AU2017301819B2 - Gene therapy for the treatment of aldehyde dehydrogenase deficiency - Google Patents

Gene therapy for the treatment of aldehyde dehydrogenase deficiency Download PDF

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Publication number
AU2017301819B2
AU2017301819B2 AU2017301819A AU2017301819A AU2017301819B2 AU 2017301819 B2 AU2017301819 B2 AU 2017301819B2 AU 2017301819 A AU2017301819 A AU 2017301819A AU 2017301819 A AU2017301819 A AU 2017301819A AU 2017301819 B2 AU2017301819 B2 AU 2017301819B2
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mammal
vector
aldh2
aav
aldehyde dehydrogenase
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AU2017301819A1 (en
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Ronald G. Crystal
Mehdi Gasmi
Odelya E. PAGOVICH
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Cornell University
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Cornell University
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/005—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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
    • C12Y101/01001—Alcohol dehydrogenase (1.1.1.1)
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    • 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
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    • C12Y102/01—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
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AU2017301819A 2016-07-26 2017-07-26 Gene therapy for the treatment of aldehyde dehydrogenase deficiency Active AU2017301819B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662367012P 2016-07-26 2016-07-26
US62/367,012 2016-07-26
PCT/US2017/043999 WO2018022783A1 (en) 2016-07-26 2017-07-26 Gene therapy for the treatment of aldehyde dehydrogenase deficiency

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AU2017301819A1 AU2017301819A1 (en) 2019-02-07
AU2017301819B2 true AU2017301819B2 (en) 2024-09-26

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AU2017301819A Active AU2017301819B2 (en) 2016-07-26 2017-07-26 Gene therapy for the treatment of aldehyde dehydrogenase deficiency

Country Status (8)

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US (1) US20190160187A1 (OSRAM)
EP (1) EP3490613A4 (OSRAM)
JP (2) JP7165357B2 (OSRAM)
KR (1) KR102621134B1 (OSRAM)
CN (1) CN109952114A (OSRAM)
AU (1) AU2017301819B2 (OSRAM)
SG (2) SG10202100632WA (OSRAM)
WO (1) WO2018022783A1 (OSRAM)

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Publication number Priority date Publication date Assignee Title
CA3141332A1 (en) * 2019-06-03 2020-12-10 Institute For Cancer Research D/B/A/ The Research Institute Of Fox Chase Cancer Center Adeno-associated virus vector delivery of cystathionine beta-synthase (cbs) enzyme for treating cbs deficiency
WO2021119142A1 (en) * 2019-12-09 2021-06-17 The Trustees Of Columbia University In The City Of New York 3d organoids for personalized oral cancer therapy
CN111471660B (zh) * 2020-03-12 2023-11-24 广州辉园苑医药科技有限公司 一种乙醛脱氢酶重组基因及其乳酸菌载体和应用
KR102557375B1 (ko) * 2020-12-16 2023-07-20 동국대학교 와이즈캠퍼스 산학협력단 미토콘드리아 전구서열 유래 펩타이드 및 그 용도
CN114891808B (zh) * 2022-06-21 2023-10-27 珠海丽凡达生物技术有限公司 编码ALDH2多肽的mRNA分子、应用及mRNA药物
GB202218090D0 (en) * 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency

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CA2530248A1 (en) * 2003-06-25 2005-01-06 Gencia Corporation Modified vectors for organelle transfection
US8124389B2 (en) * 2008-05-07 2012-02-28 The Board Of Trustees Of The Leland Stanford Junior University Crystal structure of aldehyde dehydrogenase and methods of use thereof
JP2012506856A (ja) * 2008-10-28 2012-03-22 ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティ アルデヒドデヒドロゲナーゼのモジュレーターおよびその使用方法
EP3567106A1 (en) * 2010-04-23 2019-11-13 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
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ES2877093T3 (es) * 2013-04-23 2021-11-16 Nizyme Inc Procedimientos y composiciones para el tratamiento de enfermedades
WO2015127137A1 (en) * 2014-02-19 2015-08-27 Aldea Pharmaceuticals, Inc. Mitochondrial aldehyde dehydrogenase 2 (aldh2) binding polycyclic amides and their use for the treatment of cancer
WO2017106202A2 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
AU2016370630B2 (en) * 2015-12-14 2023-04-13 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy

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Hu, X.Y. et al., Genetics and Molecular Research, 2016, vol. 15, pages 1-13 *

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Publication number Publication date
JP7558535B2 (ja) 2024-10-01
AU2017301819A1 (en) 2019-02-07
JP2022116045A (ja) 2022-08-09
CN109952114A (zh) 2019-06-28
EP3490613A4 (en) 2020-03-04
KR20190038583A (ko) 2019-04-08
WO2018022783A1 (en) 2018-02-01
KR102621134B1 (ko) 2024-01-04
US20190160187A1 (en) 2019-05-30
JP2019521692A (ja) 2019-08-08
SG11201900543SA (en) 2019-02-27
SG10202100632WA (en) 2021-03-30
EP3490613A1 (en) 2019-06-05
JP7165357B2 (ja) 2022-11-04

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