SI2497500T1 - Genska terapija za spinalno mišično atrofijo - Google Patents

Genska terapija za spinalno mišično atrofijo

Info

Publication number
SI2497500T1
SI2497500T1 SI200731873A SI200731873A SI2497500T1 SI 2497500 T1 SI2497500 T1 SI 2497500T1 SI 200731873 A SI200731873 A SI 200731873A SI 200731873 A SI200731873 A SI 200731873A SI 2497500 T1 SI2497500 T1 SI 2497500T1
Authority
SI
Slovenia
Prior art keywords
gene therapy
muscular atrophy
spinal muscular
spinal
atrophy
Prior art date
Application number
SI200731873A
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 SI2497500T1 publication Critical patent/SI2497500T1/sl

Links

Classifications

    • 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
    • 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
    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
SI200731873A 2006-10-03 2007-10-03 Genska terapija za spinalno mišično atrofijo SI2497500T1 (sl)

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
SI2497500T1 true SI2497500T1 (sl) 2017-01-31

Family

ID=39269037

Family Applications (3)

Application Number Title Priority Date Filing Date
SI200731083T SI2066791T1 (sl) 2006-10-03 2007-10-03 Genska terapija za amiotrofično lateralno sklerozo in druge motnje hrbtenjače
SI200731873A SI2497500T1 (sl) 2006-10-03 2007-10-03 Genska terapija za spinalno mišično atrofijo
SI200732128T SI3146982T1 (sl) 2006-10-03 2007-10-03 Genska terapija za amiotrofično lateralno sklerozo in druge motnje hrbtenjače

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SI200731083T SI2066791T1 (sl) 2006-10-03 2007-10-03 Genska terapija za amiotrofično lateralno sklerozo in druge motnje hrbtenjače

Family Applications After (1)

Application Number Title Priority Date Filing Date
SI200732128T SI3146982T1 (sl) 2006-10-03 2007-10-03 Genska terapija za amiotrofično lateralno sklerozo in druge motnje hrbtenjače

Country Status (13)

Country Link
US (2) US9890394B2 (sl)
EP (4) EP2497500B1 (sl)
CN (3) CN101528922A (sl)
AR (1) AR063113A1 (sl)
CY (1) CY1118477T1 (sl)
DK (2) DK2066791T3 (sl)
ES (3) ES2607060T3 (sl)
HU (2) HUE046709T2 (sl)
LT (2) LT3146982T (sl)
PL (2) PL2066791T3 (sl)
PT (3) PT2497500T (sl)
SI (3) SI2066791T1 (sl)
WO (1) WO2008042420A2 (sl)

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* Cited by examiner, † Cited by third party
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SI2066791T1 (sl) 2006-10-03 2013-01-31 Genzyme Corporation Genska terapija za amiotrofično lateralno sklerozo in druge motnje hrbtenjače
JP5186693B2 (ja) * 2008-01-16 2013-04-17 独立行政法人科学技術振興機構 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
SI2424991T1 (sl) 2009-05-02 2018-11-30 Genzyme Corporation Genska terapija za nevrodegenerativne motnje
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
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
CA2975447C (en) * 2015-01-30 2021-02-23 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.
CA3018960C (en) 2016-03-28 2023-09-26 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability
KR102392236B1 (ko) 2016-05-18 2022-05-03 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MA46863A (fr) * 2016-11-17 2019-09-25 Kevin Foust Administration intrathécale de virus adéno-associé recombinant codant pour la protéine 2 de liaison méthyl-cpg
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP2020531047A (ja) * 2017-08-16 2020-11-05 エッレジヴ１・ソチエタ・ア・レスポンサビリタ・リミタータ 神経疾患及び神経損傷における使用のためのｂｐｉｆｂ４タンパク質のｖｔｆｔアイソフォーム
US11707535B2 (en) 2017-09-08 2023-07-25 The Regents Of The University Of California Method and composition for treating neuropathic pain
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JP7502991B2 (ja) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症（ａｌｓ）の治療
US10980566B2 (en) 2019-01-10 2021-04-20 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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SI2066791T1 (sl) 2006-10-03 2013-01-31 Genzyme Corporation Genska terapija za amiotrofično lateralno sklerozo in druge motnje hrbtenjače
PT2773354T (pt) 2011-11-01 2019-07-17 Resverlogix Corp Formulações orais de libertação imediata para quinazolinonas substituídas

Also Published As

Publication number Publication date
PT2066791E (pt) 2012-12-05
US20180230490A1 (en) 2018-08-16
CY1118477T1 (el) 2017-07-12
PT2497500T (pt) 2016-11-23
PL2066791T3 (pl) 2013-02-28
EP2497500B1 (en) 2016-09-28
CN105770910A (zh) 2016-07-20
EP3146982A1 (en) 2017-03-29
ES2607060T3 (es) 2017-03-29
LT3146982T (lt) 2019-11-11
HUE031182T2 (en) 2017-06-28
PT3146982T (pt) 2019-10-31
HUE046709T2 (hu) 2020-03-30
DK2497500T3 (da) 2017-01-02
CN108853517A (zh) 2018-11-23
DK2066791T3 (da) 2012-10-15
WO2008042420A2 (en) 2008-04-10
SI3146982T1 (sl) 2019-11-29
EP2066791B1 (en) 2012-09-12
EP2066791A4 (en) 2010-11-03
CN101528922A (zh) 2009-09-09
ES2391789T3 (es) 2012-11-29
EP2497500A1 (en) 2012-09-12
ES2757723T3 (es) 2020-04-30
AR063113A1 (es) 2008-12-30
EP3146982B1 (en) 2019-08-21
EP3632472A1 (en) 2020-04-08
US20090286857A1 (en) 2009-11-19
US9890394B2 (en) 2018-02-13
LT2497500T (lt) 2016-12-27
US10920245B2 (en) 2021-02-16
PL2497500T3 (pl) 2017-07-31
SI2066791T1 (sl) 2013-01-31
EP2066791A2 (en) 2009-06-10
WO2008042420A3 (en) 2008-07-03

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