PL2497500T3 - Terapia genowa rdzeniowego zaniku mięśni - Google Patents

Terapia genowa rdzeniowego zaniku mięśni

Info

Publication number
PL2497500T3
PL2497500T3 PL12157957T PL12157957T PL2497500T3 PL 2497500 T3 PL2497500 T3 PL 2497500T3 PL 12157957 T PL12157957 T PL 12157957T PL 12157957 T PL12157957 T PL 12157957T PL 2497500 T3 PL2497500 T3 PL 2497500T3
Authority
PL
Poland
Prior art keywords
gene therapy
muscular atrophy
spinal muscular
spinal
atrophy
Prior art date
Application number
PL12157957T
Other languages
English (en)
Inventor
Catherine O'riordan
Samuel Wadsworth
Original Assignee
Genzyme Corporation
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 Genzyme Corporation filed Critical Genzyme Corporation
Publication of PL2497500T3 publication Critical patent/PL2497500T3/pl

Links

Classifications

    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • 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
    • A61P25/28—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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PL12157957T 2006-10-03 2007-10-03 Terapia genowa rdzeniowego zaniku mięśni PL2497500T3 (pl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US82797706P 2006-10-03 2006-10-03
EP12157957.7A EP2497500B1 (en) 2006-10-03 2007-10-03 Gene therapy for spinal muscular atrophy

Publications (1)

Publication Number Publication Date
PL2497500T3 true PL2497500T3 (pl) 2017-07-31

Family

ID=39269037

Family Applications (2)

Application Number Title Priority Date Filing Date
PL07839216T PL2066791T3 (pl) 2006-10-03 2007-10-03 Terapia Genowa do leczenia stwardnienia zanikowego bocznego i innych zaburzeń rdzenia kręgowego
PL12157957T PL2497500T3 (pl) 2006-10-03 2007-10-03 Terapia genowa rdzeniowego zaniku mięśni

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PL07839216T PL2066791T3 (pl) 2006-10-03 2007-10-03 Terapia Genowa do leczenia stwardnienia zanikowego bocznego i innych zaburzeń rdzenia kręgowego

Country Status (13)

Country Link
US (2) US9890394B2 (pl)
EP (4) EP3146982B1 (pl)
CN (3) CN105770910A (pl)
AR (1) AR063113A1 (pl)
CY (1) CY1118477T1 (pl)
DK (2) DK2497500T3 (pl)
ES (3) ES2391789T3 (pl)
HU (2) HUE031182T2 (pl)
LT (2) LT3146982T (pl)
PL (2) PL2066791T3 (pl)
PT (3) PT3146982T (pl)
SI (3) SI2066791T1 (pl)
WO (1) WO2008042420A2 (pl)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105770910A (zh) 2006-10-03 2016-07-20 建新公司 肌萎缩性侧索硬化症和其它脊髓病症的基因治疗
WO2009090933A1 (ja) * 2008-01-16 2009-07-23 Japan Science And Technology Agency 低酸素状態にある細胞で増殖するウイルスまたは遺伝子を発現するウイルスベクター
MX356669B (es) 2009-05-02 2018-06-08 Genzyme Corp Terapia genica para trastornos neurodegenerativos.
WO2013025963A2 (en) 2011-08-17 2013-02-21 President And Fellows Of Harvard College Conversion of somatic cells into functional spinal motor neurons, and methods and uses thereof
CN107109407A (zh) 2014-11-14 2017-08-29 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
SG10202001102XA (en) 2014-11-14 2020-03-30 Voyager Therapeutics Inc Modulatory polynucleotides
WO2016122791A1 (en) * 2015-01-30 2016-08-04 The Regents Of The University Of California Spinal subpial gene delivery system
CL2015003024A1 (es) 2015-10-09 2016-05-13 Univ Chile Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrofica.
CN108884022B (zh) 2016-03-28 2022-01-28 加利福尼亚大学董事会 用于治疗神经元的过度兴奋的方法和组合物
EP3458588B1 (en) 2016-05-18 2025-10-29 Voyager Therapeutics, Inc. Modulatory polynucleotides
KR102641846B1 (ko) * 2016-11-17 2024-02-27 네이션와이드 칠드런스 하스피탈 인코포레이티드 메틸-CpG 결합 단백질 2를 암호화하는 재조합 아데노-관련 바이러스의 경막 내 전달
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2019034723A1 (en) * 2017-08-16 2019-02-21 Lgv1 S.R.L. VTFT ISOFORM OF BPIFB4 PROTEIN INTENDED FOR USE IN NEURONAL DISEASES AND INJURIES
US11707535B2 (en) 2017-09-08 2023-07-25 The Regents Of The University Of California Method and composition for treating neuropathic pain
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CA3123293A1 (en) 2019-01-10 2020-07-16 The Regents Of The University Of California Subpial delivery system and methods of use
USD963164S1 (en) 2020-01-09 2022-09-06 The Regents Of The University Of California Surgical needle
WO2023288086A2 (en) * 2021-07-16 2023-01-19 President And Fellows Of Harvard College Enhancers driving expression in motor neurons

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SE8303626D0 (sv) 1983-06-23 1983-06-23 Kabigen Ab A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced
US5672344A (en) 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
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CN105770910A (zh) 2006-10-03 2016-07-20 建新公司 肌萎缩性侧索硬化症和其它脊髓病症的基因治疗
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Also Published As

Publication number Publication date
US10920245B2 (en) 2021-02-16
PL2066791T3 (pl) 2013-02-28
CN105770910A (zh) 2016-07-20
WO2008042420A2 (en) 2008-04-10
DK2066791T3 (da) 2012-10-15
US9890394B2 (en) 2018-02-13
PT2497500T (pt) 2016-11-23
PT2066791E (pt) 2012-12-05
DK2497500T3 (da) 2017-01-02
HUE046709T2 (hu) 2020-03-30
US20180230490A1 (en) 2018-08-16
CN101528922A (zh) 2009-09-09
CY1118477T1 (el) 2017-07-12
CN108853517A (zh) 2018-11-23
EP2066791A2 (en) 2009-06-10
PT3146982T (pt) 2019-10-31
WO2008042420A3 (en) 2008-07-03
EP3146982A1 (en) 2017-03-29
SI2066791T1 (sl) 2013-01-31
SI3146982T1 (sl) 2019-11-29
EP2497500B1 (en) 2016-09-28
LT3146982T (lt) 2019-11-11
AR063113A1 (es) 2008-12-30
ES2607060T3 (es) 2017-03-29
US20090286857A1 (en) 2009-11-19
EP2497500A1 (en) 2012-09-12
EP2066791B1 (en) 2012-09-12
HUE031182T2 (en) 2017-06-28
ES2391789T3 (es) 2012-11-29
EP2066791A4 (en) 2010-11-03
EP3146982B1 (en) 2019-08-21
EP3632472A1 (en) 2020-04-08
ES2757723T3 (es) 2020-04-30
LT2497500T (lt) 2016-12-27
SI2497500T1 (sl) 2017-01-31

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