WO2009006478A3 - 6-disubstituted bicyclic nucleic acid analogs - Google Patents

6-disubstituted bicyclic nucleic acid analogs Download PDF

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Publication number
WO2009006478A3
WO2009006478A3 PCT/US2008/068922 US2008068922W WO2009006478A3 WO 2009006478 A3 WO2009006478 A3 WO 2009006478A3 US 2008068922 W US2008068922 W US 2008068922W WO 2009006478 A3 WO2009006478 A3 WO 2009006478A3
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WO
WIPO (PCT)
Prior art keywords
disubstituted bicyclic
oligomeric compounds
nucleic acid
acid analogs
bicyclic nucleosides
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2008/068922
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French (fr)
Other versions
WO2009006478A2 (en
Inventor
Eric E Swayze
Punit P Seth
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
Original Assignee
Isis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=40087523&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=WO2009006478(A3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority to DK08781239.2T priority Critical patent/DK2176280T4/en
Priority to CA2692579A priority patent/CA2692579C/en
Priority to ES08781239.2T priority patent/ES2376507T5/en
Priority to EP08781239.2A priority patent/EP2176280B2/en
Priority to AU2008272918A priority patent/AU2008272918B2/en
Application filed by Isis Pharmaceuticals Inc filed Critical Isis Pharmaceuticals Inc
Priority to US12/667,684 priority patent/US8278283B2/en
Priority to CN200880105715.6A priority patent/CN101796062B/en
Priority to AT08781239T priority patent/ATE538127T1/en
Publication of WO2009006478A2 publication Critical patent/WO2009006478A2/en
Publication of WO2009006478A3 publication Critical patent/WO2009006478A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)

Abstract

The present disclosure describes 6-disubstituted bicyclic nucleosides, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, the 6-disubstituted bicyclic nucleosides each comprise a 2'-O-C(Ri)(R2)-4' or 2'-O- C=(R3)(R.4)-4' bridge wherein each R is, independently a substituent group and Ri and R2 include H. The 6-disubstituted bicyclic nucleosides are useful for enhancing properties of oligomeric compounds including nuclease resistance. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.
PCT/US2008/068922 2007-07-05 2008-07-01 6-disubstituted bicyclic nucleic acid analogs Ceased WO2009006478A2 (en)

Priority Applications (8)

Application Number Priority Date Filing Date Title
AT08781239T ATE538127T1 (en) 2007-07-05 2008-07-01 6-DISUBSTITUTED BICYCLIC NUCLEIC ACID ANALOGUES
CA2692579A CA2692579C (en) 2007-07-05 2008-07-01 6-disubstituted bicyclic nucleic acid analogs
ES08781239.2T ES2376507T5 (en) 2007-07-05 2008-07-01 6-disubstituted bicyclic nucleic acid analogs
EP08781239.2A EP2176280B2 (en) 2007-07-05 2008-07-01 6-disubstituted bicyclic nucleic acid analogs
AU2008272918A AU2008272918B2 (en) 2007-07-05 2008-07-01 6-disubstituted bicyclic nucleic acid analogs
DK08781239.2T DK2176280T4 (en) 2007-07-05 2008-07-01 6-Disubstituerede bicykliske nukleinsyreanaloge
US12/667,684 US8278283B2 (en) 2007-07-05 2008-07-01 6-disubstituted or unsaturated bicyclic nucleic acid analogs
CN200880105715.6A CN101796062B (en) 2007-07-05 2008-07-01 6-disubstituted bicyclic nucleic acid analogs

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US94813407P 2007-07-05 2007-07-05
US60/948,134 2007-07-05

Publications (2)

Publication Number Publication Date
WO2009006478A2 WO2009006478A2 (en) 2009-01-08
WO2009006478A3 true WO2009006478A3 (en) 2009-02-19

Family

ID=40087523

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2008/068922 Ceased WO2009006478A2 (en) 2007-07-05 2008-07-01 6-disubstituted bicyclic nucleic acid analogs

Country Status (9)

Country Link
US (1) US8278283B2 (en)
EP (1) EP2176280B2 (en)
CN (1) CN101796062B (en)
AT (1) ATE538127T1 (en)
AU (1) AU2008272918B2 (en)
CA (1) CA2692579C (en)
DK (1) DK2176280T4 (en)
ES (1) ES2376507T5 (en)
WO (1) WO2009006478A2 (en)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11077195B2 (en) 2019-02-06 2021-08-03 Synthorx, Inc. IL-2 conjugates and methods of use thereof
US11279929B2 (en) 2018-07-03 2022-03-22 Hoffmann-La Roche, Inc. Oligonucleotides for modulating Tau expression

Families Citing this family (626)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2284269B1 (en) 2002-11-18 2017-08-09 Roche Innovation Center Copenhagen A/S Antisense design
EP2188298B1 (en) 2007-08-15 2013-09-18 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
EP2265627A2 (en) * 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
US8426378B2 (en) 2008-03-21 2013-04-23 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucelosides and methods for their use
US9290534B2 (en) 2008-04-04 2016-03-22 Ionis Pharmaceuticals, Inc. Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside
WO2010036696A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
CA2740785C (en) 2008-10-15 2017-06-20 Isis Pharmaceuticals, Inc. Modulation of factor 11 expression
EP2447274B1 (en) 2008-10-24 2017-10-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds and methods
EP2358397B1 (en) 2008-10-24 2020-01-01 Ionis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
BRPI0923225A2 (en) 2008-12-02 2016-10-04 Chiralgen Ltd Phosphorus-modified nucleic acid synthesis method
EP2393825A2 (en) 2009-02-06 2011-12-14 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US8536320B2 (en) 2009-02-06 2013-09-17 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
WO2010142805A1 (en) 2009-06-12 2010-12-16 Santaris Pharma A/S New potent anti apob antisense compounds
KR20220084437A (en) 2009-06-17 2022-06-21 바이오젠 엠에이 인코포레이티드 Composition and methods for modulating of smn2 splicing in a subject
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRECURSORS AND THEIR METHODS OF USE
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
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
US8779118B2 (en) 2010-01-11 2014-07-15 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
ES2733708T3 (en) 2010-02-08 2019-12-02 Ionis Pharmaceuticals Inc Selective reduction of allelic variants
JP6018506B2 (en) 2010-02-08 2016-11-02 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Selective reduction of allelic variants
WO2011115817A1 (en) 2010-03-16 2011-09-22 Isis Pharmaceuticals, Inc. Methods of preparing 2'-o-substituted purine nucleosides
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011139702A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
WO2011139699A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
WO2011139695A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified 5' diphosphate nucleosides and oligomeric compounds prepared therefrom
ES2625689T3 (en) 2010-04-29 2017-07-20 Ionis Pharmaceuticals, Inc. Modulation of transthyretin expression
WO2011139911A2 (en) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Lipid formulated single stranded rna
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
US8846637B2 (en) 2010-06-08 2014-09-30 Isis Pharmaceuticals, Inc. Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2012007477A1 (en) 2010-07-12 2012-01-19 Santaris Pharma A/S Anti hcv oligomers
ES2756326T3 (en) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulation of myotonic dystrophy-protein kinase (DMPK) expression
WO2012034942A1 (en) 2010-09-13 2012-03-22 Santaris Pharma A/S Compounds for the modulation of aurora kinase b expression
DK2620428T3 (en) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetric help group
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
WO2012064758A2 (en) 2010-11-08 2012-05-18 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
EP3521451A1 (en) 2010-11-17 2019-08-07 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
WO2012066092A1 (en) 2010-11-19 2012-05-24 Santaris Pharma A/S Compounds for the modulation of aurora kinase a expression
WO2012066093A1 (en) 2010-11-19 2012-05-24 Santaris Pharma A/S Compounds for the modulation of pdz-binding kinase (pbk) expression
US10017764B2 (en) 2011-02-08 2018-07-10 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
KR20190092590A (en) 2011-04-01 2019-08-07 아이오니스 파마수티컬즈, 인코포레이티드 Modulation of signal transducer and activator of transcription 3 (stat3) expression
CN105886506A (en) 2011-04-13 2016-08-24 Isis制药公司 Antisense modulation of PTP1B expression
WO2012143427A1 (en) 2011-04-19 2012-10-26 Santaris Pharma A/S Anti polyomavirus compounds
SI3505528T1 (en) 2011-04-21 2021-04-30 Glaxo Group Limited Modulation of hepatitis b virus (hbv) expression
MX393138B (en) 2011-04-25 2025-03-24 Sanofi Sa MICRORNA COMPOUNDS AND METHODS FOR MODULATING MIR-21 ACTIVITY.
SG194671A1 (en) 2011-04-27 2013-12-30 Isis Pharmaceuticals Inc Modulation of apolipoprotein ciii (apociii) expression
WO2012151324A1 (en) 2011-05-02 2012-11-08 Isis 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
US9187749B2 (en) 2011-06-10 2015-11-17 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
WO2012170945A2 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
AU2012271357A1 (en) 2011-06-16 2013-05-02 Ionis Pharmaceuticals, Inc. Antisense modulation of fibroblast growth factor receptor 4 expression
WO2012175733A1 (en) 2011-06-23 2012-12-27 Santaris Pharma A/S Hcv combination therapy
AU2012275096B2 (en) 2011-06-29 2016-02-04 Ionis Pharmaceuticals, Inc. Methods for modulating kallikrein (KLKB1) expression
KR20140058536A (en) 2011-06-30 2014-05-14 스텔라 에이피에스 Hcv combination therapy
EP2726610A1 (en) 2011-06-30 2014-05-07 Stella ApS Hcv combination therapy
CN107365339A (en) 2011-07-19 2017-11-21 波涛生命科学有限公司 Methods of Synthesizing Functionalized Nucleic Acids
US20140303235A1 (en) 2011-08-11 2014-10-09 Isis Pharmaceuticals, Inc. Linkage modified gapped oligomeric compounds and uses thereof
EP2751269B1 (en) 2011-08-29 2016-03-23 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
EP2758533B1 (en) 2011-09-20 2018-04-11 Ionis Pharmaceuticals, Inc. Antisense modulation of gcgr expression
AU2012328680A1 (en) 2011-10-25 2014-05-01 Ionis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
EP2776564B1 (en) 2011-11-07 2019-10-02 Ionis Pharmaceuticals, Inc. Modulation of tmprss6 expression
WO2013068348A1 (en) 2011-11-07 2013-05-16 Santaris Pharma A/S Lna oligomers for improvement in hepatic function
EP2776590B1 (en) 2011-11-07 2016-10-26 Roche Innovation Center Copenhagen A/S Prognostic method for checking efficacy of micro rna-122 inhibitors in hcv+ patients
US20140343127A1 (en) 2011-11-11 2014-11-20 Santaris Pharma A/S a corporation Compounds for the modulation of smn2 splicing
US9243291B1 (en) 2011-12-01 2016-01-26 Isis Pharmaceuticals, Inc. Methods of predicting toxicity
ES2673960T3 (en) 2011-12-22 2018-06-26 Ionis Pharmaceuticals, Inc. Methods to modulate the expression of a transcript-1 of adenocarcinoma in lung associated with metastases (MALAT-1)
IL266882B2 (en) 2012-01-11 2025-11-01 Ionis Pharmaceuticals Inc Compositions and methods for modulating splicing of ikbkap
EP3330278A1 (en) 2012-02-08 2018-06-06 Ionis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
US9340784B2 (en) 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
CA2866392C (en) 2012-03-30 2023-10-24 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
EP3336189A1 (en) 2012-04-20 2018-06-20 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
MY173600A (en) 2012-04-25 2020-02-08 Sanofi Sa Microrna compounds and methods for modulating mir-21 activity
US9574193B2 (en) 2012-05-17 2017-02-21 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein (a) expression
US20160002624A1 (en) 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
WO2013177248A2 (en) 2012-05-22 2013-11-28 Isis Pharmaceuticals, Inc. Modulation of enhancer rna mediated gene expression
RU2624028C2 (en) 2012-05-24 2017-06-30 Ионис Фармасьютикалз, Инк. Methods and compositions for modeling expression of apolipoprotein (a)
WO2013181666A2 (en) 2012-06-01 2013-12-05 Isis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
US9828602B2 (en) 2012-06-01 2017-11-28 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
ES2809199T3 (en) 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulation of UBE3A-ATS expression
DK2872147T3 (en) 2012-07-13 2023-02-20 Wave Life Sciences Ltd METHOD FOR PREPARATION OF CHIRAL OLIGONUCLEOTIDES
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
EP3693460A1 (en) 2012-07-27 2020-08-12 Ionis Pharmaceuticals, Inc. Modulation of renin-angiotensin system (ras) related diseases by angiotensinogen
WO2014028739A1 (en) 2012-08-15 2014-02-20 Isis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified capping protocols
US9695418B2 (en) 2012-10-11 2017-07-04 Ionis 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
WO2014059364A1 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Methods of treating kennedy's disease
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
EP4086347A3 (en) 2012-10-12 2023-01-11 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US10443052B2 (en) 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
EP2906697A4 (en) 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHODS OF MONITORING C9ORF72 EXPRESSION
FI2906696T4 (en) 2012-10-15 2023-03-18 Methods for modulating c9orf72 expression
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2014066239A1 (en) 2012-10-22 2014-05-01 Idenix Pharmaceuticals, Inc. 2',4'-bridged nucleosides for hcv infection
EP3483273A1 (en) 2012-10-31 2019-05-15 Ionis Pharmaceuticals, Inc. Cancer treatment
CA2889044A1 (en) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
BR112015012051A2 (en) 2012-11-26 2017-12-12 Roche Innovation Ct Copenhagen As fgfr3 expression modulation compositions and methods
US9695475B2 (en) 2012-12-11 2017-07-04 Ionis Pharmaceuticals, Inc. Competitive modulation of microRNAs
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
CA2893801A1 (en) 2013-01-30 2014-08-07 F. Hoffmann-La Roche Ag Lna oligonucleotide carbohydrate conjugates
DK2951191T3 (en) * 2013-01-31 2019-01-14 Ionis Pharmaceuticals Inc PROCEDURE FOR MANUFACTURING OLIGOMERIC COMPOUNDS USING MODIFIED CLUTCH PROTOCOLS
EP2951304B1 (en) 2013-02-04 2020-07-08 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
KR20200123263A (en) 2013-02-14 2020-10-28 아이오니스 파마수티컬즈, 인코포레이티드 Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations
BR112015022156A2 (en) 2013-03-14 2017-11-14 Isis Pharmaceuticals Inc compositions and methods for modulating tau expression
WO2014172698A1 (en) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
PL2992098T3 (en) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating hbv and ttr expression
EP2992096B1 (en) 2013-05-01 2021-03-03 Regulus Therapeutics Inc. Compounds and methods for enhanced cellular uptake
JP2016518842A (en) 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド MicroRNA compounds and methods of modulating miR-122
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
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
WO2014205451A2 (en) 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
MX367083B (en) 2013-06-27 2019-08-05 Roche Innovation Ct Copenhagen As Antisense oligomers and conjugates targeting pcsk9.
JP6487913B2 (en) 2013-07-02 2019-03-20 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Growth hormone receptor modulators
WO2015009731A2 (en) 2013-07-15 2015-01-22 The Regents Of The University Of California Azacyclic constrained analogs of fty720
TWI702046B (en) 2013-07-19 2020-08-21 美商Ionis製藥公司 Compositions for modulating tau expression
EP3027617B1 (en) 2013-07-31 2026-02-18 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
AU2014306271A1 (en) 2013-08-08 2016-03-24 The Scripps Research Institute A method for the site-specific enzymatic labelling of nucleic acids in vitro by incorporation of unnatural nucleotides
TW201536329A (en) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc Compound and method for regulating the manifestation of dystrophic myotonic protein kinase (DMPK)
US9670492B2 (en) 2013-08-28 2017-06-06 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (PKK) expression
PL3043827T3 (en) 2013-09-13 2020-03-31 Ionis Pharmaceuticals, Inc. Modulators of complement factor b
US9943604B2 (en) 2013-09-20 2018-04-17 Ionis Pharmaceuticals, Inc. Targeted therapeutic nucleosides and their use
MY192689A (en) 2013-10-11 2022-09-01 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
WO2015061536A1 (en) 2013-10-25 2015-04-30 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-21 activity
DK3077510T3 (en) 2013-12-02 2020-06-08 Ionis Pharmaceuticals Inc ANTISENSE COMPOUNDS AND APPLICATIONS THEREOF
WO2015089368A2 (en) 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
PL3087183T3 (en) 2013-12-24 2021-01-11 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
WO2015108046A1 (en) 2014-01-15 2015-07-23 株式会社新日本科学 Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
EP3105331B1 (en) 2014-02-11 2021-06-23 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
EP3109254B1 (en) * 2014-02-18 2018-04-11 Osaka University Crosslinked nucleoside and nucleotide
EP3119789B1 (en) 2014-03-17 2020-04-22 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
SI3119888T1 (en) 2014-03-19 2021-11-30 Ionis Pharmaceuticals, Inc. Compositions for modulating ataxin 2 expression
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
HUE050704T2 (en) 2014-04-01 2020-12-28 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
WO2015164693A1 (en) 2014-04-24 2015-10-29 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
US9926556B2 (en) 2014-04-28 2018-03-27 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
WO2015168618A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
EP3137115B1 (en) 2014-05-01 2020-10-14 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
HUE052709T2 (en) 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Conjugates of modified antisense oligonucleotides and their use to modify PKK expression
WO2015168589A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
CR20160554A (en) 2014-05-01 2017-03-07 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS TO MODULATE THE EXPRESSION OF COMPLEMENT FACTOR B
TW201607559A (en) 2014-05-12 2016-03-01 阿尼拉製藥公司 Methods and compositions for treating a SERPINC1-associated disorder
GB201408623D0 (en) 2014-05-15 2014-07-02 Santaris Pharma As Oligomers and oligomer conjugates
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
SG11201609376SA (en) 2014-05-22 2016-12-29 Alnylam Pharmaceuticals Inc Angiotensinogen (agt) irna compositions and methods of use thereof
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
MA40463A (en) 2014-08-07 2017-06-14 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
WO2016040589A1 (en) 2014-09-12 2016-03-17 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
CN107109411B (en) 2014-10-03 2022-07-01 冷泉港实验室 Targeted increase in nuclear gene export
AU2015329974B2 (en) 2014-10-10 2019-06-20 F. Hoffmann-La Roche Ag GaINAc phosphoramidites, nucleic acid conjugates thereof and their use
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3904519A1 (en) 2014-10-30 2021-11-03 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
TWI769565B (en) 2014-11-10 2022-07-01 美商阿尼拉製藥公司 HEPATITIS B VIRUS (HBV) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
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
AU2015346042A1 (en) 2014-11-14 2017-04-27 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of proteins
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
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
AU2015360794B2 (en) 2014-12-08 2021-07-08 The Board Of Regents Of The University Of Texas System Lipocationic polymers and uses thereof
DK3234131T3 (en) 2014-12-16 2020-06-29 Roche Innovation Ct Copenhagen As Method of screening for chiral toxicity
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
ES2893114T3 (en) 2015-02-04 2022-02-08 Bristol Myers Squibb Co Methods for selecting therapeutic molecules
CA2975525A1 (en) 2015-02-04 2016-08-11 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
JP2018510621A (en) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Patatin-like phospholipase domain-containing 3 (PNPLA3) iRNA compositions and methods of use thereof
MX391217B (en) 2015-02-26 2025-03-21 Ionis Pharmaceuticals Inc ALLELE-SPECIFICIAL MODULATORS OF P23H RHODOPSIN.
WO2016141145A1 (en) 2015-03-03 2016-09-09 Ionis Pharmaceuticals, Inc. Methods for modulating mecp2 expression
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
EP3265098B1 (en) 2015-03-03 2025-03-26 Ionis Pharmaceuticals, Inc. Compositions for modulating mecp2 expression
CN107429250B (en) 2015-04-03 2022-03-01 Ionis制药公司 Compounds and methods for modulating expression of TMPRSS6
WO2016164746A1 (en) 2015-04-08 2016-10-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016168592A2 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
EP3307316A1 (en) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
JP2018519811A (en) 2015-06-29 2018-07-26 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． Modified CRISPR RNA and modified single CRISPR RNA and uses thereof
CA2991894A1 (en) 2015-07-10 2017-01-19 Ionis Pharmaceuticals, Inc. Modulators of diacyglycerol acyltransferase 2 (dgat2)
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
US20180201937A1 (en) 2015-08-04 2018-07-19 The University Of Chicago Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
KR20180043819A (en) 2015-08-24 2018-04-30 로슈 이노베이션 센터 코펜하겐 에이/에스 LNA-G method
US10889813B2 (en) 2015-09-02 2021-01-12 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof
KR102764250B1 (en) 2015-09-14 2025-02-07 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 Lipocationic dendrimers and uses thereof
EP3352753A4 (en) 2015-09-24 2019-03-13 The Regents of The University of California SYNTHETIC SPHINGOLIPID MOLECULES, MEDICAMENTS, METHODS FOR THEIR SYNTHESIS, AND METHODS OF TREATMENT
HK1251624A1 (en) 2015-09-24 2019-02-01 Ionis Pharmaceuticals, Inc. Modulators of kras expression
WO2017053781A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
MX392014B (en) 2015-10-08 2025-03-21 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING ANGIOTENSINOGEN EXPRESSION.
CN108603230A (en) 2015-10-09 2018-09-28 南安普敦大学 The screening of the adjusting of gene expression and protein expression imbalance
WO2017067970A1 (en) 2015-10-22 2017-04-27 Roche Innovation Center Copenhagen A/S In vitro toxicity screening assay
WO2017068087A1 (en) 2015-10-22 2017-04-27 Roche Innovation Center Copenhagen A/S Oligonucleotide detection method
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
AU2016349625B2 (en) 2015-11-06 2022-07-07 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
LT4119569T (en) 2015-11-06 2024-09-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy
HK1258434A1 (en) 2015-11-12 2019-11-08 F. Hoffmann-La Roche Ag Standardized neuronal cell assays from primate species
EP3389670A4 (en) 2015-12-04 2020-01-08 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
IL308717A (en) 2015-12-07 2024-01-01 Genzyme Corp Methods and preparations for the treatment of diseases related to SERPINC1
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CN116059236A (en) 2015-12-14 2023-05-05 冷泉港实验室 Antisense oligomers for the treatment of autosomal dominant mental retardation type 5 and Dravet syndrome
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
WO2017117496A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
EP3400300A4 (en) 2016-01-05 2019-08-07 Ionis Pharmaceuticals, Inc. METHODS FOR REDUCING LRRK2 EXPRESSION
AU2017229778A1 (en) 2016-03-09 2018-08-16 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting PMP22 expression
CA3013683C (en) 2016-03-14 2021-07-13 F. Hoffmann-La Roche Ag Oligonucleotides for reduction of pd-l1 expression
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
CA3013799A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Methods of modulating keap1
WO2017180835A1 (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 (en) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc HIGH DENSITY LIPOPROTEIN BINDING PROTEIN (HDLBP / VIGILINE) RNA COMPOSITION AND METHODS FOR USING THEM
CN109475568B (en) 2016-05-06 2026-07-17 Ionis制药公司 GLP-1 receptor ligand moiety conjugated oligonucleotides and uses thereof
CN114656378A (en) 2016-05-16 2022-06-24 得克萨斯州大学系统董事会 Cationic sulfonamide amino lipids and amphiphilic zwitterionic amino lipids
EP3469083A1 (en) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
AU2017286811A1 (en) 2016-06-17 2018-11-22 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
EP3472347B1 (en) 2016-06-17 2023-01-04 F. Hoffmann-La Roche AG In vitro nephrotoxicity screening assay
AU2017285350A1 (en) 2016-06-17 2018-12-20 F. Hoffmann-La Roche Ag Nucleic acid molecules for reduction of PAPD5 or PAPD7 mRNA for treating hepatitis B infection
US11105794B2 (en) 2016-06-17 2021-08-31 Hoffmann-La Roche Inc. In vitro nephrotoxicity screening assay
EP3475295B1 (en) 2016-06-24 2022-08-10 The Scripps Research Institute Novel nucleoside triphosphate transporter and uses thereof
RU2019101298A (en) 2016-07-01 2020-08-03 Ф. Хоффманн-Ля Рош Аг ANTI-SENSE OLIGONUCLEOTIDES FOR MODULATION OF HTRA1 EXPRESSION
EP3507367A4 (en) 2016-07-05 2020-03-25 Aduro BioTech, Inc. Locked nucleic acid cyclic dinucleotide compounds and uses thereof
US20190321387A1 (en) 2016-07-15 2019-10-24 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
PL3484524T3 (en) 2016-07-15 2023-03-20 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
MD3519572T2 (en) 2016-09-29 2024-03-31 Biogen Ma Inc Compounds and methods for reducing Tau expression
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
JOP20190104A1 (en) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn3 expression
WO2018087200A1 (en) 2016-11-11 2018-05-17 Roche Innovation Center Copenhagen A/S Therapeutic oligonucleotides capture and detection
TW202313978A (en) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 Serpina1 irna compositions and methods of use thereof
US11033570B2 (en) 2016-12-02 2021-06-15 Cold Spring Harbor Laboratory Modulation of Lnc05 expression
TWI865997B (en) 2016-12-16 2024-12-11 美商阿尼拉製藥公司 METHODS FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES USING TRANSTHYRETIN (TTR) iRNA COMPOSITIONS
WO2018130584A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb2 expression
US20190345496A1 (en) 2017-01-13 2019-11-14 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating relb expression
EP3568479A1 (en) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb1 expression
EP3568478A1 (en) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rel expression
WO2018130581A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rela expression
US11180756B2 (en) 2017-03-09 2021-11-23 Ionis Pharmaceuticals Morpholino modified oligomeric compounds
JOP20190215A1 (en) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc Modulators of pcsk9 expression
WO2018195165A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
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
JP2020530442A (en) 2017-07-10 2020-10-22 ジェンザイム・コーポレーション Methods and Compositions for Treating Bleeding Events in Subjects with Hemophilia
TWI821192B (en) 2017-07-11 2023-11-11 美商新索思股份有限公司 Incorporation of unnatural nucleotides and methods thereof
WO2019014262A1 (en) 2017-07-11 2019-01-17 The Scripps Research Institute Incorporation of unnatural nucleotides and methods of use in vivo thereof
CA3069868A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (ldha) irna compositions and methods of use thereof
IL321812A (en) 2017-08-03 2025-08-01 Synthorx Inc Cytokine conjugates for the treatment of autoimmune diseases
WO2019030313A2 (en) 2017-08-11 2019-02-14 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating 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 modulating tom1 expression
KR20260009385A (en) 2017-08-25 2026-01-19 스톡 테라퓨틱스, 인크. Antisense oligomers for treatment of conditions and diseases
US10517889B2 (en) 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
CN111226114A (en) 2017-10-13 2020-06-02 罗氏创新中心哥本哈根有限公司 Method for identifying improved variants of antisense oligonucleotides using a subset of sterically defined oligonucleotides
KR20200030594A (en) 2017-10-16 2020-03-20 에프. 호프만-라 로슈 아게 Nucleic acid molecule for reducing PAPD5 and PAPD7 mRNA to treat hepatitis B infection
CA3078971A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
TWI809004B (en) 2017-11-09 2023-07-21 美商Ionis製藥公司 Compounds and methods for reducing snca expression
EP3710587A1 (en) 2017-11-16 2020-09-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
WO2019115416A2 (en) 2017-12-11 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating fndc3b expression
WO2019115417A2 (en) 2017-12-12 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating rb1 expression
WO2019118916A1 (en) 2017-12-14 2019-06-20 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2019126097A1 (en) 2017-12-18 2019-06-27 Alnylam Pharmaceuticals, Inc. High mobility group box-1 (hmgb1) irna compositions and methods of use thereof
CA3085211A1 (en) 2017-12-21 2019-06-27 F. Hoffmann-La Roche Ag Companion diagnostic for htra1 rna antagonists
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
CN111448317B (en) 2017-12-22 2025-07-04 罗氏创新中心哥本哈根有限公司 Gapmer oligonucleotides containing phosphorodithioate internucleoside bonds
US20200339982A1 (en) 2017-12-22 2020-10-29 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorodithioate internucleoside linkage
CN111448316B (en) 2017-12-22 2025-02-14 罗氏创新中心哥本哈根有限公司 New thiophosphoramidites
JP7429642B2 (en) 2017-12-29 2024-02-08 ザ スクリプス リサーチ インスティテュート Non-natural base pair compositions and methods of use
CN111699258A (en) 2018-01-10 2020-09-22 哥本哈根罗氏创新中心 Oligonucleotides for modulating expression of PIAS4
EP3737759A1 (en) 2018-01-12 2020-11-18 Roche Innovation Center Copenhagen A/S Alpha-synuclein antisense oligonucleotides and uses thereof
US20210095275A1 (en) 2018-01-12 2021-04-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating gsk3b expression
WO2019140236A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
MY203356A (en) 2018-01-12 2024-06-25 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CA3088522A1 (en) 2018-01-15 2019-07-18 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
WO2019141656A1 (en) 2018-01-17 2019-07-25 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating erc1 expression
EP3740573A1 (en) 2018-01-18 2020-11-25 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting srebp1
WO2019145386A1 (en) 2018-01-26 2019-08-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating csnk1d expression
BR112020016170A2 (en) 2018-02-09 2020-12-15 Genentech, Inc. THERAPEUTIC AND ANTISENSE OLIGONUCLEOTIDS, CONJUGATE, PHARMACEUTICALLY ACCEPTABLE SALT, PHARMACEUTICAL COMPOSITION, IN VITRO OR IN VIVO METHOD TO MODULATE THE EXPRESSION OF TMEM106B, METHOD FOR TREATING OR PREVENTING ILLNESS, USE
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
WO2019165067A1 (en) 2018-02-21 2019-08-29 Bristol-Myers Squibb Company Camk2d antisense oligonucleotides and uses thereof
AU2019224159B2 (en) 2018-02-26 2025-05-29 Sanofi IL-15 conjugates and uses thereof
TWI840345B (en) 2018-03-02 2024-05-01 美商Ionis製藥公司 Modulators of irf4 expression
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
EP3770256A4 (en) 2018-03-20 2021-12-22 Tokyo Institute of Technology OLIGONUCLEOTIDE ANTISENS WITH REDUCED TOXICITY
WO2019183440A1 (en) 2018-03-22 2019-09-26 Ionis Pharmaceuticals, Inc. Methods for modulating fmr1 expression
MA52661A (en) 2018-04-05 2021-02-17 Centre Leon Berard USE OF FUBP1 INHIBITORS IN THE TREATMENT OF HEPATITIS B VIRUS INFECTION
WO2019200172A1 (en) 2018-04-11 2019-10-17 Ionis Pharmaceuticals, Inc. Modulators of ezh2 expression
EP3774824A1 (en) 2018-04-13 2021-02-17 Bristol-Myers Squibb Company Novel phosphorous (v)-based reagents, processes for the preparation thereof, and their use in making stereo-defined organophoshorous (v) compounds
WO2019213016A1 (en) 2018-04-30 2019-11-07 The Children's Hospital Of Philadelphia Methods of improving anemias by combining agents
JP2021523227A (en) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． Methods and Compositions for the Treatment of Cholesteryl Ester Accumulation
JP2021522815A (en) 2018-05-07 2021-09-02 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 Quality control method for LNA oligonucleotide therapy using massively parallel sequencing
US20210371860A1 (en) 2018-05-08 2021-12-02 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating myh7 expression
JP2021522800A (en) 2018-05-09 2021-09-02 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Compounds and methods for reducing ATXN3 expression
IL277970B2 (en) 2018-05-09 2025-03-01 Ionis Pharmaceuticals Inc Oligomeric compounds comprising modified oligonucleotides and conjugate groups for use in treating end stage renal disease, chronic kidney disease, and coronary artery disease
TWI851574B (en) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 ANGIOTENSINOGEN (AGT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
JP2021524450A (en) 2018-05-18 2021-09-13 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ Pharmaceutical compositions for the 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
EP3802824A1 (en) 2018-06-05 2021-04-14 F. Hoffmann-La Roche AG Oligonucleotides for modulating atxn2 expression
CA3103429A1 (en) 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
TWI833770B (en) 2018-06-27 2024-03-01 美商Ionis製藥公司 Compounds and methods for reducing lrrk2 expression
WO2020007700A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020007702A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting bcl2l11
WO2020007772A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
WO2020007826A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mbtps1
WO2020007889A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting stat1
WO2020011653A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
WO2020011743A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mafb
WO2020011744A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers5
WO2020011745A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers6
WO2020011869A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting tlr2
BR112021000538A2 (en) 2018-07-13 2021-04-06 F. Hoffmann-La Roche Ag OLIGONUCLEOTIDS TO MODULATE RTEL1 EXPRESSION
WO2020018558A1 (en) 2018-07-17 2020-01-23 Aronora, Inc. Methods for safely reducing thrombopoietin
AU2019310097A1 (en) 2018-07-25 2021-02-04 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ATXN2 expression
CA3098267A1 (en) 2018-07-31 2020-02-06 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorotrithioate internucleoside linkage
BR112021001756A2 (en) 2018-07-31 2021-05-11 Roche Innovation Center Copenhagen A/S oligonucleotides, gapmer oligonucleotide, pharmaceutically acceptable salt, conjugate, pharmaceutical composition, uses of an oligonucleotide, method for the treatment or prophylaxis of a disease, process for making an oligonucleotide and invention
WO2020036862A1 (en) 2018-08-13 2020-02-20 Alnylam Pharmaceuticals, Inc. HEPATITIS B VIRUS (HBV) dsRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
CA3110211A1 (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
JP2022500003A (en) 2018-09-18 2022-01-04 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. Ketohexokinase (KHK) iRNA composition and its usage
TWI869213B (en) 2018-09-19 2025-01-01 美商Ionis製藥公司 Modulators of pnpla3 expression
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
JP2022512877A (en) 2018-11-01 2022-02-07 エフ．ホフマン－ラ ロシュ アーゲー Antisense oligonucleotide targeting TIA1
TW202031284A (en) 2018-11-08 2020-09-01 美商新索思股份有限公司 Interleukin 10 conjugates and uses thereof
TW202028222A (en) 2018-11-14 2020-08-01 美商Ionis製藥公司 Modulators of foxp3 expression
MX2021005758A (en) 2018-11-15 2021-09-21 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
WO2020109343A1 (en) 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Combination therapy for treatment of macular degeneration
WO2020109344A1 (en) 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Occular administration device for antisense oligonucleotides
EP3894559A4 (en) 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. THERAPEUTIC METHODS FOR TRINUCLEOTIDE REPEAT EXPANSION DISEASES ASSOCIATED WITH MLH3 ACTIVITY
JP2022515744A (en) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ， インコーポレイテッド Compositions and Methods for the Treatment of KCNT1-Related Disorders
JP2022514648A (en) 2018-12-21 2022-02-14 ベーリンガー インゲルハイム インターナショナル ゲゼルシャフト ミット ベシュレンクテル ハフツング Antisense oligonucleotides targeting CARD9
SG11202106378VA (en) 2018-12-21 2021-07-29 Ionis Pharmaceuticals Inc Modulators of hsd17b13 expression
CA3126933A1 (en) 2019-01-16 2020-07-23 Genzyme Corporation Serpinc1 irna compositions and methods of use thereof
CN113365607A (en) 2019-01-25 2021-09-07 豪夫迈·罗氏有限公司 Lipid vesicles for oral drug delivery
TW202214854A (en) 2019-01-31 2022-04-16 美商Ionis製藥公司 Modulators of yap1 expression
WO2020169696A1 (en) 2019-02-20 2020-08-27 Roche Innovation Center Copenhagen A/S Novel phosphoramidites
MX2021009950A (en) 2019-02-20 2021-09-21 Roche Innovation Ct Copenhagen As Phosphonoacetate gapmer oligonucleotides.
JP7503072B2 (en) 2019-02-26 2024-06-19 ロシュ イノベーション センター コペンハーゲン エーエス Methods for formulating oligonucleotides
KR20210134003A (en) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. Antisense oligomers for the treatment of conditions and diseases
WO2020176771A1 (en) 2019-02-27 2020-09-03 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
WO2020191177A1 (en) 2019-03-21 2020-09-24 Sudhir Agrawal Antisense oligonucleotides for allele specificity
KR20210141554A (en) 2019-03-21 2021-11-23 코디악 바이오사이언시즈, 인크. Extracellular vesicle conjugates and uses thereof
TW202104595A (en) 2019-03-29 2021-02-01 日商田邊三菱製藥股份有限公司 Compound, method and pharmaceutical composition for dux4 expression adjustment
TW202536179A (en) 2019-03-29 2025-09-16 美商Ionis製藥公司 Compounds and methods for modulating ube3a-ats
JP7725370B2 (en) 2019-04-03 2025-08-19 ブリストル－マイヤーズ スクイブ カンパニー ANGPTL2 antisense oligonucleotides and uses thereof
WO2020201339A1 (en) 2019-04-04 2020-10-08 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
EP3950696A4 (en) * 2019-04-05 2023-01-04 Nippon Shokubai Co., Ltd. MANUFACTURE OF BRIDGED ARTIFICIAL NUCLEOSIDE
CN113661168A (en) 2019-04-16 2021-11-16 罗氏创新中心哥本哈根有限公司 New method for preparing nucleotide P (V) monomer
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
WO2020243292A1 (en) 2019-05-28 2020-12-03 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing fus expression
WO2020245233A1 (en) 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
WO2020252262A1 (en) 2019-06-14 2020-12-17 The Scripps Research Institute Reagents and methods for replication, transcription, and translation in semi-synthetic organisms
CN110204583B (en) * 2019-07-01 2021-03-23 中国人民解放军军事科学院军事医学研究院 Modified nucleoside, nucleotide and modified nucleic acid polymer as well as preparation method and application thereof
PL3956450T3 (en) 2019-07-26 2025-12-22 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
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
CN114729356A (en) 2019-08-14 2022-07-08 科迪亚克生物科学公司 Extracellular vesicle-ASO constructs targeting STAT6
EP4013878A1 (en) 2019-08-14 2022-06-22 Codiak BioSciences, Inc. Extracellular vesicle-aso constructs targeting cebp/beta
CN120771302A (en) 2019-08-14 2025-10-14 隆萨销售股份公司 Extracellular vesicles linked to molecules and uses thereof
CA3147365A1 (en) 2019-08-14 2021-02-18 Joanne LIM Extracellular vesicle-nlrp3 antagonist
CN114729327A (en) 2019-08-14 2022-07-08 科迪亚克生物科学公司 Extracellular vesicles with STAT3 antisense oligonucleotides
EP4013872A1 (en) 2019-08-14 2022-06-22 Codiak BioSciences, Inc. Extracellular vesicles with antisense oligonucleotides targeting kras
KR20220062517A (en) 2019-08-15 2022-05-17 아이오니스 파마수티컬즈, 인코포레이티드 Linkage-modified oligomeric compounds and uses thereof
EP4013454A1 (en) 2019-08-15 2022-06-22 Synthorx, Inc. Immuno oncology combination therapies with il-2 conjugates
CA3148135A1 (en) 2019-08-23 2021-03-04 Carolina E. CAFFARO Il-15 conjugates and uses thereof
WO2021050554A1 (en) 2019-09-10 2021-03-18 Synthorx, Inc. Il-2 conjugates and methods of use to treat autoimmune diseases
EP4034247A1 (en) 2019-09-25 2022-08-03 Codiak BioSciences, Inc. Sting agonist comprising exosomes for treating neuroimmunological disorders
CN110590886B (en) * 2019-09-26 2021-03-23 中国人民解放军军事科学院军事医学研究院 Modified nucleoside, nucleotide and nucleic acid polymer and preparation method and application thereof
US12503699B2 (en) 2019-10-04 2025-12-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing UGT1a1 gene expression
CN114728017B (en) 2019-10-14 2025-10-28 阿斯利康(瑞典)有限公司 Regulators of PNPLA3 expression
EP4045652A1 (en) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
CA3158320A1 (en) 2019-10-22 2021-04-29 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
WO2021087036A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
JP2022554272A (en) 2019-11-04 2022-12-28 シンソークス， インコーポレイテッド Interleukin 10 conjugates and uses thereof
IL292865A (en) 2019-11-13 2022-07-01 Alnylam Pharmaceuticals Inc Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
EP4061945A1 (en) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
IL293824A (en) 2019-12-13 2022-08-01 Alnylam Pharmaceuticals Inc Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
CN115176007A (en) 2019-12-16 2022-10-11 阿尔尼拉姆医药品有限公司 Compositions containing PATATIN-like phospholipase domain 3 (PNPLA 3) iRNA and methods of use thereof
WO2021122921A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of cops3 inhibitors for treating hepatitis b virus infection
JP7634542B2 (en) 2019-12-19 2025-02-21 エフ． ホフマン－ラ ロシュ アーゲー Use of SEPT9 inhibitors for treating hepatitis B virus infection - Patents.com
WO2021122910A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of sbds inhibitors for treating hepatitis b virus infection
CN114867856A (en) 2019-12-19 2022-08-05 豪夫迈·罗氏有限公司 Use of SARAF inhibitors for the treatment of hepatitis B virus infection
JP2023506540A (en) 2019-12-19 2023-02-16 エフ． ホフマン－ラ ロシュ エージー． Use of SCAMP3 inhibitors to treat hepatitis B virus infection
WO2021123086A1 (en) 2019-12-20 2021-06-24 F. Hoffmann-La Roche Ag Enhanced oligonucleotides for inhibiting scn9a expression
EP4081217A1 (en) 2019-12-24 2022-11-02 F. Hoffmann-La Roche AG Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
EP4081639A1 (en) 2019-12-24 2022-11-02 F. Hoffmann-La Roche AG Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
JP7797393B2 (en) 2020-01-16 2026-01-13 ブリストル－マイヤーズ スクイブ カンパニー Reagents for modular enantiodivergent synthesis of C-P bonds and uses thereof
WO2021158810A1 (en) 2020-02-05 2021-08-12 Bristol-Myers Squibb Company Oligonucleotides for splice modulation of camk2d
PH12022552080A1 (en) 2020-02-10 2024-02-12 Alnylam Pharmaceuticals Inc Compositions and methods for silencing vegf-a expression
EP4103715A4 (en) 2020-02-14 2024-03-06 University Of Massachusetts OLIGONUCLEOTIDES DIRECTED AGAINST FRATAXIN AND RELATED METHODS
CN115397989A (en) 2020-02-18 2022-11-25 阿尔尼拉姆医药品有限公司 Apolipoprotein C3 (APOC 3) iRNA compositions and methods of use thereof
US12606589B2 (en) 2020-02-19 2026-04-21 Osaka University Bridged nucleoside and nucleotide using same
AU2021225957A1 (en) 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2
EP4110916A1 (en) 2020-02-28 2023-01-04 F. Hoffmann-La Roche AG Oligonucleotides for modulating cd73 exon 7 splicing
JP7773730B2 (en) 2020-03-02 2025-11-20 国立大学法人京都大学 Prevention or treatment of aneurysms with miR-33b inhibitors
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
AU2021232029A1 (en) 2020-03-06 2022-10-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin (TTR)
MX2022011009A (en) 2020-03-06 2022-10-07 Alnylam Pharmaceuticals Inc KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF.
WO2021184020A1 (en) 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Methods of treating neuroinflammation
WO2021184021A1 (en) 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting pmp22
EP4121534A1 (en) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
EP4127134A4 (en) 2020-04-01 2024-07-31 Alnylam Pharmaceuticals, Inc. IRNA DRUG COMPOSITIONS CONTAINING ALPHA-2A ADRENERGEN RECEPTOR (ADRA2A) AND METHODS OF USE THEREOF
IL297121A (en) 2020-04-06 2022-12-01 Alnylam Pharmaceuticals Inc Compositions and methods for silencing myoc expression
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
AU2021251754A1 (en) 2020-04-07 2022-11-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing SCN9A expression
JP2023522957A (en) 2020-04-21 2023-06-01 フラッグシップ パイオニアリング， インコーポレイテッド BIFUNCTIONAL MOLECULES AND METHODS OF USE THEREOF
JP7844347B2 (en) 2020-04-27 2026-04-13 アルナイラム ファーマシューティカルズ， インコーポレイテッド Apolipoprotein E (ApoE) iRNA formulation and method of use thereof
MX2022013606A (en) 2020-04-30 2023-01-16 Alnylam Pharmaceuticals Inc COMPOSITIONS OF INTERFERENT RIBONUCLEIC ACID (ARNI) OF COMPLEMENT FACTOR B (CFB) AND METHODS OF USE THEREOF.
TWI901676B (en) 2020-05-01 2025-10-21 美商Ionis製藥公司 Compounds and methods for modulating atxn1
CN115551519A (en) 2020-05-11 2022-12-30 基因泰克公司 Complement Component C1S Inhibitors and Related Compositions, Systems, and Methods of Using Them for Treatment of Neurological Disorders
CN115698290A (en) 2020-05-11 2023-02-03 基因泰克公司 Complement Component 4 Inhibitors and Related Compositions, Systems, and Methods Using Them for Treating Neurological Disorders
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
CN115605592A (en) 2020-05-11 2023-01-13 基因泰克公司（Us） Complement Component C1R Inhibitors and Related Compositions, Systems, and Methods of Using Them for Treatment of Neurological Disorders
JP7776420B2 (en) 2020-05-12 2025-11-26 田辺三菱製薬株式会社 Compounds, methods and pharmaceutical compositions for modulating ataxin 3 expression
WO2021229036A1 (en) 2020-05-13 2021-11-18 F. Hoffmann-La Roche Ag Oligonucleotide agonists targeting progranulin
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)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
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)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
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)
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
US20230193269A1 (en) 2020-05-22 2023-06-22 Boehringer Ingelheim International Gmbh Oligonucleotides for splice modulation of card9
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
EP4162047A1 (en) 2020-06-09 2023-04-12 Roche Innovation Center Copenhagen A/S Guanosine analogues for use in therapeutic polynucleotides
WO2021252557A1 (en) 2020-06-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
CN116075592A (en) 2020-06-09 2023-05-05 阿尔尼拉姆医药品有限公司 SIRNA compositions and methods for silencing GPAM (mitochondrial glycerol-3-phosphate acyltransferase 1) expression
AU2021292296A1 (en) 2020-06-18 2023-01-19 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof
WO2021263026A1 (en) 2020-06-25 2021-12-30 Synthorx, Inc. Immuno oncology combination therapy with il-2 conjugates and anti-egfr antibodies
AR122731A1 (en) 2020-06-26 2022-10-05 Hoffmann La Roche IMPROVED OLIGONUCLEOTIDES TO MODULATE FUBP1 EXPRESSION
TW202216996A (en) 2020-06-29 2022-05-01 美商Ionis製藥公司 Compounds and methods for modulating plp1
EP4183416A4 (en) 2020-07-17 2025-08-27 Mitsubishi Tanabe Pharma Corp Means for the prevention or treatment of muscular diseases
WO2022018155A1 (en) 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
EP4185696A1 (en) 2020-07-23 2023-05-31 F. Hoffmann-La Roche AG Oligonucleotides targeting rna binding protein sites
MX2023001222A (en) 2020-07-28 2023-04-26 Ionis Pharmaceuticals Inc Compounds and methods for reducing app expression.
IL300258A (en) 2020-08-07 2023-03-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating scn2a
JP2023538630A (en) 2020-08-21 2023-09-08 エフ． ホフマン－ラ ロシュ アーゲー Use of A1CF inhibitors to treat hepatitis B virus infection
TW202227102A (en) 2020-09-22 2022-07-16 瑞典商阿斯特捷利康公司 Method of treating fatty liver disease
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
EP4225917A1 (en) 2020-10-05 2023-08-16 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
WO2022076596A1 (en) 2020-10-06 2022-04-14 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting stat6
IL301612A (en) 2020-10-09 2023-05-01 Synthorx Inc Immuno-oncological treatments with IL-2 couples
EP4225376A1 (en) 2020-10-09 2023-08-16 Synthorx, Inc. Immuno oncology combination therapy with il-2 conjugates and pembrolizumab
CN116761885A (en) 2020-10-23 2023-09-15 斯克利普斯研究所 Reverse transcription of polynucleotides containing unnatural nucleotides
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
CA3200595A1 (en) 2020-11-13 2022-05-19 Alnylam Pharmaceuticals, Inc. Coagulation factor v (f5) irna compositions and methods of use thereof
WO2022107025A1 (en) 2020-11-18 2022-05-27 Lemba Bv Umlilo antisense transcription inhibitors
KR20230108728A (en) 2020-11-18 2023-07-18 아이오니스 파마수티컬즈, 인코포레이티드 Compounds and methods for modulating angiotensinogen expression
AU2021382146A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
WO2022117745A1 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
TW202237843A (en) 2020-12-03 2022-10-01 瑞士商赫孚孟拉羅股份公司 Antisense oligonucleotides targeting atxn3
EP4259642A1 (en) 2020-12-08 2023-10-18 F. Hoffmann-La Roche AG Novel synthesis of phosphorodithioate oligonucleotides
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
CR20230308A (en) 2020-12-11 2023-09-08 Civi Biopharma Inc ORAL DELIVERY OF ANTISENSE CONJUGATES TARGETTING PCSK9
AR124408A1 (en) 2020-12-18 2023-03-22 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS TO MODULATE FACTOR XII
EP4263831A1 (en) 2020-12-18 2023-10-25 F. Hoffmann-La Roche AG Antisense oligonucleotides for targeting progranulin
CN116670282A (en) 2020-12-22 2023-08-29 豪夫迈·罗氏有限公司 Oligonucleotides targeting XBP1
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
TW202246500A (en) 2021-02-02 2022-12-01 瑞士商赫孚孟拉羅股份公司 Enhanced oligonucleotides for inhibiting rtel1 expression
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
WO2022174101A1 (en) 2021-02-12 2022-08-18 Synthorx, Inc. Skin cancer combination therapy with il-2 conjugates and cemiplimab
TW202302148A (en) 2021-02-12 2023-01-16 美商欣爍克斯公司 Lung cancer combination therapy with il-2 conjugates and an anti-pd-1 antibody or antigen-binding fragment thereof
JP2024512236A (en) 2021-02-17 2024-03-19 ロンザ セールス アーゲー Extracellular vesicles - NLRP3 antagonist
EP4294456A1 (en) 2021-02-17 2023-12-27 Lonza Sales AG Extracellular vesicle linked to a biologically active molecule via an optimized linker and an anchoring moiety
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
CA3211059A1 (en) 2021-02-26 2022-09-01 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
BR112023017737A2 (en) 2021-03-04 2023-10-03 Alnylam Pharmaceuticals Inc ANGIOPOIETIN-SIMILAR IRNA COMPOSITIONS 3 (ANGPTL3) AND METHODS OF USE THEREOF
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
BR112023019981A2 (en) 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc COMPOSITIONS OF HUNTINGTINA IRNA AGENT (HTT) AND METHODS OF USE THEREOF
CA3213989A1 (en) 2021-04-01 2022-10-06 Conlin O'NEIL Extracellular vesicle compositions
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
CA3216106A1 (en) 2021-04-26 2022-11-03 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4331606A4 (en) 2021-04-30 2025-05-07 Kyoto University PREVENTION OR TREATMENT OF MYOPATHY WITH MIR-33B INHIBITOR
JP2024522068A (en) 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 Sodium-glucose cotransporter 2 (SGLT2) IRNA compositions and methods of use thereof
US20240263177A1 (en) 2021-05-20 2024-08-08 Korro Bio, Inc. Methods and Compositions for Adar-Mediated Editing
MX2023014008A (en) 2021-05-27 2023-12-11 Alnylam Pharmaceuticals Inc Compositions and methods for silencing carbonic anhydrase 2 expression.
AU2022286925A1 (en) 2021-05-29 2023-12-21 1Globe Health Institute Llc Short duplex dna as a novel gene silencing technology and use thereof
EP4347829A1 (en) 2021-05-29 2024-04-10 1Globe Health Institute LLC Asymmetric 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
WO2022256395A1 (en) 2021-06-02 2022-12-08 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2022256534A1 (en) 2021-06-03 2022-12-08 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
MX2023014168A (en) 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF.
CA3222546A1 (en) 2021-06-08 2022-12-15 F. Hoffmann-La Roche Ag Oligonucleotide progranulin agonists
JP2024523000A (en) 2021-06-08 2024-06-25 アルナイラム ファーマシューティカルズ， インコーポレイテッド Compositions and methods for treating or preventing Stargardt's disease and/or retinal binding protein 4 (RBP4)-associated disorders
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
US11753644B2 (en) 2021-06-18 2023-09-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing IFNAR1 expression
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
IL309296A (en) 2021-06-30 2024-02-01 Alnylam Pharmaceuticals Inc Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
JP2024528659A (en) 2021-07-19 2024-07-30 アルナイラム ファーマシューティカルズ， インコーポレイテッド Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluric disease or disorder - Patents.com
TW202325312A (en) 2021-07-23 2023-07-01 美商艾拉倫製藥股份有限公司 Beta-catenin (ctnnb1) irna compositions and methods of use thereof
JP2024529437A (en) 2021-07-29 2024-08-06 アルナイラム ファーマシューティカルズ， インコーポレイテッド 3-hydroxy-3-methylglutaric-coa reductase (hmgcr) iRNA compositions and methods of use thereof
AR126675A1 (en) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSITIONS OF RNAi AGAINST TRANSTHYRRETIN (TTR) AND THEIR METHODS OF USE
CA3228255A1 (en) 2021-08-04 2023-02-09 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing angiotensinogen (agt)
CN118076737A (en) 2021-08-13 2024-05-24 阿尔尼拉姆医药品有限公司 Factor XII (F12) iRNA compositions and methods of use thereof
EP4388094A1 (en) 2021-08-16 2024-06-26 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
TW202325311A (en) 2021-08-31 2023-07-01 美商艾拉倫製藥公司 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
JP2024535850A (en) 2021-09-17 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド iRNA Compositions and Methods for Silencing Complement Component (C3)
JP2024535888A (en) 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 Inhibin subunit beta E (INHBE) modulator compositions and methods of use thereof
KR20240067943A (en) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 Microtubule-Associated Protein Tau (MAPT) iRNA Preparation Composition and Method of Using Same
CN118019848A (en) 2021-09-29 2024-05-10 豪夫迈·罗氏有限公司 RNA editing
AU2022357561A1 (en) 2021-10-01 2024-04-18 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
US20240401045A1 (en) 2021-10-01 2024-12-05 Adarx Pharmaceuticals, Inc. Angiotensinogen-modulating compositions and methods of use thereof
AU2022370009A1 (en) 2021-10-22 2024-05-16 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
AR127477A1 (en) 2021-10-29 2024-01-31 Alnylam Pharmaceuticals Inc COMPOSITIONS OF RNAi AGAINST COMPLEMENT FACTOR B (CFB) AND METHODS OF USE THEREOF
EP4423272A2 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2023078883A1 (en) 2021-11-03 2023-05-11 F. Hoffmann-La Roche Ag Oligonucleotides for modulating apolipoprotein e4 expression
US20250019702A1 (en) 2021-11-10 2025-01-16 University Of Rochester Gata4-targeted therapeutics for treatment of cardiac hypertrophy
EP4430187A2 (en) 2021-11-10 2024-09-18 University of Rochester Antisense oligonucleotides for modifying protein expression
AU2022384619A1 (en) 2021-11-11 2024-04-11 F. Hoffmann-La Roche Ag Pharmaceutical combinations for treatment of hbv
EP4441224A4 (en) 2021-12-03 2026-03-11 Quralis Corp GAPMER-ANTISENSE OLIGONUCLEOTIDS WITH MODIFIED RECORD CHEMISTRY
CN118434858A (en) 2021-12-07 2024-08-02 豪夫迈·罗氏有限公司 Antisense oligonucleotides targeting ACTL6B
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
EP4448763A1 (en) 2021-12-17 2024-10-23 F. Hoffmann-La Roche AG Oligonucleotide gba agonists
CN119095603A (en) 2021-12-17 2024-12-06 豪夫迈·罗氏有限公司 Combinations of oligonucleotides for regulating RTEL1 and FUBP1
EP4452327A1 (en) 2021-12-20 2024-10-30 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
JP2024547071A (en) 2021-12-20 2024-12-26 エフ． ホフマン－ラ ロシュ アーゲー Threose nucleic acid antisense oligonucleotides and methods thereof
WO2023122762A1 (en) 2021-12-22 2023-06-29 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
WO2023127857A1 (en) * 2021-12-27 2023-07-06 株式会社理研ジェネシス Novel artificial nucleic acid, method for producing same, and use of same
KR20240131441A (en) 2022-01-10 2024-08-30 오스퍼바이오 테라퓨틱스 인크. Regulation of hepatitis B virus (HBV) expression
WO2023141507A1 (en) 2022-01-20 2023-07-27 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 (en) 2022-02-21 2024-05-22 Hoffmann La Roche ANTISENSE OLIGONUCLEOTIDE
JP2025516554A (en) 2022-05-10 2025-05-30 エフ． ホフマン－ラ ロシュ アーゲー Antisense oligonucleotides targeting the CFP-ELK1 intergenic region
PE20250394A1 (en) 2022-05-18 2025-02-11 Hoffmann La Roche Improved oligonucleotides targeting RNA-binding protein sites
KR20250022763A (en) 2022-06-10 2025-02-17 캠프4 테라퓨틱스 코포레이션 Method for modulating progranulin expression using antisense oligonucleotides targeting regulatory RNA
WO2023242324A1 (en) 2022-06-17 2023-12-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for targeting progranulin
US20260049309A1 (en) 2022-08-15 2026-02-19 Novo Nordisk A/S Regulation of activity of rnai molecules
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
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
JP2025529283A (en) 2022-09-06 2025-09-04 エフ． ホフマン－ラ ロシュ アーゲー Double-stranded RNA molecules for ocular administration
CN119907856A (en) 2022-09-15 2025-04-29 阿尔尼拉姆医药品有限公司 17β-Hydroxysteroid Dehydrogenase Type 13 (HSD17B13) iRNA Compositions and Methods of Using Them
JP2025532127A (en) 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ， インコーポレーテッド Compounds and methods for reducing MECP2 expression
US20260166072A1 (en) 2022-11-04 2026-06-18 Genkardia Inc. Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure
EP4627084A1 (en) 2022-12-01 2025-10-08 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
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
WO2024160756A1 (en) 2023-01-30 2024-08-08 Vib Vzw Suppressors of tauopathies
IL322261A (en) 2023-02-09 2025-09-01 Alnylam Pharmaceuticals Inc Reversir molecules and methods of use thereof
WO2024175586A2 (en) 2023-02-21 2024-08-29 Vib Vzw Inhibitors of synaptogyrin-3 expression
WO2024175588A1 (en) 2023-02-21 2024-08-29 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
WO2024178172A1 (en) 2023-02-23 2024-08-29 University Of Rochester Agents and methods for making closed-end dna thread molecules
CN121263208A (en) 2023-03-20 2026-01-02 新索思股份有限公司 Cancer therapy using IL-2 PEG conjugates
KR20260021596A (en) 2023-04-05 2026-02-13 아이오니스 파마수티컬즈, 인코포레이티드 Compounds and methods for reducing PLN expression
IL323663A (en) 2023-04-06 2025-11-01 Shanghai Argo Biopharmaceutical Co Ltd Nucleoside analog for 5'-phosphonate modification and oligonucleotide prepared therefrom
EP4698652A2 (en) 2023-04-20 2026-02-25 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
KR20260007576A (en) 2023-04-28 2026-01-14 빔 테라퓨틱스, 인크. Modified guide RNA
CN121263524A (en) 2023-05-04 2026-01-02 豪夫迈·罗氏有限公司 Oligonucleotides capable of up-regulating glucocerebrosidase expression
WO2024233864A2 (en) 2023-05-10 2024-11-14 Dicerna Pharmaceuticals, Inc. Galnac-conjugated rnai oligonucleotides
WO2024238396A1 (en) 2023-05-12 2024-11-21 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
CN116606338B (en) * 2023-05-18 2026-06-02 中国人民解放军军事科学院军事医学研究院 6’-Cyano-modified nucleosides, nucleotides and nucleic acid polymers
WO2024249328A2 (en) 2023-05-26 2024-12-05 Adarx Pharmaceuticals, Inc. Sod1-modulating compositions and methods of use thereof
KR20260026070A (en) 2023-06-16 2026-02-25 에프. 호프만-라 로슈 아게 Double-stranded oligonucleotides for modulating JAK1 expression
CN121712891A (en) 2023-06-20 2026-03-20 阿达尔克斯制药有限公司 LRRK2 modulating compositions and methods of use thereof
JPWO2025005265A1 (en) * 2023-06-30 2025-01-02
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
KR20260044219A (en) 2023-07-21 2026-04-01 매로우 테라퓨틱스, 인크. Hematopoietic cell targeting conjugate and related methods
KR20260044217A (en) 2023-07-25 2026-04-01 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 Cas Endonuclease and Related Methods
JP2026525612A (en) 2023-07-25 2026-07-31 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー Cas endonuclease and related methods
EP4754255A1 (en) 2023-08-04 2026-06-10 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
KR20260089666A (en) 2023-09-14 2026-06-15 아이오니스 파마수티컬즈, 인코포레이티드 Compounds and methods for reducing APOCIII expression
WO2025059511A1 (en) 2023-09-15 2025-03-20 Praxis Precision Medicines, Inc. Elsunersen / prax-222 for use in the treatment of scn2a-related disorders
UY40897A (en) 2023-09-21 2025-03-31 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS TO INHIBIT LPA
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
US20250115914A1 (en) 2023-10-04 2025-04-10 Lemba Bv Compounds for inhibition of il-1beta expression
KR20260085131A (en) 2023-10-04 2026-06-08 렘바 비브이 Compounds to inhibit AMANZI
EP4791878A1 (en) 2023-10-13 2026-08-19 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
AU2024397279A1 (en) 2023-12-13 2026-07-02 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with ube3a overexpression
AR134762A1 (en) 2023-12-21 2026-04-01 Hoffmann La Roche ANTI-SENSE OLIGONUCLETIDE
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
US20250263702A1 (en) 2024-02-19 2025-08-21 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting cideb and related methods
WO2025199231A2 (en) 2024-03-20 2025-09-25 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
TW202603156A (en) 2024-03-25 2026-01-16 美商欣爍克斯公司 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
WO2025252669A1 (en) 2024-06-03 2025-12-11 Evotec International Gmbh Modified oligonucleotides for reducing atxn3 expression
WO2025255388A1 (en) 2024-06-05 2025-12-11 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2025259743A1 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dual conjugate compounds for extrahepatic delivery
TW202615058A (en) 2024-06-12 2026-04-16 美商艾拉倫製藥股份有限公司 Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof
WO2026006151A2 (en) 2024-06-24 2026-01-02 University Of Rochester Functionalization of ace-trna encoding synthetic linear picovectors
WO2026006436A1 (en) 2024-06-25 2026-01-02 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of tar dna binding protein 43 kda (tdp43)
WO2026041784A1 (en) 2024-08-23 2026-02-26 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
WO2026050243A1 (en) 2024-08-26 2026-03-05 Korro Bio, Inc. Galnac conjugated oligonucleotides for rna editing
WO2026055461A1 (en) 2024-09-05 2026-03-12 Aperture Therapeutics, Inc. Antibody oligonucleotide conjugates comprising an antisense polynucleotide agent conjugated to a cd33 antibody and methods of use thereof
WO2026057749A1 (en) 2024-09-11 2026-03-19 Sixfold Bioscience Ltd. Method and product
EP4711455A1 (en) 2024-09-11 2026-03-18 Aarhus Universitet Small artificial rna (smartrna) oligonucleotide for modulating protein expression
WO2026062247A1 (en) 2024-09-20 2026-03-26 Astrazeneca Ab Liquid-phase oligonucleotide synthesis using 2-(2-nitrophenyl)propyloxycarbonyl (nppoc) as a protecting group
WO2026080323A1 (en) 2024-10-09 2026-04-16 Quralis Corporation Treatment of neurological diseases using modulators of unc13a gene transcripts
WO2026122478A1 (en) 2024-12-02 2026-06-11 Korro Bio, Inc. Extrahepatic delivery formulations and methods
WO2026122779A1 (en) 2024-12-06 2026-06-11 Alnylam Pharmaceuticals, Inc. Cd81 irna compositions and methods of use thereof
WO2026136379A1 (en) 2024-12-17 2026-06-25 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2026132147A1 (en) 2024-12-20 2026-06-25 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting downstream of the transcripts 3' terminus
WO2026151811A1 (en) 2025-01-07 2026-07-16 Camp4 Therapeutics Corporation Compositions and methods for treating urea cycle disorders

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030105309A1 (en) * 1997-03-07 2003-06-05 Takeshi Imanishi Novel bicyclonucleoside and oligonucleotide analogue
WO2007134181A2 (en) * 2006-05-11 2007-11-22 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs

Family Cites Families (125)

* 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
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4668777A (en) * 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
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
USRE34069E (en) * 1983-08-18 1992-09-15 Biosyntech Gmbh Process for the preparation of oligonucleotides
DE3329892A1 (en) * 1983-08-18 1985-03-07 Köster, Hubert, Prof. Dr., 2000 Hamburg METHOD FOR PRODUCING 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 (en) * 1984-07-19 1989-02-17 Centre Nat Rech Scient NOVEL OLIGONUCLEOTIDES, THEIR PREPARATION PROCESS AND THEIR APPLICATIONS AS MEDIATORS IN DEVELOPING THE EFFECTS OF INTERFERONS
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (en) * 1985-01-08 1987-04-03 Pasteur Institut NOVEL ADENOSINE DERIVATIVE NUCLEOSIDES, THEIR PREPARATION AND THEIR BIOLOGICAL APPLICATIONS
US5405938A (en) * 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) * 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5034506A (en) * 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5235033A (en) * 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
EP0260032B1 (en) * 1986-09-08 1994-01-26 Ajinomoto Co., Inc. Compounds for the cleavage at a specific position of RNA, oligomers employed for the formation of said compounds, and starting materials for the synthesis of said oligomers
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
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
EP0366685B1 (en) * 1987-06-24 1994-10-19 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5188897A (en) * 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
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
JP3019994B2 (en) * 1987-11-30 2000-03-15 ユニバーシティ オブ アイオワ リサーチ ファウンデーション Novel oligodeoxynucleotide, method of blocking expression of target gene using the same, and novel oligodeoxynucleotide and method of inhibiting expression of target gene using the same
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
EP0406309A4 (en) * 1988-03-25 1992-08-19 The 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
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
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
WO1991006556A1 (en) 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides
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
US5587470A (en) * 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5623065A (en) * 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5646265A (en) * 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) * 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
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
EP0455905B1 (en) * 1990-05-11 1998-06-17 Microprobe Corporation Dipsticks for nucleic acid hybridization assays and methods for covalently immobilizing oligonucleotides
US5378825A (en) * 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5541307A (en) * 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5602240A (en) * 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5608046A (en) * 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5623070A (en) * 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5489677A (en) * 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5618704A (en) * 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5610289A (en) * 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5386023A (en) * 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
JPH0874B2 (en) * 1990-07-27 1996-01-10 アイシス・ファーマシューティカルス・インコーポレーテッド Nuclease-resistant, pyrimidine-modified oligonucleotides that detect and modulate gene expression
JPH06502300A (en) 1990-08-03 1994-03-17 サノフィ Compounds and methods for suppressing gene expression
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 (en) * 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclic nucleosides, oligonucleotides, processes for their preparation and intermediates
US5594121A (en) * 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
EP0637965B1 (en) * 1991-11-26 2002-10-16 Isis Pharmaceuticals, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) * 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified 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 (en) * 1992-02-05 1994-10-28 Centre Nat Rech Scient OLIGOTHIONUCLEOTIDES.
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
EP0577558A2 (en) * 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclic nucleosides having bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates
US5652355A (en) * 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304620D0 (en) * 1993-03-06 1993-04-21 Ciba Geigy Ag Compounds
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
HU9501974D0 (en) * 1993-03-31 1995-09-28 Sterling Winthrop Inc Oligonucleotides with amide linkages replacing phosphodiester linkages
DE4311944A1 (en) * 1993-04-10 1994-10-13 Degussa Coated sodium percarbonate particles, process for their preparation and detergent, cleaning and bleaching compositions containing them
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
US5446137B1 (en) * 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
DK0733059T3 (en) 1993-12-09 2000-10-16 Univ Jefferson Compounds and Methods for Site-directed Mutations in Eukaryotic Cells
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
JP3756313B2 (en) * 1997-03-07 2006-03-15 武 今西 Novel bicyclonucleosides and oligonucleotide analogues
EP1015469B2 (en) † 1997-09-12 2015-11-18 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
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
BRPI0008131B8 (en) 1999-02-12 2021-05-25 Daiichi Sankyo Co Ltd compound or a salt thereof, oligonucleotide analog, pharmaceutical composition, probe for a gene, initiator to begin amplification, use of an oligonucleotide analog or a pharmacologically acceptable salt thereof, antisense agent, and, antigen agent
US7084125B2 (en) * 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
ATE356824T1 (en) * 1999-05-04 2007-04-15 Santaris Pharma As L-RIBO-LNA ANALOGUE
JP4151751B2 (en) * 1999-07-22 2008-09-17 第一三共株式会社 New bicyclonucleoside analogues
WO2002018388A1 (en) * 2000-08-29 2002-03-07 Takeshi Imanishi Novel nucleoside analogs and oligonucleotide derivatives containing these analogs
US6426220B1 (en) * 2000-10-30 2002-07-30 Isis Pharmaceuticals, Inc. Antisense modulation of calreticulin expression
US7060809B2 (en) 2001-09-04 2006-06-13 Exiqon A/S LNA compositions and uses thereof
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
EP2284269B1 (en) † 2002-11-18 2017-08-09 Roche Innovation Center Copenhagen A/S Antisense design
DE602007009487D1 (en) † 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-MODIFIED BICYCLIC NUCLEIC ACID ANALOGUE
EP2505646A1 (en) † 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of CRP

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030105309A1 (en) * 1997-03-07 2003-06-05 Takeshi Imanishi Novel bicyclonucleoside and oligonucleotide analogue
WO2007134181A2 (en) * 2006-05-11 2007-11-22 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JESPER WENGEL ET AL: "Chemistry of locked nucleic acids (LNA): Design, synthesis, and bio-physical properties", INTERNATIONAL JOURNAL OF PEPTIDE RESEARCH AND THERAPEUTICS ; FORMERLY KNOWN AS LETTERS IN PEPTIDE SCIENCE, KLUWER ACADEMIC PUBLISHERS, DO, vol. 10, no. 3-4, 1 May 2003 (2003-05-01), pages 237 - 253, XP019287568, ISSN: 1573-3904 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11279929B2 (en) 2018-07-03 2022-03-22 Hoffmann-La Roche, Inc. Oligonucleotides for modulating Tau expression
US11753640B2 (en) 2018-07-03 2023-09-12 Hoffmann-La Roche Inc. Oligonucleotides for modulating Tau expression
US11077195B2 (en) 2019-02-06 2021-08-03 Synthorx, Inc. IL-2 conjugates and methods of use thereof
US12521437B2 (en) 2019-02-06 2026-01-13 Synthorx, Inc. IL-2 conjugates and methods of use thereof

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