CY1117367T1 - Αντινοηματικο νουκλεϊνικο οξυ - Google Patents
Αντινοηματικο νουκλεϊνικο οξυInfo
- Publication number
- CY1117367T1 CY1117367T1 CY20161100315T CY161100315T CY1117367T1 CY 1117367 T1 CY1117367 T1 CY 1117367T1 CY 20161100315 T CY20161100315 T CY 20161100315T CY 161100315 T CY161100315 T CY 161100315T CY 1117367 T1 CY1117367 T1 CY 1117367T1
- Authority
- CY
- Cyprus
- Prior art keywords
- antinematic
- nuclear acid
- exon
- provides
- present
- Prior art date
Links
- 239000002253 acid Substances 0.000 title 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
-
- 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
- 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
-
- 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
-
- 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
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2010196032 | 2010-09-01 | ||
| EP11821996.3A EP2612917B1 (en) | 2010-09-01 | 2011-08-31 | Antisense nucleic acid |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CY1117367T1 true CY1117367T1 (el) | 2017-04-26 |
Family
ID=45773054
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CY20161100315T CY1117367T1 (el) | 2010-09-01 | 2016-04-15 | Αντινοηματικο νουκλεϊνικο οξυ |
| CY20191101075T CY1122167T1 (el) | 2010-09-01 | 2019-10-14 | Αντινοηματικα νουκλεϊνικα οξεα |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CY20191101075T CY1122167T1 (el) | 2010-09-01 | 2019-10-14 | Αντινοηματικα νουκλεϊνικα οξεα |
Country Status (20)
| Country | Link |
|---|---|
| US (16) | US9079934B2 (enExample) |
| EP (6) | EP3018211B1 (enExample) |
| JP (12) | JP5363655B2 (enExample) |
| KR (1) | KR101310569B1 (enExample) |
| CN (1) | CN103154245B (enExample) |
| AU (1) | AU2011296882B2 (enExample) |
| CA (1) | CA2809637C (enExample) |
| CY (2) | CY1117367T1 (enExample) |
| DK (2) | DK3018211T3 (enExample) |
| ES (2) | ES2567411T3 (enExample) |
| HR (2) | HRP20160336T1 (enExample) |
| HU (2) | HUE046364T2 (enExample) |
| LT (1) | LT3018211T (enExample) |
| PL (2) | PL3018211T3 (enExample) |
| PT (1) | PT3018211T (enExample) |
| RS (2) | RS59361B1 (enExample) |
| SI (2) | SI3018211T1 (enExample) |
| SM (2) | SMT201900559T1 (enExample) |
| TW (1) | TWI541024B (enExample) |
| WO (1) | WO2012029986A1 (enExample) |
Families Citing this family (73)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| HRP20110352T1 (hr) * | 2004-06-28 | 2011-06-30 | The University Of Western Australia | Antisens olinukleotidi za induciranje zaobilaženja egzona i postupci njihove upotrebe |
| CA2596506C (en) | 2005-02-09 | 2021-04-06 | Avi Biopharma, Inc. | Antisense composition and method for treating muscle atrophy |
| CA2704049A1 (en) | 2007-10-26 | 2009-04-30 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| WO2010048586A1 (en) | 2008-10-24 | 2010-04-29 | Avi Biopharma, Inc. | Multiple exon skipping compositions for dmd |
| KR102113306B1 (ko) | 2009-11-12 | 2020-05-21 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| CN111440796A (zh) * | 2012-12-20 | 2020-07-24 | 萨雷普塔医疗公司 | 用于治疗肌营养不良症的改良的外显子跳跃组合物 |
| KR20230074606A (ko) | 2013-03-14 | 2023-05-30 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| NZ731587A (en) | 2013-03-14 | 2021-07-30 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| CN113633787A (zh) | 2013-03-15 | 2021-11-12 | 萨勒普塔医疗公司 | 改进的用于治疗肌营养不良的组合物 |
| HUE042283T2 (hu) * | 2014-03-12 | 2019-06-28 | Nippon Shinyaku Co Ltd | Antiszensz nukleinsav |
| US10174319B2 (en) | 2014-05-19 | 2019-01-08 | Knc Laboratories Co., Ltd. | Nucleic acid drug for inducing skipping of variant exon of CD44 gene and increasing expression of normal type CD44 mRNA |
| SI3159409T1 (sl) * | 2014-06-17 | 2020-02-28 | Nippon Shinyaku Co., Ltd. | Protismiselna nukleinska kislina za uporabo pri zdravljenju Duchennove mišične distrofije |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| EP3778895A1 (en) * | 2015-09-15 | 2021-02-17 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid |
| JP6875684B2 (ja) * | 2015-09-16 | 2021-05-26 | 日本新薬株式会社 | 筋萎縮症治療用アンチセンス核酸 |
| CN114533900A (zh) | 2015-10-09 | 2022-05-27 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| BR112018007066A2 (pt) | 2015-10-09 | 2018-10-23 | Sarepta Therapeutics Inc | composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados |
| FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| KR101686379B1 (ko) | 2016-02-05 | 2016-12-13 | 박정열 | 휴대용 구이기 |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| WO2017205496A1 (en) * | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| SG10202101836TA (en) | 2016-05-24 | 2021-03-30 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| WO2017205879A2 (en) * | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| LT3464306T (lt) | 2016-05-24 | 2024-05-10 | Sarepta Therapeutics, Inc. | Fosforodiamidato morfolino oligomerų gamybos būdas |
| TW201811807A (zh) | 2016-06-30 | 2018-04-01 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| JP2019522972A (ja) * | 2016-07-05 | 2019-08-22 | バイオマリン テクノロジーズ ベー．フェー． | 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド |
| KR101715572B1 (ko) | 2016-10-11 | 2017-03-22 | 박종찬 | 구이대 승강구조를 갖는 양방향 구이기 |
| HRP20221316T1 (hr) | 2016-12-19 | 2022-12-23 | Sarepta Therapeutics, Inc. | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
| PL3554553T3 (pl) | 2016-12-19 | 2022-11-07 | Sarepta Therapeutics, Inc. | Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej |
| ES2928486T3 (es) | 2016-12-19 | 2022-11-18 | Sarepta Therapeutics Inc | Conjugados de oligómeros con omisión de exón para la distrofia muscular |
| EP3630199A4 (en) | 2017-06-02 | 2021-11-10 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS FOR THEIR USE |
| TW201919655A (zh) * | 2017-08-31 | 2019-06-01 | 美商薩羅塔治療公司 | 治療肌肉萎縮症的方法 |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536057A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| JP2021521794A (ja) * | 2018-04-26 | 2021-08-30 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
| CA3098624A1 (en) * | 2018-05-11 | 2019-11-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| CA3101321A1 (en) * | 2018-06-26 | 2020-01-02 | Nippon Shinyaku Co., Ltd. | Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy |
| WO2020028864A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| CN113453723A (zh) | 2018-12-07 | 2021-09-28 | 牛津大学科技创新有限公司 | 接头 |
| JP7185239B2 (ja) * | 2018-12-25 | 2022-12-07 | 国立研究開発法人国立精神・神経医療研究センター | 随時尿中細胞を用いた筋系細胞の誘導方法 |
| GB201821269D0 (en) * | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| CN114901823A (zh) | 2019-12-26 | 2022-08-12 | 日本新药株式会社 | 诱导外显子50的跳读的反义核酸 |
| JPWO2021172498A1 (enExample) | 2020-02-28 | 2021-09-02 | ||
| JP6953053B1 (ja) | 2020-03-11 | 2021-10-27 | 隆光 矢野 | −１フレームシフトを誘導するための１本鎖核酸分子及び組成物 |
| WO2022059754A1 (ja) * | 2020-09-18 | 2022-03-24 | 天野エンザイム株式会社 | 加工ひよこ豆ミルクの製造方法 |
| CA3211038A1 (en) | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| EP4330395A1 (en) | 2021-04-30 | 2024-03-06 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| CA3226366A1 (en) | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| 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 |
| JPWO2023026994A1 (enExample) | 2021-08-21 | 2023-03-02 | ||
| IL311025A (en) | 2021-08-24 | 2024-04-01 | Peptidream Inc | Human transferrin receptor-binding antibody-peptide conjugate |
| CA3244382A1 (en) | 2021-12-27 | 2025-02-26 | Nippon Shinyaku Co., Ltd. | PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| JP2025079347A (ja) * | 2022-03-11 | 2025-05-22 | 日本新薬株式会社 | キャリアペプチドが連結された核酸 |
| AU2023235302A1 (en) | 2022-03-17 | 2024-10-24 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
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