PL1673104T5 - Dostarczanie związków terapeutycznych do mózgu i do innych tkanek - Google Patents

Dostarczanie związków terapeutycznych do mózgu i do innych tkanek

Info

Publication number
PL1673104T5
PL1673104T5 PL04782579.9T PL04782579T PL1673104T5 PL 1673104 T5 PL1673104 T5 PL 1673104T5 PL 04782579 T PL04782579 T PL 04782579T PL 1673104 T5 PL1673104 T5 PL 1673104T5
Authority
PL
Poland
Prior art keywords
brain
tissues
delivery
therapeutic compounds
therapeutic
Prior art date
Application number
PL04782579.9T
Other languages
English (en)
Other versions
PL1673104T3 (pl
Inventor
Emil D. Kakkis
Original Assignee
Biomarin Pharmaceutical Inc.
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=34217412&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PL1673104(T5) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Biomarin Pharmaceutical Inc. filed Critical Biomarin Pharmaceutical Inc.
Publication of PL1673104T3 publication Critical patent/PL1673104T3/pl
Publication of PL1673104T5 publication Critical patent/PL1673104T5/pl

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/06Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06008Cerebroside-sulfatase (3.1.6.8)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105Alpha-N-acetylglucosaminidase (3.2.1.50)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL04782579.9T 2003-08-29 2004-08-30 Dostarczanie związków terapeutycznych do mózgu i do innych tkanek PL1673104T5 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US10/651,493 US7442372B2 (en) 2003-08-29 2003-08-29 Delivery of therapeutic compounds to the brain and other tissues
PCT/US2004/028135 WO2005021064A2 (en) 2003-08-29 2004-08-30 Delivery of therapeutic compounds to the brain and other tissues
EP04782579.9A EP1673104B2 (en) 2003-08-29 2004-08-30 Delivery of therapeutic compounds to the brain and other tissues

Publications (2)

Publication Number Publication Date
PL1673104T3 PL1673104T3 (pl) 2014-03-31
PL1673104T5 true PL1673104T5 (pl) 2023-03-06

Family

ID=34217412

Family Applications (1)

Application Number Title Priority Date Filing Date
PL04782579.9T PL1673104T5 (pl) 2003-08-29 2004-08-30 Dostarczanie związków terapeutycznych do mózgu i do innych tkanek

Country Status (12)

Country Link
US (5) US7442372B2 (pl)
EP (1) EP1673104B2 (pl)
JP (1) JP5819574B2 (pl)
AU (1) AU2004268645B2 (pl)
CA (1) CA2537238C (pl)
CY (1) CY1114815T1 (pl)
DK (1) DK1673104T4 (pl)
ES (1) ES2441612T5 (pl)
PL (1) PL1673104T5 (pl)
PT (1) PT1673104E (pl)
SI (1) SI1673104T2 (pl)
WO (1) WO2005021064A2 (pl)

Families Citing this family (82)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6585971B1 (en) * 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
US7723296B2 (en) * 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
JP2008503590A (ja) * 2004-06-21 2008-02-07 メドトロニック・インコーポレーテッド 組成物を細胞にデリバリーするための医学用システム及び方法
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
MX358492B (es) 2006-02-09 2018-08-22 Genzyme Corp Star Suministro intraventricular lento.
US20080015546A1 (en) * 2006-07-13 2008-01-17 Casas Jesus W Methods and formulations for optimal local delivery of cell therapy via minimally invasive procedures
US8849372B2 (en) * 2006-11-22 2014-09-30 The General Hospital Corporation Method for magnetic resonance imaging using stimulus induced rotary saturation with a contrast agent
US20080140008A1 (en) * 2006-12-06 2008-06-12 Medtronic, Inc. Intrathecal catheter
DK2457920T3 (en) 2007-01-18 2018-01-22 Genzyme Corp Oligosaccharides comprising an amino oxy group and conjugates thereof
WO2008089339A2 (en) * 2007-01-18 2008-07-24 Genzyme Corporation Oligosaccharide conjugates for cellular targeting
LT2158322T (lt) * 2007-06-06 2017-08-25 Genzyme Corporation Lizosomų kaupimo ligų genų terapija
US8114346B2 (en) * 2007-08-17 2012-02-14 The Invention Science Fund I, Llc Event-triggered ultraviolet light sterilization of surfaces
US8753304B2 (en) 2007-08-17 2014-06-17 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having acoustically actuatable waveguide components for delivering a sterilizing stimulus to a region proximate a surface of the catheter
US8647292B2 (en) 2007-08-17 2014-02-11 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having components that are actively controllable between two or more wettability states
US8366652B2 (en) 2007-08-17 2013-02-05 The Invention Science Fund I, Llc Systems, devices, and methods including infection-fighting and monitoring shunts
US8702640B2 (en) 2007-08-17 2014-04-22 The Invention Science Fund I, Llc System, devices, and methods including catheters configured to monitor and inhibit biofilm formation
US8029740B2 (en) 2008-07-11 2011-10-04 The Invention Science Fund I, Llc Event-triggered self-sterilization of article surfaces
US20090048648A1 (en) * 2007-08-17 2009-02-19 Searete Llc, A Limited Liability Corporation Of The State Of Delaware Self-sterilizing device
US8162924B2 (en) 2007-08-17 2012-04-24 The Invention Science Fund I, Llc System, devices, and methods including actively-controllable superoxide water generating systems
US8706211B2 (en) 2007-08-17 2014-04-22 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having self-cleaning surfaces
US8734718B2 (en) 2007-08-17 2014-05-27 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having an actively controllable therapeutic agent delivery component
US8460229B2 (en) 2007-08-17 2013-06-11 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having components that are actively controllable between transmissive and reflective states
CA2696699A1 (en) * 2007-08-20 2009-02-26 Protalix Ltd. Saccharide-containing protein conjugates and uses thereof
JP5584132B2 (ja) * 2007-11-15 2014-09-03 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 疼痛の治療のための前立腺酸性ホスファターゼ
EP3272773B8 (en) 2008-05-07 2020-11-18 BioMarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
EP2384168B1 (en) 2008-12-04 2014-10-08 Searete LLC Actively-controllable sterilizing excitation delivery implants
US20120041286A1 (en) 2008-12-04 2012-02-16 Searete Llc, A Limited Liability Corporation Of The State Of Delaware Systems, devices, and methods including implantable devices with anti-microbial properties
US8585627B2 (en) 2008-12-04 2013-11-19 The Invention Science Fund I, Llc Systems, devices, and methods including catheters configured to monitor biofilm formation having biofilm spectral information configured as a data structure
BR122020010601B8 (pt) 2008-12-16 2021-07-27 Genzyme Corp conjugados de proteína-oligossacarídeo, seus usos, e composições farmacêuticas
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
HUE046598T2 (hu) 2009-08-28 2020-03-30 Icahn School Med Mount Sinai Dóziseszkalációs enzim helyettesítõ terápia savas szfingomielináz elégtelenség kezelésére
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
RS59469B1 (sr) * 2010-06-25 2019-11-29 Shire Human Genetic Therapies Postupci i kompozicije za isporuku iduronat-2-sulfataze u cns
WO2011163651A2 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. METHODS AND COMPOSITIONS FOR CNS DELIVERY OF β-GALACTOCEREBROSIDASE
KR102094094B1 (ko) * 2010-06-25 2020-03-27 샤이어 휴먼 지네틱 테라피즈 인크. 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
US20110318327A1 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
RU2012154576A (ru) * 2010-06-25 2014-07-27 Шир Хьюман Дженетик Терапис, Инк. Способы и композиции для доставки к цнс гепаран-n-сульфатазы
LT3103469T (lt) * 2010-06-25 2021-03-25 Shire Human Genetic Therapies, Inc. Terapinių agentų pristatymas cns
US9283181B2 (en) * 2010-06-25 2016-03-15 Shire Human Genetic Therapies, Inc. CNS delivery of therapeutic agents
JP2012062312A (ja) * 2010-08-19 2012-03-29 Yoshikatsu Eto ハンター症候群の治療剤
RS60129B1 (sr) 2011-01-20 2020-05-29 Protalix Ltd Kompozicije alfa-galaktozidaze
MX2014004359A (es) * 2011-10-12 2014-05-28 Synageva Biopharma Corp Proteina naglu humana recombinante y usos de la misma.
US9101678B2 (en) 2011-11-03 2015-08-11 Elwha Llc Heat-sanitization of surfaces
WO2013070760A1 (en) * 2011-11-07 2013-05-16 Shire Human Genetic Therapies, Inc. Biomarkers for sanfilippo syndrome 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
EA201792099A1 (ru) 2011-12-23 2018-05-31 Шир Хьюман Дженетик Терапис, Инк. Лечение когнитивных нарушений при синдроме хантера путем интратекальной доставки идуронат-2-сульфатазы
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
WO2014022515A1 (en) * 2012-07-31 2014-02-06 Bioasis Technologies, Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
CN104684585A (zh) * 2012-08-03 2015-06-03 美国政府(由卫生和人类服务部的部长所代表) 用于治疗溶酶体贮积症的环糊精
KR102385392B1 (ko) 2012-11-27 2022-04-11 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들
MX2015007183A (es) * 2012-12-07 2015-10-12 Shire Human Genetic Therapies Metodos y composiciones para el tratamiento de mucopolisacaridosis tipo iiia administrado de forma intratecal.
CN105189542A (zh) 2013-02-20 2015-12-23 瓦莱里昂治疗有限责任公司 用于治疗庞贝氏症的方法和组合物
WO2014186579A1 (en) * 2013-05-15 2014-11-20 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
US9994641B2 (en) 2013-12-25 2018-06-12 Jcr Pharmaceuticals Co., Ltd. Anti-human transferrin receptor antibody that passes through blood-brain barrier
TWI752907B (zh) 2015-05-08 2022-01-21 美商拜奧馬林製藥公司 用於治療cln2疾病之tpp1調配物及方法
JP2018517046A (ja) 2015-06-10 2018-06-28 ビテス, インコーポレイテッド ヒドロキシプロピル−ベータ−シクロデキストリン組成物および方法
SG11201710734UA (en) 2015-06-24 2018-01-30 Japan Chem Res Anti-human transferrin receptor antibody permeating blood-brain barrier
WO2016208696A1 (ja) 2015-06-24 2016-12-29 Jcrファーマ株式会社 Bdnfを含む融合蛋白質
WO2017079729A1 (en) 2015-11-06 2017-05-11 Biomarin Pharmaceutical Inc. Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes
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
JP7171439B2 (ja) 2016-04-15 2022-11-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ii型を処置するための遺伝子療法
JP7079243B2 (ja) * 2016-06-02 2022-06-01 エイディーエス セラピューティクス リミテッド ライアビリティ カンパニー 緑内障手術の成功率を向上させるためにニンテダニブを使用する組成物および方法
CA3024536A1 (en) 2016-06-13 2017-12-21 The University Of North Carolina At Chapel Hill Optimized cln1 genes and expression cassettes and their use
NZ752703A (en) 2016-12-26 2022-09-30 Japan Chem Res Novel anti-human transferrin receptor antibody capable of penetrating blood-brain barrier
EP3561058A4 (en) 2016-12-26 2020-07-22 JCR Pharmaceuticals Co., Ltd. FUSION PROTEIN INCLUDING BDNF
US11541005B2 (en) 2017-02-08 2023-01-03 New Hope Research Foundation, Inc. Systems and methods for enhanced distribution of a biologic agent within the brain and spinal cord
US11723865B2 (en) 2017-02-08 2023-08-15 New Hope Research Foundation, Inc. Systems and methods for enhanced distribution of a biologic agent within the brain and spinal cord
KR20200104852A (ko) 2017-09-22 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
TW201925236A (zh) 2017-10-02 2019-07-01 美商戴納立製藥公司 包含酶替代療法酶之融合蛋白
US11730767B2 (en) 2018-02-23 2023-08-22 Regeneration Biomedical, Inc. Methods, apparatuses and systems for instilling stem cells and pharmaceuticals into the human ventricular system
EP3670652A1 (en) * 2018-12-18 2020-06-24 NEUWAY Pharma GmbH Vlp for the treatment of a lysosomal storage disease
WO2020125962A1 (en) * 2018-12-18 2020-06-25 Neuway Pharma Gmbh A novel drug delivery system and methods providing thereof
US20220023434A1 (en) 2018-12-19 2022-01-27 The Board Of Trustees Of The Leland Stanford Junior University Bifunctional Molecules for Lysosomal Targeting and Related Compositions and Methods
US11446084B2 (en) 2019-07-12 2022-09-20 Neuralink Corp. Laser drilling of pia mater
MX2022002451A (es) 2019-08-29 2022-06-02 Biomarin Pharm Inc Metodos para el tratamiento de la enfermedad cln2 en sujetos pediatricos.
AU2021217816A1 (en) 2020-02-03 2022-08-11 Gt Gain Therapeutics Sa Combination therapy for treating MPS1
US20240043473A1 (en) * 2021-02-09 2024-02-08 Phoenix Nest Inc. Nootropic peptides for treating lysosomal storage diseases
WO2023156966A1 (en) 2022-02-18 2023-08-24 Beren Therapeutics P.B.C. Compositions of hydroxypropyl-beta-cyclodextrin and methods of purifying the same

Family Cites Families (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2601215A (en) 1948-03-17 1952-06-17 American Cyanamid Co Process of preparing dihydropterins
US3505329A (en) 1968-02-06 1970-04-07 Smithkline Corp Process for the synthesis of biopterin
JPS5883691A (ja) 1981-11-13 1983-05-19 Kanegafuchi Chem Ind Co Ltd 1′,2′−ジアシル−(6r,s)−5,6,7,8−テトラヒドロ−l−ビオプテリンおよびその製法
CH651755A5 (en) 1982-03-03 1985-10-15 Kanegafuchi Chemical Ind Use of pterin derivatives
JPS5925323A (ja) 1982-03-03 1984-02-09 鐘淵化学工業株式会社 プテリン誘導体からなる脳内神経伝達物質の関わる疾病の治療剤
GB8318833D0 (en) 1983-07-12 1983-08-10 Wellcome Found Chemical compounds
JPS5976086A (ja) 1982-09-20 1984-04-28 ザ ウエルカム フアウンデ−シヨン リミテツド プテリジン化合物
US5196533A (en) 1983-04-11 1993-03-23 South Alabama Medical Science Foundation, Usa Cyclization of 5 amino-pyrimidines to quinoid 6,6 disubstituted dihydropteridines
US4587340A (en) 1983-09-19 1986-05-06 Burroughs Wellcome Co. Biopterin analogs
JPS60178887A (ja) 1984-02-23 1985-09-12 Kanegafuchi Chem Ind Co Ltd 5,6,7,8−テトラヒドロ−l−ビオプテリンの製造法
JPS60199889A (ja) 1984-03-24 1985-10-09 Kanegafuchi Chem Ind Co Ltd 5,6,7,8−テトラヒドロ−l−エリスロ−ビオプテリンの硫酸塩およびその製法
US4550109A (en) 1984-05-31 1985-10-29 The Board Of Regents, The University Of Texas System Lipoidal biopterin compounds
DE3437944A1 (de) 1984-10-17 1986-07-31 Biotest-Serum-Institut Gmbh, 6000 Frankfurt Verwendung von pterinen zur steigerung der aktivitaet von lymphokinen und anderen blutfaktoren, sowie ein diagnostisches oder therapeutisches praeparat, das pterine in kombination mit lymphokinen enthaelt
US4713454A (en) 1985-01-28 1987-12-15 Shiratori Pharmaceutical Co., Ltd. Preparation process of (6R)-tetrahydro-L-biopterin
JPS61277618A (ja) 1985-06-04 1986-12-08 Suntory Ltd 自閉症治療剤
US4801575A (en) * 1986-07-30 1989-01-31 The Regents Of The University Of California Chimeric peptides for neuropeptide delivery through the blood-brain barrier
EP0318926B1 (en) 1987-11-30 1995-05-03 Kabushiki Kaisha Vitamin Kenkyusyo Intermediates for synthesizing 5,6,7,8-tetrahydro-L-erythro-biopterin and its derivatives
US6149909A (en) 1995-06-23 2000-11-21 Women's And Children's Hospital Synthetic α-L-iduronidase and genetic sequences encoding same
JP2534423B2 (ja) 1991-12-26 1996-09-18 コーネル・リサーチ・ファンデーション・インコーポレイテッド 酸化窒素の過剰生産から生じる血管失調を阻止する阻害剤
US5198547A (en) 1992-03-16 1993-03-30 South Alabama Medical Science Foundation, Usa Process for N5-formylating tetrahydropteridines
US5981194A (en) 1992-07-10 1999-11-09 University Of British Columbia Use of p97 and iron binding proteins as diagnostic and therapeutic agents
DE4308739C1 (de) 1993-03-19 1994-06-23 Henning Berlin Gmbh Pterinderivate, ihre Herstellung und ihre Verwendung
US5945452A (en) 1993-06-11 1999-08-31 The Board Of Trustees Of The Leland Stanford Junior University Treatment of vascular degenerative diseases by modulation of endogenous nitric oxide production or activity
US5502050A (en) 1993-11-29 1996-03-26 Cornell Research Foundation, Inc. Blocking utilization of tetrahydrobiopterin to block induction of nitric oxide synthesis
DE69526918T2 (de) 1994-08-05 2002-12-19 Suntory Ltd., Osaka Arzneimittel gegen spinocerebellare degeneration
US5716614A (en) * 1994-08-05 1998-02-10 Molecular/Structural Biotechnologies, Inc. Method for delivering active agents to mammalian brains in a complex with eicosapentaenoic acid or docosahexaenoic acid-conjugated polycationic carrier
WO1997010353A1 (en) 1995-09-14 1997-03-20 Virginia Tech Intellectual Property, Inc. Production of lysosomal enzymes in plant-based expression systems
CA2205710A1 (en) 1996-05-20 1997-11-20 The University Of British Columbia Murine model for generalized lysosomal storage disorder and methods of making and using same
US20020076782A1 (en) 1996-07-05 2002-06-20 John P.N. Rosazza Purified nitric oxide synthase
GB9617990D0 (en) 1996-08-29 1996-10-09 Scotia Holdings Plc Treatment of pain
US6458574B1 (en) * 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US5922713A (en) 1997-06-26 1999-07-13 Werner; Ernst Inhibition of nitric oxide synthase
JP4306825B2 (ja) 1998-02-27 2009-08-05 アスビオファーマ株式会社 インスリン抵抗性が関与する血管機能異常を伴う疾患の予防または治療剤
EP1078075A2 (en) * 1998-05-13 2001-02-28 Harbor-Ucla Research and Education Institute Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6200758B1 (en) 1999-02-19 2001-03-13 New York State Office Of Mental Health Phenylalanine hydroxylase gene variants, and amino acid and pterin homeostasis, in the definition, detection, treatment and prevention of psychotic, mood and personality disorders
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US6521212B1 (en) 1999-03-18 2003-02-18 United Therapeutics Corporation Method for treating peripheral vascular disease by administering benzindene prostaglandins by inhalation
JP2002539257A (ja) 1999-03-19 2002-11-19 イーノス・ファーマシューティカルス・インコーポレーテッド 薬剤の脳内生物学的利用率の増加
US6642038B1 (en) 1999-09-14 2003-11-04 Genzyme Glycobiology Research Institute, Inc. GlcNAc phosphotransferase of the lysosomal targeting pathway
GB2355191A (en) 1999-10-12 2001-04-18 Laxdale Ltd Combination formulations for fatigue, head injury and strokes
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6569661B1 (en) 1999-11-12 2003-05-27 Biomarin Pharmaceutical Inc. Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6585971B1 (en) 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
JP2003514024A (ja) * 1999-11-12 2003-04-15 オンコリティクス バイオテク,インコーポレーテッド 細胞増殖性疾患の治療のためのウイルス
AU1775901A (en) * 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US6972124B2 (en) * 2000-05-01 2005-12-06 Biomarin Pharmaceuticals Inc. Precursor of N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme
US20020006400A1 (en) 2000-05-11 2002-01-17 Peter Lobel Recombinant human CLN2 protein and methods of its production and use
US6544994B2 (en) 2000-06-07 2003-04-08 Eprov Ag Pharmaceutical preparation for treating or preventing cardiovascular or neurological disorders by modulating of the activity of nitric oxide synthase
AU2001269923A1 (en) * 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
EP2767291B1 (en) 2000-07-18 2016-09-14 Duke University Treatment of glycogen storage disease type II
US20050119270A1 (en) 2000-08-04 2005-06-02 Mason R. P. Synergistic effect of amlodipine and atorvastatin on aortic endothelial cell nitric oxide release
ATE426021T1 (de) 2000-08-31 2009-04-15 Asubio Pharma Co Ltd Verfahren zur herstellung von biopterinen
ATE384736T1 (de) * 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
WO2002098455A2 (en) 2001-06-07 2002-12-12 Hemebiotech A/S Production of recombinant human arylsulfatase a
US20030007961A1 (en) 2001-06-22 2003-01-09 Wilburn Michael D. Orthomolecular vitamin E derivatives
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US7658916B2 (en) 2002-04-05 2010-02-09 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy by modulation of cell surface receptor density
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
DK1503788T3 (da) 2002-04-25 2011-10-17 Shire Human Genetic Therapies Behandling af alpha-galactosidase A-deficiens
US7485314B2 (en) 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
US20050142141A1 (en) 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
DE10260263A1 (de) 2002-12-20 2004-07-15 Biocrates Life Sciences Gmbh Verwendung von Tetrahydrobiopterinderivaten zur Behandlung und Ernährung von Patienten mit Aminosäurestoffwechselstörungen
WO2004091475A2 (en) 2003-04-16 2004-10-28 Yeda Research And Development Co. Ltd. Gaucher disease drugs and methods of identifying same
US20050026823A1 (en) * 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
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
EP2436379A1 (en) 2004-11-17 2012-04-04 BioMarin Pharmaceutical Inc. Stable tablet formulation

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