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
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    • 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
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    • A61K9/0012—Galenical forms characterised by the site of application
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    • 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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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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

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

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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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AU2004252559B2 (en) * 2003-06-25 2010-07-29 Gencia Corporation Modified vectors for organelle transfection
WO2009137045A2 (en) * 2008-05-07 2009-11-12 The Board Of Trustees Of The Leland Stanford Junior University Crystal structure of aldehyde dehydrogenase and methods of use thereof
WO2010062308A1 (en) * 2008-10-28 2010-06-03 The Board Of Trustees Of The Leland Stanford Junior University Modulators of aldehyde dehydrogenase and methods of use thereof
EP3514232A1 (en) * 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
JP6091435B2 (ja) * 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達
US9066966B2 (en) * 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
EP2988737B1 (en) * 2013-04-23 2021-04-07 Nizyme Inc. Methods and compositions for treating diseases
ES2833157T3 (es) * 2014-02-19 2021-06-14 Aviv Therapeutics Inc Amidas políciclicas de unión a aldehído deshidrogenasa 2 mitocondrial (aldh2) y su uso para el tratamiento del cáncer
EP3389724B1 (en) * 2015-12-14 2020-07-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JP7082050B2 (ja) * 2015-12-14 2022-06-07 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 脊髄性筋萎縮症の処置において有用なアデノ－関連ウイルスベクター

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Haug, S. et al., Archives of Dermatological Research, 2005, vol. 296, no. 12, pages 568-572 *
Hu, X.Y. et al., Genetics and Molecular Research, 2016, vol. 15, pages 1-13 *

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

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