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

    • 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
    • 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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)
    • C12Y301/06008—Cerebroside-sulfatase (3.1.6.8)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105—Alpha-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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Obesity (AREA)
  • Diabetes (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
EP04782579.9A EP1673104B2 (en) 2003-08-29 2004-08-30 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

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 (89)

* 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
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 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
HUE040490T2 (hu) * 2006-02-09 2019-03-28 Genzyme Corp Lassú intraventrikuláris szállítás
EP2046435A4 (en) * 2006-07-13 2012-06-27 Medtronic Inc METHOD AND FORMULATIONS FOR OPTIMAL LOCAL ADMINISTRATION OF CELL THERAPY BY MEANS OF 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
US20080140056A1 (en) * 2006-12-06 2008-06-12 Medtronic, Inc. Method for delivering large molecules to the brain
WO2008089339A2 (en) * 2007-01-18 2008-07-24 Genzyme Corporation Oligosaccharide conjugates for cellular targeting
DK2121713T3 (da) 2007-01-18 2013-06-24 Genzyme Corp Oligosaccharider, omfattende en aminooxygruppe, og konjugater deraf
ES2635726T3 (es) * 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
US20090048648A1 (en) * 2007-08-17 2009-02-19 Searete Llc, A Limited Liability Corporation Of The State Of Delaware Self-sterilizing device
US8029740B2 (en) 2008-07-11 2011-10-04 The Invention Science Fund I, Llc Event-triggered self-sterilization of article surfaces
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
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
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
US8114346B2 (en) * 2007-08-17 2012-02-14 The Invention Science Fund I, Llc Event-triggered ultraviolet light sterilization of surfaces
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
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
US8366652B2 (en) 2007-08-17 2013-02-05 The Invention Science Fund I, Llc Systems, devices, and methods including infection-fighting and monitoring shunts
US8706211B2 (en) 2007-08-17 2014-04-22 The Invention Science Fund I, Llc Systems, devices, and methods including catheters having self-cleaning surfaces
BRPI0815324A2 (pt) * 2007-08-20 2015-07-14 Protalix Ltd "conjugado protêico que contém sacarídeos, processo de separação do conjugado protêico que contém sacarídeos, composição farmacêutica do conjugado protêico que contém sacarídeos, uso do conjugado protêico que contém sacarídeos e composto do conjugado protêico que contém sacarídeos"
WO2009064497A1 (en) * 2007-11-15 2009-05-22 The University Of North Carolina At Chapel Hill Prostatic acid phosphatase for the treatment of pain
BRPI0912225A2 (pt) 2008-05-07 2018-03-20 Zystor Therapeutics, Inc. peptídeos de direcionamento lisossomais e usos dos mesmos
US9474831B2 (en) 2008-12-04 2016-10-25 Gearbox, Llc 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
WO2010065135A1 (en) 2008-12-04 2010-06-10 Searete, Llc System, devices, and methods including actively-controllable sterilizing excitation delivery implants
HUE044381T2 (hu) 2008-12-16 2019-10-28 Genzyme Corp Szintetikus intermedierek oligoszacharid-fehérje konjugátumok elõállításához
EP2475376B1 (en) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
CN106047835A (zh) 2009-08-28 2016-10-26 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
IL291556B2 (en) * 2010-06-25 2024-02-01 Shire Human Genetic Therapies Methods and compositions for administration of arylsulfatase A to the central nervous system
AU2011270669B2 (en) * 2010-06-25 2017-01-05 Takeda Pharmaceutical Company Limited Methods and compositions for CNS delivery of iduronate-2-sulfatase
PT3626257T (pt) 2010-06-25 2021-11-12 Shire Human Genetic Therapies Métodos e composições de arilsulfatase a para a administração no snc
WO2011163651A2 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. METHODS AND COMPOSITIONS FOR CNS DELIVERY OF β-GALACTOCEREBROSIDASE
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
CN103269709A (zh) * 2010-06-25 2013-08-28 夏尔人类遗传性治疗公司 Sanfilippo综合症类型B的治疗
WO2011163648A1 (en) * 2010-06-25 2011-12-29 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
JP5913372B2 (ja) 2011-01-20 2016-04-27 プロタリクス リミテッド 植物および植物細胞におけるα−ガラクトシダーゼの発現のための核酸発現構築物
US9579366B2 (en) * 2011-10-12 2017-02-28 Alexion Pharmaceuticals, Inc. Recombinant human NaGlu protein and uses thereof
US9101678B2 (en) 2011-11-03 2015-08-11 Elwha Llc Heat-sanitization of surfaces
CA2854333A1 (en) * 2011-11-07 2013-05-16 Shire Human Genetic Therapies, Inc. Biomarkers for sanfilippo syndrome and uses thereof
ES2983576T3 (es) * 2011-12-02 2024-10-23 Armagen Inc Métodos y composiciones para aumentar la actividad arilsulfatasa en el SNC
US9682129B2 (en) 2011-12-23 2017-06-20 Shire Human Genetic Therapies, Inc. Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
CA2880162C (en) * 2012-07-31 2023-04-04 Bioasis Technologies, Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
JP2015524444A (ja) * 2012-08-03 2015-08-24 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ ライソゾーム蓄積症を治療するためのシクロデキストリン
PL2925776T3 (pl) 2012-11-27 2018-11-30 Biomarin Pharmaceutical Inc. Ukierunkowane terapeutyczne fuzyjne białka enzymu lizosomalnego i ich zastosowania
CA2891522A1 (en) * 2012-12-07 2014-06-12 Shire Human Genetic Therapies, Inc. Methods and compositions for intrathecally administered treatment of mucupolysaccharidosis type iiia
CA2901978A1 (en) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of pompe disease
CA3209821A1 (en) * 2013-05-15 2014-11-20 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
JP6616189B2 (ja) 2013-12-25 2019-12-04 Ｊｃｒファーマ株式会社 血液脳関門を通過する新規抗トランスフェリン受容体抗体
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
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
CN107849555B (zh) 2015-06-24 2022-01-04 Jcr制药股份有限公司 通过血脑屏障的抗人转铁蛋白受体抗体
CN108474788A (zh) 2015-11-06 2018-08-31 生物马林药物股份有限公司 用于检测中和溶酶体酶的摄取的抗体或其它因子的基于细胞的测定
IL261246B2 (en) 2016-02-24 2023-03-01 Biomarin Pharm Inc Therapeutic fusion proteins targeting lysosomal enzymes, their formulations and their uses
IL305449B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
CN119656170A (zh) * 2016-06-02 2025-03-21 拨康视云生物医药科技(广州)有限公司 使用尼达尼布来治疗具有异常新生血管形成的眼病的组合物和方法
JP2019517274A (ja) 2016-06-13 2019-06-24 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Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｎｏｒｔｈ Ｃａｒｏｌｉｎａ Ａｔ Ｃｈａｐｅｌ Ｈｉｌｌ 最適化されたｃｌｎ１遺伝子および発現カセットおよびそれらの使用
KR102497564B1 (ko) 2016-12-26 2023-02-07 제이씨알 파마 가부시키가이샤 혈액뇌관문을 통과하는 신규한 항인간 트랜스페린 수용체 항체
US12214007B2 (en) 2016-12-26 2025-02-04 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
BR112020005249A2 (pt) 2017-09-22 2020-09-24 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
PE20210347A1 (es) 2017-10-02 2021-02-25 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas
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
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
AU2019343166A1 (en) 2018-09-19 2021-05-20 Regeneration Biomedical, Inc. WNT-activated adipose-derived stem cell apparatuses, methods and systems
US11279938B2 (en) * 2018-12-18 2022-03-22 Neuway Pharma Gmbh Drug delivery system and methods providing thereof
EP3670652A1 (en) * 2018-12-18 2020-06-24 NEUWAY Pharma GmbH Vlp for the treatment of a lysosomal storage disease
CN113301925A (zh) 2018-12-19 2021-08-24 小利兰·斯坦福大学理事会 用于溶酶体靶向的双官能分子以及相关的组合物和方法
US11446084B2 (en) 2019-07-12 2022-09-20 Neuralink Corp. Laser drilling of pia mater
SMT202400315T1 (it) 2019-08-29 2024-09-16 Biomarin Pharm Inc Metodi per il trattamento della malattia cln2 in soggetti pediatrici
EP4100015A1 (en) 2020-02-03 2022-12-14 Gain Therapeutics SA Combination therapy for treating mps1
KR102880902B1 (ko) * 2021-01-29 2025-11-03 현대자동차주식회사 셀 기반 모빌리티 생산시스템의 운영방법
CA3207482A1 (en) * 2021-02-09 2022-08-18 Jill Wood Nootropic peptides for treating lysosomal storage diseases
CA3226269A1 (en) 2021-07-14 2023-01-19 Lycia Therapeutics, Inc. Asgpr cell surface receptor binding compounds and conjugates
CA3244449A1 (en) 2022-02-18 2023-08-24 Beren Therapeutics P.B.C. Hydroxypropyl-beta-cyclodextrin compositions and related purification methods
WO2025125542A1 (en) * 2023-12-15 2025-06-19 F. Hoffmann-La Roche Ag Intrathecal dosage method

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
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
CH651755A5 (en) 1982-03-03 1985-10-15 Kanegafuchi Chemical Ind Use of pterin derivatives
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
GB8318833D0 (en) 1983-07-12 1983-08-10 Wellcome Found Chemical compounds
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
CA1262347A (en) 1985-01-28 1989-10-17 Suntory Limited 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
US4937342A (en) 1987-11-30 1990-06-26 Kabushiki Kaisha Vitamin Kenkyusyo Intermediates for synthesizing BH4 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
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
ATE218345T1 (de) 1994-08-05 2002-06-15 Suntory Ltd Arzneimittel gegen spinocerebellare degeneration
EP0865499B1 (en) 1995-09-14 2009-03-18 Virginia Tech Intellectual Properties, 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
CN1300323A (zh) * 1998-05-13 2001-06-20 加州大学洛杉矶分校港口研究和教育院 重组α－L－艾杜糖苷酸酶,其生产和纯化的方法以及治疗其缺乏导致的疾病的方法
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
CA2367002A1 (en) 1999-03-19 2000-09-28 Michael A. Moskowitz Increasing cerebral bioavailability of drugs
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
CA2720361A1 (en) * 1999-11-12 2001-05-25 Oncolytics Biotech, Inc. Viruses for the treatment of cellular proliferative disorders
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
WO2001036603A2 (en) 1999-11-17 2001-05-25 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
DK1782825T3 (da) 2000-07-18 2014-11-03 Univ Duke Behandling af glycogenlagringssygdom 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
ES2320531T3 (es) 2000-08-31 2009-05-25 Asubio Pharma Co., Ltd. Proceso para producir biopterinas.
ES2300439T3 (es) * 2001-04-30 2008-06-16 Zystor Therapeutics , Inc. Reconocimiento subcelular de proteinas terapeuticas.
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
WO2003086452A2 (en) 2002-04-05 2003-10-23 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
US20040071686A1 (en) 2002-04-25 2004-04-15 Treco Douglas A. Treatment of alpha-galactosidase A deficiency
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
EP1613264A4 (en) 2003-04-16 2007-08-15 Yeda Res & Dev MEDICAMENT FOR THE TREATMENT OF GAUCHER DISEASE AND METHOD FOR IDENTIFYING IT
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
CA2525236C (en) * 2003-06-20 2015-03-24 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
EP2436379A1 (en) 2004-11-17 2012-04-04 BioMarin Pharmaceutical Inc. Stable tablet formulation

Also Published As

Publication number Publication date
US7442372B2 (en) 2008-10-28
CA2537238C (en) 2015-01-06
EP1673104B2 (en) 2021-10-27
AU2004268645B2 (en) 2010-02-18
JP5819574B2 (ja) 2015-11-24
DK1673104T4 (da) 2022-01-17
US20090017005A1 (en) 2009-01-15
US9089566B2 (en) 2015-07-28
SI1673104T1 (sl) 2014-02-28
US20050048047A1 (en) 2005-03-03
ES2441612T5 (es) 2022-04-04
US9044473B2 (en) 2015-06-02
CY1114815T1 (el) 2016-12-14
WO2005021064A3 (en) 2005-08-25
US20120148558A1 (en) 2012-06-14
PL1673104T3 (pl) 2014-03-31
JP2007504166A (ja) 2007-03-01
WO2005021064A2 (en) 2005-03-10
EP1673104B1 (en) 2013-10-09
US11040088B2 (en) 2021-06-22
CA2537238A1 (en) 2005-03-10
EP1673104A2 (en) 2006-06-28
ES2441612T3 (es) 2014-02-05
AU2004268645A1 (en) 2005-03-10
SI1673104T2 (sl) 2022-01-31
US9572870B2 (en) 2017-02-21
PT1673104E (pt) 2014-01-15
EP1673104A4 (en) 2009-08-12
DK1673104T3 (da) 2014-01-20
US20170182134A1 (en) 2017-06-29
US20150150799A1 (en) 2015-06-04

Similar Documents

Publication Publication Date Title
PL1673104T5 (pl) Dostarczanie związków terapeutycznych do mózgu i do innych tkanek
EP1638605A4 (en) DISTRIBUTION OF THERAPEUTIC COMPOUNDS TO THE BRAIN AND OTHER TISSUE
EP1737453A4 (en) METHOD FOR THE USE OF IMMUNOMODULATORY COMPOUNDS AND COMPOSITIONS CONTAINING THEREOF FOR THE TREATMENT AND SUPPLY OF SKIN DISEASES OR DISORDERS
EP1567157A4 (en) METHODS OF USE AND COMPOSITIONS USING IMMUNOMODULATORY COMPOUNDS FOR THE TREATMENT AND MANAGEMENT OF MYELOPROLIFERATIVE SYNDROMES
AU2002341558A1 (en) Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
GB0324761D0 (en) Use of compounds in therapy
AU2003286741A8 (en) Methods and compositions for diagnosis and therapy of parkin-associated disorders
EP1680145A4 (en) METHODS AND COMPOSITIONS FOR THE TREATMENT OF NEUROLOGICAL DISEASES
GB0305150D0 (en) Use of therapeutic compounds
IL173351A0 (en) Therapy of ocular disorders
PL373759A1 (pl) Nowe związki dihydropirymidynowe podstawione grupą cyjanową i ich zastosowanie do leczenia chorób
AU2003247140A1 (en) Delivering compounds to the brain by modifying properties of the bbb and cerebral circulation
PT1670482E (pt) Utilização de ciclesonida para o tratamento de doenças respiratórias
IL176227A0 (en) Compositions and methods of administering tubulin binding agents for the treatment of ocular diseases
GB0213869D0 (en) The treatment of pain
AU2003268484A8 (en) Delivery of therapeutics to the brain and spinal cord
AU2003255843A1 (en) 1,3-thiazolin-4-ones as therapeutic compounds in the treatment of pain
IL164951A0 (en) The treatment of pain with lfendropil
GB0312844D0 (en) Use of compounds in medicine
GB0325957D0 (en) The treatment of pain
GB0322014D0 (en) The treatment of pain
IL174968A0 (en) The use of ifenprodil in the treatment of pain
GB0303533D0 (en) Delivery of agents to the body
HK1091752A (en) Specific delivery of drugs to the brain
GB0418556D0 (en) The treatment of pain