BRPI0415563A - método para o tratamento do mal de parkinson, uso de um vetor viral, vetor de expressão viral, composição farmacêutica, célula hospedeira isolada, linhagem de células de empacotamento, mamìfero não humano quimérico, dispositivo de cultura celular implantável, cápsula biocompatìvel, método para tratamento de uma doença do sistema nervoso, célula de mamìfero, e, método para produzir neurturin ou um seu equivalente funcional - Google Patents

método para o tratamento do mal de parkinson, uso de um vetor viral, vetor de expressão viral, composição farmacêutica, célula hospedeira isolada, linhagem de células de empacotamento, mamìfero não humano quimérico, dispositivo de cultura celular implantável, cápsula biocompatìvel, método para tratamento de uma doença do sistema nervoso, célula de mamìfero, e, método para produzir neurturin ou um seu equivalente funcional

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Publication number
BRPI0415563A
BRPI0415563A BRPI0415563-7A BRPI0415563A BRPI0415563A BR PI0415563 A BRPI0415563 A BR PI0415563A BR PI0415563 A BRPI0415563 A BR PI0415563A BR PI0415563 A BRPI0415563 A BR PI0415563A
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BR
Brazil
Prior art keywords
disease
cell
neurturin
viral
treatment
Prior art date
Application number
BRPI0415563-7A
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English (en)
Inventor
Jens Tornoe
Carl Rosenblad
Lars Wahlberg
Original Assignee
Nsgene As
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=34524307&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=BRPI0415563(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Nsgene As filed Critical Nsgene As
Publication of BRPI0415563A publication Critical patent/BRPI0415563A/pt

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    • 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
    • A61K48/005—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2750/14141—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)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychology (AREA)
  • General Engineering & Computer Science (AREA)
  • Toxicology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Hospice & Palliative Care (AREA)
  • Physics & Mathematics (AREA)
  • Psychiatry (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)

Abstract

"MéTODO PARA O TRATAMENTO DO MAL DE PARKINSON USO DE UM VETOR VIRAL, VETOR DE EXPRESSãO VIRAL, COMPOSIçãO FARMACêUTICA, CéLULA HOSPEDEIRA ISOLADA, LINHAGEM DE CéLULAS DE EMPACOTAMENTO, MAMìFERO NãO HUMANO QUIMéRICO, DISPOSITIVO DE CULTURA CELULAR IMPLANTáVEL, CáPSULA BIOCOMPATìVEL, MéTODO PARA TRATAMENTO DE UMA DOENçA DO SISTEMA NERVOSO, CéLULA DE MAMìFERO E MéTODO PARA PRODUZIR NEURTURIN OU UM SEU EQUIVALENTE FUNCIONAL". A presente invenção diz respeito a métodos e composições para a terapia de genes, em particular a terapia de genes in vivo para liberação de Neurturin bioativo para o tratamento do mal de Parkinson. Em outro aspecto, a invenção diz respeito a construções de expressão viral compreendendo um peptídeo sinal ligado a um Neurturin maduro ou N-terminalmente truncado sem uma pró-região funcional entre o peptídeo sinal e o Neurturin. Estas construções de expressão viral são necessárias para a eficiente secreção do Neurturin bioativo na terapia de genes in vivo. A invenção também diz respeito a células de mamíferos capazes de produzir Neurturin em quantidades aumentadas, bem como ao uso destas células para produção recombinante do Neurturin bioativo e para uso terapêutico.
BRPI0415563-7A 2003-10-20 2004-10-20 método para o tratamento do mal de parkinson, uso de um vetor viral, vetor de expressão viral, composição farmacêutica, célula hospedeira isolada, linhagem de células de empacotamento, mamìfero não humano quimérico, dispositivo de cultura celular implantável, cápsula biocompatìvel, método para tratamento de uma doença do sistema nervoso, célula de mamìfero, e, método para produzir neurturin ou um seu equivalente funcional BRPI0415563A (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
DKPA200301543 2003-10-20
US51291803P 2003-10-22 2003-10-22
PCT/EP2004/052586 WO2005039643A2 (en) 2003-10-20 2004-10-20 In vivo gene therapy of parkinson's disease

Publications (1)

Publication Number Publication Date
BRPI0415563A true BRPI0415563A (pt) 2007-01-02

Family

ID=34524307

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0415563-7A BRPI0415563A (pt) 2003-10-20 2004-10-20 método para o tratamento do mal de parkinson, uso de um vetor viral, vetor de expressão viral, composição farmacêutica, célula hospedeira isolada, linhagem de células de empacotamento, mamìfero não humano quimérico, dispositivo de cultura celular implantável, cápsula biocompatìvel, método para tratamento de uma doença do sistema nervoso, célula de mamìfero, e, método para produzir neurturin ou um seu equivalente funcional

Country Status (14)

Country Link
EP (2) EP2210617A1 (pt)
JP (1) JP2007509109A (pt)
KR (1) KR20060095568A (pt)
AT (1) ATE460183T1 (pt)
AU (1) AU2004283053A1 (pt)
BR (1) BRPI0415563A (pt)
CA (1) CA2543189A1 (pt)
DE (1) DE602004025959D1 (pt)
DK (1) DK1677833T3 (pt)
ES (1) ES2342397T3 (pt)
MX (1) MXPA06004329A (pt)
NZ (1) NZ547185A (pt)
RU (1) RU2006117304A (pt)
WO (1) WO2005039643A2 (pt)

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JP2007509109A (ja) 2007-04-12
WO2005039643A3 (en) 2005-11-17
ATE460183T1 (de) 2010-03-15
ES2342397T3 (es) 2010-07-06
EP1677833A2 (en) 2006-07-12
DE602004025959D1 (de) 2010-04-22
RU2006117304A (ru) 2007-12-10
MXPA06004329A (es) 2006-06-05
CA2543189A1 (en) 2005-05-06
EP2210617A1 (en) 2010-07-28
WO2005039643A2 (en) 2005-05-06
KR20060095568A (ko) 2006-08-31
EP1677833B1 (en) 2010-03-10
DK1677833T3 (da) 2010-05-17
AU2004283053A1 (en) 2005-05-06
NZ547185A (en) 2009-03-31

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