DK1988823T3 - Langsom intraventrikulær administration - Google Patents

Langsom intraventrikulær administration Download PDF

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Publication number
DK1988823T3
DK1988823T3 DK07750236.7T DK07750236T DK1988823T3 DK 1988823 T3 DK1988823 T3 DK 1988823T3 DK 07750236 T DK07750236 T DK 07750236T DK 1988823 T3 DK1988823 T3 DK 1988823T3
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DK
Denmark
Prior art keywords
agent
disease
brain
patient
administration
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DK07750236.7T
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English (en)
Inventor
James Dodge
Marco Passini
Lamya Shihabuddin
Seng Cheng
Original Assignee
Genzyme Corp
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Publication of DK1988823T3 publication Critical patent/DK1988823T3/da

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
    • 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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P23/00—Anaesthetics
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Psychology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Molecular Biology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Pulmonology (AREA)

Claims (8)

1. Middel der er egnet til levering til en patients hjerne, ved anvendelse i en fremgangsmåde der omfatter at administrere midlet til patienten over en lateral ventrikel i hjernen med en sådan hastighed, at der til administration af en enkelt dosis forbruges mere end fire timer, hvilken administration udføres, indtil det nævnte middel kan detekteres i patientens serum, og hvor midlet er et enzym der mangler ved en lysosomal aflej ringssygdom.
2. Midlet ifølge krav 1, hvor midlet har adgang til den tredje ventrikel, Sylvius-akvedukten, den fjerde ventrikel, Luschka-Foramina, Magendie-Foramen, rygmarven, subarachnoid-rummet eller serummet.
3. Midlet ifølge krav 1 eller krav 2, hvor midlet er valgt fra gruppen, der består af sphingomyelinase, alfa-L-iduronidase, glucocerebrosidase, alfa-galactosidase A, syre-maltase, hexosaminidase og alfa-glucosidase.
4. Midlet ifølge et hvilket som helst af kravene 1 til 3, hvor patienten har en sygdom, som er valgt fra gruppen, der består af Niemann-Pick B-sygdom, Hurlers syndrom, Gauchers sygdom, Fabrys sygdom, Pompes sygdom, Tay-Sachs sygdom eller glycogen-aflejringssygdom type II.
5. Midlet ifølge krav 1, hvor hastigheden er sådan, at administrationen af en enkeltdosis forbruger mere end seks timer, mere end otte timer eller mere end ti timer.
6. Midlet ifølge krav 1, hvor hastigheden er sådan, at administrationen af en enkelt dosis forbruger op til ti timer.
7. Midlet ifølge krav 1, hvor hastigheden er sådan, at administrationen af en enkelt dosis forbruger seks til ti timer.
8. Midlet ifølge krav 1, hvor midlet er afgivet under anvendelse af et kateter, en pumpe eller en implanterbar pumpe.
DK07750236.7T 2006-02-09 2007-02-08 Langsom intraventrikulær administration DK1988823T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US77145106P 2006-02-09 2006-02-09
PCT/US2007/003382 WO2007095056A2 (en) 2006-02-09 2007-02-08 Slow intraventricular delivery

Publications (1)

Publication Number Publication Date
DK1988823T3 true DK1988823T3 (da) 2018-12-03

Family

ID=38372004

Family Applications (1)

Application Number Title Priority Date Filing Date
DK07750236.7T DK1988823T3 (da) 2006-02-09 2007-02-08 Langsom intraventrikulær administration

Country Status (19)

Country Link
US (3) US20090123451A1 (da)
EP (2) EP1988823B1 (da)
JP (4) JP2009526066A (da)
CN (1) CN101404927A (da)
AR (1) AR059372A1 (da)
BR (1) BRPI0707900A2 (da)
CA (2) CA2641359C (da)
CY (1) CY1121822T1 (da)
DK (1) DK1988823T3 (da)
ES (1) ES2697502T3 (da)
HU (1) HUE040490T2 (da)
IL (2) IL193242A (da)
LT (1) LT1988823T (da)
MX (3) MX358492B (da)
PL (1) PL1988823T3 (da)
PT (1) PT1988823T (da)
RU (1) RU2452368C2 (da)
SI (1) SI1988823T1 (da)
WO (1) WO2007095056A2 (da)

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AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
DK1988823T3 (da) 2006-02-09 2018-12-03 Genzyme Corp Langsom intraventrikulær administration
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
SI2158322T1 (sl) * 2007-06-06 2017-10-30 Genzyme Corporation Genska terapija za bolezni lizosomskega shranjevanja
CA2721134C (en) * 2007-06-13 2017-02-07 Wayne State University Board Of Governors A baclofen solution for low-volume therapeutic delivery
US8865182B2 (en) 2009-07-31 2014-10-21 Paxvax, Inc. Adenoviral-based vectors
PL3482767T3 (pl) 2009-08-28 2022-02-14 Icahn School Of Medicine At Mount Sinai Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy
ES2896060T3 (es) * 2010-06-25 2022-02-23 Shire Human Genetic Therapies Métodos y composiciones para la administración al SNC de arilsulfatasa A
MX2014015985A (es) 2012-06-19 2015-07-21 Univ Florida Composiciones y metodos para tratar enfermedades.
KR20220119187A (ko) * 2013-05-15 2022-08-26 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療

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SI2158322T1 (sl) 2007-06-06 2017-10-30 Genzyme Corporation Genska terapija za bolezni lizosomskega shranjevanja

Also Published As

Publication number Publication date
SI1988823T1 (sl) 2018-12-31
RU2008136196A (ru) 2010-03-20
EP3459441A1 (en) 2019-03-27
JP2018024664A (ja) 2018-02-15
MX358492B (es) 2018-08-22
JP6617125B2 (ja) 2019-12-11
MX2022005303A (es) 2022-05-16
IL248492A0 (en) 2016-12-29
CA2641359A1 (en) 2007-08-23
HUE040490T2 (hu) 2019-03-28
CN101404927A (zh) 2009-04-08
US20220370348A1 (en) 2022-11-24
LT1988823T (lt) 2018-12-10
US11253485B2 (en) 2022-02-22
JP6408970B2 (ja) 2018-10-17
MX361816B (es) 2018-12-17
PT1988823T (pt) 2018-11-22
JP2009526066A (ja) 2009-07-16
CA2641359C (en) 2022-10-04
ES2697502T3 (es) 2019-01-24
JP6129685B2 (ja) 2017-05-17
EP1988823A4 (en) 2009-02-11
JP2016028711A (ja) 2016-03-03
US20090123451A1 (en) 2009-05-14
PL1988823T3 (pl) 2019-01-31
IL193242A0 (en) 2009-08-03
AR059372A1 (es) 2008-03-26
RU2452368C2 (ru) 2012-06-10
CY1121822T1 (el) 2020-07-31
IL193242A (en) 2017-11-30
WO2007095056A3 (en) 2008-01-10
EP1988823B1 (en) 2018-08-15
CA3168881A1 (en) 2007-08-23
WO2007095056A2 (en) 2007-08-23
JP2014012694A (ja) 2014-01-23
BRPI0707900A2 (pt) 2011-05-10
US20190328682A1 (en) 2019-10-31
EP1988823A2 (en) 2008-11-12

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