JP2021500029A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2021500029A5 JP2021500029A5 JP2020522007A JP2020522007A JP2021500029A5 JP 2021500029 A5 JP2021500029 A5 JP 2021500029A5 JP 2020522007 A JP2020522007 A JP 2020522007A JP 2020522007 A JP2020522007 A JP 2020522007A JP 2021500029 A5 JP2021500029 A5 JP 2021500029A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- raav
- polypeptide
- composition
- tmck
- 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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023151106A JP2023166580A (ja) | 2017-10-20 | 2023-09-19 | Ｎｔ－３遺伝子治療のための方法及び材料 |
| JP2025127206A JP2025157575A (ja) | 2017-10-20 | 2025-07-30 | Ｎｔ－３遺伝子治療のための方法及び材料 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762574828P | 2017-10-20 | 2017-10-20 | |
| US62/574,828 | 2017-10-20 | ||
| US201862676687P | 2018-05-25 | 2018-05-25 | |
| US62/676,687 | 2018-05-25 | ||
| US201862741335P | 2018-10-04 | 2018-10-04 | |
| US62/741,335 | 2018-10-04 | ||
| PCT/US2018/056765 WO2019079755A1 (en) | 2017-10-20 | 2018-10-19 | METHODS AND MATERIALS FOR NT-3 GENE THERAPY |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023151106A Division JP2023166580A (ja) | 2017-10-20 | 2023-09-19 | Ｎｔ－３遺伝子治療のための方法及び材料 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021500029A JP2021500029A (ja) | 2021-01-07 |
| JP2021500029A5 true JP2021500029A5 (https=) | 2021-12-02 |
| JP7832758B2 JP7832758B2 (ja) | 2026-03-18 |
Family
ID=66173875
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020522007A Active JP7832758B2 (ja) | 2017-10-20 | 2018-10-19 | Ｎｔ－３遺伝子治療のための方法及び材料 |
| JP2023151106A Pending JP2023166580A (ja) | 2017-10-20 | 2023-09-19 | Ｎｔ－３遺伝子治療のための方法及び材料 |
| JP2025127206A Pending JP2025157575A (ja) | 2017-10-20 | 2025-07-30 | Ｎｔ－３遺伝子治療のための方法及び材料 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023151106A Pending JP2023166580A (ja) | 2017-10-20 | 2023-09-19 | Ｎｔ－３遺伝子治療のための方法及び材料 |
| JP2025127206A Pending JP2025157575A (ja) | 2017-10-20 | 2025-07-30 | Ｎｔ－３遺伝子治療のための方法及び材料 |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US11926653B2 (https=) |
| EP (1) | EP3697916A4 (https=) |
| JP (3) | JP7832758B2 (https=) |
| KR (2) | KR20200083495A (https=) |
| CN (2) | CN119193705A (https=) |
| AU (2) | AU2018351528B2 (https=) |
| BR (1) | BR112020007790A2 (https=) |
| CA (1) | CA3079416A1 (https=) |
| CL (1) | CL2020001060A1 (https=) |
| CO (1) | CO2020006148A2 (https=) |
| IL (2) | IL273976B2 (https=) |
| MA (1) | MA50412A (https=) |
| MX (2) | MX2020004035A (https=) |
| MY (1) | MY207563A (https=) |
| SA (1) | SA520411798B1 (https=) |
| SG (1) | SG11202003285PA (https=) |
| TW (1) | TWI879721B (https=) |
| WO (1) | WO2019079755A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102877920B1 (ko) | 2015-11-16 | 2025-10-30 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법 |
| AR114350A1 (es) | 2018-01-31 | 2020-08-26 | Res Institute At Nationwide ChildrenS Hospital | Terapia genética para la distrofia muscular de cinturas del tipo 2c |
| KR20210028162A (ko) | 2018-06-29 | 2021-03-11 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 |
| JP2022523038A (ja) * | 2019-02-02 | 2022-04-21 | ユニバーシティ オブ ピッツバーグ －オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション | 遺伝子置換及び抗炎症を介したデュシェンヌ型筋ジストロフィーに対する一段階遺伝子療法 |
| AU2020229340A1 (en) | 2019-02-26 | 2021-09-16 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy |
| GB201907882D0 (en) * | 2019-06-03 | 2019-07-17 | Cyprus Foundation For Muscular Dystrophy Res | Methods |
| RS65421B1 (sr) | 2019-08-21 | 2024-05-31 | Res Inst Nationwide Childrens Hospital | Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije |
| WO2021046169A1 (en) | 2019-09-03 | 2021-03-11 | University Of Cincinnati | Methods and compositions for the treatment of als |
| WO2021211713A1 (en) * | 2020-04-14 | 2021-10-21 | Research Institute At Nationwide Children's Hospital | Treatment of charcot-marie-tooth axonal type 2d using nt-3 gene therapy |
| WO2021211705A1 (en) * | 2020-04-14 | 2021-10-21 | Research Institute At Nationwide Children's Hospital | Treatment of sarcopenia using nt-3 gene therapy |
| CN116440252A (zh) * | 2020-06-16 | 2023-07-18 | 温州医科大学附属第二医院（温州医科大学附属育英儿童医院） | 神经营养因子3在制备提高睾丸中睾酮含量的药物中的应用 |
| EP4108263A3 (en) * | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| CN115838725B (zh) * | 2022-12-30 | 2023-09-08 | 广州派真生物技术有限公司 | 在哺乳动物心脏中特异性启动基因的启动子序列及其应用 |
| WO2024229259A1 (en) * | 2023-05-02 | 2024-11-07 | Research Institute At Nationwide Children's Hospital | Gene therapy for treatment of protein misfolding diseases |
| WO2024229115A1 (en) * | 2023-05-02 | 2024-11-07 | Research Institute At Nationwide Children's Hospital | Treatment of multiple sclerosis using nt-3 gene therapy |
| CN119899809A (zh) * | 2025-01-07 | 2025-04-29 | 广州派真生物技术有限公司 | 一种提高重组腺相关病毒生产效率的方法及应用 |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| JP2001500497A (ja) | 1996-09-06 | 2001-01-16 | 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 | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| WO2010089706A1 (en) * | 2009-02-04 | 2010-08-12 | Ecole Polytechnique Federale De Lausanne (Epfl) | Use of igf-1 and nt-3 in the treatment of x-linked adrenoleukodystrophy |
| WO2011094198A1 (en) | 2010-01-28 | 2011-08-04 | The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
| EP2736539B1 (en) | 2011-07-25 | 2017-08-23 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of dux4 |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| US10286084B2 (en) | 2014-02-18 | 2019-05-14 | Duke University | Compositions for the inactivation of virus replication and methods of making and using the same |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| KR20160068558A (ko) * | 2014-12-05 | 2016-06-15 | 삼성바이오에피스 주식회사 | 마우스 cmv 프로모터를 포함하는 융합 폴리뉴클레오타이드 및 이를 이용한 목적 폴리펩타이드의 생산 방법 |
| US20180263945A1 (en) | 2014-12-19 | 2018-09-20 | Research Institute At Nationwide Children's Hospital | Pyruvate compounds for treatment of peripheral neuropathy |
| IL258005B2 (en) | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
| GB2547179A (en) | 2015-10-26 | 2017-08-16 | Quethera Ltd | Genetic construct |
| ES3034857T3 (en) | 2015-11-12 | 2025-08-25 | The Res Institute At Nationwide Childrens Hospital | Methods of treating muscular dystrophy |
| WO2017165806A1 (en) | 2016-03-25 | 2017-09-28 | Periphagen, Inc. | Hsv vectors for delivery of nt3 and treatment of cipn |
| WO2017173043A1 (en) | 2016-03-30 | 2017-10-05 | Spark Therapeutics, Inc. | Cell line for recombinant protein and/or viral vector production |
| LT3442600T (lt) * | 2016-04-15 | 2024-05-27 | Research Institute At Nationwide Children's Hospital | Su adeno virusu susijusio viruso vektoriaus iš β-sarkoglikano ir mikro- rnr-29 pristatymas ir raumenų distrofijos gydymas |
| ES2977730T3 (es) * | 2016-04-15 | 2024-08-29 | Res Inst Nationwide Childrens Hospital | Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular |
| JP7676111B2 (ja) | 2017-03-17 | 2025-05-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
-
2018
- 2018-10-19 US US16/757,176 patent/US11926653B2/en active Active
- 2018-10-19 MY MYPI2020001966A patent/MY207563A/en unknown
- 2018-10-19 SG SG11202003285PA patent/SG11202003285PA/en unknown
- 2018-10-19 MX MX2020004035A patent/MX2020004035A/es unknown
- 2018-10-19 CN CN202411339680.XA patent/CN119193705A/zh active Pending
- 2018-10-19 IL IL273976A patent/IL273976B2/en unknown
- 2018-10-19 JP JP2020522007A patent/JP7832758B2/ja active Active
- 2018-10-19 BR BR112020007790-9A patent/BR112020007790A2/pt unknown
- 2018-10-19 KR KR1020207014064A patent/KR20200083495A/ko not_active Ceased
- 2018-10-19 MA MA050412A patent/MA50412A/fr unknown
- 2018-10-19 EP EP18867716.5A patent/EP3697916A4/en active Pending
- 2018-10-19 CN CN201880068238.4A patent/CN111433367A/zh active Pending
- 2018-10-19 AU AU2018351528A patent/AU2018351528B2/en active Active
- 2018-10-19 IL IL321310A patent/IL321310A/en unknown
- 2018-10-19 KR KR1020247037712A patent/KR20240164969A/ko active Pending
- 2018-10-19 WO PCT/US2018/056765 patent/WO2019079755A1/en not_active Ceased
- 2018-10-19 CA CA3079416A patent/CA3079416A1/en active Pending
- 2018-10-22 TW TW107137220A patent/TWI879721B/zh active
-
2020
- 2020-04-19 SA SA520411798A patent/SA520411798B1/ar unknown
- 2020-04-20 CL CL2020001060A patent/CL2020001060A1/es unknown
- 2020-05-19 CO CONC2020/0006148A patent/CO2020006148A2/es unknown
- 2020-07-13 MX MX2025010166A patent/MX2025010166A/es unknown
-
2023
- 2023-09-19 JP JP2023151106A patent/JP2023166580A/ja active Pending
-
2024
- 2024-03-08 US US18/600,267 patent/US20240383956A1/en active Pending
-
2025
- 2025-01-31 AU AU2025200649A patent/AU2025200649A1/en active Pending
- 2025-07-30 JP JP2025127206A patent/JP2025157575A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021500029A5 (https=) | ||
| US20240109938A1 (en) | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof | |
| Vandenberghe et al. | Tailoring the AAV vector capsid for gene therapy | |
| AU2018304194B2 (en) | Apheresis methods and uses | |
| RU2751592C2 (ru) | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе | |
| IL320714A (en) | Gene therapy for limb-girdle muscular dystrophy type 2c | |
| EP3134113A1 (en) | Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus | |
| JP2023501263A (ja) | 第ｖｉｉｉ因子ポリペプチド | |
| JP2023503850A5 (https=) | ||
| JP2024511024A (ja) | ２１－ヒドロキシラーゼ欠損症の遺伝子治療 | |
| MX2024010002A (es) | Metodo para reducir la fluctuacion del cst en amd neovascular mediante un virus adenoasociado recombinante | |
| CA3207161A1 (en) | Methods and compositions for treatment of friedreich's ataxia | |
| US20250207126A1 (en) | Modified muscle-specific promoters | |
| JP2022519596A (ja) | Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達 | |
| CN115836128A (zh) | 用于基因疗法的编码葡萄糖-6-磷酸酶(G6Pase-a)的载体 | |
| JP7684296B2 (ja) | Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法 | |
| US20220136005A1 (en) | One-step gene therapy for duchenne muscular dystrophy via gene replacement and anti-inflammation | |
| US20250144243A1 (en) | Aav capsids for improved heart transduction and detargeting of liver | |
| JP2025534651A (ja) | 呼吸困難を伴う脊髄性筋萎縮１型（ｓｍａｒｄ１）及びシャルコー・マリー・トゥース２ｓ型（ｃｍｔ２ｓ）を治療するためのアデノ随伴ウイルス送達 | |
| JP2024515623A (ja) | 髄腔内送達によってピット・ホプキンス症候群を治療するためのメチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルス | |
| JP2024513533A (ja) | 血友病における血友病性関節症の予防及び／又は治療のための方法及び手段 | |
| RU2021102985A (ru) | Генная терапия нейродегенеративных нарушений | |
| JP2021527418A (ja) | ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 | |
| NZ748736B2 (en) | Treatment of complement-mediated disorders |