CA2884608C - MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS - Google Patents
MULTIMERIC OLIGONUCLEOTIDE COMPOUNDSInfo
- Publication number
- CA2884608C CA2884608C CA2884608A CA2884608A CA2884608C CA 2884608 C CA2884608 C CA 2884608C CA 2884608 A CA2884608 A CA 2884608A CA 2884608 A CA2884608 A CA 2884608A CA 2884608 C CA2884608 C CA 2884608C
- Authority
- CA
- Canada
- Prior art keywords
- compound
- dts
- linker
- das
- oligonucleotide
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Classifications
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- 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]
- C12N2310/141—MicroRNAs, miRNAs
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- 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
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- 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
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- 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
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- 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
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- 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
- C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
-
- 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
- C12N2320/30—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
- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261701351P | 2012-09-14 | 2012-09-14 | |
| US61/701,351 | 2012-09-14 | ||
| US201361783272P | 2013-03-14 | 2013-03-14 | |
| US61/783,272 | 2013-03-14 | ||
| PCT/US2013/059772 WO2014043544A1 (en) | 2012-09-14 | 2013-09-13 | Multimeric oligonucleotide compounds |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2884608A1 CA2884608A1 (en) | 2014-03-20 |
| CA2884608C true CA2884608C (en) | 2025-05-20 |
Family
ID=50278726
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2884608A Active CA2884608C (en) | 2012-09-14 | 2013-09-13 | MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS |
Country Status (8)
| Country | Link |
|---|---|
| US (4) | US20150247141A1 (enExample) |
| EP (1) | EP2895200B1 (enExample) |
| JP (1) | JP2015529469A (enExample) |
| AU (3) | AU2013315225B2 (enExample) |
| CA (1) | CA2884608C (enExample) |
| DK (1) | DK2895200T3 (enExample) |
| IL (1) | IL237634A0 (enExample) |
| WO (1) | WO2014043544A1 (enExample) |
Families Citing this family (79)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101141544B1 (ko) | 2009-03-13 | 2012-05-03 | 한국과학기술원 | 에스아이알엔에이 다중 접합체 및 이의 제조방법 |
| JP6129844B2 (ja) | 2011-09-14 | 2017-05-17 | ラナ セラピューティクス インコーポレイテッド | 多量体オリゴヌクレオチド化合物 |
| SG11201407486PA (en) | 2012-05-16 | 2014-12-30 | Rana Therapeutics Inc | Compositions and methods for modulating utrn expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| EA201492121A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии семейства генов гемоглобина |
| JP2015523853A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Ａｔｐ２ａ２発現を調節するための組成物及び方法 |
| KR20150030205A (ko) | 2012-05-16 | 2015-03-19 | 라나 테라퓨틱스, 인크. | Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법 |
| AU2013262709A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating MECP2 expression |
| CA2884608C (en) | 2012-09-14 | 2025-05-20 | Translate Bio Ma, Inc. | MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS |
| EP3033422A4 (en) | 2013-08-16 | 2017-08-02 | Rana Therapeutics Inc. | Oligonucleotides targeting euchromatin regions of genes |
| US20150050738A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Compositions and methods for modulating rna |
| JP2017505623A (ja) | 2014-01-30 | 2017-02-23 | エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ | 生物切断性コンジュゲートを有するポリオリゴマー化合物 |
| US9487783B2 (en) | 2014-08-07 | 2016-11-08 | Regulus Therapeutics Inc. | Targeting microRNAs for metabolic disorders |
| WO2016023974A1 (de) * | 2014-08-14 | 2016-02-18 | Friedrich-Schiller-Universität Jena | Peptid zur verwendung in der reduktion von nebenwirkungen in form von immunstimulatorischen reaktionen/effekten |
| GB201421379D0 (en) * | 2014-12-02 | 2015-01-14 | Isis Innovation Ltd And Medical Res Council | Molecule |
| WO2016130943A1 (en) * | 2015-02-13 | 2016-08-18 | Rana Therapeutics, Inc. | Hybrid oligonucleotides and uses thereof |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| JP7009356B2 (ja) | 2015-04-03 | 2022-01-25 | ユニバーシティ・オブ・マサチューセッツ | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| IL316159A (en) * | 2015-06-15 | 2024-12-01 | Mpeg La Llc | Defined Oligonucleotide Multimers and Methods for Manufacture Thereof |
| US20170009230A1 (en) * | 2015-07-08 | 2017-01-12 | Idera Pharmaceuticals, Inc. | Compositions for inhibiting dux4 gene expression and uses thereof |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| CN116064544A (zh) * | 2016-01-26 | 2023-05-05 | 日产化学株式会社 | 单链寡核苷酸 |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| US11753638B2 (en) | 2016-08-12 | 2023-09-12 | University Of Massachusetts | Conjugated oligonucleotides |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| EP3576752A4 (en) | 2017-02-06 | 2020-12-16 | Mpeg La, LLC | MULTIMERIC OLIGONUCLEOTIDES WITH REDUCED RENAL CLEARANCE |
| US11572558B2 (en) | 2017-02-06 | 2023-02-07 | Nissan Chemical Corporation | Single-stranded oligonucleotide |
| US20200131231A1 (en) | 2017-02-17 | 2020-04-30 | Oxford University Innovation Limited | Cell penetrating peptides |
| JPWO2018182008A1 (ja) * | 2017-03-31 | 2020-02-06 | 株式会社ボナック | 遺伝子発現制御機能を有する環状型核酸分子 |
| JP7246729B2 (ja) * | 2017-05-18 | 2023-03-28 | 国立大学法人京都大学 | 脊髄小脳変性症３６型の予防又は治療用組成物 |
| CA3064590A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | Two-tailed self-delivering sirna and related methods |
| TWI902655B (zh) * | 2017-07-26 | 2025-11-01 | 日商日產化學股份有限公司 | 單股寡核苷酸 |
| JP6960641B2 (ja) * | 2017-11-09 | 2021-11-05 | 国立大学法人 東京大学 | ｍＲＮＡの安定化方法 |
| US20210139985A1 (en) * | 2018-04-10 | 2021-05-13 | Board Of Regents, The University Of Texas System | Dna-barcoded antigen multimers and methods of use thereof |
| KR102193874B1 (ko) * | 2018-05-31 | 2020-12-23 | 고려대학교 산학협력단 | 마이크로rna를 억제하는 변형 핵산 및 이의 용도 |
| US11168141B2 (en) * | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| JP7611825B2 (ja) | 2018-08-02 | 2025-01-10 | ダイン セラピューティクス，インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体およびそれらの使用 |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| GB201812972D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| GB201812980D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| WO2020033899A1 (en) | 2018-08-10 | 2020-02-13 | University Of Massachusetts | Modified oligonucleotides targeting snps |
| JP7627042B2 (ja) | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | Ｏ－メチルリッチ完全安定化オリゴヌクレオチド |
| IL326747A (en) | 2018-11-02 | 2026-04-01 | Biomarin Tech Bv | Bispecific antisense oligonucleotides for dystrophin exon skipping |
| US12465646B2 (en) | 2018-12-07 | 2025-11-11 | Oxford University Innovation Limited | Linkers |
| WO2020150636A1 (en) | 2019-01-18 | 2020-07-23 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| CA3133314A1 (en) | 2019-03-21 | 2020-09-24 | Codiak Biosciences, Inc. | Extracellular vesicle conjugates and uses thereof |
| US12502427B2 (en) | 2019-03-21 | 2025-12-23 | Lonza Sales Ag | Extracellular vesicles for vaccine delivery |
| US20220323519A1 (en) | 2019-04-17 | 2022-10-13 | Codiak Biosciences, Inc. | Compositions of exosomes and aav |
| US20220251200A1 (en) | 2019-07-03 | 2022-08-11 | Codiak Biosciences, Inc. | Extracellular vesicles targeting t cells and uses thereof |
| US20230114023A1 (en) * | 2019-07-30 | 2023-04-13 | Mpeg La, L.L.C. | Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity |
| CA3149835A1 (en) | 2019-08-09 | 2021-02-18 | University Of Massachusetts | Chemically modified oligonucleotides targeting snps |
| KR20220078565A (ko) | 2019-08-14 | 2022-06-10 | 코디악 바이오사이언시즈, 인크. | 분자에 연결된 세포외 소포 및 이의 용도 |
| CN114641570A (zh) | 2019-08-14 | 2022-06-17 | 科迪亚克生物科学公司 | 具有靶向kras的反义寡核苷酸的细胞外囊泡 |
| BR112022002690A2 (pt) | 2019-08-14 | 2022-08-23 | Codiak Biosciences Inc | Construtos de vesícula-aso extracelular tendo como alvo cebp/beta |
| WO2021030773A1 (en) | 2019-08-14 | 2021-02-18 | Codiak Biosciences, Inc. | Extracellular vesicle-nlrp3 antagonist |
| JP2022544289A (ja) | 2019-08-14 | 2022-10-17 | コディアック バイオサイエンシーズ， インコーポレイテッド | Ｓｔａｔ６を標的とする細胞外小胞－ａｓｏ構築物 |
| WO2021030769A1 (en) | 2019-08-14 | 2021-02-18 | Codiak Biosciences, Inc. | Extracellular vesicles with nras antisense oligonucleotides |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| KR102270700B1 (ko) * | 2019-11-01 | 2021-06-30 | 한국과학기술연구원 | 폐 섬유증의 예방 또는 치료를 위한 올리고뉴클레오티드 중합체와 생체 적합성 양이온성 펩타이드로 이루어지는 폐 특이적 약물 전달체 및 이의 이용 |
| JP7826221B2 (ja) * | 2020-04-30 | 2026-03-09 | エムペグ エルエイ リミテッド ライアビリティ カンパニー | 分割鎖を有する多量体オリゴヌクレオチド |
| EP4157289A4 (en) | 2020-05-26 | 2024-06-26 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| US20240082389A1 (en) | 2020-09-23 | 2024-03-14 | Lonza Sales Ag | Methods of producing extracellular vesicles |
| WO2022066883A1 (en) | 2020-09-23 | 2022-03-31 | Codiak Biosciences, Inc. | Extracellular vesicles comprising kras antigens and uses thereof |
| WO2022076596A1 (en) | 2020-10-06 | 2022-04-14 | Codiak Biosciences, Inc. | Extracellular vesicle-aso constructs targeting stat6 |
| KR20230118715A (ko) | 2020-10-14 | 2023-08-11 | 조지 메이슨 리서치 파운데이션 인코포레이티드 | 이온화 가능 지질 및 이의 제조 및 사용 방법 |
| JP2023548597A (ja) * | 2020-10-29 | 2023-11-17 | エー． ガルシア－ブランコ，マリアーノ | がんを処置するための可溶性インターロイキン－７受容体（ｓＩＬ７Ｒ）変調療法 |
| EP4294456A1 (en) | 2021-02-17 | 2023-12-27 | Lonza Sales AG | Extracellular vesicle linked to a biologically active molecule via an optimized linker and an anchoring moiety |
| AR126207A1 (es) | 2021-06-23 | 2023-09-27 | Univ Massachusetts | Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogénicos |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CN117980006A (zh) | 2021-07-09 | 2024-05-03 | 达因疗法公司 | 用于治疗肌养蛋白病的肌肉靶向复合物和制剂 |
| JP2025501682A (ja) * | 2021-12-03 | 2025-01-23 | クラリス コーポレーション | 改変された骨格化学を有するギャップマーアンチセンスオリゴヌクレオチド |
| CA3255691A1 (en) * | 2022-03-17 | 2023-09-21 | Aligos Therapeutics, Inc. | MODIFIED GAPMERE OLIGOMERS AND THEIR METHODS OF USE |
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| AU2013315225B2 (en) | 2018-11-08 |
| US10844375B2 (en) | 2020-11-24 |
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| EP2895200A1 (en) | 2015-07-22 |
| AU2019200789A1 (en) | 2019-02-21 |
| IL237634A0 (en) | 2015-04-30 |
| AU2021203173A1 (en) | 2021-06-10 |
| US20150247141A1 (en) | 2015-09-03 |
| WO2014043544A1 (en) | 2014-03-20 |
| US20150315586A1 (en) | 2015-11-05 |
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