JP5342881B2 - ６−修飾された二環式核酸類似体 - Google Patents

６−修飾された二環式核酸類似体 Download PDF

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Publication number
JP5342881B2
JP5342881B2 JP2008552604A JP2008552604A JP5342881B2 JP 5342881 B2 JP5342881 B2 JP 5342881B2 JP 2008552604 A JP2008552604 A JP 2008552604A JP 2008552604 A JP2008552604 A JP 2008552604A JP 5342881 B2 JP5342881 B2 JP 5342881B2
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oligomeric compound
nucleoside
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nucleosides
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Japanese (ja)
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JP2009524695A (ja
JP2009524695A5 (enExample
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イー．スワイゼ エリク
ピー．セトフ プニト
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
Isis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02P—CLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00—Technologies relating to chemical industry
    • Y02P20/50—Improvements relating to the production of bulk chemicals
    • Y02P20/55—Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2008552604A 2006-01-27 2007-01-27 ６−修飾された二環式核酸類似体 Active JP5342881B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US76272206P 2006-01-27 2006-01-27
US60/762,722 2006-01-27
US80566006P 2006-06-23 2006-06-23
US60/805,660 2006-06-23
PCT/US2007/061183 WO2007090071A2 (en) 2006-01-27 2007-01-27 6-modified bicyclic nucleic acid analogs

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2013017584A Division JP2013147498A (ja) 2006-01-27 2013-01-31 ６−修飾された二環式核酸類似体

Publications (3)

Publication Number Publication Date
JP2009524695A JP2009524695A (ja) 2009-07-02
JP2009524695A5 JP2009524695A5 (enExample) 2009-12-17
JP5342881B2 true JP5342881B2 (ja) 2013-11-13

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ID=38328111

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2008552604A Active JP5342881B2 (ja) 2006-01-27 2007-01-27 ６−修飾された二環式核酸類似体
JP2013017584A Pending JP2013147498A (ja) 2006-01-27 2013-01-31 ６−修飾された二環式核酸類似体

Family Applications After (1)

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JP2013017584A Pending JP2013147498A (ja) 2006-01-27 2013-01-31 ６−修飾された二環式核酸類似体

Country Status (13)

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US (3) US7399845B2 (enExample)
EP (3) EP1984381B1 (enExample)
JP (2) JP5342881B2 (enExample)
KR (2) KR101304071B1 (enExample)
CN (2) CN102766630B (enExample)
AT (1) ATE482965T1 (enExample)
AU (1) AU2007211080B9 (enExample)
CA (1) CA2640171C (enExample)
DE (1) DE602007009487D1 (enExample)
DK (2) DK2314594T3 (enExample)
ES (1) ES2516815T3 (enExample)
PL (2) PL1984381T3 (enExample)
WO (1) WO2007090071A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013147498A (ja) * 2006-01-27 2013-08-01 Isis Pharmaceuticals Inc ６−修飾された二環式核酸類似体

Families Citing this family (783)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7511131B2 (en) 2002-11-13 2009-03-31 Genzyme Corporation Antisense modulation of apolipoprotein B expression
DE60329220D1 (de) 2002-11-18 2009-10-22 Santaris Pharma As Antisense-entwurf
US20050287558A1 (en) 2004-05-05 2005-12-29 Crooke Rosanne M SNPs of apolipoprotein B and modulation of their expression
DE102005042073B4 (de) * 2005-08-31 2010-11-11 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Faserlaser
JP5713377B2 (ja) 2005-12-28 2015-05-07 ザ スクリプス リサーチ インスティテュート 薬物標的としての天然アンチセンスおよび非コードｒｎａ転写物
EP2505650A1 (en) * 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of PCSK9
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
DK2066684T3 (da) * 2006-05-11 2012-10-22 Isis Pharmaceuticals Inc 5´-Modificerede bicycliske nukleinsyreanaloge
US7666854B2 (en) * 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
ES2377327T5 (es) 2006-10-18 2020-04-28 Ionis Pharmaceuticals Inc Compuestos antisentido
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
EP2455471A3 (en) * 2006-11-27 2012-09-12 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
KR20090128494A (ko) 2007-03-24 2009-12-15 겐자임 코포레이션 인간 아포리포프로틴 ｂ에 상보적인 안티센스 올리고뉴클레오타이드 투여
CN101796062B (zh) * 2007-07-05 2014-07-30 Isis制药公司 6-双取代双环核酸类似物
EP2188298B1 (en) 2007-08-15 2013-09-18 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
US20110059895A1 (en) 2007-11-09 2011-03-10 Isis Pharmaceuticals, Inc. Modulation of factor 9 expression
CN102076852A (zh) 2007-11-09 2011-05-25 Isis药物公司 因子7表达的调节
EP2265627A2 (en) * 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
WO2009117589A1 (en) 2008-03-21 2009-09-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucleosides and methods for their use
EP2274423A2 (en) 2008-04-04 2011-01-19 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
WO2009124238A1 (en) * 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
EP2294181A4 (en) * 2008-05-22 2013-04-24 Isis Pharmaceuticals Inc MODULATION OF SMRT EXPRESSION
EP2323667A4 (en) * 2008-08-07 2012-07-25 Isis Pharmaceuticals Inc MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES
US8669102B2 (en) * 2008-08-14 2014-03-11 Isis Pharmaceuticals, Inc. Modulation of prion expression
EP2334319B1 (en) 2008-08-25 2017-12-06 Excaliard Pharmaceuticals, Inc. Method for reducing scarring during wound healing using antisense compounds directed to ctgf
US8946172B2 (en) * 2008-08-25 2015-02-03 Excaliard Pharmaceuticals, Inc. Method for reducing scarring during wound healing using antisense compounds directed to CTGF
MX2011002143A (es) 2008-08-25 2011-07-20 Excaliard Pharmaceuticals Inc Oligonucleotidos antisentido dirigidos contra el factor de crecimiento del tejido conectivo y usos de los mismos.
WO2010036698A1 (en) * 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP2361256B1 (en) * 2008-09-24 2013-04-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
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
US8334372B2 (en) 2008-10-15 2012-12-18 Isis Pharmaceuticals, Inc. Modulation of factor 11 expression
US8987435B2 (en) 2008-10-24 2015-03-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
WO2010064146A2 (en) 2008-12-02 2010-06-10 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
WO2010065671A2 (en) 2008-12-04 2010-06-10 Curna, Inc. Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf
CA2745811C (en) 2008-12-04 2021-07-13 Joseph Collard Treatment of tumor suppressor gene related diseases by inhibition of natural antisense transcript to the gene
JP6091752B2 (ja) 2008-12-04 2017-03-08 クルナ・インコーポレーテッド Ｅｐｏに対する天然アンチセンス転写物の抑制によるエリスロポエチン（ｅｐｏ）関連疾患の治療
WO2010076248A1 (en) 2008-12-31 2010-07-08 Santaris Pharma A/S Use of lna apob antisense oligomers for the treatment of acute coronary syndromes
WO2010090830A1 (en) * 2009-01-20 2010-08-12 Isis Pharmaceuticals, Inc. Modulation of sirt1 expression
US8536320B2 (en) 2009-02-06 2013-09-17 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
WO2010091308A2 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
KR101682735B1 (ko) 2009-02-12 2016-12-06 큐알엔에이, 인크. 뇌 유래된 신경영양성 인자 (ｂｄｎｆ)에 대한 자연 안티센스 전사체의 저해에 의한 뇌 유래된 신경영양성 인자 (ｂｄｎｆ) 관련된 질환의 치료
MX2011009751A (es) 2009-03-16 2011-09-29 Opko Curna Llc Tratamiento de enfermedades relacionadas con factor nuclear (eritroide derivado 2) tipo 2 (nrf2) por inhibicion del transcrito antisentido natural a nrf2.
WO2010107838A1 (en) 2009-03-16 2010-09-23 Isis Pharmaceuticals, Inc. Targeting apolipoprotein b for the reduction of apolipoprotein c-iii
JP5904935B2 (ja) 2009-03-17 2016-04-20 クルナ・インコーポレーテッド デルタ様１ホモログ（ｄｌｋ１）に対する天然アンチセンス転写物の抑制によるｄｌｋ１関連疾患の治療
US20120088814A1 (en) 2009-03-31 2012-04-12 Isis Pharmaceuticals, Inc. Methods of modulating an immune response to a viral infection
WO2010129799A2 (en) 2009-05-06 2010-11-11 Curna, Inc. Treatment of lipid transport and metabolism gene related diseases by inhibition of natural antisense transcript to a lipid transport and metabolism gene
EP2427552B1 (en) 2009-05-06 2016-11-16 CuRNA, Inc. Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp
CA2761248C (en) 2009-05-08 2023-03-14 Joseph Collard Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
EP2432881B1 (en) 2009-05-18 2017-11-15 CuRNA, Inc. Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor
CA2762987A1 (en) 2009-05-22 2010-11-25 Joseph Collard Treatment of transcription factor e3 (tfe3) and insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to tfe3
ES2618576T3 (es) 2009-05-28 2017-06-21 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen antivírico mediante la inhibición de una transcripción antisentido natural a un gen antivírico
CA2764822A1 (en) 2009-06-12 2010-12-16 Santaris Pharma A/S New potent anti apob antisense compounds
EP2443237B1 (en) 2009-06-16 2017-02-22 CuRNA, Inc. Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
ES2629339T3 (es) 2009-06-16 2017-08-08 Curna, Inc. Tratamiento de enfermedades relacionadas con la paraoxonasa 1 (pon1) por inhibición de transcrito antisentido natural a pon1
NZ597071A (en) 2009-06-17 2014-05-30 Isis Pharmaceuticals Inc Compositions and methods for modulation of smn2 splicing in a subject
WO2010150789A1 (ja) 2009-06-23 2010-12-29 武田薬品工業株式会社 核酸の合成法
JP6073133B2 (ja) 2009-06-24 2017-02-01 クルナ・インコーポレーテッド 腫瘍壊死因子受容体２（ｔｎｆｒ２）に対する天然アンチセンス転写物の抑制によるｔｎｆｒ２関連疾患の治療
CN102482672B (zh) 2009-06-26 2016-11-09 库尔纳公司 通过抑制唐氏综合征基因的天然反义转录物治疗唐氏综合征基因相关疾病
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
WO2011009697A1 (en) 2009-07-21 2011-01-27 Santaris Pharma A/S Antisense oligomers targeting pcsk9
JP2013500017A (ja) 2009-07-24 2013-01-07 カッパーアールエヌエー，インコーポレイテッド サ−チュイン（ｓｉｒｔ）への天然アンチセンス転写物の阻止によるサ−チュイン（ｓｉｒｔ）関連疾患の治療
KR101802536B1 (ko) 2009-08-05 2017-11-28 큐알엔에이, 인크. 인슐린 유전자 (ｉｎｓ)에 대한 자연 안티센스 전사체의 저해에 의한 인슐린 유전자 (ｉｎｓ) 관련된 질환의 치료
US9012421B2 (en) 2009-08-06 2015-04-21 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
ES2599986T3 (es) 2009-08-11 2017-02-06 Curna, Inc. Tratamiento de enfermedades relacionadas con adiponectina (ADIPOQ) mediante la inhibición de un transcrito antisentido natural de una adiponectina (ADIPOQ)
EP2982755B1 (en) 2009-08-21 2020-10-07 CuRNA, Inc. Treatment of 'c terminus of hsp70-interacting protein' (chip) related diseases by inhibition of natural antisense transcript to chip
WO2011031482A2 (en) 2009-08-25 2011-03-17 Curna, Inc. Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap
EP3626823A1 (en) 2009-09-11 2020-03-25 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression
DK2480669T3 (en) 2009-09-25 2018-02-12 Curna Inc TREATMENT OF FILAGGRIN- (FLG) RELATED DISEASES BY MODULATING FLG EXPRESSION AND ACTIVITY
US9364495B2 (en) 2009-10-20 2016-06-14 Roche Innovation Center Copenhagen A/S Oral delivery of therapeutically effective LNA oligonucleotides
WO2011054811A1 (en) 2009-11-03 2011-05-12 Santaris Pharma A/S Rna antagonists targeting hsp27 combination therapy
NO2513310T3 (enExample) 2009-12-16 2018-03-31
US9068183B2 (en) 2009-12-23 2015-06-30 Curna, Inc. Treatment of uncoupling protein 2 (UCP2) related diseases by inhibition of natural antisense transcript to UCP2
US8940708B2 (en) 2009-12-23 2015-01-27 Curna, Inc. Treatment of hepatocyte growth factor (HGF) related diseases by inhibition of natural antisense transcript to HGF
ES2585829T3 (es) 2009-12-29 2016-10-10 Curna, Inc. Tratamiento de las enfermedades relacionadas con la proteína tumoral 63 (p63) por inhibición de transcripción antisentido natural a p63
US8921334B2 (en) 2009-12-29 2014-12-30 Curna, Inc. Treatment of nuclear respiratory factor 1 (NRF1) related diseases by inhibition of natural antisense transcript to NRF1
EP2519632B1 (en) 2009-12-31 2018-04-11 CuRNA, Inc. Treatment of insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to irs2 and transcription factor e3 (tfe3)
ES2664605T3 (es) 2010-01-04 2018-04-20 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor 8 regulador del interferón (irf8) mediante inhibición del transcrito antisentido natural al gen irf8
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
CA2786071C (en) 2010-01-08 2021-01-12 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
NO2524039T3 (enExample) 2010-01-11 2018-04-28
EP2524038A4 (en) * 2010-01-12 2013-11-20 Isis Pharmaceuticals Inc MODULATION OF TRANSFORMING A GROWTH FACTOR BETA-1 EXPRESSION
US8946182B2 (en) 2010-01-25 2015-02-03 Curna, Inc. Treatment of RNASE H1 related diseases by inhibition of natural antisense transcript to RNASE H1
EP2534262B1 (en) 2010-02-08 2016-12-14 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
AU2011213562B2 (en) 2010-02-08 2016-07-07 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
CA2790506A1 (en) 2010-02-22 2011-08-25 Curna, Inc. Treatment of pyrroline-5-carboxylate reductase 1 (pycr1) related diseases by inhibition of natural antisense transcript to pycr1
US20110213011A1 (en) * 2010-02-26 2011-09-01 Dean Nicholas M Modulation of smad3 expression
WO2011115817A1 (en) 2010-03-16 2011-09-22 Isis Pharmaceuticals, Inc. Methods of preparing 2'-o-substituted purine nucleosides
US9193752B2 (en) 2010-03-17 2015-11-24 Isis Pharmaceuticals, Inc. 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
KR101877065B1 (ko) 2010-04-02 2018-07-10 큐알엔에이, 인크. 집락 자극 인자 3 (ｃｓｆ3)에 대한 자연 안티센스 전사체의 저해에 의한 집락 자극 인자 3 (ｃｓｆ3) 관련된 질환의 치료
EP3578657B1 (en) 2010-04-06 2024-03-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of cd274/pd-l1 gene
CN102858979B (zh) 2010-04-09 2018-01-26 库尔纳公司 通过抑制成纤维细胞生长因子21(fgf21)的天然反义转录物而治疗fgf21相关疾病
WO2011139699A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
US8993738B2 (en) 2010-04-28 2015-03-31 Isis Pharmaceuticals, Inc. Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
US9156873B2 (en) 2010-04-28 2015-10-13 Isis Pharmaceuticals, Inc. Modified 5′ diphosphate nucleosides and oligomeric compounds prepared therefrom
WO2011139917A1 (en) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression
WO2011139911A2 (en) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Lipid formulated single stranded rna
CN107988228B (zh) 2010-05-03 2022-01-25 库尔纳公司 通过抑制沉默调节蛋白(sirt)的天然反义转录物而治疗沉默调节蛋白(sirt)相关疾病
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
EP2576784B1 (en) 2010-05-26 2017-11-15 CuRNA, Inc. Treatment of methionine sulfoxide reductase a (msra) related diseases by inhibition of natural antisense transcript to msra
JP5973996B2 (ja) 2010-05-26 2016-08-23 カッパーアールエヌエー，インコーポレイテッド Ａｔｏｈ１に対する天然アンチセンス転写物の阻害による無調ホモログ１（ａｔｏｈ１）関連疾患の治療
EP2576579B1 (en) 2010-06-02 2018-08-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
WO2011156278A1 (en) 2010-06-07 2011-12-15 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2580228B1 (en) * 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011159836A2 (en) 2010-06-15 2011-12-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
DK2585596T3 (da) 2010-06-23 2021-04-06 Curna Inc Behandling af spændingsreguleret natriumkanal-alpha-underenhed (scna)-relaterede sygdomme ved inhibering af naturligt antisense-transkript til scna
WO2012007477A1 (en) 2010-07-12 2012-01-19 Santaris Pharma A/S Anti hcv oligomers
NO2593547T3 (enExample) 2010-07-14 2018-04-14
DK3031920T3 (da) 2010-07-19 2019-10-14 Ionis Pharmaceuticals Inc Modulation af dystrofi myotonica-protein kinase (dmpk) ekspression
EP2601611B1 (en) 2010-08-02 2020-12-09 Integrated Dna Technologies, Inc. Methods for predicting stability and melting temperatures of nucleic acid duplexes
WO2012034942A1 (en) 2010-09-13 2012-03-22 Santaris Pharma A/S Compounds for the modulation of aurora kinase b expression
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
WO2012047956A2 (en) 2010-10-06 2012-04-12 Opko Curna Llc Treatment of sialidase 4 (neu4) related diseases by inhibition of natural antisense transcript to neu4
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
CA2815212A1 (en) 2010-10-22 2012-04-26 Curna, Inc. Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
WO2012064758A2 (en) 2010-11-08 2012-05-18 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
JP6126009B2 (ja) 2010-11-17 2017-05-10 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． α−シヌクレイン発現の調節
US10000752B2 (en) 2010-11-18 2018-06-19 Curna, Inc. Antagonat compositions and methods of use
WO2012066093A1 (en) 2010-11-19 2012-05-24 Santaris Pharma A/S Compounds for the modulation of pdz-binding kinase (pbk) expression
WO2012066092A1 (en) 2010-11-19 2012-05-24 Santaris Pharma A/S Compounds for the modulation of aurora kinase a expression
CN103459599B (zh) 2010-11-23 2017-06-16 库尔纳公司 通过抑制nanog的天然反义转录物而治疗nanog相关疾病
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
US9127275B2 (en) 2010-12-10 2015-09-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
US9173894B2 (en) 2011-02-02 2015-11-03 Excaliard Pharamaceuticals, Inc. Method of treating keloids or hypertrophic scars using antisense compounds targeting connective tissue growth factor (CTGF)
EP3067421B1 (en) 2011-02-08 2018-10-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
WO2012110457A2 (en) 2011-02-14 2012-08-23 Santaris Pharma A/S Compounds for the modulation of osteopontin expression
JP6108628B2 (ja) 2011-03-29 2017-04-05 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｔｍｐｒｓｓ６遺伝子の発現を阻害する組成物および方法
WO2012135736A2 (en) 2011-04-01 2012-10-04 Isis Pharmaceuticals, Inc. Modulation of signal transducer and activator of transcription 3 (stat3) expression
KR101839177B1 (ko) 2011-04-13 2018-03-15 아이오니스 파마수티컬즈, 인코포레이티드 Ｐｔｐｉｂ 발현의 안티센스 조절
WO2012143427A1 (en) 2011-04-19 2012-10-26 Santaris Pharma A/S Anti polyomavirus compounds
CR20190207A (es) 2011-04-21 2019-06-26 Ionis Pharmaceuticals Inc MODULACIÓN DE LA EXPRESIÓN DEL VIRUS DE LA HEPATITIS B (VHB) (Divisional 2013-0551)
ES2868950T3 (es) 2011-04-25 2021-10-22 Sanofi Sa Compuestos de microARN y métodos para modular la actividad de miR-21
DK2701713T3 (en) 2011-04-27 2018-04-16 Ionis 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
JP6188686B2 (ja) 2011-06-09 2017-08-30 カッパーアールエヌエー，インコーポレイテッド フラタキシン（ｆｘｎ）への天然アンチセンス転写物の阻害によるｆｘｎ関連疾患の治療
WO2012170945A2 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
WO2012170947A2 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
EP2721156B1 (en) 2011-06-16 2016-12-21 Ionis Pharmaceuticals, Inc. Antisense modulation of fibroblast growth factor receptor 4 expression
CN107287202B (zh) 2011-06-21 2021-03-16 阿尔尼拉姆医药品有限公司 血管生成素样3(ANGPTL3)iRNA组合物及其使用方法
KR20140051271A (ko) 2011-06-23 2014-04-30 스텔라 에이피에스 Hcv 조합 치료
EP3597750B1 (en) 2011-06-23 2022-05-04 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
US9322021B2 (en) 2011-06-29 2016-04-26 Ionis Pharmaceuticals, Inc. Methods for modulating kallikrein (KLKB1) expression
JP2014523429A (ja) 2011-06-30 2014-09-11 ステラ・アンパルトセルスカブ Ｈｃｖ併用療法
JP2014520772A (ja) 2011-06-30 2014-08-25 ステラ・アンパルトセルスカブ Ｈｃｖ併用療法
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
JP2014526887A (ja) 2011-08-01 2014-10-09 アルナイラム ファーマシューティカルズ， インコーポレイテッド 造血幹細胞移植の成功率を改善する方法
WO2013022966A1 (en) 2011-08-11 2013-02-14 Isis Pharmaceuticals, Inc. Linkage modified gapped oligomeric compounds and uses thereof
US9976138B2 (en) 2011-08-29 2018-05-22 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
BR112014005234A2 (pt) 2011-09-06 2017-04-11 Curna Inc tratamento de doenças relacionadas com subunididades alfa dos canais de sódio dependentes de voltagem (scnxa) com pequenas moléculas
EP3533873A1 (en) 2011-09-14 2019-09-04 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
CN103814132B (zh) 2011-09-20 2018-06-05 苏州瑞博生物技术有限公司 Gcgr表达的反义调节
JP2015501155A (ja) 2011-10-25 2015-01-15 アイシス ファーマシューティカルズ， インコーポレーテッド Ｇｃｃｒ発現のアンチセンス調整
EP2773777B1 (en) 2011-10-31 2020-05-13 University of Utah Research Foundation Genetic alterations in glioblastoma
ES2607681T3 (es) 2011-11-07 2017-04-03 Roche Innovation Center Copenhagen A/S Método de pronóstico para comprobar la eficacia de inhibidores de micro ARN-122 en pacientes VHC+
WO2013068348A1 (en) 2011-11-07 2013-05-16 Santaris Pharma A/S Lna oligomers for improvement in hepatic function
HRP20191883T1 (hr) 2011-11-07 2019-12-27 Ionis Pharmaceuticals, Inc. Modulacija ekspresije tmprss6
CN103946380A (zh) 2011-11-11 2014-07-23 桑塔瑞斯制药公司 用于调控smn2剪接的化合物
US9243291B1 (en) 2011-12-01 2016-01-26 Isis Pharmaceuticals, Inc. Methods of predicting toxicity
WO2013090457A2 (en) 2011-12-12 2013-06-20 Oncoimmunin Inc. In vivo delivery of oligonucleotides
RU2649364C2 (ru) 2011-12-16 2018-04-02 Модерна Терапьютикс, Инк. Составы на основе модифицированного нуклеозида, нуклеотида и нуклеиновой кислоты
ES2673960T3 (es) 2011-12-22 2018-06-26 Ionis Pharmaceuticals, Inc. Métodos para modular la expresión de un transcrito-1 de adenocarcinoma en pulmón asociado a metástasis (MALAT-1)
IL321422A (en) 2012-01-11 2025-08-01 Ionis Pharmaceuticals Inc Compositions and methods for modulation of ikbkap splicing
WO2013119602A1 (en) 2012-02-06 2013-08-15 President And Fellows Of Harvard College Arrdc1-mediated microvesicles (armms) and uses thereof
US20140378533A1 (en) 2012-02-08 2014-12-25 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
ES2694592T3 (es) 2012-03-15 2018-12-21 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado del cerebro (BDNF) por inhibición del transcrito antisentido natural de BDNF
US9340784B2 (en) 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
WO2013148283A1 (en) 2012-03-30 2013-10-03 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
EP3520821A1 (en) 2012-04-02 2019-08-07 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
CN108949772A (zh) 2012-04-02 2018-12-07 现代泰克斯公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
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
US9914922B2 (en) 2012-04-20 2018-03-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US8969317B2 (en) 2012-04-25 2015-03-03 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-21 activity
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
CN104583398A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节基因表达的组合物和方法
US9574193B2 (en) 2012-05-17 2017-02-21 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein (a) expression
WO2013173789A2 (en) 2012-05-17 2013-11-21 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
EP2852606B1 (en) 2012-05-22 2019-08-07 Ionis Pharmaceuticals, Inc. Modulation of enhancer rna mediated gene expression
KR20190084354A (ko) 2012-05-24 2019-07-16 아이오니스 파마수티컬즈, 인코포레이티드 아포지질단백질 (a) 발현을 조절하는 방법들 및 조성물들
US9487780B2 (en) 2012-06-01 2016-11-08 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
WO2013181665A1 (en) 2012-06-01 2013-12-05 Isis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
EP3461895B1 (en) 2012-06-25 2020-07-01 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
JP6453212B2 (ja) 2012-07-13 2019-01-16 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． キラル制御
CA2879066C (en) 2012-07-13 2019-08-13 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
EP2877579B1 (en) 2012-07-27 2019-12-18 Ionis Pharmaceuticals, Inc. Modulation of renin-angiotensin system (ras) related diseases by angiotensinogen
KR102237882B1 (ko) 2012-08-15 2021-04-07 아이오니스 파마수티컬즈, 인코포레이티드 변형된 캡핑 프로토콜을 이용하는 올리고머 화합물 제조 방법
EP4052709A1 (en) 2012-10-11 2022-09-07 Ionis Pharmaceuticals, Inc. Methods of treating kennedy's disease
WO2014059353A2 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
EP2906256B1 (en) 2012-10-12 2018-08-22 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
DK2906696T4 (da) 2012-10-15 2023-02-27 Ionis Pharmaceuticals Inc Fremgangsmåder til modulering af c9orf72-ekspression
HK1212597A1 (zh) 2012-10-15 2016-06-17 Isis Pharmaceuticals, Inc. 用於調節c90rf72表達的組合物
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2014066239A1 (en) 2012-10-22 2014-05-01 Idenix Pharmaceuticals, Inc. 2',4'-bridged nucleosides for hcv infection
RS58576B1 (sr) 2012-10-31 2019-05-31 Ionis Pharmaceuticals Inc Tretman raka
AU2013337277B2 (en) 2012-11-05 2018-03-08 Foundation Medicine, Inc. Novel NTRK1 fusion molecules and uses thereof
CA2890346A1 (en) 2012-11-05 2014-05-08 Foundation Medicine, Inc. Novel fusion molecules and uses thereof
WO2014076196A1 (en) 2012-11-15 2014-05-22 Santaris Pharma A/S Anti apob antisense conjugate compounds
SI2922554T1 (sl) 2012-11-26 2022-06-30 Modernatx, Inc. Terminalno modificirana RNA
BR112015012051A2 (pt) 2012-11-26 2017-12-12 Roche Innovation Ct Copenhagen As composições e métodos para modulação de expressão de fgfr3
WO2014093537A1 (en) 2012-12-11 2014-06-19 Isis Pharmaceuticals, Inc. Competitive modulation of micrornas
US20150337002A1 (en) * 2013-01-15 2015-11-26 Shionogi & Co., Ltd. Nucleoside and nucleotide having sulfonamide structure
EP3434774A1 (en) 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
EP3939614A1 (en) 2013-01-18 2022-01-19 Foundation Medicine, Inc. Methods of treating cholangiocarcinoma
CA2893801A1 (en) 2013-01-30 2014-08-07 F. Hoffmann-La Roche Ag Lna oligonucleotide carbohydrate conjugates
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
DK2951191T3 (en) 2013-01-31 2019-01-14 Ionis Pharmaceuticals Inc PROCEDURE FOR MANUFACTURING OLIGOMERIC COMPOUNDS USING MODIFIED CLUTCH PROTOCOLS
CA2899924A1 (en) 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
RU2018121529A (ru) 2013-02-14 2019-03-06 Ионис Фармасьютикалз, Инк. МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD)
WO2014130922A1 (en) 2013-02-25 2014-08-28 Trustees Of Boston University Compositions and methods for treating fungal infections
WO2014159813A1 (en) 2013-03-13 2014-10-02 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
WO2014143158A1 (en) 2013-03-13 2014-09-18 The Broad Institute, Inc. Compositions and methods for labeling of agents
US9273349B2 (en) 2013-03-14 2016-03-01 Affymetrix, Inc. Detection of nucleic acids
ME03043B (me) 2013-03-14 2018-10-20 Alnylam Pharmaceuticals Inc Irnk sastavi komponente komplementa c5 i metode za njihovu upotrebu
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
RU2745324C2 (ru) 2013-03-14 2021-03-23 Ионис Фармасьютикалз, Инк. Композиции и способы модулирования экспрессии tau
JP2016519083A (ja) 2013-03-14 2016-06-30 アンデス バイオテクノロジーズ ソシエダード アノニマＡｎｄｅｓ Ｂｉｏｔｅｃｈｎｏｌｏｇｉｅｓ Ｓ．Ａ． 多発性骨髄腫を検出および処置するための方法
JP6313419B2 (ja) 2013-03-14 2018-04-18 アンデス バイオテクノロジーズ グローバル， インコーポレイテッド 癌幹細胞の処置のためのアンチセンスオリゴヌクレオチド
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014172698A1 (en) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
WO2014179445A1 (en) 2013-05-01 2014-11-06 Regulus Therapeutics Inc. Compounds and methods for enhanced cellular uptake
RU2670614C9 (ru) 2013-05-01 2018-11-23 Ионис Фармасьютикалз, Инк. Композиции и способы модулирования экспрессии hbv и ttr
US9309513B2 (en) 2013-05-01 2016-04-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-122
HUE038146T2 (hu) 2013-05-22 2018-09-28 Alnylam Pharmaceuticals Inc Serpina1 IRNS készítmények és eljárások alkalmazásukra
CN105452463B (zh) 2013-05-22 2019-06-21 阿尔尼拉姆医药品有限公司 Tmprss6 irna组合物及其使用方法
US20160130582A1 (en) 2013-05-24 2016-05-12 Roche Innovation Center Copenhagen A/S Oligonucleotide modulators of b-cell cll/lymphoma 11a (bcl11a) and uses thereof
EP3004396B1 (en) 2013-06-06 2019-10-16 The General Hospital Corporation Compositions for the treatment of cancer
WO2014205451A2 (en) 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
EP3656386A1 (en) 2013-06-21 2020-05-27 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile
RS59986B1 (sr) 2013-06-27 2020-03-31 Roche Innovation Ct Copenhagen As Antisens oligomeri i konjugati koji ciljno deluju na pcsk9
ES2787600T3 (es) 2013-07-02 2020-10-16 Ionis Pharmaceuticals Inc Moduladores del receptor de la hormona del crecimiento
SMT202100691T1 (it) 2013-07-11 2022-01-10 Modernatx Inc Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso
EP3022176B8 (en) 2013-07-15 2019-12-25 The Regents of the University of California Azacyclic constrained analogs of fty720
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
WO2015017675A2 (en) 2013-07-31 2015-02-05 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
SI3041854T1 (sl) 2013-08-08 2020-03-31 The Scripps Research Institute Postopek za mestnospecifično encimsko označevanje nukleinskih kislin in vitro z vključevanjem nenaravnih nukleotidov
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
CN105517556B (zh) 2013-08-28 2021-02-02 Ionis制药公司 前激肽释放酶(pkk)表达的调节
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
HRP20191600T1 (hr) 2013-09-13 2019-11-29 Ionis Pharmaceuticals Inc Modulatori faktora b komplementa
WO2015042447A1 (en) 2013-09-20 2015-03-26 Isis Pharmaceuticals, Inc. Targeted therapeutic nucleosides and their use
US10077444B2 (en) 2013-10-02 2018-09-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
EP3693463A1 (en) 2013-10-04 2020-08-12 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
SG11201602597YA (en) 2013-10-11 2016-05-30 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
US9758546B2 (en) 2013-10-21 2017-09-12 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
JP2016536303A (ja) 2013-10-21 2016-11-24 ザ ジェネラル ホスピタル コーポレイション 末梢循環腫瘍細胞クラスターおよび癌処置に関する方法
US9994846B2 (en) 2013-10-25 2018-06-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-21 activity
DK3066219T3 (en) 2013-11-08 2019-03-11 Ionis Pharmaceuticals Inc METHODS FOR DETECTING OIGONUCLEOTIDES
US20170268000A1 (en) 2013-12-02 2017-09-21 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
CA2844640A1 (en) 2013-12-06 2015-06-06 The University Of British Columbia Method for treatment of castration-resistant prostate cancer
KR102736538B1 (ko) 2013-12-12 2024-12-03 알닐람 파마슈티칼스 인코포레이티드 보체 성분 ｉＲＮＡ 조성물 및 이의 이용 방법
WO2015095527A1 (en) 2013-12-20 2015-06-25 The General Hosptial Corporation Methods and assays relating to circulating tumor cells
NZ720286A (en) 2013-12-24 2022-12-23 Ionis Pharmaceuticals Inc Modulation of angiopoietin-like 3 expression
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
KR20230152178A (ko) 2014-01-16 2023-11-02 웨이브 라이프 사이언시스 리미티드 키랄 디자인
EP3960860A3 (en) 2014-02-11 2022-06-08 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
US10036019B2 (en) 2014-03-17 2018-07-31 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
ES2890677T3 (es) 2014-03-19 2022-01-21 Ionis Pharmaceuticals Inc Composiciones para modular la expresión de ataxina 2
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
NZ724100A (en) 2014-04-01 2022-12-23 Biogen Ma Inc Compositions for modulating sod-1 expression
EP3129493B1 (en) 2014-04-09 2021-07-07 The Scripps Research Institute Import of unnatural or modified nucleoside triphosphates into cells via nucleic acid triphosphate transporters
EP4162940A1 (en) 2014-04-17 2023-04-12 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
WO2015164693A1 (en) 2014-04-24 2015-10-29 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
EP3647318B1 (en) 2014-04-28 2021-06-30 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
ES2849600T3 (es) 2014-05-01 2021-08-19 Ionis Pharmaceuticals Inc Conjugados de oligonucleótidos antisentido modificados y su uso para modular la expresión de PKK
US9382540B2 (en) 2014-05-01 2016-07-05 Isis Pharmaceuticals, Inc Compositions and methods for modulating angiopoietin-like 3 expression
CA2943894A1 (en) 2014-05-01 2015-11-05 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating complement factor b expression
JP6667453B2 (ja) 2014-05-01 2020-03-18 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 成長ホルモン受容体発現を調節するための組成物及び方法
EP4223315A3 (en) 2014-05-01 2023-08-23 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
WO2015175510A1 (en) 2014-05-12 2015-11-19 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating a serpinc1-associated disorder
GB201408623D0 (en) 2014-05-15 2014-07-02 Santaris Pharma As Oligomers and oligomer conjugates
EP3739048A1 (en) 2014-05-22 2020-11-18 Alnylam Pharmaceuticals, Inc. Angiotensinogen (agt) irna compositions and methods of use thereof
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3148564B1 (en) 2014-06-02 2020-01-08 Children's Medical Center Corporation Methods and compositions for immunomodulation
USD766120S1 (en) 2014-06-09 2016-09-13 Gambro Lundia Ab Status light bar
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20170210788A1 (en) 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
US9487783B2 (en) 2014-08-07 2016-11-08 Regulus Therapeutics Inc. Targeting microRNAs for metabolic disorders
WO2016024205A1 (en) 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
EP3185910A4 (en) 2014-08-29 2018-01-10 Children's Medical Center Corporation Methods and compositions for the treatment of cancer
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
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))
RU2017114156A (ru) 2014-10-10 2018-11-15 Ф. Хоффманн-Ля Рош Аг N-ацетилгалактозаминовые (galnac) фосфорамитиды, их конъюгаты с нуклеиновыми кислотами и их применение
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3212794B1 (en) 2014-10-30 2021-04-07 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
US10287584B2 (en) 2014-11-12 2019-05-14 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of COMP
WO2016077704A1 (en) 2014-11-14 2016-05-19 The Regents Of The University Of California Modulation of agpat5 expression
EP3020813A1 (en) 2014-11-16 2016-05-18 Neurovision Pharma GmbH Antisense-oligonucleotides as inhibitors of TGF-R signaling
WO2016081444A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
US10400243B2 (en) 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
EP4088741A1 (en) 2014-12-08 2022-11-16 The Board of Regents of the University of Texas System Lipocationic polymers and uses thereof
CN107208092B (zh) 2014-12-16 2021-09-10 罗氏创新中心哥本哈根有限公司 手性毒性筛选方法
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
CN115181778A (zh) 2015-02-04 2022-10-14 百时美施贵宝公司 选择治疗性分子的方法
US11077132B2 (en) 2015-02-04 2021-08-03 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
WO2016130943A1 (en) 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
WO2016130806A2 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
AU2016222546B2 (en) 2015-02-26 2020-01-23 Ionis Pharmaceuticals, Inc. Allele specific modulators of P23H rhodopsin
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
EP3265564B1 (en) 2015-03-03 2022-01-26 Ionis Pharmaceuticals, Inc. Methods for modulating mecp2 expression
EP4636085A2 (en) 2015-03-03 2025-10-22 Ionis Pharmaceuticals, Inc. Compositions for modulating mecp2 expression
CA2981068C (en) 2015-03-26 2021-12-14 Women & Infants Hospital Of Rhode Island Therapy for malignant disease comprising the inhibition of human epididymal secretory protein e4 and immune checkpoint inhibitors
RU2017137410A (ru) 2015-04-03 2019-05-06 Ионис Фармасьютикалз, Инк. Соединения и способы модулирования экспрессии tmprss6
US10745702B2 (en) 2015-04-08 2020-08-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
US10407678B2 (en) 2015-04-16 2019-09-10 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
SG11201708468YA (en) 2015-04-16 2017-11-29 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
EP4365291A3 (en) 2015-06-12 2024-08-14 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016205681A1 (en) 2015-06-19 2016-12-22 University Of Rochester Septin proteins as novel biomarkers for detection and treatment of müllerian cancers
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
AU2016285724A1 (en) 2015-06-29 2017-11-16 Ionis Pharmaceuticals, Inc. Modified CRISPR RNA and modified single CRISPR RNA and uses thereof
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
ES2917181T3 (es) 2015-07-10 2022-07-07 Ionis Pharmaceuticals Inc Moduladores de diacilglicerol aciltransferasa 2 (DGAT2)
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017024111A1 (en) 2015-08-04 2017-02-09 The University Of Chicago Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
JP6835826B2 (ja) 2015-08-24 2021-02-24 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 Ｌｎａ−ｇプロセス
JP7028764B2 (ja) 2015-09-02 2022-03-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド プログラム細胞死１リガンド１（ＰＤ－Ｌ１）ｉＲＮＡ組成物およびその使用方法
ES2899308T3 (es) 2015-09-14 2022-03-10 Univ Texas Dendrímeros lipocatiónicos y usos de los mismos
CA2999177A1 (en) 2015-09-24 2017-03-30 The Regents Of The University Of California Synthetic sphingolipid-like molecules, drugs, methods of their synthesis and methods of treatment
CN108513588A (zh) 2015-09-24 2018-09-07 Ionis制药公司 Kras表达的调节剂
WO2017053781A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
JP2018530325A (ja) 2015-10-08 2018-10-18 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． アンジオテンシノーゲンの発現を調節するための化合物及び方法
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
US11078528B2 (en) 2015-10-12 2021-08-03 Advanced Cell Diagnostics, Inc. In situ detection of nucleotide variants in high noise samples, and compositions and methods related thereto
WO2017068087A1 (en) 2015-10-22 2017-04-27 Roche Innovation Center Copenhagen A/S Oligonucleotide detection method
EP3394258B1 (en) 2015-10-22 2021-09-22 Roche Innovation Center Copenhagen A/S In vitro toxicity screening assay
WO2017075038A1 (en) 2015-10-26 2017-05-04 Rana Therapeutics, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
CN113952353A (zh) 2015-11-06 2022-01-21 Ionis制药公司 调节载脂蛋白(a)表达
ES2989704T3 (es) 2015-11-06 2024-11-27 Ionis Pharmaceuticals Inc Compuestos antisentido conjugados para uso terapéutico
PE20181157A1 (es) 2015-11-12 2018-07-19 Hoffmann La Roche Oligonucleotidos para inducir la expresion paterna de ube3a
EP3389670A4 (en) 2015-12-04 2020-01-08 Ionis Pharmaceuticals, Inc. METHODS OF TREATING BREAST CANCER
RU2021124592A (ru) 2015-12-07 2021-11-01 Джензим Корпорейшн Способы и композиции для лечения ассоциированного с serpinc1 расстройства
ES2882500T3 (es) 2015-12-14 2021-12-02 Cold Spring Harbor Laboratory Oligómeros antisentido para el tratamiento del síndrome de Dravet
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
WO2017111137A1 (ja) * 2015-12-22 2017-06-29 味の素株式会社 オリゴヌクレオチドの製造方法
US20190002887A1 (en) 2015-12-31 2019-01-03 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
WO2017120365A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
EP4354140A3 (en) 2016-02-26 2024-07-24 The Board of Trustees of the Leland Stanford Junior University Multiplexed single molecule rna visualization with a two-probe proximity ligation system
US11364258B2 (en) 2016-03-04 2022-06-21 Rhode Island Hospital Methods for treating chondrosarcoma using microrna(miR)
US11136577B2 (en) 2016-03-09 2021-10-05 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting PMP22 expression
PE20230157A1 (es) 2016-03-14 2023-02-01 Hoffmann La Roche Oligonucleotidos para reducir la expresion 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
US11267843B2 (en) 2016-03-18 2022-03-08 Roche Innovation Center Copenhagen A/S Stereodefining L-monomers
CA3017532A1 (en) 2016-04-13 2017-10-19 Ionis Pharmaceuticals, Inc. Methods for reducing c9orf72 expression
WO2017178656A1 (en) 2016-04-14 2017-10-19 Roche Innovation Center Copenhagen A/S TRITYL-MONO-GalNAc COMPOUNDS AND THEIR USE
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
AU2017260003B2 (en) 2016-05-06 2019-12-05 Astrazeneca Ab GLP-1 receptor ligand moiety conjugated oligonucleotides and uses thereof
WO2017194498A1 (en) 2016-05-12 2017-11-16 Roche Innovation Center Copenhagen A/S Enhanced coupling of stereodefined oxazaphospholidine phosphoramidite monomers to nucleoside or oligonucleotide
CN109414408B (zh) 2016-05-16 2022-03-29 得克萨斯州大学系统董事会 阳离子磺酰胺氨基脂质和两亲性两性离子氨基脂质
JP6876383B2 (ja) * 2016-06-07 2021-05-26 富士通オプティカルコンポーネンツ株式会社 波長可変光源
EP3469083A1 (en) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
KR102461234B1 (ko) 2016-06-14 2022-10-28 바이오젠 엠에이 인코포레이티드 올리고뉴클레오타이드의 정제를 위한 소수성 상호 작용 크로마토그래피
WO2017216340A1 (en) 2016-06-17 2017-12-21 F. Hoffmann-La Roche Ag In vitro nephrotoxicity screening assay
US11236339B2 (en) 2016-06-17 2022-02-01 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
US11479818B2 (en) 2016-06-17 2022-10-25 Hoffmann-La Roche Inc. In vitro nephrotoxicity screening assay
US20190218257A1 (en) 2016-06-24 2019-07-18 The Scripps Research Institute Novel nucleoside triphosphate transporter and uses thereof
BR112018077321A2 (pt) 2016-07-01 2019-04-24 F. Hoffmann-La Roche Ag oligonucleotídeos anti-sentido para modular a expressão de htra1
US11098077B2 (en) 2016-07-05 2021-08-24 Chinook Therapeutics, Inc. Locked nucleic acid cyclic dinucleotide compounds and uses thereof
US11253601B2 (en) 2016-07-11 2022-02-22 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
DK3484524T3 (da) 2016-07-15 2022-12-12 Ionis Pharmaceuticals Inc Forbindelser og fremgangsmåder til modulation af smn2
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
WO2018067546A1 (en) 2016-10-03 2018-04-12 President And Fellows Of Harvard College Delivery of therapeutic rnas via arrdc1-mediated microvesicles
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
EP3538671A1 (en) 2016-11-11 2019-09-18 Roche Innovation Center Copenhagen A/S Therapeutic oligonucleotides capture and detection
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
JP2019535839A (ja) 2016-11-29 2019-12-12 ピュアテック ヘルス エルエルシー 治療剤の送達のためのエクソソーム
US11033570B2 (en) 2016-12-02 2021-06-15 Cold Spring Harbor Laboratory Modulation of Lnc05 expression
TWI865997B (zh) 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
US20190345496A1 (en) 2017-01-13 2019-11-14 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating relb expression
US20190345495A1 (en) 2017-01-13 2019-11-14 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb2 expression
WO2018130581A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rela expression
US20190338286A1 (en) 2017-01-13 2019-11-07 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rel expression
WO2018130583A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb1 expression
WO2018165564A1 (en) 2017-03-09 2018-09-13 Ionis Pharmaceuticals, Inc. Morpholino modified oligomeric compounds
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
US11591362B2 (en) 2017-03-29 2023-02-28 Roche Innovation Center Copenhagen A/S Orthogonal protecting groups for the preparation of stereodefined phosphorothioate oligonucleotides
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
US20190055564A1 (en) 2017-06-01 2019-02-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
US11401519B2 (en) 2017-06-07 2022-08-02 University Of Massachusetts Anti-ADAM33 oligonucleotides and related methods
JP7492829B2 (ja) 2017-06-28 2024-05-30 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 多重カップリングおよび酸化の方法
WO2019014187A1 (en) 2017-07-10 2019-01-17 Genzyme Corporation METHODS AND COMPOSITIONS FOR TREATING A BLEEDING EVENT IN A SUBJECT WITH HEMOPHILIA
CA3069321A1 (en) 2017-07-11 2019-01-17 Synthorx, Inc. Incorporation of unnatural nucleotides and methods thereof
AU2018301477A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof
AR112969A1 (es) 2017-08-03 2020-01-15 Synthorx Inc Conjugados de citoquina para el tratamiento de enfermedades proliferativas e infecciosas
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
RS65031B1 (sr) 2017-08-25 2024-02-29 Stoke Therapeutics Inc Antisens oligomeri za lečenje stanja i bolesti
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
EP3694995A1 (en) 2017-10-13 2020-08-19 Roche Innovation Center Copenhagen A/S Methods for identifying improved stereodefined phosphorothioate oligonucleotide variants of antisense oligonucleotides utilising sub-libraries of partially stereodefined oligonucleotides
KR102585898B1 (ko) 2017-10-16 2023-10-10 에프. 호프만-라 로슈 아게 B형 간염 감염을 치료하기 위한 PAPD5 및 PAPD7 mRNA의 감소용 핵산 분자
AU2018360697A1 (en) 2017-11-01 2020-05-14 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
EP3710587A1 (en) 2017-11-16 2020-09-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
EP3714054A1 (en) 2017-11-20 2020-09-30 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
JP2021505175A (ja) 2017-12-11 2021-02-18 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 Ｆｎｄｃ３ｂの発現を調節するためのオリゴヌクレオチド
WO2019115417A2 (en) 2017-12-12 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating rb1 expression
EP3724206B1 (en) 2017-12-14 2023-06-28 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3728593A1 (en) 2017-12-18 2020-10-28 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
CA3085211A1 (en) 2017-12-21 2019-06-27 F. Hoffmann-La Roche Ag Companion diagnostic for htra1 rna antagonists
JP2021508327A (ja) 2017-12-22 2021-03-04 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 新規のチオホスホラミダイト
EP3728591B1 (en) 2017-12-22 2025-07-23 Roche Innovation Center Copenhagen A/S Gapmer oligonucleotides comprising a phosphorodithioate internucleoside linkage
AU2018386524A1 (en) 2017-12-22 2020-07-02 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorodithioate internucleoside linkage
AU2018395054B2 (en) 2017-12-29 2025-04-24 The Scripps Research Institute Unnatural base pair compositions and methods of use
CN111699258A (zh) 2018-01-10 2020-09-22 哥本哈根罗氏创新中心 用于调控pias4表达的寡核苷酸
US12178855B2 (en) 2018-01-10 2024-12-31 Translate Bio Ma, Inc. Compositions and methods for facilitating delivery of synthetic nucleic acids to cells
US20210095275A1 (en) 2018-01-12 2021-04-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating gsk3b expression
JP7547201B2 (ja) 2018-01-12 2024-09-09 ブリストル－マイヤーズ スクイブ カンパニー アルファ－シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用
PE20201501A1 (es) 2018-01-12 2020-12-29 Bristol Myers Squibb Co Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos
PE20210172A1 (es) 2018-01-12 2021-01-29 Roche Innovation Ct Copenhagen As Oligonucleotidos antisentido para alfa-sinucleina y usos de los mismos
CN111902537A (zh) 2018-01-15 2020-11-06 Ionis制药公司 Dnm2表达的调节剂
WO2019141656A1 (en) 2018-01-17 2019-07-25 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating erc1 expression
JP2021511029A (ja) 2018-01-18 2021-05-06 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 Ｓｒｅｂｐ１を標的とするアンチセンスオリゴヌクレオチド
WO2019145386A1 (en) 2018-01-26 2019-08-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating csnk1d expression
AU2019219194A1 (en) 2018-02-09 2020-08-06 Genentech, Inc. Oligonucleotides for modulating TMEM106B expression
CA3090901A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
JP7500426B2 (ja) 2018-02-21 2024-06-17 ブリストル－マイヤーズ スクイブ カンパニー Ｃａｍｋ２ｄアンチセンスオリゴヌクレオチドおよびその使用
MX2020008772A (es) 2018-02-26 2020-10-01 Synthorx Inc Conjugados de il-15, y sus usos.
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
CA3094303A1 (en) 2018-03-20 2019-09-26 Tokyo Institute Of Technology Antisense oligonucleotide reduced in toxicity
EP3768694A4 (en) 2018-03-22 2021-12-29 Ionis Pharmaceuticals, Inc. Methods for modulating fmr1 expression
EP3775208A1 (en) 2018-04-05 2021-02-17 F. Hoffmann-La Roche AG Use of fubp1 inhibitors for treating hepatitis b virus infection
WO2019199643A1 (en) 2018-04-09 2019-10-17 Bio-Techne Corporation Methods to further enhance signal amplification for the in situ detection of nucleic acids
EP3775204A4 (en) 2018-04-11 2021-12-29 Ionis Pharmaceuticals, Inc. Modulators of ezh2 expression
EP3788065B1 (en) 2018-04-30 2025-08-27 The Children's Hospital Of Philadelphia Improving anemias by combining agents
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
CN112105745A (zh) 2018-05-07 2020-12-18 罗氏创新中心哥本哈根有限公司 用于寡核苷酸治疗剂的大规模平行发现方法
US20210371860A1 (en) 2018-05-08 2021-12-02 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating myh7 expression
JOP20200283A1 (ar) 2018-05-09 2020-11-08 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير الوراثي عن atxn3
CA3098136A1 (en) 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing fxi expression
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
WO2019219723A1 (en) 2018-05-18 2019-11-21 F. Hoffmann-La Roche Ag Pharmaceutical compositions for treatment of microrna related diseases
WO2019224172A1 (en) 2018-05-25 2019-11-28 Roche Innovation Center Copenhagen A/S Novel process for making allofuranose from glucofuranose
WO2019233922A1 (en) 2018-06-05 2019-12-12 F. Hoffmann-La Roche Ag Oligonucleotides for modulating atxn2 expression
US11833168B2 (en) 2018-06-14 2023-12-05 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
WO2020007702A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting bcl2l11
WO2020007700A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020007772A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
AU2019297391B2 (en) 2018-07-03 2023-01-12 F. Hoffmann-La Roche Ag Oligonucleotides for modulating Tau expression
WO2020007889A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting stat1
WO2020007826A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mbtps1
WO2020011743A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mafb
WO2020011653A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
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
WO2020011744A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers5
MX2021000404A (es) 2018-07-13 2021-03-25 Hoffmann La Roche Oligonucleotidos para modular la expresion de rtel1.
MX2021000922A (es) 2018-07-25 2021-03-31 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de la atxn2.
AU2019313443A1 (en) 2018-07-31 2020-11-26 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorotrithioate internucleoside linkage
EP4122943B1 (en) 2018-07-31 2024-05-29 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorotrithioate internucleoside linkage
CN119265191A (zh) 2018-08-13 2025-01-07 阿尔尼拉姆医药品有限公司 乙型肝炎病毒(HBV)dsRNA剂组合物及其使用方法
EP3837381A1 (en) 2018-08-15 2021-06-23 Illumina, Inc. Compositions and methods for improving library enrichment
AR115960A1 (es) 2018-08-16 2021-03-17 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen lect2
WO2020041348A1 (en) 2018-08-20 2020-02-27 Rogcon, Inc. Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies
WO2020038973A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting sptlc1
WO2020038971A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting vcan
EP3841220A1 (en) 2018-08-23 2021-06-30 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
WO2020038976A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting usp8
US20220160870A1 (en) 2018-08-28 2022-05-26 Roche Innovation Center Copenhagen A/S Neoantigen engineering using splice modulating compounds
EP3620519A1 (en) 2018-09-04 2020-03-11 F. Hoffmann-La Roche AG Use of isolated milk extracellular vesicles for delivering oligonucleotides orally
EP3620520A1 (en) 2018-09-10 2020-03-11 Universidad del Pais Vasco Novel target to treat a metabolic disease in an individual
CA3105385A1 (en) 2018-09-18 2020-03-26 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
TWI869213B (zh) 2018-09-19 2025-01-01 美商Ionis製藥公司 Pnpla3表現之調節劑
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 (en) 2018-11-01 2021-09-08 F. Hoffmann-La Roche AG Antisense oligonucleotides targeting tia1
AU2019376079A1 (en) * 2018-11-08 2021-05-27 Aligos Therapeutics, Inc. S-antigen transport inhibiting oligonucleotide polymers and methods
EP3876973B1 (en) 2018-11-08 2025-10-22 Synthorx, Inc. Interleukin 10 conjugates and uses thereof
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
CA3119456A1 (en) 2018-11-15 2020-05-22 Ionis Pharmaceuticals, Inc. Modulators of irf5 expression
US12281305B2 (en) 2018-11-21 2025-04-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing prion expression
WO2020104492A1 (en) 2018-11-22 2020-05-28 Roche Innovation Center Copenhagen A/S Pyridinium salts as activators in the synthesis of stereodefined oligonucleotides
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
WO2020117706A1 (en) 2018-12-03 2020-06-11 Triplet Therapeutics, Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity
SI3897672T1 (sl) 2018-12-20 2024-02-29 Humabs Biomed Sa Kombinirana terapija HBV
CA3123617A1 (en) 2018-12-20 2020-06-25 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
AU2019403447B2 (en) 2018-12-21 2023-07-27 Ionis Pharmaceuticals, Inc. Modulators of HSD17B13 expression
EP3898975A2 (en) 2018-12-21 2021-10-27 Boehringer Ingelheim International GmbH Antisense oligonucleotides targeting card9
AU2020209186A1 (en) 2019-01-16 2021-09-09 Genzyme Corporation Serpinc1 iRNA compositions and methods of use thereof
WO2020152303A1 (en) 2019-01-25 2020-07-30 F. Hoffmann-La Roche Ag Lipid vesicle for oral drug delivery
MX2021008918A (es) 2019-01-31 2021-08-24 Ionis Pharmaceuticals Inc Moduladores de expresion yap1.
US20200246467A1 (en) 2019-02-06 2020-08-06 Synthorx, Inc. Il-2 conjugates and methods of use thereof
CN114555825A (zh) 2019-02-15 2022-05-27 领先细胞医疗诊断有限公司 用于通过原位杂交进行核酸的多路复用检测的方法
JP2022521510A (ja) 2019-02-20 2022-04-08 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 ホスホノアセテートギャップマー型オリゴヌクレオチド
TW202045521A (zh) 2019-02-20 2020-12-16 丹麥商羅氏創新中心哥本哈根有限公司 新穎亞磷醯胺化物
WO2020173845A1 (en) 2019-02-26 2020-09-03 Roche Innovation Center Copenhagen A/S Oligonucleotide formulation method
SG11202108450SA (en) 2019-02-27 2021-09-29 Ionis Pharmaceuticals Inc Modulators of malat1 expression
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
WO2020178258A1 (en) 2019-03-05 2020-09-10 F. Hoffmann-La Roche Ag Intracellular targeting of molecules
KR20210141554A (ko) 2019-03-21 2021-11-23 코디악 바이오사이언시즈, 인크. 세포외 소포 접합체 및 이의 용도
EP3941483A4 (en) 2019-03-21 2023-10-18 Arnay Sciences, Llc ANTISEN OLIGONUCLEOTIDES FOR ALLEL SPECIFICITY
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
EP3950006A4 (en) 2019-03-29 2024-06-12 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for dux4 expression adjustment
SG11202110745VA (en) 2019-04-03 2021-10-28 Bristol Myers Squibb Co Angptl2 antisense oligonucleotides and uses thereof
EP3947677A1 (en) 2019-04-04 2022-02-09 F. Hoffmann-La Roche AG Oligonucleotides for modulating atxn2 expression
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
US12421268B2 (en) 2019-04-16 2025-09-23 Roche Innovation Center Copenhagen A/S Process for preparing nucleotide P(V) monomers
EP3962918A1 (en) 2019-04-30 2022-03-09 Roche Innovation Center Copenhagen A/S Novel process for preparing rhenium chelated mag3 oligonucleotides
WO2020226960A1 (en) 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
US11629347B2 (en) 2019-05-06 2023-04-18 University Of Massachusetts Anti-C9ORF72 oligonucleotides and related methods
US20220339256A1 (en) 2019-05-13 2022-10-27 Vir Biotechnology, Inc. Compositions and methods for treating hepatitis b virus (hbv) infection
HRP20250468T1 (hr) 2019-05-28 2025-07-18 Ionis Pharmaceuticals. Inc. SPOJEVI I METODE ZA SMANJENJE POKAZATELJA FUS-a
EP3980539A1 (en) 2019-06-06 2022-04-13 F. Hoffmann-La Roche AG Antisense oligonucleotides targeting atxn3
CA3143330A1 (en) 2019-06-14 2020-12-17 The Scripps Research Institute Reagents and methods for replication, transcription, and translation in semi-synthetic organisms
HRP20251033T1 (hr) 2019-07-26 2025-10-24 Ionis Pharmaceuticals, Inc. Spojevi i postupci za moduliranje gfap
WO2021022109A1 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. SERPIN FAMILY F MEMBER 2 (SERPINF2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
JP2022544935A (ja) 2019-08-14 2022-10-24 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 細胞外小胞－ｎｌｒｐ３アンタゴニスト
WO2021030776A1 (en) 2019-08-14 2021-02-18 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting stat6
JP2022544288A (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 ＣＥＢＰ／βを標的とする細胞外小胞－ＡＳＯ構築物
BR112022002599A2 (pt) 2019-08-14 2022-07-19 Codiak Biosciences Inc Vesícula extracelular ligada a moléculas e usos da mesma
CN114641570A (zh) 2019-08-14 2022-06-17 科迪亚克生物科学公司 具有靶向kras的反义寡核苷酸的细胞外囊泡
US20230193274A1 (en) 2019-08-14 2023-06-22 Codiak Biosciences, Inc. Extracellular vesicles with stat3-antisense oligonucleotides
WO2021030778A1 (en) 2019-08-15 2021-02-18 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds and uses thereof
WO2021030706A1 (en) 2019-08-15 2021-02-18 Synthorx, Inc. Immuno oncology combination therapies with il-2 conjugates
MX2022002053A (es) 2019-08-23 2022-03-17 Synthorx Inc Conjugados de il-15 y sus usos.
US20220290152A1 (en) 2019-09-03 2022-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
CN114746122A (zh) 2019-09-10 2022-07-12 新索思股份有限公司 Il-2缀合物和治疗自身免疫性疾病的使用方法
US20230241089A1 (en) 2019-09-25 2023-08-03 Codiak Biosciences, Inc. Sting agonist comprising exosomes for treating neuroimmunological disorders
US20220389429A1 (en) 2019-10-04 2022-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
CN114728017B (zh) 2019-10-14 2025-10-28 阿斯利康(瑞典)有限公司 Pnpla3表达的调节剂
US12486512B2 (en) 2019-10-18 2025-12-02 Alnylam Pharmaceuticals, Inc. Solute carrier family member iRNA compositions and methods of use thereof
AR120267A1 (es) 2019-10-22 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA EL COMPONENTE C3 DEL COMPLEMENTO Y SUS MÉTODOS DE USO
KR20220094213A (ko) 2019-11-01 2022-07-05 알닐람 파마슈티칼스 인코포레이티드 헌팅틴 (HTT) iRNA 제제 조성물 및 이의 사용 방법
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
IL291786A (en) 2019-11-04 2022-06-01 Synthorx Inc Interleukin 10 conjugates and their use
WO2021096763A1 (en) 2019-11-13 2021-05-20 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
US20230041016A1 (en) 2019-12-04 2023-02-09 Athma A. PAI Anti-slc6a1 oligonucleotides and related methods
KR20220115995A (ko) 2019-12-13 2022-08-19 알닐람 파마슈티칼스 인코포레이티드 인간 염색체 ９ 개방 판독 프레임 72 (C9orf72) iRNA 제제 조성물 및 이의 사용 방법
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
WO2021122993A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of saraf inhibitors for treating hepatitis b virus infection
WO2021122869A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of scamp3 inhibitors for treating hepatitis b virus infection
WO2021122921A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of cops3 inhibitors for treating hepatitis b virus infection
WO2021122735A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of sept9 inhibitors for treating hepatitis b virus infection
WO2021122910A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of sbds inhibitors for treating hepatitis b virus infection
CN114829603A (zh) 2019-12-20 2022-07-29 豪夫迈·罗氏有限公司 用于抑制scn9a表达的增强寡核苷酸
WO2021130266A1 (en) 2019-12-24 2021-07-01 F. Hoffmann-La Roche Ag Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
MX2022007909A (es) 2019-12-24 2022-07-21 Hoffmann La Roche Combinacion farmaceutica de agentes antivirales que actuan sobre hbv y/o un inmunomodulador para el tratamiento de 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
US20230136787A1 (en) 2020-02-10 2023-05-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing vegf-a expression
WO2021163564A1 (en) 2020-02-14 2021-08-19 University Of Massachusetts Oligonucleotides targeting frataxin and related methods
IL295496A (en) 2020-02-18 2022-10-01 Alnylam Pharmaceuticals Inc Apolipoprotein c3 (apoc3) irna preparations and methods of using them
AR121446A1 (es) 2020-02-28 2022-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2
JP2023516142A (ja) 2020-02-28 2023-04-18 エフ・ホフマン－ラ・ロシュ・アクチェンゲゼルシャフト Ｃｄ７３エクソン７スプライシングを調節するためのオリゴヌクレオチド
AR121490A1 (es) 2020-03-02 2022-06-08 Mitsubishi Tanabe Pharma Corp Prevención o tratamiento de aneurismas con substancia inhibidora de mir-33b
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
WO2021178778A1 (en) 2020-03-06 2021-09-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin (ttr)
IL296109A (en) 2020-03-06 2022-11-01 Alnylam Pharmaceuticals Inc Ketohexokinase (khk) irna compositions and methods of use thereof
WO2021184021A1 (en) 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting pmp22
WO2021184020A1 (en) 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Methods of treating neuroinflammation
EP4121534A1 (en) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
CN116209759A (zh) 2020-03-26 2023-06-02 阿尔尼拉姆医药品有限公司 冠状病毒iRNA组合物及其使用方法
WO2021202443A2 (en) 2020-03-30 2021-10-07 Alnylam Pharmaceucticals, Inc. Compositions and methods for silencing dnajc15 gene expression
EP4133078A1 (en) 2020-04-06 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
US20230159933A1 (en) 2020-04-07 2023-05-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
CN115916262A (zh) 2020-04-21 2023-04-04 旗舰创业股份有限公司 双官能分子及其使用方法
IL297702A (en) 2020-04-27 2022-12-01 Alnylam Pharmaceuticals Inc Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
EP4142738A1 (en) 2020-04-30 2023-03-08 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
WO2021222768A2 (en) 2020-05-01 2021-11-04 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atxn1
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
WO2021231204A1 (en) 2020-05-11 2021-11-18 Genentech, Inc. Complement component 4 inhibitors for treating neurological diseases, and related compositons, systems and methods of using same
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
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
US20230227824A1 (en) 2020-05-12 2023-07-20 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for regulating expression of ataxin 3
TW202208628A (zh) 2020-05-13 2022-03-01 瑞士商赫孚孟拉羅股份公司 靶向顆粒蛋白前體之寡核苷酸促效劑
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
EP4150087A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
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)
EP4150088A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
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
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
CN115702243A (zh) 2020-06-09 2023-02-14 罗氏创新中心哥本哈根有限公司 用于治疗性多核苷酸的鸟苷类似物
EP4162033A4 (en) 2020-06-09 2024-12-25 Alnylam Pharmaceuticals, Inc. Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression
EP4168546A1 (en) 2020-06-18 2023-04-26 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof
TW202208422A (zh) 2020-06-24 2022-03-01 美商維爾生物科技股份有限公司 經工程化之b型肝炎病毒中和抗體及其用途
TW202216203A (zh) 2020-06-25 2022-05-01 美商欣爍克斯公司 Il-2接合物及抗egfr抗體之免疫腫瘤學組合治療
AR122731A1 (es) 2020-06-26 2022-10-05 Hoffmann La Roche Oligonucleótidos mejorados para modular la expresión de fubp1
BR112022024206A2 (pt) 2020-06-29 2023-01-03 Ionis Pharmaceuticals Inc Compostos e métodos para modular plp1
CN116209478A (zh) 2020-07-17 2023-06-02 田边三菱制药株式会社 肌病的预防或治疗剂
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
UY39344A (es) 2020-07-28 2022-02-25 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de app
TW202221123A (zh) 2020-08-07 2022-06-01 美商Ionis製藥公司 調節ｓｃｎ２ａ之化合物及方法
EP4200419A2 (en) 2020-08-21 2023-06-28 F. Hoffmann-La Roche AG Use of a1cf inhibitors for treating hepatitis b virus infection
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
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
TW202229552A (zh) 2020-10-05 2022-08-01 美商艾拉倫製藥股份有限公司 Ｇ蛋白－偶合受體７５（ＧＰＲ７５）ｉＲＮＡ組成物及其使用方法
WO2022076596A1 (en) 2020-10-06 2022-04-14 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting stat6
KR20230084204A (ko) 2020-10-09 2023-06-12 신톡스, 인크. Il-2 콘쥬게이트를 사용한 면역 종양학 치료법
AU2021356693A1 (en) 2020-10-09 2023-06-15 Synthorx, Inc. Immuno oncology combination therapy with il-2 conjugates and pembrolizumab
AU2021365822A1 (en) 2020-10-21 2023-06-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
EP4232570A4 (en) 2020-10-23 2024-10-16 The Scripps Research Institute REVERSE TRANSCRIPTION OF POLYNUCLEOTIDES WITH UNNATURAL NUCLEOTIDES
IL302709A (en) 2020-11-13 2023-07-01 Alnylam Pharmaceuticals Inc COAGULATION FACTOR V (F5) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CA3202569A1 (en) 2020-11-18 2022-05-27 Lemba Bv Umlilo antisense transcription inhibitors
MX2023005736A (es) 2020-11-18 2023-05-25 Ionis Pharmaceuticals Inc Compuestos y metodos para modular la expresion de angiotensinogeno.
US20240002853A1 (en) 2020-11-23 2024-01-04 Alpha Anomeric Sas Nucleic acid duplexes
US11987795B2 (en) 2020-11-24 2024-05-21 The Broad Institute, Inc. Methods of modulating SLC7A11 pre-mRNA transcripts for diseases and conditions associated with expression of SLC7A11
CA3201452A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
US20230060373A1 (en) 2020-12-03 2023-03-02 Hoffmann-La Roche Inc. Antisense Oligonucleotides Targeting ATXN3
TW202237842A (zh) 2020-12-03 2022-10-01 瑞士商赫孚孟拉羅股份公司 靶向ａｔｘｎ３之反義寡核苷酸
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
CN116568696A (zh) 2020-12-08 2023-08-08 豪夫迈·罗氏有限公司 二硫代磷酸酯寡核苷酸的新颖合成
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
CR20230308A (es) 2020-12-11 2023-09-08 Civi Biopharma Inc Entrega oral de conjugados antisentido que tienen por blanco a pcsk9
IL303800A (en) 2020-12-18 2023-08-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating factor xii
JP2023553710A (ja) 2020-12-18 2023-12-25 エフ． ホフマン－ラ ロシュ アーゲー プログラニュリンを標的とするためのアンチセンスオリゴヌクレオチド
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
EP4271816A4 (en) 2020-12-31 2025-04-02 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND RELATED USES FOR THE TREATMENT OF MYOTONIC DYSTROPHY
WO2022150260A1 (en) 2021-01-05 2022-07-14 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
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
AU2022220704A1 (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
EP4291243A1 (en) 2021-02-12 2023-12-20 Synthorx, Inc. Lung cancer combination therapy with il-2 conjugates and an anti-pd-1 antibody or antigen-binding fragment thereof
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
KR20230146603A (ko) 2021-02-17 2023-10-19 론자 세일즈 아게 최적화 링커와 고정 모이어티를 통해 생물학적 활성 분자에 연결된 세포외 소포체
KR20230147125A (ko) 2021-02-17 2023-10-20 론자 세일즈 아게 세포외 소포-nlrp3 길항제
CN117222739A (zh) 2021-02-25 2023-12-12 阿尔尼拉姆医药品有限公司 朊病毒蛋白(prnp)irna组合物和其使用方法
KR20230150844A (ko) 2021-02-26 2023-10-31 알닐람 파마슈티칼스 인코포레이티드 케토헥소키나아제(KHK) iRNA 조성물 및 이의 사용 방법
MX2023010249A (es) 2021-03-04 2023-09-15 Alnylam Pharmaceuticals Inc Composiciones de arni de proteina similar a la angiopoyetina 3 (angptl3) y metodos para usarlas.
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
CN117203336A (zh) 2021-03-29 2023-12-08 阿尔尼拉姆医药品有限公司 亨廷顿蛋白(HTT)iRNA药剂组合物以及其使用方法
MX2023011612A (es) 2021-03-31 2023-12-15 Entrada Therapeutics Inc Peptidos de penetracion celular ciclicos.
CN117120041A (zh) 2021-04-01 2023-11-24 隆萨销售股份有限公司 胞外囊泡组合物
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
WO2022214632A1 (en) 2021-04-07 2022-10-13 Neoplants Sas Compositions and methods for indoor air remediation
IL307926A (en) 2021-04-26 2023-12-01 Alnylam Pharmaceuticals Inc Transmembrane assemblies, serine 6 ((TMPRSS6 IRNA) and methods of using them
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
JPWO2022230987A1 (enExample) 2021-04-30 2022-11-03
EP4337263A1 (en) 2021-05-10 2024-03-20 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
US20240358845A1 (en) 2021-05-10 2024-10-31 Entrada Therapeutics, Inc. Compositions and methods for intracellular therapeutics
US20250051780A1 (en) 2021-05-10 2025-02-13 Entrada Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING
JP2024522068A (ja) 2021-05-18 2024-06-11 アルナイラム ファーマシューティカルズ， インコーポレイテッド ナトリウム－グルコース共輸送体２（ｓｇｌｔ２）ｉｒｎａ組成物およびその使用方法
WO2022246023A1 (en) 2021-05-20 2022-11-24 Korro Bio, Inc. Methods and compositions for adar-mediated editing
JP2024520555A (ja) 2021-05-29 2024-05-24 １グローブ ヘルス インスティテュート エルエルシー 新規遺伝子サイレンシング技術としての非対称短鎖二重鎖ｄｎａおよびその使用
AU2022286925A1 (en) 2021-05-29 2023-12-21 1Globe Health Institute Llc Short duplex dna as a novel gene silencing technology and use thereof
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
TW202317762A (zh) 2021-06-02 2023-05-01 美商艾拉倫製藥股份有限公司 含有類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）的ｉＲＮＡ組成物及其使用方法
WO2022256534A1 (en) 2021-06-03 2022-12-08 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
KR20240017911A (ko) 2021-06-04 2024-02-08 알닐람 파마슈티칼스 인코포레이티드 인간 염색체 9 개방 해독 프레임 72(C9orf72) iRNA 제제 조성물 및 이의 사용 방법
WO2022260939A2 (en) 2021-06-08 2022-12-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
KR20240019228A (ko) 2021-06-08 2024-02-14 에프. 호프만-라 로슈 아게 올리고뉴클레오티드 프로그래눌린 효현제
EP4105328A1 (en) 2021-06-15 2022-12-21 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Antisense-oligonucleotides for prevention of kidney dysfunction promoted by endothelial dysfunction by ephrin-b2 suppression
AU2022293556A1 (en) 2021-06-18 2024-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
EP4359006A1 (en) 2021-06-23 2024-05-01 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
US20250034564A1 (en) 2021-06-29 2025-01-30 Korro Bio, Inc. Methods and Compositions for ADAR-Mediated Editing
CA3225469A1 (en) 2021-06-30 2023-01-05 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
TW202333748A (zh) 2021-07-19 2023-09-01 美商艾拉倫製藥股份有限公司 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物
IL309905A (en) 2021-07-23 2024-03-01 Alnylam Pharmaceuticals Inc IRNA compositions in β-catenin (CTNNB1) and methods of using them
EP4377458A1 (en) 2021-07-29 2024-06-05 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
JP2024530018A (ja) 2021-08-03 2024-08-14 アルナイラム ファーマシューティカルズ， インコーポレイテッド トランスサイレチン（ＴＴＲ）ｉＲＮＡ組成物およびその使用方法
EP4381071A1 (en) 2021-08-04 2024-06-12 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing angiotensinogen (agt)
KR20240045300A (ko) 2021-08-13 2024-04-05 알닐람 파마슈티칼스 인코포레이티드 인자 XII (F12) iRNA 조성물 및 이의 사용 방법
CA3229305A1 (en) 2021-08-16 2023-02-23 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
CA3230404A1 (en) 2021-08-31 2023-03-09 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
US11833221B2 (en) 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
IL311139A (en) 2021-09-01 2024-04-01 Entrada Therapeutics Inc Compounds and methods for skipping exon 44 in Duchenne muscular dystrophy
EP4401742A2 (en) 2021-09-17 2024-07-24 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
JP2024535888A (ja) 2021-09-20 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド インヒビンサブユニットベータｅ（ｉｎｈｂｅ）モジュレーター組成物およびその使用方法
CN118019848A (zh) 2021-09-29 2024-05-10 豪夫迈·罗氏有限公司 Rna编辑
KR20240067112A (ko) 2021-09-30 2024-05-16 아카우오스, 인크. Kcnq4-연관 청력손실을 치료하기 위한 조성물 및 방법
EP4409003A1 (en) 2021-10-01 2024-08-07 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
EP4419683A1 (en) 2021-10-22 2024-08-28 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
KR20240095325A (ko) 2021-10-29 2024-06-25 알닐람 파마슈티칼스 인코포레이티드 보체 인자 b (cfb) irna 조성물 및 이의 사용 방법
TW202334418A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 杭丁頓（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法
CN118318042A (zh) 2021-11-03 2024-07-09 豪夫迈·罗氏有限公司 用于调节载脂蛋白e4表达的寡核苷酸
EP4430187A2 (en) 2021-11-10 2024-09-18 University of Rochester Antisense oligonucleotides for modifying protein expression
CA3237769A1 (en) 2021-11-10 2023-05-19 University Of Rochester Gata4-targeted therapeutics for treatment of cardiac hypertrophy
JP2024544532A (ja) 2021-11-11 2024-12-03 エフ． ホフマン－ラ ロシュ アーゲー Ｈｂｖの処置のための医薬組合せ
WO2023102188A1 (en) 2021-12-03 2023-06-08 Quralis Corporation Gapmer antisense oligonucleotides with modified backbone chemistries
CN118434858A (zh) 2021-12-07 2024-08-02 豪夫迈·罗氏有限公司 靶向actl6b的反义寡核苷酸
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
CN118489009A (zh) 2021-12-17 2024-08-13 豪夫迈·罗氏有限公司 寡核苷酸gba激动剂
WO2023111210A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Combination of oligonucleotides for modulating rtel1 and fubp1
WO2023117738A1 (en) 2021-12-20 2023-06-29 F. Hoffmann-La Roche Ag Threose nucleic acid antisense oligonucleotides and methods thereof
US20250302950A1 (en) 2021-12-20 2025-10-02 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
MX2024007790A (es) 2021-12-22 2024-09-06 Camp4 Therapeutics Corp Modulación de la transcripción génica utilizando oligonucleótidos antisentido dirigidos a arn reguladores.
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
WO2023127857A1 (ja) * 2021-12-27 2023-07-06 株式会社理研ジェネシス 新規人工核酸、その製造方法及び用途
EP4465994A1 (en) 2022-01-20 2024-11-27 Genentech, Inc. Antisense oligonucleotides for modulating tmem106b expression
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
AR128558A1 (es) 2022-02-21 2024-05-22 Hoffmann La Roche Oligonucleótido antisentido
CN119213005A (zh) 2022-03-16 2024-12-27 艾姆皮瑞克公司 用于提高siRNA生物利用度的GALNAC组合物
WO2023192830A2 (en) 2022-03-28 2023-10-05 Empirico Inc. Modified oligonucleotides
CN119173632A (zh) 2022-05-10 2024-12-20 豪夫迈·罗氏有限公司 靶向cfp-elk1基因间区域的反义寡核苷酸
WO2023222858A1 (en) 2022-05-18 2023-11-23 F. Hoffmann-La Roche Ag Improved oligonucleotides targeting rna binding protein sites
CA3258307A1 (en) 2022-06-10 2023-12-14 Camp4 Therapeutics Corp METHODS FOR MODULATING PROGRANULIN EXPRESSION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATOR RNAS
JP2025520514A (ja) 2022-06-17 2025-07-03 エフ． ホフマン－ラ ロシュ アーゲー プログラニュリンを標的とするためのアンチセンスオリゴヌクレオチド
KR20250040656A (ko) 2022-07-06 2025-03-24 몰레큘라 악시옴, 엘엘씨 췌장암을 치료하기 위한 조성물 및 방법
EP4561636A1 (en) 2022-07-29 2025-06-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024040041A1 (en) 2022-08-15 2024-02-22 Dicerna Pharmaceuticals, Inc. Regulation of activity of rnai molecules
EP4573198A2 (en) 2022-08-18 2025-06-25 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
EP4332221A1 (en) 2022-08-29 2024-03-06 Roche Innovation Center Copenhagen A/S Threose nucleic acid antisense oligonucleotides and methods thereof
EP4581143A1 (en) 2022-08-29 2025-07-09 University of Rochester Antisense oligonucleotide-based anti-fibrotic therapeutics
IL318655A (en) 2022-09-06 2025-03-01 Hoffmann La Roche Double RNA molecule for ocular administration
JP2025532593A (ja) 2022-09-15 2025-10-01 リジェネロン・ファーマシューティカルズ・インコーポレイテッド １７ｂ－ヒドロキシステロイドデヒドロゲナーゼ１３型（ｈｓｄ１７ｂ１３）ｉｒｎａ組成物およびその使用方法
KR20250075637A (ko) 2022-09-23 2025-05-28 아이오니스 파마수티컬즈, 인코포레이티드 Mecp2 발현을 감소시키기 위한 화합물 및 방법(compounds and methods for reducing mecp2 expression)
WO2024098061A2 (en) 2022-11-04 2024-05-10 Genkardia Inc. Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure
AU2023371614A1 (en) 2022-11-04 2025-05-15 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
CN120500498A (zh) 2022-11-14 2025-08-15 瑞泽恩制药公司 用于成纤维细胞生长因子受体3介导的至星形胶质细胞的递送的组合物及方法
AU2023403208A1 (en) 2022-12-01 2025-06-19 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2024124041A1 (en) 2022-12-07 2024-06-13 Advanced Cell Diagnostics, Inc. Multiplexed detection of nucleic acid targets
WO2024126654A1 (en) 2022-12-14 2024-06-20 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting actl6b
WO2024136899A1 (en) 2022-12-21 2024-06-27 Synthorx, Inc. Cancer therapy with il-2 conjugates and chimeric antigen receptor therapies
EP4658281A1 (en) 2023-01-30 2025-12-10 Vib Vzw Suppressors of tauopathies
AR131799A1 (es) 2023-02-09 2025-04-30 Alnylam Pharmaceuticals Inc Moléculas de reversir y métodos para su uso
WO2024175588A1 (en) 2023-02-21 2024-08-29 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
WO2024175586A2 (en) 2023-02-21 2024-08-29 Vib Vzw Inhibitors of synaptogyrin-3 expression
CN120769910A (zh) 2023-02-23 2025-10-10 罗切斯特大学 用于制备封闭端dna线分子的药剂和方法
TW202444349A (zh) 2023-03-20 2024-11-16 美商欣爍克斯公司 使用ｉｌ－２綴合物之癌症療法
US12383615B2 (en) 2023-03-23 2025-08-12 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods
WO2024196965A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2024208249A1 (zh) 2023-04-06 2024-10-10 上海舶望制药有限公司 5'-膦酸酯修饰的核苷类似物及其制备的寡核苷酸
WO2024220930A2 (en) 2023-04-20 2024-10-24 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024227047A2 (en) 2023-04-28 2024-10-31 Beam Therapeutics Inc. Modified guide rna
WO2024227765A2 (en) 2023-05-04 2024-11-07 F. Hoffmann-La Roche Ag Oligonucleotides capable of upregulating glucocerebrosidase expression
WO2024233864A2 (en) 2023-05-10 2024-11-14 Dicerna Pharmaceuticals, Inc. Galnac-conjugated rnai oligonucleotides
AU2024273075A1 (en) 2023-05-12 2025-11-20 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
CN116606338A (zh) * 2023-05-18 2023-08-18 中国人民解放军军事科学院军事医学研究院 6’-氰基修饰的锁核苷、核苷酸和核酸聚合物
WO2024249328A2 (en) 2023-05-26 2024-12-05 Adarx Pharmaceuticals, Inc. Sod1-modulating compositions and methods of use thereof
WO2024256707A1 (en) 2023-06-16 2024-12-19 F. Hoffmann-La Roche Ag Double stranded oligonucleotide for modulating jak1 expression
TW202506137A (zh) 2023-06-20 2025-02-16 美商雅迪克斯製藥公司 Ｌｒｒｋ２調節組合物及其使用方法
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025024334A1 (en) 2023-07-21 2025-01-30 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods
TW202516001A (zh) 2023-07-25 2025-04-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 Ｃａｓ內切酶及相關方法
WO2025024493A1 (en) 2023-07-25 2025-01-30 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025054459A1 (en) 2023-09-08 2025-03-13 Dicerna Pharmaceuticals, Inc. Rnai oligonucleotide conjugates
TW202526017A (zh) 2023-09-14 2025-07-01 美商Ionis製藥公司 用於減少ａｐｏｃｉｉｉ表現的化合物及方法
TW202530406A (zh) 2023-09-21 2025-08-01 美商Ionis製藥公司 用於抑制ｌｐａ的化合物及方法
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025076031A2 (en) 2023-10-03 2025-04-10 Alnylam Pharmaceuticals, Inc. Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof
WO2025080939A1 (en) 2023-10-13 2025-04-17 Ultragenyx Pharmaceutical, Inc. Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025128853A2 (en) 2023-12-13 2025-06-19 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with ube3a overexpression
TW202535425A (zh) 2023-12-21 2025-09-16 瑞士商赫孚孟拉羅股份公司 反義寡核苷酸
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
WO2025178854A2 (en) 2024-02-19 2025-08-28 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting cideb and related methods
WO2025199231A2 (en) 2024-03-20 2025-09-25 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
WO2025207517A2 (en) 2024-03-25 2025-10-02 Synthorx, Inc. Synthetic trna synthetases and cells comprising synthetic molecules for production of polypeptides
GB202404290D0 (en) 2024-03-26 2024-05-08 Senisca Ltd Novel oligoncleotides
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods

Family Cites Families (138)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
DE3329892A1 (de) 1983-08-18 1985-03-07 Köster, Hubert, Prof. Dr., 2000 Hamburg Verfahren zur herstellung von oligonucleotiden
USRE34069E (en) 1983-08-18 1992-09-15 Biosyntech Gmbh Process for the preparation of oligonucleotides
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
DE3788914T2 (de) 1986-09-08 1994-08-25 Ajinomoto Kk Verbindungen zur Spaltung von RNS an eine spezifische Position, Oligomere, verwendet bei der Herstellung dieser Verbindungen und Ausgangsprodukte für die Synthese dieser Oligomere.
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
JP2828642B2 (ja) 1987-06-24 1998-11-25 ハワード フローレイ インスティテュト オブ イクスペリメンタル フィジオロジー アンド メディシン ヌクレオシド誘導体
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
ATE151467T1 (de) 1987-11-30 1997-04-15 Univ Iowa Res Found Durch modifikationen an der 3'-terminalen phosphodiesterbindung stabilisierte dna moleküle, ihre verwendung als nukleinsäuresonden sowie als therapeutische mittel zur hemmung der expression spezifischer zielgene
US5403711A (en) 1987-11-30 1995-04-04 University Of Iowa Research Foundation Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved
WO1989009221A1 (en) 1988-03-25 1989-10-05 University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5194599A (en) 1988-09-23 1993-03-16 Gilead Sciences, Inc. Hydrogen phosphonodithioate compositions
JP2863922B2 (ja) 1988-11-28 1999-03-03 マツダ株式会社 後輪操舵車両の後輪懸架装置
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5721218A (en) 1989-10-23 1998-02-24 Gilead Sciences, Inc. Oligonucleotides with inverted polarity
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
EP0497875B1 (en) 1989-10-24 2000-03-22 Isis Pharmaceuticals, Inc. 2' modified oligonucleotides
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5623065A (en) 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5220007A (en) 1990-02-15 1993-06-15 The Worcester Foundation For Experimental Biology Method of site-specific alteration of RNA and production of encoded polypeptides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
ATE167523T1 (de) 1990-05-11 1998-07-15 Microprobe Corp Teststreifen zum eintauchen für nukleinsäure- hybridisierungsassays und verfahren zur kovalenten immobilisierung von oligonucleotiden
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5223618A (en) 1990-08-13 1993-06-29 Isis Pharmaceuticals, Inc. 4'-desmethyl nucleoside analog compounds
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
BR9106702A (pt) 1990-07-27 1993-06-08 Isis Pharmaceuticals Inc Analogo de oligonucleotideos e processos para modular a producao de uma proteina por um organismo e para tratar um organismo
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
IL99066A (en) 1990-08-03 1996-01-31 Sterling Winthrop Inc Nuclease resistant compounds comprising an oligonucleotide sequence having a diol at either or both termini and compositions containing them
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
WO1992005186A1 (en) 1990-09-20 1992-04-02 Gilead Sciences Modified internucleoside linkages
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5672697A (en) 1991-02-08 1997-09-30 Gilead Sciences, Inc. Nucleoside 5'-methylene phosphonates
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
DE59208572D1 (de) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
EP1256589A3 (en) 1991-11-26 2003-09-17 Isis Pharmaceuticals, Inc. Oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5792608A (en) 1991-12-12 1998-08-11 Gilead Sciences, Inc. Nuclease stable and binding competent oligomers and methods for their use
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5700922A (en) 1991-12-24 1997-12-23 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
DE69332304T2 (de) * 1992-03-12 2003-01-23 Temple University Of The Commonwealth System Of Higher Education, Philadelphia Antivirale 2',5'-oligoadenylatderivate mit doppelter wirkung und ihre verwendungen
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
NL9300058A (nl) 1992-06-18 1994-01-17 Stichting Rega V Z W 1,5-anhydrohexitol nucleoside analoga en farmaceutisch gebruik daarvan.
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
DE69316369D1 (en) 1992-07-27 1998-02-19 Hybridon Inc Oligonukleotid alkylphosphonothiate
WO1994017093A1 (en) 1993-01-25 1994-08-04 Hybridon, Inc. Oligonucleotide alkylphosphonates and alkylphosphonothioates
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
GB9304620D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Compounds
ATE155467T1 (de) 1993-03-30 1997-08-15 Sanofi Sa Acyclische nucleosid analoge und sie enthaltende oligonucleotidsequenzen
EP0691977B1 (en) 1993-03-31 1997-11-26 Sanofi Oligonucleotides with amide linkages replacing phosphodiester linkages
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
NZ278490A (en) 1993-12-09 1998-03-25 Univ Jefferson Chimeric polynucleotide with both ribo- and deoxyribonucleotides in one strand and deoxyribonucleotides in a second strand
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5646269A (en) 1994-04-28 1997-07-08 Gilead Sciences, Inc. Method for oligonucleotide analog synthesis
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5792747A (en) 1995-01-24 1998-08-11 The Administrators Of The Tulane Educational Fund Highly potent agonists of growth hormone releasing hormone
US5652356A (en) 1995-08-17 1997-07-29 Hybridon, Inc. Inverted chimeric and hybrid oligonucleotides
AU4966997A (en) 1996-11-18 1998-06-10 Takeshi Imanishi Novel nucleotide analogues
US6770748B2 (en) * 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
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
ATE293123T1 (de) 1997-09-12 2005-04-15 Exiqon As Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
HK1040084B (en) 1999-02-12 2005-04-22 Daiichi Sankyo Company, Limited Novel nucleosides and oligonucleotide analogues
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US6436640B1 (en) 1999-03-18 2002-08-20 Exiqon A/S Use of LNA in mass spectrometry
CA2372085C (en) 1999-05-04 2009-10-27 Exiqon A/S L-ribo-lna analogues
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
WO2002018388A1 (fr) 2000-08-29 2002-03-07 Takeshi Imanishi Analogues de nucleosides et derives d'oligonucleotides renfermant ces analogues
US6426220B1 (en) 2000-10-30 2002-07-30 Isis Pharmaceuticals, Inc. Antisense modulation of calreticulin expression
JP2005514005A (ja) 2001-09-04 2005-05-19 エクシコン エ／エス 新規のｌｎａ組成物およびその使用
JP3712374B2 (ja) * 2001-12-06 2005-11-02 富士写真フイルム株式会社 小型磁気ディスクカートリッジ
US7655456B2 (en) 2002-01-18 2010-02-02 Arkray, Inc. Analytical device having temperature detection unit
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
US7456393B2 (en) 2003-04-10 2008-11-25 Ge Homeland Protection, Inc. Device for testing surfaces of articles for traces of explosives and/or drugs
CA2569419A1 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
EP1984381B1 (en) * 2006-01-27 2010-09-29 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
US8935416B2 (en) 2006-04-21 2015-01-13 Fortinet, Inc. Method, apparatus, signals and medium for enforcing compliance with a policy on a client computer
US9400902B2 (en) 2012-05-22 2016-07-26 Trimble Navigation Limited Multi-modal entity tracking and display

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013147498A (ja) * 2006-01-27 2013-08-01 Isis Pharmaceuticals Inc ６−修飾された二環式核酸類似体

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CN102908630A (zh) 2013-02-06
ES2516815T3 (es) 2014-10-31
US20090012281A1 (en) 2009-01-08
WO2007090071A2 (en) 2007-08-09
CA2640171A1 (en) 2007-08-09
US20070249049A1 (en) 2007-10-25
AU2007211080A1 (en) 2007-08-09
DK1984381T3 (da) 2010-11-01
DE602007009487D1 (de) 2010-11-11
AU2007211080B9 (en) 2012-05-03
JP2013147498A (ja) 2013-08-01
WO2007090071A3 (en) 2008-01-24
EP1984381A2 (en) 2008-10-29

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