JP2020526514A5 - - Google Patents

Download PDF

Info

Publication number
JP2020526514A5
JP2020526514A5 JP2020500131A JP2020500131A JP2020526514A5 JP 2020526514 A5 JP2020526514 A5 JP 2020526514A5 JP 2020500131 A JP2020500131 A JP 2020500131A JP 2020500131 A JP2020500131 A JP 2020500131A JP 2020526514 A5 JP2020526514 A5 JP 2020526514A5
Authority
JP
Japan
Prior art keywords
immunosuppressant
composition
use according
months
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.)
Pending
Application number
JP2020500131A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020526514A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/040957 external-priority patent/WO2019010335A1/en
Publication of JP2020526514A publication Critical patent/JP2020526514A/ja
Publication of JP2020526514A5 publication Critical patent/JP2020526514A5/ja
Priority to JP2023108011A priority Critical patent/JP7670759B2/ja
Pending legal-status Critical Current

Links

JP2020500131A 2017-07-06 2018-07-05 ムコ多糖症i型を治療するための遺伝子治療 Pending JP2020526514A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023108011A JP7670759B2 (ja) 2017-07-06 2023-06-30 ムコ多糖症i型を治療するための遺伝子治療

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762529385P 2017-07-06 2017-07-06
US62/529,385 2017-07-06
US201762530614P 2017-07-10 2017-07-10
US62/530,614 2017-07-10
US201862616106P 2018-01-11 2018-01-11
US62/616,106 2018-01-11
PCT/US2018/040957 WO2019010335A1 (en) 2017-07-06 2018-07-05 AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023108011A Division JP7670759B2 (ja) 2017-07-06 2023-06-30 ムコ多糖症i型を治療するための遺伝子治療

Publications (2)

Publication Number Publication Date
JP2020526514A JP2020526514A (ja) 2020-08-31
JP2020526514A5 true JP2020526514A5 (https=) 2021-08-12

Family

ID=63036365

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020500131A Pending JP2020526514A (ja) 2017-07-06 2018-07-05 ムコ多糖症i型を治療するための遺伝子治療
JP2023108011A Active JP7670759B2 (ja) 2017-07-06 2023-06-30 ムコ多糖症i型を治療するための遺伝子治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023108011A Active JP7670759B2 (ja) 2017-07-06 2023-06-30 ムコ多糖症i型を治療するための遺伝子治療

Country Status (11)

Country Link
US (2) US11890329B2 (https=)
EP (1) EP3676385A1 (https=)
JP (2) JP2020526514A (https=)
KR (2) KR20200053468A (https=)
AU (1) AU2018298133A1 (https=)
BR (1) BR112020000063A2 (https=)
CA (1) CA3068328A1 (https=)
IL (1) IL271780A (https=)
MA (1) MA50016A (https=)
SG (1) SG11201912631PA (https=)
WO (1) WO2019010335A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2984166T1 (sl) 2013-03-15 2020-09-30 The Trustees Of The University Of Pennsylvania Sestavki za zdravljenje MPSI
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
EP3411488A1 (en) 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
MA50016A (fr) 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
WO2020113034A1 (en) * 2018-11-30 2020-06-04 Avexis, Inc. Aav viral vectors and uses thereof
WO2021081217A1 (en) * 2019-10-23 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2021150570A1 (en) * 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
IL300407A (en) * 2020-08-10 2023-04-01 Prevail Therapeutics Inc Gene therapies for lysosomal disorders
US20230346979A1 (en) * 2020-08-10 2023-11-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022170082A1 (en) * 2021-02-05 2022-08-11 Regents Of The University Of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses
EP4294461A4 (en) * 2021-02-22 2025-06-25 Decibel Therapeutics, Inc. METHODS AND COMPOSITIONS FOR REDUCING NUCLEIC ACID VECTOR-INDUCED TOXICITY IN THE INNER EAR
WO2024205528A1 (en) * 2023-03-31 2024-10-03 Erci̇yes Üni̇versi̇tesi̇ Strateji̇ Geli̇şti̇rme Dai̇re Başkanliği A multi-head for use in electrohydrodynamic

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU649897B2 (en) 1991-11-14 1994-06-02 Women's And Children's Hospital Synthetic alpha-L-iduronidase and genetic sequences encoding same
US6149909A (en) 1995-06-23 2000-11-21 Women's And Children's Hospital Synthetic α-L-iduronidase and genetic sequences encoding same
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
CA2328518A1 (en) 1998-05-13 1999-11-18 Harbor-Ucla Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
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
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
RU2196988C2 (ru) 2000-04-19 2003-01-20 Государственный новосибирский областной клинический диагностический центр Способ диагностики мукополисахаридозов
AU2001267337A1 (en) 2000-06-30 2002-01-14 Maxygen Aps Peptide extended glycosylated polypeptides
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
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
AU2003302724A1 (en) 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
US7155804B2 (en) 2003-09-17 2007-01-02 Moog Inc. Method of forming an electric motor
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
US20090062144A1 (en) 2007-04-03 2009-03-05 Nancy Lan Guo Gene signature for prognosis and diagnosis of lung cancer
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
JP2014503173A (ja) 2009-11-05 2014-02-13 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 制御された発現系
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
DK2675902T3 (da) 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
HRP20171334T1 (hr) 2011-04-22 2017-11-17 The Regents Of The University Of California Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe
DK3444342T3 (da) 2012-07-11 2020-08-24 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme
WO2014125647A1 (ja) 2013-02-18 2014-08-21 富士通オプティカルコンポーネンツ株式会社 光受信装置
SI2984166T1 (sl) 2013-03-15 2020-09-30 The Trustees Of The University Of Pennsylvania Sestavki za zdravljenje MPSI
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
CA2915296A1 (en) 2013-06-11 2015-02-26 Portage Pharmaceuticals Ltd. Structure, manufacturing and uses of human-derived cell-permeable peptides conjugated with specific biologically active cargo peptides
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
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
CN107002096A (zh) 2014-10-06 2017-08-01 阿罗根有限公司 基于aav的基因疗法
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
US10604747B2 (en) * 2015-08-06 2020-03-31 Academia Sinica Engineered enzyme for enzyme replacement therapy
EP3332012A4 (en) 2015-08-06 2019-01-16 The Trustees of The University of Pennsylvania GLP-1 AND USE THEREOF IN COMPOSITIONS FOR THE TREATMENT OF METABOLISM DISEASES
US20170059692A1 (en) 2015-08-28 2017-03-02 Laufer Wind Group Llc Mitigation of Small Unmanned Aircraft Systems Threats
HK1257024A1 (zh) * 2015-08-31 2019-10-11 宾夕法尼亚大学 用於治疗伴侣动物的aav-epo
US20180236105A1 (en) * 2015-10-23 2018-08-23 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
CA3010738A1 (en) * 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
EP3411488A1 (en) 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
EP3541946A1 (en) 2016-11-15 2019-09-25 Regents Of The University Of Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
CA3049915A1 (en) 2017-01-31 2018-08-09 Stephen YOO Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
MA50016A (fr) 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
IL280198B2 (en) 2018-07-18 2026-01-01 Regenxbio Inc Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus)
WO2021150570A1 (en) 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
WO2022170082A1 (en) 2021-02-05 2022-08-11 Regents Of The University Of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses

Similar Documents

Publication Publication Date Title
JP2020526514A5 (https=)
JP2020537637A5 (https=)
CN108310396B (zh) 用于心血管疾病和其它病况的旁分泌基因的全身性递送和受调节的表达
ES2916344T3 (es) Composiciones y métodos para reducir la neovascularización ocular
JP7175506B2 (ja) 低血圧治療のための単独または併用使用されるアンギオテンシンii
IL273427B1 (en) Gene therapy for treating mucopolysaccharidosis type ii
US10098969B2 (en) Methods and compositions for increasing sialic acid production and treating sialic related disease conditions
US20240002462A1 (en) Treatment of neuropathy with igf-1-encoding dna constructs and hgf-encoding dna constructs
KR20160137908A (ko) 당뇨병-연관 심기능 장애 및 울혈성 심부전을 치료하기 위한 우로코르틴-2 및 연관 유전자들을 인코딩하는 바이러스 벡터들의 전신 전달
BR112021013876A2 (pt) Codificação de rhabdovírus recombinante para ccl21
WO2023091960A1 (en) Vectors for increasing nprl2 expression in cancer cells and methods of use thereof
JP7413629B2 (ja) Igf-1異型体を発現させるdnaコンストラクトを用いた神経病症の治療
CA3178591A1 (en) Immunosuppressive agents and viral delivery re-dosing methods for gene therapy
EP4509606A1 (en) Nucleic acid construct for treating hereditary coagulation factor deficiency and use thereof
EP3570896B1 (en) Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use
JP2022523038A (ja) 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
JPWO2022232141A5 (https=)
JPWO2023150566A5 (https=)
US20220088223A1 (en) Methods and compositions for increasing sialic acid production and treating sialic related disease conditions
WO2020097150A1 (en) Treatment of traumatic brain injury
CA2978917C (en) Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression
TW202503064A (zh) 用編碼阿柏西普之aav2變異體治療眼部新生血管性疾病之方法
JPWO2023020802A5 (https=)
JPWO2021155337A5 (https=)
Dagueta et al. Evolving knowledge in pharmacologic treatments of age-related macular degeneration