JP6623213B2 - リソソーム酵素に融合したマンノース−６−リン酸含有ペプチド - Google Patents
リソソーム酵素に融合したマンノース−６−リン酸含有ペプチド Download PDFInfo
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- JP6623213B2 JP6623213B2 JP2017507791A JP2017507791A JP6623213B2 JP 6623213 B2 JP6623213 B2 JP 6623213B2 JP 2017507791 A JP2017507791 A JP 2017507791A JP 2017507791 A JP2017507791 A JP 2017507791A JP 6623213 B2 JP6623213 B2 JP 6623213B2
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- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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- C—CHEMISTRY; METALLURGY
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- C07K—PEPTIDES
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- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
- C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
- C12N9/1288—Transferases for other substituted phosphate groups (2.7.8)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/08—Transferases for other substituted phosphate groups (2.7.8)
- C12Y207/08017—UDP-N-acetylglucosamine--lysosomal-enzyme N-acetylglucosaminephosphotransferase (2.7.8.17)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
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- 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)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
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- Wood Science & Technology (AREA)
- Toxicology (AREA)
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- Diabetes (AREA)
- Endocrinology (AREA)
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- Hematology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (3)
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| US201462036082P | 2014-08-11 | 2014-08-11 | |
| US62/036,082 | 2014-08-11 | ||
| PCT/US2015/044713 WO2016025519A1 (en) | 2014-08-11 | 2015-08-11 | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
Publications (3)
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| JP2017529326A JP2017529326A (ja) | 2017-10-05 |
| JP2017529326A5 JP2017529326A5 (OSRAM) | 2018-09-13 |
| JP6623213B2 true JP6623213B2 (ja) | 2019-12-18 |
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| WO (1) | WO2016025519A1 (OSRAM) |
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| HUE056884T2 (hu) | 2010-06-25 | 2022-03-28 | Shire Human Genetic Therapies | Eljárások és készítmények arilszulfatáz-A CNS-be történõ beadására |
| US20220133863A1 (en) * | 2010-06-25 | 2022-05-05 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
| WO2019070577A1 (en) | 2017-10-02 | 2019-04-11 | Denali Therapeutics Inc. | FUSION PROTEINS COMPRISING SUBSTITUTE ENZYM THERAPY ENZYMES |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | 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 |
| CA3123086A1 (en) * | 2018-12-19 | 2020-06-25 | The Board Of Trustees Of The Leland Stanford Junior University | Bifunctional molecules for lysosomal targeting and related compositions and methods |
| TW202122579A (zh) * | 2019-07-02 | 2021-06-16 | 美商Ｍ６Ｐ生物醫藥公司 | 用於治療溶酶體儲積症之載體組合物及其使用方法 |
| CN111455004B (zh) * | 2020-02-29 | 2023-05-05 | 浙江农林大学 | 一种脂肪酶催化在线合成香豆素-3-羧酸-6’-o-d-甘露糖酯的方法 |
| US20230211001A1 (en) * | 2020-07-30 | 2023-07-06 | The Board Of Trustees Of The Leland Stanford Junior University | Lysosomal Targeting Molecules Comprising Knottin Peptides And Related Compositions And Methods |
| WO2023288033A1 (en) | 2021-07-14 | 2023-01-19 | Lycia Therapeutics, Inc. | Asgpr cell surface receptor binding compounds and conjugates |
| WO2023150387A1 (en) * | 2022-02-07 | 2023-08-10 | M6P Therapeutics, Inc. | Compositions comprising acid alpha glucosidase and methods of use thereof |
| CA3255934A1 (en) | 2022-04-15 | 2023-10-19 | Dyne Therapeutics, Inc. | MUSCLE TARGETTING COMPLEXES AND FORMULATIONS FOR THE TREATMENT OF MYOTONIC DYSTROPHY |
| WO2025238106A1 (en) * | 2024-05-15 | 2025-11-20 | M6P Therapeutics (Switzerland) GmbH | Lysosome-targeting degradation fusion design |
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| US20040142474A1 (en) * | 2000-09-14 | 2004-07-22 | Expression Genetics, Inc. | Novel cationic lipopolymer as a biocompatible gene delivery agent |
| US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20050158296A1 (en) * | 2002-01-11 | 2005-07-21 | Starr Christopher M. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
| US20110027267A1 (en) * | 2007-11-09 | 2011-02-03 | Anaphore, Inc. | Fusion Proteins of Mannose Binding Lectins for Treatment of Disease |
| BRPI0912225A2 (pt) * | 2008-05-07 | 2018-03-20 | Zystor Therapeutics, Inc. | peptídeos de direcionamento lisossomais e usos dos mesmos |
| EP3103469B1 (en) * | 2010-06-25 | 2020-12-09 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
| MX2013000324A (es) | 2010-06-25 | 2013-02-01 | Shire Human Genetic Therapies | Tratamiento del sindrome de sanfilippo tipo b. |
| US8775146B2 (en) * | 2010-07-22 | 2014-07-08 | Shire Human Genetic Therapies, Inc. | Crystal structure of human alpha-N-acetylglucosaminidase |
| WO2012157136A1 (ja) * | 2011-05-19 | 2012-11-22 | 財団法人東京都医学総合研究所 | リン酸化糖鎖を含有する組換えヒトサポシンｂタンパク質及びその用途 |
| DK2714752T3 (en) * | 2011-05-27 | 2018-02-26 | Amicus Therapeutics Inc | PROCEDURES FOR THE CONNECTION OF TARGETING PEPTIDES TO RECOMBINANT LYSOSOMAL ENZYMES FOR IMPROVED TREATMENT OF LYSOSOMAL DISPOSAL DISEASES |
| IN2014MN00740A (OSRAM) * | 2011-10-12 | 2015-05-22 | Synageva Biopharma Corp | |
| DK3115372T3 (da) * | 2012-11-27 | 2019-06-11 | Biomarin Pharm Inc | Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf |
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2020
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| US10722559B2 (en) | 2020-07-28 |
| CN106573039A (zh) | 2017-04-19 |
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| MX2017001898A (es) | 2017-04-11 |
| US20170232076A1 (en) | 2017-08-17 |
| BR112017002741A2 (pt) | 2018-02-27 |
| AU2015301809A1 (en) | 2017-02-02 |
| JP2017529326A (ja) | 2017-10-05 |
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