JP2024519451A5 - - Google Patents
Info
- Publication number
- JP2024519451A5 JP2024519451A5 JP2023563991A JP2023563991A JP2024519451A5 JP 2024519451 A5 JP2024519451 A5 JP 2024519451A5 JP 2023563991 A JP2023563991 A JP 2023563991A JP 2023563991 A JP2023563991 A JP 2023563991A JP 2024519451 A5 JP2024519451 A5 JP 2024519451A5
- Authority
- JP
- Japan
- Prior art keywords
- approximately
- conjugate
- pharmaceutically acceptable
- antisense oligomer
- acceptable salt
- 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.)
- Pending
Links
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163182327P | 2021-04-30 | 2021-04-30 | |
| US63/182,327 | 2021-04-30 | ||
| US202163249721P | 2021-09-29 | 2021-09-29 | |
| US63/249,721 | 2021-09-29 | ||
| PCT/US2022/026887 WO2022232478A1 (en) | 2021-04-30 | 2022-04-29 | Treatment methods for muscular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024519451A JP2024519451A (ja) | 2024-05-14 |
| JP2024519451A5 true JP2024519451A5 (enExample) | 2025-05-02 |
Family
ID=81749386
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023563991A Pending JP2024519451A (ja) | 2021-04-30 | 2022-04-29 | 筋ジストロフィーのための治療法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US12286630B2 (enExample) |
| EP (1) | EP4330395A1 (enExample) |
| JP (1) | JP2024519451A (enExample) |
| KR (1) | KR20240004609A (enExample) |
| AU (1) | AU2022267324A1 (enExample) |
| BR (1) | BR112023021849A2 (enExample) |
| CA (1) | CA3216839A1 (enExample) |
| CO (1) | CO2023016203A2 (enExample) |
| IL (1) | IL307937A (enExample) |
| MX (1) | MX2023012559A (enExample) |
| WO (1) | WO2022232478A1 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2020028832A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| EP3830259A4 (en) | 2018-08-02 | 2022-05-04 | Dyne Therapeutics, Inc. | MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF 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 |
| 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 |
| CA3211038A1 (en) | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| WO2026112568A1 (en) * | 2024-11-25 | 2026-05-28 | Pepgen Inc. | Methods and compositions for treating duchenne muscular dystrophy |
Family Cites Families (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| 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 |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| JP2528107B2 (ja) | 1985-03-15 | 1996-08-28 | サマ−トン，ジエ−ムス | ポリヌクレオチド測定試薬と方法 |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| JPH07509133A (ja) | 1992-07-17 | 1995-10-12 | リボザイム・ファーマシューティカルズ・インコーポレイテッド | 動物疾患の処置のための方法および剤 |
| 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 |
| 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 |
| EP0998945A1 (en) * | 1998-09-30 | 2000-05-10 | Transgene S.A. | Use of magnesium (Mg2+) for the enhancement of gene delivery in gene therapy |
| 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 |
| EP2530155B1 (en) | 2002-11-25 | 2016-03-16 | Masafumi Matsuo | ENA nucleic acid drugs modifying splicing in mRNA precursor |
| AU2004235396B2 (en) | 2003-04-29 | 2010-11-25 | Sarepta Therapeutics, Inc. | Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells |
| WO2005120526A1 (ja) * | 2004-06-11 | 2005-12-22 | Combi Corporation | 筋ジストロフィー進行抑制組成物 |
| CA2691673A1 (en) | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
| 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 |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| CN103154009B (zh) | 2010-09-30 | 2015-06-10 | 日本新药株式会社 | 吗啉基核酸衍生物 |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| AU2011367230B2 (en) | 2011-05-05 | 2017-08-10 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| CN108588073A (zh) | 2011-12-28 | 2018-09-28 | 日本新药株式会社 | 反义核酸 |
| BR112016020618B1 (pt) | 2014-03-12 | 2023-04-04 | Nippon Shinyaku Co., Ltd. | Oligômero antissentido, composição farmacêutica, e, uso de um oligômero antissentido |
| PT3159409T (pt) | 2014-06-17 | 2020-01-21 | Nippon Shinyaku Co Ltd | Ácido nucleico anti-sentido para utilização no tratamento da distrofia muscular de duchenne |
| HUE050061T2 (hu) | 2015-09-15 | 2020-12-28 | Nippon Shinyaku Co Ltd | Antiszensz nukleinsav |
| MA46427A (fr) | 2015-10-09 | 2018-08-15 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| AU2017291960B2 (en) | 2016-07-05 | 2024-03-14 | Biomarin Technologies B.V. | Pre-mRNA splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders |
| BR112019009953A2 (pt) | 2016-11-16 | 2019-10-08 | Academisch Ziekenhuis Leiden | substâncias direcionadas a vários órgãos ou tecidos selecionados |
| ES3051834T3 (en) | 2016-12-19 | 2025-12-30 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| NZ755416A (en) | 2016-12-19 | 2023-05-26 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| PT4122497T (pt) | 2016-12-19 | 2024-06-12 | Sarepta Therapeutics Inc | Conjugados oligoméricos de salto de exão para a distrofia muscular |
| KR20240172236A (ko) | 2017-01-06 | 2024-12-09 | 어비디티 바이오사이언시스 인크. | 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법 |
| SG11202002517RA (en) | 2017-09-22 | 2020-04-29 | Avidity Biosciences Inc | Nucleic acid-polypeptide compositions and methods of inducing exon skipping |
| JP7345466B2 (ja) | 2018-06-26 | 2023-09-15 | 日本新薬株式会社 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
| WO2020028832A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12331293B2 (en) | 2018-11-02 | 2025-06-17 | Biomarin Technologies B.V. | Bispecific antisense oligonucleotides for dystrophin exon skipping |
| CN113660938A (zh) | 2019-01-30 | 2021-11-16 | 国立研究开发法人国立精神·神经医疗研究中心 | 核酸递送复合物 |
| WO2020214763A1 (en) * | 2019-04-18 | 2020-10-22 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| WO2020219820A1 (en) | 2019-04-25 | 2020-10-29 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
-
2022
- 2022-04-29 WO PCT/US2022/026887 patent/WO2022232478A1/en not_active Ceased
- 2022-04-29 IL IL307937A patent/IL307937A/en unknown
- 2022-04-29 JP JP2023563991A patent/JP2024519451A/ja active Pending
- 2022-04-29 BR BR112023021849A patent/BR112023021849A2/pt unknown
- 2022-04-29 KR KR1020237040520A patent/KR20240004609A/ko active Pending
- 2022-04-29 AU AU2022267324A patent/AU2022267324A1/en not_active Abandoned
- 2022-04-29 CA CA3216839A patent/CA3216839A1/en active Pending
- 2022-04-29 EP EP22724552.9A patent/EP4330395A1/en active Pending
- 2022-04-29 US US17/732,757 patent/US12286630B2/en active Active
- 2022-04-29 MX MX2023012559A patent/MX2023012559A/es unknown
-
2023
- 2023-11-29 CO CONC2023/0016203A patent/CO2023016203A2/es unknown
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