AU2017355502B2 - Gene transfer compositions, methods and uses for treating neurodegenerative diseases - Google Patents

Gene transfer compositions, methods and uses for treating neurodegenerative diseases Download PDF

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Publication number
AU2017355502B2
AU2017355502B2 AU2017355502A AU2017355502A AU2017355502B2 AU 2017355502 B2 AU2017355502 B2 AU 2017355502B2 AU 2017355502 A AU2017355502 A AU 2017355502A AU 2017355502 A AU2017355502 A AU 2017355502A AU 2017355502 B2 AU2017355502 B2 AU 2017355502B2
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Australia
Prior art keywords
aav
mammal
tpp1
particles
polypeptide
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AU2017355502A
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AU2017355502A1 (en
Inventor
Yong Hong Chen
Beverly L. Davidson
Luis TECEDOR
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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365—Lactones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/12—Cyclic peptides, e.g. bacitracins; Polymyxins; Gramicidins S, C; Tyrocidins A, B or C
    • A61K38/13—Cyclosporins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Mycology (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2017355502A 2016-11-04 2017-11-03 Gene transfer compositions, methods and uses for treating neurodegenerative diseases Ceased AU2017355502B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662418033P 2016-11-04 2016-11-04
US62/418,033 2016-11-04
PCT/US2017/059986 WO2018085688A1 (en) 2016-11-04 2017-11-03 Gene transfer compositions, methods and uses for treating neurodegenerative diseases

Publications (2)

Publication Number Publication Date
AU2017355502A1 AU2017355502A1 (en) 2019-05-16
AU2017355502B2 true AU2017355502B2 (en) 2023-08-31

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AU2017355502A Ceased AU2017355502B2 (en) 2016-11-04 2017-11-03 Gene transfer compositions, methods and uses for treating neurodegenerative diseases

Country Status (9)

Country Link
US (1) US20190269797A1 (enExample)
EP (1) EP3534892A4 (enExample)
JP (2) JP2019537576A (enExample)
CN (1) CN110198712A (enExample)
AU (1) AU2017355502B2 (enExample)
BR (1) BR112019009074A2 (enExample)
CA (1) CA3041548A1 (enExample)
MX (1) MX2019005266A (enExample)
WO (1) WO2018085688A1 (enExample)

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US9818239B2 (en) 2015-08-20 2017-11-14 Zendrive, Inc. Method for smartphone-based accident detection
ES3065816T3 (en) 2017-06-07 2026-05-08 Regeneron Pharma Compositions and methods for internalizing enzymes
EP3698340A4 (en) 2017-10-20 2021-07-14 Zendrive, Inc. PROCEDURE AND SYSTEM FOR VEHICLE ASSOCIATED COMMUNICATION
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
BR112020023145A2 (pt) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto
SG11202103102WA (en) 2018-09-26 2021-04-29 California Inst Of Techn Adeno-associated virus compositions for targeted gene therapy
IL327339A (en) * 2018-11-14 2026-05-01 Regenxbio Inc Gene therapy for neural ceroid lipofuscinosis
CN113891712A (zh) * 2019-02-01 2022-01-04 星火治疗有限公司 用于晚期婴儿神经元蜡样脂褐质沉积症2型的aav载体治疗方法
EP4042297A4 (en) 2019-12-03 2023-11-22 Zendrive, Inc. METHOD AND SYSTEM FOR DETERMINING THE RISK OF A ROUTE
WO2021180118A1 (en) * 2020-03-11 2021-09-16 Shanghai Belief-Delivery Biomed Co., Ltd. Novel use of aspirin compound in increasing nucleic acid expression
WO2025231406A1 (en) * 2024-05-02 2025-11-06 The Children's Hospital Of Philadelphia Methods to increase transduction of ependyma cells in brain

Citations (5)

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WO2005120581A2 (en) * 2004-06-07 2005-12-22 Genzyme Corporation Gene therapy for neurometabolic disorders
WO2015077473A1 (en) * 2013-11-20 2015-05-28 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits
US20150151007A1 (en) * 2007-06-06 2015-06-04 Genzyme Corporation Gene therapy for lysosomal storage diseases
US20160166709A1 (en) * 2013-07-26 2016-06-16 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2017070678A1 (en) * 2015-10-23 2017-04-27 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection

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AU2003221745A1 (en) * 2002-04-19 2003-11-03 University Of Florida rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES
KR20070004637A (ko) * 2004-01-22 2007-01-09 가부시키가이샤 디나벡크 겐큐쇼 바이러스 벡터의 제조방법
ES2700048T3 (es) * 2006-02-08 2019-02-13 Genzyme Corp Terapia génica par la enfermedad de Niemann-Pick tipo A
CA2832151C (en) * 2011-03-31 2021-06-15 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
PT3024498T (pt) * 2013-07-22 2020-03-06 Childrens Hospital Philadelphia Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos
WO2015060722A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
BR112019023303A2 (pt) * 2017-05-11 2020-06-16 The Trustees Of The University Of Pennsylvania Terapia de gene para lipofuscinoses ceroides neuronais

Patent Citations (5)

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WO2005120581A2 (en) * 2004-06-07 2005-12-22 Genzyme Corporation Gene therapy for neurometabolic disorders
US20150151007A1 (en) * 2007-06-06 2015-06-04 Genzyme Corporation Gene therapy for lysosomal storage diseases
US20160166709A1 (en) * 2013-07-26 2016-06-16 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2015077473A1 (en) * 2013-11-20 2015-05-28 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits
WO2017070678A1 (en) * 2015-10-23 2017-04-27 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection

Non-Patent Citations (1)

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Title
RONALD G CRYSTAL, SONDHI DOLAN, PH D, HACKETT NEIL R, PH D, KAMINSKY STEPHEN M, PH D, WORGALL STEFAN, M D, STIEG PHILIP, M D, : "Administration of a Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinosis", HUMAN GENE THERAPY, MARY ANN LIEBERT, INC, vol. 15, no. 11, pages 1131 - 1154, XP055687428, DOI: 10.1089/hum.2004.15.1131 *

Also Published As

Publication number Publication date
JP2023002721A (ja) 2023-01-10
CN110198712A (zh) 2019-09-03
US20190269797A1 (en) 2019-09-05
EP3534892A4 (en) 2020-05-27
RU2019117062A3 (enExample) 2021-03-11
AU2017355502A1 (en) 2019-05-16
BR112019009074A2 (pt) 2019-07-16
EP3534892A1 (en) 2019-09-11
MX2019005266A (es) 2019-09-27
WO2018085688A1 (en) 2018-05-11
RU2019117062A (ru) 2020-12-04
CA3041548A1 (en) 2018-05-11
JP2019537576A (ja) 2019-12-26

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