JPWO2022115745A5 - - Google Patents
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- Publication number
- JPWO2022115745A5 JPWO2022115745A5 JP2023532456A JP2023532456A JPWO2022115745A5 JP WO2022115745 A5 JPWO2022115745 A5 JP WO2022115745A5 JP 2023532456 A JP2023532456 A JP 2023532456A JP 2023532456 A JP2023532456 A JP 2023532456A JP WO2022115745 A5 JPWO2022115745 A5 JP WO2022115745A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- viral vector
- nucleic acid
- vector according
- 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
- 239000013603 viral vector Substances 0.000 claims 13
- 108020004707 nucleic acids Proteins 0.000 claims 10
- 150000007523 nucleic acids Chemical class 0.000 claims 10
- 102000039446 nucleic acids Human genes 0.000 claims 10
- 102100021158 Double homeobox protein 4 Human genes 0.000 claims 7
- 101000968549 Homo sapiens Double homeobox protein 4 Proteins 0.000 claims 7
- 239000000203 mixture Substances 0.000 claims 7
- 239000013598 vector Substances 0.000 claims 7
- 210000001808 exosome Anatomy 0.000 claims 5
- 239000002105 nanoparticle Substances 0.000 claims 5
- 239000002773 nucleotide Substances 0.000 claims 5
- 125000003729 nucleotide group Chemical group 0.000 claims 5
- 206010028980 Neoplasm Diseases 0.000 claims 4
- 201000011510 cancer Diseases 0.000 claims 4
- 239000003814 drug Substances 0.000 claims 4
- 201000006938 muscular dystrophy Diseases 0.000 claims 4
- 208000037149 Facioscapulohumeral dystrophy Diseases 0.000 claims 3
- 208000008570 facioscapulohumeral muscular dystrophy Diseases 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 206010039491 Sarcoma Diseases 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 239000003184 complementary RNA Substances 0.000 claims 2
- 230000002401 inhibitory effect Effects 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 238000010255 intramuscular injection Methods 0.000 claims 2
- 239000007927 intramuscular injection Substances 0.000 claims 2
- 210000003205 muscle Anatomy 0.000 claims 2
- -1 transdermal delivery Substances 0.000 claims 2
- 230000037317 transdermal delivery Effects 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 241000700584 Simplexvirus Species 0.000 claims 1
- 101150023087 UNC45B gene Proteins 0.000 claims 1
- 241000700618 Vaccinia virus Species 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000009977 dual effect Effects 0.000 claims 1
- 230000002452 interceptive effect Effects 0.000 claims 1
- 239000013645 rAAV1 vector Substances 0.000 claims 1
- 239000013648 rAAV12 vector Substances 0.000 claims 1
- 239000013646 rAAV2 vector Substances 0.000 claims 1
- 239000013647 rAAV8 vector Substances 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241000701447 unidentified baculovirus Species 0.000 claims 1
- 241001430294 unidentified retrovirus Species 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063119268P | 2020-11-30 | 2020-11-30 | |
| US63/119,268 | 2020-11-30 | ||
| PCT/US2021/061109 WO2022115745A1 (en) | 2020-11-30 | 2021-11-30 | Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd) |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023551279A JP2023551279A (ja) | 2023-12-07 |
| JPWO2022115745A5 true JPWO2022115745A5 (https=) | 2024-11-14 |
| JP2023551279A5 JP2023551279A5 (https=) | 2024-11-14 |
Family
ID=79021122
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023532456A Pending JP2023551279A (ja) | 2020-11-30 | 2021-11-30 | 顔面肩甲上腕筋ジストロフィー（ｆｓｈｄ）を治療するための組成物及び方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20240026356A1 (https=) |
| EP (1) | EP4251752A1 (https=) |
| JP (1) | JP2023551279A (https=) |
| KR (1) | KR20230128470A (https=) |
| AU (1) | AU2021385595A1 (https=) |
| CA (1) | CA3203585A1 (https=) |
| IL (1) | IL303230A (https=) |
| WO (1) | WO2022115745A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| AU2019419494B2 (en) * | 2018-12-31 | 2026-03-26 | Research Institute At Nationwide Children's Hospital | DUX4 RNA silencing using RNA targeting CRISPR-Cas13b |
| 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 |
| JP2025507958A (ja) | 2022-03-04 | 2025-03-21 | ロックアネイビオ， インコーポレイテッド | 操作された核内低分子ＲＮＡ（ｓｎＲＮＡ）を含む組成物および方法 |
| WO2025225719A1 (en) * | 2024-04-26 | 2025-10-30 | Chugai Seiyaku Kabushiki Kaisha | Treatment and prevention of muscular dystrophy and its related diseases or disorders with satralizumab |
Family Cites Families (25)
| 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 |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| 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 |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | 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 |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved 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 |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| JP2001514845A (ja) | 1997-09-05 | 2001-09-18 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法 |
| 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 |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| AU2012286994B2 (en) | 2011-07-25 | 2017-08-10 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of DUX4 |
| 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 |
| JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 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プログラム |
| 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 |
| AU2017222653B2 (en) * | 2016-02-26 | 2023-06-01 | Research Institute At Nationwide Children's Hospital | Recombinant virus products and methods for inducing DUX4 exon skipping |
| WO2020028134A1 (en) * | 2018-07-30 | 2020-02-06 | Fred Hutchinson Cancer Research Center | Methods and compositions for treating cancer |
| AU2019419494B2 (en) * | 2018-12-31 | 2026-03-26 | Research Institute At Nationwide Children's Hospital | DUX4 RNA silencing using RNA targeting CRISPR-Cas13b |
-
2021
- 2021-11-30 IL IL303230A patent/IL303230A/en unknown
- 2021-11-30 WO PCT/US2021/061109 patent/WO2022115745A1/en not_active Ceased
- 2021-11-30 EP EP21830568.8A patent/EP4251752A1/en active Pending
- 2021-11-30 JP JP2023532456A patent/JP2023551279A/ja active Pending
- 2021-11-30 KR KR1020237022057A patent/KR20230128470A/ko active Pending
- 2021-11-30 US US18/038,878 patent/US20240026356A1/en active Pending
- 2021-11-30 AU AU2021385595A patent/AU2021385595A1/en active Pending
- 2021-11-30 CA CA3203585A patent/CA3203585A1/en active Pending