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 enzimas

Info

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
Application number
CONC2020/0003128A
Other languages
English (en)
Inventor
Giuseppe Astarita
Mark S Dennis
Jennifer A Getz
Anastasia Henry
Mihalis Kariolis
Cathal Mahon
Adam P Silverman
Ankita Srivastava
Julie Ullman
Junhua Wang
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 CO2020003128A2 publication Critical patent/CO2020003128A2/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/68Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K19/00Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18Carboxylic ester hydrolases (3.1.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405Glucanases
    • C12N9/2434Glucanases acting on beta-1,4-glucosidic bonds
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/01Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01001Carboxylesterase (3.1.1.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012Sphingomyelin phosphodiesterase (3.1.4.12)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/06Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013Iduronate-2-sulfatase (3.1.6.13)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y310/00Hydrolases acting on sulfur-nitrogen bonds (3.10)
    • C12Y310/01Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
    • C12Y310/01001N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/50Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55Fab or Fab'
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/60Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/90Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/92Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/30Non-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.
CONC2020/0003128A 2017-10-02 2020-03-16 Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas CO2020003128A2 (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
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)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP4291246A4 (en) * 2021-02-11 2024-12-11 Denali Therapeutics Inc. ANTI-TRANSFERRIN RECEPTOR FUSION PROTEINS AND METHODS OF USE THEREOF
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
EP4508206A1 (en) * 2022-04-12 2025-02-19 F. Hoffmann-La Roche AG Fusion proteins targeted to the central nervous system
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

Family Cites Families (148)

* 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
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
WO2002087510A2 (en) 2001-04-30 2002-11-07 Symbiontics, Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
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
US20060130158A1 (en) * 2002-08-30 2006-06-15 Turner Andrew J Modified transferrin fusion proteins comprising duplicate transferrin amino or carboxy terminal domains
US7388079B2 (en) 2002-11-27 2008-06-17 The Regents Of The University Of California Delivery of pharmaceutical agents via the human insulin receptor
US20050142141A1 (en) * 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
CA2515708A1 (en) 2003-02-11 2004-08-26 Transkaryotic Therapies, Inc. Diagnosis and treatment of multiple sulfatase deficiency and other sulfatase deficiencies
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
AU2003286870A1 (en) 2003-06-05 2005-01-04 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
ES2383300T3 (es) 2003-11-13 2012-06-20 Hanmi Holdings Co., Ltd Fragmento Fc de IgG para un vehículo de fármacos y procedimiento para su preparación
EP1697520A2 (en) 2003-12-22 2006-09-06 Xencor, Inc. Fc polypeptides with novel fc ligand binding sites
EP1713904B1 (en) 2004-01-30 2016-06-29 Shire Pharmaceuticals Ireland Limited Production and purification of recombinant arylsulfatase a
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
EP1699826B1 (en) 2005-01-05 2009-03-11 f-star Biotechnologische Forschungs- und Entwicklungsges.m.b.H. Synthetic immunoglobulin domains with binding properties engineered in regions of the molecule different from the complementarity determining regions
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
CA2641070C (en) 2006-02-06 2017-01-03 Ramot At Tel-Aviv University Ltd. 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
CA2711590C (en) 2008-01-18 2021-03-23 Biomarin Pharmaceutical Inc. Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
JP5627571B2 (ja) 2008-05-07 2014-11-19 バイオマリン ファーマシューティカル インコーポレイテッド リソソーム標的化ペプチドおよびその使用
SI3998078T1 (sl) 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
KR102000383B1 (ko) 2009-09-29 2019-07-15 유니버시테이트 젠트 만노스-1-포스포-6-만노스 결합의 포스포-6-만노스로의 가수분해
EP2485761B1 (en) * 2009-10-09 2019-02-27 Armagen, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
KR20230159646A (ko) 2010-06-25 2023-11-21 샤이어 휴먼 지네틱 테라피즈 인크. 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들
LT2588130T (lt) 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
JP2013530993A (ja) 2010-07-02 2013-08-01 アンジオケム インコーポレーテッド 治療用コンジュゲートのための短く且つd−アミノ酸を含有するポリペプチドおよびその使用
CA2812872C (en) 2010-09-29 2021-08-03 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
CN103201382A (zh) 2010-11-08 2013-07-10 日本化学研究株式会社 新表达载体
CA2818173C (en) 2010-11-30 2022-05-03 Genentech, Inc. Low affinity blood brain barrier receptor antibodies and uses therefor
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
WO2012159164A1 (en) 2011-05-23 2012-11-29 Phylogica Limited Method of determining, identifying or isolating cell-penetrating peptides
HRP20180291T1 (hr) 2011-05-27 2018-03-23 Amicus Therapeutics, Inc. Postupci za sparivanje ciljajućeg peptida na rekombinantne lizosomalne enzime za poboljšano liječenje lizosomskih bolesti nakupljanja
CA2840224C (en) 2011-06-22 2019-08-13 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
AU2012294673B2 (en) 2011-08-05 2015-11-26 Bioasis Technologies Inc. p97 fragments with transfer activity
SG10201510106PA (en) 2011-10-12 2016-01-28 Synageva Biopharma Corp Recombinant human naglu protein and uses thereof
BR112014013250A2 (pt) 2011-12-01 2019-09-24 Angiochem Inc compostos com enzima alvo e usos dos mesmos
CN104145015A (zh) 2011-12-01 2014-11-12 安吉奥开米公司 靶向的溶酶体酶化合物
AU2012346448B2 (en) 2011-12-02 2017-09-14 Armagen, Inc. Methods and compositions for increasing arylsulfatase A activity in the CNS
US9682129B2 (en) 2011-12-23 2017-06-20 Shire Human Genetic Therapies, Inc. Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase
MX2014011836A (es) 2012-03-30 2015-03-09 Shire Human Genetic Therapies Administracion subcutanea de iduronato-2-sulfatasa.
CA2871468C (en) 2012-04-23 2021-09-21 Nrl Pharma, Inc. Lactoferrin fusion protein and method for preparation thereof
US9657310B2 (en) 2012-04-27 2017-05-23 Jcr Pharmaceuticals Co., Ltd. Expression vector
WO2013177062A2 (en) 2012-05-21 2013-11-28 Genentech, Inc. Methods for improving safety of blood-brain barrier transport
EP2854834A4 (en) 2012-05-30 2016-05-18 Biostrategies LC PLANT LENSES AS CARRIER CORRESPONDING AGENTS IN 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
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
US9150841B2 (en) 2012-06-29 2015-10-06 Shire Human Genetic Therapies, Inc. Cells for producing recombinant iduronate-2-sulfatase
CA3140358A1 (en) 2012-07-31 2014-02-06 Bioasis Technologies, Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
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
HUE045144T2 (hu) 2012-08-29 2019-12-30 Hoffmann La Roche Véragygát-shuttle
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
ES2729997T3 (es) 2012-11-27 2019-11-07 Biomarin Pharm Inc Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas
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
AU2014243816B2 (en) 2013-03-13 2019-01-31 Bioasis Technologies Inc. Fragments of p97 and uses thereof
EP2970940B1 (en) 2013-03-14 2018-07-25 Translate Bio, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
EP3594240B1 (en) 2013-05-20 2023-12-06 F. Hoffmann-La Roche AG Anti-transferrin receptor antibodies and methods of use
WO2014194427A1 (en) 2013-06-06 2014-12-11 Angiochem Inc. Targeted iduronate-2-sulfatase fusion proteins
ES2762608T3 (es) 2013-06-07 2020-05-25 Genzyme Corp Marcador para trastornos de esfingomielinasa ácida y usos del mismo
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
WO2015033223A2 (en) 2013-09-03 2015-03-12 Novimmune S.A. 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
EP3088518A4 (en) 2013-12-25 2017-09-20 JCR Pharmaceuticals Co., Ltd. Novel anti-transferrin receptor antibody that passes through blood-brain barrier
CN105899540B (zh) 2014-01-03 2020-02-07 豪夫迈·罗氏有限公司 双特异性抗-半抗原/抗-血脑屏障受体的抗体、其复合物及它们作为血脑屏障穿梭物的应用
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
SI2970413T1 (sl) 2014-04-01 2018-10-30 Swedish Orphan Biovitrum Ab (Publ) Modificirana sulfamidaza in proizvodja le-te
AU2015252906C1 (en) 2014-05-01 2020-09-17 Bioasis Technologies Inc. p97-polynucleotide conjugates
EP3166626B1 (en) 2014-07-11 2021-04-28 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
EP3185889B1 (en) 2014-08-11 2020-07-15 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
DK3215528T3 (da) 2014-11-06 2019-10-07 Hoffmann La Roche Fc-regionvarianter med modificeret FcRn-binding og anvendelsesfremgangsmåder
US20180185495A1 (en) 2014-11-10 2018-07-05 Alexion Pharmaceuticals, Inc. Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof
EP3218411B1 (en) 2014-11-14 2022-01-12 Ossianix, Inc. Variable new antigen receptors (vnars) directed against transferrin receptor (tfr) and their use
MA40954A (fr) * 2014-11-14 2017-09-19 Shire Human Genetic Therapies Détermination de niveaux de glycosaminoglycane par spectrométrie de masse
WO2016081640A1 (en) 2014-11-19 2016-05-26 Genentech, Inc. Anti-transferrin receptor / anti-bace1 multispecific antibodies and methods of use
US10508151B2 (en) 2014-11-19 2019-12-17 Genentech, Inc. Anti-transferrin receptor 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
JP6785777B2 (ja) 2015-02-05 2020-11-18 アール.ピー. シェーラー テクノロジーズ エルエルシー 活性化ホルミルグリシン生成酵素ならびにその生成方法及び使用方法
EP3303403A4 (en) 2015-06-01 2019-01-16 The Hospital for Sick Children RELEASE OF STRUCTURELY DIFFERENT POLYPEPTIDES IN MAMMALIAN CELLS BY MEANS OF BACTERIAL TOXIN
EA039366B1 (ru) 2015-06-24 2022-01-19 Джей Си Ар ФАРМАСЬЮТИКАЛЗ КО., ЛТД. Антитело к рецептору трансферрина человека, проникающее через гематоэнцефалический барьер
CN107771156B (zh) 2015-06-24 2020-10-27 伯曼集团股份公司 线分拣机
HUE057952T2 (hu) * 2015-06-24 2022-06-28 Hoffmann La Roche Anti-transzferrin receptor antitestek testreszabott affinitással
WO2017003270A1 (ko) 2015-07-02 2017-01-05 주식회사 녹십자 헌터증후군 치료제 및 치료방법
KR20170004814A (ko) 2015-07-02 2017-01-11 주식회사 녹십자 헌터증후군 치료제
EP3355912A1 (en) 2015-10-01 2018-08-08 Swedish Orphan Biovitrum AB (Publ) Modified iduronate 2-sulfatase and production thereof
BR112018006278A2 (pt) 2015-10-01 2018-10-16 Swedish Orphan Biovitrum Ab Publ proteína lisossômica modificada e produção da mesma
US20180221459A1 (en) 2015-10-06 2018-08-09 LA BioMedical Research Institute at Harbor-UCLA Medical Center Enzyme replacement therapy for mucopolysaccharidosis iiid
WO2017062666A2 (en) 2015-10-06 2017-04-13 Patricia Dickson Enzyme replacement therapy for mucopolysaccharidosis iiid
DK3386534T3 (da) 2015-12-08 2020-11-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
GB201522243D0 (en) 2015-12-16 2016-01-27 Ucl Business Plc Treatment
PL3397270T3 (pl) 2015-12-30 2024-08-19 Green Cross Corporation Kompozycje do zastosowania w leczeniu zespołu huntera
AR107483A1 (es) 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
MX2018010196A (es) 2016-02-24 2018-11-19 Biomarin Pharm Inc Proteinas de fusion de enzimas lisosomales terapeuticas dirigidas, formulaciones asociadas y usos de las mismas.
US20190046637A1 (en) 2016-03-01 2019-02-14 Biostrategies LC Materials and methods for mitigating immune-sensitization
SG10201912761UA (en) 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
US10169252B2 (en) 2016-07-15 2019-01-01 International Business Machines Corporation Configuring functional capabilities of a computer system
EP3494216B1 (en) 2016-08-06 2025-10-15 Ossianix, Inc. In vivo methods for selecting peptides that cross the blood brain barrier, related compositions and methods of use
SG11201901495YA (en) 2016-08-25 2019-03-28 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
KR102497564B1 (ko) * 2016-12-26 2023-02-07 제이씨알 파마 가부시키가이샤 혈액뇌관문을 통과하는 신규한 항인간 트랜스페린 수용체 항체
AU2017386888B2 (en) 2016-12-28 2024-08-08 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
US10457717B2 (en) 2017-02-17 2019-10-29 Denali Therapeutics Inc. Engineered polypeptides
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
SG11201907422RA (en) * 2017-02-17 2019-09-27 Denali Therapeutics Inc Anti-tau antibodies and methods of use thereof
CA3053381A1 (en) 2017-02-17 2018-08-23 Denali Therapeutics Inc. Engineered polypeptides
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
AU2018295994B2 (en) 2017-07-07 2025-02-06 Hanmi Pharm. Co., Ltd. Novel therapeutic enzyme fusion protein and use thereof
JP2020528756A (ja) 2017-07-28 2020-10-01 ハンミ ファーマシューティカル カンパニー リミテッド イズロン酸−2−スルファターゼ結合体
EP3665194A4 (en) 2017-08-10 2021-07-07 Denali Therapeutics Inc. AFFINITY BASED METHODS OF USING TRANSFERRIN RECEPTOR BINDING PROTEINS
CN111278453A (zh) 2017-09-07 2020-06-12 Jcr制药股份有限公司 水性药物组合物
CR20200129A (es) 2017-10-02 2020-08-22 Denali Therapeutics Inc Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas
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
WO2019246071A1 (en) 2018-06-18 2019-12-26 Denali Therapeutics Inc. Fusion proteins comprising progranulin
WO2020037150A2 (en) 2018-08-16 2020-02-20 Denali Therapeutics Inc. Engineered bispecific proteins
JP2021534220A (ja) 2018-08-22 2021-12-09 デナリ セラピューティクス インコーポレイテッドDenali Therapeutics Inc. 抗her2ポリペプチド及びそれらの使用の方法
KR20200094577A (ko) 2019-01-30 2020-08-07 주식회사 모비스 인공신경망 시뮬레이터와 강화학습 제어기를 사용한 실시간 가속기 제어 시스템
CA3134538A1 (en) 2019-04-03 2020-10-08 Denali Therapeutics Inc. Formulations of protein molecules comprising iduronate 2-sulfatase
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

Also Published As

Publication number Publication date
MA50746A (fr) 2020-08-12
TW201925236A (zh) 2019-07-01
PE20210347A1 (es) 2021-02-25
EP3692070A1 (en) 2020-08-12
US20210198640A1 (en) 2021-07-01
SA520411662B1 (ar) 2023-12-03
CA3076369A1 (en) 2019-04-11
CL2020000810A1 (es) 2020-08-14
MY209029A (en) 2025-06-17
US20190249155A1 (en) 2019-08-15
AU2018345303B2 (en) 2025-09-18
IL273439B2 (en) 2025-10-01
IL273439A (en) 2020-05-31
MX2023009913A (es) 2024-05-21
SG11202002730XA (en) 2020-04-29
CN121780481A (zh) 2026-04-03
CR20200129A (es) 2020-08-22
KR20200058510A (ko) 2020-05-27
JP2023179600A (ja) 2023-12-19
BR112020005271A2 (pt) 2020-09-15
MX2024006720A (es) 2025-04-02
EP3904389A1 (en) 2021-11-03
US11866742B2 (en) 2024-01-09
JP2021500857A (ja) 2021-01-14
JP7854267B2 (ja) 2026-05-01
MX2020002918A (es) 2020-07-22
AU2018345303A1 (en) 2020-04-09
AU2025283436A1 (en) 2026-01-22
PH12020550510A1 (en) 2021-04-19
US20200277584A1 (en) 2020-09-03
CN111278859A (zh) 2020-06-12
US10870837B2 (en) 2020-12-22
IL273439B1 (en) 2025-06-01
WO2019070577A1 (en) 2019-04-11

Similar Documents

Publication Publication Date Title
MX2024006720A (es) Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas
EA201891277A1 (ru) Композиции и способы интернализации ферментов
CL2024003234A1 (es) Polipéptidos modificados y usos de los mismos.
MX2020006635A (es) Proteína de fusión enzimática terapéutica que tiene una nueva estructura y uso de la misma.
CL2020003255A1 (es) Proteínas de fusión que comprenden progranulina
PH12019550261A1 (en) Compositions and methods for internalizing enzymes
DOP2020000254A (es) Sistema de comunicación por satélite de banda ancha con enlaces de alimentador óptico
CY1122555T1 (el) Στοχευμενες θεραπευτικες πρωτεϊνες συντηξης λυσωσομικων ενζυμων και χρησεις αυτων
CR20170139A (es) Proteínas de fusión, bacterias recombinantes y métodos de uso de las bacterias recombinantes
AU2017241807A1 (en) Medium, methods, cells and secreted factors for stem cell culture and therapy
MX2018010196A (es) Proteinas de fusion de enzimas lisosomales terapeuticas dirigidas, formulaciones asociadas y usos de las mismas.
BR112019000693A2 (pt) composições de muc1- car e métodos para uso
EA201790178A1 (ru) Пептиды, несущие маннозо-6-фосфат, слитые с лизосомальным ферментом
DOP2016000220A (es) PROTEÍNAS DE FUSIÓN TATk-CDKL5, COMPOSICIONES, FORMULACIONES Y USO DE ESTAS.
ECSP22049014A (es) Variantes de progranulina
AR113339A1 (es) Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas (ert) y una región fc
EA202090882A1 (ru) Слитые белки, содержащие ферменты для заместительной ферментной терапии
CO2019007583A2 (es) Cabezal para encuentros de vigas
UA111147U (uk) Спосіб моделювання гострого асептичного панкреатиту у щурів
AR115565A1 (es) Proteínas de fusión que comprenden progranulina
杨扬 A Report on EC Translation of Cultural Factor in Information about Study Abroad
AR105635A1 (es) Heterómeros con actividad enzimática