KR20130043166A - 산필리포 증후군 b형의 치료 - Google Patents

산필리포 증후군 b형의 치료 Download PDF

Info

Publication number
KR20130043166A
KR20130043166A KR1020137001751A KR20137001751A KR20130043166A KR 20130043166 A KR20130043166 A KR 20130043166A KR 1020137001751 A KR1020137001751 A KR 1020137001751A KR 20137001751 A KR20137001751 A KR 20137001751A KR 20130043166 A KR20130043166 A KR 20130043166A
Authority
KR
South Korea
Prior art keywords
naglu
nmol
protein
certain embodiments
brain
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
KR1020137001751A
Other languages
English (en)
Korean (ko)
Inventor
마이클 에프. 콘치노
페리클레스 칼리아스
징 판
케빈 홀름스
파올로 마르티니
알라 로마쉬코
무쑤라만 메이얍판
보홍 장
안드레아 이스켄드리안
다이아나 룬드버그
앤젤라 노턴
베티나 스트랙-로그
얀 후앙
마리 알레산드리니
리차드 파이퍼
Original Assignee
샤이어 휴먼 지네틱 테라피즈 인크.
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 샤이어 휴먼 지네틱 테라피즈 인크. filed Critical 샤이어 휴먼 지네틱 테라피즈 인크.
Publication of KR20130043166A publication Critical patent/KR20130043166A/ko
Ceased legal-status Critical Current

Links

Images

Classifications

    • 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

Landscapes

  • 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)
KR1020137001751A 2010-06-25 2011-06-25 산필리포 증후군 b형의 치료 Ceased KR20130043166A (ko)

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

Publications (1)

Publication Number Publication Date
KR20130043166A true KR20130043166A (ko) 2013-04-29

Family

ID=46888842

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020137001751A Ceased KR20130043166A (ko) 2010-06-25 2011-06-25 산필리포 증후군 b형의 치료

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)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2007285763B2 (en) 2006-08-18 2011-12-15 Armagen Technologies, Inc. Agents for blood-brain barrier delivery
CA3184105A1 (en) 2007-07-27 2009-02-05 Armagen Inc. Methods and compositions for increasing alpha-l-iduronidase activity in the cns
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
MA41022A (fr) 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
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
CN112566652A (zh) 2018-06-25 2021-03-26 Jcr制药股份有限公司 含有蛋白的水性液体制剂
CA3192771A1 (en) * 2020-08-28 2022-03-03 Jcr Pharmaceuticals Co., Ltd. Mutant of .alpha.-n-acetylglucosaminidase

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
DK2020438T3 (en) 1998-12-07 2018-07-23 Genzyme Corp Treatment of Pompe disease
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US7560424B2 (en) * 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
WO2003102583A1 (en) 2002-05-29 2003-12-11 Symbiontics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
EP1436316B1 (en) 2001-04-30 2008-01-23 ZyStor Therapeutics , Inc. Subcellular targeting of therapeutic proteins
WO2003032913A2 (en) 2001-10-16 2003-04-24 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
PT1587923E (pt) 2003-01-22 2011-12-07 Univ Duke Construções melhoradas para a expressão de polipéptidos lisossomais
WO2005002515A2 (en) * 2003-06-20 2005-01-13 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
DE602005020745D1 (de) 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
GB0611463D0 (en) * 2006-06-09 2006-07-19 Novartis Ag Organic compounds
WO2008109677A2 (en) 2007-03-06 2008-09-12 Saint Louis University Modified enzyme and treatment method
WO2009137721A2 (en) * 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
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

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
AU2011270672B2 (en) 2017-01-12
NZ702801A (en) 2016-08-26
CN103269709A (zh) 2013-08-28

Similar Documents

Publication Publication Date Title
US11065307B2 (en) Therapeutic fusion protein comprising an alpha-n-acetylglucosaminidase and a lysosomal targeting moiety
EP2585105B1 (en) Treatment of sanfilippo syndrome type b
KR102356192B1 (ko) 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들
EP3626257B1 (en) Methods and compositions for cns delivery of arylsulfatase a
KR20140005842A (ko) 헤파란 n-설파타제의 cns 전달을 위한 방법들 및 조성물들
US20220133863A1 (en) Treatment of sanfilippo syndrome type b
RU2783380C2 (ru) Способы и композиции для доставки в цнс арилсульфатазы а
HK40022682A (en) Methods and compositions for cns delivery of arylsulfatase a
HK40022682B (en) Methods and compositions for cns delivery of arylsulfatase a

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20130123

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20160624

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20170804

Patent event code: PE09021S01D

E601 Decision to refuse application
PE0601 Decision on rejection of patent

Patent event date: 20180201

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D

Patent event date: 20170804

Comment text: Notification of reason for refusal

Patent event code: PE06011S01I