PE20210347A1 - Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas - Google Patents

Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas

Info

Publication number
PE20210347A1
PE20210347A1 PE2020000415A PE2020000415A PE20210347A1 PE 20210347 A1 PE20210347 A1 PE 20210347A1 PE 2020000415 A PE2020000415 A PE 2020000415A PE 2020000415 A PE2020000415 A PE 2020000415A PE 20210347 A1 PE20210347 A1 PE 20210347A1
Authority
PE
Peru
Prior art keywords
fusion proteins
replacement therapy
enzyme replacement
proteins including
including enzymes
Prior art date
Application number
PE2020000415A
Other languages
English (en)
Inventor
Giuseppe Astarita
Jennifer A Getz
Anastasia Henry
Mihalis Kariolis
Cathal Mahon
Adam P Silverman
Ankita Srivastava
Julie Ullman
Junhua Wang
Joy Yu Zuchero
Mark S Dennis
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 PE20210347A1 publication Critical patent/PE20210347A1/es

Links

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 [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], 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 [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IGs], 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)
    • C12Y301/06—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
    • 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
    • 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
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Obesity (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)

Abstract

En la presente se proporcionan proteinas de fusion que comprenden una enzima de terapia de reemplazo de enzimas y una region Fc, asi como metodos de uso de dichas proteinas para tratar un trastorno de almacenamiento lisosomico. En la presente tambien se proporcionan metodos para transportar agentes a traves de la barrera hematoencefalica
PE2020000415A 2017-10-02 2018-10-01 Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas PE20210347A1 (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
PE20210347A1 true PE20210347A1 (es) 2021-02-25

Family

ID=64267892

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2020000415A PE20210347A1 (es) 2017-10-02 2018-10-01 Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas

Country Status (20)

Country Link
US (3) US20200277584A1 (es)
EP (2) EP3904389A1 (es)
JP (2) JP2021500857A (es)
KR (1) KR20200058510A (es)
CN (1) CN111278859A (es)
AU (1) AU2018345303A1 (es)
BR (1) BR112020005271A2 (es)
CA (1) CA3076369A1 (es)
CL (1) CL2020000810A1 (es)
CO (1) CO2020003128A2 (es)
CR (1) CR20200129A (es)
IL (1) IL273439A (es)
MA (1) MA50746A (es)
MX (1) MX2020002918A (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 (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL307373A (en) 2017-02-17 2023-11-01 Denali Therapeutics Inc Polypeptides that bind to a transgenic transferrin receptor
US20200277584A1 (en) 2017-10-02 2020-09-03 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
MX2021001711A (es) * 2018-08-16 2021-05-27 Denali Therapeutics Inc Proteínas biespecíficas modificadas.
US20220025065A1 (en) 2018-12-10 2022-01-27 Denali Therapeutics Inc. Lysosomal storage disorder biomarkers and methods of use thereof
JP2022526799A (ja) * 2019-04-03 2022-05-26 デナリ セラピューティクス インコーポレイテッド イズロン酸２－スルファターゼを含むタンパク質分子の製剤
CN110111326B (zh) * 2019-05-15 2021-01-15 西安科技大学 基于ert***的重建图像质量评价方法
CN114981297A (zh) 2019-12-23 2022-08-30 戴纳立制药公司 颗粒蛋白前体变体
MX2022008582A (es) 2020-01-13 2022-08-10 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
JP2023545707A (ja) 2020-10-14 2023-10-31 デナリ セラピューティクス インコーポレイテッド スルホグルコサミンスルホヒドロラーゼ酵素を含む融合タンパク質及びその方法
KR20220073454A (ko) * 2020-11-26 2022-06-03 한미약품 주식회사 신규한 뇌 표적 지속형 단백질 결합체 및 이의 제조방법
US20240148866A1 (en) * 2021-02-11 2024-05-09 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
AR128003A1 (es) 2021-12-17 2024-03-20 Denali Therapeutics Inc PROTEÍNAS DE FUSIÓN QUE COMPRENDEN ENZIMAS DE a-L-IDURONIDASA Y MÉTODOS PARA LAS MISMAS
WO2023198661A1 (en) * 2022-04-12 2023-10-19 F. Hoffmann-La Roche Ag Fusion proteins targeted to the central nervous system
WO2023224956A1 (en) * 2022-05-16 2023-11-23 Denali Therapeutics Inc. Transferrin receptor-binding domains and proteins comprising the same
WO2023244540A1 (en) 2022-06-13 2023-12-21 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

Family Cites Families (144)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
EP1436316B1 (en) 2001-04-30 2008-01-23 ZyStor Therapeutics , Inc. Subcellular targeting of therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 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
EP1463512B1 (en) 2002-01-11 2014-05-28 biOasis Technologies Inc. Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
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
HUE027210T2 (en) 2003-02-11 2016-10-28 Shire Human Genetic Therapies Cells expressing the enzyme producing sulfatase and C-formylglycine together and their uses
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
PT2256134E (pt) 2003-11-13 2014-03-05 Hanmi Science Co Ltd Fragmento fc de igg para um veículo de fármaco e método para a preparação do mesmo
EP1697520A2 (en) 2003-12-22 2006-09-06 Xencor, Inc. Fc polypeptides with novel fc ligand binding sites
WO2005073367A1 (en) 2004-01-30 2005-08-11 Zymenex A/S Production and purification of recombinant arylsulfatase a
WO2006007560A2 (en) 2004-07-01 2006-01-19 University Of Pennsylvania Targeted protein replacement for the treatment of lysosomal storage disorders
WO2006072620A1 (en) 2005-01-05 2006-07-13 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
US7341720B2 (en) 2005-04-06 2008-03-11 Genzyme Corporation Targeting of glycoprotein therapeutics
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
US8536319B2 (en) 2006-06-29 2013-09-17 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
AR070198A1 (es) 2008-01-18 2010-03-17 Biomarin Pharm Inc Elaboracion de enzimas lisosomales sulfatasa humanas activas y altamente fosforiladas y sus usos
HUE034850T2 (en) 2008-05-07 2018-03-28 Biomarin Pharm Inc Lysosomal targeting peptides and their use
SI3482767T1 (sl) 2009-08-28 2022-03-31 Icahn School Of Medicine At Mount Sinai Encimska nadomestna terapija s povečevanjem odmerkov za zdravljenje pomanjkanja kisle sfingomielinaze
WO2011039634A2 (en) 2009-09-29 2011-04-07 Universiteit Gent Hydrolysis of mannose-1-phospho-6-mannose linkage to phospho-6-mannose
WO2011044542A1 (en) 2009-10-09 2011-04-14 Armagen Technologies, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
LT2593131T (lt) 2010-06-25 2019-10-25 Shire Human Genetic Therapies Būdai ir kompozicijos, skirti iduronato 2-sulfatazės pristatymui į cns
NZ605865A (en) 2010-06-25 2015-07-31 Shire Human Genetic Therapies Cns delivery of therapeutic agents
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
JP6131190B2 (ja) 2010-09-29 2017-05-17 オキシレイン ユーケー リミテッド リン酸化ｎ−グリカンの脱マンノシル化
EP2628797B1 (en) 2010-10-15 2016-07-13 JCR Pharmaceuticals CO., LTD. Method for producing glycoprotein having mannose residue as non-reducing end of sugar chain
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
NZ724348A (en) 2010-11-30 2019-12-20 Genentech Inc Low affinity blood brain barrier receptor antibodies and uses therefor
WO2012085622A1 (en) 2010-12-22 2012-06-28 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
EA201892619A1 (ru) 2011-04-29 2019-04-30 Роше Гликарт Аг Иммуноконъюгаты, содержащие мутантные полипептиды интерлейкина-2
CA2834577A1 (en) 2011-05-23 2012-11-29 Phylogica Limited Method of determining, identifying or isolating cell-penetrating peptides
PL2714752T3 (pl) 2011-05-27 2018-04-30 Amicus Therapeutics, Inc. Sposoby sprzęgania peptydów nakierowanych na rekombinowane enzymy lizosomalne dla poprawy leczenia lizosomalnych chorób spichrzeniowych
US9845327B2 (en) 2011-06-22 2017-12-19 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
RU2014117291A (ru) 2011-10-12 2015-11-20 Синаджева Биофарма Корп. Рекомбинантный человеческий белок naglu и его применение
CN104136606A (zh) 2011-12-01 2014-11-05 安吉奥开米公司 靶向的艾杜糖-2-硫酸酯酶化合物
US20150037311A1 (en) 2011-12-01 2015-02-05 Angiochem Inc. Targeted lysosomal enzyme compounds
CA2857647C (en) 2011-12-02 2022-04-19 Armagen Technologies, 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
EA201491578A1 (ru) 2012-03-30 2015-04-30 Шир Хьюман Дженетик Терапис, Инк. Подкожное введение идуронат-2-сульфатазы
JP5855239B2 (ja) 2012-04-23 2016-02-09 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
JP6279466B2 (ja) 2012-04-27 2018-02-14 Ｊｃｒファーマ株式会社 新規な発現ベクター
EA201491920A1 (ru) 2012-05-21 2016-03-31 Дженентек, Инк. Способы повышения безопасности транспорта через гематоэнцефалический барьер
WO2013181454A1 (en) 2012-05-30 2013-12-05 Biostrategies LC Plant lectins as carriers of associated drug substances into animal and human cells
SG11201407017WA (en) 2012-06-04 2014-12-30 Irm Llc 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
US20140004097A1 (en) 2012-06-29 2014-01-02 Shire Human Genetic Therapies, Inc. Method of producing recombinant iduronate-2-sulfatase
KR101380740B1 (ko) 2012-06-29 2014-04-11 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 이듀로네이트-2-설파타제의 정제
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
KR20190121874A (ko) 2012-08-29 2019-10-28 에프. 호프만-라 로슈 아게 혈액 뇌 장벽 셔틀
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
BR112015012152B1 (pt) 2012-11-27 2023-04-25 Biomarin Pharmaceutical Inc Proteína de fusão terapêutica direcionada que compreende uma enzima lisossomal, ácido nucleico, composição farmacêutica, método para produzir a dita proteína e usos da proteína para tratar uma doença de armazenamento lisossomal e mucopolissacaridose tipo iiib
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
CN105027138B (zh) 2012-12-27 2018-01-26 施耐德电气It公司 可视化气流的***和方法
WO2014160438A1 (en) 2013-03-13 2014-10-02 Bioasis Technologies Inc. Fragments of p97 and uses thereof
EP3431592A1 (en) 2013-03-14 2019-01-23 Translate Bio, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
KR102293064B1 (ko) 2013-05-20 2021-08-23 제넨테크, 인크. 항-트랜스페린 수용체 항체 및 사용 방법
WO2014194427A1 (en) 2013-06-06 2014-12-11 Angiochem Inc. Targeted iduronate-2-sulfatase fusion proteins
EP3584580A1 (en) 2013-06-07 2019-12-25 Genzyme Corporation Recombinant human acid sphingomyelinase for use in treating human asmd
WO2015009052A1 (ko) * 2013-07-16 2015-01-22 일동제약 주식회사 하이브리드 면역글로불린 fc와 효소의 융합단백질
US10906981B2 (en) 2013-07-19 2021-02-02 The Regents Of The University Of California Compositions and methods related to structures that cross the blood brain barrier
US20150023956A1 (en) 2013-07-22 2015-01-22 Armagen Technologies, Inc. Methods and compositions for increasing enzyme activity in the cns
WO2015031673A2 (en) * 2013-08-28 2015-03-05 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
JP6616189B2 (ja) 2013-12-25 2019-12-04 Ｊｃｒファーマ株式会社 血液脳関門を通過する新規抗トランスフェリン受容体抗体
BR112016013849A2 (pt) 2014-01-03 2017-10-10 Hoffmann La Roche conjugados de anticorpos biespecíficos antihapteno/antirreceptores da barreira hematoencefálica, seus usos, e formulação farmacêutica
JP6557664B2 (ja) 2014-01-06 2019-08-07 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 一価の血液脳関門シャトルモジュール
WO2015126729A1 (en) 2014-02-19 2015-08-27 Bioasis Technologies, Inc. P97-ids fusion proteins
LT2970413T (lt) 2014-04-01 2018-10-10 Swedish Orphan Biovitrum Ab (Publ) Modifikuota sulfamidazė ir jos gamyba
EP3137610B1 (en) 2014-05-01 2019-03-06 Bioasis Technologies, Inc. P97-polynucleotide conjugates
WO2016007909A2 (en) 2014-07-11 2016-01-14 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
MX2017001898A (es) 2014-08-11 2017-04-11 Shire Human Genetic Therapies Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales.
MX2017005148A (es) 2014-11-06 2017-08-08 Hoffmann La Roche Variantes de region fc con union del receptor fc neonatal (fcrn) modificado y metodos de uso.
WO2016077356A2 (en) 2014-11-10 2016-05-19 Synageva Biopharma Corp. Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof
CA2967830A1 (en) 2014-11-14 2016-05-19 Ossianix, Inc. Tfr selective binding compounds and related methods
MA40954A (fr) * 2014-11-14 2017-09-19 Shire Human Genetic Therapies Détermination de niveaux de glycosaminoglycane par spectrométrie de masse
US10508151B2 (en) 2014-11-19 2019-12-17 Genentech, Inc. Anti-transferrin receptor antibodies and methods of use
US11008403B2 (en) 2014-11-19 2021-05-18 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
CN107250372B (zh) 2015-02-05 2022-11-01 R.P.谢勒技术有限责任公司 活化的甲酰甘氨酸生成酶及其生成和使用方法
EP3303403A4 (en) 2015-06-01 2019-01-16 The Hospital for Sick Children ADMINISTRATION OF POLYPEPTIDE CARGO OF VARIOUS STRUCTURES IN MAMMALIAN CELLS BY BACTERIAL TOXIN
BR112017027965A2 (pt) 2015-06-24 2018-08-28 Japan Chem Res anticorpo receptor, proteína de fusão, fragmento de dna, vetor de expressão, célula, complexo de anticorpo receptor, usos de um anticorpo receptor e de uma proteína de fusão, e, métodos para tratamento de um distúrbio e de uma doença.
PE20221007A1 (es) * 2015-06-24 2022-06-15 Hoffmann La Roche Anticuerpos anti-receptor de transferrina con afinidad disenada
ES2849248T3 (es) 2015-06-24 2021-08-17 Beumer Group As Clasificador de línea
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
AU2016329507A1 (en) 2015-10-01 2018-04-19 Swedish Orphan Biovitrum Ab (Publ) Modified iduronate 2-sulfatase and production thereof
WO2017055570A1 (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
JP7065770B2 (ja) 2015-12-08 2022-05-12 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 酵素の内部移行のための組成物および方法
GB201522243D0 (en) 2015-12-16 2016-01-27 Ucl Business Plc Treatment
DK3397270T3 (da) 2015-12-30 2024-05-06 Green Cross Corp Sammensætninger til anvendelse i behandlingen af hunters syndrom
AR107483A1 (es) 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
RU2751235C2 (ru) 2016-02-24 2021-07-12 Байомарин Фармасьютикал Инк. Терапевтические слитые белки направленного действия на основе лизосомных ферментов, соответствующие составы и их применения
WO2017151791A1 (en) 2016-03-01 2017-09-08 Biostrategies LC Materials and methods for mitigating immune-sensitization
CA3019427A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania 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
AU2017311040B2 (en) 2016-08-06 2023-08-31 Ossianix, Inc. In vivo methods for selecting peptides that cross the blood brain barrier, related compositions and methods of use
MX2019002252A (es) 2016-08-25 2019-07-04 Japan Chem Res Metodo para producir una proteina de fusion de anticuerpo.
CN110312532A (zh) * 2016-12-19 2019-10-08 韩美药品株式会社 脑靶向长效蛋白质缀合物
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
BR112019013202A2 (pt) 2016-12-28 2019-12-10 Japan Chem Res formulação liofilizada
GB201701968D0 (en) 2017-02-07 2017-03-22 Univ Manchester Gene therapy
KR102637590B1 (ko) 2017-02-17 2024-02-15 데날리 테라퓨틱스 인크. 트란스페린 수용체 유전자삽입 모델
US10457717B2 (en) 2017-02-17 2019-10-29 Denali Therapeutics Inc. Engineered polypeptides
CA3053381A1 (en) 2017-02-17 2018-08-23 Denali Therapeutics Inc. Engineered polypeptides
IL307373A (en) 2017-02-17 2023-11-01 Denali Therapeutics Inc Polypeptides that bind to a transgenic transferrin receptor
CA3053379A1 (en) * 2017-02-17 2018-08-23 Denali Therapeutics Inc. Anti-tau antibodies and methods of use thereof
WO2018155443A1 (ja) 2017-02-21 2018-08-30 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
IL271837B2 (en) 2017-07-07 2024-01-01 Hanmi Pharm Ind Co Ltd A new medical fusion protein with an enzyme and its uses
US20200230253A1 (en) 2017-07-28 2020-07-23 HANMl PHARM. CO., LTD. Conjugate of iduronate-2-sulfatase
SI3665192T1 (sl) 2017-08-10 2023-11-30 Denali Therapeutics Inc. Inženiring polipeptidov, ki se vežejo na receptorje za transferine
CN111278453A (zh) 2017-09-07 2020-06-12 Jcr制药股份有限公司 水性药物组合物
US20200277584A1 (en) 2017-10-02 2020-09-03 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
BR112020013921A2 (pt) 2018-01-10 2020-12-01 Denali Therapeutics Inc. polipeptídeos de ligação ao receptor de transferrina e usos destes
BR112020025306A2 (pt) 2018-06-18 2021-03-09 Denali Therapeutics Inc. Proteínas de fusão compreendendo progranulina
MX2021001711A (es) 2018-08-16 2021-05-27 Denali Therapeutics Inc Proteínas biespecíficas modificadas.
WO2020041604A1 (en) 2018-08-22 2020-02-27 Denali Therapeutics Inc. Anti-her2 polypeptides and methods of use thereof
JP2022526799A (ja) 2019-04-03 2022-05-26 デナリ セラピューティクス インコーポレイテッド イズロン酸２－スルファターゼを含むタンパク質分子の製剤
CN114981297A (zh) 2019-12-23 2022-08-30 戴纳立制药公司 颗粒蛋白前体变体
MX2022008582A (es) 2020-01-13 2022-08-10 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
JP2023545707A (ja) 2020-10-14 2023-10-31 デナリ セラピューティクス インコーポレイテッド スルホグルコサミンスルホヒドロラーゼ酵素を含む融合タンパク質及びその方法

Also Published As

Publication number Publication date
EP3692070A1 (en) 2020-08-12
MA50746A (fr) 2020-08-12
CA3076369A1 (en) 2019-04-11
IL273439A (en) 2020-05-31
US20200277584A1 (en) 2020-09-03
JP2023179600A (ja) 2023-12-19
MX2020002918A (es) 2020-07-22
CN111278859A (zh) 2020-06-12
US20190249155A1 (en) 2019-08-15
PH12020550510A1 (en) 2021-04-19
KR20200058510A (ko) 2020-05-27
EP3904389A1 (en) 2021-11-03
JP2021500857A (ja) 2021-01-14
CO2020003128A2 (es) 2020-06-19
US10870837B2 (en) 2020-12-22
AU2018345303A1 (en) 2020-04-09
WO2019070577A1 (en) 2019-04-11
TW201925236A (zh) 2019-07-01
BR112020005271A2 (pt) 2020-09-15
SG11202002730XA (en) 2020-04-29
US11866742B2 (en) 2024-01-09
SA520411662B1 (ar) 2023-12-03
CL2020000810A1 (es) 2020-08-14
US20210198640A1 (en) 2021-07-01
CR20200129A (es) 2020-08-22

Similar Documents

Publication Publication Date Title
PE20210347A1 (es) Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas
CO2019014682A2 (es) Composiciones y métodos para enzimas de internalización
EA201891277A1 (ru) Композиции и способы интернализации ферментов
CL2019000261A1 (es) Polipéptidos modificados y usos de los mismos.
AU2017241807A1 (en) Medium, methods, cells and secreted factors for stem cell culture and therapy
CR20170139A (es) Proteínas de fusión, bacterias recombinantes y métodos de uso de las bacterias recombinantes
WO2012139028A3 (en) Anti-viral combination therapy
MX2019009191A (es) Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas.
CL2015002472A1 (es) Como inhibidores de mutant idh-yl-oxazolidin-2-onas 3-pirimidin-4.
CY1124761T1 (el) Ρυθμιστες της δραστικοτητας του συμπληρωματος
BR112017016897A2 (pt) cepas fúngicas e métodos de uso
EP2507267A4 (en) COMPOSITIONS AND METHODS FOR INCREASING THE SERUM DURABILITY OF FC FUSION PROTEINS
MX2019004487A (es) Metodos y composiciones para el tratamiento de la enfermedad de fabry.
MX2018010196A (es) Proteinas de fusion de enzimas lisosomales terapeuticas dirigidas, formulaciones asociadas y usos de las mismas.
EA201790178A1 (ru) Пептиды, несущие маннозо-6-фосфат, слитые с лизосомальным ферментом
DOP2016000220A (es) PROTEÍNAS DE FUSIÓN TATk-CDKL5, COMPOSICIONES, FORMULACIONES Y USO DE ESTAS.
EA202091239A1 (ru) Слитый белок на основе терапевтического фермента, имеющий новую структуру, и его применение
CR20190480A (es) Uso de antagonistas de klk5 para el tratamiento de una enfermedad
CO2022008648A2 (es) Variantes de progranulina
BR112021014944A2 (pt) Toxinas apxia, apxiia e apxiiia inativas
MX2020008408A (es) Imagenologia y terapia dirigidas por la granzima b.
SG11202000115SA (en) Novel therapeutic enzyme fusion protein and use thereof
AR113339A1 (es) Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas (ert) y una región fc
ECSP22049014A (es) Variantes de progranulina
EA202090882A1 (ru) Слитые белки, содержащие ферменты для заместительной ферментной терапии