CR20200129A - 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 enzimas

Info

Publication number
CR20200129A
CR20200129A CR20200129A CR20200129A CR20200129A CR 20200129 A CR20200129 A CR 20200129A CR 20200129 A CR20200129 A CR 20200129A CR 20200129 A CR20200129 A CR 20200129A CR 20200129 A CR20200129 A CR 20200129A
Authority
CR
Costa Rica
Prior art keywords
fusion proteins
replacement therapy
enzyme replacement
proteins including
including enzymes
Prior art date
Application number
CR20200129A
Other languages
English (en)
Inventor
Anastasia Henry
Adam P Silverman
Cathal Mahon
Julie Ullman
Jennifer A Getz
Mihalis Kariolis
Junhua Wang
Giuseppe Astarita
Mark S Dennis
Ankita Srivastava
Joy Yu Zuchero
Original Assignee
Denali Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Denali Therapeutics Inc filed Critical Denali Therapeutics Inc
Publication of CR20200129A publication Critical patent/CR20200129A/es

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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)
    • 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
    • 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)
    • 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)
    • 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)
    • 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
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (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)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicinal Preparation (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.
CR20200129A 2017-10-02 2018-10-01 Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas CR20200129A (es)

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
CR20200129A true CR20200129A (es) 2020-08-22

Family

ID=64267892

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20200129A CR20200129A (es) 2017-10-02 2018-10-01 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 (1) JP2023179600A (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)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112019016989A2 (pt) 2017-02-17 2020-05-26 Denali Therapeutics Inc. Polipeptídeos de ligação ao receptor de transferrina manipulados
AU2018345303B2 (en) 2017-10-02 2025-09-18 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
MA53616A (fr) * 2018-08-16 2022-05-04 Denali Therapeutics Inc Protéines bispécifiques modifiées
EP3894864A2 (en) 2018-12-10 2021-10-20 Denali Therapeutics Inc. Lysosomal storage disorder biomarkers and methods of use thereof
PH12021552002A1 (en) 2019-02-20 2022-09-19 Denali Therapeutics Inc Anti-trem2 antibodies and methods of use thereof
US20220184186A1 (en) * 2019-04-03 2022-06-16 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
CA3165846A1 (en) 2019-12-23 2021-07-01 Denali Therapeutics Inc. Progranulin variants
PE20221465A1 (es) 2020-01-13 2022-09-21 Denali Therapeutics Inc Anticuerpos anti-trem2 y metodos para utilizarlos
EP4100419A4 (en) * 2020-02-07 2024-05-29 Denali Therapeutics Inc. METHODS OF TREATING HUNTER SYNDROME
PE20231931A1 (es) 2020-10-14 2023-12-01 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas
KR20220073454A (ko) * 2020-11-26 2022-06-03 한미약품 주식회사 신규한 뇌 표적 지속형 단백질 결합체 및 이의 제조방법
EP4291246A4 (en) * 2021-02-11 2024-12-11 Denali Therapeutics Inc. ANTI-TRANSFERRIN RECEPTOR FUSION PROTEINS AND METHODS OF USE THEREOF
US20250018015A1 (en) 2021-07-23 2025-01-16 Denali Therapeutics Inc. Methods for the treatment of hunter syndrome
JP2024546129A (ja) 2021-12-17 2024-12-17 デナリ セラピューティクス インコーポレイテッド アルファ－ｌ－イズロニダーゼ酵素を含む融合タンパク質及びその方法
WO2023198661A1 (en) * 2022-04-12 2023-10-19 F. Hoffmann-La Roche Ag Fusion proteins targeted to the central nervous system
US20250333528A1 (en) * 2022-05-16 2025-10-30 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

Family Cites Families (147)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6015555A (en) * 1995-05-19 2000-01-18 Alkermes, Inc. Transferrin receptor specific antibody-neuropharmaceutical or diagnostic agent conjugates
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
ATE384736T1 (de) 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
AU2003222568B2 (en) 2002-01-11 2009-05-07 Bioasis Technologies, Inc. Use of P97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
EP1545595B1 (en) * 2002-08-30 2010-07-07 Biorexis Pharmaceutical Corporation Modified transferrin fusion proteins comprising duplicate transferrin amino or carboxy terminal domains
US20050142141A1 (en) * 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
US7388079B2 (en) 2002-11-27 2008-06-17 The Regents Of The University Of California Delivery of pharmaceutical agents via the human insulin receptor
EP2325301B1 (en) 2003-02-11 2015-09-02 Shire Human Genetic Therapies, Inc. Diagnosis and treatment of multiple sulfatase deficiency and others using a formylglycine generating enzyme (FGE)
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
WO2004108071A2 (en) 2003-06-05 2004-12-16 Salk Institute For Biological Studies Targeting polypeptides to the central nervous system
US20050026823A1 (en) 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
ATE555133T1 (de) 2003-11-13 2012-05-15 Hanmi Holdings Co Ltd Igg fc fragment für einen arzneimittelträger und verfahren zu dessen herstellung
US20050249723A1 (en) 2003-12-22 2005-11-10 Xencor, Inc. Fc polypeptides with novel Fc ligand binding sites
US8536315B2 (en) 2004-01-30 2013-09-17 Shire Pharmaceuticals Ireland Limited Production and purification of recombinant arylsulftase
US8889127B2 (en) 2004-07-01 2014-11-18 Icahn School Of Medicine At Mount Sinai Targeted protein replacement for the treatment of lysosomal storage disorders
CN103555733A (zh) 2005-01-05 2014-02-05 F-星生物技术研究与开发有限公司 分子中互补决定区以外的区域中工程改造了的具有结合特性的合成免疫球蛋白结构域
EP1877099B1 (en) 2005-04-06 2012-09-19 Genzyme Corporation Therapeutic conjugates comprising a lysosomal enzyme, polysialic acid and a targeting moiety
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
EP1981546B1 (en) 2006-02-06 2014-06-04 The Medical Research and Infrastructure Fund of the Tel-Aviv Sourasky Medical Center Enzyme replacement therapy for treating lysosomal storage diseases
WO2008054544A2 (en) 2006-05-22 2008-05-08 Immune Disease Institute, Inc. Method for delivery across the blood brain barrier
US7871624B2 (en) * 2006-06-27 2011-01-18 Saint Louis University Chimeral polypeptide composition for cross-placenta delivery
WO2008105796A2 (en) 2006-06-29 2008-09-04 Albert Einstein College Of Medicine Of Yeshiva University Compounds and methods for detecting ricin and uses thereof
AT503889B1 (de) 2006-07-05 2011-12-15 Star Biotechnologische Forschungs Und Entwicklungsges M B H F Multivalente immunglobuline
WO2008089339A2 (en) 2007-01-18 2008-07-24 Genzyme Corporation Oligosaccharide conjugates for cellular targeting
CN105274072A (zh) 2008-01-18 2016-01-27 生物马林药物股份有限公司 制备活性高度磷酸化人溶酶体硫酸酯酶及其应用
HRP20170698T1 (hr) 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lizosomski usmjereni peptidi i njihove uporabe
SI3998078T1 (sl) 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
US9598682B2 (en) 2009-09-29 2017-03-21 Vib Vzw Hydrolysis of mannose-1-phospho-6-mannose linkage to phospho-6-mannose
JP2013507131A (ja) 2009-10-09 2013-03-04 アーメイゲン・テクノロジーズ・インコーポレイテッド Ｃｎｓにおけるイズロン酸２−スルファターゼ活性を増加させるための方法および組成物
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
CN103179980B (zh) 2010-06-25 2016-09-28 夏尔人类遗传性治疗公司 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物
RU2013103763A (ru) 2010-07-02 2014-08-10 Ангиохем Инк. Короткие и содержащие d-аминокислоты полипептиды для терапевтических конъюгатов и их применения
WO2012042387A2 (en) 2010-09-29 2012-04-05 Oxyrane Uk Limited De-mannosylation of phosphorylated n-glycans
WO2012050175A1 (ja) 2010-10-15 2012-04-19 日本ケミカルリサーチ株式会社 糖鎖の非還元末端がマンノース残基である糖蛋白質の製造方法
WO2012063799A1 (ja) 2010-11-08 2012-05-18 日本ケミカルリサーチ株式会社 新規発現ベクター
AR084020A1 (es) 2010-11-30 2013-04-17 Genentech Inc Anticuerpos para el receptor de la barrera hematoencefalica de baja afinidad y sus usos
AU2010366066B2 (en) 2010-12-22 2016-01-14 Fondazione Telethon Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses
WO2012101671A1 (en) 2011-01-25 2012-08-02 Jcr Pharmaceuticals Co., Ltd. Method for production of human recombinant iduronate 2-sulfatase
WO2012136790A1 (en) * 2011-04-07 2012-10-11 Glaxo Group Limited Compositions comprising fusion proteins or conjugates with an improved half -life
EA201892619A1 (ru) 2011-04-29 2019-04-30 Роше Гликарт Аг Иммуноконъюгаты, содержащие мутантные полипептиды интерлейкина-2
AU2012260434A1 (en) 2011-05-23 2013-10-31 Phylogica Limited Method of determining, identifying or isolating cell-penetrating peptides
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
WO2012177997A1 (en) 2011-06-22 2012-12-27 The General Hospital Corporation Treatment of proteinopathies
US20160145589A1 (en) 2011-06-24 2016-05-26 Green Cross Corporation Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof
KR101158673B1 (ko) 2011-06-24 2012-07-03 주식회사 지씨바이오 재조합 인간 이듀로네이트-2-설파타아제를 포함하는 조성물, 제제 및 이의 제조방법
CA2842492A1 (en) 2011-08-05 2013-02-14 Bioasis Technologies, Inc. P97 fragments with transfer activity
JP6329483B2 (ja) 2011-10-12 2018-05-23 シナジェバ・バイオファーマ・コーポレイションＳｙｎａｇｅｖａ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐ． 組換えヒトｎａｇｌｕタンパク質およびその利用
EP2785838A4 (en) 2011-12-01 2015-07-01 Angiochem Inc VECTORIZED LYSOSOMAL ENZYME COMPOUNDS
WO2013078562A2 (en) 2011-12-01 2013-06-06 Angiochem Inc. Targeted enzyme compounds and uses thereof
AU2012346448B2 (en) 2011-12-02 2017-09-14 Armagen, Inc. Methods and compositions for increasing arylsulfatase A activity in the CNS
EP2793933B1 (en) 2011-12-23 2019-04-17 Shire Human Genetic Therapies, Inc. Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase
EA201491578A1 (ru) 2012-03-30 2015-04-30 Шир Хьюман Дженетик Терапис, Инк. Подкожное введение идуронат-2-сульфатазы
WO2013162050A1 (ja) 2012-04-23 2013-10-31 株式会社Ｎｒｌファーマ ラクトフェリン融合タンパク質及びその製造方法
WO2013161958A1 (ja) 2012-04-27 2013-10-31 日本ケミカルリサーチ株式会社 新規な発現ベクター
AU2013266611B2 (en) * 2012-05-21 2016-08-11 Genentech, Inc. Methods for improving safety of blood-brain barrier transport
WO2013181454A1 (en) 2012-05-30 2013-12-05 Biostrategies LC Plant lectins as carriers of associated drug substances into animal and human cells
EP2855520B1 (en) 2012-06-04 2018-09-26 Novartis AG Site-specific labeling methods and molecules produced thereby
AU2013273894A1 (en) 2012-06-15 2015-02-05 Angiochem Inc. Targeted iduronidase compounds
US9150841B2 (en) 2012-06-29 2015-10-06 Shire Human Genetic Therapies, Inc. Cells for producing recombinant iduronate-2-sulfatase
KR101380740B1 (ko) 2012-06-29 2014-04-11 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 이듀로네이트-2-설파타제의 정제
US20140004097A1 (en) 2012-06-29 2014-01-02 Shire Human Genetic Therapies, Inc. Method of producing recombinant iduronate-2-sulfatase
ES2647082T3 (es) 2012-07-31 2017-12-19 Bioasis Technologies Inc Proteínas desfosforiladas de la enfermedad de depósito lisosómico y métodos de uso de las mismas
US10138474B2 (en) 2012-08-17 2018-11-27 Research Foundation Of The City University Of New York Scavenger receptor uptake for fabry disease enzyme replacement therapy
RU2711552C2 (ru) 2012-08-29 2020-01-17 Ф. Хоффманн-Ля Рош Аг Шаттл для гематоэнцефалического барьера
WO2014082080A2 (en) 2012-11-26 2014-05-30 Callidus Biopharma, Inc. Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
HUE039334T2 (hu) 2012-11-27 2018-12-28 Biomarin Pharm Inc Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai
JP2015536658A (ja) 2012-11-30 2015-12-24 アンジオケム インコーポレーテッド 標的化イズロン酸−２−スルファターゼ化合物
EP2939176A4 (en) 2012-12-27 2016-07-27 Schneider Electric It Corp Systems and methods of visualizing airflow
JP6586412B2 (ja) 2013-03-13 2019-10-02 バイオアシス テクノロジーズ インコーポレイテッド ｐ９７のフラグメントおよびその使用
US20160184458A1 (en) 2013-03-14 2016-06-30 Shire Human Genetic Therapies, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
WO2014189973A2 (en) 2013-05-20 2014-11-27 Genentech, Inc. Anti-transferrin receptor antibodies and methods of use
WO2014194427A1 (en) 2013-06-06 2014-12-11 Angiochem Inc. Targeted iduronate-2-sulfatase fusion proteins
LT3004896T (lt) 2013-06-07 2020-01-10 Genzyme Corporation Rūgštinės sfingomielinazės sutrikimų žymuo ir jo panaudojimas
WO2015009052A1 (ko) * 2013-07-16 2015-01-22 일동제약 주식회사 하이브리드 면역글로불린 fc와 효소의 융합단백질
WO2015009961A1 (en) 2013-07-19 2015-01-22 Armagen Technologies, Inc. Compositions and methods related to structures that cross the blood brain barrier
JP6692293B2 (ja) 2013-07-22 2020-05-13 アーマジェン・インコーポレイテッドＡｒｍａｇｅｎ， Ｉｎｃ． Ｃｎｓにおける酵素活性を増大するための方法および組成物
US20150093399A1 (en) 2013-08-28 2015-04-02 Bioasis Technologies, Inc. Cns-targeted conjugates having modified fc regions and methods of use thereof
US11261262B2 (en) 2013-09-03 2022-03-01 Novimmune Sa Readily isolated bispecific binding molecules with native format having mutated constant regions
JP6309427B2 (ja) 2013-10-09 2018-04-11 Ｊｃｒファーマ株式会社 イズロン酸−２−スルファターゼの活性測定法
US9994641B2 (en) 2013-12-25 2018-06-12 Jcr Pharmaceuticals Co., Ltd. Anti-human transferrin receptor antibody that passes through blood-brain barrier
CA2933384A1 (en) 2014-01-03 2015-07-09 F. Hoffmann-La Roche Ag Bispecific anti-hapten/anti-blood brain barrier receptor antibodies, complexes thereof and their use as blood brain barrier shuttles
CA2932547C (en) 2014-01-06 2023-05-23 F. Hoffmann-La Roche Ag Monovalent blood brain barrier shuttle modules
DK3107562T3 (da) 2014-02-19 2019-12-16 Bioasis Technologies Inc P97-ids-fusionsproteiner
HUE039691T2 (hu) 2014-04-01 2019-01-28 Swedish Orphan Biovitrum Ab Publ Módosított szulfamidáz és elõállítása
US10058619B2 (en) 2014-05-01 2018-08-28 Bioasis Technologies, Inc. P97-polynucleotide conjugates
US20170191041A1 (en) 2014-07-11 2017-07-06 Biostrategies LC Materials and methods for treating disorders associated with sulfatase enzymes
US10068619B1 (en) 2014-07-25 2018-09-04 Western Digital Technologies, Inc. Hand held storage device
US10722559B2 (en) 2014-08-11 2020-07-28 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
RS59340B1 (sr) 2014-11-06 2019-10-31 Hoffmann La Roche Varijante fc regiona sa modifikovanim vezivanjem za fcrn i metode upotrebe
EP3218000A2 (en) 2014-11-10 2017-09-20 Alexion Pharmaceuticals, Inc. Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof
MA40954A (fr) * 2014-11-14 2017-09-19 Shire Human Genetic Therapies Détermination de niveaux de glycosaminoglycane par spectrométrie de masse
WO2016077840A2 (en) 2014-11-14 2016-05-19 Ossianix, Inc. TfR SELECTIVE BINDING COMPOUNDS AND RELATED METHODS
EP3221362B1 (en) 2014-11-19 2019-07-24 F.Hoffmann-La Roche Ag Anti-transferrin receptor antibodies and methods of use
EP3221361B1 (en) 2014-11-19 2021-04-21 Genentech, Inc. Anti-transferrin receptor / anti-bace1 multispecific antibodies and methods of use
MA41198A (fr) 2014-12-18 2017-10-24 Shire Human Genetic Therapies Essais d'activité enzymatique pour i2s
US10538589B2 (en) 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
CA2973343C (en) 2015-02-05 2023-08-08 R.P. Scherer Technologies, Llc Activated formylglycine-generating enzymes and methods of producing and using the same
CA2987684A1 (en) 2015-06-01 2016-12-08 The Hospital For Sick Children Delivery of structurally diverse polypeptide cargo into mammalian cells by a bacterial toxin
TWI769982B (zh) 2015-06-24 2022-07-11 日商Ｊｃｒ製藥股份有限公司 通過血腦障壁之抗人類運鐵蛋白受體抗體
CN107771156B (zh) 2015-06-24 2020-10-27 伯曼集团股份公司 线分拣机
MX391086B (es) * 2015-06-24 2025-03-21 Hoffmann La Roche Anticuerpos anti-receptor de transferrina con afinidad diseñada.
KR20170004814A (ko) 2015-07-02 2017-01-11 주식회사 녹십자 헌터증후군 치료제
WO2017003270A1 (ko) 2015-07-02 2017-01-05 주식회사 녹십자 헌터증후군 치료제 및 치료방법
US20180264089A1 (en) 2015-10-01 2018-09-20 Swedish Orphan Biovitrum Ab (Publ) Modified iduronate 2-sulfatase and production thereof
CA3000289A1 (en) 2015-10-01 2017-04-06 Swedish Orphan Biovitrum Ab (Publ) Modified lysosomal protein and production thereof
WO2017062666A2 (en) 2015-10-06 2017-04-13 Patricia Dickson Enzyme replacement therapy for mucopolysaccharidosis iiid
US20180221459A1 (en) 2015-10-06 2018-08-09 LA BioMedical Research Institute at Harbor-UCLA Medical Center Enzyme replacement therapy for mucopolysaccharidosis iiid
AU2016365834B2 (en) 2015-12-08 2023-09-28 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
GB201522243D0 (en) 2015-12-16 2016-01-27 Ucl Business Plc Treatment
WO2017116066A1 (en) 2015-12-30 2017-07-06 Green Cross Corporation Methods and compositions for treating hunter syndrome
AR107483A1 (es) 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
EP4275751A3 (en) 2016-02-24 2023-12-27 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
US20190046637A1 (en) 2016-03-01 2019-02-14 Biostrategies LC Materials and methods for mitigating immune-sensitization
IL305449B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
US10169252B2 (en) 2016-07-15 2019-01-01 International Business Machines Corporation Configuring functional capabilities of a computer system
CA3033082A1 (en) 2016-08-06 2018-02-15 Ossianix, Inc. In vivo methods for selecting peptides that cross the blood brain barrier, related compositions and methods of use
AU2017316955B2 (en) 2016-08-25 2022-03-03 Jcr Pharmaceuticals Co., Ltd. Method for producing antibody fusion protein
US20190352335A1 (en) * 2016-12-19 2019-11-21 Hanmi Pharm. Co., Ltd. Brain targeting long-acting protein conjugate
TWI761413B (zh) * 2016-12-26 2022-04-21 日商Ｊｃｒ製藥股份有限公司 通過血腦障壁之新穎抗人類運鐵蛋白受體抗體
US10940185B2 (en) 2016-12-28 2021-03-09 Jcr Pharmaceuticals Co., Ltd. Lyophilized preparation
GB201701968D0 (en) 2017-02-07 2017-03-22 Univ Manchester Gene therapy
WO2018152285A1 (en) 2017-02-17 2018-08-23 Denali Therapeutics Inc. Transferrin receptor transgenic models
BR112019017021A2 (pt) * 2017-02-17 2020-04-14 Denali Therapeutics Inc anticorpos anti-tau e métodos de uso dos mesmos
EP3583119A2 (en) 2017-02-17 2019-12-25 Denali Therapeutics Inc. Engineered polypeptides
US10457717B2 (en) 2017-02-17 2019-10-29 Denali Therapeutics Inc. Engineered polypeptides
BR112019016989A2 (pt) 2017-02-17 2020-05-26 Denali Therapeutics Inc. Polipeptídeos de ligação ao receptor de transferrina manipulados
JP7106289B2 (ja) 2017-02-21 2022-07-26 Ｊｃｒファーマ株式会社 グリコサミノグリカンの分析法
GB201702863D0 (en) 2017-02-22 2017-04-05 Evox Therapeutics Ltd Improved loading of EVs with therapeutic proteins
WO2019009884A1 (en) 2017-07-03 2019-01-10 Mark Anthony Allen VISUAL DRIVING AID FOR VEHICLE VISOR
SG11202000115SA (en) 2017-07-07 2020-02-27 Hanmi Pharmaceutical Co Ltd Novel therapeutic enzyme fusion protein and use thereof
WO2019022563A2 (ko) 2017-07-28 2019-01-31 한미약품 주식회사 이두로네이트 2-설파타제 결합체
SMT202300347T1 (it) 2017-08-10 2023-11-13 Denali Therapeutics Inc Polipeptidi ingegnerizzati che si legano al recettore della transferrina
KR102655498B1 (ko) 2017-09-07 2024-04-11 제이씨알 파마 가부시키가이샤 수성 의약 조성물
AU2018345303B2 (en) 2017-10-02 2025-09-18 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
WO2019094608A1 (en) 2017-11-08 2019-05-16 Denali Therapeutics Inc. Anti-bace1 antibodies and methods of use thereof
TW201934573A (zh) 2018-01-10 2019-09-01 美商戴納立製藥公司 轉鐵蛋白受體結合性多肽及其用途
CA3101202A1 (en) 2018-06-18 2019-12-26 Denali Therapeutics Inc. Fusion proteins comprising progranulin
MA53616A (fr) 2018-08-16 2022-05-04 Denali Therapeutics Inc Protéines bispécifiques modifiées
KR20210074279A (ko) 2018-08-22 2021-06-21 데날리 테라퓨틱스 인크. 항-her2 폴리펩타이드 및 이의 사용방법
US20220184186A1 (en) 2019-04-03 2022-06-16 Denali Therapeutics Inc. Formulations of protein molecules comprising iduronate 2-sulfatase
CA3165846A1 (en) 2019-12-23 2021-07-01 Denali Therapeutics Inc. Progranulin variants
PE20221465A1 (es) 2020-01-13 2022-09-21 Denali Therapeutics Inc Anticuerpos anti-trem2 y metodos para utilizarlos
EP4100419A4 (en) 2020-02-07 2024-05-29 Denali Therapeutics Inc. METHODS OF TREATING HUNTER SYNDROME
PE20231931A1 (es) 2020-10-14 2023-12-01 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas

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