JPWO2020176562A5 - - Google Patents
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- Publication number
- JPWO2020176562A5 JPWO2020176562A5 JP2021549986A JP2021549986A JPWO2020176562A5 JP WO2020176562 A5 JPWO2020176562 A5 JP WO2020176562A5 JP 2021549986 A JP2021549986 A JP 2021549986A JP 2021549986 A JP2021549986 A JP 2021549986A JP WO2020176562 A5 JPWO2020176562 A5 JP WO2020176562A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- seq
- aavhu68
- galactosylceramidase
- composition
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024208739A JP2025032175A (ja) | 2019-02-26 | 2024-11-29 | クラッベ病の治療に有用な組成物 |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962810708P | 2019-02-26 | 2019-02-26 | |
| US62/810,708 | 2019-02-26 | ||
| US201962817482P | 2019-03-12 | 2019-03-12 | |
| US62/817,482 | 2019-03-12 | ||
| US201962877707P | 2019-07-23 | 2019-07-23 | |
| US62/877,707 | 2019-09-26 | ||
| US201962916652P | 2019-10-17 | 2019-10-17 | |
| US62/916,652 | 2019-10-17 | ||
| PCT/US2020/019794 WO2020176562A1 (en) | 2019-02-26 | 2020-02-26 | Compositions useful in treatment of krabbe disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024208739A Division JP2025032175A (ja) | 2019-02-26 | 2024-11-29 | クラッベ病の治療に有用な組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022525848A JP2022525848A (ja) | 2022-05-20 |
| JPWO2020176562A5 true JPWO2020176562A5 (https=) | 2023-02-22 |
| JP2022525848A5 JP2022525848A5 (https=) | 2023-02-22 |
Family
ID=69941503
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549986A Pending JP2022525848A (ja) | 2019-02-26 | 2020-02-26 | クラッベ病の治療に有用な組成物 |
| JP2024208739A Pending JP2025032175A (ja) | 2019-02-26 | 2024-11-29 | クラッベ病の治療に有用な組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024208739A Pending JP2025032175A (ja) | 2019-02-26 | 2024-11-29 | クラッベ病の治療に有用な組成物 |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20220118108A1 (https=) |
| EP (1) | EP3931337A1 (https=) |
| JP (2) | JP2022525848A (https=) |
| KR (1) | KR20210132095A (https=) |
| CN (1) | CN114040980A (https=) |
| AU (1) | AU2020229772A1 (https=) |
| BR (1) | BR112021016566A2 (https=) |
| CA (1) | CA3130055A1 (https=) |
| CL (1) | CL2021002248A1 (https=) |
| CO (1) | CO2021011090A2 (https=) |
| IL (1) | IL285776A (https=) |
| MX (1) | MX2021010266A (https=) |
| PE (1) | PE20212072A1 (https=) |
| SG (1) | SG11202108480UA (https=) |
| TW (1) | TW202045728A (https=) |
| WO (1) | WO2020176562A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3177924A1 (en) * | 2020-05-12 | 2021-11-18 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
| KR20230170022A (ko) * | 2021-04-12 | 2023-12-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물 |
| EP4695389A2 (en) * | 2023-04-10 | 2026-02-18 | M6P Therapeutics, Inc. | Compositions comprising modified human galactocerebrosidase |
Family Cites Families (35)
| 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 |
| 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 |
| 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 |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| 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 |
| 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 |
| PL3252161T3 (pl) * | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| 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 |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| EA030305B1 (ru) * | 2012-08-07 | 2018-07-31 | КЬЕЗИ ФАРМАЧЕУТИЧИ С.п.А. | Животная модель болезни краббе |
| US9719106B2 (en) * | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| 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 |
| US10195746B2 (en) | 2014-09-26 | 2019-02-05 | Teradyne, Inc. | Grasping gripper |
| 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 |
| HK1257801A1 (zh) * | 2015-10-28 | 2019-11-01 | 宾夕法尼亚州大学信托人 | 用於基因治疗的腺伴随病毒载体的鞘内给药 |
| US12599636B2 (en) * | 2017-01-20 | 2026-04-14 | University of Pittsburgh—of the Commonwealth System of Higher Education | Treatment of Krabbe disease with umbilical cord blood transplantion (UCBT) and increased galactocerebrosidase (GALC) expression |
| 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 |
| WO2018160852A1 (en) | 2017-03-03 | 2018-09-07 | Cook Medical Technologies, LLC | Rapid exchange multiple catheter system |
| KR102709597B1 (ko) * | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
-
2020
- 2020-02-26 TW TW109106249A patent/TW202045728A/zh unknown
- 2020-02-26 US US17/433,195 patent/US20220118108A1/en active Pending
- 2020-02-26 PE PE2021001391A patent/PE20212072A1/es unknown
- 2020-02-26 EP EP20713474.3A patent/EP3931337A1/en active Pending
- 2020-02-26 JP JP2021549986A patent/JP2022525848A/ja active Pending
- 2020-02-26 CA CA3130055A patent/CA3130055A1/en active Pending
- 2020-02-26 MX MX2021010266A patent/MX2021010266A/es unknown
- 2020-02-26 BR BR112021016566A patent/BR112021016566A2/pt unknown
- 2020-02-26 WO PCT/US2020/019794 patent/WO2020176562A1/en not_active Ceased
- 2020-02-26 AU AU2020229772A patent/AU2020229772A1/en active Pending
- 2020-02-26 SG SG11202108480UA patent/SG11202108480UA/en unknown
- 2020-02-26 KR KR1020217029570A patent/KR20210132095A/ko active Pending
- 2020-02-26 CN CN202080017113.6A patent/CN114040980A/zh active Pending
-
2021
- 2021-08-22 IL IL285776A patent/IL285776A/en unknown
- 2021-08-24 CO CONC2021/0011090A patent/CO2021011090A2/es unknown
- 2021-08-25 CL CL2021002248A patent/CL2021002248A1/es unknown
-
2024
- 2024-11-29 JP JP2024208739A patent/JP2025032175A/ja active Pending
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