JP2021511803A5 - - Google Patents
Info
- Publication number
- JP2021511803A5 JP2021511803A5 JP2020541767A JP2020541767A JP2021511803A5 JP 2021511803 A5 JP2021511803 A5 JP 2021511803A5 JP 2020541767 A JP2020541767 A JP 2020541767A JP 2020541767 A JP2020541767 A JP 2020541767A JP 2021511803 A5 JP2021511803 A5 JP 2021511803A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- pharmaceutical composition
- composition according
- grna
- aav
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862624748P | 2018-01-31 | 2018-01-31 | |
| US62/624,748 | 2018-01-31 | ||
| PCT/US2019/015988 WO2019152609A1 (en) | 2018-01-31 | 2019-01-31 | Compositions and methods for correcting dystrophin mutations in human cardiomyocytes |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021511803A JP2021511803A (ja) | 2021-05-13 |
| JP2021511803A5 true JP2021511803A5 (https=) | 2022-01-27 |
Family
ID=65657512
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020541767A Pending JP2021511803A (ja) | 2018-01-31 | 2019-01-31 | ヒト心筋細胞におけるジストロフィン変異を修正するための組成物および方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20200370042A1 (https=) |
| EP (1) | EP3746557A1 (https=) |
| JP (1) | JP2021511803A (https=) |
| KR (1) | KR20200116933A (https=) |
| CN (1) | CN111836893A (https=) |
| AU (1) | AU2019216321A1 (https=) |
| BR (1) | BR112020015617A2 (https=) |
| CA (1) | CA3088547A1 (https=) |
| EA (1) | EA202091828A1 (https=) |
| IL (1) | IL276139A (https=) |
| WO (1) | WO2019152609A1 (https=) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| AR118668A1 (es) * | 2019-04-14 | 2021-10-20 | Univ Duke | Composiciones para editar genoma a base de crispr / cas para restaurar la función de distrofina |
| WO2021064162A1 (en) * | 2019-10-02 | 2021-04-08 | Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) | Treatment of diseases caused by frame shift mutations |
| EP4069314A4 (en) * | 2019-12-03 | 2024-06-26 | Duke University | SYSTEMS AND METHODS FOR LIPID NANOPARTICLE DELIVERY OF GENE EDITING MACHINERY |
| US20230272428A1 (en) * | 2019-12-16 | 2023-08-31 | President And Fellows Of Harvard College | Methods and compositions for correction of dmd mutations |
| CN115011598A (zh) * | 2020-09-02 | 2022-09-06 | 西湖大学 | 杜氏肌营养不良症相关的外显子剪接增强子、sgRNA、基因编辑工具及应用 |
| US20240254509A1 (en) * | 2021-03-04 | 2024-08-01 | Research Institute At Nationwide Children's Hospital | Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications |
| AU2022262420A1 (en) * | 2021-04-23 | 2023-11-16 | Research Institute At Nationwide Children's Hospital | Products and methods for treating muscular dystrophy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| JP2024525608A (ja) | 2021-07-09 | 2024-07-12 | 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製剤 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2023039444A2 (en) * | 2021-09-08 | 2023-03-16 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exon 51 for treatment of duchenne muscular dystrophy |
| WO2023172926A1 (en) * | 2022-03-08 | 2023-09-14 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exons for treatment of duchenne muscular dystrophy |
| WO2023172927A1 (en) * | 2022-03-08 | 2023-09-14 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exon 44, 50, and 53 for treatment of duchenne muscular dystrophy |
| KR20230134098A (ko) * | 2022-03-10 | 2023-09-20 | 주식회사 진코어 | 듀센 근이영양증 치료를 위한 유전자 편집 시스템 및 이를 이용한 질병 치료 방법 |
| WO2023240157A2 (en) * | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of dmd |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0273085A1 (en) | 1986-12-29 | 1988-07-06 | IntraCel Corporation | A method for internalizing nucleic acids into eukaryotic cells |
| BR122020021379B1 (pt) * | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| CA2758189C (en) * | 2009-04-10 | 2020-12-29 | Association Institut De Myologie | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| US20140329762A1 (en) * | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| CN106714845A (zh) * | 2014-08-11 | 2017-05-24 | 得克萨斯州大学系统董事会 | 通过crispr/cas9介导的基因编辑预防肌营养不良 |
| EP3748004A1 (en) * | 2015-04-01 | 2020-12-09 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy |
| WO2017049407A1 (en) * | 2015-09-23 | 2017-03-30 | UNIVERSITé LAVAL | Modification of the dystrophin gene and uses thereof |
| EP3858993A1 (en) * | 2015-10-09 | 2021-08-04 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| JP7075597B2 (ja) * | 2016-05-05 | 2022-05-26 | デューク ユニバーシティ | デュシェンヌ型筋ジストロフィーを治療するためのｃｒｉｓｐｒ／ｃａｓ関連の方法および組成物 |
| US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
| JP7490211B2 (ja) * | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Ｃｐｆ１に基づくゲノム編集の治療適用 |
| RU2625003C1 (ru) * | 2016-10-04 | 2017-07-11 | Федеральное государственное бюджетное научное учреждение "Медико-генетический научный центр" | Набор последовательностей нуклеотидов для медицинской технологии детекции наиболее частых в россии делеций гена dmd методом мультиплексного пцр/пдаф анализа |
| US10687520B2 (en) * | 2017-03-07 | 2020-06-23 | The Board Of Regents Of The University Of Texas System | Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44 |
-
2019
- 2019-01-31 AU AU2019216321A patent/AU2019216321A1/en not_active Abandoned
- 2019-01-31 EA EA202091828A patent/EA202091828A1/ru unknown
- 2019-01-31 WO PCT/US2019/015988 patent/WO2019152609A1/en not_active Ceased
- 2019-01-31 KR KR1020207022943A patent/KR20200116933A/ko not_active Ceased
- 2019-01-31 US US16/966,274 patent/US20200370042A1/en not_active Abandoned
- 2019-01-31 BR BR112020015617-5A patent/BR112020015617A2/pt not_active Application Discontinuation
- 2019-01-31 EP EP19708910.5A patent/EP3746557A1/en not_active Withdrawn
- 2019-01-31 CA CA3088547A patent/CA3088547A1/en active Pending
- 2019-01-31 JP JP2020541767A patent/JP2021511803A/ja active Pending
- 2019-01-31 CN CN201980011157.5A patent/CN111836893A/zh active Pending
-
2020
- 2020-07-19 IL IL276139A patent/IL276139A/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021511803A5 (https=) | ||
| JP7374119B2 (ja) | 減少した肝臓向性を有するＡＡＶ９とＡＡＶｒｈ７４とのハイブリッド組換えアデノ随伴ウイルス血清型 | |
| EP4211231B1 (en) | Peptide-modified aav capsid | |
| CN115023242B (zh) | 腺相关病毒载体变体 | |
| CN105579465B (zh) | 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途 | |
| JP6619454B2 (ja) | キャプシド | |
| JP2022508182A5 (https=) | ||
| BR112020021903A2 (pt) | terapia genética para degeneração do snc | |
| JP2020503869A5 (https=) | ||
| JP2021512649A5 (https=) | ||
| TW201629225A (zh) | 第九因子基因療法 | |
| JP2020054367A (ja) | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 | |
| JP2020509786A5 (https=) | ||
| EP4536686B1 (en) | Peptide-modified aav capsid with enhanced muscle transduction efficiency | |
| WO2020208032A1 (en) | Hybrid promoters for muscle expression | |
| JP2023515795A5 (https=) | ||
| US20230272428A1 (en) | Methods and compositions for correction of dmd mutations | |
| KR20250120301A (ko) | 심장 질환 및 심장 기능 장애 관련 질환 치료를 위해 간 친화성 감소 및 심장 세포 형질도입이 향상된 재조합-아데노 관련 바이러스 유전자 치료 벡터 | |
| KR102874560B1 (ko) | 아데노연관바이러스 변이체 | |
| US20240398985A1 (en) | Novel transcription factors | |
| US12480098B1 (en) | Adeno-associated virus variant | |
| KR20260057323A (ko) | 아데노연관바이러스 변이체 | |
| WO2026041758A1 (en) | Modified aav capsid with enhanced transduction efficiency | |
| BR112023004176B1 (pt) | Proteína de capsídeo de aav, polinucleotídeo, plasmídeo recombinante, partícula de vetor de aav, seu uso para tratamento de doenças musculares e composição farmacêutica | |
| HK40091751A (en) | Peptide-modified aav capsid |