JP2024515720A5 - - Google Patents
Info
- Publication number
- JP2024515720A5 JP2024515720A5 JP2023564657A JP2023564657A JP2024515720A5 JP 2024515720 A5 JP2024515720 A5 JP 2024515720A5 JP 2023564657 A JP2023564657 A JP 2023564657A JP 2023564657 A JP2023564657 A JP 2023564657A JP 2024515720 A5 JP2024515720 A5 JP 2024515720A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide sequence
- test
- composition
- combination
- raav
- 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 |
|---|---|---|---|
| US202163178648P | 2021-04-23 | 2021-04-23 | |
| US63/178,648 | 2021-04-23 | ||
| PCT/US2022/025986 WO2022226334A1 (en) | 2021-04-23 | 2022-04-22 | Products and methods for treating muscular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024515720A JP2024515720A (ja) | 2024-04-10 |
| JP2024515720A5 true JP2024515720A5 (https=) | 2025-04-28 |
Family
ID=81854546
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023564657A Pending JP2024515720A (ja) | 2021-04-23 | 2022-04-22 | 筋ジストロフィーを治療するための製品及び方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20240200066A1 (https=) |
| EP (1) | EP4326752A1 (https=) |
| JP (1) | JP2024515720A (https=) |
| AU (1) | AU2022262420A1 (https=) |
| CA (1) | CA3217491A1 (https=) |
| WO (1) | WO2022226334A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2023228712A1 (en) | 2022-03-04 | 2024-10-17 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising engineered small nuclear rna (snrna) |
| EP4680741A2 (en) * | 2023-03-15 | 2026-01-21 | RegenxBio Inc. | Exon skipping gene therapy constructs, vectors and uses thereof |
| WO2025179121A1 (en) * | 2024-02-21 | 2025-08-28 | Research Institute At Nationwide Children's Hospital | Exon 17-targeted nucleic acids, compositions, and methods for treatment of dystrophin-based myopathies |
| CN120130955B (zh) * | 2025-05-16 | 2025-09-09 | 杭州市第一人民医院(西湖大学附属杭州市第一人民医院) | 一种多源数据融合的肌少症检测系统及方法 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| JP2001500497A (ja) | 1996-09-06 | 2001-01-16 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| EP2933332A1 (en) * | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| 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 |
| US20140080896A1 (en) * | 2011-08-30 | 2014-03-20 | The Regents Of The University Of California | Identification of small molecules that facilitate therapeutic exon skipping |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| CA2957661A1 (en) * | 2014-08-09 | 2016-02-18 | Kevin FLANIGAN | Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| AU2019216321A1 (en) * | 2018-01-31 | 2020-07-30 | The Board Of Regents Of The University Of Texas System | Compositions and methods for correcting dystrophin mutations in human cardiomyocytes |
| EP3801638A1 (en) * | 2018-06-08 | 2021-04-14 | Novartis AG | Cell-based assay for measuring drug product potency |
| WO2021026075A1 (en) * | 2019-08-02 | 2021-02-11 | Research Institute At Nationwide Children's Hospital | Exon 44-targeted nucleic acids and recombinant adeno-associated virus comprising said nucleic acids for treatment of dystrophin-based myopathies |
-
2022
- 2022-04-22 AU AU2022262420A patent/AU2022262420A1/en not_active Abandoned
- 2022-04-22 US US18/287,677 patent/US20240200066A1/en active Pending
- 2022-04-22 EP EP22726853.9A patent/EP4326752A1/en active Pending
- 2022-04-22 JP JP2023564657A patent/JP2024515720A/ja active Pending
- 2022-04-22 CA CA3217491A patent/CA3217491A1/en active Pending
- 2022-04-22 WO PCT/US2022/025986 patent/WO2022226334A1/en not_active Ceased
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2024515720A5 (https=) | ||
| Khoshandam et al. | Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine | |
| JP5711666B2 (ja) | 白血球への効率的輸送 | |
| Kabanov et al. | Pluronic block copolymers for gene delivery | |
| ES2502915T3 (es) | Composición que comprende un ARN(m) complejado y un ARNm desnudo para proporcionar o mejorar la respuesta inmunoestimuladora en un mamífero y usos de la misma | |
| HRP20250932T1 (hr) | Dostava mišićno specifičnog mikrodistrofina putem vektora adeno-asociranog virusa za liječenje mišićne distrofije | |
| EP4218828A3 (en) | Adeno-associated virus variant capsids and methods of use thereof | |
| JP2022544627A5 (https=) | ||
| AU1386697A (en) | Polymers | |
| EP3416634A1 (en) | Immunomodulatory agents and methods of use thereof | |
| WO2011115862A1 (en) | Endosomolytic poly(amidoamine) disulfide polymers for the delivery of oligonucleotides | |
| KR102075393B1 (ko) | 중증 열성 혈소판 감소 증후군(sfts) 바이러스 감염 질환 예방 또는 치료용 백신 조성물 | |
| JP2024527340A5 (https=) | ||
| JP2021513365A (ja) | スクレロスチンに対するアプタマー及びその使用 | |
| KR20200041310A (ko) | 인간에서의 ａａｖ 유전자 요법을 위한 수단 및 방법 | |
| JP2023543029A5 (https=) | ||
| AU2004313242A1 (en) | HIV gp41 HR2-derived synthetic peptides, and their use in therapy to inhibit transmission of human immunodeficiency virus | |
| CN114276419B (zh) | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 | |
| CN114958850A (zh) | 一种基因组件、含有此基因组件的递送系统及其应用 | |
| JP2022532098A5 (https=) | ||
| WO2025043010A1 (en) | Nucleic acid compositions for the generation of a zika virus-like particle | |
| CA2423045A1 (en) | Adenoviral vectors for gene delivery in skeletal muscle cells or myoblasts | |
| CN117017996B (zh) | Navitoclax（ABT263）在制备广谱抗病毒抑制剂中的应用 | |
| Ustinova et al. | Comparative analysis of polyethyleneimine efficiency for improvement of plasmid DNA bioavailability | |
| US11299537B2 (en) | Methods for treatment of motor neuron diseases |