JP2021520811A - ヘキソサミニダーゼアルファおよびベータサブユニットをコードしているバイシストロニックａａｖベクターならびにその使用 - Google Patents

ヘキソサミニダーゼアルファおよびベータサブユニットをコードしているバイシストロニックａａｖベクターならびにその使用 Download PDF

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JP2021520811A
JP2021520811A JP2020555467A JP2020555467A JP2021520811A JP 2021520811 A JP2021520811 A JP 2021520811A JP 2020555467 A JP2020555467 A JP 2020555467A JP 2020555467 A JP2020555467 A JP 2020555467A JP 2021520811 A JP2021520811 A JP 2021520811A
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nucleic acid
promoter
isolated nucleic
aav
expression cassette
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JP2021520811A5 (https=
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エステベス，ミゲル，セナ
ゴエンビオフスキー，ダイアン
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ユニバーシティ オブ マサチューセッツ
ユニバーシティ オブ マサチューセッツ
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01052—Beta-N-acetylhexosaminidase (3.2.1.52)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
    • C12N2830/205—Vector systems having a special element relevant for transcription transcription of more than one cistron bidirectional
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020555467A 2018-04-13 2019-04-12 ヘキソサミニダーゼアルファおよびベータサブユニットをコードしているバイシストロニックａａｖベクターならびにその使用 Withdrawn JP2021520811A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862657243P 2018-04-13 2018-04-13
US62/657,243 2018-04-13
PCT/US2019/027271 WO2019200286A1 (en) 2018-04-13 2019-04-12 Bicistronic aav vectors encoding hexosaminidase alpha and beta-subunits and uses thereof

Publications (2)

Publication Number Publication Date
JP2021520811A true JP2021520811A (ja) 2021-08-26
JP2021520811A5 JP2021520811A5 (https=) 2022-04-18

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JP2020555467A Withdrawn JP2021520811A (ja) 2018-04-13 2019-04-12 ヘキソサミニダーゼアルファおよびベータサブユニットをコードしているバイシストロニックａａｖベクターならびにその使用

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Country Link
US (2) US12486518B2 (https=)
EP (1) EP3773748A4 (https=)
JP (1) JP2021520811A (https=)
CN (1) CN112203697A (https=)
BR (1) BR112020020836A2 (https=)
CA (1) CA3096102A1 (https=)
WO (1) WO2019200286A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12448629B2 (en) 2018-10-05 2025-10-21 University Of Massachusetts rAAV vectors for the treatment of GM1 and GM2 gangliosidosis
CA3164335A1 (en) * 2020-01-10 2021-07-15 Fatih Ozsolak Viral vector for combination therapy
WO2023150051A1 (en) * 2022-02-04 2023-08-10 M6P Therapeutics, Inc. Compositions and methods of using two-promoter vector for treatment of lysosomal storage disorders
KR20250158063A (ko) * 2023-03-16 2025-11-05 젠자임 코포레이션 건식 연령-관련 황반 변성의 치료
WO2026064462A1 (en) * 2024-09-18 2026-03-26 Genzyme Corporation Production of recombinant aav

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US5217865A (en) 1986-07-25 1993-06-08 The United States Of America As Represented By The Department Of Health And Human Services Screening for Tay-Sachs disease with cloned DNA for beta-hexosaminidase
ATE404683T1 (de) 1992-09-25 2008-08-15 Aventis Pharma Sa Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
WO2000012740A2 (en) 1998-08-28 2000-03-09 Duke University ADENOVIRUSES DELETED IN THE IVa2, 100K AND/OR PRETERMINAL PROTEIN SEQUENCES
AU2013202568B2 (en) 2001-11-13 2015-09-17 The Trustees Of The University Of Pennsylvania Adeno-associated virus rh.8 (AAVrh.8) sequences and recombinant AAVs comprising same
CN1429905A (zh) * 2002-01-04 2003-07-16 北京大学 治疗帕金森病的可调控双基因表达的重组腺病毒伴随病毒载体
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
CA2483915A1 (en) 2002-05-02 2003-11-13 University Of Rochester Vectors having both isoforms of b-hexosaminidase
US20040192630A1 (en) * 2002-05-02 2004-09-30 Stephanos Kyrkanides Vectors having both isoforms of beta-hexosaminidase and uses of the same
EP1620133B1 (en) 2003-05-01 2015-12-09 Genzyme Corporation Gene therapy for neurometabolic disorders
ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
AU2009288657A1 (en) 2008-09-04 2010-03-11 Galenea Corp. Synaptic vesicle cycling assays and systems
KR20120089743A (ko) 2009-11-09 2012-08-13 제네포드 테라퓨틱스 에이비 생체 내에서 최적화된 뉴런 특이적 연속 도파 합성을 위한 신규한 바이러스 벡터 구성물
CA2781332C (en) 2009-11-19 2018-09-04 National University Corporation Okayama University System for increasing gene expression and vector comprising the system
US20130090374A1 (en) 2011-04-20 2013-04-11 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis
CA2840224C (en) 2011-06-22 2019-08-13 The General Hospital Corporation Treatment of proteinopathies
CA2888628A1 (en) 2012-10-19 2014-04-24 Tokushima University Novel high functional enzyme having modified substrate specificity of human beta-hexosaminidase b and exhibiting protease resistance
CN115120746A (zh) 2013-05-15 2022-09-30 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
CN105745326A (zh) 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
WO2015150922A2 (en) 2014-03-17 2015-10-08 The Hospital For Sick Children β-HEXOSAMINIDASE PROTEIN VARIANTS AND ASSOCIATED METHODS FOR TREATING GM2 GANGLIOSIDOSES
CN107530447A (zh) 2015-02-10 2018-01-02 建新公司 病毒颗粒至纹状体和皮质的增强递送
EP3285788B1 (en) 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
US10016514B2 (en) 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
US11045557B2 (en) 2016-06-09 2021-06-29 Queen's University At Kingston Methods and gene therapy constructs for treating GM2 gangliosidoses
WO2019222314A1 (en) 2018-05-15 2019-11-21 University Of Massachusetts Raav vectors encoding of lysosomal beta-galactorsidase (glb1) and cathepsin a
US12448629B2 (en) 2018-10-05 2025-10-21 University Of Massachusetts rAAV vectors for the treatment of GM1 and GM2 gangliosidosis

Also Published As

Publication number Publication date
EP3773748A1 (en) 2021-02-17
CN112203697A (zh) 2021-01-08
CA3096102A1 (en) 2019-10-17
US20210095314A1 (en) 2021-04-01
EP3773748A4 (en) 2022-02-23
US12486518B2 (en) 2025-12-02
US20260049336A1 (en) 2026-02-19
BR112020020836A2 (pt) 2021-01-19
WO2019200286A1 (en) 2019-10-17

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