CA2892146A1 - Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof - Google Patents
Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof Download PDFInfo
- Publication number
- CA2892146A1 CA2892146A1 CA2892146A CA2892146A CA2892146A1 CA 2892146 A1 CA2892146 A1 CA 2892146A1 CA 2892146 A CA2892146 A CA 2892146A CA 2892146 A CA2892146 A CA 2892146A CA 2892146 A1 CA2892146 A1 CA 2892146A1
- Authority
- CA
- Canada
- Prior art keywords
- seq
- gap
- nos
- gggga
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- Prior art date
- Legal status (The legal status 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 status listed.)
- Pending
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- 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
- 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
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- 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)
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Toxicology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261730378P | 2012-11-27 | 2012-11-27 | |
| US61/730,378 | 2012-11-27 | ||
| US201361788968P | 2013-03-15 | 2013-03-15 | |
| US61/788,968 | 2013-03-15 | ||
| PCT/US2013/072287 WO2014085621A1 (en) | 2012-11-27 | 2013-11-27 | Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2892146A1 true CA2892146A1 (en) | 2014-06-05 |
Family
ID=49753532
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2892146A Pending CA2892146A1 (en) | 2012-11-27 | 2013-11-27 | Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof |
Country Status (26)
| Country | Link |
|---|---|
| US (9) | US9376480B2 (enExample) |
| EP (2) | EP2925776B1 (enExample) |
| JP (2) | JP6831176B2 (enExample) |
| KR (4) | KR102262882B1 (enExample) |
| CN (1) | CN104822701B (enExample) |
| AR (1) | AR093626A1 (enExample) |
| AU (1) | AU2013352184B2 (enExample) |
| BR (1) | BR112015012152B1 (enExample) |
| CA (1) | CA2892146A1 (enExample) |
| CL (1) | CL2015001371A1 (enExample) |
| CY (1) | CY1122555T1 (enExample) |
| DK (2) | DK2925776T3 (enExample) |
| ES (2) | ES2679374T3 (enExample) |
| HR (2) | HRP20181351T1 (enExample) |
| HU (2) | HUE039334T2 (enExample) |
| IL (3) | IL238824B (enExample) |
| LT (1) | LT3115372T (enExample) |
| MX (2) | MX367024B (enExample) |
| PL (2) | PL3115372T3 (enExample) |
| PT (2) | PT2925776T (enExample) |
| RS (1) | RS58916B1 (enExample) |
| RU (1) | RU2680581C2 (enExample) |
| SI (1) | SI3115372T1 (enExample) |
| TW (2) | TWI626250B (enExample) |
| WO (1) | WO2014085621A1 (enExample) |
| ZA (1) | ZA201503509B (enExample) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009137721A2 (en) | 2008-05-07 | 2009-11-12 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides 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 |
| JP2016523835A (ja) | 2013-05-15 | 2016-08-12 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入 |
| WO2016025519A1 (en) * | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| WO2016049510A2 (en) * | 2014-09-25 | 2016-03-31 | The General Hospital Corporation | Cell-based targeted delivery of pseudonomas exotoxin |
| WO2016065319A1 (en) * | 2014-10-24 | 2016-04-28 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting of enzymes, and uses thereof |
| 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 |
| JP6993958B2 (ja) | 2015-07-06 | 2022-02-04 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 多重特異性抗原結合分子およびその使用 |
| HK1253529A1 (zh) | 2015-11-06 | 2019-06-21 | Biomarin Pharmaceutical Inc. | 用於检测中和溶酶体酶的摄取的抗体或其它因子的基於细胞的测定 |
| EP3419651B1 (en) | 2016-02-24 | 2023-10-25 | BioMarin Pharmaceutical Inc. | Improved naglu fusion protein formulation |
| US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| CL2016003282A1 (es) | 2016-12-21 | 2017-08-18 | Univ Chile | Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington |
| AU2018281280B2 (en) | 2017-06-07 | 2024-12-19 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| 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 |
| CN112040985A (zh) | 2018-02-07 | 2020-12-04 | 瑞泽恩制药公司 | 用于递送治疗性蛋白质的方法和组合物 |
| EP3762505A4 (en) * | 2018-03-09 | 2021-12-01 | Avrobio, Inc. | COMPOSITIONS AND METHODS OF TREATMENT FOR PARKINSON'S DISEASE |
| TW202344691A (zh) * | 2018-04-30 | 2023-11-16 | 美商阿米庫斯醫療股份有限公司 | 基因治療構築體及使用方法 |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| JP2021526026A (ja) | 2018-06-08 | 2021-09-30 | エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ | 翻訳後修飾が低減したペプチドリンカー |
| JP7577650B2 (ja) | 2018-10-10 | 2024-11-05 | アミカス セラピューティックス インコーポレイテッド | ジスルフィド結合により安定したポリペプチド組成物及びその使用方法 |
| CA3120087A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| JP2022552254A (ja) * | 2019-10-10 | 2022-12-15 | アミカス セラピューティックス インコーポレイテッド | バリアントｉｇｆ２構築体 |
| EP4081241A4 (en) | 2019-12-24 | 2024-05-01 | Juvena Therapeutics, Inc. | REGENERATIVE POLYPEPTIDES AND USES THEREOF |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| EP4359425B1 (en) | 2021-06-21 | 2026-08-05 | Juvena Therapeutics, Inc. | Igf2 fusion polypeptide and use in the treatment of muscular dystrophy, sarcopenia, cachexia, or muscle injury |
| EP4359430A4 (en) * | 2021-06-23 | 2025-07-16 | Lycia Therapeutics Inc | BIFUNCTIONAL COMPOUNDS CONTAINING IGF-2 POLYPEPTIDES |
| CN117343197A (zh) * | 2021-10-15 | 2024-01-05 | 中山大学 | 重组融合抗体 |
| EP4654982A1 (en) * | 2023-01-27 | 2025-12-03 | Regeneron Pharmaceuticals, Inc. | Modified rhabdovirus glycoproteins and uses thereof |
| CN117122690A (zh) * | 2023-08-28 | 2023-11-28 | 河北工业大学 | 一种溶酶体靶向嵌合体及其制备方法和应用 |
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| US5206161A (en) | 1991-02-01 | 1993-04-27 | Genentech, Inc. | Human plasma carboxypeptidase B |
| AUPN674895A0 (en) | 1995-11-23 | 1995-12-14 | Women's And Children's Hospital | Synthetic mammalian alpha-N-acetylglucosaminidase and genetic sequences encoding same |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| DE10035433C2 (de) | 2000-07-20 | 2002-07-18 | Tuma Wolfgang | Schonende Hochanreicherung von fetalen Zellen aus pripherem Blut und Verwendung derselben |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
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| EP1436316B1 (en) * | 2001-04-30 | 2008-01-23 | ZyStor Therapeutics , Inc. | Subcellular targeting of therapeutic proteins |
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| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
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| ES2371072T3 (es) | 2003-06-12 | 2011-12-27 | Eli Lilly And Company | Proteínas de fusión análogas de glp-1. |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| DE602005020745D1 (de) | 2004-02-10 | 2010-06-02 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
| WO2006096653A2 (en) | 2005-03-04 | 2006-09-14 | Biogen Idec Ma Inc. | Methods of humanizing immunoglobulin variable regions through rational modification of complementarity determining residues |
| WO2009137721A2 (en) | 2008-05-07 | 2009-11-12 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| US8697654B2 (en) * | 2008-12-18 | 2014-04-15 | E I Du Pont De Nemours And Company | Peptide linkers for effective multivalent peptide binding |
| US9029530B2 (en) | 2009-01-02 | 2015-05-12 | Biomarin Pharmaceutical Inc. | Detection of oligosaccharides |
| US8785168B2 (en) * | 2009-06-17 | 2014-07-22 | Biomarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| 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 |
| MX335966B (es) | 2010-06-25 | 2016-01-06 | Amcor Ltd | Sistema de depuración de oxígeno para un recipiente. |
| US9409950B2 (en) * | 2010-12-23 | 2016-08-09 | Biogen Ma Inc. | Linker peptides and polypeptides comprising same |
| BR112013022557A2 (pt) * | 2011-03-04 | 2017-08-01 | Shire Human Genetic Therapies | ligantes de peptídeo composições de polipeptídeo e métodos para uso dos mesmos |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| WO2012122477A1 (en) * | 2011-03-10 | 2012-09-13 | Novozymes A/S | Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same |
| ES2679374T3 (es) | 2012-11-27 | 2018-08-24 | Biomarin Pharmaceutical Inc. | Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas |
-
2013
- 2013-11-27 ES ES13802834.5T patent/ES2679374T3/es active Active
- 2013-11-27 LT LTEP16182420.6T patent/LT3115372T/lt unknown
- 2013-11-27 CN CN201380061935.4A patent/CN104822701B/zh active Active
- 2013-11-27 KR KR1020157017224A patent/KR102262882B1/ko active Active
- 2013-11-27 HU HUE13802834A patent/HUE039334T2/hu unknown
- 2013-11-27 WO PCT/US2013/072287 patent/WO2014085621A1/en not_active Ceased
- 2013-11-27 MX MX2015006644A patent/MX367024B/es active IP Right Grant
- 2013-11-27 TW TW102143329A patent/TWI626250B/zh active
- 2013-11-27 RU RU2015125491A patent/RU2680581C2/ru active
- 2013-11-27 MX MX2019009191A patent/MX377150B/es active IP Right Grant
- 2013-11-27 PT PT138028345T patent/PT2925776T/pt unknown
- 2013-11-27 JP JP2015544209A patent/JP6831176B2/ja active Active
- 2013-11-27 AR ARP130104367A patent/AR093626A1/es active IP Right Grant
- 2013-11-27 DK DK13802834.5T patent/DK2925776T3/en active
- 2013-11-27 PT PT16182420T patent/PT3115372T/pt unknown
- 2013-11-27 AU AU2013352184A patent/AU2013352184B2/en active Active
- 2013-11-27 ES ES16182420T patent/ES2729997T3/es active Active
- 2013-11-27 HU HUE16182420A patent/HUE043679T2/hu unknown
- 2013-11-27 TW TW107114660A patent/TWI711632B/zh active
- 2013-11-27 PL PL16182420T patent/PL3115372T3/pl unknown
- 2013-11-27 CA CA2892146A patent/CA2892146A1/en active Pending
- 2013-11-27 KR KR1020237012007A patent/KR20230054482A/ko not_active Ceased
- 2013-11-27 KR KR1020217017007A patent/KR102385392B1/ko active Active
- 2013-11-27 PL PL13802834T patent/PL2925776T3/pl unknown
- 2013-11-27 RS RS20190585A patent/RS58916B1/sr unknown
- 2013-11-27 DK DK16182420.6T patent/DK3115372T3/da active
- 2013-11-27 EP EP13802834.5A patent/EP2925776B1/en active Active
- 2013-11-27 EP EP16182420.6A patent/EP3115372B1/en active Active
- 2013-11-27 KR KR1020227011416A patent/KR102521039B1/ko active Active
- 2013-11-27 US US14/092,336 patent/US9376480B2/en active Active
- 2013-11-27 BR BR112015012152-7A patent/BR112015012152B1/pt active IP Right Grant
- 2013-11-27 SI SI201331450T patent/SI3115372T1/sl unknown
- 2013-11-27 HR HRP20181351TT patent/HRP20181351T1/hr unknown
-
2015
- 2015-05-14 IL IL238824A patent/IL238824B/en active IP Right Grant
- 2015-05-19 ZA ZA2015/03509A patent/ZA201503509B/en unknown
- 2015-05-20 CL CL2015001371A patent/CL2015001371A1/es unknown
- 2015-10-14 US US14/883,169 patent/US9845346B2/en active Active
- 2015-10-14 US US14/883,211 patent/US9771408B2/en active Active
- 2015-10-14 US US14/883,193 patent/US9834587B2/en active Active
- 2015-10-14 US US14/883,203 patent/US9834588B2/en active Active
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2017
- 2017-08-28 US US15/688,438 patent/US10301369B2/en active Active
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2018
- 2018-11-13 IL IL262976A patent/IL262976B/en active IP Right Grant
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2019
- 2019-04-08 US US16/378,163 patent/US11254725B2/en active Active
- 2019-05-17 HR HRP20190918TT patent/HRP20190918T1/hr unknown
- 2019-06-06 CY CY20191100600T patent/CY1122555T1/el unknown
- 2019-07-24 JP JP2019136013A patent/JP6913719B2/ja active Active
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2020
- 2020-02-23 IL IL272854A patent/IL272854B/en unknown
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2022
- 2022-01-10 US US17/572,018 patent/US20220127326A1/en not_active Abandoned
-
2025
- 2025-01-17 US US19/030,525 patent/US20250388644A1/en active Pending
Also Published As
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Legal Events
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| EEER | Examination request |
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