NZ631132A - Exon skipping compositions for treating muscular dystrophy - Google Patents
Exon skipping compositions for treating muscular dystrophyInfo
- Publication number
- NZ631132A NZ631132A NZ631132A NZ63113214A NZ631132A NZ 631132 A NZ631132 A NZ 631132A NZ 631132 A NZ631132 A NZ 631132A NZ 63113214 A NZ63113214 A NZ 63113214A NZ 631132 A NZ631132 A NZ 631132A
- Authority
- NZ
- New Zealand
- Prior art keywords
- muscular dystrophy
- oligomer
- exon skipping
- morpholino
- treating muscular
- Prior art date
Links
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
-
- 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
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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
-
- 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
- C12N2310/3233—Morpholino-type ring
-
- 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
-
- 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
- C12N2310/3513—Protein; Peptide
-
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3535—Nitrogen
-
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (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)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361782706P | 2013-03-14 | 2013-03-14 | |
| PCT/US2014/029766 WO2014153240A2 (en) | 2013-03-14 | 2014-03-14 | Exon skipping compositions for treating muscular dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NZ631132A true NZ631132A (en) | 2017-06-30 |
Family
ID=50694008
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ631132A NZ631132A (en) | 2013-03-14 | 2014-03-14 | Exon skipping compositions for treating muscular dystrophy |
Country Status (26)
| Country | Link |
|---|---|
| US (5) | US20140315977A1 (enExample) |
| EP (3) | EP3760720A1 (enExample) |
| JP (4) | JP2016521119A (enExample) |
| KR (5) | KR20230074606A (enExample) |
| CN (2) | CN105392884B (enExample) |
| AU (3) | AU2014236160A1 (enExample) |
| BR (1) | BR112015023038A2 (enExample) |
| CA (1) | CA2903872A1 (enExample) |
| CY (1) | CY1121876T1 (enExample) |
| DK (1) | DK2970964T3 (enExample) |
| EA (1) | EA201591694A1 (enExample) |
| ES (1) | ES2714290T3 (enExample) |
| HR (1) | HRP20190382T1 (enExample) |
| HU (1) | HUE042218T2 (enExample) |
| IL (2) | IL241047B (enExample) |
| LT (1) | LT2970964T (enExample) |
| MX (2) | MX366274B (enExample) |
| NZ (1) | NZ631132A (enExample) |
| PL (1) | PL2970964T3 (enExample) |
| PT (1) | PT2970964T (enExample) |
| RS (1) | RS58529B1 (enExample) |
| SA (1) | SA515361121B1 (enExample) |
| SI (1) | SI2970964T1 (enExample) |
| SM (1) | SMT201900139T1 (enExample) |
| TR (1) | TR201903009T4 (enExample) |
| WO (1) | WO2014153240A2 (enExample) |
Families Citing this family (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3808845A1 (en) | 2004-06-28 | 2021-04-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| PT2203173E (pt) * | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| CA3066050A1 (en) | 2008-10-24 | 2010-04-29 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
| SI2499249T1 (sl) | 2009-11-12 | 2019-02-28 | The University Of Western Australia, | Protismiselne molekule in postopki zdravljenja patologij |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| IL280443B (en) | 2013-03-14 | 2022-07-01 | Sarepta Therapeutics Inc | Preparations that skip axon for the treatment of muscular dystrophy |
| MX373959B (es) | 2013-03-15 | 2020-07-13 | Sarepta Therapeutics Inc | Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd). |
| FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| JOP20200228A1 (ar) | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
| WO2017205879A2 (en) | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino 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 |
| MD3464305T2 (ro) * | 2016-05-24 | 2025-01-31 | Sarepta Therapeutics Inc | Procedee de preparare de oligomeri |
| MA45362A (fr) * | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| KR102504759B1 (ko) * | 2016-05-24 | 2023-02-28 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| CN109311919A (zh) | 2016-06-30 | 2019-02-05 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体 |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| CN109757108A (zh) * | 2016-07-05 | 2019-05-14 | 比奥马林技术公司 | 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸 |
| US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
| CN116804031A (zh) * | 2016-09-20 | 2023-09-26 | 科罗拉多州立大学董事会法人团体 | 使用亚磷酰胺化学法合成主链修饰的吗啉代寡核苷酸和嵌合体 |
| DK3554552T3 (da) * | 2016-12-19 | 2022-10-24 | Sarepta Therapeutics Inc | Exon-skipping-oligomerkonjugater mod muskeldystrofi |
| MA65895B1 (fr) | 2016-12-19 | 2024-06-28 | Sarepta Therapeutics, Inc. | Conjugués d'oligomères induisant le saut d'exons pour la dystrophie musculaire |
| MD3554553T2 (ro) | 2016-12-19 | 2022-10-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
| EP3675836A4 (en) * | 2017-08-31 | 2021-05-26 | Sarepta Therapeutics, Inc. | METHODS FOR TREATING MUSCLE DYSTROPHY |
| EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| MA49986A (fr) * | 2017-09-25 | 2020-07-01 | Massachusetts Inst Technology | Procédés de préparation d'oligomères morpholino phosphorodiamidate par l'intermédiaire d'une synthèse à flux rapide |
| EP3687519A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| US12370261B2 (en) | 2018-03-16 | 2025-07-29 | Sarepta Therapeutics, Inc. | Chimeric peptides for antisense delivery |
| 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 |
| US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| US20210220386A1 (en) * | 2018-06-14 | 2021-07-22 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| PE20210630A1 (es) * | 2018-06-26 | 2021-03-23 | Nippon Shinyaku Co Ltd | Composicion que comprende el oligonucleotido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne |
| CN112955153A (zh) | 2018-08-02 | 2021-06-11 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗肌养蛋白病的用途 |
| 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CN112912499A (zh) | 2018-08-02 | 2021-06-04 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 |
| 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 |
| CN113677374A (zh) * | 2019-04-08 | 2021-11-19 | 国立大学法人东京医科齿科大学 | 肌疾病治疗用药物组合物 |
| WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| WO2022006871A1 (en) * | 2020-07-10 | 2022-01-13 | Changzhou Syntheall Pharmaceuticals Co., Ltd. | Processes for preparing oligonucleotides |
| WO2022171972A1 (en) * | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| MX2023011612A (es) | 2021-03-31 | 2023-12-15 | Entrada Therapeutics Inc | Peptidos de penetracion celular ciclicos. |
| WO2022217366A1 (en) * | 2021-04-14 | 2022-10-20 | The Royal Institution For The Advancement Of Learning/Mcgill University | Modified oligonucleotides for the treatment of duchenne muscular dystrophy |
| US20240358845A1 (en) | 2021-05-10 | 2024-10-31 | Entrada Therapeutics, Inc. | Compositions and methods for intracellular therapeutics |
| IL309936A (en) | 2021-07-09 | 2024-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of dystrophinopathy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| EP4458833A1 (en) | 2021-12-27 | 2024-11-06 | Nippon Shinyaku Co., Ltd. | Method for producing oligonucleic acid compound |
| IL315280A (en) * | 2022-03-17 | 2024-10-01 | Sarepta Therapeutics Inc | Phosphorodiamidate morpholino oligomer conjugates |
| WO2024073588A1 (en) * | 2022-09-28 | 2024-04-04 | Board Of Regents, The University Of Texas System | Respiratory dry powder delivery |
Family Cites Families (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CH445129A (fr) | 1964-04-29 | 1967-10-15 | Nestle Sa | Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé |
| US3459731A (en) | 1966-12-16 | 1969-08-05 | Corn Products Co | Cyclodextrin polyethers and their production |
| US3453257A (en) | 1967-02-13 | 1969-07-01 | Corn Products Co | Cyclodextrin with cationic properties |
| US3426011A (en) | 1967-02-13 | 1969-02-04 | Corn Products Co | Cyclodextrins with anionic properties |
| US3453259A (en) | 1967-03-22 | 1969-07-01 | Corn Products Co | Cyclodextrin polyol ethers and their oxidation products |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| JP3022967B2 (ja) | 1985-03-15 | 2000-03-21 | アンチバイラルズ インコーポレイテッド | 立体規則性ポリヌクレオチド結合ポリマー |
| US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
| KR0166088B1 (ko) | 1990-01-23 | 1999-01-15 | . | 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도 |
| US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
| WO1994002595A1 (en) | 1992-07-17 | 1994-02-03 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for treatment of animal diseases |
| DE69435005T2 (de) | 1993-05-11 | 2008-04-17 | The University Of North Carolina At Chapel Hill | Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung |
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| US5820873A (en) | 1994-09-30 | 1998-10-13 | The University Of British Columbia | Polyethylene glycol modified ceramide lipids and liposome uses thereof |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
| IL115849A0 (en) | 1994-11-03 | 1996-01-31 | Merz & Co Gmbh & Co | Tangential filtration preparation of liposomal drugs and liposome product thereof |
| WO1999042091A2 (en) | 1998-02-19 | 1999-08-26 | Massachusetts Institute Of Technology | Use of polycations as endosomolytic agents |
| US6683173B2 (en) | 1998-04-03 | 2004-01-27 | Epoch Biosciences, Inc. | Tm leveling methods |
| US6210892B1 (en) | 1998-10-07 | 2001-04-03 | Isis Pharmaceuticals, Inc. | Alteration of cellular behavior by antisense modulation of mRNA processing |
| CA2394758A1 (en) | 1999-12-29 | 2001-07-05 | A. James Mixson | Histidine-containing copolymers enhance pharmaceutical agent delivery |
| US7070807B2 (en) | 1999-12-29 | 2006-07-04 | Mixson A James | Branched histidine copolymers and methods for using same |
| DE60231975D1 (de) | 2001-05-24 | 2009-05-28 | Genzyme Corp | Muskelspezifische expressionsvektoren |
| WO2004083432A1 (en) | 2003-03-21 | 2004-09-30 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| EP3808845A1 (en) * | 2004-06-28 | 2021-04-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| ES2852549T3 (es) * | 2005-02-09 | 2021-09-13 | Sarepta Therapeutics Inc | Composición antisentido para tratamiento de la atrofia muscular |
| US8067571B2 (en) * | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| CA2629323A1 (en) | 2005-11-10 | 2007-05-24 | The University Of North Carolina At Chapel Hill | Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease |
| PL2735568T3 (pl) | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| US20070265215A1 (en) * | 2006-05-11 | 2007-11-15 | Iversen Patrick L | Antisense restenosis composition and method |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| PT2203173E (pt) * | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
| WO2009127230A1 (en) | 2008-04-16 | 2009-10-22 | Curevac Gmbh | MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION |
| US8084601B2 (en) * | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| CA3066050A1 (en) * | 2008-10-24 | 2010-04-29 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
| SI2499249T1 (sl) * | 2009-11-12 | 2019-02-28 | The University Of Western Australia, | Protismiselne molekule in postopki zdravljenja patologij |
| KR102095478B1 (ko) | 2010-05-28 | 2020-04-01 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| KR102339196B1 (ko) | 2011-05-05 | 2021-12-15 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 펩타이드 올리고뉴클레오타이드 접합체 |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| WO2013033407A2 (en) * | 2011-08-30 | 2013-03-07 | The Regents Of The University Of California | Identification of small molecules that enhance therapeutic exon skipping |
| CA2862628C (en) * | 2012-01-27 | 2021-08-24 | Prosensa Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| CA2868174A1 (en) * | 2012-03-20 | 2013-09-26 | Sarepta Therapeutics, Inc. | Boronic acid conjugates of oligonucleotide analogues |
| EP3885439A1 (en) * | 2012-12-20 | 2021-09-29 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
-
2014
- 2014-03-14 EP EP20182238.4A patent/EP3760720A1/en not_active Withdrawn
- 2014-03-14 PL PL14723583T patent/PL2970964T3/pl unknown
- 2014-03-14 EP EP18215621.6A patent/EP3495485A3/en not_active Withdrawn
- 2014-03-14 SM SM20190139T patent/SMT201900139T1/it unknown
- 2014-03-14 KR KR1020237016315A patent/KR20230074606A/ko not_active Ceased
- 2014-03-14 JP JP2016503219A patent/JP2016521119A/ja not_active Withdrawn
- 2014-03-14 RS RS20190277A patent/RS58529B1/sr unknown
- 2014-03-14 ES ES14723583T patent/ES2714290T3/es active Active
- 2014-03-14 US US14/213,641 patent/US20140315977A1/en not_active Abandoned
- 2014-03-14 US US14/776,533 patent/US20160040162A1/en not_active Abandoned
- 2014-03-14 LT LTEP14723583.2T patent/LT2970964T/lt unknown
- 2014-03-14 EA EA201591694A patent/EA201591694A1/ru unknown
- 2014-03-14 KR KR1020247018517A patent/KR20240094032A/ko not_active Ceased
- 2014-03-14 CN CN201480027371.7A patent/CN105392884B/zh active Active
- 2014-03-14 HR HRP20190382TT patent/HRP20190382T1/hr unknown
- 2014-03-14 BR BR112015023038A patent/BR112015023038A2/pt not_active Application Discontinuation
- 2014-03-14 MX MX2015012149A patent/MX366274B/es active IP Right Grant
- 2014-03-14 SI SI201431107T patent/SI2970964T1/sl unknown
- 2014-03-14 KR KR1020217000648A patent/KR20210006516A/ko not_active Ceased
- 2014-03-14 NZ NZ631132A patent/NZ631132A/en unknown
- 2014-03-14 HU HUE14723583A patent/HUE042218T2/hu unknown
- 2014-03-14 TR TR2019/03009T patent/TR201903009T4/tr unknown
- 2014-03-14 DK DK14723583.2T patent/DK2970964T3/en active
- 2014-03-14 CA CA2903872A patent/CA2903872A1/en active Pending
- 2014-03-14 EP EP14723583.2A patent/EP2970964B8/en active Active
- 2014-03-14 WO PCT/US2014/029766 patent/WO2014153240A2/en not_active Ceased
- 2014-03-14 AU AU2014236160A patent/AU2014236160A1/en not_active Abandoned
- 2014-03-14 CN CN201811579820.5A patent/CN110066793A/zh active Pending
- 2014-03-14 PT PT14723583T patent/PT2970964T/pt unknown
- 2014-03-14 KR KR1020157028188A patent/KR20150133752A/ko not_active Ceased
- 2014-03-14 KR KR1020227014526A patent/KR20220061280A/ko not_active Ceased
-
2015
- 2015-09-02 IL IL241047A patent/IL241047B/en active IP Right Grant
- 2015-09-10 MX MX2019000224A patent/MX2019000224A/es unknown
- 2015-09-14 SA SA515361121A patent/SA515361121B1/ar unknown
-
2017
- 2017-01-27 US US15/417,401 patent/US20180002689A1/en not_active Abandoned
-
2019
- 2019-03-05 CY CY20191100267T patent/CY1121876T1/el unknown
- 2019-03-19 US US16/358,169 patent/US20200040336A1/en not_active Abandoned
- 2019-11-25 JP JP2019212294A patent/JP2020039362A/ja not_active Withdrawn
-
2020
- 2020-02-02 IL IL272415A patent/IL272415A/en unknown
- 2020-05-27 AU AU2020203473A patent/AU2020203473A1/en not_active Abandoned
-
2021
- 2021-12-17 JP JP2021204872A patent/JP2022031948A/ja not_active Withdrawn
-
2022
- 2022-06-03 US US17/805,402 patent/US20220364093A1/en not_active Abandoned
- 2022-10-13 AU AU2022252769A patent/AU2022252769A1/en not_active Abandoned
-
2024
- 2024-05-22 JP JP2024083541A patent/JP2024112929A/ja not_active Withdrawn
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| NZ631132A (en) | Exon skipping compositions for treating muscular dystrophy | |
| MX373959B (es) | Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd). | |
| CY1124197T1 (el) | Antinohmatika πολυνουκλεοτιδια για να διεγειρεται παραλειψη εξονιου και μεθοδοι θεραπειας δυστροφιων | |
| PH12015501953A1 (en) | Substituted nucleotide analogs | |
| NZ631512A (en) | Compositions and methods for modulating apolipoprotein (a) expression | |
| UA117098C2 (uk) | Сполука, що містить модифікований олігонуклеотид | |
| PH12014502094A1 (en) | Substituted nucleosides, nucleotides and analogs thereof | |
| MX2015007925A (es) | Nucleosidos sustituidos, nucleotidos y analogos de los mismos. | |
| NZ734019A (en) | Novel micro-dystrophins and related methods of use | |
| PH12015502799A1 (en) | Substituted nucleosides, nucleotides and analogs thereof | |
| SG10201804835VA (en) | Substituted nucleosides, nucleotides and analogs thereof | |
| MX2015008035A (es) | Composiciones mejoradas para el salto de exones para tratar distrofia muscular. | |
| PH12014500557A1 (en) | Methods for treating hcv | |
| PH12013500005A1 (en) | Indolizine derivatives, process for the preparation thereof and therapeutic use thereof | |
| NZ630890A (en) | Microrna compounds and methods for modulating mir-122 | |
| PH12015501088A1 (en) | Dimeric compounds | |
| MX2015007097A (es) | Nuevas fenil-piridinas/pirazinas bi-anulares para el tratamiento de cancer. | |
| WO2015184375A3 (en) | Compositions and methods for promoting intestinal stem cell and/or non-stem progenitor cell function | |
| MX2013005875A (es) | Inhibidores de microarn que comprenden nucleotidos bloqueados. | |
| PH12017501539A1 (en) | Disacetoxytubulysin h and analogs thereof | |
| WO2013112699A3 (en) | Proteasome activity enhancing compounds | |
| WO2013112651A3 (en) | Proteasome activity modulating compounds | |
| MX359337B (es) | Oligonucleotidos antisentido para el tratamiento de celulas madre del cancer. | |
| GB2507700A (en) | Compositions and methods for treatment of metastatic cancer | |
| GB201008123D0 (en) | Novel compounds |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PSEA | Patent sealed | ||
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 14 MAR 2019 BY DAVIES COLLISON CAVE PTY LTD Effective date: 20171221 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 14 MAR 2020 BY DENNEMEYER Effective date: 20190219 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 14 MAR 2021 BY DENNEMEYER + CO. Effective date: 20200219 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 14 MAR 2022 BY DENNEMEYER + CO. S.A.R.L. Effective date: 20210302 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 14 MAR 2023 BY PETRA OLK Effective date: 20220307 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 14 MAR 2024 BY DENNEMEYER + CO. Effective date: 20230307 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 14 MAR 2025 BY DENNEMEYER + CO. S.A.R.L. Effective date: 20240304 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 14 MAR 2026 BY DENNEMEYER AND CO. SARL Effective date: 20250304 |