AU2020236735A1 - Gene therapy compositions and methods for treating parkinson's disease - Google Patents

Gene therapy compositions and methods for treating parkinson's disease Download PDF

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Publication number
AU2020236735A1
AU2020236735A1 AU2020236735A AU2020236735A AU2020236735A1 AU 2020236735 A1 AU2020236735 A1 AU 2020236735A1 AU 2020236735 A AU2020236735 A AU 2020236735A AU 2020236735 A AU2020236735 A AU 2020236735A AU 2020236735 A1 AU2020236735 A1 AU 2020236735A1
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AU
Australia
Prior art keywords
subject
nucleotide sequence
vector
administration
aadc
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
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AU2020236735A
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English (en)
Inventor
Gavin CORCORAN
Kyriacos Mitrophanous
Hannah STEWART
Michele Stone
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Oxford Biomedica UK Ltd
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Oxford Biomedica UK Ltd
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Publication date
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Publication of AU2020236735A1 publication Critical patent/AU2020236735A1/en
Assigned to OXFORD BIOMEDICA (UK) LIMITED reassignment OXFORD BIOMEDICA (UK) LIMITED Request for Assignment Assignors: OXFORD BIOMEDICA (UK) LIMITED, Sio Gene Therapies Inc.
Abandoned legal-status Critical Current

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Classifications

    • 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
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44Oxidoreductases (1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16002Tyrosine 3-monooxygenase (1.14.16.2)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04016GTP cyclohydrolase I (3.5.4.16)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y401/00Carbon-carbon lyases (4.1)
    • C12Y401/01Carboxy-lyases (4.1.1)
    • C12Y401/01028Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00Modified animals
    • A01K2207/20Animals treated with compounds which are neither proteins nor nucleic acids
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/106Primate
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • A01K2267/0318Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6072Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses
    • C12N2810/6081Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses rhabdoviridae, e.g. VSV

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2020236735A 2019-03-10 2020-03-10 Gene therapy compositions and methods for treating parkinson's disease Abandoned AU2020236735A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962816170P 2019-03-10 2019-03-10
US62/816,170 2019-03-10
US201962871007P 2019-07-05 2019-07-05
US62/871,007 2019-07-05
PCT/IB2020/052073 WO2020183374A1 (en) 2019-03-10 2020-03-10 Gene therapy compositions and methods for treating parkinson's disease

Publications (1)

Publication Number Publication Date
AU2020236735A1 true AU2020236735A1 (en) 2021-10-14

Family

ID=70057171

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020236735A Abandoned AU2020236735A1 (en) 2019-03-10 2020-03-10 Gene therapy compositions and methods for treating parkinson's disease

Country Status (8)

Country Link
US (1) US20220175962A1 (enExample)
EP (1) EP3937985A1 (enExample)
JP (1) JP2022525136A (enExample)
KR (1) KR20220034719A (enExample)
CN (1) CN115835887A (enExample)
AU (1) AU2020236735A1 (enExample)
CA (1) CA3133188A1 (enExample)
WO (1) WO2020183374A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024251679A1 (fr) * 2023-06-06 2024-12-12 Centre D'etude Des Cellules Souches (Cecs) Vecteur lentiviral pour la synthese de dopamine

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US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
DE4006630A1 (de) 1990-03-03 1991-09-12 Behringwerke Ag Humane monoklonale antikoerper gegen tollwutviren, ihre herstellung und verwendung
US5512421A (en) 1991-02-19 1996-04-30 The Regents Of The University Of California Generation, concentration and efficient transfer of VSV-G pseudotyped retroviral vectors
EP0796657B1 (en) 1991-06-21 2002-01-16 Phillips Petroleum Company Alkylation catalyst containing hydrofluoric acid and a sulfone
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
PT904392E (pt) 1996-10-17 2001-06-29 Oxford Biomedica Ltd Vectores retrovirais
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
DE69941049D1 (de) 1998-05-22 2009-08-13 Oxford Biomedica Ltd Retrovirales verabreichungssystem
GB9904905D0 (en) 1999-03-03 1999-04-28 Oxford Biomedica Ltd Cells
JP2003504541A (ja) 1999-07-13 2003-02-04 ペリセル カルロス フラデラ 建築パネル及びその製造設備
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
GB0024550D0 (enExample) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
SI1504108T1 (sl) 2002-02-01 2013-07-31 Oxford Biomedica (Uk) Limited Lentiviralni vektor
SE0302509D0 (sv) 2003-09-19 2003-09-19 Amersham Biosciences Ab Matrix for separation of polyethers and method of separation
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
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GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
DK2307551T3 (en) 2008-06-18 2017-03-20 Oxford Biomedica (Uk) Ltd CLEANING RETROVIRAL VECTORS
WO2011148194A1 (en) * 2010-05-28 2011-12-01 Oxford Biomedica (Uk) Ltd Delivery of lentiviral vectors to the brain
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Also Published As

Publication number Publication date
US20220175962A1 (en) 2022-06-09
KR20220034719A (ko) 2022-03-18
CA3133188A1 (en) 2020-09-17
CN115835887A (zh) 2023-03-21
JP2022525136A (ja) 2022-05-11
WO2020183374A1 (en) 2020-09-17
EP3937985A1 (en) 2022-01-19

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Free format text: THE NATURE OF THE AMENDMENT IS: TO AMEND THE NAME OF THE CO-APPLICANT TO READ OXFORD BIOMEDICA (UK) LIMITED

PC1 Assignment before grant (sect. 113)

Owner name: OXFORD BIOMEDICA (UK) LIMITED

Free format text: FORMER APPLICANT(S): OXFORD BIOMEDICA (UK) LIMITED; SIO GENE THERAPIES INC.

MK1 Application lapsed section 142(2)(a) - no request for examination in relevant period