JPWO2020023857A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020023857A5 JPWO2020023857A5 JP2021527020A JP2021527020A JPWO2020023857A5 JP WO2020023857 A5 JPWO2020023857 A5 JP WO2020023857A5 JP 2021527020 A JP2021527020 A JP 2021527020A JP 2021527020 A JP2021527020 A JP 2021527020A JP WO2020023857 A5 JPWO2020023857 A5 JP WO2020023857A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide sequence
- seq
- hgalns
- raav
- encoding
- 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 (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862711238P | 2018-07-27 | 2018-07-27 | |
| US62/711,238 | 2018-07-27 | ||
| US201862756880P | 2018-11-07 | 2018-11-07 | |
| US62/756,880 | 2018-11-07 | ||
| US201962799834P | 2019-02-01 | 2019-02-01 | |
| US62/799,834 | 2019-02-01 | ||
| PCT/US2019/043631 WO2020023857A1 (en) | 2018-07-27 | 2019-07-26 | Treatment of mucopolysaccharidosis iva |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021531044A JP2021531044A (ja) | 2021-11-18 |
| JP2021531044A5 JP2021531044A5 (https=) | 2022-08-02 |
| JPWO2020023857A5 true JPWO2020023857A5 (https=) | 2022-08-02 |
Family
ID=69180546
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021527020A Pending JP2021531044A (ja) | 2018-07-27 | 2019-07-26 | ムコ多糖症ｉｖａ型の治療 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US12247213B2 (https=) |
| EP (1) | EP3829619A4 (https=) |
| JP (1) | JP2021531044A (https=) |
| KR (1) | KR20210040984A (https=) |
| AU (1) | AU2019312301A1 (https=) |
| BR (1) | BR112021001498A2 (https=) |
| CA (1) | CA3107800A1 (https=) |
| CO (1) | CO2021002505A2 (https=) |
| IL (1) | IL280343A (https=) |
| SG (1) | SG11202100781VA (https=) |
| TW (1) | TW202039857A (https=) |
| WO (1) | WO2020023857A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW202039857A (zh) | 2018-07-27 | 2020-11-01 | 美商銳進科斯生物股份有限公司 | 黏多醣病iva之治療 |
| US12290574B2 (en) | 2018-08-24 | 2025-05-06 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| JP2022544004A (ja) * | 2019-07-26 | 2022-10-17 | リジェネックスバイオ インコーポレイテッド | 操作された核酸調節エレメントならびにその使用方法 |
| AR121201A1 (es) * | 2020-01-29 | 2022-04-27 | Regenxbio Inc | Tratamiento de mucopolisacaridosis iva |
| EP4423285A1 (en) * | 2021-10-28 | 2024-09-04 | RegenxBio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7863238B2 (en) * | 2004-06-10 | 2011-01-04 | Saint Louis University | Proteins with an attached short peptide of acidic amino acids |
| US7972593B2 (en) | 2004-06-10 | 2011-07-05 | Saint Louis University | Delivery of therapeutic agents to the bone |
| US20070207139A1 (en) | 2004-06-10 | 2007-09-06 | Saint Louis University, A Non-Profit Organization | Enhancing the effect of therapeutic proteins on the central nervous system |
| ES2429142T3 (es) | 2009-01-30 | 2013-11-13 | Uniqure Ip B.V. | Agentes terapéuticos de alanina-glioxilato aminotransferasa |
| US20140271550A1 (en) | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| CN120174012A (zh) * | 2013-10-24 | 2025-06-20 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| EP3101125A1 (en) * | 2015-06-05 | 2016-12-07 | Laboratorios Del Dr. Esteve, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
| HK1257024A1 (zh) | 2015-08-31 | 2019-10-11 | 宾夕法尼亚大学 | 用於治疗伴侣动物的aav-epo |
| UA128788C2 (uk) | 2018-05-30 | 2024-10-23 | Естеве Фармасьютікалз, С.А. | Вектор аденоасоційованого вірусу для лікування мукополісахаридозу типу iva |
| TW202039857A (zh) | 2018-07-27 | 2020-11-01 | 美商銳進科斯生物股份有限公司 | 黏多醣病iva之治療 |
| AR121201A1 (es) | 2020-01-29 | 2022-04-27 | Regenxbio Inc | Tratamiento de mucopolisacaridosis iva |
-
2019
- 2019-07-26 TW TW108126686A patent/TW202039857A/zh unknown
- 2019-07-26 SG SG11202100781VA patent/SG11202100781VA/en unknown
- 2019-07-26 CA CA3107800A patent/CA3107800A1/en active Pending
- 2019-07-26 EP EP19841900.4A patent/EP3829619A4/en not_active Withdrawn
- 2019-07-26 KR KR1020217004895A patent/KR20210040984A/ko not_active Withdrawn
- 2019-07-26 WO PCT/US2019/043631 patent/WO2020023857A1/en not_active Ceased
- 2019-07-26 BR BR112021001498-5A patent/BR112021001498A2/pt not_active IP Right Cessation
- 2019-07-26 US US17/263,049 patent/US12247213B2/en active Active
- 2019-07-26 JP JP2021527020A patent/JP2021531044A/ja active Pending
- 2019-07-26 AU AU2019312301A patent/AU2019312301A1/en not_active Abandoned
-
2021
- 2021-01-21 IL IL280343A patent/IL280343A/en unknown
- 2021-02-25 CO CONC2021/0002505A patent/CO2021002505A2/es unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7796702B2 (ja) | スペーサーを含む核酸分子およびその使用の方法 | |
| JP4860886B2 (ja) | 二本鎖パルボウイルスベクター | |
| JP6174318B2 (ja) | アデノ随伴ウイルス構築物を含んでなるコンダクティング気道細胞を標的とするための組成物 | |
| Tal | Adeno-associated virus-based vectors in gene therapy | |
| JP2021531044A5 (https=) | ||
| JP2020511127A5 (https=) | ||
| US20020045264A1 (en) | Production of chimeric capsid vectors | |
| Flotte et al. | Adeno-associated virus: a ubiquitous commensal of mammals | |
| WO2004020600A2 (en) | Modified aav | |
| JP7598333B2 (ja) | 組換えウイルスベクターの製造方法 | |
| CA2469053A1 (en) | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor | |
| CN114981444A (zh) | Aav5的分离的修饰的vp1衣壳蛋白 | |
| JP2021517168A5 (https=) | ||
| JPWO2020023857A5 (https=) | ||
| Xiao et al. | Adeno-associated virus (AAV) vectors for gene transfer | |
| Smith et al. | Transposase-mediated construction of an integrated adeno-associated virus type 5 helper plasmid | |
| JPWO2019173756A5 (https=) | ||
| JP2001502549A (ja) | ヘルパーウイルス配列を含有するａａｖ ｄｎａ | |
| Zhao et al. | Hot topics in adeno-associated virus as a gene transfer vector | |
| WO2023022634A1 (ru) | Выделенный модифицированный белок vp1 капсида aav9 | |
| CA2983593C (en) | Nucleic acid molecules containing spacers and methods of use thereof | |
| RU2023121263A (ru) | Вектор на основе рнк-содержащего аденоассоциированного вируса (raav) и пути его применения | |
| HK40112375A (zh) | 获得修饰的腺相关病毒衣壳的方法 | |
| Zhou et al. | 90. Evaluation of Novel Gene Transfer Vectors Derived from Infectious Molecular Clones of Primate AAVs | |
| Im | Adenovirus vs AAV Vectors for Gene Delivery: Their Advantages and Disadvantages |