen receptor therapies
IL322261A (en) 2023-02-09 2025-09-01 Alnylam Pharmaceuticals Inc Reverser molecules and methods of using them
WO2024170778A1 (en) 2023-02-17 2024-08-22 Anjarium Biosciences Ag Methods of making dna molecules and compositions and uses thereof
CN121263208A (zh) 2023-03-20 2026-01-02 新索思股份有限公司 使用il-2 peg缀合物的癌症疗法
KR20260021596A (ko) 2023-04-05 2026-02-13 아이오니스 파마수티컬즈, 인코포레이티드 Pln 발현을 감소시키는 화합물 및 방법
JP2026514045A (ja) 2023-04-12 2026-05-01 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 二本鎖rna剤の肝外デリバリー
EP4698652A2 (en) 2023-04-20 2026-02-25 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024226499A1 (en) 2023-04-24 2024-10-31 The Broad Institute, Inc. Compositions and methods for modifying fertility
WO2024238396A1 (en) 2023-05-12 2024-11-21 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
CN121712891A (zh) 2023-06-20 2026-03-20 阿达尔克斯制药有限公司 Lrrk2调节组合物及其使用方法
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
KR20260044219A (ko) 2023-07-21 2026-04-01 매로우 테라퓨틱스, 인크. 조혈 세포 표적화 접합체 및 관련 방법
EP4754255A1 (en) 2023-08-04 2026-06-10 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025059511A1 (en) 2023-09-15 2025-03-20 Praxis Precision Medicines, Inc. Elsunersen / prax-222 for use in the treatment of scn2a-related disorders
EP4784172A1 (en) 2023-09-27 2026-08-05 Judo Bio Inc. Polymyxins for delivery of agents to the kidney
EP4784753A2 (en) 2023-09-27 2026-08-05 Judo Bio Inc. Slc6a19-targeting modulatory nucleic acid agents
AU2024348221A1 (en) 2023-09-27 2026-04-30 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
US20250263702A1 (en) 2024-02-19 2025-08-21 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting cideb and related methods
WO2025199231A2 (en) 2024-03-20 2025-09-25 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
TW202603156A (zh) 2024-03-25 2026-01-16 美商欣爍克斯公司 用於生產多肽之合成tRNA合成酶及包含合成分子的細胞
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
TW202615058A (zh) 2024-06-12 2026-04-16 美商艾拉倫製藥股份有限公司 失養強直性肌肉蛋白質激酶(dmpk)irna組成物及其使用方法
WO2025259743A1 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dual conjugate compounds for extrahepatic delivery
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)
WO2026050243A1 (en) 2024-08-26 2026-03-05 Korro Bio, Inc. Galnac conjugated oligonucleotides for rna editing
WO2026055461A1 (en) 2024-09-05 2026-03-12 Aperture Therapeutics, Inc. Antibody oligonucleotide conjugates comprising an antisense polynucleotide agent conjugated to a cd33 antibody and methods of use thereof
WO2026122478A1 (en) 2024-12-02 2026-06-11 Korro Bio, Inc. Extrahepatic delivery formulations and methods
WO2026136379A1 (en) 2024-12-17 2026-06-25 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof

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 (enExample) * 1987-02-12 1990-05-19 Takeda Chemical Industries Ltd
WO1989008146A1 (en) * 1988-02-26 1989-09-08 Worcester Foundation For Experimental Biology Inhibition of htlv-iii by exogenous oligonucleotides
WO1991006556A1 (en) * 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides

Also Published As

Publication number Publication date
JPH06100562A (ja) 1994-04-12
EP0577558A3 (enExample) 1994-04-20
CA2099712A1 (en) 1994-01-02
US5700920A (en) 1997-12-23
US5461152A (en) 1995-10-24
US5610300A (en) 1997-03-11
EP0577558A2 (de) 1994-01-05

Similar Documents

Publication Publication Date Title
MX9205988A (es) Nucleosidos biciclicos, oligonucleotidos, procedimiento para su preparacion y productos intermedios.
ES2131116T3 (es) Analogos de nucleotidos enantiomericos antirretrovirales.
AU551324B2 (en) Phosphoramidite derivatives of nucleotides and their use in preparation of oligonucleotides
PA8483101A1 (es) Compuestos quimicos
ES2113881T3 (es) Derivados y analogos de acido 2-desoxi-2,3-didehidro-n-acetil neuraminico y su utilizacion como agentes antivirales.
ES2056096T3 (es) Purinil-ciclobutanos.
EP0288163A3 (en) Oligodeoxynucleotides as inhibitors of the replication of retroviruses and the expression of oncogenes
ATE92326T1 (de) Adenosin-deaminase-stabile anti-retroviralnukleoside.
ES8602840A1 (es) Un procedimiento para la preparacion de un nucleosido.
ES2036194T3 (es) Derivados n-fosfonilmetoxialquilicos de bases de pirimidina y purina, metodos para su preparacion y composiciones farmaceuticas basadas en ellos, con actividad antivirica.
ES2181725T3 (es) Inhibidores de hiv proteasa.
MX9405383A (es) Imidazo(4,5-c) piridin-4-aminas.
ES2052629T3 (es) Pirazolo(3,4-d)piridinas, procedimiento para su preparacion y utilizacion como medicamentos.
CO4940451A1 (es) Derivados de acido barbiturico con actividad antimetastica y antitumoral
ES2085446T3 (es) Derivados de acido 9-purinil fosfonico.
ES2143487T3 (es) 1,2,4-oxadiazolil-fenoxialquilisoxazoles y su empleo como agentes antivirales.
ES2066806T3 (es) Uso de la adenosina 5'-fosfato para el tratamiento de caquexia de cancer.
FI871322A7 (fi) Nya benzotiazinderivat.
ES8802363A1 (es) Procedimiento para la fabricacion de derivados nucleosidos
ES2164366T3 (es) Derivados de la distamicina acriloil sustituidos, procedimiento de fabricacion asociado y su utilizacion como agentes antitumorales y antivirales.
ES2105684T3 (es) Derivados insaturados de fosfonato de purinas y pirimidinas.
AR248011A1 (es) Procedimiento para la fabricacion de un derivado de diamidin-difenil-alquileno.
ES2022192B3 (es) Eteres de oximas sustituidos, su utilizacion como biorreguladores para la reduccion del nivel de etileno endogeno en las plantas.
DK615788D0 (da) Nucleosidforbindelser til anvendelse mod virale infektioner
ES2132419T3 (es) Nuevos agentes de nucleosidos 9-n-biciclicos utiles como inhibidores selectivos de citocinas proinflamatorias.