CO2020003128A2 - Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas - Google Patents
Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimasInfo
- Publication number
- CO2020003128A2 CO2020003128A2 CONC2020/0003128A CO2020003128A CO2020003128A2 CO 2020003128 A2 CO2020003128 A2 CO 2020003128A2 CO 2020003128 A CO2020003128 A CO 2020003128A CO 2020003128 A2 CO2020003128 A2 CO 2020003128A2
- Authority
- CO
- Colombia
- Prior art keywords
- fusion proteins
- replacement therapy
- enzyme replacement
- enzymes
- therapy enzymes
- Prior art date
Links
Classifications
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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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- 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
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2881—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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)
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
-
- 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)
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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
- C12N9/14—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)
- C12N9/2405—Glucanases
- C12N9/2434—Glucanases acting on beta-1,4-glucosidic bonds
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- 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)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01001—Carboxylesterase (3.1.1.1)
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- 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)
- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
- C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)
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- 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)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
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- 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)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y310/00—Hydrolases acting on sulfur-nitrogen bonds (3.10)
- C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
- C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/55—Fab or Fab'
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
- C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
- C07K2317/622—Single chain antibody (scFv)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
- C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
Abstract
En la presente se proporcionan proteínas de fusión que comprenden una enzima de terapia de reemplazo de enzimas y una región Fc, así como métodos de uso de dichas proteínas para tratar un trastorno de almacenamiento lisosómico. En la presente también se proporcionan métodos para transportar agentes a través de la barrera hematoencefálica.
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762566898P | 2017-10-02 | 2017-10-02 | |
| US201762583276P | 2017-11-08 | 2017-11-08 | |
| US201862626365P | 2018-02-05 | 2018-02-05 | |
| US201862678183P | 2018-05-30 | 2018-05-30 | |
| US201862721396P | 2018-08-22 | 2018-08-22 | |
| PCT/US2018/053747 WO2019070577A1 (en) | 2017-10-02 | 2018-10-01 | FUSION PROTEINS COMPRISING SUBSTITUTE ENZYM THERAPY ENZYMES |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2020003128A2 true CO2020003128A2 (es) | 2020-06-19 |
Family
ID=64267892
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2020/0003128A CO2020003128A2 (es) | 2017-10-02 | 2020-03-16 | Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas |
Country Status (21)
| Country | Link |
|---|---|
| US (3) | US20200277584A1 (es) |
| EP (2) | EP3904389A1 (es) |
| JP (2) | JP7854267B2 (es) |
| KR (1) | KR20200058510A (es) |
| CN (2) | CN111278859A (es) |
| AU (2) | AU2018345303B2 (es) |
| BR (1) | BR112020005271A2 (es) |
| CA (1) | CA3076369A1 (es) |
| CL (1) | CL2020000810A1 (es) |
| CO (1) | CO2020003128A2 (es) |
| CR (1) | CR20200129A (es) |
| IL (1) | IL273439B2 (es) |
| MA (1) | MA50746A (es) |
| MX (3) | MX2020002918A (es) |
| MY (1) | MY209029A (es) |
| PE (1) | PE20210347A1 (es) |
| PH (1) | PH12020550510A1 (es) |
| SA (1) | SA520411662B1 (es) |
| SG (1) | SG11202002730XA (es) |
| TW (1) | TW201925236A (es) |
| WO (1) | WO2019070577A1 (es) |
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| CN110506055A (zh) | 2017-02-17 | 2019-11-26 | 戴纳立制药公司 | 工程改造的转铁蛋白受体结合多肽 |
| CR20200129A (es) | 2017-10-02 | 2020-08-22 | Denali Therapeutics Inc | Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas |
| WO2020037150A2 (en) * | 2018-08-16 | 2020-02-20 | Denali Therapeutics Inc. | Engineered bispecific proteins |
| US20220025065A1 (en) | 2018-12-10 | 2022-01-27 | Denali Therapeutics Inc. | Lysosomal storage disorder biomarkers and methods of use thereof |
| BR112021015656A2 (pt) | 2019-02-20 | 2021-10-05 | Denali Therapeutics Inc. | Anticorpos anti-trem2 e métodos de uso dos mesmos |
| CA3134538A1 (en) * | 2019-04-03 | 2020-10-08 | Denali Therapeutics Inc. | Formulations of protein molecules comprising iduronate 2-sulfatase |
| CN110111326B (zh) * | 2019-05-15 | 2021-01-15 | 西安科技大学 | 基于ert系统的重建图像质量评价方法 |
| PH12022551598A1 (en) | 2019-12-23 | 2023-11-20 | Denali Therapeutics Inc | Progranulin variants |
| JP2023512450A (ja) | 2020-01-13 | 2023-03-27 | デナリ セラピューティクス インコーポレイテッド | 抗trem2抗体及びその使用方法 |
| WO2021158986A1 (en) * | 2020-02-07 | 2021-08-12 | Denali Therapeutics Inc. | Methods for the treatment of hunter syndrome |
| EP4229192A1 (en) * | 2020-10-14 | 2023-08-23 | Denali Therapeutics Inc. | Fusion proteins comprising sulfoglucosamine sulfohydrolase enzymes and methods thereof |
| KR20220073454A (ko) * | 2020-11-26 | 2022-06-03 | 한미약품 주식회사 | 신규한 뇌 표적 지속형 단백질 결합체 및 이의 제조방법 |
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| EP4373511A1 (en) | 2021-07-23 | 2024-05-29 | Denali Therapeutics Inc. | Methods for the treatment of hunter syndrome |
| AU2022415476A1 (en) | 2021-12-17 | 2024-07-04 | Denali Therapeutics Inc. | Fusion proteins comprising alpha-l-iduronidase enzymes and methods |
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| EP4526344A1 (en) * | 2022-05-16 | 2025-03-26 | Denali Therapeutics Inc. | Transferrin receptor-binding domains and proteins comprising the same |
| US20250372225A1 (en) | 2022-06-13 | 2025-12-04 | Denali Therapeutics Inc. | A quantitative pharmacological model of enzyme replacement therapy |
| CN115032377B (zh) * | 2022-08-11 | 2022-11-01 | 裕菁科技(上海)有限公司 | 一种粘多糖贮积症生物标记物及应用 |
| WO2024211624A1 (en) * | 2023-04-04 | 2024-10-10 | University Of Cincinnati | Novel treatment strategies for pancreatic cancer using recombinant acid sphingomyelinase |
| EP4698671A2 (en) | 2023-04-21 | 2026-02-25 | Denali Therapeutics Inc. | Methods of increasing sialic acid levels in recombinant glycosylated proteins |
| TW202542190A (zh) | 2023-12-20 | 2025-11-01 | 美商戴納立製藥公司 | 包含酸性α—葡萄糖苷酶之融合蛋白及其方法 |
| WO2025166045A1 (en) * | 2024-01-31 | 2025-08-07 | Alector Llc | β-GLUCOCEREBROSIDASE ENZYMES, FUSION PROTEINS AND COMPLEXES COMPRISING THE SAME, AND METHODS OF USE THEREOF |
| WO2026006173A1 (en) | 2024-06-24 | 2026-01-02 | Denali Therapeutics Inc. | Fusion proteins comprising modified glucocerebrosidase polypeptides and methods thereof |
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