CA3176986A1 - Oligonucleotide compositions and methods thereof - Google Patents

Oligonucleotide compositions and methods thereof Download PDF

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Publication number
CA3176986A1
CA3176986A1 CA3176986A CA3176986A CA3176986A1 CA 3176986 A1 CA3176986 A1 CA 3176986A1 CA 3176986 A CA3176986 A CA 3176986A CA 3176986 A CA3176986 A CA 3176986A CA 3176986 A1 CA3176986 A1 CA 3176986A1
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CA
Canada
Prior art keywords
independently
optionally substituted
heteroatoms
membered
optionally
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.)
Pending
Application number
CA3176986A
Other languages
English (en)
French (fr)
Inventor
Pachamuthu Kandasamy
Jayakanthan Kumarasamy
Chandra Vargeese
Subramanian Marappan
Gopal Reddy Bommineni
Mamoru Shimizu
Naoki Iwamoto
Stephany Michelle STANDLEY
Yuanjing LIU
Amy Jada ANDREUCCI
Genliang Lu
Onanong CHIVATAKARN
Akbar Husain KHAN
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.)
Wave Life Sciences Pte Ltd
Original Assignee
Wave Life Sciences Pte Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Wave Life Sciences Pte Ltd filed Critical Wave Life Sciences Pte Ltd
Publication of CA3176986A1 publication Critical patent/CA3176986A1/en
Pending legal-status Critical Current

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Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D295/00Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/02Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/06Phosphorus compounds without P—C bonds
    • C07F9/16Esters of thiophosphoric acids or thiophosphorous acids
    • C07F9/165Esters of thiophosphoric acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H1/00Processes for the preparation of sugar derivatives
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3176986A 2020-05-22 2021-05-24 Oligonucleotide compositions and methods thereof Pending CA3176986A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063029387P 2020-05-22 2020-05-22
US63/029,387 2020-05-22
PCT/US2021/033945 WO2021237223A1 (en) 2020-05-22 2021-05-24 Oligonucleotide compositions and methods thereof

Publications (1)

Publication Number Publication Date
CA3176986A1 true CA3176986A1 (en) 2021-11-25

Family

ID=78707682

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3176986A Pending CA3176986A1 (en) 2020-05-22 2021-05-24 Oligonucleotide compositions and methods thereof

Country Status (6)

Country Link
US (1) US20230203087A1 (cg-RX-API-DMAC7.html)
EP (1) EP4153604A4 (cg-RX-API-DMAC7.html)
JP (1) JP2023526975A (cg-RX-API-DMAC7.html)
AU (1) AU2021277407A1 (cg-RX-API-DMAC7.html)
CA (1) CA3176986A1 (cg-RX-API-DMAC7.html)
WO (1) WO2021237223A1 (cg-RX-API-DMAC7.html)

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ES2940887T3 (es) 2012-07-13 2023-05-12 Wave Life Sciences Ltd Método de preparación de oligonucleótidos quirales
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017210647A1 (en) 2016-06-03 2017-12-07 Wave Life Sciences Ltd. Oligonucleotides, compositions and methods thereof
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
DK3507366T3 (da) 2016-09-01 2020-11-02 Proqr Therapeutics Ii Bv Kemisk modificeret, enkeltstrengede rna-modificerende oligonukleotider
JP7296882B2 (ja) 2016-11-23 2023-06-23 ウェイブ ライフ サイエンシズ リミテッド ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法
JP7557941B2 (ja) 2017-06-02 2024-09-30 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその使用方法
JP2020522510A (ja) 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. オリゴヌクレオチド組成物及びその使用方法
JP7402696B2 (ja) 2017-06-21 2023-12-21 ウェイブ ライフ サイエンシズ リミテッド 合成のための化合物、組成物、及び方法
CN111032057A (zh) 2017-08-08 2020-04-17 波涛生命科学有限公司 寡核苷酸组合物及其方法
KR20250123934A (ko) 2017-09-18 2025-08-18 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 제조 기술
EP3694530A4 (en) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
US12391942B2 (en) 2018-05-11 2025-08-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
EP4555085A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Chemically modified oligonucleotides for adar-mediated rna editing
JP2025524566A (ja) 2022-07-15 2025-07-30 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ Adar媒介rna編集のためのオリゴヌクレオチドおよびその使用
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
AU2023390632A1 (en) 2022-12-09 2025-07-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
EP4669753A1 (en) 2023-02-20 2025-12-31 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
WO2024200472A1 (en) 2023-03-27 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
TW202516003A (zh) 2023-06-16 2025-04-16 荷蘭商Proqr治療上市公司Ii 用於治療神經退化性疾病之反義寡核苷酸
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025070241A1 (ja) * 2023-09-25 2025-04-03 藤本化学製品株式会社 化合物又はその塩、並びにその製造方法
WO2025072886A1 (en) * 2023-09-28 2025-04-03 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
AU2024369609A1 (en) * 2023-10-31 2025-12-11 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
WO2025151895A1 (en) * 2024-01-12 2025-07-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease

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US5216168A (en) * 1992-04-01 1993-06-01 G. D. Searle & Co. 2- and 3- amino and azido derivatives of 1,5-iminosugars
WO2011005860A2 (en) * 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
RU2708237C2 (ru) * 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
SG10202101836TA (en) * 2016-05-24 2021-03-30 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
US20220306573A1 (en) * 2018-04-12 2022-09-29 Jason Jingxi ZHANG Oligonucleotide compositions and methods of use thereof
US12391942B2 (en) * 2018-05-11 2025-08-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof

Also Published As

Publication number Publication date
AU2021277407A1 (en) 2022-11-17
JP2023526975A (ja) 2023-06-26
WO2021237223A1 (en) 2021-11-25
EP4153604A1 (en) 2023-03-29
US20230203087A1 (en) 2023-06-29
EP4153604A4 (en) 2024-11-27

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