AU2011270672B2 - Treatment of Sanfilippo Syndrome type B - Google Patents

Treatment of Sanfilippo Syndrome type B Download PDF

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Publication number
AU2011270672B2
AU2011270672B2 AU2011270672A AU2011270672A AU2011270672B2 AU 2011270672 B2 AU2011270672 B2 AU 2011270672B2 AU 2011270672 A AU2011270672 A AU 2011270672A AU 2011270672 A AU2011270672 A AU 2011270672A AU 2011270672 B2 AU2011270672 B2 AU 2011270672B2
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AU
Australia
Prior art keywords
naglu
protein
brain
nmol
lysosomal
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.)
Ceased
Application number
AU2011270672A
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English (en)
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AU2011270672A1 (en
Inventor
Mary Alessandrini
Pericles Calias
Michael F. Concino
Kevin Holmes
Andrea Iskenderian
Dianna Lundberg
Paolo Martini
Muthuraman Meiyappan
Angela Norton
Jing Pan
Richard Pfeifer
Alla Romashko
Bettina Strack-Logue
Huang Yan
Bohong Zhang
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.)
Shire Human Genetics Therapies Inc
Original Assignee
Shire Human Genetics Therapies Inc
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Filing date
Publication date
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Publication of AU2011270672A1 publication Critical patent/AU2011270672A1/en
Application granted granted Critical
Publication of AU2011270672B2 publication Critical patent/AU2011270672B2/en
Priority to AU2017202344A priority Critical patent/AU2017202344A1/en
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

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    • 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
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • A61P25/20—Hypnotics; Sedatives
    • 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
    • A61P27/16—Otologicals
    • 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
    • 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
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Psychology (AREA)
  • Inorganic Chemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Psychiatry (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Anesthesiology (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
AU2011270672A 2010-06-25 2011-06-25 Treatment of Sanfilippo Syndrome type B Ceased AU2011270672B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2017202344A AU2017202344A1 (en) 2010-06-25 2017-04-10 Treatment of sanfilippo syndrome type b

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US35885710P 2010-06-25 2010-06-25
US61/358,857 2010-06-25
US36078610P 2010-07-01 2010-07-01
US61/360,786 2010-07-01
US38786210P 2010-09-29 2010-09-29
US61/387,862 2010-09-29
US201161435710P 2011-01-24 2011-01-24
US61/435,710 2011-01-24
US201161442115P 2011-02-11 2011-02-11
US61/442,115 2011-02-11
US201161476210P 2011-04-15 2011-04-15
US61/476,210 2011-04-15
US201161495268P 2011-06-09 2011-06-09
PCT/US2011/041928 WO2011163652A2 (en) 2010-06-25 2011-06-25 Treatment of sanfilippo syndrome type b
US61/495,268 2012-06-09

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2017202344A Division AU2017202344A1 (en) 2010-06-25 2017-04-10 Treatment of sanfilippo syndrome type b

Publications (2)

Publication Number Publication Date
AU2011270672A1 AU2011270672A1 (en) 2013-02-14
AU2011270672B2 true AU2011270672B2 (en) 2017-01-12

Family

ID=46888842

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2011270672A Ceased AU2011270672B2 (en) 2010-06-25 2011-06-25 Treatment of Sanfilippo Syndrome type B
AU2017202344A Abandoned AU2017202344A1 (en) 2010-06-25 2017-04-10 Treatment of sanfilippo syndrome type b

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2017202344A Abandoned AU2017202344A1 (en) 2010-06-25 2017-04-10 Treatment of sanfilippo syndrome type b

Country Status (11)

Country Link
US (1) US20110318327A1 (cg-RX-API-DMAC10.html)
EP (1) EP2585105B1 (cg-RX-API-DMAC10.html)
JP (1) JP6063380B2 (cg-RX-API-DMAC10.html)
KR (1) KR20130043166A (cg-RX-API-DMAC10.html)
CN (1) CN103269709A (cg-RX-API-DMAC10.html)
AU (2) AU2011270672B2 (cg-RX-API-DMAC10.html)
CA (1) CA2805449A1 (cg-RX-API-DMAC10.html)
ES (1) ES2643015T3 (cg-RX-API-DMAC10.html)
MX (1) MX2013000324A (cg-RX-API-DMAC10.html)
NZ (2) NZ702801A (cg-RX-API-DMAC10.html)
WO (1) WO2011163652A2 (cg-RX-API-DMAC10.html)

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WO2009137721A2 (en) 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
JP2013507131A (ja) 2009-10-09 2013-03-04 アーメイゲン・テクノロジーズ・インコーポレイテッド Ｃｎｓにおけるイズロン酸２−スルファターゼ活性を増加させるための方法および組成物
CA2803166C (en) 2010-06-25 2023-03-07 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
JP6045493B2 (ja) 2010-06-25 2016-12-14 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド アリールスルファターゼａのｃｎｓ送達の方法および組成物
JP6073783B2 (ja) 2010-06-25 2017-02-01 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物
BR112012033214B1 (pt) 2010-06-25 2020-11-03 Shire Human Genetic Therapies, Inc. uso de uma formulação estável compreendendo uma proteína de iduronato-2-sulfatase (i2s), formulação compreendendo a mesma e recipiente
EP2596123B1 (en) 2010-07-22 2015-11-25 Shire Human Genetic Therapies, Inc. Crystal structure of human alpha-n-acetylglucosaminidase
BR112013022557A2 (pt) * 2011-03-04 2017-08-01 Shire Human Genetic Therapies ligantes de peptídeo composições de polipeptídeo e métodos para uso dos mesmos
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
WO2013055888A2 (en) * 2011-10-12 2013-04-18 Synageva Biopharma Corp. Recombinant human naglu protein and uses thereof
US8486399B2 (en) 2011-12-02 2013-07-16 Armagen Technologies, Inc. Methods and compositions for increasing arylsulfatase A activity in the CNS
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR101380740B1 (ko) 2012-06-29 2014-04-11 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 이듀로네이트-2-설파타제의 정제
ES2679374T3 (es) * 2012-11-27 2018-08-24 Biomarin Pharmaceutical Inc. Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas
FR3011468B1 (fr) * 2013-10-04 2015-12-04 Inventiva Utilisation de l'odiparcil dans le traitement d'une mucopolysaccharidose
EP3160494B1 (en) * 2014-06-25 2019-11-20 Shire Human Genetic Therapies, Inc. Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase
WO2016025523A1 (en) 2014-08-11 2016-02-18 Shire Human Genetic Therapies, Inc. Lysosomal targeting and uses thereof
WO2016025519A1 (en) 2014-08-11 2016-02-18 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
AU2015323966A1 (en) 2014-09-29 2017-03-16 Alexion Pharmaceuticals, Inc. Methods of treating mucopolysaccharidosis IIIB (MPSIIIB)
WO2016065319A1 (en) * 2014-10-24 2016-04-28 Shire Human Genetic Therapies, Inc. Lysosomal targeting of enzymes, and uses thereof
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US10538589B2 (en) * 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
EP3419651B1 (en) 2016-02-24 2023-10-25 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
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Also Published As

Publication number Publication date
NZ605871A (en) 2015-02-27
EP2585105A2 (en) 2013-05-01
US20110318327A1 (en) 2011-12-29
AU2011270672A1 (en) 2013-02-14
ES2643015T3 (es) 2017-11-21
JP2013542913A (ja) 2013-11-28
EP2585105A4 (en) 2014-07-09
CA2805449A1 (en) 2011-12-29
AU2017202344A1 (en) 2017-04-27
WO2011163652A3 (en) 2013-07-11
MX2013000324A (es) 2013-02-01
JP6063380B2 (ja) 2017-01-18
EP2585105B1 (en) 2017-08-09
WO2011163652A2 (en) 2011-12-29
NZ702801A (en) 2016-08-26
CN103269709A (zh) 2013-08-28
KR20130043166A (ko) 2013-04-29

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