JP2021500029A5 - - Google Patents

Download PDF

Info

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
Application number
JP2020522007A
Other languages
English (en)
Japanese (ja)
Other versions
JP7832758B2 (ja
JP2021500029A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/056765 external-priority patent/WO2019079755A1/en
Publication of JP2021500029A publication Critical patent/JP2021500029A/ja
Publication of JP2021500029A5 publication Critical patent/JP2021500029A5/ja
Priority to JP2023151106A priority Critical patent/JP2023166580A/ja
Priority to JP2025127206A priority patent/JP2025157575A/ja
Application granted granted Critical
Publication of JP7832758B2 publication Critical patent/JP7832758B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2020522007A 2017-10-20 2018-10-19 Ｎｔ－３遺伝子治療のための方法及び材料 Active JP7832758B2 (ja)

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) IL321310A (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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3005633C (en) 2015-11-16 2023-11-21 Research Institute Of Nationwide Children's Hospital Materials and methods for treatment of titin-based myopathies and other titinopathies
MX2020007876A (es) 2018-01-31 2020-12-03 Res Inst Nationwide Childrens Hospital Terapia genica para la distrofia muscular de cinturas tipo 2c.
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
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
MX2021010356A (es) 2019-02-26 2022-03-04 Res Inst Nationwide Childrens Hospital SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR.
GB201907882D0 (en) * 2019-06-03 2019-07-17 Cyprus Foundation For Muscular Dystrophy Res Methods
PT4017871T (pt) 2019-08-21 2024-04-22 Res Inst Nationwide Childrens Hospital Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular
EP4025258A4 (en) * 2019-09-03 2023-09-06 University of Cincinnati METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
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
CN111514279B (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在制备治疗男性性腺功能减退症的药物中的应用
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
JP2023059858A (ja) 2021-10-15 2023-04-27 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 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
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
WO2024229115A1 (en) * 2023-05-02 2024-11-07 Research Institute At Nationwide Children's Hospital Treatment of multiple sclerosis using nt-3 gene therapy
AU2024265705A1 (en) 2023-05-02 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases
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)

* Cited by examiner, † Cited by third party
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
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
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
AU707866B2 (en) 1994-12-06 1999-07-22 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
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved 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
EP0932418B1 (en) 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法
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
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
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
DK2529020T3 (en) 2010-01-28 2018-08-06 Childrens Hospital Philadelphia SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY
AU2012286994B2 (en) 2011-07-25 2017-08-10 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
JP2017506893A (ja) 2014-02-18 2017-03-16 デューク ユニバーシティ ウイルス複製不活化組成物並びにその製造方法及び使用
JP6202701B2 (ja) 2014-03-21 2017-09-27 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プログラム
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
WO2016100963A1 (en) 2014-12-19 2016-06-23 Research Institute At Nationwide Children's Hospital Pyruvate compounds for treatment of peripheral neuropathy
EA201890543A1 (ru) 2015-09-17 2018-08-31 Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл Способы и материалы для генной терапии galgt2
GB2547179A (en) 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
FI3373980T3 (fi) 2015-11-12 2025-07-10 The Res Institute At Nationwide Childrens Hospital Menetelmiä lihasdystrofian hoitamiseksi
US10799560B2 (en) 2016-03-25 2020-10-13 Periphagen, Inc. HSV vectors for delivery of NT3 and treatment of CIPN
MX2018011928A (es) 2016-03-30 2019-03-28 Spark Therapeutics Inc Linea celular para produccion de proteina recombinante y/o vector viral.
PT3442600T (pt) * 2016-04-15 2024-05-09 Res Inst Nationwide Childrens Hospital Distribuição de vetor de vírus adeno-associado de bsarcoglicano e microrna-29 e o tratamento da distrofia muscular
AU2017250791B2 (en) * 2016-04-15 2022-11-24 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
MX2019011046A (es) 2017-03-17 2019-10-17 Res Inst Nationwide Childrens Hospital Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular.

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
US10308957B2 (en) rAAV vectors and methods for transduction of photoreceptors and RPE cells
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
WO2015164758A1 (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
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
JP2024156872A (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
RU2021102985A (ru) Генная терапия нейродегенеративных нарушений
NZ748736B2 (en) Treatment of complement-mediated disorders
JPWO2023183594A5 (https=)