JP2021521851A5 - - Google Patents
Info
- Publication number
- JP2021521851A5 JP2021521851A5 JP2020560355A JP2020560355A JP2021521851A5 JP 2021521851 A5 JP2021521851 A5 JP 2021521851A5 JP 2020560355 A JP2020560355 A JP 2020560355A JP 2020560355 A JP2020560355 A JP 2020560355A JP 2021521851 A5 JP2021521851 A5 JP 2021521851A5
- Authority
- JP
- Japan
- Prior art keywords
- gene therapy
- therapy vector
- vector according
- disease
- seq
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024068431A JP2024119788A (ja) | 2018-04-30 | 2024-04-19 | 遺伝子治療構築物及び使用方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862664741P | 2018-04-30 | 2018-04-30 | |
| US62/664,741 | 2018-04-30 | ||
| US201862688640P | 2018-06-22 | 2018-06-22 | |
| US62/688,640 | 2018-06-22 | ||
| US201862744068P | 2018-10-10 | 2018-10-10 | |
| US62/744,068 | 2018-10-10 | ||
| PCT/US2019/030076 WO2019213180A1 (en) | 2018-04-30 | 2019-04-30 | Gene therapy constructs and methods of use |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024068431A Division JP2024119788A (ja) | 2018-04-30 | 2024-04-19 | 遺伝子治療構築物及び使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021521851A JP2021521851A (ja) | 2021-08-30 |
| JP2021521851A5 true JP2021521851A5 (https=) | 2022-05-11 |
| JP7749322B2 JP7749322B2 (ja) | 2025-10-06 |
Family
ID=66669057
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020560355A Active JP7749322B2 (ja) | 2018-04-30 | 2019-04-30 | 遺伝子治療構築物及び使用方法 |
| JP2024068431A Withdrawn JP2024119788A (ja) | 2018-04-30 | 2024-04-19 | 遺伝子治療構築物及び使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024068431A Withdrawn JP2024119788A (ja) | 2018-04-30 | 2024-04-19 | 遺伝子治療構築物及び使用方法 |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US10874750B2 (https=) |
| EP (1) | EP3788064A1 (https=) |
| JP (2) | JP7749322B2 (https=) |
| KR (1) | KR20210005154A (https=) |
| CN (1) | CN112513071A (https=) |
| AU (1) | AU2019261982A1 (https=) |
| BR (1) | BR112020021962A2 (https=) |
| CA (1) | CA3098674A1 (https=) |
| CL (1) | CL2020002809A1 (https=) |
| CO (1) | CO2020014729A2 (https=) |
| MX (1) | MX2020011514A (https=) |
| PH (1) | PH12020551818A1 (https=) |
| SG (1) | SG11202010533WA (https=) |
| TW (2) | TWI808169B (https=) |
| WO (1) | WO2019213180A1 (https=) |
| ZA (1) | ZA202006905B (https=) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112020021962A2 (pt) * | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| CN113631182B (zh) | 2018-10-10 | 2025-03-21 | 阿米库斯治疗学公司 | 二硫键稳定的多肽组合物和使用方法 |
| KR20220077921A (ko) * | 2019-10-10 | 2022-06-09 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 변이체 igf2 작제물 |
| KR20220098384A (ko) * | 2019-11-19 | 2022-07-12 | 아스클레피오스 바이오파마슈티컬, 인크. | 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스 |
| CN115379850A (zh) * | 2019-12-24 | 2022-11-22 | 裘美娜治疗公司 | 再生性多肽及其用途 |
| JP7443761B2 (ja) | 2019-12-25 | 2024-03-06 | 東洋紡エムシー株式会社 | 中空糸膜モジュール |
| WO2021231863A1 (en) * | 2020-05-14 | 2021-11-18 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of pompe disease |
| WO2022026410A2 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
| CN111733174B (zh) * | 2020-08-07 | 2021-02-09 | 北京大学第三医院（北京大学第三临床医学院） | 一种分离的核酸分子及其用途 |
| US20220119549A1 (en) * | 2020-10-15 | 2022-04-21 | Tavotek Biotherapeutics (Hong Kong) Limited | Shielded biologics with masking domains to shield antigen binding capability of biologics and uses thereof |
| WO2022104186A1 (en) * | 2020-11-16 | 2022-05-19 | Tavotek Biotherapeutics (Hong Kong) Limited | A shielded and homing bispecific antibody that simultaneously inhibits angiogenic pathway targets and her2 family proteins and uses thereof |
| IL303239A (en) | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | Compositions and their uses for the treatment of Engelmann syndrome |
| WO2022271466A1 (en) | 2021-06-21 | 2022-12-29 | Juvena Therapeutics, Inc. | Regenerative polypeptides and uses thereof |
| WO2023086939A1 (en) * | 2021-11-12 | 2023-05-19 | Amicus Therapeutics, Inc. | Compositions and methods for treating mucopolysaccharidosis iiia |
| US20250049955A1 (en) | 2021-11-17 | 2025-02-13 | Voyager Therapeutics, Inc. | Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
| WO2024151982A1 (en) | 2023-01-13 | 2024-07-18 | Amicus Therapeutics, Inc. | Gene therapy constructs for the treatment of pompe disease |
| AU2023427408A1 (en) | 2023-02-02 | 2025-09-04 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| WO2024226761A2 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| WO2026006281A1 (en) * | 2024-06-24 | 2026-01-02 | Remedium Bio, Inc. | A system for delivering genetic material subcutaneously with elements enabling subsequent post-treatment identification, localization, quantification, evaluation, or targeting for up- and/or down-titration |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6087164A (en) * | 1997-10-03 | 2000-07-11 | Yissum Research Development Company Of The Hebrew University Of Jerusalem | Methods and compositions for inducing tumor-specific cytotoxicity |
| HK1047109A1 (zh) | 1999-10-15 | 2003-02-07 | University Of Massachusetts | 作为指定基因干预工具的rna干预轨迹基因 |
| US6326193B1 (en) | 1999-11-05 | 2001-12-04 | Cambria Biosciences, Llc | Insect control agent |
| EP1118334A1 (en) | 2000-01-11 | 2001-07-25 | Aventis Behring Gesellschaft mit beschränkter Haftung | Method for the production of conjugates and uses thereof for the prevention and treatment of allergic reactions and autoimmune diseases |
| AU2001275474A1 (en) | 2000-06-12 | 2001-12-24 | Akkadix Corporation | Materials and methods for the control of nematodes |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| WO2002087510A2 (en) | 2001-04-30 | 2002-11-07 | Symbiontics, Inc. | Subcellular targeting of therapeutic proteins |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| AU2002347910A1 (en) * | 2001-10-16 | 2003-04-28 | Symbiontics Inc. | Methods and compositions for targeting proteins across the blood brain barrier |
| DE10237639A1 (de) * | 2002-08-13 | 2004-02-26 | Martin-Luther-Universität Halle-Wittenberg | Verfahren zur Herstellung von virusanalogen Partikeln durch in vitro-Assemblierung von Kapsomeren oder von Kapsomeren, an die biologisch aktive Makromoleküle gebunden sind, bei gleichzeitiger Verpackung der Makromoleküle |
| ES2371913T3 (es) | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
| EP1620133B1 (en) * | 2003-05-01 | 2015-12-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| GB0314856D0 (en) * | 2003-06-25 | 2003-07-30 | Unitargeting Res As | Protein expression system |
| US7355018B2 (en) | 2003-09-30 | 2008-04-08 | Regeneron Pharmaceuticals, Inc. | Modified IGF1 polypeptides with increased stability and potency |
| CA2553955C (en) | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| ES2242542B1 (es) * | 2004-04-30 | 2006-12-16 | Consejo Superior De Investigaciones Cientificas | Procedimiento para la produccion en levaduras de capsidas virales vacias compuestas por proteinas derivadas de pvp2 del virus causante de la enfermedad de la bursitis infecciosa (ibdv). |
| US8889127B2 (en) | 2004-07-01 | 2014-11-18 | Icahn School Of Medicine At Mount Sinai | Targeted protein replacement for the treatment of lysosomal storage disorders |
| CN1903374A (zh) * | 2005-07-27 | 2007-01-31 | 中国人民解放军军事医学科学院生物医学分析中心 | 大肠杆菌在制备肿瘤诊断或治疗药物中的用途 |
| EP2099523A2 (en) | 2006-11-13 | 2009-09-16 | ZyStor Therapeutics , Inc. | Methods for treating pompe disease |
| JP5627571B2 (ja) | 2008-05-07 | 2014-11-19 | バイオマリン ファーマシューティカル インコーポレイテッド | リソソーム標的化ペプチドおよびその使用 |
| ES3000111T3 (en) | 2009-02-13 | 2025-02-27 | Immunomedics Inc | Intermediates for preparing conjugates with an intracellularly-cleavable linkage |
| ES2829199T3 (es) * | 2010-11-22 | 2021-05-31 | Amicus Therapeutics Inc | Nuevas secuencias de señalización para mejorar la expresión y segregación de proteínas de enzimas recombinantes y otras proteínas |
| RU2013125923A (ru) * | 2010-11-30 | 2015-01-10 | Орфазиме Апс | СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70 |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| CN103443273B (zh) | 2011-03-16 | 2016-10-26 | 天野酶制品株式会社 | 修饰型α-葡萄糖苷酶及其用途 |
| HRP20180291T1 (hr) | 2011-05-27 | 2018-03-23 | Amicus Therapeutics, Inc. | Postupci za sparivanje ciljajućeg peptida na rekombinantne lizosomalne enzime za poboljšano liječenje lizosomskih bolesti nakupljanja |
| US9387236B2 (en) * | 2011-06-10 | 2016-07-12 | Prothera Inc. | Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases |
| ES2729997T3 (es) * | 2012-11-27 | 2019-11-07 | Biomarin Pharm Inc | Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas |
| AU2014228231B2 (en) * | 2013-03-15 | 2019-02-21 | Amicus Therapeutics, Inc. | Chemical crosslinkers |
| CN105745326A (zh) | 2013-10-24 | 2016-07-06 | 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 |
| WO2015085238A1 (en) | 2013-12-05 | 2015-06-11 | The Regents Of The University Of California, A California Corporation | Inhibitors of lpxc |
| BR112020021962A2 (pt) | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| KR20220077921A (ko) * | 2019-10-10 | 2022-06-09 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 변이체 igf2 작제물 |
-
2019
- 2019-04-30 BR BR112020021962-2A patent/BR112020021962A2/pt not_active IP Right Cessation
- 2019-04-30 EP EP19727170.3A patent/EP3788064A1/en active Pending
- 2019-04-30 KR KR1020207033891A patent/KR20210005154A/ko not_active Ceased
- 2019-04-30 WO PCT/US2019/030076 patent/WO2019213180A1/en not_active Ceased
- 2019-04-30 JP JP2020560355A patent/JP7749322B2/ja active Active
- 2019-04-30 MX MX2020011514A patent/MX2020011514A/es unknown
- 2019-04-30 TW TW108115137A patent/TWI808169B/zh not_active IP Right Cessation
- 2019-04-30 US US16/399,979 patent/US10874750B2/en active Active
- 2019-04-30 TW TW112122938A patent/TW202344691A/zh unknown
- 2019-04-30 AU AU2019261982A patent/AU2019261982A1/en not_active Abandoned
- 2019-04-30 SG SG11202010533WA patent/SG11202010533WA/en unknown
- 2019-04-30 CA CA3098674A patent/CA3098674A1/en active Pending
- 2019-04-30 CN CN201980029113.5A patent/CN112513071A/zh active Pending
-
2020
- 2020-10-29 CL CL2020002809A patent/CL2020002809A1/es unknown
- 2020-10-30 PH PH12020551818A patent/PH12020551818A1/en unknown
- 2020-11-05 ZA ZA2020/06905A patent/ZA202006905B/en unknown
- 2020-11-27 CO CONC2020/0014729A patent/CO2020014729A2/es unknown
- 2020-12-18 US US17/127,001 patent/US11491243B2/en active Active
-
2022
- 2022-11-07 US US18/053,160 patent/US20230233711A1/en active Pending
-
2024
- 2024-04-19 JP JP2024068431A patent/JP2024119788A/ja not_active Withdrawn
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