NZ605871A - Treatment of sanfilippo syndrome type b - Google Patents
Treatment of sanfilippo syndrome type bInfo
- Publication number
- NZ605871A NZ605871A NZ605871A NZ60587111A NZ605871A NZ 605871 A NZ605871 A NZ 605871A NZ 605871 A NZ605871 A NZ 605871A NZ 60587111 A NZ60587111 A NZ 60587111A NZ 605871 A NZ605871 A NZ 605871A
- Authority
- NZ
- New Zealand
- Prior art keywords
- treatment
- syndrome type
- sanfilippo syndrome
- sanfilippo
- type
- Prior art date
Links
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 title 1
- 208000036709 mucopolysaccharidosis type 3B Diseases 0.000 title 1
- 208000012227 mucopolysaccharidosis type IIIB Diseases 0.000 title 1
- 238000009472 formulation Methods 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 102100034561 Alpha-N-acetylglucosaminidase Human genes 0.000 abstract 1
- 229910019142 PO4 Inorganic materials 0.000 abstract 1
- 108010009380 alpha-N-acetyl-D-glucosaminidase Proteins 0.000 abstract 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 abstract 1
- 239000010452 phosphate Substances 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
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
- A61K38/43—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
- 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)
- A61K38/465—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)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Inorganic Chemistry (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Zoology (AREA)
- Psychiatry (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Hospice & Palliative Care (AREA)
- Anesthesiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| NZ702801A NZ702801A (en) | 2010-06-25 | 2011-06-25 | Treatment of sanfilippo syndrome type b |
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US35885710P | 2010-06-25 | 2010-06-25 | |
| US36078610P | 2010-07-01 | 2010-07-01 | |
| US38786210P | 2010-09-29 | 2010-09-29 | |
| US201161435710P | 2011-01-24 | 2011-01-24 | |
| US201161442115P | 2011-02-11 | 2011-02-11 | |
| US201161476210P | 2011-04-15 | 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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NZ605871A true NZ605871A (en) | 2015-02-27 |
Family
ID=46888842
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ605871A NZ605871A (en) | 2010-06-25 | 2011-06-25 | Treatment of sanfilippo syndrome type b |
| NZ702801A NZ702801A (en) | 2010-06-25 | 2011-06-25 | Treatment of sanfilippo syndrome type b |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ702801A NZ702801A (en) | 2010-06-25 | 2011-06-25 | Treatment of sanfilippo syndrome type b |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20110318327A1 (enExample) |
| EP (1) | EP2585105B1 (enExample) |
| JP (1) | JP6063380B2 (enExample) |
| KR (1) | KR20130043166A (enExample) |
| CN (1) | CN103269709A (enExample) |
| AU (2) | AU2011270672B2 (enExample) |
| CA (1) | CA2805449A1 (enExample) |
| ES (1) | ES2643015T3 (enExample) |
| MX (1) | MX2013000324A (enExample) |
| NZ (2) | NZ605871A (enExample) |
| WO (1) | WO2011163652A2 (enExample) |
Families Citing this family (27)
| 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 |
| AU2008282496B2 (en) | 2007-07-27 | 2013-04-04 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-iduronidase activity in the CNS |
| CN102066422B (zh) | 2008-05-07 | 2015-07-22 | 宝玛瑞制药公司 | 溶酶体靶向肽及其应用 |
| PL2485761T3 (pl) | 2009-10-09 | 2019-10-31 | Armagen Inc | Sposoby i kompozycje do zwiększania aktywności 2-sulfatazy iduronianu w cns |
| EP3875107A1 (en) | 2010-06-25 | 2021-09-08 | Shire Human Genetic Therapies, Inc. | Pharmaceutical formulation comprising a replacement enzyme for a lysosomal enzyme for use in treating lysosomal storage disease intrathecally |
| CN103260637B (zh) | 2010-06-25 | 2016-04-06 | 夏尔人类遗传性治疗公司 | 乙酰肝素n-硫酸酯酶cns递送的方法和组合物 |
| ES2858726T3 (es) | 2010-06-25 | 2021-09-30 | Shire Human Genetic Therapies | Administración de agentes terapéuticos al SNC |
| DK2585104T3 (da) | 2010-06-25 | 2019-10-14 | Shire Human Genetic Therapies | Fremgangsmåder og sammensætninger tilcns-levering af arylsulfatase a |
| EP2975134A3 (en) | 2010-07-22 | 2016-02-24 | Shire Human Genetic Therapies, Inc. | Crystal structure of human alpha-n-acetylglucosaminidase |
| CN106279433A (zh) * | 2011-03-04 | 2017-01-04 | 夏尔人类遗传治疗公司 | 用于多肽组成物的肽连接物及其使用方法 |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| CA2852027A1 (en) * | 2011-10-12 | 2013-04-18 | Synageva Biopharma Corp. | Recombinant human naglu protein and uses thereof |
| WO2013081706A1 (en) | 2011-12-02 | 2013-06-06 | Armagen Technologies, Inc. | Methods and compositions for increasing arylsulfatase a activity in the cns |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | 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 |
| MX377150B (es) | 2012-11-27 | 2025-03-07 | Biomarin Pharm Inc | Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas. |
| FR3011468B1 (fr) * | 2013-10-04 | 2015-12-04 | Inventiva | Utilisation de l'odiparcil dans le traitement d'une mucopolysaccharidose |
| US20170267986A1 (en) * | 2014-06-25 | 2017-09-21 | Shire Human Genetic Therapies, Inc. | Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase |
| EP3185889B1 (en) | 2014-08-11 | 2020-07-15 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| WO2016025523A1 (en) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| JP2017531632A (ja) * | 2014-09-29 | 2017-10-26 | アレクシオン ファーマシューティカルズ， インコーポレイテッド | ムコ多糖症ｉｉｉｂ型（ｍｐｓｉｉｉｂ）を治療する方法 |
| 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 |
| JP7109369B2 (ja) * | 2016-02-24 | 2022-07-29 | バイオマリン ファーマシューティカル インコーポレイテッド | ターゲティングされた治療用リソソーム酵素融合タンパク質、関連する製剤、およびその使用 |
| KR20210024082A (ko) | 2018-06-25 | 2021-03-04 | 제이씨알 파마 가부시키가이샤 | 단백질 함유 수성 액제 |
| TW202227630A (zh) * | 2020-08-28 | 2022-07-16 | 日商Ｊｃｒ製藥股份有限公司 | α-N-乙醯葡萄糖胺苷酶之變異體 |
Family Cites Families (19)
| 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 |
| DK1137762T3 (da) | 1998-12-07 | 2009-02-02 | Genzyme Corp | Behandling af Pompes sygdom |
| 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 |
| JP4641705B2 (ja) | 2001-04-30 | 2011-03-02 | ザイストール セラピューティクス， インコーポレイテッド | 治療的タンパク質の亜細胞標的化 |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | 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 |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| CA2463473A1 (en) * | 2001-10-16 | 2003-04-24 | Symbiontics Inc. | Targeted therapeutic proteins |
| US20040248262A1 (en) | 2003-01-22 | 2004-12-09 | Koeberl Dwight D. | Constructs for expressing lysomal polypeptides |
| EP1638605B1 (en) * | 2003-06-20 | 2014-01-08 | Raptor 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 |
| ES2344302T3 (es) | 2004-02-10 | 2010-08-24 | Zystor Therapeutics , Inc. | Alfa glucosidasa acida y fragmentos de la misma. |
| 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 |
| CN102066422B (zh) * | 2008-05-07 | 2015-07-22 | 宝玛瑞制药公司 | 溶酶体靶向肽及其应用 |
| CN103260637B (zh) * | 2010-06-25 | 2016-04-06 | 夏尔人类遗传性治疗公司 | 乙酰肝素n-硫酸酯酶cns递送的方法和组合物 |
-
2011
- 2011-06-25 JP JP2013516847A patent/JP6063380B2/ja not_active Expired - Fee Related
- 2011-06-25 KR KR1020137001751A patent/KR20130043166A/ko not_active Ceased
- 2011-06-25 EP EP11799039.0A patent/EP2585105B1/en active Active
- 2011-06-25 CN CN2011800407953A patent/CN103269709A/zh active Pending
- 2011-06-25 WO PCT/US2011/041928 patent/WO2011163652A2/en not_active Ceased
- 2011-06-25 NZ NZ605871A patent/NZ605871A/en not_active IP Right Cessation
- 2011-06-25 MX MX2013000324A patent/MX2013000324A/es unknown
- 2011-06-25 NZ NZ702801A patent/NZ702801A/en not_active IP Right Cessation
- 2011-06-25 CA CA2805449A patent/CA2805449A1/en not_active Abandoned
- 2011-06-25 ES ES11799039.0T patent/ES2643015T3/es active Active
- 2011-06-25 AU AU2011270672A patent/AU2011270672B2/en not_active Ceased
- 2011-06-25 US US13/168,969 patent/US20110318327A1/en not_active Abandoned
-
2017
- 2017-04-10 AU AU2017202344A patent/AU2017202344A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP2585105A2 (en) | 2013-05-01 |
| CN103269709A (zh) | 2013-08-28 |
| WO2011163652A2 (en) | 2011-12-29 |
| AU2017202344A1 (en) | 2017-04-27 |
| US20110318327A1 (en) | 2011-12-29 |
| NZ702801A (en) | 2016-08-26 |
| AU2011270672B2 (en) | 2017-01-12 |
| CA2805449A1 (en) | 2011-12-29 |
| JP6063380B2 (ja) | 2017-01-18 |
| ES2643015T3 (es) | 2017-11-21 |
| EP2585105B1 (en) | 2017-08-09 |
| EP2585105A4 (en) | 2014-07-09 |
| JP2013542913A (ja) | 2013-11-28 |
| AU2011270672A1 (en) | 2013-02-14 |
| WO2011163652A3 (en) | 2013-07-11 |
| MX2013000324A (es) | 2013-02-01 |
| KR20130043166A (ko) | 2013-04-29 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PSEA | Patent sealed | ||
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 25 JUN 2016 BY COMPUTER PACKAGES INC Effective date: 20150701 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 25 JUN 2017 BY COMPUTER PACKAGES INC Effective date: 20160601 |
|
| RENW | Renewal (renewal fees accepted) |
Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 25 JUN 2018 BY COMPUTER PACKAGES INC Effective date: 20170531 |
|
| LAPS | Patent lapsed |