PT3277814T - Compostos de oligonucleótidos para visar marn de huntingtina - Google Patents
Compostos de oligonucleótidos para visar marn de huntingtinaInfo
- Publication number
- PT3277814T PT3277814T PT167164128T PT16716412T PT3277814T PT 3277814 T PT3277814 T PT 3277814T PT 167164128 T PT167164128 T PT 167164128T PT 16716412 T PT16716412 T PT 16716412T PT 3277814 T PT3277814 T PT 3277814T
- Authority
- PT
- Portugal
- 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)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Neurology (AREA)
- Psychology (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (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)
- Saccharide Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (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 |
|---|---|
| PT3277814T true PT3277814T (pt) | 2020-07-28 |
Family
ID=55752785
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT167164128T PT3277814T (pt) | 2015-04-03 | 2016-04-01 | Compostos de oligonucleótidos para visar marn de huntingtina |
Country Status (18)
| Country | Link |
|---|---|
| US (5) | US9809817B2 (OSRAM) |
| EP (2) | EP3766973A1 (OSRAM) |
| JP (5) | JP7009356B2 (OSRAM) |
| CN (2) | CN107980062B (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 |
|---|---|---|---|---|
| CN104975020B (zh) | 2008-02-11 | 2020-01-17 | 菲奥医药公司 | 经修饰的RNAi多核苷酸及其用途 |
| US8796443B2 (en) | 2008-09-22 | 2014-08-05 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering RNAi compounds |
| WO2010059226A2 (en) | 2008-11-19 | 2010-05-27 | Rxi Pharmaceuticals Corporation | Inhibition of map4k4 through rnai |
| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| WO2010078536A1 (en) | 2009-01-05 | 2010-07-08 | Rxi Pharmaceuticals Corporation | Inhibition of pcsk9 through rnai |
| WO2010090762A1 (en) | 2009-02-04 | 2010-08-12 | Rxi Pharmaceuticals Corporation | Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| CA2794187C (en) | 2010-03-24 | 2020-07-14 | Rxi Pharmaceuticals Corporation | Rna interference in ocular indications |
| KR20180044433A (ko) | 2010-03-24 | 2018-05-02 | 알엑스아이 파마슈티칼스 코포레이션 | 진피 및 섬유증성 적응증에서의 ｒｎａ 간섭 |
| US9080171B2 (en) | 2010-03-24 | 2015-07-14 | RXi Parmaceuticals Corporation | Reduced size self-delivering RNAi compounds |
| 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 |
| CN112007045A (zh) | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
| BR112015000723A2 (pt) | 2012-07-13 | 2017-06-27 | Shin Nippon Biomedical Laboratories Ltd | adjuvante de ácido nucléico quiral |
| ES2862073T3 (es) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Grupo auxiliar asimétrico |
| WO2015084897A2 (en) | 2013-12-02 | 2015-06-11 | Mirimmune, Llc | Immunotherapy of cancer |
| 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 |
| 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 |
| 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 |
| US11279934B2 (en) | 2014-04-28 | 2022-03-22 | Phio Pharmaceuticals Corp. | Methods for treating cancer using nucleic acids targeting MDM2 or MYCN |
| US10900039B2 (en) | 2014-09-05 | 2021-01-26 | Phio Pharmaceuticals Corp. | Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| WO2016077689A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| 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 |
| PL3277814T3 (pl) | 2015-04-03 | 2020-11-30 | University Of Massachusetts | Związki oligonukleotydowe ukierunkowane na mrna huntingtyny |
| WO2016161378A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders |
| 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 |
| KR20180026739A (ko) | 2015-07-06 | 2018-03-13 | 알엑스아이 파마슈티칼스 코포레이션 | 슈퍼옥시드 디스뮤타제 1 (sod1)을 표적화하는 핵산 분자 |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| WO2017030973A1 (en) * | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| EP3365446A4 (en) | 2015-10-19 | 2019-06-26 | Phio Pharmaceuticals Corp. | SMALL SELF-LEANING NUCLEIC ACID COMPOUNDS TURNED AGAINST LONG NON-CODING RNA |
| WO2017132669A1 (en) | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| 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 |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| BR112018073472A2 (pt) * | 2016-05-18 | 2019-08-27 | Voyager Therapeutics Inc | composições e métodos de tratamento da doença de huntington |
| US11299733B2 (en) | 2016-07-15 | 2022-04-12 | University Of Massachusetts | Process of delivering small RNAs to sperm |
| US11753638B2 (en) | 2016-08-12 | 2023-09-12 | University Of Massachusetts | Conjugated oligonucleotides |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| ES2912270T3 (es) * | 2016-11-23 | 2022-05-25 | Mayo Found Medical Education & Res | Transporte de productos biológicos por medio de partículas |
| KR20190090836A (ko) * | 2016-11-28 | 2019-08-02 | 나파젠 파마, 인크. | 화학 수식 siRNA |
| IL321221A (en) | 2017-04-05 | 2025-07-01 | Silence Therapeutics Gmbh | Products and compositions |
| SG11201909868YA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| WO2018204786A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JP7557941B2 (ja) | 2017-06-02 | 2024-09-30 | 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及びその使用方法 |
| WO2018223056A1 (en) * | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| JP7406793B2 (ja) * | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法 |
| AU2018346507B2 (en) | 2017-10-04 | 2025-03-13 | Avidity Biosciences, Inc. | Nucleic acid-polypeptide compositions and uses thereof |
| WO2019079240A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | 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 |
| AU2019225992A1 (en) * | 2018-02-21 | 2020-08-20 | Alcyone Therapeutics, Inc. | Fluid delivery systems and methods |
| WO2019181946A1 (ja) * | 2018-03-19 | 2019-09-26 | 国立大学法人東京医科歯科大学 | 毒性が軽減した核酸 |
| US11981892B2 (en) | 2018-04-16 | 2024-05-14 | University Of Massachusetts | Compositions and methods for improved gene editing |
| AU2019265904A1 (en) | 2018-05-11 | 2020-11-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| AU2019316640A1 (en) * | 2018-08-10 | 2021-03-18 | University Of Massachusetts | Modified oligonucleotides targeting SNPs |
| US11279930B2 (en) | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| CA3125441A1 (en) | 2019-01-18 | 2020-07-23 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| CN113924364A (zh) * | 2019-03-08 | 2022-01-11 | 罗格斯新泽西州立大学 | 治疗亨廷顿病的组合物和方法 |
| JP2022528840A (ja) * | 2019-03-26 | 2022-06-16 | ユニバーシティ・オブ・マサチューセッツ | 安定性が増加した修飾オリゴヌクレオチド |
| WO2020227395A2 (en) * | 2019-05-06 | 2020-11-12 | University Of Massachusetts | Anti-c9orf72 oligonucleotides and related methods |
| US11578090B2 (en) | 2019-06-06 | 2023-02-14 | Avidity Biosciences, Inc. | Nucleic acid-polypeptide compositions and uses thereof |
| CA3142337A1 (en) | 2019-06-06 | 2020-12-10 | Avidity Biosciences, Inc. | Una amidites 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 |
| CA3149835A1 (en) | 2019-08-09 | 2021-02-18 | University Of Massachusetts | Chemically modified oligonucleotides targeting snps |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| CA3155921A1 (en) * | 2019-11-01 | 2021-05-06 | Alnylam Pharmaceuticals, Inc. | HUNTINGTINE (HTT) TARGETING RNAI AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| 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 |
| CA3171757A1 (en) * | 2020-03-18 | 2021-09-23 | Anastasia Khvorova | Oligonucleotides for snca modulation |
| AU2021244600A1 (en) | 2020-03-26 | 2022-11-03 | University Of Massachusetts | Synthesis of modified oligonucleotides with increased stability |
| AU2021260581A1 (en) * | 2020-04-20 | 2022-11-24 | University Of Massachusetts | Oligonucleotides for MSH3 modulation |
| WO2022031591A2 (en) * | 2020-08-03 | 2022-02-10 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
| AU2022246147A1 (en) * | 2021-03-24 | 2023-11-09 | Atalanta Therapeutics, Inc. | Microglial gene silencing using double-stranded sirna |
| TW202305134A (zh) | 2021-03-29 | 2023-02-01 | 美商艾拉倫製藥股份有限公司 | 杭丁頓蛋白（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法 |
| CA3174095A1 (en) | 2021-06-23 | 2022-12-29 | Vignesh Narayan HARIHARAN | Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders |
| US20230340475A1 (en) * | 2021-12-17 | 2023-10-26 | University Of Massachusetts | Oligonucleotides for mlh1 modulation |
| AU2023354001A1 (en) * | 2022-09-28 | 2025-04-17 | Atalanta Therapeutics, Inc. | Compositions and methods for treatment of huntington's disease |
| TW202517782A (zh) * | 2023-06-30 | 2025-05-01 | 美商星火治療公司 | 靶向亨丁頓蛋白(huntingtin)表現之抑制性rna |
| 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 |
| JPH05503841A (ja) | 1989-11-16 | 1993-06-24 | デューク ユニバーシティ | 動物組織細胞の微片仲介トランスフォーメーション |
| 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 |
| US6291438B1 (en) | 1993-02-24 | 2001-09-18 | Jui H. Wang | Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use |
| 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 |
| 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 |
| 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 |
| IL161100A0 (en) | 2001-09-28 | 2004-08-31 | Max Planck Gesellschaft | Identification of novel genes coding for small temporal rnas |
| 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) |
| AU2003223775A1 (en) | 2002-04-30 | 2003-11-17 | Duke University | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
| PT2308507E (pt) | 2002-07-19 | 2015-05-11 | Beth Israel Hospital | Métodos de tratamento de pré-eclâmpsia |
| EP2957568B1 (en) | 2002-11-05 | 2016-12-21 | Ionis Pharmaceuticals, Inc. | Compositions comprising alternating 2'-modified nucleosides for use in gene modulation |
| EP2213738B1 (en) * | 2002-11-14 | 2012-10-10 | Dharmacon, Inc. | siRNA molecules targeting Bcl-2 |
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| AU2004248136B2 (en) | 2003-06-02 | 2011-09-15 | University Of Massachusetts | Methods and compositions for controlling efficacy of RNA silencing |
| ATE485394T1 (de) | 2003-06-02 | 2010-11-15 | Univ Massachusetts | Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai |
| CN1906308B (zh) | 2003-12-19 | 2011-09-14 | 诺华疫苗和诊断公司 | 小干扰rna的细胞转染制剂、相关组合物以及制备和使用方法 |
| WO2005077333A2 (en) | 2004-02-10 | 2005-08-25 | University Of Florida Research Foundation, Inc. | Gel-based delivery of recombinant adeno-associated virus vectors |
| AU2005222902B2 (en) | 2004-03-12 | 2010-06-10 | Alnylam Pharmaceuticals, Inc. | iRNA agents targeting VEGF |
| EP1773998A2 (en) | 2004-04-20 | 2007-04-18 | Nastech Pharmaceutical Company Inc. | Methods and compositions for enhancing delivery of double-stranded rna or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells |
| CA2566286A1 (en) * | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same |
| CA2569419A1 (en) | 2004-06-03 | 2005-12-22 | Isis Pharmaceuticals, Inc. | Double strand compositions comprising differentially modified strands for use in gene modulation |
| CN101124328A (zh) | 2004-12-15 | 2008-02-13 | 北卡罗来纳查佩尔山大学 | 嵌合载体 |
| CA2619534A1 (en) * | 2005-08-18 | 2007-02-22 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating neurological disease |
| EP2325315B1 (en) * | 2005-10-28 | 2014-05-07 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of huntingtin gene |
| 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 |
| JPWO2007094218A1 (ja) | 2006-02-16 | 2009-07-02 | 国立大学法人岐阜大学 | 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 |
| EP2046993A4 (en) | 2006-07-07 | 2010-11-17 | Univ Massachusetts | RNA SILENCING COMPOSITIONS, AND METHODS OF TREATING HUNTINGTON CHOREA |
| 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 |
| WO2009099991A2 (en) * | 2008-01-31 | 2009-08-13 | 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 |
| US8796443B2 (en) * | 2008-09-22 | 2014-08-05 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering RNAi compounds |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| EP2470656B1 (en) | 2009-08-27 | 2015-05-06 | Idera Pharmaceuticals, Inc. | Composition for inhibiting gene expression and uses thereof |
| US9080171B2 (en) | 2010-03-24 | 2015-07-14 | RXi Parmaceuticals Corporation | Reduced size self-delivering RNAi compounds |
| WO2012005898A2 (en) * | 2010-06-15 | 2012-01-12 | Alnylam Pharmaceuticals, Inc. | Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof |
| WO2012118911A1 (en) | 2011-03-03 | 2012-09-07 | Quark Pharmaceuticals, Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| WO2012177906A1 (en) * | 2011-06-21 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Assays and methods for determining activity of a therapeutic agent in a subject |
| EP3453762B1 (en) | 2012-05-02 | 2021-04-21 | Sirna Therapeutics, Inc. | Short interfering nucleic acid (sina) compositions |
| EP3514236A1 (en) | 2012-05-22 | 2019-07-24 | Olix Pharmaceuticals, Inc. | Rna-interference-inducing nucleic acid molecule able to penetrate into cells, and use therefor |
| CN104540525A (zh) | 2012-07-10 | 2015-04-22 | 贝瑟克里科有限公司 | 大麻素修饰的核酸分子 |
| WO2014043544A1 (en) | 2012-09-14 | 2014-03-20 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| CA2889044A1 (en) | 2012-11-15 | 2014-05-22 | Roche Innovation Center Copenhagen A/S | Anti apob antisense conjugate compounds |
| US10513710B2 (en) | 2014-04-18 | 2019-12-24 | University Of Massachusetts | Exosomal loading using hydrophobically modified oligonucleotides |
| PL3277814T3 (pl) | 2015-04-03 | 2020-11-30 | University Of Massachusetts | Związki oligonukleotydowe ukierunkowane na mrna huntingtyny |
| 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 |
| WO2016161378A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| WO2017132669A1 (en) | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
-
2016
- 2016-04-01 PL PL16716412T patent/PL3277814T3/pl unknown
- 2016-04-01 JP JP2018502624A patent/JP7009356B2/ja active Active
- 2016-04-01 CN CN201680031524.4A patent/CN107980062B/zh active Active
- 2016-04-01 CA CA2980337A patent/CA2980337A1/en active Pending
- 2016-04-01 AU AU2016244027A patent/AU2016244027B2/en active Active
- 2016-04-01 CN CN202211430603.6A patent/CN116004624A/zh active Pending
- 2016-04-01 RS RS20200929A patent/RS60762B1/sr unknown
- 2016-04-01 LT LTEP16716412.8T patent/LT3277814T/lt unknown
- 2016-04-01 HU HUE16716412A patent/HUE050441T2/hu unknown
- 2016-04-01 EP EP20164108.1A patent/EP3766973A1/en active Pending
- 2016-04-01 EP EP16716412.8A patent/EP3277814B1/en active Active
- 2016-04-01 HR HRP20201200TT patent/HRP20201200T1/hr unknown
- 2016-04-01 WO PCT/US2016/025722 patent/WO2016161374A1/en not_active Ceased
- 2016-04-01 US US15/089,319 patent/US9809817B2/en active Active
- 2016-04-01 SM SM20200454T patent/SMT202000454T1/it unknown
- 2016-04-01 SI SI201630878T patent/SI3277814T1/sl unknown
- 2016-04-01 PT PT167164128T patent/PT3277814T/pt unknown
- 2016-04-01 ES ES16716412T patent/ES2808750T3/es active Active
- 2016-04-01 DK DK16716412.8T patent/DK3277814T3/da active
-
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 AU2022204719A patent/AU2022204719A1/en not_active Abandoned
- 2022-06-30 AU AU2022204722A patent/AU2022204722A1/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 |
|---|---|---|
| PL3277814T3 (pl) | Związki oligonukleotydowe ukierunkowane na mrna huntingtyny | |
| 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 | |
| IL257831B2 (en) | Diplosporic 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 |