LT3554554T - Egzoną praleidžiantys oligomero konjugatai nuo raumenų distrofijos - Google Patents
Egzoną praleidžiantys oligomero konjugatai nuo raumenų distrofijosInfo
- Publication number
- LT3554554T LT3554554T LTEPPCT/US2017/066351T LT17066351T LT3554554T LT 3554554 T LT3554554 T LT 3554554T LT 17066351 T LT17066351 T LT 17066351T LT 3554554 T LT3554554 T LT 3554554T
- Authority
- LT
- Lithuania
- Prior art keywords
- muscular dystrophy
- exon skipping
- oligomer conjugates
- skipping oligomer
- conjugates
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
- A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
- A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
-
- 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
-
- 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
- C12N2310/32—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
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662436199P | 2016-12-19 | 2016-12-19 | |
| US201762443481P | 2017-01-06 | 2017-01-06 | |
| US201762479177P | 2017-03-30 | 2017-03-30 | |
| US201762562119P | 2017-09-22 | 2017-09-22 | |
| PCT/US2017/066351 WO2018118627A1 (en) | 2016-12-19 | 2017-12-14 | Exon skipping oligomer conjugates for muscular dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| LT3554554T true LT3554554T (lt) | 2022-11-25 |
Family
ID=61006319
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| LTEPPCT/US2017/066351T LT3554554T (lt) | 2016-12-19 | 2017-12-14 | Egzoną praleidžiantys oligomero konjugatai nuo raumenų distrofijos |
Country Status (27)
| Country | Link |
|---|---|
| US (2) | US11382981B2 (lt) |
| EP (2) | EP4122497B1 (lt) |
| JP (2) | JP7125940B2 (lt) |
| KR (2) | KR102639633B1 (lt) |
| CN (2) | CN117298290A (lt) |
| AU (1) | AU2017382741A1 (lt) |
| BR (1) | BR112019012651A2 (lt) |
| CA (1) | CA3046801A1 (lt) |
| CO (1) | CO2019007398A2 (lt) |
| DK (1) | DK3554554T3 (lt) |
| ES (1) | ES2929498T3 (lt) |
| HR (1) | HRP20221316T1 (lt) |
| HU (1) | HUE060065T2 (lt) |
| IL (2) | IL267245B2 (lt) |
| LT (1) | LT3554554T (lt) |
| MD (1) | MD3554554T2 (lt) |
| MX (1) | MX2019006989A (lt) |
| NZ (1) | NZ755422A (lt) |
| PL (1) | PL3554554T3 (lt) |
| PT (1) | PT3554554T (lt) |
| RS (1) | RS63705B1 (lt) |
| SA (1) | SA519402143B1 (lt) |
| SG (1) | SG10202100491QA (lt) |
| SI (1) | SI3554554T1 (lt) |
| TW (1) | TWI780095B (lt) |
| WO (1) | WO2018118627A1 (lt) |
| ZA (1) | ZA201903940B (lt) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| MX2021008539A (es) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics Inc | Procesos para preparar oligomeros. |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| MX2019006989A (es) | 2016-12-19 | 2019-08-16 | Sarepta Therapeutics Inc | Conjugados de oligomeros de omision de exon para distrofia muscular. |
| GB201821269D0 (en) | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| MX2021011498A (es) * | 2019-03-28 | 2022-01-04 | Sarepta Therapeutics Inc | Metodos para tratar la distrofia muscular con casimersin. |
| US20220193246A1 (en) * | 2019-04-18 | 2022-06-23 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| US20220296633A1 (en) * | 2019-06-19 | 2022-09-22 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
| TW202138560A (zh) | 2019-12-26 | 2021-10-16 | 日商日本新藥股份有限公司 | 誘導外顯子５０的跳讀的反義核酸 |
| EP4112083A1 (en) | 2020-02-28 | 2023-01-04 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid inducing skipping of exon 51 |
| EP4361269A1 (en) | 2021-06-23 | 2024-05-01 | Nippon Shinyaku Co., Ltd. | Combination of antisense oligomers |
| TW202308664A (zh) | 2021-07-08 | 2023-03-01 | 日商日本新藥股份有限公司 | 腎毒性減輕劑 |
| JPWO2023282345A1 (lt) | 2021-07-08 | 2023-01-12 | ||
| TW202317147A (zh) | 2021-07-08 | 2023-05-01 | 日商日本新藥股份有限公司 | 析出抑制劑 |
Family Cites Families (107)
| 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 |
| DE3687030T2 (de) | 1985-03-15 | 1993-03-11 | James Summerton | Stereoregulare polynukleotiden bindende polymere. |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| 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 |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| 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 |
| US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| EP1251170A3 (en) | 1992-07-17 | 2002-10-30 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for treatment of NF-kappaB dependent animal diseases |
| EP1897942A1 (en) | 1993-05-11 | 2008-03-12 | The University Of North Carolina At Chapel Hill | Antisense oligonucleotides which combat aberrant splicing and methods of using the same |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
| US5820873A (en) | 1994-09-30 | 1998-10-13 | The University Of British Columbia | Polyethylene glycol modified ceramide lipids and liposome uses thereof |
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| IL115849A0 (en) | 1994-11-03 | 1996-01-31 | Merz & Co Gmbh & Co | Tangential filtration preparation of liposomal drugs and liposome product thereof |
| US7572582B2 (en) | 1997-09-12 | 2009-08-11 | Exiqon A/S | Oligonucleotide analogues |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| 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 |
| JP2000125448A (ja) | 1998-10-14 | 2000-04-28 | Yazaki Corp | 電気接続箱 |
| JP2000256547A (ja) | 1999-03-10 | 2000-09-19 | Sumitomo Dow Ltd | 耐熱性プラスチックカード用樹脂組成物 |
| US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
| ATE356824T1 (de) | 1999-05-04 | 2007-04-15 | Santaris Pharma As | L-ribo-lna analoge |
| JP2000325085A (ja) | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
| US7070807B2 (en) | 1999-12-29 | 2006-07-04 | Mixson A James | Branched histidine copolymers and methods for using same |
| WO2001047496A1 (en) | 1999-12-29 | 2001-07-05 | Mixson A James | Histidine copolymer and methods for using same |
| US6653467B1 (en) | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
| US6727355B2 (en) | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| EP1446412B1 (en) | 2001-09-04 | 2012-03-07 | Exiqon A/S | Novel lna compositions and uses thereof |
| KR100464261B1 (ko) | 2002-01-24 | 2005-01-03 | 주식회사 파나진 | Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법 |
| KR20030084444A (ko) | 2002-04-26 | 2003-11-01 | 주식회사 파나진 | Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법 |
| US7569575B2 (en) | 2002-05-08 | 2009-08-04 | Santaris Pharma A/S | Synthesis of locked nucleic acid derivatives |
| AU2003291682A1 (en) | 2002-11-05 | 2004-06-03 | Isis Pharmaceuticals, Inc. | 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations |
| ES2509140T3 (es) | 2002-11-25 | 2014-10-17 | Masafumi Matsuo | Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm |
| CA2524255C (en) | 2003-03-21 | 2014-02-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| US7211668B2 (en) | 2003-07-28 | 2007-05-01 | Panagene, Inc. | PNA monomer and precursor |
| SI2206781T1 (sl) | 2004-06-28 | 2016-05-31 | The University Of Western Australia | Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe |
| EP1877555A2 (en) | 2005-04-22 | 2008-01-16 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secondary rna structure. |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| WO2007058894A2 (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 |
| DE212006000097U1 (de) | 2006-05-17 | 2009-02-26 | Sokolova, Svetlana Anatolevna | Transportmittel |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| JP5864100B2 (ja) | 2007-06-29 | 2016-02-17 | サレプタ セラピューティクス インコーポレイテッド | 組織特異的ペプチドコンジュゲートおよび方法 |
| JP2010533170A (ja) | 2007-07-12 | 2010-10-21 | プロセンサ テクノロジーズ ビー．ブイ． | 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子 |
| WO2009054725A2 (en) | 2007-10-26 | 2009-04-30 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| EP2207779B1 (en) | 2007-11-15 | 2014-04-09 | Sarepta Therapeutics, 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 |
| US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| WO2009127230A1 (en) | 2008-04-16 | 2009-10-22 | Curevac Gmbh | MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION |
| 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 |
| AU2009310557B2 (en) | 2008-10-27 | 2014-09-11 | Academisch Ziekenhuis Leiden | Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA |
| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| PT2417257E (pt) | 2009-04-10 | 2016-06-03 | Universität Bern | Oligonucleótidos triciclo-dna anti-sentido, composições, e métodos para o tratamento de doença |
| JP2012524540A (ja) | 2009-04-24 | 2012-10-18 | プロセンサ テクノロジーズ ビー．ブイ． | Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド |
| IN2012DN00720A (lt) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
| CN102574888A (zh) | 2009-09-16 | 2012-07-11 | 株式会社启拉坚 | 用于rna及其衍生物的合成的新型保护基 |
| KR102487132B1 (ko) | 2009-11-12 | 2023-01-10 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
| 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 |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| KR20140052963A (ko) | 2011-02-08 | 2014-05-07 | 더 샬롯테-맥클렌버그 하스피털 오쏘러티 디/비/에이 카롤리나스 헬스케어 시스템 | 안티센스 올리고뉴클레오티드 |
| CA2834128A1 (en) * | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US9607308B2 (en) | 2011-06-29 | 2017-03-28 | American Express Travel Related Services Company, Inc. | Spend based digital ad targeting and measurement |
| PT2581448E (pt) | 2011-10-13 | 2015-05-21 | Institut National De La Santé Et De La Rech Médicale (Inserm) | Dna triciclo-fosforotioato |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| CA2861247C (en) | 2011-12-28 | 2021-11-16 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acids |
| ES2907250T3 (es) | 2012-01-27 | 2022-04-22 | Biomarin Tech Bv | Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker |
| DE102012101676A1 (de) | 2012-02-29 | 2013-08-29 | Klaus-Dieter Rösler | Verfahren und Vorrichtung zum Bearbeiten von Formularen mit einer Datenverarbeitungsanlage |
| JP6460983B2 (ja) * | 2012-07-03 | 2019-01-30 | バイオマリン テクノロジーズ ベー．フェー． | 筋ジストロフィー患者の治療のためのオリゴヌクレオチド |
| EP2872147B1 (en) | 2012-07-13 | 2022-12-21 | Wave Life Sciences Ltd. | Method for making chiral oligonucleotides |
| BR112015000784A8 (pt) | 2012-07-13 | 2018-04-03 | Wave Life Sciences Japan | Grupo auxiliar assimétrico |
| US10233495B2 (en) | 2012-09-27 | 2019-03-19 | The Hospital For Sick Children | Methods and compositions for screening and treating developmental disorders |
| AU2013364158A1 (en) | 2012-12-20 | 2015-07-09 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| EP2946013A1 (en) * | 2013-01-16 | 2015-11-25 | Iowa State University Research Foundation, Inc. | A deep intronic target for splicing correction on spinal muscular atrophy gene |
| BR122020016865B1 (pt) | 2013-03-14 | 2022-12-27 | Sarepta Therapeutics, Inc. | Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd) |
| PL2970964T3 (pl) | 2013-03-14 | 2019-06-28 | Sarepta Therapeutics, Inc. | Kompozycje pomijające eksony do leczenia dystrofii mięśniowej |
| CA2906812A1 (en) * | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| KR20230152178A (ko) | 2014-01-16 | 2023-11-02 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
| US20150329609A1 (en) | 2014-03-03 | 2015-11-19 | Sujata Acharjee | Chimeric dystrophin proteins to treat dystrophinopathies |
| AU2015227733B2 (en) | 2014-03-12 | 2019-10-31 | National Center Of Neurology And Psychiatry | Antisense nucleic acids |
| RS59764B1 (sr) | 2014-06-17 | 2020-02-28 | Nippon Shinyaku Co Ltd | Antismisaona nukleinska kiselina za upotrebu u lečenju dišenove mišićne distrofije |
| WO2016070166A2 (en) | 2014-11-02 | 2016-05-06 | Arcturus Therapeutics, Inc. | Messenger una molecules and uses thereof |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| EP3858993A1 (en) * | 2015-10-09 | 2021-08-04 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| MX2018003992A (es) | 2015-10-09 | 2018-08-01 | Wave Life Sciences Ltd | Composiciones oligonucleotidicas y sus metodos. |
| NZ747685A (en) * | 2016-04-29 | 2023-05-26 | Sarepta Therapeutics Inc | Oligonucleotide analogues targeting human lmna |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| MA45290A (fr) | 2016-05-04 | 2019-03-13 | Wave Life Sciences Ltd | Procédés et compositions d'agents biologiquement actifs |
| BR112019012664A2 (pt) | 2016-12-19 | 2020-01-21 | Sarepta Therapeutics Inc | conjugados de oligômero de salto de éxon para distrofia muscular |
| MD3554553T2 (ro) | 2016-12-19 | 2022-10-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
| MX2019006989A (es) * | 2016-12-19 | 2019-08-16 | Sarepta Therapeutics Inc | Conjugados de oligomeros de omision de exon para distrofia muscular. |
-
2017
- 2017-12-14 MX MX2019006989A patent/MX2019006989A/es unknown
- 2017-12-14 EP EP22180767.0A patent/EP4122497B1/en active Active
- 2017-12-14 AU AU2017382741A patent/AU2017382741A1/en active Pending
- 2017-12-14 HR HRP20221316TT patent/HRP20221316T1/hr unknown
- 2017-12-14 KR KR1020197020409A patent/KR102639633B1/ko active IP Right Grant
- 2017-12-14 JP JP2019531379A patent/JP7125940B2/ja active Active
- 2017-12-14 PT PT178324059T patent/PT3554554T/pt unknown
- 2017-12-14 CN CN202311010831.2A patent/CN117298290A/zh active Pending
- 2017-12-14 MD MDE20191208T patent/MD3554554T2/ro unknown
- 2017-12-14 US US16/469,104 patent/US11382981B2/en active Active
- 2017-12-14 WO PCT/US2017/066351 patent/WO2018118627A1/en unknown
- 2017-12-14 SG SG10202100491QA patent/SG10202100491QA/en unknown
- 2017-12-14 CN CN201780086303.1A patent/CN110337308B/zh active Active
- 2017-12-14 SI SI201731254T patent/SI3554554T1/sl unknown
- 2017-12-14 CA CA3046801A patent/CA3046801A1/en active Pending
- 2017-12-14 DK DK17832405.9T patent/DK3554554T3/da active
- 2017-12-14 NZ NZ755422A patent/NZ755422A/en unknown
- 2017-12-14 RS RS20221008A patent/RS63705B1/sr unknown
- 2017-12-14 BR BR112019012651A patent/BR112019012651A2/pt active Search and Examination
- 2017-12-14 ES ES17832405T patent/ES2929498T3/es active Active
- 2017-12-14 IL IL267245A patent/IL267245B2/en unknown
- 2017-12-14 HU HUE17832405A patent/HUE060065T2/hu unknown
- 2017-12-14 LT LTEPPCT/US2017/066351T patent/LT3554554T/lt unknown
- 2017-12-14 PL PL17832405.9T patent/PL3554554T3/pl unknown
- 2017-12-14 EP EP17832405.9A patent/EP3554554B1/en active Active
- 2017-12-14 KR KR1020237039425A patent/KR20240006023A/ko active Search and Examination
- 2017-12-18 TW TW106144430A patent/TWI780095B/zh active
-
2019
- 2019-06-17 SA SA519402143A patent/SA519402143B1/ar unknown
- 2019-06-18 ZA ZA2019/03940A patent/ZA201903940B/en unknown
- 2019-07-10 CO CONC2019/0007398A patent/CO2019007398A2/es unknown
-
2022
- 2022-05-02 JP JP2022075930A patent/JP2022101695A/ja active Pending
- 2022-05-26 US US17/804,280 patent/US20220387601A1/en active Pending
-
2023
- 2023-07-05 IL IL304284A patent/IL304284A/en unknown
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL287047A (en) | Exon-skipping oligomer conjugates for muscular dystrophy | |
| IL267246A (en) | Exon-skipping oligomer conjugates for muscular dystrophy | |
| IL304284A (en) | Exon-skipping oligomer conjugates for muscular dystrophy | |
| IL273289A (en) | Exon-skipping oligomer conjugates for muscular dystrophy | |
| IL285516B (en) | Amatoxin antibody conjugates | |
| IL263892A (en) | Exon skipping oligomers for muscular dystrophy | |
| GB201513607D0 (en) | Pyrrolobenzodiazepine-antibody conjugates | |
| IL278966A (en) | Exon-skipping oligomer conjugates for muscular dystrophy | |
| PL3528829T3 (pl) | Koniugaty bacytracyna-oligomer alginianowy | |
| IL283646A (en) | Exon-skipping oligomer conjugates for muscular dystrophy | |
| HK1257229A1 (zh) | Cck2r-藥物偶聯物 | |
| PL3270711T3 (pl) | Koniugaty cukier-dipeptyd | |
| GB201617860D0 (en) | Polymyxin-alginate oligomer conjugates | |
| GB201518839D0 (en) | Conjugates | |
| GB201513605D0 (en) | Pyrrolobenzodiazepine-antibody conjugates |