ES2679374T3 - Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas - Google Patents
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- ES2679374T3 ES2679374T3 ES13802834.5T ES13802834T ES2679374T3 ES 2679374 T3 ES2679374 T3 ES 2679374T3 ES 13802834 T ES13802834 T ES 13802834T ES 2679374 T3 ES2679374 T3 ES 2679374T3
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- A61K38/46—Hydrolases (3)
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-
- 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
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- 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)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (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)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Toxicology (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- Obesity (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)
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| US201261730378P | 2012-11-27 | 2012-11-27 | |
| US201261730378P | 2012-11-27 | ||
| US201361788968P | 2013-03-15 | 2013-03-15 | |
| US201361788968P | 2013-03-15 | ||
| PCT/US2013/072287 WO2014085621A1 (en) | 2012-11-27 | 2013-11-27 | Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof |
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| ES16182420T Active ES2729997T3 (es) | 2012-11-27 | 2013-11-27 | Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas |
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| HUE034850T2 (en) * | 2008-05-07 | 2018-03-28 | Biomarin Pharm Inc | Lysosomal targeting peptides and their use |
| KR102521039B1 (ko) | 2012-11-27 | 2023-04-12 | 바이오마린 파머수티컬 인크. | 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들 |
| CN115120746A (zh) * | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| MX2017001898A (es) * | 2014-08-11 | 2017-04-11 | Shire Human Genetic Therapies | Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales. |
| US20180303877A1 (en) * | 2014-09-25 | 2018-10-25 | The General Hospital Corporation | Cell-based targeted delivery of pseudonomas exotoxin |
| EP3220958A1 (en) * | 2014-10-24 | 2017-09-27 | 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 |
| ES2971045T3 (es) | 2015-07-06 | 2024-06-03 | Regeneron Pharma | Moléculas multiespecíficas de unión a antígeno y usos de las mismas |
| EP3371593B1 (en) | 2015-11-06 | 2020-10-14 | BioMarin Pharmaceutical Inc. | Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes |
| AU2017222620B2 (en) * | 2016-02-24 | 2022-06-16 | Biomarin Pharmaceutical Inc. | Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof |
| SG11201808176TA (en) | 2016-04-15 | 2018-10-30 | The Trustees Of The Univ Of Pennsyvania | 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 |
| KR102760654B1 (ko) | 2017-06-07 | 2025-02-04 | 리제너론 파마슈티칼스 인코포레이티드 | 효소 내재화를 위한 조성물 및 방법 |
| AU2018335752B2 (en) | 2017-09-22 | 2025-04-10 | Christian HINDERER | Gene therapy for treating Mucopolysaccharidosis type ii |
| MX2020002918A (es) | 2017-10-02 | 2020-07-22 | Denali Therapeutics Inc | Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas. |
| MA51796A (fr) | 2018-02-07 | 2020-12-16 | Regeneron Pharma | Procédés et compositions pour l'administration de protéines thérapeutiques |
| JP2021517168A (ja) * | 2018-03-09 | 2021-07-15 | アブロバイオ，インコーポレーテッド | パーキンソン病を処置するための組成物及び方法 |
| US10874750B2 (en) | 2018-04-30 | 2020-12-29 | Amicus Therapeutics, Inc. | Gene therapy constructs and methods of use |
| US12252544B2 (en) | 2018-05-17 | 2025-03-18 | Regeneron Pharmaceuticals, Inc. | Anti-CD63 antibodies, conjugates, and uses thereof |
| EP3802619A1 (en) | 2018-06-08 | 2021-04-14 | F. Hoffmann-La Roche AG | Peptidic linker with reduced post-translational modification |
| WO2020077114A2 (en) | 2018-10-10 | 2020-04-16 | Amicus Therapeutics, Inc. | Disulfide bond stabilized polypeptide compositions and methods of use |
| AU2019381803A1 (en) * | 2018-11-16 | 2021-06-10 | AskBio Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| AU2020361703A1 (en) * | 2019-10-10 | 2022-04-28 | Amicus Therapeutics, Inc. | Variant IGF2 constructs |
| EP4081241A4 (en) | 2019-12-24 | 2024-05-01 | Juvena Therapeutics, Inc. | REGENERATIVE POLYPEPTIDES AND THEIR USES |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| CN117999278A (zh) | 2021-06-21 | 2024-05-07 | 裘美娜治疗公司 | 再生性多肽及其用途 |
| US20240279360A1 (en) | 2021-06-23 | 2024-08-22 | Lycia Therapeutics, Inc. | Bifunctional compounds containing igf-2 polypeptides |
| CN115975039A (zh) * | 2021-10-15 | 2023-04-18 | 中山大学 | 重组融合抗体和抗体-药物偶联物及其用途 |
| CN120936364A (zh) * | 2023-01-27 | 2025-11-11 | 瑞泽恩制药公司 | 修饰的弹状病毒糖蛋白及其用途 |
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| KR102521039B1 (ko) | 2012-11-27 | 2023-04-12 | 바이오마린 파머수티컬 인크. | 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들 |
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2013
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2017
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2018
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2019
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2020
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2022
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