AU2019392928A1 - Oligonucleotide compositions and methods thereof - Google Patents
Oligonucleotide compositions and methods thereof Download PDFInfo
- Publication number
- AU2019392928A1 AU2019392928A1 AU2019392928A AU2019392928A AU2019392928A1 AU 2019392928 A1 AU2019392928 A1 AU 2019392928A1 AU 2019392928 A AU2019392928 A AU 2019392928A AU 2019392928 A AU2019392928 A AU 2019392928A AU 2019392928 A1 AU2019392928 A1 AU 2019392928A1
- Authority
- AU
- Australia
- Prior art keywords
- composition
- dmd
- oligonucleotide
- sfu
- oligonucleotides
- 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
Links
Classifications
-
- 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
-
- 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
-
- 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
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862776432P | 2018-12-06 | 2018-12-06 | |
| US62/776,432 | 2018-12-06 | ||
| PCT/US2019/027109 WO2019200185A1 (en) | 2018-04-12 | 2019-04-11 | Oligonucleotide compositions and methods of use thereof |
| AUPCT/US2019/027109 | 2019-04-11 | ||
| PCT/US2019/031672 WO2019217784A1 (en) | 2018-05-11 | 2019-05-10 | Oligonucleotide compositions and methods of use thereof |
| AUPCT/US2019/031672 | 2019-05-10 | ||
| US201962916192P | 2019-10-16 | 2019-10-16 | |
| US201962916194P | 2019-10-16 | 2019-10-16 | |
| US62/916,194 | 2019-10-16 | ||
| US62/916,192 | 2019-10-16 | ||
| PCT/US2019/065058 WO2020118246A1 (en) | 2018-12-06 | 2019-12-06 | Oligonucleotide compositions and methods thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2019392928A1 true AU2019392928A1 (en) | 2021-06-17 |
Family
ID=70973999
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2019392928A Pending AU2019392928A1 (en) | 2018-12-06 | 2019-12-06 | Oligonucleotide compositions and methods thereof |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20220186217A1 (https=) |
| EP (1) | EP3891284A4 (https=) |
| JP (2) | JP2022513719A (https=) |
| CN (1) | CN113383078A (https=) |
| AU (1) | AU2019392928A1 (https=) |
| CA (1) | CA3122271A1 (https=) |
| SG (1) | SG11202105626TA (https=) |
| WO (1) | WO2020118246A1 (https=) |
Families Citing this family (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| JP6453212B2 (ja) | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | キラル制御 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| RU2708237C2 (ru) | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| CN108135921B (zh) | 2015-07-22 | 2023-10-17 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| CA3015823A1 (en) | 2016-03-13 | 2017-09-21 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| CN109562122A (zh) | 2016-06-03 | 2019-04-02 | 波涛生命科学有限公司 | 寡核苷酸、组合物及其方法 |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| US10941402B2 (en) | 2016-09-01 | 2021-03-09 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded RNA-editing oligonucleotides |
| MA46905A (fr) | 2016-11-23 | 2019-10-02 | 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 |
| US11597927B2 (en) | 2017-06-02 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US11718638B2 (en) | 2017-06-21 | 2023-08-08 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
| TW201920672A (zh) | 2017-08-08 | 2019-06-01 | 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 |
| CN111108096A (zh) | 2017-09-18 | 2020-05-05 | 波涛生命科学有限公司 | 寡核苷酸制备技术 |
| EP3694530A4 (en) | 2017-10-12 | 2021-06-30 | Wave Life Sciences Ltd. | COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES |
| IL277889B2 (en) | 2018-04-12 | 2025-01-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods of using them |
| EP3790596A4 (en) | 2018-05-11 | 2022-04-06 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| CN113748116A (zh) * | 2019-03-20 | 2021-12-03 | 波涛生命科学有限公司 | 可用于寡核苷酸制备的技术 |
| WO2021113270A1 (en) | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Therapeutic editing |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| EP4476202A1 (en) * | 2022-02-11 | 2024-12-18 | Wave Life Sciences Ltd. | Stereoselective technologies for chiral compounds |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| AR128918A1 (es) * | 2022-03-30 | 2024-06-26 | Biomarin Pharm Inc | Oligonucleótidos que omiten el exón 51 de distrofina y sus aplicaciones |
| EP4555086A1 (en) | 2022-07-15 | 2025-05-21 | 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 |
| AR131145A1 (es) | 2022-11-24 | 2025-02-19 | Proqr Therapeutics Ii Bv | Oligonucleótidos antisentido para el tratamiento de hemocromatosis hfe hereditaria |
| 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 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 |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| WO2024206175A1 (en) | 2023-03-24 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| AU2024246572A1 (en) | 2023-03-27 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| WO2024256620A1 (en) | 2023-06-16 | 2024-12-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurodegenerative disease |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2025072879A1 (en) * | 2023-09-28 | 2025-04-03 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2025072881A2 (en) * | 2023-09-28 | 2025-04-03 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| 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 |
| GB202404661D0 (en) | 2024-04-02 | 2024-05-15 | Proqr Therapeutics Ii Bv | Antisense oligoncleotides for the treatment of liver disease |
| WO2025213142A2 (en) * | 2024-04-05 | 2025-10-09 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2025224230A1 (en) | 2024-04-25 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of fatty liver disease |
| GB202410081D0 (en) | 2024-07-11 | 2024-08-28 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of cardiovascular disease |
| WO2026022136A1 (en) | 2024-07-23 | 2026-01-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2026060374A2 (en) | 2024-09-16 | 2026-03-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| WO2026068781A1 (en) | 2024-09-30 | 2026-04-02 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| WO2026080323A1 (en) | 2024-10-09 | 2026-04-16 | Quralis Corporation | Treatment of neurological diseases using modulators of unc13a gene transcripts |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6617442B1 (en) * | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
| AU2002362312A1 (en) * | 2001-09-17 | 2003-04-01 | Mayo Foundation For Medical Education And Research | Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof |
| AU2008317566B2 (en) * | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| JP5786109B2 (ja) * | 2008-10-27 | 2015-09-30 | プロセンサ テクノロジーズ ベー．フェー． | Ｄｕｃｈｅｎｎｅ型筋ジストロフィーｍＲＮＡ前駆体のエクソン４５の効率的なスキッピングのための方法及び手段 |
| WO2011109427A2 (en) * | 2010-03-01 | 2011-09-09 | Alnylam Pharmaceuticals, Inc. | Improving the biological activity of sirna through modulation of its thermodynamic profile |
| JP6453212B2 (ja) * | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | キラル制御 |
| ES2917473T3 (es) * | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| SMT201900169T1 (it) * | 2014-03-12 | 2019-05-10 | Nippon Shinyaku Co Ltd | Acido nucleico antisenso |
| RU2708237C2 (ru) * | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| EP3858993A1 (en) * | 2015-10-09 | 2021-08-04 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| JP7511326B2 (ja) * | 2015-10-09 | 2024-07-05 | 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およびその方法 |
| CA3029772A1 (en) * | 2016-07-05 | 2018-01-11 | Biomarin Technologies B.V. | Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders |
-
2019
- 2019-12-06 SG SG11202105626TA patent/SG11202105626TA/en unknown
- 2019-12-06 EP EP19891722.1A patent/EP3891284A4/en active Pending
- 2019-12-06 JP JP2021531966A patent/JP2022513719A/ja not_active Ceased
- 2019-12-06 AU AU2019392928A patent/AU2019392928A1/en active Pending
- 2019-12-06 CN CN201980090833.2A patent/CN113383078A/zh active Pending
- 2019-12-06 WO PCT/US2019/065058 patent/WO2020118246A1/en not_active Ceased
- 2019-12-06 US US17/311,285 patent/US20220186217A1/en not_active Abandoned
- 2019-12-06 CA CA3122271A patent/CA3122271A1/en active Pending
-
2024
- 2024-12-02 JP JP2024209791A patent/JP2025041652A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020118246A1 (en) | 2020-06-11 |
| SG11202105626TA (en) | 2021-06-29 |
| US20220186217A1 (en) | 2022-06-16 |
| CA3122271A1 (en) | 2020-06-11 |
| EP3891284A1 (en) | 2021-10-13 |
| JP2022513719A (ja) | 2022-02-09 |
| CN113383078A (zh) | 2021-09-10 |
| JP2025041652A (ja) | 2025-03-26 |
| EP3891284A4 (en) | 2023-04-12 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| DA3 | Amendments made section 104 |
Free format text: THE NATURE OF THE AMENDMENT IS: AMEND THE NAME OF THE INVENTOR TO READ ZHANG, JASON JINGXIN; VARGEESE, CHANDRA; IWAMOTO, NAOKI; SHIVALILA, CHIKDU SHAKTI; KOTHARI, NAYANTARA; DURBIN, ANN FIEGEN; RAMASAMY, SELVI; KANDASAMY, PACHAMUTHU; KUMARASAMY, JAYAKANTHAN; BOMMINENI, GOPAL REDDY; MARAPPAN, SUBRAMANIAN; DIVAKARAMENON, SETHUMADHAVAN; BUTLER, DAVID CHARLES DONNELL; LU, GENLIANG; YANG, HAILIN; SHIMIZU, MAMORU; MONIAN, PRASHANT AND LUU, KHOA NGOC DANG |