HUE050441T2 - Huntingtin-mRNS célzására szolgáló oligonukleotid-vegyületek - Google Patents
Huntingtin-mRNS célzására szolgáló oligonukleotid-vegyületekInfo
- Publication number
- HUE050441T2 HUE050441T2 HUE16716412A HUE16716412A HUE050441T2 HU E050441 T2 HUE050441 T2 HU E050441T2 HU E16716412 A HUE16716412 A HU E16716412A HU E16716412 A HUE16716412 A HU E16716412A HU E050441 T2 HUE050441 T2 HU E050441T2
- Authority
- HU
- Hungary
- Prior art keywords
- huntingtin mrna
- oligonucleotide compounds
- targeting huntingtin
- targeting
- oligonucleotide
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title 1
- 150000001875 compounds Chemical class 0.000 title 1
- 108020004999 messenger RNA Proteins 0.000 title 1
- 230000008685 targeting Effects 0.000 title 1
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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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 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
- C12N2310/32—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
- 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
-
- 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
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- 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
- C12N2310/3517—Marker; Tag
-
- 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
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2310/50—Physical structure
- C12N2310/52—Physical structure branched
-
- 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
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (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)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562142731P | 2015-04-03 | 2015-04-03 | |
| US201662289274P | 2016-01-31 | 2016-01-31 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE050441T2 true HUE050441T2 (hu) | 2020-12-28 |
Family
ID=55752785
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE16716412A HUE050441T2 (hu) | 2015-04-03 | 2016-04-01 | Huntingtin-mRNS célzására szolgáló oligonukleotid-vegyületek |
Country Status (18)
| Country | Link |
|---|---|
| US (5) | US9809817B2 (OSRAM) |
| EP (2) | EP3766973A1 (OSRAM) |
| JP (5) | JP7009356B2 (OSRAM) |
| CN (2) | CN116004624A (OSRAM) |
| AU (4) | AU2016244027B2 (OSRAM) |
| CA (1) | CA2980337A1 (OSRAM) |
| CY (1) | CY1123289T1 (OSRAM) |
| DK (1) | DK3277814T3 (OSRAM) |
| ES (1) | ES2808750T3 (OSRAM) |
| HR (1) | HRP20201200T1 (OSRAM) |
| HU (1) | HUE050441T2 (OSRAM) |
| LT (1) | LT3277814T (OSRAM) |
| PL (1) | PL3277814T3 (OSRAM) |
| PT (1) | PT3277814T (OSRAM) |
| RS (1) | RS60762B1 (OSRAM) |
| SI (1) | SI3277814T1 (OSRAM) |
| SM (1) | SMT202000454T1 (OSRAM) |
| WO (1) | WO2016161374A1 (OSRAM) |
Families Citing this family (82)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP5697993B2 (ja) | 2008-02-11 | 2015-04-08 | アールエックスアイ ファーマシューティカルズ コーポレーション | 修飾ＲＮＡｉポリヌクレオチドおよびその使用 |
| CA2753338A1 (en) | 2008-09-22 | 2010-03-25 | Rxi Pharmaceuticals Corporation | Neutral nanotransporters |
| US9074211B2 (en) | 2008-11-19 | 2015-07-07 | Rxi Pharmaceuticals Corporation | Inhibition of MAP4K4 through RNAI |
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| WO2010078536A1 (en) | 2009-01-05 | 2010-07-08 | Rxi Pharmaceuticals Corporation | Inhibition of pcsk9 through rnai |
| US9745574B2 (en) | 2009-02-04 | 2017-08-29 | Rxi Pharmaceuticals Corporation | RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
| IN2012DN00720A (OSRAM) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
| AU2011232365A1 (en) | 2010-03-24 | 2012-10-25 | Rxi Pharmaceuticals Corporation | RNA interference in dermal and fibrotic indications |
| EP3578183B1 (en) | 2010-03-24 | 2021-09-08 | Phio Pharmaceuticals Corp. | Rna interference in ocular indications |
| RU2615143C2 (ru) | 2010-03-24 | 2017-04-04 | Адвирна | Самодоставляющие PHKi соединения уменьшенного размера |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| MX347361B (es) | 2011-07-19 | 2017-04-12 | Wave Life Sciences Ltd | Metodos para la sintesis de acidos nucleicos funcionalizados. |
| EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| AU2013287630B2 (en) | 2012-07-13 | 2017-05-25 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| EP2872147B1 (en) | 2012-07-13 | 2022-12-21 | Wave Life Sciences Ltd. | Method for making chiral oligonucleotides |
| JP6772062B2 (ja) | 2013-12-02 | 2020-10-21 | フィオ ファーマシューティカルズ コーポレーションＰｈｉｏ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｃｏｒｐ． | 癌の免疫療法 |
| 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 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| CN106068325B (zh) | 2014-01-16 | 2021-07-09 | 波涛生命科学有限公司 | 手性设计 |
| EP3137119B1 (en) | 2014-04-28 | 2020-07-01 | Phio Pharmaceuticals Corp. | Methods for treating cancer using a nucleic acid targeting mdm2 |
| KR102506169B1 (ko) | 2014-09-05 | 2023-03-08 | 피오 파마슈티칼스 코프. | Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법 |
| US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ | 十分に安定化された非対称ｓｉｒｎａ |
| DK3277814T3 (da) | 2015-04-03 | 2020-08-24 | Univ Massachusetts | Oligonukleotidforbindelser til målretning mod huntingtin-mrna |
| DK3277815T3 (da) | 2015-04-03 | 2021-12-13 | Univ Massachusetts | Oligonukleotidforbindelser til behandling af præeklampsi og andre angiogene lidelser |
| US10808247B2 (en) | 2015-07-06 | 2020-10-20 | Phio Pharmaceuticals Corp. | Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach |
| CA2991598A1 (en) | 2015-07-06 | 2017-01-12 | Rxi Pharmaceuticals Corporation | Nucleic acid molecules targeting superoxide dismutase 1 (sod1) |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| WO2017070151A1 (en) | 2015-10-19 | 2017-04-27 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering nucleic acid compounds targeting long non-coding rna |
| JP7749201B6 (ja) | 2016-01-31 | 2025-10-21 | ユニバーシティー オブ マサチューセッツ | 分岐オリゴヌクレオチド |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| MX2018014152A (es) * | 2016-05-18 | 2019-03-28 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la enfermedad de huntington. |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11299733B2 (en) | 2016-07-15 | 2022-04-12 | University Of Massachusetts | Process of delivering small RNAs to sperm |
| WO2018031933A2 (en) * | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| EP3973955B1 (en) * | 2016-11-23 | 2025-05-21 | Mayo Foundation for Medical Education and Research | Particle-mediated delivery of inhibitory rna |
| CN110121558B (zh) * | 2016-11-28 | 2023-06-13 | 日商那帕洁制药股份有限公司 | 化学修饰siRNA |
| CN110520531A (zh) * | 2017-04-05 | 2019-11-29 | 赛伦斯治疗有限责任公司 | 产品和组合物 |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| AU2018261003A1 (en) | 2017-05-05 | 2019-11-14 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's Disease |
| US11603532B2 (en) * | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| TW201904587A (zh) | 2017-06-02 | 2019-02-01 | 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 |
| US10844377B2 (en) * | 2017-06-23 | 2020-11-24 | University Of Massachusetts | Two-tailed self-delivering siRNA |
| MX2020003596A (es) | 2017-10-04 | 2020-07-22 | Avidity Biosciences Inc | Composiciones de acido nucleico-polipeptido y usos de las mismos. |
| WO2019079240A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| MA51583A (fr) * | 2018-01-04 | 2020-11-11 | Avidity Biosciences Inc | Molécules d'acide nucléique hétéroduplex et leurs utilisations |
| KR20200123201A (ko) * | 2018-02-21 | 2020-10-28 | 알시온 라이프사이언스 인크. | 유체 전달 시스템 및 방법 |
| US11851654B2 (en) * | 2018-03-19 | 2023-12-26 | National University Corporation Tokyo Medical And Dental University | Nucleic acid with reduced toxicity |
| EP3781677A4 (en) | 2018-04-16 | 2022-01-19 | University of Massachusetts | COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITTING |
| AU2019265904A1 (en) | 2018-05-11 | 2020-11-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| KR20210093227A (ko) * | 2018-08-10 | 2021-07-27 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 변형된 올리고뉴클레오티드 |
| WO2020041769A1 (en) | 2018-08-23 | 2020-02-27 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| WO2020150636A1 (en) | 2019-01-18 | 2020-07-23 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| CA3132388A1 (en) * | 2019-03-08 | 2020-09-17 | Rutgers, The State University Of New Jersey | Compositions and methods for treating huntington's disease |
| WO2020198509A2 (en) * | 2019-03-26 | 2020-10-01 | University Of Massachusetts | Modified oligonucleotides with increased stability |
| CN114072501A (zh) * | 2019-05-06 | 2022-02-18 | 马萨诸塞大学 | 抗c9orf72寡核苷酸及相关方法 |
| WO2020247818A1 (en) | 2019-06-06 | 2020-12-10 | Avidity Biosciences, Inc. | Una amidites and uses thereof |
| CA3142283A1 (en) | 2019-06-06 | 2020-12-10 | Avidity Biosciences, Inc. | Nucleic acid-polypeptide compositions and uses thereof |
| US20220307024A1 (en) * | 2019-06-17 | 2022-09-29 | Alnylam Pharmaceuticals, Inc. | Delivery of oligonucleotides to the striatum |
| EP3987024A4 (en) | 2019-06-20 | 2023-11-01 | University Of Massachusetts | COMPOSITIONS AND METHODS FOR ENHANCED GENOMIC EDITING |
| MX2022001710A (es) | 2019-08-09 | 2022-05-10 | Univ Massachusetts | Oligonucleótidos modificados químicamente dirigidos a los snp. |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| JP2023500661A (ja) * | 2019-11-01 | 2023-01-10 | 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 | ハンチンチン（ＨＴＴ）ｉＲＮＡ剤組成物およびその使用方法 |
| CN112980837B (zh) * | 2019-12-13 | 2023-07-04 | 深圳艾码生物科技有限公司 | 一种抑制HTT基因表达的siRNA及其前体和应用 |
| EP4121538A4 (en) * | 2020-03-18 | 2024-07-31 | University Of Massachusetts | OLIGONUCLEOTIDES FOR SNCA MODULATION |
| AU2021244600A1 (en) | 2020-03-26 | 2022-11-03 | University Of Massachusetts | Synthesis of modified oligonucleotides with increased stability |
| WO2021216556A2 (en) * | 2020-04-20 | 2021-10-28 | University Of Massachusetts | Oligonucleotides for msh3 modulation |
| WO2022031591A2 (en) * | 2020-08-03 | 2022-02-10 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
| JP2024512612A (ja) * | 2021-03-24 | 2024-03-19 | アタランタ セラピューティクス，インコーポレーテッド | 二本鎖ｓｉＲＮＡを使用したミクログリア遺伝子サイレンシング |
| AU2022246786A1 (en) * | 2021-03-29 | 2023-10-05 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| IL309334A (en) | 2021-06-23 | 2024-02-01 | Univ Massachusetts | Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders |
| WO2023114959A2 (en) * | 2021-12-17 | 2023-06-22 | University Of Massachusetts | Oligonucleotides for mlh1 modulation |
| EP4594497A2 (en) * | 2022-09-28 | 2025-08-06 | Atalanta Therapeutics, Inc. | Compositions and methods for treatment of huntington's disease |
| WO2025006937A2 (en) * | 2023-06-30 | 2025-01-02 | Spark Therapeutics, Inc. | Inhibitory rna targeting huntingtin expression |
| WO2025073871A1 (en) | 2023-10-03 | 2025-04-10 | Bicycletx Limited | Bicyclic peptide ligand complexes specific for tfr1 |
| WO2025160434A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting huntington's disease |
Family Cites Families (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
| EP0500799B1 (en) | 1989-11-16 | 1998-01-14 | Duke University | Particle mediated transformation of animal skin tissue cells |
| US5252479A (en) * | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| US5587308A (en) * | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US5858988A (en) | 1993-02-24 | 1999-01-12 | Wang; Jui H. | Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use |
| US6291438B1 (en) | 1993-02-24 | 2001-09-18 | Jui H. Wang | Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use |
| TW404844B (en) | 1993-04-08 | 2000-09-11 | Oxford Biosciences Ltd | Needleless syringe |
| WO1996033761A1 (en) | 1995-04-28 | 1996-10-31 | Medtronic, Inc. | Intraparenchymal infusion catheter system |
| US5684143A (en) | 1996-02-21 | 1997-11-04 | Lynx Therapeutics, Inc. | Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates |
| US5735814A (en) | 1996-04-30 | 1998-04-07 | Medtronic, Inc. | Techniques of treating neurodegenerative disorders by brain infusion |
| WO2005121370A2 (en) | 2004-06-03 | 2005-12-22 | Isis Pharmaceuticals, Inc. | Oligomeric compounds that facilitate risc loading |
| US5898031A (en) | 1996-06-06 | 1999-04-27 | Isis Pharmaceuticals, Inc. | Oligoribonucleotides for cleaving RNA |
| US6177403B1 (en) | 1996-10-21 | 2001-01-23 | The Trustees Of The University Of Pennsylvania | Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal |
| US6042820A (en) | 1996-12-20 | 2000-03-28 | Connaught Laboratories Limited | Biodegradable copolymer containing α-hydroxy acid and α-amino acid units |
| US6472375B1 (en) | 1998-04-16 | 2002-10-29 | John Wayne Cancer Institute | DNA vaccine and methods for its use |
| WO2002081628A2 (en) | 2001-04-05 | 2002-10-17 | Ribozyme Pharmaceuticals, Incorporated | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies |
| WO2003029459A2 (en) | 2001-09-28 | 2003-04-10 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Micro-rna molecules |
| US20050096284A1 (en) | 2002-02-20 | 2005-05-05 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| WO2003092594A2 (en) | 2002-04-30 | 2003-11-13 | Duke University | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
| OA12890A (en) | 2002-07-19 | 2006-10-13 | Beth Israel Hospital | Methods of diagnosing and treating pre-eclampsia or eclampsia. |
| WO2004044136A2 (en) | 2002-11-05 | 2004-05-27 | Isis Pharmaceuticals, Inc. | Compositions comprising alternating 2’-modified nucleosides for use in gene modulation |
| EP1560931B1 (en) * | 2002-11-14 | 2011-07-27 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| AU2004252442B2 (en) | 2003-06-02 | 2010-04-08 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of FNAi |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| PT1633767T (pt) | 2003-06-02 | 2019-02-27 | Univ Massachusetts | Métodos e composições para controlar a eficácia do silenciamento de arn |
| WO2005060697A2 (en) | 2003-12-19 | 2005-07-07 | Chiron Corporation | Cell transfecting formulations of small interfering rna, related compositions and methods of making and use |
| WO2005077333A2 (en) | 2004-02-10 | 2005-08-25 | University Of Florida Research Foundation, Inc. | Gel-based delivery of recombinant adeno-associated virus vectors |
| EP2636739B1 (en) | 2004-03-12 | 2014-12-10 | Alnylam Pharmaceuticals Inc. | iRNA agents targeting VEGF |
| MXPA06012076A (es) | 2004-04-20 | 2007-01-25 | Nastech Pharm Co | Metodos y composiciones para mejorar el suministro de arn bicatenario o un acido nucleico hibrido bicatenario para regular la expresion genetica en celulas de mamifero. |
| EP1752536A4 (en) * | 2004-05-11 | 2008-04-16 | Alphagen Co Ltd | POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| US20070161591A1 (en) | 2005-08-18 | 2007-07-12 | University Of Massachusetts | Methods and compositions for treating neurological disease |
| JP5111385B2 (ja) * | 2005-10-28 | 2013-01-09 | 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 | ハンチンチン遺伝子の発現を抑制するための組成物および方法 |
| US20070259827A1 (en) | 2006-01-25 | 2007-11-08 | University Of Massachusetts | Compositions and methods for enhancing discriminatory RNA interference |
| US9198981B2 (en) | 2006-02-01 | 2015-12-01 | The University Of Kentucky | Modulation of angiogenesis |
| WO2007094218A1 (ja) | 2006-02-16 | 2007-08-23 | Gifu University | 修飾オリゴヌクレオチド |
| WO2007112414A2 (en) | 2006-03-27 | 2007-10-04 | Isis Pharmaceuticals, Inc. | Conjugated double strand compositions for use in gene modulation |
| CA2662704A1 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
| US20080039415A1 (en) | 2006-08-11 | 2008-02-14 | Gregory Robert Stewart | Retrograde transport of sirna and therapeutic uses to treat neurologic disorders |
| WO2008154482A2 (en) | 2007-06-08 | 2008-12-18 | Sirnaomics, Inc. | Sirna compositions and methods of use in treatment of ocular diseases |
| CN101199858A (zh) | 2007-10-18 | 2008-06-18 | 广州拓谱基因技术有限公司 | 治疗眼科疾病的多靶点小干扰rna鸡尾酒制剂及制备方法 |
| TW200927177A (en) | 2007-10-24 | 2009-07-01 | Nat Inst Of Advanced Ind Scien | Lipid-modified double-stranded RNA having potent RNA interference effect |
| US20110081362A1 (en) | 2008-01-31 | 2011-04-07 | The Brigham And Women's Hospital, Inc. | Treatment of cancer |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| CA2753338A1 (en) | 2008-09-22 | 2010-03-25 | Rxi Pharmaceuticals Corporation | Neutral nanotransporters |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| PL2470656T3 (pl) | 2009-08-27 | 2015-08-31 | Idera Pharmaceuticals Inc | Kompozycja do hamowania ekspresji genów i jej zastosowanie |
| RU2615143C2 (ru) | 2010-03-24 | 2017-04-04 | Адвирна | Самодоставляющие PHKi соединения уменьшенного размера |
| WO2012005898A2 (en) * | 2010-06-15 | 2012-01-12 | Alnylam Pharmaceuticals, Inc. | Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof |
| SG193280A1 (en) * | 2011-03-03 | 2013-10-30 | Quark Pharmaceuticals Inc | Oligonucleotide modulators of the toll-like receptor pathway |
| AU2012272908A1 (en) * | 2011-06-21 | 2013-12-19 | Alnylam Pharmaceuticals, Inc. | Assays and methods for determining activity of a therapeutic agent in a subject |
| EP3358013B1 (en) | 2012-05-02 | 2020-06-24 | Sirna Therapeutics, Inc. | Short interfering nucleic acid (sina) compositions |
| KR101567576B1 (ko) | 2012-05-22 | 2015-11-10 | 올릭스 주식회사 | 세포 내 관통능을 가지고 ｒｎａ 간섭을 유도하는 핵산 분자 및 그 용도 |
| KR102144140B1 (ko) | 2012-07-10 | 2020-08-12 | 바제클리크 게엠베하 | 아난다미드-변형된 핵산 분자 |
| US20150247141A1 (en) | 2012-09-14 | 2015-09-03 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| RU2015119411A (ru) | 2012-11-15 | 2017-01-10 | Рош Инновейшен Сентер Копенгаген А/С | Конъюгаты антисмысловых соединений, направленные на аполипопротеин в |
| US10513710B2 (en) * | 2014-04-18 | 2019-12-24 | University Of Massachusetts | Exosomal loading using hydrophobically modified oligonucleotides |
| DK3277814T3 (da) | 2015-04-03 | 2020-08-24 | Univ Massachusetts | Oligonukleotidforbindelser til målretning mod huntingtin-mrna |
| DK3277815T3 (da) | 2015-04-03 | 2021-12-13 | Univ Massachusetts | Oligonukleotidforbindelser til behandling af præeklampsi og andre angiogene lidelser |
| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ | 十分に安定化された非対称ｓｉｒｎａ |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| JP7749201B6 (ja) | 2016-01-31 | 2025-10-21 | ユニバーシティー オブ マサチューセッツ | 分岐オリゴヌクレオチド |
-
2016
- 2016-04-01 DK DK16716412.8T patent/DK3277814T3/da active
- 2016-04-01 LT LTEP16716412.8T patent/LT3277814T/lt unknown
- 2016-04-01 AU AU2016244027A patent/AU2016244027B2/en active Active
- 2016-04-01 EP EP20164108.1A patent/EP3766973A1/en active Pending
- 2016-04-01 SM SM20200454T patent/SMT202000454T1/it unknown
- 2016-04-01 SI SI201630878T patent/SI3277814T1/sl unknown
- 2016-04-01 RS RS20200929A patent/RS60762B1/sr unknown
- 2016-04-01 PT PT167164128T patent/PT3277814T/pt unknown
- 2016-04-01 HU HUE16716412A patent/HUE050441T2/hu unknown
- 2016-04-01 ES ES16716412T patent/ES2808750T3/es active Active
- 2016-04-01 HR HRP20201200TT patent/HRP20201200T1/hr unknown
- 2016-04-01 EP EP16716412.8A patent/EP3277814B1/en active Active
- 2016-04-01 US US15/089,319 patent/US9809817B2/en active Active
- 2016-04-01 CN CN202211430603.6A patent/CN116004624A/zh active Pending
- 2016-04-01 JP JP2018502624A patent/JP7009356B2/ja active Active
- 2016-04-01 PL PL16716412T patent/PL3277814T3/pl unknown
- 2016-04-01 CN CN201680031524.4A patent/CN107980062B/zh active Active
- 2016-04-01 WO PCT/US2016/025722 patent/WO2016161374A1/en not_active Ceased
- 2016-04-01 CA CA2980337A patent/CA2980337A1/en active Pending
-
2017
- 2017-09-06 US US15/697,120 patent/US10435688B2/en active Active
-
2019
- 2019-01-31 US US16/263,200 patent/US10774327B2/en active Active
-
2020
- 2020-03-06 US US16/811,580 patent/US11230713B2/en active Active
- 2020-08-24 CY CY20201100791T patent/CY1123289T1/el unknown
-
2021
- 2021-11-29 US US17/536,647 patent/US20220251554A1/en not_active Abandoned
-
2022
- 2022-01-12 JP JP2022003057A patent/JP2022064900A/ja active Pending
- 2022-01-12 JP JP2022003059A patent/JP7504482B2/ja active Active
- 2022-06-30 AU AU2022204722A patent/AU2022204722A1/en not_active Abandoned
- 2022-06-30 AU AU2022204719A patent/AU2022204719A1/en not_active Abandoned
-
2023
- 2023-12-07 JP JP2023206985A patent/JP2024028909A/ja active Pending
-
2024
- 2024-06-05 JP JP2024091478A patent/JP2024152725A/ja not_active Revoked
-
2025
- 2025-06-20 AU AU2025204686A patent/AU2025204686A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| HUE050441T2 (hu) | Huntingtin-mRNS célzására szolgáló oligonukleotid-vegyületek | |
| IL257091B (en) | Targeted oligonucleotides | |
| IL268422A (en) | Targeted oligonucleotides | |
| SG11202000276YA (en) | Technologies for oligonucleotide preparation | |
| IL252291A0 (en) | A cationic lipid that undergoes ionization to provide RNA | |
| IL256169A (en) | Multi-conjugated oligonucleotides are defined | |
| ZA201700142B (en) | Antisense nucleic acids | |
| ZA201605864B (en) | Antisense nucleic acids | |
| GB201711809D0 (en) | Antisense oligonucleotide | |
| IL258065B (en) | Nucleic acids against the coding strand | |
| GB201503408D0 (en) | Oligonucleotides | |
| IL257831A (en) | Diplospore garden | |
| GB201504124D0 (en) | Oligonucleotides | |
| SG10201913477UA (en) | G-quadruplex-containing antisense oligonucleotides | |
| SG10201609048RA (en) | Antisense oligonucleotides | |
| EP3315507A4 (en) | An oligonucleotide | |
| SG11201700652PA (en) | Modified antimir-138 oligonucleotides | |
| GB201516505D0 (en) | Antisense oligonucleotides | |
| GB201508733D0 (en) | Antisense oligonucleotides | |
| EP3323892A4 (en) | Antisense oligonucleotide inhibition of 2GPI expression | |
| GB201507125D0 (en) | Gene delivery | |
| GB201403353D0 (en) | Novel oligonucleotides | |
| GB201403087D0 (en) | Novel oligonucleotides |