CA3176986A1 - Oligonucleotide compositions and methods thereof - Google Patents

Oligonucleotide compositions and methods thereof Download PDF

Info

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
Authority
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

Links

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 (enExample)
EP (1) EP4153604A4 (enExample)
JP (1) JP2023526975A (enExample)
AU (1) AU2021277407A1 (enExample)
CA (1) CA3176986A1 (enExample)
WO (1) WO2021237223A1 (enExample)

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
MX2016009290A (es) 2014-01-16 2017-02-28 Wave Life Sciences Ltd Diseño quiral.
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US11013757B2 (en) 2016-06-03 2021-05-25 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
ES2837076T3 (es) 2016-09-01 2021-06-29 Proqr Therapeutics Ii Bv Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente
WO2018098264A1 (en) 2016-11-23 2018-05-31 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2018223081A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
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
EP3664816A4 (en) 2017-08-08 2021-05-19 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES
AU2018333065B2 (en) 2017-09-18 2024-04-18 Wave Life Sciences Ltd. Technologies for oligonucleotide preparation
WO2019075357A1 (en) 2017-10-12 2019-04-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
CA3098624A1 (en) 2018-05-11 2019-11-14 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
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
EP4623084A1 (en) 2022-11-24 2025-10-01 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CA3276262A1 (en) 2022-12-09 2024-06-13 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (en) 2023-02-20 2024-08-29 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
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
AR132964A1 (es) 2023-06-16 2025-08-13 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de enfermedades neurodegenerativas
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
WO2025096809A1 (en) * 2023-10-31 2025-05-08 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

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
IL263044B2 (en) * 2016-05-24 2024-06-01 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
AU2019252680B2 (en) * 2018-04-12 2025-11-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CA3098624A1 (en) * 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof

Also Published As

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

Similar Documents

Publication Publication Date Title
CA3176986A1 (en) Oligonucleotide compositions and methods thereof
WO2021234459A9 (en) Double stranded oligonucleotide compositions and methods relating thereto
BR112020020670A2 (pt) composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo
US20250262235A1 (en) Oligonucleotide compositions and methods relating thereto
EP4240849A1 (en) Oligonucleotide compositions and methods thereof
WO2021071858A1 (en) Oligonucleotide compositions and methods of use thereof
CA3187220A1 (en) Systemic delivery of oligonucleotides
US20240026358A1 (en) Oligonucleotide compositions and methods thereof
US20250302995A1 (en) Oligonucleotide compositions and methods thereof
AU2022349529A1 (en) Compositions for editing mecp2 transcripts and methods thereof
CA3232070A1 (en) Oligonucleotide compositions and methods thereof
WO2025072862A1 (en) Oligonucleotide compositions and methods thereof
CA3232068A1 (en) Double stranded oligonucleotide compositions and methods relating thereto
CN121039141A (zh) 用于rna干扰的双链寡核苷酸组合物及其相关方法
CN118139629A (zh) 用于编辑mecp2转录物的组合物及其方法