JPWO2020227166A5 - - Google Patents
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- Publication number
- JPWO2020227166A5 JPWO2020227166A5 JP2021564955A JP2021564955A JPWO2020227166A5 JP WO2020227166 A5 JPWO2020227166 A5 JP WO2020227166A5 JP 2021564955 A JP2021564955 A JP 2021564955A JP 2021564955 A JP2021564955 A JP 2021564955A JP WO2020227166 A5 JPWO2020227166 A5 JP WO2020227166A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- raav
- harsa
- seq
- sequence
- 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 |
|---|---|---|---|
| US201962843091P | 2019-05-03 | 2019-05-03 | |
| US62/843,091 | 2019-05-03 | ||
| PCT/US2020/031207 WO2020227166A1 (en) | 2019-05-03 | 2020-05-02 | Compositions useful in treatment of metachromatic leukodystrophy |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022531861A JP2022531861A (ja) | 2022-07-12 |
| JP2022531861A5 JP2022531861A5 (https=) | 2023-05-12 |
| JPWO2020227166A5 true JPWO2020227166A5 (https=) | 2023-05-12 |
| JP7715640B2 JP7715640B2 (ja) | 2025-07-30 |
Family
ID=73050896
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021564955A Active JP7715640B2 (ja) | 2019-05-03 | 2020-05-02 | 異染性白質ジストロフィーの治療に有用な組成物 |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US12338451B2 (https=) |
| EP (1) | EP3962454A4 (https=) |
| JP (1) | JP7715640B2 (https=) |
| KR (1) | KR20220020261A (https=) |
| CN (1) | CN114072129A (https=) |
| AR (1) | AR118850A1 (https=) |
| AU (1) | AU2020270421A1 (https=) |
| BR (1) | BR112021021908A2 (https=) |
| CA (1) | CA3135539A1 (https=) |
| CL (1) | CL2021002881A1 (https=) |
| CO (1) | CO2021016323A2 (https=) |
| IL (1) | IL287715A (https=) |
| MX (1) | MX2021013420A (https=) |
| PE (1) | PE20220930A1 (https=) |
| SG (1) | SG11202111867XA (https=) |
| TW (1) | TW202108760A (https=) |
| WO (1) | WO2020227166A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3980447A4 (en) * | 2019-06-10 | 2023-07-26 | Homology Medicines, Inc. | ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR ARSA GENE TRANSFER AND METHODS OF USE THEREOF |
| US20230304034A1 (en) * | 2020-05-12 | 2023-09-28 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| KR20230170022A (ko) * | 2021-04-12 | 2023-12-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물 |
| RU2769577C1 (ru) * | 2021-06-01 | 2022-04-04 | федеральное государственное автономное образовательное учреждение высшего образования "Казанский (Приволжский) федеральный университет" (ФГАОУ ВО КФУ) | Препарат для лечения метахроматической лейкодистрофии и способ ее лечения |
| IL308787A (en) * | 2021-06-08 | 2024-01-01 | Univ Pennsylvania | Recombinant adeno-associated viruses for Leish-Nihan disorders and their uses |
| US20250177495A1 (en) * | 2022-01-10 | 2025-06-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| WO2023196863A1 (en) * | 2022-04-05 | 2023-10-12 | Astellas Gene Therapies, Inc. | Adeno-associated virus potency assay and uses thereof |
| AU2023273809A1 (en) * | 2022-05-16 | 2025-01-02 | Genzyme Corporation | Methods of treating metachromatic leukodystrophy |
| WO2024036250A2 (en) * | 2022-08-10 | 2024-02-15 | Homology Medicines, Inc. | Adeno-associated virus compositions for arsa gene transfer and methods of use thereof |
| WO2024079361A1 (en) * | 2022-10-14 | 2024-04-18 | Neuway Pharma Gmbh | A PROTEIN- OR PEPTIDE-BASED CAPSULE (PPC), PREFERABLY A VLP, LOADED WITH A MESSENGER RNA (mRNA) AND A METHOD OF PRODUCTION AND PURIFICATION THEREOF |
| CN118389675B (zh) * | 2024-05-27 | 2025-06-27 | 南方医科大学珠江医院 | 芳香族氨基酸脱羧酶作为标志物在肝性脑病的检测产品中的应用 |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| WO1998046728A1 (en) | 1997-04-14 | 1998-10-22 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
| AU1458200A (en) | 1998-10-31 | 2000-05-22 | University Of Medicine And Dentistry Of New Jersey | A myeloid precursor cell useful for gene therapy and for modulation of immune responses |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| PT1916258E (pt) | 1999-08-09 | 2014-07-29 | Genzyme Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| AU2002317700A1 (en) * | 2001-06-07 | 2002-12-16 | Hemebiotech A/S | Production of recombinant human arylsulfatase a |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| US8536315B2 (en) * | 2004-01-30 | 2013-09-17 | Shire Pharmaceuticals Ireland Limited | Production and purification of recombinant arylsulftase |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| NZ605873A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of arylsulfatase a |
| AU2012346448B2 (en) * | 2011-12-02 | 2017-09-14 | Armagen, Inc. | Methods and compositions for increasing arylsulfatase A activity in the CNS |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| AU2013243948A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| CN105377039A (zh) * | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| BR112017005892A2 (pt) | 2014-09-24 | 2017-12-12 | Hope City | variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma |
| HK1256341A1 (zh) | 2015-05-15 | 2019-09-20 | 明尼苏达大学董事会 | 用於治疗性递送到中枢神经系统的腺相关物 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| EP3416678A1 (en) * | 2016-02-17 | 2018-12-26 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of arylsulfatase a |
| JP7455579B2 (ja) * | 2017-02-28 | 2024-03-26 | 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 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| AU2018329741B2 (en) * | 2017-09-08 | 2025-02-20 | Poseida Therapeutics, Inc. | Compositions and methods for chimeric ligand receptor (CLR)-mediated conditional gene expression |
| WO2020132455A1 (en) | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
-
2020
- 2020-05-02 BR BR112021021908A patent/BR112021021908A2/pt unknown
- 2020-05-02 CN CN202080048744.4A patent/CN114072129A/zh active Pending
- 2020-05-02 PE PE2021001824A patent/PE20220930A1/es unknown
- 2020-05-02 AU AU2020270421A patent/AU2020270421A1/en active Pending
- 2020-05-02 KR KR1020217039381A patent/KR20220020261A/ko active Pending
- 2020-05-02 SG SG11202111867XA patent/SG11202111867XA/en unknown
- 2020-05-02 JP JP2021564955A patent/JP7715640B2/ja active Active
- 2020-05-02 MX MX2021013420A patent/MX2021013420A/es unknown
- 2020-05-02 WO PCT/US2020/031207 patent/WO2020227166A1/en not_active Ceased
- 2020-05-02 US US17/608,493 patent/US12338451B2/en active Active
- 2020-05-02 EP EP20803017.1A patent/EP3962454A4/en active Pending
- 2020-05-02 CA CA3135539A patent/CA3135539A1/en active Pending
- 2020-05-04 TW TW109114778A patent/TW202108760A/zh unknown
- 2020-05-04 AR ARP200101259A patent/AR118850A1/es unknown
-
2021
- 2021-10-31 IL IL287715A patent/IL287715A/en unknown
- 2021-11-03 CL CL2021002881A patent/CL2021002881A1/es unknown
- 2021-11-30 CO CONC2021/0016323A patent/CO2021016323A2/es unknown
-
2025
- 2025-05-07 US US19/201,601 patent/US20250313859A1/en active Pending
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