AU2018351528B2 - Methods and materials for NT-3 gene therapy - Google Patents

Methods and materials for NT-3 gene therapy Download PDF

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AU2018351528B2
AU2018351528B2 AU2018351528A AU2018351528A AU2018351528B2 AU 2018351528 B2 AU2018351528 B2 AU 2018351528B2 AU 2018351528 A AU2018351528 A AU 2018351528A AU 2018351528 A AU2018351528 A AU 2018351528A AU 2018351528 B2 AU2018351528 B2 AU 2018351528B2
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muscle
aav
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neuropathy
nucleic acid
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AU2018351528A1 (en
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Zarife SAHENK
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Nationwide Childrens Hospital Inc
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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AU2018351528A 2017-10-20 2018-10-19 Methods and materials for NT-3 gene therapy Active AU2018351528B2 (en)

Priority Applications (1)

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AU2025200649A AU2025200649A1 (en) 2017-10-20 2025-01-31 Methods and materials for NT-3 gene therapy

Applications Claiming Priority (7)

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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

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EP (1) EP3697916A4 (enExample)
JP (3) JP2021500029A (enExample)
KR (2) KR20240164969A (enExample)
CN (2) CN119193705A (enExample)
AU (2) AU2018351528B2 (enExample)
BR (1) BR112020007790A2 (enExample)
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Families Citing this family (16)

* Cited by examiner, † Cited by third party
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
SG11202006722RA (en) 2018-01-31 2020-08-28 Res Inst Nationwide Childrens Hospital Gene therapy for limb-girdle muscular dystrophy type 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
CA3131390A1 (en) 2019-02-26 2020-09-03 Research Institute Of 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
HRP20240505T1 (hr) 2019-08-21 2024-07-05 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana vektorom povezanim s adenovirusom i liječenje mišićne distrofije
EP4025258A4 (en) * 2019-09-03 2023-09-06 University of Cincinnati METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
WO2021211705A1 (en) * 2020-04-14 2021-10-21 Research Institute At Nationwide Children's Hospital Treatment of sarcopenia using nt-3 gene therapy
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
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
AU2024265705A1 (en) 2023-05-02 2025-11-20 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 (32)

* 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
ATE272123T1 (de) 1993-11-09 2004-08-15 Ohio Med College Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
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
EP0796339A1 (en) 1994-12-06 1997-09-24 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
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
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
ATE465267T1 (de) 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen
WO1999011764A2 (en) 1997-09-05 1999-03-11 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of 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
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
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
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
PT2529020T (pt) 2010-01-28 2018-07-30 Childrens Hospital Philadelphia Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica
ES2990023T3 (es) 2011-07-25 2024-11-28 Nationwide Childrens Hospital Inc Productos de virus recombinantes y métodos para inhibición de la expresión de DUX4
KR20160130392A (ko) 2014-02-18 2016-11-11 듀크 유니버시티 바이러스 복제의 불활성화를 위한 조성물 및 그의 제조 및 사용 방법
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
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
IL259178B (en) 2015-11-12 2022-08-01 Res Inst Nationwide Childrens Hospital A vector encoding ano5 for the treatment of muscular dystrophy
WO2017165806A1 (en) 2016-03-25 2017-09-28 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.
MA45477A (fr) * 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
HUE066442T2 (hu) 2016-04-15 2024-08-28 Res Institute At Nationwide ß-szarkoglikán és mikroRNS-29 adenoasszociáltvírus-vektorral történõ bejuttatása és izomdisztrófia kezelése
WO2018170408A1 (en) 2017-03-17 2018-09-20 Research Institute At Nationwide Children's Hospital, Inc. Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
YALVAC M E ET AL, GENE THERAPY, vol. 23, no. 1, 1 January 2016 (2016-01-01), GB, pages 95 - 102, XP055817812, ISSN: 0969-7128, Retrieved from the Internet <URL:https://www.nature.com/articles/gt201567.pdf> DOI: 10.1038/gt.2015.67 *

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