JP2018517414A5 - - Google Patents
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- Publication number
- JP2018517414A5 JP2018517414A5 JP2017563119A JP2017563119A JP2018517414A5 JP 2018517414 A5 JP2018517414 A5 JP 2018517414A5 JP 2017563119 A JP2017563119 A JP 2017563119A JP 2017563119 A JP2017563119 A JP 2017563119A JP 2018517414 A5 JP2018517414 A5 JP 2018517414A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- seq
- plasmid
- nucleotide sequence
- cag
- 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
- 239000013598 vector Substances 0.000 claims 14
- 239000013612 plasmid Substances 0.000 claims 10
- 239000002773 nucleotide Substances 0.000 claims 9
- 125000003729 nucleotide group Chemical group 0.000 claims 9
- 108090000623 proteins and genes Proteins 0.000 claims 5
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims 2
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 101150044789 Cap gene Proteins 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- 239000012531 culture fluid Substances 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 230000029087 digestion Effects 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 108010030074 endodeoxyribonuclease MluI Proteins 0.000 claims 1
- 239000013604 expression vector Substances 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 1
- 101150066583 rep gene Proteins 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP15382297.8A EP3101125A1 (en) | 2015-06-05 | 2015-06-05 | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
| EP15382297.8 | 2015-06-05 | ||
| PCT/EP2016/062655 WO2016193431A1 (en) | 2015-06-05 | 2016-06-03 | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018517414A JP2018517414A (ja) | 2018-07-05 |
| JP2018517414A5 true JP2018517414A5 (OSRAM) | 2019-07-04 |
| JP6850736B2 JP6850736B2 (ja) | 2021-03-31 |
Family
ID=53398018
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017563119A Active JP6850736B2 (ja) | 2015-06-05 | 2016-06-03 | ムコ多糖症治療用のアデノ随伴ウイルスベクター |
Country Status (21)
| Country | Link |
|---|---|
| US (1) | US10617771B2 (OSRAM) |
| EP (2) | EP3101125A1 (OSRAM) |
| JP (1) | JP6850736B2 (OSRAM) |
| KR (1) | KR102609858B1 (OSRAM) |
| CN (1) | CN108138154B (OSRAM) |
| AU (1) | AU2016273343B2 (OSRAM) |
| CA (1) | CA2988201C (OSRAM) |
| CL (1) | CL2017003096A1 (OSRAM) |
| CO (1) | CO2017012562A2 (OSRAM) |
| ES (1) | ES2790834T3 (OSRAM) |
| IL (1) | IL255939B (OSRAM) |
| MX (1) | MX386615B (OSRAM) |
| MY (1) | MY184586A (OSRAM) |
| NZ (1) | NZ738018A (OSRAM) |
| PH (1) | PH12017502192B1 (OSRAM) |
| PT (1) | PT3303576T (OSRAM) |
| RU (1) | RU2744593C2 (OSRAM) |
| TN (1) | TN2017000506A1 (OSRAM) |
| UA (1) | UA125373C2 (OSRAM) |
| WO (1) | WO2016193431A1 (OSRAM) |
| ZA (1) | ZA201708039B (OSRAM) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2605798A1 (en) | 2010-07-12 | 2013-06-26 | Universidad Autònoma de Barcelona | Gene therapy composition for use in diabetes treatment |
| EP2692868A1 (en) | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
| EP3194430A1 (en) | 2014-09-16 | 2017-07-26 | Universitat Autònoma De Barcelona | Adeno-associated viral vectors for the gene therapy of metabolic diseases |
| WO2016087678A1 (en) | 2014-12-05 | 2016-06-09 | Universitat Autònoma De Barcelona | Viral vectors for the treatment of diabetes |
| EP3242945B1 (en) | 2015-01-07 | 2021-09-01 | Universitat Autònoma de Barcelona | Single-vector gene construct comprising insulin and glucokinase genes |
| IL262211B2 (en) | 2016-04-15 | 2024-01-01 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| UY37679A (es) * | 2017-04-14 | 2018-11-30 | Regenxbio Inc | Tratamiento de mucopolisacaridosis ii con iduronato-2-sulfatasa (ids) humana recombinante producida por células neurales o gliales humanas |
| TWI835747B (zh) * | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| CN108795985A (zh) * | 2018-05-31 | 2018-11-13 | 深圳市免疫基因治疗研究院 | 一种黏多糖贮积症慢病毒载体、慢病毒及其制备方法和应用 |
| CN108715867A (zh) * | 2018-05-31 | 2018-10-30 | 深圳市免疫基因治疗研究院 | 一种Sanfilippo B综合症慢病毒载体、慢病毒及其制备方法和应用 |
| EP3829619A4 (en) * | 2018-07-27 | 2022-05-18 | REGENXBIO Inc. | TREATMENT OF IVA MUCOPOLYSACCHARIDOSIS |
| US12201095B2 (en) * | 2018-11-15 | 2025-01-21 | Esteve Pharmaceuticals, S.A. | Animal model of mucopolysaccharidoses type IVA |
| TW202039856A (zh) * | 2019-03-28 | 2020-11-01 | 西班牙商艾斯提夫製藥股份有限公司 | 製造重組病毒載體之方法 |
| EP3914720B9 (en) | 2019-07-10 | 2023-09-27 | Masonic Medical Research Laboratory | Vgll4 with ucp-1 cis-regulatory element and method of use thereof |
| IL293684A (en) * | 2019-12-10 | 2022-08-01 | Takeda Pharmaceuticals Co | Adeno associated virus vectors for the treatment of hunter disease |
| BR112022019182A2 (pt) * | 2020-04-06 | 2022-11-29 | Homology Medicines Inc | Composições de vírus adeno-associadas para transferência do gene ids e métodos de uso da mesma |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| CN114480454B (zh) * | 2020-10-27 | 2024-03-29 | 华东理工大学 | 用于治疗粘多糖贮积症ⅱ型的腺相关病毒载体、药物及其应用 |
| US20240117328A1 (en) | 2021-02-10 | 2024-04-11 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) |
| WO2022221421A2 (en) * | 2021-04-13 | 2022-10-20 | Capsida, Inc. | Aav compositions with high brain expression for treating mucopolysaccharidosis ii |
| US20240350653A1 (en) * | 2021-08-18 | 2024-10-24 | Joint-Stock Company "Generium" | Targeted delivery of therapeutic enzymes |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR100762945B1 (ko) * | 2004-09-08 | 2007-10-04 | 진동규 | 동물 세포주를 이용한 이듀로네이트-설파타제의 발현 방법및 그 발현을 위한 세포주 |
| BRPI0906948B1 (pt) * | 2008-01-18 | 2023-05-16 | Biomarin Pharmaceutical, Inc | Composições compreendendo uma enzima nacetilgalactosamina-6- sulfatase (galns) recombinante humana e uso das mesmas para tratar mucopolissacaridose tipo iva, síndrome de morquio a, ou deficiência múltipla de sulfatase |
| HRP20190690T1 (hr) * | 2009-10-09 | 2019-06-28 | Armagen, Inc. | Postupci i pripravci za povećanje aktiviteta iduronat-2-sulfataze u centralnom nervnom sustavu |
| JPWO2011108451A1 (ja) * | 2010-03-01 | 2013-06-27 | 日本ケミカルリサーチ株式会社 | 遺伝子ノックアウト細胞を用いた組換え体リソソーム酵素の製造方法 |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| KR101535791B1 (ko) * | 2010-11-12 | 2015-07-10 | 주식회사 녹십자 | 개량형 이듀로네이트-2-설파타제 및 이의 용도 |
| WO2012101671A1 (en) * | 2011-01-25 | 2012-08-02 | Jcr Pharmaceuticals Co., Ltd. | Method for production of human recombinant iduronate 2-sulfatase |
| US10086043B2 (en) * | 2011-04-03 | 2018-10-02 | The General Hospital Corporation | Efficient protein expression in vivo using modified RNA (MOD-RNA) |
| AU2013243949A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of biologics and proteins associated with human disease |
| US9150841B2 (en) * | 2012-06-29 | 2015-10-06 | Shire Human Genetic Therapies, Inc. | Cells for producing recombinant iduronate-2-sulfatase |
| JP6605482B2 (ja) * | 2014-02-19 | 2019-11-13 | バイオアシス テクノロジーズ インコーポレイテッド | Ｐ９７−ｉｄｓ融合タンパク質 |
-
2015
- 2015-06-05 EP EP15382297.8A patent/EP3101125A1/en not_active Withdrawn
-
2016
- 2016-06-03 RU RU2017142006A patent/RU2744593C2/ru active
- 2016-06-03 MX MX2017015725A patent/MX386615B/es unknown
- 2016-06-03 CA CA2988201A patent/CA2988201C/en active Active
- 2016-06-03 WO PCT/EP2016/062655 patent/WO2016193431A1/en not_active Ceased
- 2016-06-03 MY MYPI2017704615A patent/MY184586A/en unknown
- 2016-06-03 NZ NZ738018A patent/NZ738018A/en unknown
- 2016-06-03 US US15/578,366 patent/US10617771B2/en active Active
- 2016-06-03 ES ES16730716T patent/ES2790834T3/es active Active
- 2016-06-03 PH PH1/2017/502192A patent/PH12017502192B1/en unknown
- 2016-06-03 JP JP2017563119A patent/JP6850736B2/ja active Active
- 2016-06-03 EP EP16730716.4A patent/EP3303576B1/en active Active
- 2016-06-03 TN TNP/2017/000506A patent/TN2017000506A1/en unknown
- 2016-06-03 UA UAA201711584A patent/UA125373C2/uk unknown
- 2016-06-03 PT PT167307164T patent/PT3303576T/pt unknown
- 2016-06-03 CN CN201680032931.7A patent/CN108138154B/zh active Active
- 2016-06-03 AU AU2016273343A patent/AU2016273343B2/en not_active Ceased
- 2016-06-03 KR KR1020187000048A patent/KR102609858B1/ko active Active
-
2017
- 2017-11-27 ZA ZA2017/08039A patent/ZA201708039B/en unknown
- 2017-11-27 IL IL255939A patent/IL255939B/en active IP Right Grant
- 2017-12-04 CL CL2017003096A patent/CL2017003096A1/es unknown
- 2017-12-05 CO CONC2017/0012562A patent/CO2017012562A2/es unknown
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