MA45270A - Compositions d'oligonucléotides et procédés associés - Google Patents
Compositions d'oligonucléotides et procédés associésInfo
- Publication number
- MA45270A MA45270A MA045270A MA45270A MA45270A MA 45270 A MA45270 A MA 45270A MA 045270 A MA045270 A MA 045270A MA 45270 A MA45270 A MA 45270A MA 45270 A MA45270 A MA 45270A
- Authority
- MA
- Morocco
- Prior art keywords
- oligonucleotides
- compositions
- related processes
- processes
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Public Health (AREA)
- Psychology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662331960P | 2016-05-04 | 2016-05-04 | |
PCT/US2016/043542 WO2017015555A1 (fr) | 2015-07-22 | 2016-07-22 | Compositions d'oligonucléotides et procédés associés |
US201762447832P | 2017-01-18 | 2017-01-18 |
Publications (1)
Publication Number | Publication Date |
---|---|
MA45270A true MA45270A (fr) | 2017-11-09 |
Family
ID=60203309
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA045270A MA45270A (fr) | 2016-05-04 | Compositions d'oligonucléotides et procédés associés |
Country Status (6)
Country | Link |
---|---|
US (2) | US10724035B2 (fr) |
EP (1) | EP3452596A4 (fr) |
JP (1) | JP2019516680A (fr) |
CN (1) | CN109477108A (fr) |
MA (1) | MA45270A (fr) |
WO (1) | WO2017192664A1 (fr) |
Families Citing this family (64)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2011005761A1 (fr) | 2009-07-06 | 2011-01-13 | Ontorii, Inc | Nouveaux précurseurs d'acide nucléique et leurs méthodes d'utilisation |
CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
EP2872485B1 (fr) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Groupe auxiliaire asymétrique |
CN112007045A (zh) | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
BR112017011510B1 (pt) | 2014-12-17 | 2023-12-05 | Proqr Therapeutics Ii B.V. | Construto de oligonucleotídeo para a edição direcionada ao sítio de um nucleotídeo adenosina em uma sequência de rna alvo em uma célula eucariótica e uso do mesmo |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
CR20180233A (es) | 2015-10-09 | 2018-05-25 | Wave Life Sciences Ltd | Composiciones oligonucleotídicas y sus métodos |
LT3386511T (lt) | 2015-12-10 | 2021-08-25 | Ptc Therapeutics, Inc. | Hantingtono ligos gydymo būdai |
CN114685589A (zh) | 2016-03-13 | 2022-07-01 | 波涛生命科学有限公司 | 用于亚磷酰胺和寡核苷酸合成的组合物和方法 |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
EP3463386A4 (fr) | 2016-06-03 | 2020-03-04 | Wave Life Sciences Ltd. | Oligonucléotides, compositions et méthodes associées |
EP3475424A1 (fr) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Oligonucléotides d'édition d'arn monocaténaire |
PL3507366T3 (pl) | 2016-09-01 | 2021-05-04 | Proqr Therapeutics Ii B.V. | Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna |
WO2018098264A1 (fr) | 2016-11-23 | 2018-05-31 | Wave Life Sciences Ltd. | Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides |
MD3554553T2 (ro) | 2016-12-19 | 2022-10-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
EP4122497B1 (fr) | 2016-12-19 | 2024-04-10 | Sarepta Therapeutics, Inc. | Conjugués d'oligomères induisant le saut d'exons pour la dystrophie musculaire |
NZ755438A (en) | 2016-12-19 | 2023-05-26 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
TW201904587A (zh) | 2017-06-02 | 2019-02-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
IL300875A (en) | 2017-06-05 | 2023-04-01 | Ptc Therapeutics Inc | Compounds for the treatment of Huntington's disease |
EP3642182A4 (fr) | 2017-06-21 | 2020-12-09 | Wave Life Sciences Ltd. | Composés, compositions et procédés de synthèse |
MX2019015580A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
CA3067591A1 (fr) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | Procedes de traitement de la maladie de huntington |
AU2018316159A1 (en) | 2017-08-08 | 2020-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
WO2019055951A1 (fr) | 2017-09-18 | 2019-03-21 | Wave Life Sciences Ltd. | Technologies de préparation d'oligonucléotides |
EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
WO2019067975A1 (fr) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | Polythérapies pour traiter une dystrophie musculaire |
US20200254002A1 (en) | 2017-09-28 | 2020-08-13 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
JP2020536058A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
SG11202001783YA (en) | 2017-10-12 | 2020-03-30 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
BR112020013994A2 (pt) | 2018-01-12 | 2020-12-08 | Bristol-Myers Squibb Company | Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos |
KR20210005559A (ko) | 2018-03-27 | 2021-01-14 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 화합물 |
CN112218664A (zh) * | 2018-05-11 | 2021-01-12 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
EP3806868A4 (fr) | 2018-06-13 | 2022-06-22 | Sarepta Therapeutics, Inc. | Oligomères induisant un saut d'exon pour la dystrophie musculaire |
CN112805280B (zh) | 2018-06-27 | 2024-12-06 | Ptc医疗公司 | 用于治疗亨廷顿氏病的杂芳基化合物 |
RS65727B1 (sr) | 2018-06-27 | 2024-08-30 | Ptc Therapeutics Inc | Heterociklična i heteroaril jedinjenja za lečenje hantingtonove bolesti |
TW202020153A (zh) | 2018-07-27 | 2020-06-01 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
US20200190516A1 (en) | 2018-12-13 | 2020-06-18 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystropy |
MA54875A (fr) * | 2019-02-01 | 2021-12-08 | Wave Life Sciences Ltd | Compositions oligonucléotidiques et procédés associés |
JP2022528725A (ja) | 2019-04-18 | 2022-06-15 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーを治療するための組成物 |
AU2020262485A1 (en) * | 2019-04-25 | 2021-11-11 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
KR20220093335A (ko) | 2019-11-01 | 2022-07-05 | 노파르티스 아게 | 헌팅턴병의 진행을 늦추는 치료를 위한 스플라이싱 조절제의 용도 |
AU2021274944A1 (en) | 2020-05-22 | 2022-12-15 | Wave Life Sciences Ltd. | Double stranded oligonucleotide compositions and methods relating thereto |
TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
CA3232070A1 (fr) * | 2021-09-26 | 2023-03-30 | Prashant MONIAN | Compositions d'oligonucleotides et procedes associes |
CA3233242A1 (fr) | 2021-09-30 | 2023-04-06 | Sarepta Therapeutics, Inc. | Oligonucleotides antisens ayant une ou plusieurs unites abasiques |
EP4479535A1 (fr) | 2022-02-14 | 2024-12-25 | ProQR Therapeutics II B.V. | Oligonucléotides guides pour l'édition d'acides nucléiques dans le traitement de l'hypercholestérolémie |
WO2024013360A1 (fr) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucléotides chimiquement modifiés pour édition d'arn médiée par adar |
WO2024013361A1 (fr) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucléotides pour édition d'arn médiée par adar et leur utilisation |
WO2024064237A2 (fr) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Efficacité du saut d'exon médié par un oligonucléotide antisens dans le traitement de la dmd |
GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
TW202435897A (zh) | 2022-11-24 | 2024-09-16 | 荷蘭商Proqr治療上市公司Ii | 用於治療遺傳性hfe血色沉著病之反義寡核苷酸 |
GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
WO2024121373A1 (fr) | 2022-12-09 | 2024-06-13 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire |
GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
WO2024175550A1 (fr) | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire athérosclérotique |
GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
WO2024206175A1 (fr) | 2023-03-24 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement de troubles neurologiques |
WO2024200472A1 (fr) | 2023-03-27 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour traitement des maladies du foie |
TW202442246A (zh) | 2023-04-27 | 2024-11-01 | 美商薩羅塔治療公司 | 用於治療慢性腎病之反義寡聚物 |
WO2024256620A1 (fr) | 2023-06-16 | 2024-12-19 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement des maladies neurodégénératives |
Family Cites Families (99)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5512668A (en) * | 1991-03-06 | 1996-04-30 | Polish Academy Of Sciences | Solid phase oligonucleotide synthesis using phospholane intermediates |
US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
EP0729474A4 (fr) | 1993-11-16 | 1998-10-21 | Genta Inc | Oligomeres synthetiques ayant des liaisons internucleosidyle phosphonate chiralement pures melangees avec des liaisons internucleosidyle non phosphonate |
US6172209B1 (en) | 1997-02-14 | 2001-01-09 | Isis Pharmaceuticals Inc. | Aminooxy-modified oligonucleotides and methods for making same |
US6639062B2 (en) | 1997-02-14 | 2003-10-28 | Isis Pharmaceuticals, Inc. | Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom |
US6242589B1 (en) * | 1998-07-14 | 2001-06-05 | Isis Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides having modified internucleoside linkages |
US6867294B1 (en) | 1998-07-14 | 2005-03-15 | Isis Pharmaceuticals, Inc. | Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages |
US20020082227A1 (en) | 1999-09-30 | 2002-06-27 | Scott Henry | Use of oligonucleotides for inhibition of complement activation |
DE10019756A1 (de) | 2000-04-20 | 2001-10-25 | Bayer Ag | Verfahren zur Herstellung von superabsorbierenden Polymeren aus Polyacrylnitrilen |
US20050277133A1 (en) | 2001-05-18 | 2005-12-15 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
JP4348044B2 (ja) | 2002-02-12 | 2009-10-21 | 株式会社キラルジェン | 立体規則性の高いジヌクレオシドホスホロチオエートの製造法 |
US20050096284A1 (en) | 2002-02-20 | 2005-05-05 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
US7045306B2 (en) | 2003-04-28 | 2006-05-16 | The General Hospital Corporation | Method for identifying compounds in vitro that modulate the dysregulation of transcription of transcription mediated by mutant huntingtin protein |
JP4179562B2 (ja) | 2003-05-14 | 2008-11-12 | 独立行政法人科学技術振興機構 | ハンチンチン遺伝子の発現抑制 |
JP2005089441A (ja) | 2003-08-08 | 2005-04-07 | Toudai Tlo Ltd | 立体規則性の高いリン原子修飾ヌクレオチド類縁体の製造法 |
WO2005023828A1 (fr) | 2003-09-02 | 2005-03-17 | Takeshi Wada | Procede de production de ribonucleotide ou de derive de ribonucleotide |
WO2005028494A1 (fr) | 2003-09-02 | 2005-03-31 | Takeshi Wada | Procede de production d'un monomere 5'-phosphityle et d'un derive d'oligonucleotide h-phosphonate |
EP2821085B1 (fr) | 2003-09-12 | 2020-04-29 | University of Massachusetts | Interférence arn pour le traitement de troubles à gain |
WO2008005562A2 (fr) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Compositions de silençage de l'arn, et méthodes de traitement de la chorée de huntington |
US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
WO2005070859A1 (fr) | 2004-01-27 | 2005-08-04 | Takeshi Wada | Supports fluores et procedes de production de derives d'oligonucleotide utilisant ces supports |
WO2005085272A1 (fr) | 2004-03-05 | 2005-09-15 | Takeshi Wada | Monomere de boranophosphate et procede de production de derive oligonucleotide |
WO2005092909A1 (fr) | 2004-03-25 | 2005-10-06 | Toudai Tlo, Ltd. | Procédés de fabrication d'analogue ribonucléotide de forte stéréorégularité et analogue désoxyribonucléotide |
EP1885854B1 (fr) | 2005-05-06 | 2012-10-17 | Medtronic, Inc. | Procedes et sequences permettant de supprimer l'expression du gene de huntington chez les primates |
US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
US9133517B2 (en) | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
EP2316967A1 (fr) | 2005-06-28 | 2011-05-04 | Medtronic, Inc. | Méthodes et séquences pour supprimer préférentiellement l'expression du gène huntingtin muté. |
US7320965B2 (en) | 2005-10-28 | 2008-01-22 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of Huntingtin gene |
US7951934B2 (en) | 2006-01-26 | 2011-05-31 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to huntingtin |
CA2667055C (fr) | 2006-10-18 | 2017-05-09 | Isis Pharmaceuticals, Inc. | Composes antisens |
WO2008151833A2 (fr) | 2007-06-13 | 2008-12-18 | Hochschule Mannheim | Composés pour la modulation de l'agrégation de l'huntingtine, procédés et moyens d'identification de tels composés |
EP2014769B1 (fr) | 2007-06-18 | 2010-03-31 | Commissariat à l'Energie Atomique | Amortissement du bruit à base de sirna réversible de gène huntingtin de type sauvage endogènes et mutés et son application pour le traitement de la maladie de Hungtington |
JP2011515653A (ja) | 2008-02-04 | 2011-05-19 | ガラパゴス・ナムローゼ・フェンノートシャップ | 神経変性疾患の治療に有用な標的配列及びそれらの同定方法 |
CA2726866A1 (fr) * | 2008-05-09 | 2009-11-12 | The University Of British Columbia | Procedes et compositions pour le traitement de la maladie de huntington |
US8679750B2 (en) | 2008-05-09 | 2014-03-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington'S disease |
WO2009143391A2 (fr) | 2008-05-22 | 2009-11-26 | Isis Pharmaceuticals, Inc | Procédés de modulation de l’expression de creb |
WO2009148605A2 (fr) | 2008-06-04 | 2009-12-10 | Isis Pharmaceuticals, Inc. | Procédés pour traiter une hypercholestérolémie |
US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
WO2010118263A1 (fr) | 2009-04-08 | 2010-10-14 | University Of Massachusetts | Thérapies ciblant le polymorphisme nucléotidique (snp) destinées à traiter la maladie de huntington |
WO2011005761A1 (fr) | 2009-07-06 | 2011-01-13 | Ontorii, Inc | Nouveaux précurseurs d'acide nucléique et leurs méthodes d'utilisation |
WO2011015572A1 (fr) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Cibles et composés moléculaires, et procédés pour leur identification utiles dans le traitement de maladies neurodégénératives |
WO2011015573A1 (fr) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Cibles et composés moléculaires, et procédés pour les identifier, utiles dans le traitement de maladies neurodégénératives |
US8927553B2 (en) | 2009-08-10 | 2015-01-06 | Daljit Singh Dhanoa | Deuterium-enriched alkyl sulfonamides and uses thereof |
EP3178933B1 (fr) | 2009-09-11 | 2019-11-13 | Ionis Pharmaceuticals, Inc. | Modulation de l'expression d'huntingtine |
CN102574888A (zh) | 2009-09-16 | 2012-07-11 | 株式会社启拉坚 | 用于rna及其衍生物的合成的新型保护基 |
US20120289583A1 (en) | 2009-12-31 | 2012-11-15 | Curna, Inc. | Treatment of insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to irs2 and transcription factor e3 (tfe3) |
WO2011097643A1 (fr) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Réduction sélective de variants alléliques |
WO2011097644A2 (fr) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Réduction sélective de variants alléliques |
CN102918052A (zh) | 2010-03-05 | 2013-02-06 | 国立大学法人东京大学 | 硫代磷酸核糖核苷的制造方法 |
JP2011184318A (ja) | 2010-03-05 | 2011-09-22 | Univ Of Tokyo | リボヌクレシドh−ボラノホスホネート |
EP2620428B1 (fr) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Groupe auxiliaire asymétrique |
US9328346B2 (en) | 2010-11-12 | 2016-05-03 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
US8822671B2 (en) | 2010-11-30 | 2014-09-02 | The University Of Tokyo | 2'-O-modified RNA |
US10017764B2 (en) | 2011-02-08 | 2018-07-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
WO2012109667A1 (fr) | 2011-02-12 | 2012-08-16 | University Of Iowa Research Foundation | Composés thérapeutiques |
CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
DK2742136T3 (da) * | 2011-08-11 | 2017-11-20 | Ionis Pharmaceuticals Inc | Gapmerforbindelser omfattende 5'-modificerede deoxyribonukleosider i gap og anvendelser deraf |
ES2832531T3 (es) | 2011-11-30 | 2021-06-10 | Sarepta Therapeutics Inc | Oligonucleótidos para el tratamiento de enfermedades por expansión de repeticiones |
CN112007045A (zh) * | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
EP2872485B1 (fr) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Groupe auxiliaire asymétrique |
KR101835401B1 (ko) | 2012-07-13 | 2018-03-08 | 신 니뽄 바이오메디칼 라보라토리즈, 엘티디. | 키랄 핵산 어쥬번트 |
DK2906256T3 (en) | 2012-10-12 | 2018-11-19 | Ionis Pharmaceuticals Inc | SELECTIVE ANTISENSE COMPOUNDS AND APPLICATIONS THEREOF |
CA2899924A1 (fr) | 2013-02-04 | 2014-08-07 | Isis Pharmaceuticals, Inc. | Composes antisens selectifs et leurs utilisations |
JP6387084B2 (ja) | 2013-05-01 | 2018-09-05 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | アポリポタンパク質c−iiiの発現を調節するための組成物および方法 |
CA2926218A1 (fr) | 2013-10-03 | 2015-04-09 | Moderna Therapeutics, Inc. | Polynucleotides codant pour un recepteur de lipoproteines de faible densite |
JP2016537341A (ja) | 2013-11-11 | 2016-12-01 | サンガモ バイオサイエンシーズ, インコーポレイテッド | ハンチントン病を処置するための方法および組成物 |
EP3095459A4 (fr) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral |
JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
US9926556B2 (en) | 2014-04-28 | 2018-03-27 | Ionis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
CN106413760B (zh) | 2014-05-08 | 2020-01-14 | 桑格摩生物科学股份有限公司 | 用于治疗亨廷顿病的方法和组合物 |
AU2015264263B2 (en) | 2014-05-20 | 2021-08-05 | University Of Iowa Research Foundation | Huntington's disease therapeutic compounds |
US20160017327A1 (en) | 2014-07-11 | 2016-01-21 | The Johns Hopkins University | Phosphorodiamidate morpholino oligomers (pmos) and their use in suppression of mutant huntingtin expression and attenuation of neurotoxicity |
CA2955250A1 (fr) | 2014-07-16 | 2016-01-21 | Moderna Therapeutics, Inc. | Polynucleotides chimeriques |
RU2708237C2 (ru) * | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
JP2017536366A (ja) | 2014-11-19 | 2017-12-07 | ロシュ イノベーション センター コペンハーゲン エーエス | Lnaキラルホスホロチオエート |
LT3237618T (lt) | 2014-12-24 | 2019-07-10 | Uniqure Ip B.V. | Rnri sukeltas hantingtino geno slopinimas |
WO2016127002A1 (fr) | 2015-02-04 | 2016-08-11 | Bristol-Myers Squibb Company | Oligonucléotides lna à flancs alternés |
CA2976075A1 (fr) | 2015-02-10 | 2016-08-18 | Genzyme Corporation | Variant d'arni |
LT3277814T (lt) | 2015-04-03 | 2020-10-26 | University Of Massachusetts | Oligonukleotidų junginiai, skirti nukreipimui į hantingtino mrnr |
MA43072A (fr) * | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
CR20180233A (es) | 2015-10-09 | 2018-05-25 | Wave Life Sciences Ltd | Composiciones oligonucleotídicas y sus métodos |
CN114685589A (zh) | 2016-03-13 | 2022-07-01 | 波涛生命科学有限公司 | 用于亚磷酰胺和寡核苷酸合成的组合物和方法 |
MA45290A (fr) | 2016-05-04 | 2019-03-13 | Wave Life Sciences Ltd | Procédés et compositions d'agents biologiquement actifs |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
EP3463386A4 (fr) | 2016-06-03 | 2020-03-04 | Wave Life Sciences Ltd. | Oligonucléotides, compositions et méthodes associées |
WO2018022473A1 (fr) | 2016-07-25 | 2018-02-01 | Wave Life Sciences Ltd. | Phasage |
WO2018098264A1 (fr) | 2016-11-23 | 2018-05-31 | Wave Life Sciences Ltd. | Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides |
US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
WO2018223073A1 (fr) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Compositions d'oligonucléotides et leurs procédés d'utilisation |
TW201904587A (zh) | 2017-06-02 | 2019-02-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
EP3642182A4 (fr) | 2017-06-21 | 2020-12-09 | Wave Life Sciences Ltd. | Composés, compositions et procédés de synthèse |
EP3646836B1 (fr) | 2017-06-26 | 2021-10-06 | Yamaha Hatsudoki Kabushiki Kaisha | Fauteuil roulant à assistance motorisée, unité d'assistance motorisée pour fauteuil roulant, dispositif de commande pour fauteuil roulant à assistance motorisée, procédé de commande pour fauteuil roulant à assistance motorisée, et programme |
AU2018316159A1 (en) | 2017-08-08 | 2020-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
WO2019055951A1 (fr) | 2017-09-18 | 2019-03-21 | Wave Life Sciences Ltd. | Technologies de préparation d'oligonucléotides |
SG11202001783YA (en) | 2017-10-12 | 2020-03-30 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
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0
- MA MA045270A patent/MA45270A/fr unknown
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2017
- 2017-05-03 US US16/098,836 patent/US10724035B2/en not_active Expired - Fee Related
- 2017-05-03 CN CN201780038913.4A patent/CN109477108A/zh active Pending
- 2017-05-03 EP EP17793215.9A patent/EP3452596A4/fr not_active Withdrawn
- 2017-05-03 WO PCT/US2017/030753 patent/WO2017192664A1/fr unknown
- 2017-05-03 JP JP2018557087A patent/JP2019516680A/ja active Pending
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2020
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US20200056173A1 (en) | 2020-02-20 |
EP3452596A4 (fr) | 2020-03-18 |
US20220162598A1 (en) | 2022-05-26 |
WO2017192664A1 (fr) | 2017-11-09 |
US10724035B2 (en) | 2020-07-28 |
CN109477108A (zh) | 2019-03-15 |
JP2019516680A (ja) | 2019-06-20 |
EP3452596A1 (fr) | 2019-03-13 |
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