JPWO2020010042A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2020010042A5
JPWO2020010042A5 JP2020570046A JP2020570046A JPWO2020010042A5 JP WO2020010042 A5 JPWO2020010042 A5 JP WO2020010042A5 JP 2020570046 A JP2020570046 A JP 2020570046A JP 2020570046 A JP2020570046 A JP 2020570046A JP WO2020010042 A5 JPWO2020010042 A5 JP WO2020010042A5
Authority
JP
Japan
Prior art keywords
seq
nucleotide sequence
vector genome
sequence
aav vector
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
JP2020570046A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021529513A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/040230 external-priority patent/WO2020010042A1/en
Publication of JP2021529513A publication Critical patent/JP2021529513A/ja
Publication of JPWO2020010042A5 publication Critical patent/JPWO2020010042A5/ja
Pending legal-status Critical Current

Links

JP2020570046A 2018-07-02 2019-07-02 筋萎縮性側索硬化症および脊髄に関連する障害の治療 Pending JP2021529513A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862693040P 2018-07-02 2018-07-02
US62/693,040 2018-07-02
US201862746104P 2018-10-16 2018-10-16
US62/746,104 2018-10-16
PCT/US2019/040230 WO2020010042A1 (en) 2018-07-02 2019-07-02 Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord

Publications (2)

Publication Number Publication Date
JP2021529513A JP2021529513A (ja) 2021-11-04
JPWO2020010042A5 true JPWO2020010042A5 (hr) 2022-07-11

Family

ID=67551691

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020570046A Pending JP2021529513A (ja) 2018-07-02 2019-07-02 筋萎縮性側索硬化症および脊髄に関連する障害の治療

Country Status (7)

Country Link
US (1) US20210254103A1 (hr)
EP (1) EP3818161A1 (hr)
JP (1) JP2021529513A (hr)
CN (1) CN112567035A (hr)
AU (1) AU2019299861A1 (hr)
CA (1) CA3103963A1 (hr)
WO (1) WO2020010042A1 (hr)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
CN110913866A (zh) 2017-05-05 2020-03-24 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
JP7502991B2 (ja) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症(als)の治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2020223296A1 (en) * 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
IL304880A (en) 2021-02-12 2023-10-01 Alnylam Pharmaceuticals Inc Superoxide dismutase 1 (SOD1) IRNA compositions and methods of using them to treat or prevent superoxide dismutase 1- (SOD1-) associated neurodegenerative diseases

Family Cites Families (121)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4650764A (en) 1983-04-12 1987-03-17 Wisconsin Alumni Research Foundation Helper cell
US4980289A (en) 1987-04-27 1990-12-25 Wisconsin Alumni Research Foundation Promoter deficient retroviral vector
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
US5124263A (en) 1989-01-12 1992-06-23 Wisconsin Alumni Research Foundation Recombination resistant retroviral helper cell and products produced thereby
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5387484A (en) 1992-07-07 1995-02-07 International Business Machines Corporation Two-sided mask for patterning of materials with electromagnetic radiation
CA2592997A1 (en) 1992-12-03 1994-06-09 Genzyme Corporation Pseudo-adenovirus vectors
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
FR2705686B1 (fr) 1993-05-28 1995-08-18 Transgene Sa Nouveaux adénovirus défectifs et lignées de complémentation correspondantes.
DK0667912T3 (da) 1993-07-13 2008-11-10 Centelion Defekte adenovirusvektorer og anvendelse heraf i genterapi
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
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
US6676935B2 (en) 1995-06-27 2004-01-13 Cell Genesys, Inc. Tissue specific adenoviral vectors
US6197293B1 (en) 1997-03-03 2001-03-06 Calydon, Inc. Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof
US6143548A (en) 1995-08-30 2000-11-07 Genzyme Corporation Chromatographic purification of adeno-associated virus (AAV)
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
ATE550429T1 (de) 1996-11-20 2012-04-15 Crucell Holland Bv Adenovirus-zusammensetzungen erhältlich durch ein verbessertes produktions- und reinigungsverfahren
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
EP0950091A2 (en) 1996-12-18 1999-10-20 Targeted Genetics Corporation Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP1015619A1 (en) 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP2002506652A (ja) 1998-03-20 2002-03-05 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のヘルパーを含まない製造のための組成物及び方法
FR2778413B1 (fr) 1998-05-07 2000-08-04 Immunotech Sa Nouveaux reactifs et methode de lyse des erythrocytes
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
GB2338236B (en) 1998-06-13 2003-04-09 Aea Technology Plc Microbiological cell processing
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
MXPA01004169A (es) 1998-10-27 2002-06-04 Crucell Holland Bv Produccion mejorada de vector de virus adenoasociados.
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
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
AU2409200A (en) 1999-06-02 2000-12-28 Trustees Of The University Of Pennsylvania, The Compositions and methods useful for production of recombinant viruses which require helper viruses
US6399385B1 (en) 1999-09-29 2002-06-04 The Trustees Of The University Of Pennsylvania Methods for rapid PEG-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
US6334998B1 (en) 1999-12-07 2002-01-01 Parker Hughes Institute Estrogens for treating ALS
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
EP1302542B1 (en) 2000-07-18 2007-06-13 Takeda Pharmaceutical Company Limited Novel physiologically active peptide and use thereof
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
FR2813891B1 (fr) 2000-09-14 2005-01-14 Immunotech Sa Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications
ATE303808T1 (de) 2000-10-24 2005-09-15 Mitsubishi Pharma Corp Mittel zur behandlung der amyotrophischen lateralsklerose (als)
EP2017338A1 (en) 2001-05-24 2009-01-21 Genzyme Corporation Muscle-specific expression vectors
EP2270024B1 (en) 2001-06-21 2018-10-24 Ionis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
NZ600121A (en) 2001-11-13 2013-12-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
CA2469623C (en) 2001-12-12 2012-05-29 F H Faulding & Co Limited Composition for the preservation of viruses
CN1617938A (zh) 2002-01-16 2005-05-18 戴诺生物技术有限公司 从单个样品中分离核酸和蛋白质的方法
MXPA04010603A (es) 2002-04-30 2004-12-13 Oncolytics Biotech Inc Metodos mejorados de purificacion viral.
US20050196854A1 (en) 2002-05-14 2005-09-08 Konz John O.Jr. Methods of adenovirus purification
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
US20050196862A1 (en) * 2002-08-30 2005-09-08 Wooddell Christine I. DNA cassette for cellular expression of small RNA
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
CA2505514A1 (en) 2002-11-06 2004-05-27 Mount Sinai School Of Medicine Treatment of amyotrophic lateral sclerosis with nimesulide
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
CA2509494A1 (en) 2002-12-11 2004-06-24 University Of Massachusetts Method of introducing sirna into adipocytes
ES2521682T3 (es) 2003-05-21 2014-11-13 Genzyme Corporation Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías
PL1633772T3 (pl) 2003-06-19 2016-10-31 Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US7491508B2 (en) 2003-06-20 2009-02-17 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2005007676A2 (en) 2003-07-10 2005-01-27 Rajadhyaksha V J Glycopeptides for the treatment of als and other metabolic and autoimmune disorders
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
PT1714960T (pt) 2004-02-09 2018-05-02 Mitsubishi Tanabe Pharma Corp Um agente terapêutico inovador para a esclerose lateral amiotrófica (ela) ou doenças provocadas pela ela
WO2005096781A2 (en) 2004-04-06 2005-10-20 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using rna interference
ES2647477T3 (es) 2004-06-01 2017-12-21 Genzyme Corporation Composiciones y métodos para prevenir la agregación del vector AAV
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
WO2006066203A2 (en) 2004-12-16 2006-06-22 Alsgen, Llc Small interfering rna (sirna) molecules for modulating superoxide dismutase (sod)
US8614101B2 (en) 2008-05-20 2013-12-24 Rapid Pathogen Screening, Inc. In situ lysis of cells in lateral flow immunoassays
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
EP1986609A2 (en) 2006-01-26 2008-11-05 University of Massachusetts Rna silencing agents for use in therapy and nanotransporters for efficient delivery of same
WO2007098607A1 (en) 2006-03-03 2007-09-07 Amorfix Life Sciences Ltd. Methods and compositions to treat and detect misfolded-sod1 mediated diseases
ES2400235T3 (es) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania Método de producción escalable de AAV
AU2007257093A1 (en) 2006-05-05 2007-12-13 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of PCSK9
US8183219B2 (en) 2007-01-03 2012-05-22 Medtronic, Inc. Therapeuting compositions comprising an RNAi agent and a neurotrophic factor and methods of use thereof
EP2680007A1 (en) 2007-02-08 2014-01-01 Biogen Idec MA Inc. Nrf2 screening assays and related methods and compositions
US20090036395A1 (en) 2007-04-26 2009-02-05 Davidson Beverly L Rna interference suppression of neurodegenerative diseases and methods of use thereof
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
WO2009014445A2 (en) 2007-07-26 2009-01-29 Amsterdam Molecular Therapeutics B.V. Baculoviral vectors comprising repeated coding sequences with differential codon biases
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
CA2713338C (en) 2008-01-29 2021-10-26 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
JP5328244B2 (ja) 2008-07-07 2013-10-30 株式会社ニュージェン・ファーマ 筋萎縮性側索硬化症治療剤
US8664189B2 (en) 2008-09-22 2014-03-04 Rxi Pharmaceuticals Corporation RNA interference in skin indications
CA2744016C (en) 2008-11-20 2018-12-11 Cambria Pharmaceuticals, Inc. Treatment of amyotrophic lateral sclerosis
EP2435575A2 (en) 2009-05-28 2012-04-04 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
WO2011005786A2 (en) 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for enhancing production of a biological product
EP2568999B1 (en) 2010-05-11 2018-07-11 Mallinckrodt Ard Ip Limited Acth for treatment of amyotrophic lateral sclerosis
EP2422787A1 (en) 2010-08-17 2012-02-29 Neurotec Pharma, S.L. Diazoxide for use in the treatment of amyotrophic lateral sclerosis (als)
ES2739804T3 (es) 2011-02-12 2020-02-04 Univ Iowa Res Found Compuestos terapéuticos
US9867837B2 (en) 2011-03-01 2018-01-16 Pharnext Compositions for treating neurological disorders
AU2012266754B2 (en) 2011-06-06 2016-04-21 Biocartis Nv Selective lysis of cells by ionic surfactants
KR101862203B1 (ko) 2011-12-27 2018-05-29 (주)바이오팜솔루션즈 페닐알킬카바메이트 유도체 화합물 및 이를 포함하는 약학 조성물
WO2013119880A1 (en) * 2012-02-07 2013-08-15 Global Bio Therapeutics Usa, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
TWI775096B (zh) 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
AU2013287261B2 (en) * 2012-07-06 2019-03-07 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions
CN105612253A (zh) * 2013-03-15 2016-05-25 费城儿童医院 含有填充者/填充物多核苷酸序列的载体及其制备方法
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
JP6663859B2 (ja) 2014-05-20 2020-03-13 ユニバーシティー オブ アイオワ リサーチ ファウンデーション ハンチントン病の治療化合物
PT3198018T (pt) * 2014-09-24 2021-02-24 Hope City Variantes dos vetores de vírus adenoassociados para edição do genoma de alta eficácia e métodos da mesma
US10597660B2 (en) * 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
SG11201703419UA (en) * 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
LT3237618T (lt) 2014-12-24 2019-07-10 Uniqure Ip B.V. Rnri sukeltas hantingtino geno slopinimas
WO2017189963A1 (en) * 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
SG11201809643UA (en) * 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
JP2020518266A (ja) * 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 調節性ポリヌクレオチド
CN110913866A (zh) * 2017-05-05 2020-03-24 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
WO2019079242A1 (en) * 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)

Similar Documents

Publication Publication Date Title
JP2021502060A5 (hr)
JP7416443B2 (ja) 高形質導入効率rAAVベクター、組成物、および使用方法
US20230414724A1 (en) Treatment of hyperbilirubinemia
JP2020537544A5 (hr)
JP6495273B2 (ja) 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
AU2013221212B2 (en) AAV vector compositions and methods for gene transfer to cells, organs and tissues
JP2018516978A5 (hr)
JPWO2020132455A5 (hr)
JP2020537542A5 (hr)
JP2021508241A5 (hr)
JP2020535804A5 (hr)
RU2018145010A (ru) Оптимизированные гены и экспрессионные кассеты cln1, и их применение
WO2020142714A1 (en) Aav expression cassette and aav vectors comprising the same
IL292605A (en) CRISPR and AAV strategies for the treatment of x-linked childhood retinoschisis
JPWO2020010042A5 (hr)
JPWO2020082046A5 (hr)
RU2019130004A (ru) Генная терапия при глазных заболеваниях
JPWO2020069461A5 (hr)
JPWO2020227166A5 (hr)
CA3146364A1 (en) One-step gene therapy for duchenne muscular dystrophy via gene replacement and anti-inflammation
JPWO2020219766A5 (hr)
JPWO2021092513A5 (hr)
JPWO2020132115A5 (hr)
JPWO2020172490A5 (hr)
JPWO2019195727A5 (hr)