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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- 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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- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- C—CHEMISTRY; METALLURGY
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- 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
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- 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.)
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- 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
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- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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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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Landscapes
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
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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 |
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| US20220133863A1 (en) * | 2010-06-25 | 2022-05-05 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
| 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 |
| BR112020005271A2 (pt) | 2017-10-02 | 2020-09-15 | Denali Therapeutics Inc. | proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica |
| 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 |
| EP3830259A4 (en) | 2018-08-02 | 2022-05-04 | Dyne Therapeutics, Inc. | MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY |
| EP3897747A4 (en) | 2018-12-19 | 2022-12-07 | The Board of Trustees of the Leland Stanford Junior University | BIFUNCTIONAL MOLECULES FOR LYSOSOMA TARGETING AND RELATED COMPOSITIONS AND METHODS |
| BR112021026866A2 (pt) * | 2019-07-02 | 2022-03-03 | M6P Therapeutics | Composições de vetor e métodos de seu uso para o tratamento de distúrbios do armazenamento lisossômico |
| 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 |
| JP2024524614A (ja) | 2021-07-14 | 2024-07-05 | ライシア セラピューティクス， インコーポレイテッド | Ａｓｇｐｒ細胞表面受容体結合化合物及びコンジュゲート |
| WO2023150387A1 (en) * | 2022-02-07 | 2023-08-10 | M6P Therapeutics, Inc. | Compositions comprising acid alpha glucosidase and methods of use thereof |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting 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 |
| WO2026159194A1 (en) * | 2025-01-22 | 2026-07-30 | Vdjip B.V. | Targeting the lysosome |
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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 |
| WO2003057179A2 (en) | 2002-01-11 | 2003-07-17 | Biomarin Pharmaceutical, Inc. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
| EP2225269A2 (en) * | 2007-11-09 | 2010-09-08 | Anaphore, Inc. | Fusion proteins of mannose binding lectins for treatment of disease |
| WO2009137721A2 (en) * | 2008-05-07 | 2009-11-12 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| CA2803166C (en) * | 2010-06-25 | 2023-03-07 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
| 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 |
| EP2596123B1 (en) | 2010-07-22 | 2015-11-25 | 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 |
| LT2714752T (lt) * | 2011-05-27 | 2018-03-12 | Amicus Therapeutics, Inc. | Nukreipiančių peptidų kopuliavimo ant rekombinantinių lizosominių fermentų būdai, skirti lizosominio kaupimo ligų gydymo pagerinimui |
| WO2013055888A2 (en) * | 2011-10-12 | 2013-04-18 | Synageva Biopharma Corp. | Recombinant human naglu protein and uses thereof |
| ES2679374T3 (es) * | 2012-11-27 | 2018-08-24 | Biomarin Pharmaceutical Inc. | Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas |
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2020
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| US20170232076A1 (en) | 2017-08-17 |
| US10722559B2 (en) | 2020-07-28 |
| EP3185889A1 (en) | 2017-07-05 |
| BR112017002741A2 (pt) | 2018-02-27 |
| US20200376095A1 (en) | 2020-12-03 |
| JP2017529326A (ja) | 2017-10-05 |
| WO2016025519A1 (en) | 2016-02-18 |
| AU2015301809A1 (en) | 2017-02-02 |
| MX2017001898A (es) | 2017-04-11 |
| EP3185889B1 (en) | 2020-07-15 |
| EA201790178A1 (ru) | 2017-07-31 |
| CN106573039A (zh) | 2017-04-19 |
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