SG10201913786SA - Modified rna agents with reduced off-target effect - Google Patents
Modified rna agents with reduced off-target effectInfo
- Publication number
- SG10201913786SA SG10201913786SA SG10201913786SA SG10201913786SA SG10201913786SA SG 10201913786S A SG10201913786S A SG 10201913786SA SG 10201913786S A SG10201913786S A SG 10201913786SA SG 10201913786S A SG10201913786S A SG 10201913786SA SG 10201913786S A SG10201913786S A SG 10201913786SA
- Authority
- SG
- Singapore
- Prior art keywords
- reduced
- modified rna
- target effect
- rna agents
- agents
- Prior art date
Links
Classifications
-
- 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
- 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
- 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
-
- 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.
-
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
-
- 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
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Plant Pathology (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (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 |
|---|---|---|---|
| US201662425907P | 2016-11-23 | 2016-11-23 | |
| US201762548589P | 2017-08-22 | 2017-08-22 | |
| US201762561514P | 2017-09-21 | 2017-09-21 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG10201913786SA true SG10201913786SA (en) | 2020-03-30 |
Family
ID=60888585
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG10201913786SA SG10201913786SA (en) | 2016-11-23 | 2017-11-22 | Modified rna agents with reduced off-target effect |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11504391B1 (zh) |
| EP (1) | EP3544617A4 (zh) |
| JP (2) | JP7288852B2 (zh) |
| KR (2) | KR20240035907A (zh) |
| CN (2) | CN117757791A (zh) |
| AU (2) | AU2017363892B2 (zh) |
| CA (1) | CA3044598A1 (zh) |
| IL (1) | IL266780A (zh) |
| MX (2) | MX2019005816A (zh) |
| SG (1) | SG10201913786SA (zh) |
| WO (1) | WO2018098328A1 (zh) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| BR112017021967A2 (pt) | 2015-05-06 | 2018-07-31 | Alnylam Pharmaceuticals Inc | composições do fator xii (fator hageman) (f12), kallikrein b, plasma (fator fletcher) 1 (klkb1), e kininogen 1 (kng1) irna e métodos de uso dos mesmos |
| CN117757791A (zh) | 2016-11-23 | 2024-03-26 | 阿尔尼拉姆医药品有限公司 | 具有降低的脱靶效应的修饰的rna试剂 |
| FR3060947A1 (fr) * | 2016-12-28 | 2018-06-29 | Ynsect | Procede de traitement d'insectes comprenant la separation des cuticules de la partie molle des insectes puis la separation de la partie molle en trois fractions |
| WO2019010342A1 (en) * | 2017-07-07 | 2019-01-10 | Alnylam Pharmaceuticals, Inc. | METHODS OF TREATING OR PREVENTING DISEASES ASSOCIATED WITH THE CONTACT ACTIVATION PATHWAY USING FIBER XII-TARGETING RNAI COMPOSITIONS (FACTOR HAGEMAN) (F12) |
| EP3790557A4 (en) * | 2018-05-07 | 2022-03-02 | Alnylam Pharmaceuticals Inc. | COMPOSITIONS AND METHODS FOR ENHANCING STRAND DISTORTION |
| KR20210018267A (ko) | 2018-05-07 | 2021-02-17 | 알닐람 파마슈티칼스 인코포레이티드 | 간외 전달 |
| US20210238595A1 (en) * | 2018-05-16 | 2021-08-05 | Alnylam Pharmaceuticals, Inc. | Modified rna agents with reduced off-target effect |
| US11066669B2 (en) | 2018-06-05 | 2021-07-20 | Hoffmann-La Roche Inc. | Oligonucleotides for modulating ATXN2 expression |
| BR112021000538A2 (pt) | 2018-07-13 | 2021-04-06 | F. Hoffmann-La Roche Ag | Oligonucleotídeos para modular a expressão de rtel1 |
| MX2021001056A (es) * | 2018-08-13 | 2021-04-12 | Alnylam Pharmaceuticals Inc | Composiciones de agente de acido ribonucleico bicatenario (arnbc) de virus de la hepatitis b (vhb) y metodos de uso de las mismas. |
| WO2020206115A2 (en) | 2019-04-03 | 2020-10-08 | Bristol-Myers Squibb Company | Angptl2 antisense oligonucleotides and uses thereof |
| US20220411800A1 (en) * | 2019-07-16 | 2022-12-29 | The University Of Tokyo | Rna molecule, chimeric na molecule, double-stranded rna molecule, and double-stranded chimeric na molecule |
| CN114555621A (zh) | 2019-08-15 | 2022-05-27 | Ionis制药公司 | 键修饰的寡聚化合物及其用途 |
| BR112022003860A2 (pt) * | 2019-09-03 | 2022-08-16 | Alnylam Pharmaceuticals Inc | Composições e métodos para inibir a expressão do gene lect2 |
| BR112022009216A2 (pt) * | 2019-11-13 | 2022-08-02 | Alnylam Pharmaceuticals Inc | Métodos e composições para tratar um distúrbio associado a angiotensinogênio (agt) |
| JP2023506550A (ja) | 2019-12-19 | 2023-02-16 | エフ． ホフマン－ラ ロシュ エージー． | Ｂ型肝炎ウイルス感染症を処置するためのｓｂｄｓ阻害剤の使用 |
| CN114829599A (zh) | 2019-12-19 | 2022-07-29 | 豪夫迈·罗氏有限公司 | Scamp3抑制剂用于治疗乙型肝炎病毒感染的用途 |
| WO2021122921A1 (en) | 2019-12-19 | 2021-06-24 | F. Hoffmann-La Roche Ag | Use of cops3 inhibitors for treating hepatitis b virus infection |
| CN114901821A (zh) | 2019-12-19 | 2022-08-12 | 豪夫迈·罗氏有限公司 | Sept9抑制剂用于治疗乙型肝炎病毒感染的用途 |
| WO2021122993A1 (en) | 2019-12-19 | 2021-06-24 | F. Hoffmann-La Roche Ag | Use of saraf inhibitors for treating hepatitis b virus infection |
| TW202137987A (zh) | 2019-12-24 | 2021-10-16 | 瑞士商赫孚孟拉羅股份公司 | 用於治療ｈｂｖ之靶向ｈｂｖ的治療性寡核苷酸及ｔｌｒ７促效劑之醫藥組合 |
| JP2023509872A (ja) | 2019-12-24 | 2023-03-10 | エフ． ホフマン－ラ ロシュ アーゲー | Ｈｂｖを標的とする抗ウイルス剤及び／又はｈｂｖの処置のための免疫調節剤の医薬組合せ |
| CA3172111A1 (en) | 2020-03-19 | 2021-09-23 | Barbora MALECOVA | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| WO2021231211A1 (en) | 2020-05-11 | 2021-11-18 | Genentech, Inc. | Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same |
| WO2021231204A1 (en) | 2020-05-11 | 2021-11-18 | Genentech, Inc. | Complement component 4 inhibitors for treating neurological diseases, and related compositons, systems and methods of using same |
| CN115605592A (zh) | 2020-05-11 | 2023-01-13 | 基因泰克公司（Us） | 用于治疗神经系统疾病的补体组分c1r抑制剂以及相关的组合物、系统和使用它们的方法 |
| EP4200419A2 (en) | 2020-08-21 | 2023-06-28 | F. Hoffmann-La Roche AG | Use of a1cf inhibitors for treating hepatitis b virus infection |
| AU2021345266A1 (en) * | 2020-09-17 | 2023-04-27 | Q-State Biosciences, Inc. | Therapeutic compositions for treating pain via multiple targets |
| EP4279591A1 (en) * | 2021-01-15 | 2023-11-22 | The University of Tokyo | Rna molecule, chimeric na molecule, double-stranded rna molecule, and double-stranded chimeric na molecule |
| KR20230146048A (ko) | 2021-02-12 | 2023-10-18 | 알닐람 파마슈티칼스 인코포레이티드 | 슈퍼옥사이드 디스뮤타제 1(sod1) irna 조성물 및 슈퍼옥사이드 디스뮤타제 1- (sod1-) 관련 신경퇴행성 질환을 치료하거나 예방하기 위한 이의 사용 방법 |
| MX2023011466A (es) | 2021-03-29 | 2024-02-01 | Alnylam Pharmaceuticals Inc | Composiciones de agentes de ácido ribonucleico de interferencia (arni) de huntingtina (htt) y métodos de uso de estas. |
| AU2022328347A1 (en) | 2021-08-13 | 2024-02-08 | Alnylam Pharmaceuticals, Inc. | Factor xii (f12) irna compositions and methods of use thereof |
| EP4401792A1 (en) | 2021-09-16 | 2024-07-24 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| IL312635A (en) | 2021-11-11 | 2024-07-01 | Hoffmann La Roche | Pharmaceutical combinations for the treatment of HBV |
| WO2023111210A1 (en) | 2021-12-17 | 2023-06-22 | F. Hoffmann-La Roche Ag | Combination of oligonucleotides for modulating rtel1 and fubp1 |
| CN116003494A (zh) * | 2022-01-05 | 2023-04-25 | 大睿生物医药科技(上海)有限公司 | 具有核苷酸类似物的双链rna |
| WO2024098061A2 (en) | 2022-11-04 | 2024-05-10 | Genkardia Inc. | Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure |
| WO2024108217A1 (en) | 2022-11-18 | 2024-05-23 | Genkardia Inc. | Methods and compositions for preventing, treating, or reversing cardiac diastolic dysfunction |
| WO2024140101A1 (zh) * | 2022-12-28 | 2024-07-04 | 北京炫景瑞医药科技有限公司 | 修饰的双链寡核苷酸分子、修饰的双链寡核苷酸缀合物及其用途 |
| US20240254485A1 (en) * | 2023-01-13 | 2024-08-01 | Hemoshear Therapeutics, Inc. | Sirnas targeting slc10a1 transcripts, compositions and uses thereof |
| WO2024175588A1 (en) | 2023-02-21 | 2024-08-29 | Vib Vzw | Oligonucleotides for modulating synaptogyrin-3 expression |
| WO2024175586A2 (en) | 2023-02-21 | 2024-08-29 | Vib Vzw | Inhibitors of synaptogyrin-3 expression |
| WO2024176153A1 (en) * | 2023-02-22 | 2024-08-29 | Auris Medical Ag | Compositions and methods for kras inhibition for the treatment of disease |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN103045601B (zh) | 2004-04-22 | 2015-04-01 | 雷加多生物科学公司 | 改良的凝血因子调节物 |
| EP1681347A1 (en) * | 2005-01-18 | 2006-07-19 | Metanomics GmbH & Co. KGaA | Improved methods for double-stranded RNA mediated gene silencing |
| AU2007229161B2 (en) * | 2006-03-23 | 2012-07-12 | Roche Innovation Center Copenhagen A/S | Small internally segmented interfering RNA |
| US20140170191A1 (en) | 2008-10-23 | 2014-06-19 | Hemispher Biopharma, Inc. | Novel double-stranded ribonucleic acids with rugged physico-chemical structure and highly specific biologic activity |
| JP2012528596A (ja) | 2009-06-03 | 2012-11-15 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | ペプチドダイサー基質剤およびその特異的な遺伝子発現阻害のための方法 |
| WO2011109427A2 (en) * | 2010-03-01 | 2011-09-09 | Alnylam Pharmaceuticals, Inc. | Improving the biological activity of sirna through modulation of its thermodynamic profile |
| US10913767B2 (en) * | 2010-04-22 | 2021-02-09 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising acyclic and abasic nucleosides and analogs |
| CN103502449B (zh) * | 2010-12-02 | 2016-08-10 | 关键基因股份有限公司 | 用寡核苷酸靶向改变dna |
| WO2013013068A2 (en) * | 2011-07-19 | 2013-01-24 | University Of Idaho | Embodiments of a probe and method for targeting nucleic acids |
| KR20220159478A (ko) | 2013-10-04 | 2022-12-02 | 알닐람 파마슈티칼스 인코포레이티드 | Ａｌａｓ1 유전자의 발현을 억제하기 위한 조성물 및 방법 |
| BR122020024443B1 (pt) * | 2014-05-01 | 2022-02-22 | Ionis Pharmaceuticals, Inc | Composto e composição farmacêutica para modulação da expressão de angptl3 |
| EP3812462A1 (en) * | 2014-08-20 | 2021-04-28 | Alnylam Pharmaceuticals, Inc. | Modified double-stranded rna agents |
| JP2018504380A (ja) | 2014-12-18 | 2018-02-15 | アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | Ｒｅｖｅｒｓｉｒ（商標）化合物 |
| CN117757791A (zh) | 2016-11-23 | 2024-03-26 | 阿尔尼拉姆医药品有限公司 | 具有降低的脱靶效应的修饰的rna试剂 |
| EP4041248A4 (en) | 2019-10-11 | 2024-03-20 | Alnylam Pharmaceuticals Inc. | MODIFIED OLIGONUCLEOTIDES |
-
2017
- 2017-11-22 CN CN202311754990.3A patent/CN117757791A/zh active Pending
- 2017-11-22 EP EP17873158.4A patent/EP3544617A4/en active Pending
- 2017-11-22 CN CN201780084435.0A patent/CN110582283B/zh active Active
- 2017-11-22 US US16/461,523 patent/US11504391B1/en active Active
- 2017-11-22 JP JP2019527517A patent/JP7288852B2/ja active Active
- 2017-11-22 MX MX2019005816A patent/MX2019005816A/es unknown
- 2017-11-22 WO PCT/US2017/063078 patent/WO2018098328A1/en unknown
- 2017-11-22 CA CA3044598A patent/CA3044598A1/en active Pending
- 2017-11-22 SG SG10201913786SA patent/SG10201913786SA/en unknown
- 2017-11-22 KR KR1020247007251A patent/KR20240035907A/ko not_active Application Discontinuation
- 2017-11-22 AU AU2017363892A patent/AU2017363892B2/en active Active
- 2017-11-22 KR KR1020197018071A patent/KR102645243B1/ko active IP Right Grant
-
2019
- 2019-05-17 MX MX2023010958A patent/MX2023010958A/es unknown
- 2019-05-21 IL IL266780A patent/IL266780A/en unknown
-
2022
- 2022-09-30 US US17/937,256 patent/US20230256001A1/en active Pending
-
2023
- 2023-01-20 JP JP2023007453A patent/JP2023053963A/ja active Pending
- 2023-09-05 AU AU2023226646A patent/AU2023226646A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20190086001A (ko) | 2019-07-19 |
| MX2023010958A (es) | 2023-12-07 |
| US11504391B1 (en) | 2022-11-22 |
| CN110582283A (zh) | 2019-12-17 |
| JP2023053963A (ja) | 2023-04-13 |
| MX2019005816A (es) | 2019-10-07 |
| CA3044598A1 (en) | 2018-05-31 |
| CN117757791A (zh) | 2024-03-26 |
| AU2023226646A1 (en) | 2023-11-16 |
| EP3544617A4 (en) | 2020-08-05 |
| JP2019535288A (ja) | 2019-12-12 |
| EP3544617A1 (en) | 2019-10-02 |
| KR20240035907A (ko) | 2024-03-18 |
| KR102645243B1 (ko) | 2024-03-11 |
| US20230256001A1 (en) | 2023-08-17 |
| IL266780A (en) | 2019-08-29 |
| JP7288852B2 (ja) | 2023-06-08 |
| WO2018098328A1 (en) | 2018-05-31 |
| AU2017363892B2 (en) | 2023-06-15 |
| CN110582283B (zh) | 2024-01-02 |
| AU2017363892A1 (en) | 2019-06-13 |
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