CA3118537A1 - Modified double stranded oligonucleotides - Google Patents
Modified double stranded oligonucleotides Download PDFInfo
- Publication number
- CA3118537A1 CA3118537A1 CA3118537A CA3118537A CA3118537A1 CA 3118537 A1 CA3118537 A1 CA 3118537A1 CA 3118537 A CA3118537 A CA 3118537A CA 3118537 A CA3118537 A CA 3118537A CA 3118537 A1 CA3118537 A1 CA 3118537A1
- Authority
- CA
- Canada
- Prior art keywords
- nucleotides
- length
- strand
- ligand
- antisense strand
- 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
- C12N15/111—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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
-
- 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 nucleic acids [NA]
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- 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
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862758094P | 2018-11-09 | 2018-11-09 | |
| US62/758,094 | 2018-11-09 | ||
| PCT/US2019/059818 WO2020097044A1 (en) | 2018-11-09 | 2019-11-05 | Modified double stranded oligonucleotides |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3118537A1 true CA3118537A1 (en) | 2020-05-14 |
Family
ID=70612465
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3118537A Pending CA3118537A1 (en) | 2018-11-09 | 2019-11-05 | Modified double stranded oligonucleotides |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US12600965B2 (https=) |
| EP (1) | EP3877524A4 (https=) |
| JP (2) | JP7536007B2 (https=) |
| AU (2) | AU2019376628B2 (https=) |
| CA (1) | CA3118537A1 (https=) |
| WO (1) | WO2020097044A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3216595A1 (en) | 2013-03-14 | 2014-10-02 | Kevin Fitzgerald | Complement component c5 irna compositions and methods of use thereof |
| TWI851574B (zh) | 2018-05-14 | 2024-08-11 | 美商阿尼拉製藥公司 | 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法 |
| CA3118537A1 (en) * | 2018-11-09 | 2020-05-14 | Alnylam Pharmaceuticals, Inc. | Modified double stranded oligonucleotides |
| KR20220115946A (ko) * | 2019-11-13 | 2022-08-19 | 알닐람 파마슈티칼스 인코포레이티드 | 안지오텐시노겐 (agt) 관련 장애를 치료하기 위한 방법 및 조성물 |
| MX2023008469A (es) * | 2021-01-22 | 2023-11-22 | Alnylam Pharmaceuticals Inc | Oligonucleotidos de doble hebra modificados. |
| WO2022174000A2 (en) | 2021-02-12 | 2022-08-18 | 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 |
| WO2022212231A2 (en) | 2021-03-29 | 2022-10-06 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| KR20240067943A (ko) | 2021-09-24 | 2024-05-17 | 알닐람 파마슈티칼스 인코포레이티드 | 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법 |
| EP4493570A2 (en) | 2022-03-16 | 2025-01-22 | Empirico Inc. | Galnac compositions for improving sirna bioavailability |
| KR20240163743A (ko) | 2022-03-28 | 2024-11-19 | 엠피리코 인크. | 변형된 올리고뉴클레오티드 |
| EP4662311A2 (en) * | 2023-02-09 | 2025-12-17 | Alnylam Pharmaceuticals, Inc. | Reversir molecules and methods of use thereof |
| WO2026046152A1 (zh) * | 2024-08-26 | 2026-03-05 | 倍臻生物技术（苏州）有限公司 | 修饰的双链核糖核酸及其应用 |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2652134B1 (en) * | 2010-12-17 | 2017-03-01 | Arrowhead Pharmaceuticals, Inc. | Galactose cluster-pharmacokinetic modulator targeting moiety for sirna |
| EP2780454A2 (en) | 2011-11-18 | 2014-09-24 | Alnylam Pharmaceuticals, Inc. | Modified rnai agents |
| HUE048622T2 (hu) | 2011-11-18 | 2020-08-28 | Alnylam Pharmaceuticals Inc | RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére |
| US9127274B2 (en) | 2012-04-26 | 2015-09-08 | Alnylam Pharmaceuticals, Inc. | Serpinc1 iRNA compositions and methods of use thereof |
| KR102486617B1 (ko) | 2013-05-22 | 2023-01-12 | 알닐람 파마슈티칼스 인코포레이티드 | Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법 |
| TW201610151A (zh) | 2013-09-23 | 2016-03-16 | 阿尼拉製藥公司 | 治療或預防與甲狀腺素運載蛋白相關疾病之方法 |
| TWI768330B (zh) | 2013-10-04 | 2022-06-21 | 美國西奈山伊坎醫學院 | 抑制alas1基因表現的組合物及方法 |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| TWI864340B (zh) | 2014-10-10 | 2024-12-01 | 美商艾爾妮蘭製藥公司 | 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法 |
| TWI710633B (zh) | 2014-11-10 | 2020-11-21 | 美商阿尼拉製藥公司 | Ｂ型肝炎病毒（ＨＢＶ）ｉＲＮＡ組成物及其用途方法 |
| HK1244843A1 (zh) | 2014-11-17 | 2018-08-17 | Alnylam Pharmaceuticals, Inc. | 载脂蛋白c3(apoc3)irna组合物及其使用方法 |
| WO2016085852A1 (en) | 2014-11-24 | 2016-06-02 | Alnylam Pharmaceuticals, Inc. | Tmprss6 irna compositions and methods of use thereof |
| EA201792263A1 (ru) | 2015-04-13 | 2018-08-31 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ НА ОСНОВЕ iRNA ПРОТИВ АНГИОПОЭТИН-ПОДОБНОГО БЕЛКА 3 (ANGPTL3) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| 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 |
| CN108138182B (zh) | 2015-07-31 | 2022-08-19 | 阿尔尼拉姆医药品有限公司 | 甲状腺素运载蛋白(TTR)iRNA组合物及其治疗或预防TTR相关疾病的使用方法 |
| CN118697894A (zh) | 2015-08-25 | 2024-09-27 | 阿尔尼拉姆医药品有限公司 | 用于治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物 |
| US11260134B2 (en) | 2016-09-29 | 2022-03-01 | National University Corporation Tokyo Medical And Dental University | Double-stranded nucleic acid complex having overhang |
| TW202313978A (zh) | 2016-11-23 | 2023-04-01 | 美商阿尼拉製藥公司 | 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法 |
| KR102645243B1 (ko) | 2016-11-23 | 2024-03-11 | 알닐람 파마슈티칼스 인코포레이티드 | 표적외 효과가 감소된 변형 rna 작용제 |
| AU2017376950B2 (en) | 2016-12-16 | 2024-02-22 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing TTR-associated diseases using transthyretin (TTR) iRNA compositions |
| IL269927B2 (en) | 2017-04-18 | 2025-04-01 | Alnylam Pharmaceuticals Inc | Methods of treating patients with hepatitis B virus infection |
| AU2018301477A1 (en) | 2017-07-13 | 2020-02-27 | Alnylam Pharmaceuticals Inc. | Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof |
| JP7507093B2 (ja) | 2018-03-21 | 2024-06-27 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法 |
| CA3106701A1 (en) | 2018-08-13 | 2020-02-20 | Alnylam Pharmaceuticals, Inc. | Hepatitis b virus (hbv) dsrna agent compositions and methods of use thereof |
| CA3118537A1 (en) * | 2018-11-09 | 2020-05-14 | Alnylam Pharmaceuticals, Inc. | Modified double stranded oligonucleotides |
-
2019
- 2019-11-05 CA CA3118537A patent/CA3118537A1/en active Pending
- 2019-11-05 WO PCT/US2019/059818 patent/WO2020097044A1/en not_active Ceased
- 2019-11-05 US US17/291,775 patent/US12600965B2/en active Active
- 2019-11-05 AU AU2019376628A patent/AU2019376628B2/en active Active
- 2019-11-05 JP JP2021523902A patent/JP7536007B2/ja active Active
- 2019-11-05 EP EP19882543.2A patent/EP3877524A4/en active Pending
-
2024
- 2024-08-06 JP JP2024129696A patent/JP2024149701A/ja active Pending
-
2025
- 2025-10-29 AU AU2025259857A patent/AU2025259857A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3877524A1 (en) | 2021-09-15 |
| JP7536007B2 (ja) | 2024-08-19 |
| US20210388356A1 (en) | 2021-12-16 |
| AU2019376628A1 (en) | 2021-06-03 |
| JP2022506503A (ja) | 2022-01-17 |
| WO2020097044A1 (en) | 2020-05-14 |
| AU2019376628B2 (en) | 2025-08-07 |
| JP2024149701A (ja) | 2024-10-18 |
| AU2025259857A1 (en) | 2026-01-22 |
| EP3877524A4 (en) | 2023-10-11 |
| US12600965B2 (en) | 2026-04-14 |
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