IL296476B2 - TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES - Google Patents
TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASESInfo
- Publication number
- IL296476B2 IL296476B2 IL296476A IL29647622A IL296476B2 IL 296476 B2 IL296476 B2 IL 296476B2 IL 296476 A IL296476 A IL 296476A IL 29647622 A IL29647622 A IL 29647622A IL 296476 B2 IL296476 B2 IL 296476B2
- Authority
- IL
- Israel
- Prior art keywords
- double stranded
- rnai agent
- ttr
- nucleotides
- stranded rnai
- Prior art date
Links
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- 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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P5/00—Drugs for disorders of the endocrine system
- A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
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- 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]
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/35—Nature of the modification
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- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- 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
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/158—Expression markers
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- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA
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- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
- G01N2333/46—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
- G01N2333/47—Assays involving proteins of known structure or function as defined in the subgroups
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- G01N2800/00—Detection or diagnosis of diseases
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- G—PHYSICS
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- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/28—Neurological disorders
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- G—PHYSICS
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- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
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- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
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- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
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- Diabetes (AREA)
- Hematology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
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- Hospice & Palliative Care (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562199563P | 2015-07-31 | 2015-07-31 | |
| US201662287518P | 2016-01-27 | 2016-01-27 | |
| PCT/US2016/044359 WO2017023660A1 (en) | 2015-07-31 | 2016-07-28 | TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| IL296476A IL296476A (en) | 2022-11-01 |
| IL296476B1 IL296476B1 (en) | 2024-06-01 |
| IL296476B2 true IL296476B2 (en) | 2024-10-01 |
Family
ID=56682264
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL296476A IL296476B2 (en) | 2015-07-31 | 2016-07-28 | TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES |
| IL313082A IL313082A (en) | 2015-07-31 | 2016-07-28 | TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES |
| IL256614A IL256614B2 (en) | 2015-07-31 | 2017-12-27 | Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated diseases |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL313082A IL313082A (en) | 2015-07-31 | 2016-07-28 | TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES |
| IL256614A IL256614B2 (en) | 2015-07-31 | 2017-12-27 | Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated diseases |
Country Status (35)
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010048228A2 (en) | 2008-10-20 | 2010-04-29 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of transthyretin |
| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| JP5896175B2 (ja) | 2010-04-29 | 2016-03-30 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | トランスサイレチン発現の調節 |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
| AU2012340159B2 (en) | 2011-11-18 | 2017-09-07 | Alnylam Pharmaceuticals, Inc. | RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases |
| FI3366775T4 (fi) | 2011-11-18 | 2025-11-14 | Alnylam Pharmaceuticals Inc | Modifioituja rnai-agensseja |
| CN112007045A (zh) | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
| ES2862073T3 (es) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Grupo auxiliar asimétrico |
| US10144933B2 (en) | 2014-01-15 | 2018-12-04 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| EP3095459A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| DK3329002T3 (da) | 2015-07-31 | 2021-01-11 | Alnylam Pharmaceuticals Inc | Transthyretin (ttr)-irna-sammensætninger og fremgangsmåder til anvendelse deraf til behandling eller forebyggelse af ttr-forbundne sygdomme |
| JOP20210043A1 (ar) * | 2015-10-01 | 2017-06-16 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
| TWI790217B (zh) * | 2016-12-16 | 2023-01-21 | 美商阿尼拉製藥公司 | 使用甲狀腺素運載蛋白(TTR)iRNA組成物於治療或預防TTR相關疾病之方法 |
| KR20250046363A (ko) | 2017-09-19 | 2025-04-02 | 알닐람 파마슈티칼스 인코포레이티드 | 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법 |
| EP3719128B1 (en) * | 2017-12-01 | 2025-01-15 | Suzhou Ribo Life Science Co., Ltd. | Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof |
| TW202028465A (zh) * | 2018-09-28 | 2020-08-01 | 美商阿尼拉製藥公司 | 甲狀腺素運載蛋白(TTR)iRNA組成物及其治療或預防TTR相關眼部疾病之使用方法 |
| EP4022062A1 (en) | 2019-08-30 | 2022-07-06 | Alnylam Pharmaceuticals, Inc. | Neurofilament light chain (nfl) as a biomarker for transthyretin amyloidosis polyneuropathy |
| KR20220152270A (ko) * | 2020-03-06 | 2022-11-15 | 알닐람 파마슈티칼스 인코포레이티드 | 트랜스티레틴 (ttr)의 발현을 억제하기 위한 조성물 및 방법 |
| JP2023548005A (ja) * | 2020-10-28 | 2023-11-15 | ノヴォ ノルディスク アー/エス | 抗トランスサイレチン抗体およびその使用方法 |
| AU2022242899A1 (en) * | 2021-03-24 | 2023-11-09 | Atalanta Therapeutics, Inc. | Double-stranded sirna having patterned chemical modifications |
| AR126070A1 (es) | 2021-06-08 | 2023-09-06 | Alnylam Pharmaceuticals Inc | Composiciones y métodos para tratar o prevenir la enfermedad de stargardt y/o trastornos asociados con la proteína transportadora de retinol 4 (rbp4) |
| CA3227852A1 (en) | 2021-08-03 | 2023-02-09 | Alnylam Pharmaceuticals, Inc. | Transthyretin (ttr) irna compositions and methods of use thereof |
| WO2024130142A2 (en) * | 2022-12-16 | 2024-06-20 | Amgen Inc. | Rnai constructs for inhibiting ttr expression and methods of use thereof |
| TW202530408A (zh) | 2023-09-29 | 2025-08-01 | 美商英西特羅公司 | 用於治療非酒精性脂肪肝病的組合物與方法 |
| WO2025077916A1 (zh) * | 2023-10-13 | 2025-04-17 | 北京安龙生物医药有限公司 | 靶向转甲状腺素蛋白的寡核苷酸及其用途 |
| WO2025098117A1 (zh) * | 2023-11-06 | 2025-05-15 | 成都倍特药业股份有限公司 | 一种抑制甲状腺素转运蛋白(TTR)表达的RNAi试剂、组合物及应用 |
| WO2025113470A1 (en) * | 2023-11-27 | 2025-06-05 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of transthyretin (ttr) |
| WO2025226980A2 (en) | 2024-04-25 | 2025-10-30 | Insitro, Inc. | Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013075035A1 (en) * | 2011-11-18 | 2013-05-23 | Alnylam Pharmaceuticals | Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases |
Family Cites Families (79)
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