SG10202100632WA - Gene therapy for the treatment of aldehyde dehydrogenase deficiency - Google Patents

Gene therapy for the treatment of aldehyde dehydrogenase deficiency

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Publication number
SG10202100632WA
SG10202100632WA SG10202100632WA SG10202100632WA SG10202100632WA SG 10202100632W A SG10202100632W A SG 10202100632WA SG 10202100632W A SG10202100632W A SG 10202100632WA SG 10202100632W A SG10202100632W A SG 10202100632WA SG 10202100632W A SG10202100632W A SG 10202100632WA
Authority
SG
Singapore
Prior art keywords
treatment
gene therapy
aldehyde dehydrogenase
dehydrogenase deficiency
deficiency
Prior art date
Application number
SG10202100632WA
Inventor
Mehdi Gasmi
Ronald G Crystal
Odelya E Pagovich
Original Assignee
Univ Cornell
Adverum Biotechnologies Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ Cornell, Adverum Biotechnologies Inc filed Critical Univ Cornell
Publication of SG10202100632WA publication Critical patent/SG10202100632WA/en

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    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
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    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
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    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P9/06Antiarrhythmics
    • AHUMAN NECESSITIES
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0006Oxidoreductases (1.) acting on CH-OH groups as donors (1.1)
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    • C12Q1/26Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving oxidoreductase
    • C12Q1/32Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving oxidoreductase involving dehydrogenase
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    • C12Y101/00Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/01Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
    • C12Y101/01001Alcohol dehydrogenase (1.1.1.1)
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/106Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
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    • C12Y102/00Oxidoreductases acting on the aldehyde or oxo group of donors (1.2)
    • C12Y102/01Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
    • C12Y102/01003Aldehyde dehydrogenase (NAD+) (1.2.1.3)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y401/00Carbon-carbon lyases (4.1)
    • C12Y401/01Carboxy-lyases (4.1.1)
    • C12Y401/01001Pyruvate decarboxylase (4.1.1.1)

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  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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SG10202100632WA 2016-07-26 2017-07-26 Gene therapy for the treatment of aldehyde dehydrogenase deficiency SG10202100632WA (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662367012P 2016-07-26 2016-07-26

Publications (1)

Publication Number Publication Date
SG10202100632WA true SG10202100632WA (en) 2021-03-30

Family

ID=61016742

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10202100632WA SG10202100632WA (en) 2016-07-26 2017-07-26 Gene therapy for the treatment of aldehyde dehydrogenase deficiency
SG11201900543SA SG11201900543SA (en) 2016-07-26 2017-07-26 Gene therapy for the treatment of aldehyde dehydrogenase deficiency

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201900543SA SG11201900543SA (en) 2016-07-26 2017-07-26 Gene therapy for the treatment of aldehyde dehydrogenase deficiency

Country Status (8)

Country Link
US (1) US20190160187A1 (en)
EP (1) EP3490613A4 (en)
JP (2) JP7165357B2 (en)
KR (1) KR102621134B1 (en)
CN (1) CN109952114A (en)
AU (1) AU2017301819A1 (en)
SG (2) SG10202100632WA (en)
WO (1) WO2018022783A1 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
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 (en) * 2020-03-12 2023-11-24 广州辉园苑医药科技有限公司 Acetaldehyde dehydrogenase recombinant gene, lactic acid bacteria carrier and application thereof
KR102557375B1 (en) * 2020-12-16 2023-07-20 동국대학교 와이즈캠퍼스 산학협력단 Peptides Derived from Mitochondrial Presequence and Uses thereof
CN114891808B (en) * 2022-06-21 2023-10-27 珠海丽凡达生物技术有限公司 mRNA molecule encoding ALDH2 polypeptide, application and mRNA medicament
GB202218090D0 (en) * 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
KR20110082180A (en) * 2008-10-28 2011-07-18 더 보드 오브 트러스티스 오브 더 리랜드 스탠포드 쥬니어 유니버시티 Modulators of aldehyde dehydrogenase and methods of use thereof
DK2561073T3 (en) * 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
JP6091435B2 (en) * 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 Protein delivery using adeno-associated virus (AAV) vectors
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
ES2877093T3 (en) * 2013-04-23 2021-11-16 Nizyme Inc Procedures and compositions for the treatment of diseases
PL3107899T3 (en) * 2014-02-19 2021-01-25 Aviv Therapeutics, Inc. Mitochondrial aldehyde dehydrogenase 2 (aldh2) binding polycyclic amides and their use for the treatment of cancer
CN109072254A (en) * 2015-12-14 2018-12-21 宾夕法尼亚州大学信托人 For treating the gland relevant viral vector of Duchenne-Arandisease
BR112018011838A2 (en) * 2015-12-14 2018-12-04 The Trustees Of The University Of Pennsylvania gene therapy for eye disorders

Also Published As

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

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