JPWO2019193119A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2019193119A5 JPWO2019193119A5 JP2020554181A JP2020554181A JPWO2019193119A5 JP WO2019193119 A5 JPWO2019193119 A5 JP WO2019193119A5 JP 2020554181 A JP2020554181 A JP 2020554181A JP 2020554181 A JP2020554181 A JP 2020554181A JP WO2019193119 A5 JPWO2019193119 A5 JP WO2019193119A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- sequence
- recombinant
- aav9
- aavrh74
- 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.)
- Granted
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18305399.0 | 2018-04-05 | ||
| EP18305399 | 2018-04-05 | ||
| PCT/EP2019/058560 WO2019193119A1 (en) | 2018-04-05 | 2019-04-04 | HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021520203A JP2021520203A (ja) | 2021-08-19 |
| JPWO2019193119A5 true JPWO2019193119A5 (he) | 2022-04-25 |
| JP7374119B2 JP7374119B2 (ja) | 2023-11-06 |
Family
ID=62063468
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020554181A Active JP7374119B2 (ja) | 2018-04-05 | 2019-04-04 | 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 |
| JP2021558887A Withdrawn JP2022526405A (ja) | 2018-04-05 | 2019-10-04 | 肝向性の低減及び筋形質導入の増大を有するＡＡＶ９とＡＡＶｒｈ７４とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021558887A Withdrawn JP2022526405A (ja) | 2018-04-05 | 2019-10-04 | 肝向性の低減及び筋形質導入の増大を有するＡＡＶ９とＡＡＶｒｈ７４とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型 |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20210107948A1 (he) |
| EP (2) | EP3775173A1 (he) |
| JP (2) | JP7374119B2 (he) |
| KR (2) | KR20210015770A (he) |
| CN (2) | CN112004925A (he) |
| AU (2) | AU2019249890A1 (he) |
| BR (2) | BR112020020223A2 (he) |
| CA (2) | CA3093347A1 (he) |
| IL (2) | IL277740A (he) |
| MA (1) | MA51353B1 (he) |
| TN (1) | TN2020000187A1 (he) |
| WO (2) | WO2019193119A1 (he) |
| ZA (1) | ZA202005422B (he) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3784288A1 (en) * | 2018-04-27 | 2021-03-03 | Universität Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
| US20230270885A2 (en) | 2019-09-19 | 2023-08-31 | Genethon | Gene therapy expression system alleviating cardiac toxicity of fkrp |
| WO2021092514A1 (en) * | 2019-11-08 | 2021-05-14 | The Board Of Regents Of The University Of Texas | Recombinant adeno-associated viral vector for gene delivery |
| WO2021116138A1 (en) * | 2019-12-09 | 2021-06-17 | Ucl Business Ltd | Gene therapy composition and treatment for myh7-linked cardiomyopathy |
| US20230032826A1 (en) | 2019-12-18 | 2023-02-02 | Genethon | Production of recombinant viral vectors from plant hairy roots |
| WO2021229255A1 (en) | 2020-05-14 | 2021-11-18 | Genethon | Tools and method for preventing a a¥ neutralization by antibodies |
| EP3913060A1 (en) | 2020-05-22 | 2021-11-24 | Genethon | Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy |
| JP2023528661A (ja) | 2020-06-09 | 2023-07-05 | ジェネトン | 拡張型心筋症の処置における使用のためのｎｒｆ２活性化剤 |
| WO2021250076A1 (en) | 2020-06-09 | 2021-12-16 | Genethon | Treatment of genetic dilated cardiomyopathies |
| JP2023528662A (ja) | 2020-06-09 | 2023-07-05 | ジェネトン | 拡張型心筋症の処置における使用のためのｃｉｌｐ－１阻害剤 |
| CN115996758A (zh) | 2020-06-19 | 2023-04-21 | 吉尼松公司 | 使sgcg在肌肉和心脏中充分表达的基因治疗表达系统 |
| CA3187635A1 (en) | 2020-07-03 | 2022-01-06 | Genethon | Method for engineering novel hybrid aav capsids through hypervariable regions swapping |
| BR112023000305A2 (pt) | 2020-07-10 | 2023-03-21 | Genethon | Promotor, sistema de expressão e composição farmacêutica |
| JP2023544683A (ja) | 2020-09-10 | 2023-10-25 | ジェネトン | ペプチド改変されたａａｖカプシド |
| WO2022069598A1 (en) | 2020-09-29 | 2022-04-07 | Genethon | Enhancing utrophin expression in cell by inducing mutations within utrophin regulatory elements and therapeutic use thereof |
| JP2024504914A (ja) * | 2020-12-29 | 2024-02-02 | 江▲蘇▼金斯瑞蓬勃生物科技有限公司 | 高分散性ｈｅｋ２９３ｔ細胞株及びそのスクリーニング方法 |
| US20240165271A1 (en) | 2021-03-26 | 2024-05-23 | The Board Of Regents Of The University Of Texas System | Nucleotide editing to reframe dmd transcripts by base editing and prime editing |
| WO2022226301A1 (en) * | 2021-04-23 | 2022-10-27 | The University Of North Carolina At Chapel Hill | Chimeric heart tropic aav capsids |
| CN117897492A (zh) | 2021-08-04 | 2024-04-16 | 吉尼松公司 | 用于肌肉和cns中的基因表达的杂合启动子 |
| CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| WO2023147304A1 (en) | 2022-01-25 | 2023-08-03 | The Trustees Of The University Of Pennsylvania | Aav capsids for improved heart transduction and detargeting of liver |
| CN114657152A (zh) * | 2022-03-31 | 2022-06-24 | 苏州博腾生物制药有限公司 | 一种新型aav血清型及其制备方法 |
| WO2023237731A1 (en) | 2022-06-09 | 2023-12-14 | Genethon | N-terminal truncated gde for the treatment of glycogen storage disease iii |
| WO2023237748A1 (en) | 2022-06-10 | 2023-12-14 | Genethon | Peptide-modified aav capsid with enhanced muscle transduction efficiency |
| WO2024074727A1 (en) | 2022-10-07 | 2024-04-11 | Genethon | Immunotherapy of skeletal myopathies using anti-fap car-t cells |
| WO2024079249A1 (en) | 2022-10-12 | 2024-04-18 | Genethon | Hybrid aav vector enhancing transgene expression in the liver |
| CN116041443B (zh) * | 2022-12-30 | 2023-09-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN116693633B (zh) * | 2023-02-21 | 2023-12-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN117031014A (zh) * | 2023-08-14 | 2023-11-10 | 陕西脉元生物科技有限公司 | 抗matr3自身抗体的试剂在制备检测神经系统自身免疫疾病的产品中的应用及试剂盒 |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP5054975B2 (ja) | 2003-09-30 | 2012-10-24 | ザ・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 | アデノ随伴ウイルス（ａａｖ）の同源系統群、配列、それらを含有するベクターおよびそれらの用途 |
| JP5702519B2 (ja) | 2005-04-07 | 2015-04-15 | ザ・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 | Ａａｖベクターの機能を上げる方法 |
| ES2724800T3 (es) | 2011-02-17 | 2019-09-16 | Univ Pennsylvania | Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9 |
| ES2752191T3 (es) | 2012-02-17 | 2020-04-03 | Childrens Hospital Philadelphia | Composiciones y métodos con vectores de AAV para la transferencia de genes a células, órganos y tejidos |
| IN2014DN08812A (he) | 2012-04-18 | 2015-05-22 | Philadelphia Children Hospital | |
| EP2660325A3 (en) | 2012-05-02 | 2014-02-12 | Christian Medical College | AAV vectors and corresponding nucleotide sequences and methods |
| CN105612253A (zh) * | 2013-03-15 | 2016-05-25 | 费城儿童医院 | 含有填充者/填充物多核苷酸序列的载体及其制备方法 |
| AU2014253730B2 (en) * | 2013-04-20 | 2018-09-13 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus delivery of exon 2-targeted U7snRNA polynucleotide constructs |
| US11408899B2 (en) * | 2013-07-12 | 2022-08-09 | The Children's Hospital Of Philadelphia | AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies |
| KR102380265B1 (ko) | 2013-07-22 | 2022-03-29 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| PT3198018T (pt) | 2014-09-24 | 2021-02-24 | Hope City | Variantes dos vetores de vírus adenoassociados para edição do genoma de alta eficácia e métodos da mesma |
| WO2017096164A1 (en) | 2015-12-02 | 2017-06-08 | The Board Of Trustees Of The Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
| IL305149A (he) * | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | חלבוני קפסיד וירוס קשור–אדנו חדשים |
| CN112533644A (zh) * | 2018-04-03 | 2021-03-19 | 斯特里迪比奥公司 | 靶向眼组织的病毒载体 |
-
2019
- 2019-04-04 BR BR112020020223-1A patent/BR112020020223A2/pt unknown
- 2019-04-04 JP JP2020554181A patent/JP7374119B2/ja active Active
- 2019-04-04 CA CA3093347A patent/CA3093347A1/en active Pending
- 2019-04-04 US US16/981,086 patent/US20210107948A1/en active Pending
- 2019-04-04 CN CN201980023352.XA patent/CN112004925A/zh active Pending
- 2019-04-04 MA MA51353A patent/MA51353B1/fr unknown
- 2019-04-04 WO PCT/EP2019/058560 patent/WO2019193119A1/en active Application Filing
- 2019-04-04 KR KR1020207032024A patent/KR20210015770A/ko unknown
- 2019-04-04 AU AU2019249890A patent/AU2019249890A1/en active Pending
- 2019-04-04 TN TNP/2020/000187A patent/TN2020000187A1/en unknown
- 2019-04-04 EP EP19714230.0A patent/EP3775173A1/en active Pending
- 2019-10-04 AU AU2019439673A patent/AU2019439673A1/en not_active Withdrawn
- 2019-10-04 WO PCT/EP2019/076958 patent/WO2020200499A1/en unknown
- 2019-10-04 BR BR112021019821A patent/BR112021019821A2/pt not_active Application Discontinuation
- 2019-10-04 US US17/600,715 patent/US20220228167A1/en not_active Abandoned
- 2019-10-04 CN CN201980095204.9A patent/CN113795574A/zh active Pending
- 2019-10-04 KR KR1020217031502A patent/KR20210153047A/ko not_active Application Discontinuation
- 2019-10-04 CA CA3134183A patent/CA3134183A1/en not_active Withdrawn
- 2019-10-04 JP JP2021558887A patent/JP2022526405A/ja not_active Withdrawn
- 2019-10-04 EP EP19782603.5A patent/EP3947653A1/en not_active Withdrawn
-
2020
- 2020-08-31 ZA ZA2020/05422A patent/ZA202005422B/en unknown
- 2020-10-01 IL IL277740A patent/IL277740A/he unknown
-
2021
- 2021-10-03 IL IL286926A patent/IL286926A/he unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JPWO2019193119A5 (he) | ||
| 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 | |
| RU2020113681A (ru) | Капсиды вариантов аденоассоциированных вирусов и методы их применения | |
| M Mitchell et al. | AAV's anatomy: roadmap for optimizing vectors for translational success | |
| TW202102526A (zh) | 重組腺相關病毒及其用途 | |
| JP2020534788A5 (he) | ||
| FI3445773T3 (fi) | Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä | |
| JP2020528734A5 (he) | ||
| JP2017536116A5 (he) | ||
| JP2020510428A5 (he) | ||
| JP2021508241A5 (he) | ||
| JP2018510620A5 (he) | ||
| JP2023088902A5 (he) | ||
| JP2020512822A5 (he) | ||
| JP2017529395A5 (he) | ||
| JP2019524090A5 (he) | ||
| WO2023124740A1 (zh) | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 | |
| KR20220004072A (ko) | 뇌에 지향성을 가지는 aav 돌연변이체 | |
| JP2023126658A5 (he) | ||
| WO2022222869A1 (zh) | 重组腺相关病毒及其应用 | |
| IL293431A (he) | קסטות טרנסגן שמיועדות לבטא גן mecp2 אנושי | |
| RU2019126509A (ru) | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе | |
| JP2023509895A (ja) | 肝臓特異的プロモータ及びその使用 | |
| JP2020533968A5 (he) | ||
| IL298772A (he) | חומצת גרעין שעברה אופטימיזצית קידוד המקודדת לחלבון smn1 ושימוש בה |