AU2011235890B2 - Methods for treating Parkinson's disease and other disorders of dopaminergic neurons of the brain - Google Patents

Methods for treating Parkinson's disease and other disorders of dopaminergic neurons of the brain Download PDF

Info

Publication number
AU2011235890B2
AU2011235890B2 AU2011235890A AU2011235890A AU2011235890B2 AU 2011235890 B2 AU2011235890 B2 AU 2011235890B2 AU 2011235890 A AU2011235890 A AU 2011235890A AU 2011235890 A AU2011235890 A AU 2011235890A AU 2011235890 B2 AU2011235890 B2 AU 2011235890B2
Authority
AU
Australia
Prior art keywords
growth factor
disease
nerve growth
substantia nigra
striatum
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.)
Withdrawn - After Issue
Application number
AU2011235890A
Other languages
English (en)
Other versions
AU2011235890A1 (en
Inventor
Raymond T. Bartus
Joao Siffert
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.)
Ceregene Inc
Original Assignee
Ceregene 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 Ceregene Inc filed Critical Ceregene Inc
Publication of AU2011235890A1 publication Critical patent/AU2011235890A1/en
Application granted granted Critical
Publication of AU2011235890B2 publication Critical patent/AU2011235890B2/en
Withdrawn - After Issue legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/48—Nerve growth factor [NGF]
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Psychology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2011235890A 2010-04-02 2011-04-01 Methods for treating Parkinson's disease and other disorders of dopaminergic neurons of the brain Withdrawn - After Issue AU2011235890B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US32065410P 2010-04-02 2010-04-02
US61/320,654 2010-04-02
PCT/US2011/031027 WO2011123842A2 (en) 2010-04-02 2011-04-01 Methods for treating parkinson's disease and other disorders of dopaminergic neurons of the brain

Publications (2)

Publication Number Publication Date
AU2011235890A1 AU2011235890A1 (en) 2012-09-27
AU2011235890B2 true AU2011235890B2 (en) 2014-07-31

Family

ID=44712866

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2011235890A Withdrawn - After Issue AU2011235890B2 (en) 2010-04-02 2011-04-01 Methods for treating Parkinson's disease and other disorders of dopaminergic neurons of the brain

Country Status (7)

Country Link
US (1) US20120082650A1 (enExample)
EP (1) EP2552473A4 (enExample)
JP (1) JP2013523776A (enExample)
CN (1) CN102821781A (enExample)
AU (1) AU2011235890B2 (enExample)
CA (1) CA2793692A1 (enExample)
WO (1) WO2011123842A2 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017011519A1 (en) * 2015-07-13 2017-01-19 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
BR112020021903A2 (pt) 2018-04-27 2021-03-02 Rocket Pharmaceuticals, Ltd. terapia genética para degeneração do snc
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
IL322484A (en) 2023-02-03 2025-09-01 Janssen Pharmaceutica Nv Gene therapy vectors for use in Parkinson's disease

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020187951A1 (en) * 2000-11-09 2002-12-12 Patrick Aebischer Lentiviral-mediated growth factor gene therapy for neurodegenerative diseases

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060239966A1 (en) * 2003-10-20 2006-10-26 Tornoee Jens In vivo gene therapy of parkinson's disease

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020187951A1 (en) * 2000-11-09 2002-12-12 Patrick Aebischer Lentiviral-mediated growth factor gene therapy for neurodegenerative diseases

Also Published As

Publication number Publication date
JP2013523776A (ja) 2013-06-17
CN102821781A (zh) 2012-12-12
CA2793692A1 (en) 2011-10-06
WO2011123842A2 (en) 2011-10-06
EP2552473A2 (en) 2013-02-06
WO2011123842A9 (en) 2012-01-12
AU2011235890A1 (en) 2012-09-27
US20120082650A1 (en) 2012-04-05
EP2552473A4 (en) 2013-10-23

Similar Documents

Publication Publication Date Title
Domanskyi et al. Prospects of neurotrophic factors for Parkinson's disease: comparison of protein and gene therapy
Leone et al. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease
JP2020079234A (ja) 筋萎縮性側索硬化症および他の脊髄障害のための遺伝子治療
Federici et al. Gene‐based treatment of motor neuron diseases
JP2020050660A (ja) 脊髄疾患の遺伝子療法
US10017553B2 (en) Splice variants of GDNF and uses thereof
JP2013006846A (ja) 脳の神経変性疾患の治療法
AU2011235890B2 (en) Methods for treating Parkinson's disease and other disorders of dopaminergic neurons of the brain
Kakoty et al. Unraveling the role of glial cell line–derived neurotrophic factor in the treatment of Parkinson’s disease
JP6957614B2 (ja) 疼痛を軽減または処置する組成物
CN114516901B (zh) 一种神经系统高亲和性的aav载体及其应用
CN101014359A (zh) 通过将神经胶质细胞系来源的神经营养因子直接注入到未定带内治疗人帕金森病的方法
US20230330267A1 (en) Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain
Melchior et al. Ectopic expression of the TrkA receptor in adult dopaminergic mesencephalic neurons promotes retrograde axonal NGF transport and NGF-dependent neuroprotection
CA2266619A1 (en) Non-invasive administration of adeno-associated viral vectors
KR20250097939A (ko) 투여 경로
WO2023275558A1 (en) Delivery of gene therapies into muscle via the central nervous system
Kirik et al. Delivery of GDNF for Parkinson's disease: transition of a neuroprotective treatment strategy from basic sciences to clinical application
WO2024175604A1 (en) Methods and pharmaceutical composition for the treatment of sanfilippo syndrome type iiib
JP2023554198A (ja) 発現ベクター組成物
KIRIK et al. Neuroprotective Treatment Strategy
Leone et al. Aspartoacylase Gene Transfer to the Mammalian Central Nervous System with Therapeutic Implications for
Zhang et al. Advancing Parkinson's Prevention: Brain-Specific Lipopolyplex Delivery of Gdnf as a Neuroprotective Gene Therapy
HK1247867A1 (zh) 脊髓软膜下基因递送系统
AU2007203173A1 (en) Genetic vectors for increasing or decreasing innervation

Legal Events

Date Code Title Description
MK12 Application lapsed section 141(1)/reg 8.3(2) - applicant filed a written notice of withdrawal