ATE356824T1 - L-ribo-lna analoge - Google Patents

L-ribo-lna analoge

Info

Publication number
ATE356824T1
ATE356824T1 AT00925080T AT00925080T ATE356824T1 AT E356824 T1 ATE356824 T1 AT E356824T1 AT 00925080 T AT00925080 T AT 00925080T AT 00925080 T AT00925080 T AT 00925080T AT E356824 T1 ATE356824 T1 AT E356824T1
Authority
AT
Austria
Prior art keywords
ribo
compounds
lna analogue
lna
analogue
Prior art date
Application number
AT00925080T
Other languages
English (en)
Inventor
Jesper Wengel
Original Assignee
Santaris Pharma As
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=27220811&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ATE356824(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Santaris Pharma As filed Critical Santaris Pharma As
Application granted granted Critical
Publication of ATE356824T1 publication Critical patent/ATE356824T1/de

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H9/00Compounds containing a hetero ring sharing at least two hetero atoms with a saccharide radical
    • C07H9/02Compounds containing a hetero ring sharing at least two hetero atoms with a saccharide radical the hetero ring containing only oxygen as ring hetero atoms
    • C07H9/04Cyclic acetals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06Pyrimidine radicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
AT00925080T 1999-05-04 2000-05-04 L-ribo-lna analoge ATE356824T1 (de)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
DKPA199900603 1999-05-04
DKPA199901225 1999-09-01
DKPA200000032 2000-01-11

Publications (1)

Publication Number Publication Date
ATE356824T1 true ATE356824T1 (de) 2007-04-15

Family

ID=27220811

Family Applications (1)

Application Number Title Priority Date Filing Date
AT00925080T ATE356824T1 (de) 1999-05-04 2000-05-04 L-ribo-lna analoge

Country Status (16)

Country Link
US (1) US7053207B2 (de)
EP (1) EP1178999B1 (de)
JP (1) JP2002543214A (de)
KR (1) KR100782896B1 (de)
CN (2) CN102180924A (de)
AT (1) ATE356824T1 (de)
AU (1) AU776362B2 (de)
CA (1) CA2372085C (de)
DE (1) DE60033927T2 (de)
DK (1) DK1178999T3 (de)
ES (1) ES2283298T3 (de)
HK (1) HK1048322A1 (de)
IL (1) IL145497A0 (de)
NZ (1) NZ514348A (de)
PT (1) PT1178999E (de)
WO (1) WO2000066604A2 (de)

Families Citing this family (853)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7119184B2 (en) * 1991-08-12 2006-10-10 Isis Pharmaceuticals, Inc. Oligonucleotides having A-DNA form and B-DNA form conformational geometry
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
AU758956B2 (en) 1999-02-12 2003-04-03 Daiichi Sankyo Company, Limited Novel nucleosides and oligonucleotide analogues
US6734291B2 (en) 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
WO2000056746A2 (en) * 1999-03-24 2000-09-28 Exiqon A/S Improved synthesis of [2.2.1]bicyclo nucleosides
AU3041701A (en) * 1999-12-23 2001-07-09 Exiqon A/S Therapeutic uses of lna-modified oligonucleotides
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
EP1268768A2 (de) 2000-03-27 2003-01-02 University Of Delaware Gezielte chromosomale genom-änderungen mit modifizierten einzelsträngigen oligonukleotiden
US6936467B2 (en) 2000-03-27 2005-08-30 University Of Delaware Targeted chromosomal genomic alterations with modified single stranded oligonucleotides
EP1414840A4 (de) 2001-03-27 2005-04-13 Univ Delaware Genomics-anwendungen für modifizierte oligonukleotide
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
GB0114719D0 (en) * 2001-06-15 2001-08-08 Glaxo Group Ltd Compound
US9261460B2 (en) * 2002-03-12 2016-02-16 Enzo Life Sciences, Inc. Real-time nucleic acid detection processes and compositions
CA2459347C (en) 2001-09-04 2012-10-09 Exiqon A/S Locked nucleic acid (lna) compositions and uses thereof
US7112405B2 (en) 2001-09-27 2006-09-26 University Of Delaware Compositions and methods for enhancing oligonucleotide-mediated gene alteration
US7468244B2 (en) 2001-09-27 2008-12-23 University Of Delaware Polymorphism detection and separation
WO2003039523A2 (en) * 2001-11-05 2003-05-15 Exiqon A/S OLIGONUCLEOTIDES MODIFIED WITH NOVEL α-L-RNA ANALOGUES
US20030198980A1 (en) * 2001-12-21 2003-10-23 Applera Corporation Heteroconfigurational polynucleotides and methods of use
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US20030166282A1 (en) * 2002-02-01 2003-09-04 David Brown High potency siRNAS for reducing the expression of target genes
CA2474910A1 (en) 2002-02-01 2003-08-07 Sequitur, Inc. Oligonucleotide compositions with enhanced efficiency
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US7566535B2 (en) 2002-03-07 2009-07-28 University Of Delaware Enhanced oligonucleotide-mediated nucleic acid sequence alteration
US9353405B2 (en) 2002-03-12 2016-05-31 Enzo Life Sciences, Inc. Optimized real time nucleic acid detection processes
CA2480673A1 (en) * 2002-03-29 2003-10-09 Genentech, Inc. Methods and compositions for detection and quantitation of nucleic acid analytes
SI2264172T1 (sl) 2002-04-05 2017-12-29 Roche Innovation Center Copenhagen A/S Oligomerne spojine za modulacijo izražanja HIF-1alfa
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
ES2290448T3 (es) * 2002-05-08 2008-02-16 Santaris Pharma A/S Sistesis de derivados de acidos nucleicos bloqueados.
US20040248094A1 (en) * 2002-06-12 2004-12-09 Ford Lance P. Methods and compositions relating to labeled RNA molecules that reduce gene expression
US20100075423A1 (en) * 2002-06-12 2010-03-25 Life Technologies Corporation Methods and compositions relating to polypeptides with rnase iii domains that mediate rna interference
EP1532271A4 (de) * 2002-06-12 2006-10-18 Ambion Inc Verfahren und zusammensetzungen im zusammenhang mit polypeptiden mit rnase-iii-domänen, die rna-interferenz vermitteln
US7005265B1 (en) 2002-06-20 2006-02-28 Wenhong Fan Nonenzymatic catalytic signal amplification for nucleic acid hybridization assays
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
AU2003281969B2 (en) 2002-11-18 2011-01-27 Roche Innovation Center Copenhagen A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
AU2013201786B2 (en) * 2002-11-18 2015-04-02 Roche Innovation Center Copenhagen A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
EP1426448A1 (de) 2002-12-03 2004-06-09 bioMérieux BV Methode zur Reduktion des Effektes von Sequenzvariationen in einem diagnostischen Hybridisierungsverfahren, Sonde zur Verwendung in einem solchen Verfahren, sowie das Verfahren selbst
US20060292438A1 (en) * 2002-12-23 2006-12-28 Applera Corporation; Applied Biosystems Group Heteroconfigurational Polynucleotides and Methods of Use
ES2329131T3 (es) 2003-02-10 2009-11-23 Santaris Pharma A/S Compuestos oligomericos para modular la expresion de survivina.
US7713738B2 (en) 2003-02-10 2010-05-11 Enzon Pharmaceuticals, Inc. Oligomeric compounds for the modulation of survivin expression
WO2004083430A2 (en) 2003-03-21 2004-09-30 Santaris Pharma A/S SHORT INTERFERING RNA (siRNA) ANALOGUES
WO2004106356A1 (en) * 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
ES2385828T3 (es) * 2003-06-20 2012-08-01 Exiqon A/S Sondas, bibliotecas y kits para análisis de mezclas de ácidos nucleicos y procedimientos para construirlos
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US20050053981A1 (en) * 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US7480382B2 (en) * 2003-09-30 2009-01-20 Microsoft Corporation Image file container
ES2313104T3 (es) * 2003-11-07 2009-03-01 Daiichi Sankyo Company, Limited Metodo para detectar polimorfismo genico.
CA2550258A1 (en) 2003-12-23 2005-07-07 Santaris Pharma A/S Oligomeric compounds for the modulation of bcl-2
GB0406864D0 (en) 2004-03-26 2004-04-28 Qiagen As Nucleic acid sequencing
US8192937B2 (en) 2004-04-07 2012-06-05 Exiqon A/S Methods for quantification of microRNAs and small interfering RNAs
WO2005105157A2 (en) 2004-04-23 2005-11-10 The Trustees Of Columbia University In The City Ofnew York INHIBITION OF HAIRLESS PROTEIN mRNA
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
CA2572450A1 (en) 2004-05-28 2005-12-15 Ambion, Inc. Methods and compositions involving microrna
ATE482271T1 (de) * 2004-11-09 2010-10-15 Santaris Pharma As Lna-oligonukleotide und krebsbehandlung
EA014097B1 (ru) 2004-11-09 2010-08-30 Сантарис Фарма А/С ОЛИГОНУКЛЕОТИДЫ ЗАМКНУТОЙ НУКЛЕИНОВОЙ КИСЛОТЫ (LNA) ДЛЯ МОДУЛИРОВАНИЯ ЭКСПРЕССИИ HIF-1a И ИХ ПРИМЕНЕНИЕ
US9447138B2 (en) 2004-11-09 2016-09-20 Roche Innovation Center Copenhagen A/S Potent LNA oligonucleotides for the inhibition of HIF-1a expression
NZ555645A (en) 2004-11-09 2009-08-28 Santaris Pharma As LNA oligonucleotides and the treatment of cancer
EP2302055B1 (de) 2004-11-12 2014-08-27 Asuragen, Inc. Verfahren und Zusammensetzungen, die miRNAs und miRNAa-inhibitorischen Molekülen verbunden sind
US20060166238A1 (en) * 2004-12-22 2006-07-27 Ramsing Niels B Probes, libraries and kits for analysis of mixtures of nucleic acids and methods for constructing the same
US20060142228A1 (en) * 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
EP1959012A3 (de) 2004-12-29 2009-12-30 Exiqon A/S Neuartige Oligonukleotid-Zusammensetzungen und Probensequenzen für die Erkennung und Analyse von Mikro-RNAs und ihre Ziel-MRNAs
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
DK1866414T3 (da) 2005-03-31 2012-04-23 Calando Pharmaceuticals Inc Inhibitorer af ribonukleotidreduktase-underenhed 2 og anvendelser deraf.
US7838502B2 (en) 2005-05-06 2010-11-23 University Of Massachusetts Medical School Compositions and methods to modulate H. influenzae pathogenesis
DK3308788T3 (da) 2005-06-23 2019-01-02 Biogen Ma Inc Sammensætninger og fremgangsmåder til modulering af smn2-splejsning
AU2006281569A1 (en) 2005-08-17 2007-02-22 Medexis S.A. Composition and method for determination of CK19 expression
US20070054872A1 (en) * 2005-08-24 2007-03-08 Mirus Bio Corporation Regulatable or conditional expression systems
EP1931780B1 (de) 2005-08-29 2016-01-06 Regulus Therapeutics Inc. Antisense-verbindungen mit verbesserter anti-mikrorna-aktivität
JP5523705B2 (ja) 2005-08-29 2014-06-18 レグルス・セラピューティクス・インコーポレイテッド Mir−122aをモジュレートする使用方法
JP5623016B2 (ja) 2005-12-01 2014-11-12 プロネイ・セラピューティクス・インコーポレイテッドPronaitherapeutics, Inc. 癌治療法およびそれに用いる医薬組成物
WO2007073149A1 (en) 2005-12-22 2007-06-28 Keygene N.V. Alternative nucleotides for improved targeted nucleotide exchange
WO2007087113A2 (en) 2005-12-28 2007-08-02 The Scripps Research Institute Natural antisense and non-coding rna transcripts as drug targets
CA2640171C (en) * 2006-01-27 2014-10-28 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
CA2640058C (en) 2006-01-27 2018-04-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
ES2550099T3 (es) 2006-01-27 2015-11-04 Biogen Ma Inc. Antagonistas del receptor Nogo
EA015563B1 (ru) 2006-03-23 2011-08-30 Сантарис Фарма А/С Короткая внутренне сегментированная интерферирующая рнк
EP3431602A1 (de) 2006-04-03 2019-01-23 Roche Innovation Center Copenhagen A/S Pharmazeutische zusammensetzung mit anti-mirna-antisense-oligonukleotiden
EP2194129A3 (de) 2006-04-03 2012-12-26 Santaris Pharma A/S Pharmazeutische Zusammensetzungen mit Anti-miRNA-Antisense-Oligonukleotiden
AU2007258117B2 (en) * 2006-05-05 2013-05-30 Isis Pharmaceuticals, Inc. Compounds and methods for modulating gene expression
EP2527442A3 (de) 2006-05-05 2013-03-06 Isis Pharmaceuticals, Inc. Verbindungen und Verfahren zur Modulation von Genexpression
US7666854B2 (en) * 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
ES2389737T3 (es) * 2006-05-11 2012-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en 5'
EP2054415A1 (de) * 2006-07-14 2009-05-06 Santaris Pharma A/S Adenosinrezeptorantagonisten
US9550988B2 (en) 2006-10-18 2017-01-24 Ionis Pharmaceuticals, Inc. Antisense compounds
WO2008046911A2 (en) 2006-10-20 2008-04-24 Exiqon A/S Novel human micrornas associated with cancer
US8188255B2 (en) 2006-10-20 2012-05-29 Exiqon A/S Human microRNAs associated with cancer
WO2008052774A2 (en) 2006-10-31 2008-05-08 Noxxon Pharma Ag Methods for detection of a single- or double-stranded nucleic acid molecule
WO2008060613A1 (en) 2006-11-15 2008-05-22 The J. David Gladstone Institutes Methods and compositions for reducing amyloid beta levels
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
EP2455471A3 (de) * 2006-11-27 2012-09-12 Isis Pharmaceuticals, Inc. Verfahren zur Behandlung von Hypercholesterinämie
US8048998B2 (en) 2007-01-19 2011-11-01 Exiqon A/S Mediated cellular delivery of LNA oligonucleotides
US20080206850A1 (en) * 2007-02-28 2008-08-28 Dellinger Douglas J Methods and compositions for DNA synthesis
WO2008113830A1 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S Rna antagonist compounds for the inhibition of apo-b100 expression
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
US8999634B2 (en) 2007-04-27 2015-04-07 Quest Diagnostics Investments Incorporated Nucleic acid detection combining amplification with fragmentation
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (de) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbozyklische bizyklische nukleinsäureanaloga
US20100261175A1 (en) * 2007-06-15 2010-10-14 Exiqon A/S Use of short oligonucleotides for reagent redundancy experiments in rna functional analysis
US8278283B2 (en) * 2007-07-05 2012-10-02 Isis Pharmaceuticals, Inc. 6-disubstituted or unsaturated bicyclic nucleic acid analogs
US8361714B2 (en) 2007-09-14 2013-01-29 Asuragen, Inc. Micrornas differentially expressed in cervical cancer and uses thereof
WO2009039173A2 (en) 2007-09-19 2009-03-26 Applied Biosystems Inc. SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF
MX2010003465A (es) 2007-10-02 2010-07-05 Amgen Inc Incremento de eritropoyetina utilizando acidos nucleicos que hibridan para micro-arn y precursores de los mismos.
EP2205737B1 (de) 2007-10-04 2013-02-13 Santaris Pharma A/S Mikromir-moleküle
WO2009067647A1 (en) * 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
EP4053546A1 (de) 2007-12-06 2022-09-07 Genalyte, Inc. Vorrichtung und verfahren zur kennzeichnungsfreien überwachung von prozessen.
BRPI0820738A2 (pt) 2007-12-14 2015-06-16 Minitube America Inc Separação específica do gênero de células de esperma e embriões
EP2265627A2 (de) * 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclische cyclohexitolnukleinsäureanaloga
US8361980B2 (en) 2008-03-07 2013-01-29 Santaris Pharma A/S Pharmaceutical compositions for treatment of microRNA related diseases
US8227431B2 (en) 2008-03-17 2012-07-24 Hetero Drugs Limited Nucleoside derivatives
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
JP2011516065A (ja) 2008-04-04 2011-05-26 カランド ファーマシューティカルズ, インコーポレイテッド Epas1阻害剤の組成物および使用
EP2990487A1 (de) 2008-05-08 2016-03-02 Asuragen, INC. Zusammensetzungen und verfahren in zusammenhang mit der mirna-modulation von neovaskularisation oder angiogenese
US8222221B2 (en) 2008-06-04 2012-07-17 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small RNA targeting of gene promoters
CA2635187A1 (en) 2008-06-05 2009-12-05 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes and uses thereof
AU2009276763B2 (en) 2008-07-29 2015-07-16 The Board Of Regents Of The University Of Texas Sytem Selective inhibition of polyglutamine protein expression
EP2315832B1 (de) 2008-08-01 2015-04-08 Roche Innovation Center Copenhagen A/S Micro-rna-vermittelte modulation koloniestimulierender faktoren
WO2010017509A1 (en) * 2008-08-07 2010-02-11 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
EP2356129B1 (de) * 2008-09-24 2013-04-03 Isis Pharmaceuticals, Inc. Substituierte alpha-l-bicyclische nukleoside
ES2727549T3 (es) * 2008-10-03 2019-10-17 Curna Inc Tratamiento de las enfermedades relacionadas con la apolipoproteína a1 por inhibición del transcrito antisentido natural a la apolipoproteína a1
KR101979134B1 (ko) 2008-10-15 2019-05-15 아이오니스 파마수티컬즈, 인코포레이티드 인자 11 발현의 조정
US8987435B2 (en) 2008-10-24 2015-03-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US9846126B2 (en) 2008-10-27 2017-12-19 Genalyte, Inc. Biosensors based on optical probing and sensing
MX2011005910A (es) * 2008-12-04 2011-06-17 Opko Curna Llc Tratamiento de enfermedades relacionadas con eritropoyetina (epo) mediante inhibicion del transcrito antisentido natural a eritropoyetina.
US8927511B2 (en) * 2008-12-04 2015-01-06 Curna, Inc. Treatment of vascular endothelial growth factor (VEGF) related diseases by inhibition of natural antisense transcript to VEGF
KR101840618B1 (ko) 2008-12-04 2018-03-20 큐알엔에이, 인크. 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료
CN102257147A (zh) 2008-12-22 2011-11-23 凯津公司 在植物原生质体中使用双链rna提高靶基因改变的效率
WO2010091308A2 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
CN102387817B (zh) 2009-02-12 2018-01-30 库尔纳公司 通过抑制针对脑衍生神经营养因子(bdnf)的天然反义转录物来治疗bdnf相关的疾病
EP2408919B1 (de) 2009-03-16 2017-10-18 CuRNA, Inc. Behandlung von erkrankungen im zusammenhang mit dem nuklearfaktor (erythroid-derived)-like 2 (nrf2) mittels hemmung des natürlichen antisense-transkripts gegen nrf2
CA2755404C (en) 2009-03-17 2020-03-24 Joseph Collard Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
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
EP2421970B1 (de) 2009-04-24 2016-09-07 Roche Innovation Center Copenhagen A/S Pharmazeutische zusammensetzungen zur behandlung von nicht auf interferon ansprechende hcv-patienten
EP2427553A4 (de) 2009-05-06 2012-11-07 Opko Curna Llc Behandlung von lipidtransport- und stoffwechselgenbedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts gegen ein lipidtransport- und stoffwechselgen
CN102803492B (zh) 2009-05-06 2016-06-29 库尔纳公司 通过抑制针对三重四脯氨酸(ttp)的天然反义转录物来治疗ttp相关疾病
KR101742334B1 (ko) 2009-05-08 2017-06-01 큐알엔에이, 인크. Dmd 패밀리에 대한 천연 안티센스 전사체의 억제에 의한 디스트로핀 패밀리 관련된 질환의 치료
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
US8895527B2 (en) 2009-05-22 2014-11-25 Curna, Inc. Treatment of transcription factor E3 (TFE3) and insulin receptor substrate 2(IRS2) related diseases by inhibition of natural antisense transcript to TFE3
WO2010138806A2 (en) 2009-05-28 2010-12-02 Curna, Inc. Treatment of antiviral gene related diseases by inhibition of natural antisense transcript to an antiviral gene
US9200276B2 (en) 2009-06-01 2015-12-01 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent RNA interference, compositions and methods of use thereof
US8951981B2 (en) 2009-06-16 2015-02-10 Curna, Inc. Treatment of paraoxonase 1 (PON1) related diseases by inhibition of natural antisense transcript to PON1
KR101801404B1 (ko) 2009-06-16 2017-12-20 큐알엔에이, 인크. 콜라겐 유전자에 대한 천연 안티센스 전사체의 억제에 의한 콜라겐 유전자 관련된 질환의 치료
EP3643783A1 (de) 2009-06-17 2020-04-29 Biogen MA Inc. Zusammensetzungen und verfahren zur modulation der smn2-aufspaltung in einem patienten
KR101807323B1 (ko) 2009-06-24 2017-12-08 큐알엔에이, 인크. Tnfr2에 대한 천연 안티센스 전사체의 억제에 의한 종양 괴사 인자 수용체 2(tnfr2) 관련된 질환의 치료
CA2765815A1 (en) 2009-06-26 2010-12-29 Opko Curna, Llc Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
US8563528B2 (en) 2009-07-21 2013-10-22 Santaris Pharma A/S Antisense oligomers targeting PCSK9
US20120252869A1 (en) 2009-07-24 2012-10-04 Opko Curna, Llc Treatment of sirtuin (sirt) related diseases by inhibition of natural antisense transcript to a sirtuin (sirt)
ES2585360T3 (es) 2009-08-05 2016-10-05 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen de la insulina (INS) por inhibición de la transcripción antisentido natural en un gen de la insulina (INS)
EP2462153B1 (de) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclische cyclohexosenukleinsäureanaloga
CN104313027B (zh) 2009-08-11 2018-11-20 库尔纳公司 通过抑制脂连蛋白(adipoq)的天然反义转录物治疗脂连蛋白(adipoq)相关疾病
KR101805213B1 (ko) 2009-08-21 2017-12-06 큐알엔에이, 인크. Chip에 대한 천연 안티센스 전사체의 억제에 의한 “hsp70-상호작용 단백질(chip)의 c-말단” 관련된 질환의 치료
JP5964232B2 (ja) 2009-08-25 2016-08-03 カッパーアールエヌエー,インコーポレイテッド ‘iqモチーフ含有gtpアーゼ活性化タンパク質’(iqgap)に対する天然アンチセンス転写産物の阻害によるiqgap関連疾患の治療
US20110196141A1 (en) * 2009-09-07 2011-08-11 Council Of Scientific & Industrial Research Locked and unlocked 2'-o phosphoramidite nucleosides, process of preparation thereof and oligomers comprising the nucleosides
WO2011032034A2 (en) * 2009-09-10 2011-03-17 University Of Idaho Nucleobase-functionalized conformationally restricted nucleotides and oligonucleotides for targeting nucleic acids
CA2775111C (en) 2009-09-25 2019-12-31 Opko Curna, Llc Treatment of filaggrin (flg) related diseases by modulation of flg expression and activity
BR122022001178B1 (pt) 2009-10-26 2022-10-04 F. Hoffmann-La Roche Ag Método para a produção de uma imunoglobulina glicosilada e seu uso
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
KR101944119B1 (ko) 2009-11-13 2019-01-30 사렙타 쎄러퓨틱스 인코퍼레이티드 안티센스 항바이러스 화합물 및 인플루엔자 바이러스 감염을 치료하는 방법
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
EP2515947B1 (de) 2009-12-23 2021-10-06 CuRNA, Inc. Behandlung von durch das uncoupling protein 2 (ucp2) bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts für ucp2
CA2782373C (en) 2009-12-23 2019-03-26 Opko Curna, Llc Treatment of hepatocyte growth factor (hgf) related diseases by inhibition of natural antisense transcript to hgf
EP2519634B1 (de) 2009-12-29 2016-06-01 CuRNA, Inc. Behandlung von durch das tumorprotein 63 (p63) vermittelten erkrankungen durch das natürliche antisense-transkript für p63
EP2519633B1 (de) 2009-12-29 2017-10-25 CuRNA, Inc. Behandlung von erkrankungen im zusammenhang mit dem nuclear-respirator-faktor 1 (nrf1) mittels hemmung des natürlichen antisense-transkripts gegen nrf1
DK2519632T3 (en) 2009-12-31 2018-07-23 Curna Inc TREATMENT OF INSULIN RECEPTOR SUBSTRATE 2- (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPTION TO IRS2 AND TRANSCRIPTION FACTOR E3 (TFE3)
JP5886757B2 (ja) 2010-01-04 2016-03-16 カッパーアールエヌエー,インコーポレイテッド インターフェロン調節因子8(irf8)に対する天然アンチセンス転写物の阻害によるインターフェロン調節因子8(irf8)関連疾患の治療
US8912157B2 (en) 2010-01-06 2014-12-16 Curna, Inc. Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene
WO2011085271A2 (en) 2010-01-08 2011-07-14 Isis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
US8779118B2 (en) 2010-01-11 2014-07-15 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
US9200277B2 (en) 2010-01-11 2015-12-01 Curna, Inc. Treatment of sex hormone binding globulin (SHBG) related diseases by inhibition of natural antisense transcript to SHBG
US20110172296A1 (en) * 2010-01-12 2011-07-14 Bennett C Frank Modulation of transforming growth factor-beta 1 expression
DK2529015T3 (en) 2010-01-25 2018-02-26 Curna Inc TREATMENT OF RNASE H1-RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO RNASE H1
US9574191B2 (en) 2010-02-03 2017-02-21 The Board Of Regents Of The University Of Texas System Selective inhibition of polyglutamine protein expression
US9006198B2 (en) 2010-02-08 2015-04-14 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP3321361B1 (de) 2010-02-08 2019-03-27 Ionis Pharmaceuticals, Inc. Selektive reduktion von allelvarianten
JP5976548B2 (ja) 2010-02-22 2016-08-23 カッパーアールエヌエー,インコーポレイテッド Pycr1に対する天然アンチセンス転写物の阻害によるピロリン−5−カルボン酸レダクターゼ1(pycr1)関連疾患の治療
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011105900A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-alpha (c8-alpha) and uses thereof
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
US20110213011A1 (en) * 2010-02-26 2011-09-01 Dean Nicholas M Modulation of smad3 expression
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
WO2011113015A2 (en) 2010-03-12 2011-09-15 Avi Biopharma, Inc. Antisense modulation of nuclear hormone receptors
WO2011112732A2 (en) 2010-03-12 2011-09-15 The Brigham And Women's Hospital, Inc. Methods of treating vascular inflammatory disorders
US9193752B2 (en) 2010-03-17 2015-11-24 Isis Pharmaceuticals, Inc. 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
CA2795145C (en) 2010-04-02 2019-01-22 Curna, Inc. Treatment of colony-stimulating factor 3 (csf3) related diseases by inhibition of natural antisense transcript to csf3
KR101900962B1 (ko) 2010-04-09 2018-09-20 큐알엔에이, 인크. 섬유아세포 성장 인자 21 (fgf21)에 대한 자연 안티센스 전사체의 저해에 의한 섬유아세포 성장 인자 21 (fgf21) 관련된 질환의 치료
US8980253B2 (en) 2010-04-26 2015-03-17 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of cysteinyl-tRNA synthetase
WO2011139799A2 (en) 2010-04-27 2011-11-10 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of isoleucyl trna synthetases
EP2625186B1 (de) 2010-04-28 2016-07-27 Ionis Pharmaceuticals, Inc. 5'-modifizierte nukleoside und daraus hergestellte oligomere verbindungen
WO2011139702A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
WO2011139695A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified 5' diphosphate nucleosides and oligomeric compounds prepared therefrom
US8993723B2 (en) 2010-04-28 2015-03-31 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of alanyl-tRNA synthetases
EP2563912B1 (de) 2010-04-29 2018-09-05 aTyr Pharma, Inc. Innovative entdeckung therapeutischer, diagnostischer und antikörperhaltiger zusammensetzungen im zusammenhang mit proteinfragmenten von asparaginyl-trna-synthetasen
US20130156845A1 (en) 2010-04-29 2013-06-20 Alnylam Pharmaceuticals, Inc. Lipid formulated single stranded rna
BR112012027547B1 (pt) 2010-04-29 2022-06-14 Ionis Pharmaceuticals, Inc Oligonucleotídeo modificado de fita simples, composição, e seus usos para tratar amiloidose transtirretina, reduzir os seus sintomas e para reduzir a expressão de mrna ou de proteína de transtirretina
EP2563383B1 (de) 2010-04-29 2017-03-01 Atyr Pharma, Inc. Innovative entdeckung therapeutischer, diagnostischer und antikörperhaltiger zusammensetzungen im zusammenhang mit proteinfragmenten von valyl-trna-synthetasen
ES2465948T3 (es) 2010-04-30 2014-06-09 Exiqon A/S Procedimiento de hibridación in situ y tampón
WO2011140132A2 (en) 2010-05-03 2011-11-10 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of phenylalanyl-alpha-trna synthetases
CN103096925A (zh) 2010-05-03 2013-05-08 Atyr医药公司 与精氨酰-tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的创新发现
KR101936011B1 (ko) 2010-05-03 2019-01-07 큐알엔에이, 인크. 시르투인 (sirt)에 대한 자연 안티센스 전사체의 저해에 의한 시르투인 (sirt) 관련된 질환의 치료
WO2011140135A2 (en) 2010-05-03 2011-11-10 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of methionyl-trna synthetases
CN102985103A (zh) 2010-05-04 2013-03-20 Atyr医药公司 与p38多-tRNA合成酶复合物相关的治疗、诊断和抗体组合物的创新发现
US9238042B2 (en) 2010-05-13 2016-01-19 Sarepta Therapeutics, Inc. Antisense modulation of interleukins 17 and 23 signaling
WO2011143482A2 (en) 2010-05-14 2011-11-17 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of phenylalanyl-beta-trna synthetases
TWI531370B (zh) 2010-05-14 2016-05-01 可娜公司 藉由抑制par4天然反股轉錄本治療par4相關疾病
JP6027965B2 (ja) 2010-05-17 2016-11-16 エータイアー ファーマ, インコーポレイテッド ロイシルtRNA合成酵素のタンパク質フラグメントに関連した治療用、診断用および抗体組成物の革新的発見
KR101902197B1 (ko) 2010-05-26 2018-10-01 큐알엔에이, 인크. 메티오닌 설폭시드 환원효소 a (msra)에 대한 자연 안티센스 전사체의 저해에 의한 메티오닌 설폭시드 환원효소 a (msra) 관련된 질환의 치료
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
AU2011258106B2 (en) 2010-05-27 2017-02-23 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glutaminyl-tRNA synthetases
JP6081910B2 (ja) 2010-06-02 2017-02-15 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. 肝線維症治療用組成物および肝線維症の治療法
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011156202A1 (en) 2010-06-08 2011-12-15 Isis Pharmaceuticals, Inc. Substituted 2 '-amino and 2 '-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011159836A2 (en) 2010-06-15 2011-12-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
WO2011163499A2 (en) 2010-06-23 2011-12-29 Opko Curna, Llc Treatment of sodium channel, voltage-gated, alpha subunit (scna) related diseases by inhibition of natural antisense transcript to scna
AU2011289831C1 (en) 2010-07-12 2017-06-15 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glycyl-tRNA synthetases
JP5998131B2 (ja) 2010-07-14 2016-09-28 カッパーアールエヌエー,インコーポレイテッド Discslargehomolog(dlg)dlg1への天然アンチセンス転写物の阻害によるdlg関連疾患の治療
KR20180105730A (ko) 2010-07-19 2018-09-28 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
KR20130137160A (ko) 2010-08-24 2013-12-16 머크 샤프 앤드 돔 코포레이션 내부의 비-핵산 스페이서를 함유하는 단일-가닥 RNAi 작용제
EP2608801B1 (de) 2010-08-25 2019-08-21 aTyr Pharma, Inc. Innovative erkennung therapeutischer, diagnostischer und antikörperhaltiger zusammensetzungen in zusammenhang mit proteinfragmenten von tyrosyl-trna-synthetasen
US9938590B2 (en) * 2010-09-16 2018-04-10 Gen-Probe Incorporated Capture probes immobilizable via L-nucleotide tail
WO2012040124A1 (en) 2010-09-22 2012-03-29 Alios Biopharma, Inc. Azido nucleosides and nucleotide analogs
KR101886457B1 (ko) 2010-10-06 2018-08-07 큐알엔에이, 인크. 시알리다아제 4 (neu4)에 대한 자연 안티센스 전사체의 저해에 의한 neu4 관련된 질환의 치료
WO2012048113A2 (en) 2010-10-07 2012-04-12 The General Hospital Corporation Biomarkers of cancer
US8648053B2 (en) 2010-10-20 2014-02-11 Rosalind Franklin University Of Medicine And Science Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
JP6049623B2 (ja) 2010-10-22 2016-12-21 カッパーアールエヌエー,インコーポレイテッド α‐L‐イズロニダーゼ(IDUA)への天然アンチセンス転写物の阻害によるIDUA関連疾患の治療
EP3327125B1 (de) 2010-10-29 2020-08-05 Sirna Therapeutics, Inc. Rna-interferenz-vermittelte inhibition von genexpression unter verwendung kurzer interferierender nukleinsäure (sina)
CA3060724A1 (en) 2010-11-05 2012-05-10 Genalyte, Inc. Optical analyte detection systems and methods of use
WO2012064758A2 (en) 2010-11-08 2012-05-18 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
CA2817960C (en) 2010-11-17 2020-06-09 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
US10000752B2 (en) 2010-11-18 2018-06-19 Curna, Inc. Antagonat compositions and methods of use
CA2818824A1 (en) 2010-11-23 2012-05-31 Joseph Collard Treatment of nanog related diseases by inhibition of natural antisense transcript to nanog
CA2819431C (en) 2010-12-02 2021-09-28 Keygene N.V. Targeted alteration of dna with oligonucleotides.
EP2857512B1 (de) 2010-12-02 2016-06-29 Keygene N.V. Gezielte Änderung von DNA
WO2012078967A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (epo) production
EP2648763A4 (de) 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur expressionshemmung der gene klf-1 und bcl11a
WO2012097261A2 (en) 2011-01-14 2012-07-19 The General Hospital Corporation Methods targeting mir-128 for regulating cholesterol/lipid metabolism
EP3467109A1 (de) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomere verbindungen mit bicyclischen nukleotiden und verwendungen davon
KR102104401B1 (ko) 2011-03-29 2020-04-27 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
CA2831915C (en) 2011-04-01 2021-01-12 Isis Pharmaceuticals, Inc. Modulation of signal transducer and activator of transcription 3 (stat3) expression
JP5951752B2 (ja) 2011-04-13 2016-07-13 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. Ptp1b発現のアンチセンス調節
UA118951C2 (uk) 2011-04-21 2019-04-10 Айоніс Фармасьютікалз, Інк. Модулювання експресії вірусу гепатиту b (hbv)
WO2012149495A1 (en) 2011-04-27 2012-11-01 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein ciii (apociii) expression
US9353371B2 (en) 2011-05-02 2016-05-31 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with usher syndrome
WO2012170347A1 (en) 2011-06-09 2012-12-13 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
RU2620980C2 (ru) 2011-06-09 2017-05-30 Курна, Инк. Лечение заболеваний, связанных с фратаксином (fxn), путем ингибирования природного антисмыслового транскрипта fxn
WO2012170947A2 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
CA2838984A1 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
EP2721156B1 (de) 2011-06-16 2016-12-21 Ionis Pharmaceuticals, Inc. Antisense-modulierung der expression des fibroblastenwachstumsfaktor-rezeptors 4
AU2012272970A1 (en) 2011-06-21 2014-02-06 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
WO2012178033A2 (en) 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
WO2013003808A1 (en) 2011-06-29 2013-01-03 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
US10100305B2 (en) 2011-07-15 2018-10-16 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation of start sites
EP2734525A4 (de) 2011-07-19 2015-03-04 Univ Idaho Ausgestaltungen einer sonde und verfahren zum anzielen von nukleinsäuren
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
DK2742136T3 (da) 2011-08-11 2017-11-20 Ionis Pharmaceuticals Inc Gapmerforbindelser omfattende 5'-modificerede deoxyribonukleosider i gap og anvendelser deraf
EP2751269B1 (de) 2011-08-29 2016-03-23 Ionis Pharmaceuticals, Inc. Verfahren und verbindungen für erkrankungen im zusammenhang mit repeat-expansion
DK2751270T3 (en) 2011-08-29 2018-10-29 Ionis Pharmaceuticals Inc OLIGOMER-CONJUGATE COMPLEXES AND THEIR USE
EA029151B1 (ru) 2011-09-06 2018-02-28 Курна, Инк. ЛЕЧЕНИЕ ЗАБОЛЕВАНИЙ, СВЯЗАННЫХ С АЛЬФА-СУБЪЕДИНИЦАМИ ПОТЕНЦИАЛЗАВИСИМЫХ НАТРИЕВЫХ КАНАЛОВ (SCNxA), С ПОМОЩЬЮ МАЛЫХ МОЛЕКУЛ
US9644241B2 (en) 2011-09-13 2017-05-09 Interpace Diagnostics, Llc Methods and compositions involving miR-135B for distinguishing pancreatic cancer from benign pancreatic disease
BR112014006587B1 (pt) 2011-09-20 2021-07-27 Ionis Pharmaceuticals, Inc Composto compreendendo oligonucleotídeo que modula a expressão de gcgr, composição, bem como seu uso no tratamento ou prevenção de diabetes
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
WO2013055865A1 (en) 2011-10-11 2013-04-18 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
US8901098B2 (en) 2011-10-25 2014-12-02 Isis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
US10202643B2 (en) 2011-10-31 2019-02-12 University Of Utah Research Foundation Genetic alterations in glioma
ES2761343T3 (es) 2011-11-07 2020-05-19 Ionis Pharmaceuticals Inc Modulación de la expresión de TMPRSS6
US9243291B1 (en) 2011-12-01 2016-01-26 Isis Pharmaceuticals, Inc. Methods of predicting toxicity
DK2788487T3 (en) 2011-12-08 2018-07-23 Sarepta Therapeutics Inc Oligonucleotide Analogs Targeted against HUMAN LMNA
WO2013090648A1 (en) 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
US9546368B2 (en) 2011-12-22 2017-01-17 Ionis Pharmaceuticals, Inc. Methods for modulating metastasis-associated-in-lung-adenocarcinoma-transcript-1 (MALAT-1) expression
EP3466959A1 (de) * 2011-12-22 2019-04-10 Janssen BioPharma, Inc. Substituierte nukleoside, nukleotide und analoga davon
EP2797948B1 (de) 2011-12-27 2019-02-20 The J. David Gladstone Institutes Zusammensetzungen zur verwendung in der regulierung des glukosemetabolismus
ES2842938T3 (es) 2012-01-11 2021-07-15 Ionis Pharmaceuticals Inc Composiciones y métodos para la modulación del empalme de IKBKAP
US20140378533A1 (en) 2012-02-08 2014-12-25 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
EP2825885B1 (de) 2012-03-12 2021-05-12 The Board of Trustees of the University of Illinois Optische analytdetektionssysteme mit magnetischer verstärkung
WO2013138374A2 (en) 2012-03-15 2013-09-19 Curna, Inc. Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2013142514A1 (en) 2012-03-19 2013-09-26 Isis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
US9441007B2 (en) 2012-03-21 2016-09-13 Alios Biopharma, Inc. Substituted nucleosides, nucleotides and analogs thereof
USRE48171E1 (en) 2012-03-21 2020-08-25 Janssen Biopharma, Inc. Substituted nucleosides, nucleotides and analogs thereof
JP6492003B2 (ja) 2012-03-30 2019-03-27 ワシントン・ユニバーシティWashington University 発作を減少させるためおよび神経変性症候群を改質するためにタウ発現を調節する方法
JP2015516143A (ja) 2012-04-02 2015-06-08 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. ヒト疾患に関連するタンパク質の産生のための修飾ポリヌクレオチド
JP2015518705A (ja) 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
US9221864B2 (en) 2012-04-09 2015-12-29 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
WO2013154799A1 (en) * 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP3336189A1 (de) 2012-04-20 2018-06-20 Ionis Pharmaceuticals, Inc. Oligomere verbindungen mit bicyclischen nukleotiden und verwendungen davon
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
US9574193B2 (en) 2012-05-17 2017-02-21 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein (a) expression
US20160002624A1 (en) 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
WO2013177248A2 (en) 2012-05-22 2013-11-28 Isis Pharmaceuticals, Inc. Modulation of enhancer rna mediated gene expression
NZ631071A (en) 2012-05-24 2017-02-24 Ionis Pharmaceuticals Inc Methods and compositions for modulating apolipoprotein(a) expression
ES2597757T3 (es) 2012-05-25 2017-01-20 Janssen Sciences Ireland Uc Nucleósidos de uracilespirooxetano
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
HUE051698T2 (hu) 2012-06-25 2021-03-29 Ionis Pharmaceuticals Inc UBE3A-ATS expresszió modulálása
EP2877579B1 (de) 2012-07-27 2019-12-18 Ionis Pharmaceuticals, Inc. Modulation von mit dem reninangiotensinsystem (ras) assoziierten erkrankungen durch angiotensinogen
EP2885312A4 (de) 2012-08-15 2016-01-20 Isis Pharmaceuticals Inc Verfahren zur herstellung oligomerer verbindungen unter verwendung von modifizierten kappungsprotokollen
EP2897633B1 (de) 2012-09-18 2020-01-01 UTI Limited Partnership Behandlung von schmerzen durch hemmung von usp5-deubiquitinase
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
WO2014059364A1 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Methods of treating kennedy's disease
US9695418B2 (en) 2012-10-11 2017-07-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
US20160138014A1 (en) 2012-10-12 2016-05-19 Isis Pharmaceuticals, Inc. Antisense compounds and uses thereof
EP3459549B1 (de) 2012-10-12 2022-04-06 Ionis Pharmaceuticals, Inc. Selektive antisense-verbindungen und verwendungen davon
PT2906696T (pt) 2012-10-15 2019-12-16 Univ California Métodos para modular a expressão de c9orf72
US10577604B2 (en) 2012-10-15 2020-03-03 Ionis Pharmaceuticals, Inc. Methods for monitoring C9ORF72 expression
US10443052B2 (en) 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
SI2920308T1 (sl) 2012-10-31 2019-05-31 Ionis Pharmaceuticals, Inc. Zdravljenje raka
US20150299803A1 (en) 2012-11-05 2015-10-22 Pronai Therapeutics, Inc. Methods of Using Biomarkers for the Treatment of Cancer by Modulation of BCL2 Expression
EP2914621B1 (de) 2012-11-05 2023-06-07 Foundation Medicine, Inc. Neuartige ntrk1-fusionsmoleküle und verwendungen davon
EP2920304B1 (de) 2012-11-15 2019-03-06 Roche Innovation Center Copenhagen A/S Oligonukleotidkonjugate
WO2014080004A1 (en) 2012-11-26 2014-05-30 Santaris Pharma A/S Compositions and methods for modulation of fgfr3 expression
LT2922554T (lt) 2012-11-26 2022-06-27 Modernatx, Inc. Terminaliai modifikuota rnr
WO2014093537A1 (en) 2012-12-11 2014-06-19 Isis Pharmaceuticals, Inc. Competitive modulation of micrornas
EA201590943A1 (ru) 2012-12-21 2016-01-29 Алиос Биофарма, Инк. Замещенные нуклеозиды, нуклеотиды и их аналоги
US9926559B2 (en) 2013-01-09 2018-03-27 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject
EP2946014A2 (de) 2013-01-17 2015-11-25 Moderna Therapeutics, Inc. Signal-sensor-polynukleotide zur veränderung zellulärer phänotypen
EP3939614A1 (de) 2013-01-18 2022-01-19 Foundation Medicine, Inc. Verfahren zur behandlung cholangiokarzinom
DK2951305T3 (en) 2013-01-30 2018-10-29 Hoffmann La Roche LNA oligonucleotide KULHYDRATKONJUGATER
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
KR102190852B1 (ko) 2013-01-31 2020-12-14 아이오니스 파마수티컬즈, 인코포레이티드 변형된 커플링 프로토콜을 이용하는 올리고머 화합물의 제조 방법
EP3778618A1 (de) 2013-02-04 2021-02-17 Ionis Pharmaceuticals, Inc. Selektive antisense-verbindungen und verwendungen davon
AU2014216137B2 (en) 2013-02-14 2018-05-10 Ionis Pharmaceuticals, Inc. Modulation of Apolipoprotein C-III (ApoCIII) expression in lipoprotein lipase deficient (LPLD) populations
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
CN105143470B (zh) 2013-02-28 2020-06-09 德克萨斯大学系统董事会 用于将癌症分类为易感于tmepai定向疗法以及治疗所述癌症的方法
EP2961386B1 (de) 2013-02-28 2019-07-10 The General Hospital Corporation Mirna-profilierungszusammensetzungen und verfahren zur verwendung
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
CN114015692A (zh) 2013-03-14 2022-02-08 阿尔尼拉姆医药品有限公司 补体组分C5 iRNA组合物及其使用方法
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US9273349B2 (en) 2013-03-14 2016-03-01 Affymetrix, Inc. Detection of nucleic acids
AU2014236156C1 (en) 2013-03-14 2020-12-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating Tau expression
US9983206B2 (en) 2013-03-15 2018-05-29 The Board Of Trustees Of The University Of Illinois Methods and compositions for enhancing immunoassays
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
NZ631512A (en) 2013-05-01 2016-10-28 Ionis Pharmaceuticals Inc Compositions and methods for modulating apolipoprotein (a) expression
EP2999786A1 (de) 2013-05-22 2016-03-30 Alnylam Pharmaceuticals, Inc. Tmprss6-irna-zusammensetzungen und verfahren zur verwendung davon
HUE038146T2 (hu) 2013-05-22 2018-09-28 Alnylam Pharmaceuticals Inc Serpina1 IRNS készítmények és eljárások alkalmazásukra
US20160113911A1 (en) 2013-06-06 2016-04-28 The General Hospital Corporation Methods and compositions for the treatment of cancer
US20160122761A1 (en) 2013-06-21 2016-05-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
US9909124B2 (en) 2013-06-21 2018-03-06 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein C-III expression for improving a diabetic profile
MY183049A (en) 2013-06-27 2021-02-09 Roche Innovation Ct Copenhagen As Antisense oligomers and conjugates targeting pcsk9
CN105452461B (zh) 2013-07-02 2021-04-13 Ionis制药公司 生长激素受体的调节剂
RS62529B1 (sr) 2013-07-11 2021-11-30 Modernatx Inc Kompozicije koje sadrže sintetičke polinukleotide koji kodiraju proteine povezane sa crispr i sintetičke sgrnk i postupci upotrebe
JP6617702B2 (ja) 2013-07-15 2019-12-11 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Fty720のアザサイクリック拘束アナログ
TWI657819B (zh) 2013-07-19 2019-05-01 美商Ionis製藥公司 用於調節τ蛋白表現之組合物
US10435430B2 (en) 2013-07-31 2019-10-08 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
EA202191796A2 (ru) 2013-08-08 2022-03-31 Дзе Скриппс Рисёч Инститьют Способ сайт-специфического ферментативного мечения нуклеиновых кислот in vitro введением не встречающихся в природе нуклеотидов
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
WO2015031679A2 (en) 2013-08-28 2015-03-05 Isis Pharmaceuticals, Inc. Modulation of prekallikrein (pkk) expression
EP3041938A1 (de) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Kreisförmige polynukleotide
WO2015034928A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
KR20210088009A (ko) 2013-09-05 2021-07-13 사렙타 쎄러퓨틱스, 인코퍼레이티드 산성 알파-글루코시다제 내 안티센스-유도된 엑손2 포함
DK3043827T3 (da) 2013-09-13 2019-08-26 Ionis Pharmaceuticals Inc Modulatorer af komplement faktor b
US9943604B2 (en) 2013-09-20 2018-04-17 Ionis Pharmaceuticals, Inc. Targeted therapeutic nucleosides and their use
CN105793423A (zh) 2013-10-02 2016-07-20 阿尔尼拉姆医药品有限公司 用于抑制lect2基因表达的组合物和方法
AU2014329452B2 (en) 2013-10-03 2019-06-20 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
NZ757749A (en) 2013-10-04 2022-07-01 Icahn School Med Mount Sinai Compositions and methods for inhibiting expression of the alas1 gene
CN112080502A (zh) 2013-10-11 2020-12-15 Ionis制药公司 用于调节c9orf72表达的组合物
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
WO2015061246A1 (en) 2013-10-21 2015-04-30 Isis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
CA2928779A1 (en) 2013-10-21 2015-04-30 The General Hospital Corporation Methods relating to circulating tumor cell clusters and the treatment of cancer
WO2015075166A1 (en) 2013-11-22 2015-05-28 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for treatment of a bacterial infection
ES2797679T3 (es) 2013-12-02 2020-12-03 Ionis Pharmaceuticals Inc Compuestos antisentido y usos de los mismos
CA3107872A1 (en) 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
WO2015095527A1 (en) 2013-12-20 2015-06-25 The General Hosptial Corporation Methods and assays relating to circulating tumor cells
JP6643996B2 (ja) 2013-12-24 2020-02-12 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. アンジオポエチン様3発現の調節
WO2015118407A2 (en) 2014-01-29 2015-08-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Oligonucleotides and methods for inhibiting or reducing bacterial biofilms
EA201691587A1 (ru) 2014-02-11 2017-01-30 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ КЕТОГЕКСОКИНАЗЫ (KHK) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
US10036019B2 (en) 2014-03-17 2018-07-31 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
CN116970607A (zh) 2014-03-19 2023-10-31 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
EP3757214B1 (de) 2014-04-01 2022-06-15 Biogen MA Inc. Zusammensetzungen zur modulierung der sod-1-expression
US10513706B2 (en) 2014-04-09 2019-12-24 The Scripps Research Institute Import of unnatural or modified nucleoside triphosphates into cells via nucleic acid triphosphate transporters
ES2932304T3 (es) 2014-04-17 2023-01-17 Biogen Ma Inc Composiciones y métodos para la modulación del empalme de SMN2 en un sujeto
WO2015164693A1 (en) 2014-04-24 2015-10-29 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
EP3137476B1 (de) 2014-04-28 2019-10-09 Ionis Pharmaceuticals, Inc. Verbindungsmodifizierte oligomerverbindungen
PT3137605T (pt) 2014-05-01 2020-12-18 Ionis Pharmaceuticals Inc Composições e métodos para modulação da expressão de angiopoietina de tipo 3
RU2703411C2 (ru) 2014-05-01 2019-10-16 Ионис Фармасьютикалз, Инк. Композиции и способы модулирования экспрессии пкп
EP3608406B1 (de) 2014-05-01 2023-02-15 Ionis Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur modulation der expression vom komplementfaktor b
KR20200102553A (ko) 2014-05-01 2020-08-31 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
EP3137115B1 (de) 2014-05-01 2020-10-14 Ionis Pharmaceuticals, Inc. Verfahren zur synthese von reaktiven konjugatclustern
WO2015175510A1 (en) 2014-05-12 2015-11-19 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating a serpinc1-associated disorder
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
SG11201609376SA (en) 2014-05-22 2016-12-29 Alnylam Pharmaceuticals Inc Angiotensinogen (agt) irna compositions and methods of use thereof
WO2015187541A1 (en) 2014-06-02 2015-12-10 Children's Medical Center Corporation Methods and compositions for immunomodulation
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US10301624B2 (en) 2014-06-25 2019-05-28 The General Hospital Corporation Targeting human satellite II (HSATII)
CA2955250A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
JP6940404B2 (ja) 2014-08-19 2021-09-29 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 核酸検出のための方法および組成物
US10385343B2 (en) 2014-08-29 2019-08-20 Children's Medical Center Corporation Methods and compositions for the treatment of cancer
EP3191591A1 (de) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete polynukleotidwirkstoffe und verfahren zur verwendung davon
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
EP3201350B1 (de) * 2014-09-30 2020-12-02 Global Life Sciences Solutions USA LLC Verfahren zur nukleinsäureanalyse direkt aus einer ungereinigten biologischen probe
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
WO2016061131A1 (en) 2014-10-14 2016-04-21 The J. David Gladstone Institutes Compositions and methods for reactivating latent immunodeficiency virus
EP3207138B1 (de) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Gegen aminolävulinsäuresynthase-1 (alas1) gerichtete polynukleotidmittel und verwendungen davon
EP3212794B1 (de) 2014-10-30 2021-04-07 Genzyme Corporation Polynukleotidwirkstoffe mit abzielung auf serpinc1 (at3) und verfahren zur verwendung davon
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
WO2016077704A1 (en) 2014-11-14 2016-05-19 The Regents Of The University Of California Modulation of agpat5 expression
WO2016081444A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
EP3221445B1 (de) 2014-11-20 2021-07-14 The Regents of The University of California Zusammensetzungen und verfahren in zusammenhang mit hämatologischer erholung
US10400243B2 (en) 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
EP3229842B1 (de) 2014-12-08 2022-07-06 The Board of Regents of The University of Texas System Lipokationische polymere und verwendungen davon
DK3234134T3 (da) 2014-12-17 2020-07-27 Proqr Therapeutics Ii Bv Målrettet rna-redigering
AU2015364508A1 (en) 2014-12-18 2017-07-06 Alnylam Pharmaceuticals, Inc. ReversirTM compounds
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2016115490A1 (en) 2015-01-16 2016-07-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
CA2976445A1 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
EP3261643B1 (de) 2015-02-26 2020-11-25 Ionis Pharmaceuticals, Inc. Allelspezifische modulatoren von p23h-rhodopsin
WO2016141236A1 (en) 2015-03-03 2016-09-09 Ionis Pharmaceuticals, Inc. Compositions for modulating mecp2 expression
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
EP3265564B1 (de) 2015-03-03 2022-01-26 Ionis Pharmaceuticals, Inc. Verfahren zur modulierung der mecp2-expression
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
EP3277817A4 (de) 2015-04-03 2018-12-05 Ionis Pharmaceuticals, Inc. Verbindungen und verfahren zur modulation der tmprss6-expression
WO2016164463A1 (en) 2015-04-07 2016-10-13 The General Hospital Corporation Methods for reactivating genes on the inactive x chromosome
US10745702B2 (en) 2015-04-08 2020-08-18 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
KR102104163B1 (ko) 2015-04-16 2020-04-23 아이오니스 파마수티컬즈, 인코포레이티드 C9orf72 발현을 조절하기 위한 조성물
MA50829A (fr) 2015-06-01 2018-04-11 Sarepta Therapeutics Inc Exclusion d'exon induite pat technologie antisens dans le collagène de type vii
EP3307316A1 (de) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete irna-zusammensetzungen und verfahren zur verwendung davon
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
EP3314250A4 (de) 2015-06-26 2018-12-05 Beth Israel Deaconess Medical Center, Inc. Auf tetraspanin 33 (tspann33) in myeloidabgeleiteten suppressorzellen abzielende krebstherapie
CA2990699A1 (en) 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
ES2917181T3 (es) 2015-07-10 2022-07-07 Ionis Pharmaceuticals Inc Moduladores de diacilglicerol aciltransferasa 2 (DGAT2)
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
JP2018520683A (ja) 2015-07-17 2018-08-02 アルニラム ファーマスーティカルズ インコーポレイテッドAlnylam Pharmaceuticals, Inc. 多標的単一体コンジュゲート
EP3331536A4 (de) 2015-08-03 2019-03-27 The Regents of The University of California Zusammensetzungen und verfahren zur modulierung der abhd2-aktivität
WO2017032726A1 (en) 2015-08-24 2017-03-02 Roche Innovation Center Copenhagen A/S Lna-g process
WO2017035278A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
EP3344769B1 (de) 2015-09-02 2024-04-17 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (pd-l1) irna-kompositionen und methoden zu deren verwendung
PT3349802T (pt) 2015-09-14 2021-10-15 Univ Texas Dendrímeros lipocatiónicos e suas utilizações
TW201718857A (zh) 2015-09-14 2017-06-01 艾爾妮蘭製藥公司 用於抑制alas1基因表現之組合物及方法
EP3353328A4 (de) 2015-09-24 2019-06-12 Ionis Pharmaceuticals, Inc. Modulatoren der kras-expression
MX2018003685A (es) 2015-09-24 2018-08-15 Univ California Moleculas tipo esfingolipidos sinteticas, farmacos, metodos de su sintesis y metodos de tratamiento.
PT3353303T (pt) 2015-09-25 2023-10-10 Academisch Ziekenhuis Leiden Composições e métodos para modulação da expressão de ataxina 3
BR122020023854B1 (pt) 2015-10-08 2022-11-29 Ionis Pharmaceuticals, Inc Composto compreendendo oligonucleotídeo modificado e seu uso, uso do oligonucleotídeo modificado, composição e seu uso
MA45819A (fr) 2015-10-09 2018-08-15 Sarepta Therapeutics Inc Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés
WO2017060731A1 (en) 2015-10-09 2017-04-13 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
WO2017068087A1 (en) 2015-10-22 2017-04-27 Roche Innovation Center Copenhagen A/S Oligonucleotide detection method
US11260073B2 (en) 2015-11-02 2022-03-01 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating C90RF72
BR122023026882A2 (pt) 2015-11-06 2024-01-23 Ionis Pharmaceuticals, Inc Uso de um composto oligomérico
WO2017079745A1 (en) 2015-11-06 2017-05-11 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy
CN116064539A (zh) 2015-11-12 2023-05-05 豪夫迈·罗氏有限公司 用于诱导父本ube3a表达的寡核苷酸
AU2016359629B2 (en) 2015-11-23 2023-03-09 Ranjan BATRA Tracking and manipulating cellular RNA via nuclear delivery of CRISPR/Cas9
WO2017096395A1 (en) 2015-12-04 2017-06-08 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3933041B1 (de) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense-oligomere zur behandlung von autosomaldominierender geistiger behinderung
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
CA3006599A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
CA3054284A1 (en) 2016-02-25 2017-08-31 The Brigham And Women's Hospital, Inc. Treatment methods for fibrosis targeting smoc2
AU2017229778A1 (en) 2016-03-09 2018-08-16 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting PMP22 expression
ES2857702T3 (es) 2016-03-14 2021-09-29 Hoffmann La Roche Oligonucleótidos para la reducción de la expresión de PD-L1
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
AU2017234678A1 (en) 2016-03-16 2018-08-16 Ionis Pharmaceuticals, Inc. Methods of modulating KEAP1
AU2017248637A1 (en) 2016-04-13 2018-09-27 Ionis Pharmaceuticals, Inc. Methods for reducing C9ORF72 expression
JP6985288B2 (ja) 2016-04-14 2021-12-22 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft トリチル−モノ−GalNAc化合物とその利用
KR102522059B1 (ko) 2016-04-18 2023-04-14 사렙타 쎄러퓨틱스 인코퍼레이티드 안티센스 올리고머, 및 산성 알파-글루코시다제 유전자와 연관된 질환을 치료하기 위한 이의 사용 방법
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
WO2017188898A1 (en) 2016-04-29 2017-11-02 Nanyang Technological University G-quadruplex-containing antisense oligonucleotides
EA201892467A1 (ru) 2016-04-29 2019-05-31 Сарепта Терапьютикс, Инк. Олигонуклеотидные аналоги, нацеленные на lmna человека
BR112018073699A2 (pt) 2016-05-16 2019-05-07 The Board Of Regents Of The University Of Texas System amino lipídios de sulfonamida catiônica e amino lipídios zwiteriônico anfifílicos
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
JP6987081B2 (ja) 2016-05-24 2021-12-22 サレプタ セラピューティクス, インコーポレイテッド ホスホロジアミダートモルホリノオリゴマーを調製するためのプロセス
EP3564248A1 (de) 2016-05-24 2019-11-06 Sarepta Therapeutics, Inc. Verfahren zur herstellung von phosphordiamidat-morpholino-oligomeren
US10905710B2 (en) 2016-05-24 2021-02-02 Emory University Particles with RNA cleaving nucleobase polymers and uses for managing inflammatory disorders
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
US20190256845A1 (en) 2016-06-10 2019-08-22 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
WO2017219017A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
MA45496A (fr) 2016-06-17 2019-04-24 Hoffmann La Roche Molécules d'acide nucléique pour la réduction de l'arnm de padd5 ou pad7 pour le traitement d'une infection par l'hépatite b
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
HUE060123T2 (hu) 2016-06-24 2023-01-28 Scripps Research Inst Új nukleozid-trifoszfát-transzporter és alkalmazásai
EP3478697A1 (de) 2016-06-30 2019-05-08 Sarepta Therapeutics, Inc. Exon-skipping-oligomere für muskeldystrophie
BR112018077321A2 (pt) 2016-07-01 2019-04-24 F. Hoffmann-La Roche Ag oligonucleotídeos anti-sentido para modular a expressão de htra1
PL3484524T3 (pl) 2016-07-15 2023-03-20 Ionis Pharmaceuticals, Inc. Związki i sposoby modulacji smn2
WO2018017814A1 (en) 2016-07-20 2018-01-25 President And Fellows Of Harvard College Peptidoglycan glycosyltransferase inhibitors of sed proteins for treating bacterial infections
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
WO2018053142A2 (en) 2016-09-14 2018-03-22 President And Fellows Of Harvard College Methods and compositions for modulating erythropoiesis
US10793859B2 (en) 2016-09-14 2020-10-06 Janssen Biopharma, Inc. Modified oligonucleotides and methods of use
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
US11400161B2 (en) 2016-10-06 2022-08-02 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
EP3532638A4 (de) 2016-10-31 2020-07-29 University of Massachusetts Targeting von microrna-101-3p in der krebstherapie
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
JP7033591B2 (ja) 2016-11-11 2022-03-10 ロシュ イノベーション センター コペンハーゲン エーエス 治療用オリゴヌクレオチドの捕捉および検出
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
US11033570B2 (en) 2016-12-02 2021-06-15 Cold Spring Harbor Laboratory Modulation of Lnc05 expression
WO2018112032A1 (en) 2016-12-13 2018-06-21 President And Fellows Of Harvard College Methods and compositions for targeting tumor-infiltrating tregs using inhibitors of ccr8 and tnfrsf8
WO2018112320A1 (en) 2016-12-16 2018-06-21 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
CN110337308B (zh) 2016-12-19 2023-09-01 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物
KR102552428B1 (ko) 2016-12-19 2023-07-06 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
WO2018118662A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3568480A1 (de) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense-oligonukleotide zur modulierung der nfkb2-expression
WO2018130585A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating relb expression
US20190338286A1 (en) 2017-01-13 2019-11-07 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rel expression
US20200216845A1 (en) 2017-01-13 2020-07-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rela expression
WO2018130583A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb1 expression
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
US11180756B2 (en) 2017-03-09 2021-11-23 Ionis Pharmaceuticals Morpholino modified oligomeric compounds
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
BR112019021852A2 (pt) 2017-04-18 2020-06-02 Alnylam Pharmaceuticals, Inc. Agente de rnai e uma vacina contra hbv, uso ou método e kit para tratamento
CN110536694A (zh) 2017-04-20 2019-12-03 Atyr 医药公司 用于治疗肺部炎症的组合物和方法
CA3062595A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
US20190055564A1 (en) 2017-06-01 2019-02-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
EP3652316A4 (de) 2017-07-11 2021-04-07 Synthorx, Inc. Einbindung von unnatürlichen nukleotiden und verfahren dafür
US11622993B2 (en) 2017-08-03 2023-04-11 Synthorx, Inc. Cytokine conjugates for the treatment of autoimmune diseases
WO2019030313A2 (en) 2017-08-11 2019-02-14 Roche Innovation Center Copenhagen A/S OLIGONUCLEOTIDES FOR MODULATION OF UBE3C EXPRESSION
CA3071033A1 (en) 2017-08-18 2019-02-21 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
WO2019038228A1 (en) 2017-08-22 2019-02-28 Roche Innovation Center Copenhagen A/S OLIGONUCLEOTIDES FOR MODULATION OF TOM1 EXPRESSION
SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
US10517889B2 (en) 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
SG11202002149XA (en) 2017-09-14 2020-04-29 Janssen Biopharma Inc Galnac derivatives
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
WO2019067981A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
EP3687547A1 (de) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Kombinationstherapien zur behandlung von muskeldystrophie
JP2020536057A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを処置するための併用療法
JP2021502059A (ja) 2017-10-13 2021-01-28 ロシュ イノベーション センター コペンハーゲン エーエス 部分的に立体定義されたオリゴヌクレオチドのサブライブラリーを使用することによる、アンチセンスオリゴヌクレオチドの、改良された立体定義されたホスホロチオエートオリゴヌクレオチド変異体を同定するための方法
MA52151A (fr) 2017-10-16 2020-05-06 Hoffmann La Roche Molécule d'acide nucléique pour la réduction de l'arnm de papd5 et de papd7 pour le traitement d'une infection par l'hépatite b
EP3697904A1 (de) 2017-10-17 2020-08-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Kombinationsbehandlung für zystische fibrose
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
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
WO2019099610A1 (en) 2017-11-16 2019-05-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
EP3714054A1 (de) 2017-11-20 2020-09-30 Alnylam Pharmaceuticals, Inc. Serum-amyloid p-komponente (apcs) irna zusammensetzungen und verfahren zu deren verwendung
WO2019115416A2 (en) 2017-12-11 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating fndc3b expression
WO2019115417A2 (en) 2017-12-12 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating rb1 expression
WO2019118916A1 (en) 2017-12-14 2019-06-20 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
CA3086343A1 (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
WO2019121838A1 (en) 2017-12-21 2019-06-27 F. Hoffmann-La Roche Ag Companion diagnostic for htra1 rna antagonists
BR112020010090A2 (pt) 2017-12-22 2020-11-03 Roche Innovation Center Copenhagen A/S oligonucleotídeo gapmer, sal farmaceuticamente aceitável do mesmo, conjugado, composição farmacêutica e uso dos mesmos
EP3728590A1 (de) 2017-12-22 2020-10-28 Roche Innovation Center Copenhagen A/S Neuartige thiophosphoramidite
AU2018386524A1 (en) 2017-12-22 2020-07-02 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorodithioate internucleoside linkage
EP3737758A1 (de) 2018-01-10 2020-11-18 Roche Innovation Center Copenhagen A/S Oligonukleotide zur modulierung der pias4-expression
EP3737761A1 (de) 2018-01-12 2020-11-18 Bristol-Myers Squibb Company Antisense oligonukleotide zum targeting von alpha-synuklein und deren verwendung
US20210095275A1 (en) 2018-01-12 2021-04-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating gsk3b expression
SG11202006526YA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
EP3737759A1 (de) 2018-01-12 2020-11-18 Roche Innovation Center Copenhagen A/S Alpha-synnuclein-antisense-oligonukleotide und verwendungen davon
CN111902537A (zh) 2018-01-15 2020-11-06 Ionis制药公司 Dnm2表达的调节剂
US20210095276A1 (en) 2018-01-17 2021-04-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating erc1 expression
WO2019141723A1 (en) 2018-01-18 2019-07-25 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting srebp1
WO2019145386A1 (en) 2018-01-26 2019-08-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating csnk1d expression
KR20200108315A (ko) 2018-02-09 2020-09-17 제넨테크, 인크. Tmem106b의 발현을 조절하기 위한 올리고뉴클레오티드
EP3752612A4 (de) 2018-02-12 2021-11-10 Ionis Pharmaceuticals, Inc. Modifizierte verbindungen und verwendungen davon
KR20200140805A (ko) 2018-02-21 2020-12-16 브리스톨-마이어스 스큅 컴퍼니 Camk2d 안티센스 올리고뉴클레오티드 및 그의 용도
MA52426A (fr) 2018-02-26 2021-06-02 Synthorx Inc Conjugués d'il-15 et leurs utilisations
TW202000199A (zh) 2018-03-02 2020-01-01 美商Ionis製藥公司 Irf4 表現之調節劑
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
AU2019233612A1 (en) 2018-03-13 2020-09-10 Janssen Pharmaceutica Nv Modified oligonucleotides for use in treatment of tauopathies
US11661601B2 (en) 2018-03-22 2023-05-30 Ionis Pharmaceuticals, Inc. Methods for modulating FMR1 expression
AU2019247645A1 (en) 2018-04-05 2020-10-15 Centre Leon Berard Use of FUBP1 inhibitors for treating hepatitis B virus infection
US11365416B2 (en) 2018-04-11 2022-06-21 Ionis Pharmaceuticals, Inc. Modulators of EZH2 expression
WO2019215067A1 (en) 2018-05-07 2019-11-14 Roche Innovation Center Copenhagen A/S Massively parallel discovery methods for oligonucleotide therapeutics
WO2019217459A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
EP3790971A1 (de) 2018-05-08 2021-03-17 Roche Innovation Center Copenhagen A/S Oligonukleotide zur modulierung der myh7-expression
EP3799604A4 (de) 2018-05-09 2022-09-07 Ionis Pharmaceuticals, Inc. Verbindungen und verfahren zur verminderung der fxi-expression
BR112020021253A2 (pt) 2018-05-09 2021-02-02 Ionis Pharmaceuticals, Inc. compostos e métodos para redução da expressão de atxn3
TW202016304A (zh) 2018-05-14 2020-05-01 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
JP2021524450A (ja) 2018-05-18 2021-09-13 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft マイクロrna関連疾患の処置のための薬学的組成物
WO2019224172A1 (en) 2018-05-25 2019-11-28 Roche Innovation Center Copenhagen A/S Novel process for making allofuranose from glucofuranose
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
JP7379387B2 (ja) 2018-06-05 2023-11-14 エフ. ホフマン-ラ ロシュ アーゲー Atxn2発現を制御するためのオリゴヌクレオチド
EP4219717A3 (de) 2018-06-13 2023-12-20 Sarepta Therapeutics, Inc. Exon-skipping-oligomere für muskeldystrophie
EP3807411A4 (de) 2018-06-14 2022-08-03 Ionis Pharmaceuticals, Inc. Verbindungen und verfahren zur verminderung der atxn3-expression
WO2019243391A1 (en) 2018-06-21 2019-12-26 F. Hoffmann-La Roche Ag Hybridizing all-lna oligonucleotides
SG11202011864XA (en) 2018-06-27 2020-12-30 Ionis Pharmaceuticals Inc Compounds and methods for reducing lrrk2 expression
WO2020007772A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
WO2020007700A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020007702A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting bcl2l11
SG11202012759XA (en) 2018-07-03 2021-01-28 Hoffmann La Roche Oligonucleotides for modulating tau expression
WO2020007826A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mbtps1
WO2020007889A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting stat1
WO2020011653A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
WO2020011743A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mafb
WO2020011744A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers5
WO2020011869A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting tlr2
WO2020011745A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers6
CA3106288A1 (en) 2018-07-13 2020-01-16 F.Hoffmann-La Roche Ag Oligonucleotides for modulating rtel1 expression
SG11202100077PA (en) 2018-07-25 2021-02-25 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn2 expression
TW202020153A (zh) 2018-07-27 2020-06-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
MX2020013270A (es) 2018-07-31 2021-02-18 Roche Innovation Ct Copenhagen As Oligonucleotidos que comprenden enlace internucleosidico de fosforotritioato.
JP7470097B2 (ja) 2018-07-31 2024-04-17 ロシュ イノベーション センター コペンハーゲン エーエス ホスホロトリチオアートヌクレオシド間結合を含むオリゴヌクレオチド
AU2019321375A1 (en) 2018-08-13 2021-03-11 Alnylam Pharmaceuticals, Inc. Hepatitis B virus (HBV) dsRNA agent compositions and methods of use thereof
EP3837367A1 (de) 2018-08-16 2021-06-23 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur hemmung der lect2-genexpression
EA202190581A1 (ru) 2018-08-20 2021-07-13 Рогкон, Инк. Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a
WO2020038976A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting usp8
WO2020038973A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting sptlc1
CN112585280A (zh) 2018-08-23 2021-03-30 罗氏创新中心哥本哈根有限公司 微小rna-134生物标志物
WO2020038971A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting vcan
WO2020043750A1 (en) 2018-08-28 2020-03-05 Roche Innovation Center Copenhagen A/S Neoantigen engineering using splice modulating compounds
EP3843845A4 (de) 2018-08-29 2022-05-11 University Of Massachusetts Hemmung von proteinkinasen zur behandlung von friedreich-ataxie
US11273137B2 (en) 2018-09-04 2022-03-15 Board Of Trustees Of Michigan State University Methods and compositions to prevent and treat disorders associated with mutations in the ODC1 gene
EP3620519A1 (de) 2018-09-04 2020-03-11 F. Hoffmann-La Roche AG Verwendung von isolierten extrazellulären vesikeln aus milch zur oralen verabreichung von oligonukleotiden
EP3620520A1 (de) 2018-09-10 2020-03-11 Universidad del Pais Vasco Neuartiges target zur behandlung einer stoffwechselerkrankung in einem individuum
US20210332367A1 (en) 2018-09-18 2021-10-28 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
TW202023573A (zh) 2018-09-19 2020-07-01 美商Ionis製藥公司 Pnpla3表現之調節劑
TW202028465A (zh) 2018-09-28 2020-08-01 美商阿尼拉製藥公司 甲狀腺素運載蛋白(TTR)iRNA組成物及其治療或預防TTR相關眼部疾病之使用方法
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
EP3873920A1 (de) 2018-11-01 2021-09-08 F. Hoffmann-La Roche AG Gegen tia1 gerichtete antisense-oligonukleotide
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
JOP20210108A1 (ar) 2018-11-15 2023-01-30 Ionis Pharmaceuticals Inc معدِّلات تعبير irf5
TW202039841A (zh) 2018-11-21 2020-11-01 美商Ionis製藥公司 用於減少朊病毒表現之化合物及方法
CN113166185A (zh) 2018-11-22 2021-07-23 罗氏创新中心哥本哈根有限公司 作为立体限定的寡核苷酸合成中的活化剂的吡啶鎓盐
WO2020109344A1 (en) 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Occular administration device for antisense oligonucleotides
WO2020109343A1 (en) 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Combination therapy for treatment of macular degeneration
WO2020123574A1 (en) 2018-12-13 2020-06-18 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
KR20210104555A (ko) 2018-12-17 2021-08-25 일루미나 케임브리지 리미티드 서열분석을 위한 프라이머 올리고뉴클레오타이드
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
EP4285929A3 (de) 2018-12-20 2024-03-06 Humabs Biomed SA Kombinierte hbv-therapie
CN113330118A (zh) 2018-12-21 2021-08-31 勃林格殷格翰国际有限公司 靶向card9的反义寡核苷酸
MX2021008628A (es) 2019-01-16 2021-11-17 Genzyme Corp Composiciones de arni para serpinc1 y metodos de uso de las mismas.
CN113365607A (zh) 2019-01-25 2021-09-07 豪夫迈·罗氏有限公司 用于口服药物递送的脂质囊泡
CA3128093A1 (en) 2019-01-31 2020-08-06 Ionis Pharmaceuticals, Inc. Modulators of yap1 expression
CN113660946A (zh) 2019-02-06 2021-11-16 新索思股份有限公司 Il-2缀合物及其使用方法
WO2020169695A1 (en) 2019-02-20 2020-08-27 Roche Innovation Center Copenhagen A/S Phosphonoacetate gapmer oligonucleotides
MX2021009949A (es) 2019-02-20 2021-09-21 Roche Innovation Ct Copenhagen As Fosforamiditas novedosas.
JP2022522430A (ja) 2019-02-26 2022-04-19 ロシュ イノベーション センター コペンハーゲン エーエス オリゴヌクレオチドの製剤化方法
TW202045724A (zh) 2019-02-27 2020-12-16 美商Ionis製藥公司 Malat1表現之調節劑
CN113507942A (zh) 2019-03-05 2021-10-15 豪夫迈·罗氏有限公司 分子的细胞内靶向
CN114040799A (zh) 2019-03-12 2022-02-11 哈佛大学校长及研究员协会 治疗癌症的方法和组合物
WO2020191377A1 (en) 2019-03-21 2020-09-24 Codiak Biosciences, Inc. Extracellular vesicle conjugates and uses thereof
CN117431244A (zh) 2019-03-29 2024-01-23 Ionis制药公司 用于调节ube3a-ats的化合物和方法
CN113906139A (zh) 2019-04-03 2022-01-07 百时美施贵宝公司 Angptl2反义寡核苷酸及其用途
CN113785060A (zh) 2019-04-04 2021-12-10 豪夫迈·罗氏有限公司 用于调节atxn2表达的寡核苷酸
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
JP2022529342A (ja) 2019-04-16 2022-06-21 ロシュ イノベーション センター コペンハーゲン エーエス ヌクレオチドp(v)モノマーを調製するための新規の方法
US20220193246A1 (en) 2019-04-18 2022-06-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
CN113767108A (zh) 2019-04-30 2021-12-07 罗氏创新中心哥本哈根有限公司 制备铼螯合mag3寡核苷酸的新方法
KR20220004675A (ko) 2019-05-03 2022-01-11 다이서나 파마수이티컬, 인크. 단축 센스 가닥을 갖는 이중 가닥 핵산 억제제 분자
KR20220036914A (ko) 2019-05-13 2022-03-23 비르 바이오테크놀로지, 인코포레이티드 B형 간염 바이러스(hbv) 감염을 치료하기 위한 조성물 및 방법
CA3138915A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
TW202111123A (zh) 2019-05-28 2021-03-16 美商Ionis製藥公司 用於減少fus 表現之化合物及方法
JP7155302B2 (ja) 2019-06-06 2022-10-18 エフ.ホフマン-ラ ロシュ アーゲー Atxn3を標的とするアンチセンスオリゴヌクレオチド
CN113939599A (zh) 2019-06-07 2022-01-14 豪夫迈·罗氏有限公司 杂交全lna寡核苷酸
US20220226269A1 (en) 2019-06-12 2022-07-21 President And Fellows Of Harvard College Methods and compositions for modulation of an interspecies gut bacterial pathway for levodopa metabolism
CA3143330A1 (en) 2019-06-14 2020-12-17 The Scripps Research Institute Reagents and methods for replication, transcription, and translation in semi-synthetic organisms
US20220251567A1 (en) 2019-07-10 2022-08-11 Inserm (Institut National De La Santè Et De La Recherche Médicale) Methods for the treatment of epilepsy
US11786546B2 (en) 2019-07-26 2023-10-17 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating GFAP
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021022109A1 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. SERPIN FAMILY F MEMBER 2 (SERPINF2) 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
JP2022544289A (ja) 2019-08-14 2022-10-17 コディアック バイオサイエンシーズ, インコーポレイテッド Stat6を標的とする細胞外小胞-aso構築物
US20230002764A1 (en) 2019-08-14 2023-01-05 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting cebp/beta
CA3147701A1 (en) 2019-08-14 2021-02-18 Codiak Biosciences, Inc. Extracellular vesicles with antisense oligonucleotides targeting kras
EP4013876A1 (de) 2019-08-14 2022-06-22 Codiak BioSciences, Inc. Extrazelluläre vesikel mit nlrp3-antagonist
WO2021030769A1 (en) 2019-08-14 2021-02-18 Codiak Biosciences, Inc. Extracellular vesicles with nras antisense oligonucleotides
CA3145924A1 (en) 2019-08-14 2021-02-18 Yi Zhang Extracellular vesicle linked to molecules and uses thereof
CN114555128A (zh) 2019-08-15 2022-05-27 新索思股份有限公司 采用il-2缀合物的免疫肿瘤学组合疗法
CN114555621A (zh) 2019-08-15 2022-05-27 Ionis制药公司 键修饰的寡聚化合物及其用途
EP4017540A1 (de) 2019-08-23 2022-06-29 Synthorx, Inc. Il-15-konjugate und verwendungen davon
EP4025694A1 (de) 2019-09-03 2022-07-13 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zum hemmen der lect2-genexpression
KR20220061158A (ko) 2019-09-10 2022-05-12 신톡스, 인크. 자가면역질환을 치료하기 위한 il-2 접합체 및 사용 방법
WO2021062058A1 (en) 2019-09-25 2021-04-01 Codiak Biosciences, Inc. Sting agonist comprising exosomes for treating neuroimmunological disorders
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
CN114728017A (zh) 2019-10-14 2022-07-08 阿斯利康(瑞典)有限公司 Pnpla3表达的调节剂
WO2021074657A1 (en) 2019-10-17 2021-04-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Combination treatment for cystic fibrosis
EP4045652A1 (de) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Irna-zusammensetzungen eines elements der solute-carrier-familie und verwendungsverfahren dafür
KR20220084399A (ko) 2019-10-22 2022-06-21 알닐람 파마슈티칼스 인코포레이티드 보체 성분 C3 iRNA 조성물 및 이의 사용 방법
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
AR120341A1 (es) 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
MX2022005251A (es) 2019-11-04 2022-06-08 Synthorx Inc Conjugados de interleuquina 10 y sus usos.
CN114945669A (zh) 2019-11-06 2022-08-26 阿尔尼拉姆医药品有限公司 肝外递送
EP4055165A1 (de) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr)-irna-zusammensetzungen und verfahren zur verwendung davon für die behandlung oder prävention ttr-assoziierter augenerkrankungen
PE20230179A1 (es) 2019-11-13 2023-02-01 Alnylam Pharmaceuticals Inc Metodos y composiciones para el tratamiento de un trastorno asociado con angiotensinogeno (agt)
US20230016983A1 (en) 2019-11-19 2023-01-19 lNSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) Antisense oligonucleotides and thier use for the treatment of cancer
EP4061945A1 (de) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3)-rnai-wirkstoffzusammensetzungen und verfahren zu deren verwendung
WO2021119226A1 (en) 2019-12-13 2021-06-17 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
WO2021126734A1 (en) 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2021123074A1 (en) 2019-12-18 2021-06-24 F. Hoffmann-La Roche Ag Methods of sequencing by synthesis using a consecutive labeling scheme
EP4077671A1 (de) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Verwendung von saraf-inhibitoren zum behandeln von hepatitis-b-virus-infektionen
WO2021122910A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of sbds inhibitors for treating hepatitis b virus infection
EP4077668A1 (de) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Verwendung von scamp3-inhibitoren zum behandeln von hepatitis-b-virus-infektionen
EP4077670A1 (de) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Verwendung von cops3-inhibitoren zum behandeln von hepatitis-b-virus-infektionen
WO2021122735A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of sept9 inhibitors for treating hepatitis b virus infection
TW202136510A (zh) 2019-12-20 2021-10-01 瑞士商赫孚孟拉羅股份公司 用於抑制scn9a表現之增強型寡核苷酸
CA3163490A1 (en) 2019-12-24 2021-07-01 F. Hoffman-La Roche Ag Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
CA3163646A1 (en) 2019-12-24 2021-07-01 F. Hoffman-La Roche Ag Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
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)
WO2021158810A1 (en) 2020-02-05 2021-08-12 Bristol-Myers Squibb Company Oligonucleotides for splice modulation of camk2d
WO2021163066A1 (en) 2020-02-10 2021-08-19 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing vegf-a expression
AU2021224778A1 (en) 2020-02-18 2022-09-29 Alnylam Pharmaceuticals, Inc. Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
TW202322824A (zh) 2020-02-18 2023-06-16 美商基利科學股份有限公司 抗病毒化合物
US20220064638A1 (en) 2020-02-28 2022-03-03 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
WO2021170697A1 (en) 2020-02-28 2021-09-02 F. Hoffmann-La Roche Ag Oligonucleotides for modulating cd73 exon 7 splicing
AU2021232598A1 (en) 2020-03-04 2022-09-08 Regeneron Pharmaceuticals, Inc. Methods and compositions for sensitization of tumor cells to immune therapy
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
JP2023516095A (ja) 2020-03-06 2023-04-17 アルナイラム ファーマシューティカルズ, インコーポレイテッド ケトヘキソキナーゼ(KHK)iRNA組成物およびその使用方法
JP2023516748A (ja) 2020-03-06 2023-04-20 アルナイラム ファーマシューティカルズ, インコーポレイテッド トランスサイレチン(ttr)の発現を阻害するための組成物および方法
WO2021184020A1 (en) 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Methods of treating neuroinflammation
WO2021184021A1 (en) 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting pmp22
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
TW202204615A (zh) 2020-03-26 2022-02-01 美商阿尼拉製藥公司 冠狀病毒iRNA組成物及其使用方法
WO2021202443A2 (en) 2020-03-30 2021-10-07 Alnylam Pharmaceucticals, Inc. Compositions and methods for silencing dnajc15 gene expression
JP2023520201A (ja) 2020-03-31 2023-05-16 ヤンセン バイオファーマ インク. オリゴヌクレオチド及び関連化合物の合成
EP4133078A1 (de) 2020-04-06 2023-02-15 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur ausschaltung der myoc-expression
EP4133076A1 (de) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Irna-zusammensetzungen aus angiotensin-konvertierendem enzym 2 (ace2) und verfahren zur verwendung davon
TW202204617A (zh) 2020-04-07 2022-02-01 美商艾爾妮蘭製藥公司 用於靜默scn9a表現之組合物及方法
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
EP4143319A1 (de) 2020-04-27 2023-03-08 Alnylam Pharmaceuticals, Inc. Apolipoprotein e (apoe) irna-wirkstoffzusammensetzungen und verfahren zur verwendung davon
BR112022021136A2 (pt) 2020-04-30 2022-11-29 Alnylam Pharmaceuticals Inc Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas
CA3181546A1 (en) 2020-05-01 2021-11-04 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atxn1
JP2023527684A (ja) 2020-05-11 2023-06-30 ジェネンテック, インコーポレイテッド 神経疾患を治療するための補体成分c1s阻害剤、並びにそれを使用する関連組成物、システム及び方法
CN115698290A (zh) 2020-05-11 2023-02-03 基因泰克公司 用于治疗神经系统疾病的补体组分4抑制剂以及使用它们的相关组合物、系统和方法
JP2023525799A (ja) 2020-05-11 2023-06-19 ストーク セラピューティクス,インク. 状態及び疾患の治療のためのopa1アンチセンスオリゴマー
WO2021231210A1 (en) 2020-05-11 2021-11-18 Genentech, Inc. Complement component c1r inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
US20210388357A1 (en) 2020-05-13 2021-12-16 Hoffmann-La Roche Inc. Oligonucleotide agonists targeting progranulin
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231685A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
EP4150076A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von methyl-cpg-bindendem protein 2 (mecp2)
WO2021231673A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
WO2021231679A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
JP2023526096A (ja) 2020-05-22 2023-06-20 エフ. ホフマン-ラ ロシュ アーゲー Card9のスプライス調節のためのオリゴヌクレオチド
US20230285357A1 (en) 2020-05-27 2023-09-14 The Regents Of The University Of California Compositions and methods for transdifferentiating cells
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
WO2021249993A1 (en) 2020-06-09 2021-12-16 Roche Innovation Center Copenhagen A/S Guanosine analogues for use in therapeutic polynucleotides
EP4162050A1 (de) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai-zusammensetzungen und verfahren zur verwendung davon zur verabreichung durch inhalation
CA3184289A1 (en) 2020-06-18 2021-12-23 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof
KR20230042023A (ko) 2020-06-24 2023-03-27 비르 바이오테크놀로지, 인코포레이티드 조작된 b형 간염 바이러스 중화 항체 및 이의 용도
AU2021296622A1 (en) 2020-06-25 2023-02-23 Synthorx, Inc. Immuno oncology combination therapy with IL-2 conjugates and anti-EGFR antibodies
AR122731A1 (es) 2020-06-26 2022-10-05 Hoffmann La Roche Oligonucleótidos mejorados para modular la expresión de fubp1
JP2023532518A (ja) 2020-06-29 2023-07-28 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 Plp1を調節するための化合物及び方法
EP4179091A1 (de) 2020-07-10 2023-05-17 Institut National De La Sante Et De La Recherche Medicale - Inserm Verfahren und zusammensetzungen zur behandlung von epilepsie
WO2022011214A1 (en) 2020-07-10 2022-01-13 Alnylam Pharmaceuticals, Inc. Circular sirnas
CN116209761A (zh) 2020-07-23 2023-06-02 豪夫迈·罗氏有限公司 靶向rna结合蛋白位点的寡核苷酸
WO2022018155A1 (en) 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
WO2022038211A2 (en) 2020-08-21 2022-02-24 F. Hoffmann-La Roche Ag Use of a1cf inhibitors for treating hepatitis b virus infection
WO2022056454A2 (en) 2020-09-14 2022-03-17 President And Fellows Of Harvard College Methods and compositions for treating hpv-positive cancers
EP4213947A2 (de) 2020-09-16 2023-07-26 President and Fellows of Harvard College Verfahren zur behandlung einer person mit fehlgeschlagener anti-pd-1/anti-pd-l1-therapie
TW202227102A (zh) 2020-09-22 2022-07-16 瑞典商阿斯特捷利康公司 治療脂肪肝病之方法
EP4217489A1 (de) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidylpeptidase 4 (dpp4)-irna-zusammensetzungen und verfahren zur verwendung davon
JP2023544413A (ja) 2020-10-05 2023-10-23 アルナイラム ファーマシューティカルズ, インコーポレイテッド Gタンパク質共役受容体75(GPR75)iRNA組成物およびその使用方法
WO2022076596A1 (en) 2020-10-06 2022-04-14 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting stat6
EP4225376A1 (de) 2020-10-09 2023-08-16 Synthorx, Inc. Immunonkologische kombinationstherapie mit il-2-konjugaten und pembrolizumab
CA3194880A1 (en) 2020-10-09 2022-04-14 Carolina E. CAFFARO Immuno oncology therapies with il-2 conjugates
EP4232581A1 (de) 2020-10-21 2023-08-30 Alnylam Pharmaceuticals, Inc. Verfahren und zusammensetzungen zur behandlung von primärer hyperoxalurie
EP4232582A1 (de) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin-5b (muc5b)-irna-zusammensetzungen und verfahren zur verwendung davon
AU2021380809A1 (en) 2020-11-13 2023-06-22 Alnylam Pharmaceuticals, Inc. COAGULATION FACTOR V (F5) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4136092A4 (de) 2020-11-18 2023-10-11 Ionis Pharmaceuticals, Inc. Verbindungen und verfahren zur modulation der angiotensinogenexpression
WO2022106695A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
EP4256053A1 (de) 2020-12-01 2023-10-11 Alnylam Pharmaceuticals, Inc. Verfahren und zusammensetzungen zur hemmung der hao1 (hydroxysäureoxidase 1 (glycolatoxidase))-genexpression
WO2022117745A1 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
WO2022117747A2 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
EP4259795A1 (de) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Gerinnungsfaktor x (f10)-irna-zusammensetzungen und verfahren zur verwendung davon
EP4259642A1 (de) 2020-12-08 2023-10-18 F. Hoffmann-La Roche AG Synthese von phosphordithioat-oligonukleotiden
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
TW202229553A (zh) 2020-12-18 2022-08-01 瑞士商赫孚孟拉羅股份公司 靶向顆粒蛋白前體之反義寡核苷酸
JP2024501662A (ja) 2020-12-22 2024-01-15 エフ. ホフマン-ラ ロシュ アーゲー Xbp1を標的とするオリゴヌクレオチド
EP4271695A2 (de) 2020-12-31 2023-11-08 Alnylam Pharmaceuticals, Inc. Auf 2'-modifiziertem nukleosid basierende oligonukleotid-prodrugs
CA3207125A1 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
EP4274896A1 (de) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Komplementkomponente 9 (c9)-irna-zusammensetzungen und verfahren zur verwendung davon
TW202246500A (zh) 2021-02-02 2022-12-01 瑞士商赫孚孟拉羅股份公司 用於抑制 rtel1 表現之增強型寡核苷酸
CA3210763A1 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
TW202245843A (zh) 2021-02-12 2022-12-01 美商欣爍克斯公司 Il-2接合物及西米普利單抗(cemiplimab)之皮膚癌組合療法
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
IL305171A (en) 2021-02-17 2023-10-01 Lonza Sales Ag EXTRACELLULAR VESILE-NLRP3 antagonist
KR20230146603A (ko) 2021-02-17 2023-10-19 론자 세일즈 아게 최적화 링커와 고정 모이어티를 통해 생물학적 활성 분자에 연결된 세포외 소포체
EP4298220A1 (de) 2021-02-25 2024-01-03 Alnylam Pharmaceuticals, Inc. Prionprotein (prnp)-irna-zusammensetzungen und verfahren zur verwendung davon
TW202302847A (zh) 2021-02-26 2023-01-16 美商艾拉倫製藥股份有限公司 己酮糖激酶(KHK)iRNA組成物及其使用方法
KR20230150843A (ko) 2021-03-04 2023-10-31 알닐람 파마슈티칼스 인코포레이티드 안지오포이에틴-유사 3(ANGPTL3) iRNA 조성물 및 이의 사용 방법
EP4305169A1 (de) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glykogensynthasekinase-3-alpha (gsk3a)-irna-zusammensetzungen und verfahren zur verwendung davon
KR20230162024A (ko) 2021-03-29 2023-11-28 알닐람 파마슈티칼스 인코포레이티드 헌팅틴(HTT) iRNA 제제 조성물 및 이의 사용 방법
IL307298A (en) 2021-03-31 2023-11-01 Entrada Therapeutics Inc Cell-penetrating circular peptides
EP4314293A1 (de) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Prolindehydrogenase 2 (prodh2)-irna-zusammensetzungen und verfahren zur verwendung davon
CA3213989A1 (en) 2021-04-01 2022-10-06 Conlin O'NEIL Extracellular vesicle compositions
CN117120444A (zh) 2021-04-16 2023-11-24 吉利德科学公司 使用酰胺制备卡巴核苷的方法
WO2022231999A1 (en) 2021-04-26 2022-11-03 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
WO2022240721A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
CA3218805A1 (en) 2021-05-10 2022-11-17 Ziqing QIAN Compositions and methods for intracellular therapeutics
WO2022240760A2 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING
WO2022245583A1 (en) 2021-05-18 2022-11-24 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
EP4341405A1 (de) 2021-05-20 2024-03-27 Korro Bio, Inc. Verfahren und zusammensetzungen für adar-vermittelte bearbeitung
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
EP4346904A1 (de) 2021-06-03 2024-04-10 Synthorx, Inc. Kopf-hals-krebskombinationstherapie mit einem il-2-konjugat und cetuximab
AR126000A1 (es) 2021-06-04 2023-08-30 Alnylam Pharmaceuticals Inc Agentes de arni del marco de lectura abierto 72 del cromosoma 9 humano (c9orf72), composiciones y métodos de uso de los mismos
AU2022288115A1 (en) 2021-06-08 2023-12-07 F. Hoffmann-La Roche Ag Oligonucleotide progranulin agonists
EP4351541A2 (de) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur behandlung oder prävention von erkrankungen im zusammenhang mit morbus stargardt und/oder retinal-bindendem protein 4 (rbp4)
CA3223192A1 (en) 2021-06-18 2022-12-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
KR20240037226A (ko) 2021-06-22 2024-03-21 아큐라스템 인코포레이티드 Pikfyve 안티센스 올리고뉴클레오티드
KR20240038967A (ko) 2021-06-23 2024-03-26 엔트라다 테라퓨틱스, 인크. Cug 반복을 표적화하기 위한 안티센스 화합물 및 방법
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
CA3225469A1 (en) 2021-06-30 2023-01-05 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
WO2023003805A1 (en) 2021-07-19 2023-01-26 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder
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
CA3227115A1 (en) 2021-07-21 2023-01-26 Wen-Hsuan Chang Unc13a antisense oligonucleotides
KR20240037293A (ko) 2021-07-23 2024-03-21 알닐람 파마슈티칼스 인코포레이티드 베타-카테닌(CTNNB1) iRNA 조성물 및 이의 사용 방법
WO2023009687A1 (en) 2021-07-29 2023-02-02 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
AU2022323090A1 (en) 2021-08-03 2024-02-01 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
WO2023014765A1 (en) 2021-08-04 2023-02-09 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
TW202334413A (zh) 2021-08-13 2023-09-01 美商艾拉倫製藥股份有限公司 第十二因子(F12)iRNA組成物及其使用方法
WO2023021046A1 (en) 2021-08-16 2023-02-23 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
IL311139A (en) 2021-09-01 2024-04-01 Entrada Therapeutics Inc Compounds and methods for skipping exon 44 in Duchenne muscular dystrophy
WO2023034870A2 (en) 2021-09-01 2023-03-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dmpk expression
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
CA3232420A1 (en) 2021-09-20 2023-03-23 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
WO2023052317A1 (en) 2021-09-29 2023-04-06 F. Hoffmann-La Roche Ag Rna editing
CA3233242A1 (en) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
AU2022364838A1 (en) 2021-10-15 2024-04-11 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
WO2023070086A1 (en) 2021-10-22 2023-04-27 Sarepta Therapeutics, Inc. Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
CA3234835A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
TW202333749A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 補體因子b(cfb)irna組成物及其使用方法
WO2023078883A1 (en) 2021-11-03 2023-05-11 F. Hoffmann-La Roche Ag Oligonucleotides for modulating apolipoprotein e4 expression
WO2023086292A2 (en) 2021-11-10 2023-05-19 University Of Rochester Gata4-targeted therapeutics for treatment of cardiac hypertrophy
WO2023083906A2 (en) 2021-11-11 2023-05-19 F. Hoffmann-La Roche Ag Pharmaceutical combinations for treatment of hbv
WO2023091704A1 (en) 2021-11-18 2023-05-25 Circularis Biotechnologies, Inc. Compositions and methods for production of circular nucleic acid molecules
WO2023092102A1 (en) 2021-11-19 2023-05-25 Sanegene Bio Usa Inc. Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof
WO2023096847A2 (en) 2021-11-24 2023-06-01 Prime Medicine, Inc. Methods and compositions for inhibiting mismatch repair
WO2023104693A1 (en) 2021-12-07 2023-06-15 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting actl6b
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
WO2023111210A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Combination of oligonucleotides for modulating rtel1 and fubp1
WO2023111336A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Oligonucleotide gba agonists
WO2023122573A1 (en) 2021-12-20 2023-06-29 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
WO2023117738A1 (en) 2021-12-20 2023-06-29 F. Hoffmann-La Roche Ag Threose nucleic acid antisense oligonucleotides and methods thereof
WO2023122762A1 (en) 2021-12-22 2023-06-29 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
WO2023141507A1 (en) 2022-01-20 2023-07-27 Genentech, Inc. Antisense oligonucleotides for modulating tmem106b expression
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023150181A1 (en) 2022-02-01 2023-08-10 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2023152369A1 (en) 2022-02-14 2023-08-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Nucleic acid mir-9 inhibitor for the treatment of cystic fibrosis
WO2023156652A1 (en) 2022-02-21 2023-08-24 F. Hoffmann-La Roche Ag Antisense oligonucleotide
WO2023212625A1 (en) 2022-04-28 2023-11-02 AcuraStem Incorporated Syf2 antisense oligonucleotides
US20240002857A1 (en) 2022-05-09 2024-01-04 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
WO2023217890A1 (en) 2022-05-10 2023-11-16 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting cfp-elk1 intergene region
WO2023220744A2 (en) 2022-05-13 2023-11-16 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2023222858A1 (en) 2022-05-18 2023-11-23 F. Hoffmann-La Roche Ag Improved oligonucleotides targeting rna binding protein sites
WO2023240277A2 (en) 2022-06-10 2023-12-14 Camp4 Therapeutics Corporation Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory rnas
WO2023242324A1 (en) 2022-06-17 2023-12-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for targeting progranulin
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024017990A1 (en) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024031101A1 (en) 2022-08-05 2024-02-08 Sanegene Bio Usa Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
WO2024040041A1 (en) 2022-08-15 2024-02-22 Dicerna Pharmaceuticals, Inc. Regulation of activity of rnai molecules
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
WO2024042489A1 (en) 2022-08-25 2024-02-29 LifeEDIT Therapeutics, Inc. Chemical modification of guide rnas with locked nucleic acid for rna guided nuclease-mediated gene editing
WO2024050261A1 (en) 2022-08-29 2024-03-07 University Of Rochester Antisense oligonucleotide-based anti-fibrotic therapeutics
EP4332221A1 (de) 2022-08-29 2024-03-06 Roche Innovation Center Copenhagen A/S Threose-nukleinsäure-antisense-oligonukleotide und verfahren dafür
WO2024052403A1 (en) 2022-09-06 2024-03-14 F. Hoffmann-La Roche Ag Double-stranded rna molecule for administration to the eye
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
WO2024073732A1 (en) 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4837312A (en) * 1985-01-25 1989-06-06 California Institute Of Technology Chelator-functionalized nucleosides and nucleotides and methods for making same
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
DE69233046T2 (de) 1991-10-24 2004-03-04 Isis Pharmaceuticals, Inc., Carlsfeld Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
US5559101A (en) * 1994-10-24 1996-09-24 Genencor International, Inc. L-ribofuranosyl nucleosides
UA63915C2 (uk) 1996-10-16 2004-02-16 Ай-Сі-Ен Фармасьютикалз, Інк. МОНОЦИКЛІЧНИЙ L-НУКЛЕОЗИД, ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ, СПОСІБ ЛІКУВАННЯ ТА СПОСІБ ІНВЕРСІЙНОГО МОДУЛЮВАННЯ АКТИВНОСТІ Th1 ТА Th2
ATE232880T1 (de) 1996-11-18 2003-03-15 Takeshi Imanishi Neue nucleotidanaloga
JP3756313B2 (ja) * 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) * 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
EP2341057A3 (de) 1997-09-12 2011-11-23 Exiqon A/S Oligonukleotidanaloga
US6436640B1 (en) * 1999-03-18 2002-08-20 Exiqon A/S Use of LNA in mass spectrometry
US6525191B1 (en) * 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides

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