SG11202002730XA - Fusion proteins comprising enzyme replacement therapy enzymes - Google Patents

Fusion proteins comprising enzyme replacement therapy enzymes

Info

Publication number
SG11202002730XA
SG11202002730XA SG11202002730XA SG11202002730XA SG11202002730XA SG 11202002730X A SG11202002730X A SG 11202002730XA SG 11202002730X A SG11202002730X A SG 11202002730XA SG 11202002730X A SG11202002730X A SG 11202002730XA SG 11202002730X A SG11202002730X A SG 11202002730XA
Authority
SG
Singapore
Prior art keywords
fusion proteins
replacement therapy
enzyme replacement
enzymes
therapy enzymes
Prior art date
Application number
SG11202002730XA
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 SG11202002730XA publication Critical patent/SG11202002730XA/en

Links

Classifications

    • 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)
    • 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
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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)
  • Zoology (AREA)
  • Wood Science & Technology (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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
SG11202002730XA 2017-10-02 2018-10-01 Fusion proteins comprising enzyme replacement therapy enzymes SG11202002730XA (en)

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
SG11202002730XA true SG11202002730XA (en) 2020-04-29

Family

ID=64267892

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202002730XA SG11202002730XA (en) 2017-10-02 2018-10-01 Fusion proteins comprising enzyme replacement therapy enzymes

Country Status (20)

Country Link
US (3) US20200277584A1 (ko)
EP (2) EP3692070A1 (ko)
JP (2) JP2021500857A (ko)
KR (1) KR20200058510A (ko)
CN (1) CN111278859A (ko)
AU (1) AU2018345303A1 (ko)
BR (1) BR112020005271A2 (ko)
CA (1) CA3076369A1 (ko)
CL (1) CL2020000810A1 (ko)
CO (1) CO2020003128A2 (ko)
CR (1) CR20200129A (ko)
IL (1) IL273439A (ko)
MA (1) MA50746A (ko)
MX (2) MX2020002918A (ko)
PE (1) PE20210347A1 (ko)
PH (1) PH12020550510A1 (ko)
SA (1) SA520411662B1 (ko)
SG (1) SG11202002730XA (ko)
TW (1) TW201925236A (ko)
WO (1) WO2019070577A1 (ko)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2932759T3 (es) 2017-02-17 2023-01-25 Denali Therapeutics Inc Polipéptidos de unión al receptor de transferrina diseñados
KR20200058510A (ko) 2017-10-02 2020-05-27 데날리 테라퓨틱스 인크. 효소 대체 요법 효소를 포함하는 융합 단백질
MA53616A (fr) * 2018-08-16 2022-05-04 Denali Therapeutics Inc Protéines bispécifiques modifiées
WO2020123511A2 (en) 2018-12-10 2020-06-18 Denali Therapeutics Inc. Lysosomal storage disorder biomarkers and methods of use thereof
KR20220004651A (ko) * 2019-04-03 2022-01-11 데날리 테라퓨틱스 인크. 이두로네이트 2-설파타제를 포함하는 단백질 분자의 제제
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
AU2020411480B2 (en) 2019-12-23 2023-12-21 Denali Therapeutics Inc. Progranulin variants
MX2022008582A (es) 2020-01-13 2022-08-10 Denali Therapeutics Inc Anticuerpos anti-trem2 y metodos para utilizarlos.
US20230092681A1 (en) * 2020-02-07 2023-03-23 Denali Therapeutics Inc. Methods for the treatment of hunter syndrome
AU2021362209A1 (en) 2020-10-14 2023-05-18 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
CN116997364A (zh) * 2021-02-11 2023-11-03 戴纳立制药公司 抗转铁蛋白受体融合蛋白及其使用方法
WO2023004156A1 (en) 2021-07-23 2023-01-26 Denali Therapeutics Inc. Methods for the treatment of hunter syndrome
CN118401660A (zh) 2021-12-17 2024-07-26 戴纳立制药公司 包含α-L-艾杜糖醛酸酶的融合蛋白和方法
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
WO2024220986A2 (en) 2023-04-21 2024-10-24 Denali Therapeutics Inc. Methods of increasing sialic acid levels in recombinant glycosylated proteins

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
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7396811B2 (en) 2001-04-30 2008-07-08 Zystor Therapeutics, Inc. Subcellular targeting of therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
EP2399586A1 (en) 2002-01-11 2011-12-28 Jefferies, Dr., Wilfred 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
ATE534730T1 (de) 2003-02-11 2011-12-15 Shire Human Genetic Therapies Diagnose und behandlung von multiplen sulfatase- erkrankungen und anderen mit hilfe eines formylglycin bildenden enzyms
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
BRPI0406606A (pt) 2003-11-13 2005-12-06 Hanmi Pharm Ind Co Ltd Composição farmacêutica compreendendo uma região fc de imunoglobulina como um veìculo
US20050249723A1 (en) 2003-12-22 2005-11-10 Xencor, Inc. Fc polypeptides with novel Fc ligand binding sites
ES2687671T3 (es) 2004-01-30 2018-10-26 Shire Pharmaceuticals Ireland Limited Arilsulfatasa A recombinante para reducir los niveles de galatosil sulfátidos en un sujeto
WO2006007560A2 (en) 2004-07-01 2006-01-19 University Of Pennsylvania Targeted protein replacement for the treatment of lysosomal storage disorders
RS50752B (sr) 2005-01-05 2010-08-31 F-Star Biotechnologische Forschungs-Und Entwicklungsges M.B.H. Domeni sintetskih imunoglobulina sa svojstvima vezivanja, konstruisani u regionima molekula, koji se razlikuju od regiona, koji određuju komplementarnost
PL1877099T3 (pl) 2005-04-06 2013-02-28 Genzyme Corp Terapeutyczne koniugaty zawierające enzym lizosomalny, kwas polisialowy i grupę kierującą
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
WO2007091250A2 (en) 2006-02-06 2007-08-16 The Medical Research And Infrastructure Fund Of The Tel-Aviv Sourasky Medical Center Enzyme replacement therapy for treating lysosomal storage diseases
US8748567B2 (en) 2006-05-22 2014-06-10 Children's Medical Center Corporation 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
PL2245145T3 (pl) 2008-01-18 2017-07-31 Biomarin Pharmaceutical Inc. Wytwarzanie aktywnych wysoce fosforylowanych ludzkich enzymów sulfatazy lizosomalnej i ich zastosowania
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
CN105999238A (zh) 2009-08-28 2016-10-12 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法
KR101824743B1 (ko) 2009-09-29 2018-02-05 유니버시테이트 젠트 만노스-1-포스포-6-만노스 결합의 포스포-6-만노스로의 가수분해
EP4273164A3 (en) 2009-10-09 2024-01-24 Armagen, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
KR102283671B1 (ko) 2010-06-25 2021-07-30 샤이어 휴먼 지네틱 테라피즈 인크. 치료제들의 cns 전달
KR102151889B1 (ko) 2010-06-25 2020-09-04 샤이어 휴먼 지네틱 테라피즈 인크. 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들
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
US9689015B2 (en) 2010-09-29 2017-06-27 Oxyrane Uk Limited De-mannosylation of phosphorylated N-glycans
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
US20130244231A1 (en) 2010-11-08 2013-09-19 Jcr Pharmaceuticals Co., Ltd. Novel expression vector
AU2011336716B2 (en) 2010-11-30 2016-01-21 Genentech, Inc. Low affinity blood brain barrier receptor antibodies and uses therefor
JP6023073B2 (ja) 2010-12-22 2016-11-09 フォンダッツィオーネ・テレソン ムコ多糖症におけるｃｎｓ病変を処置するための治療戦略
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
AU2012260434A1 (en) 2011-05-23 2013-10-31 Phylogica Limited Method of determining, identifying or isolating cell-penetrating peptides
SI2714752T1 (en) 2011-05-27 2018-03-30 Amicus Therapeutics, Inc. Methods for combining target peptides for recombinant lysosomal enzymes for improved treatment of lysosomal storage diseases
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
US8722019B2 (en) 2011-08-05 2014-05-13 Bioasis Technologies, Inc. P97 fragments with transfer activity
EP2766477B1 (en) 2011-10-12 2018-09-26 Alexion Pharmaceuticals, Inc. Recombinant human naglu protein and uses thereof
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
BR112014013161A2 (pt) 2011-12-01 2019-09-24 Angiochem Inc compostos com enzima lisossomal alvo
ES2983576T3 (es) 2011-12-02 2024-10-23 Armagen Inc Métodos y composiciones para aumentar la actividad arilsulfatasa en el SNC
WO2013096912A2 (en) 2011-12-23 2013-06-27 Shire Human Genetic Therapies, Inc. Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase
WO2013148277A1 (en) 2012-03-30 2013-10-03 Shire Human Genetic Therapies, Inc. Subcutaneous administration of iduronate- 2-sulfatase
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
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
CN104520325A (zh) * 2012-05-21 2015-04-15 霍夫曼-拉罗奇有限公司 用于提高血脑屏障转运的安全性的方法
US20130323221A1 (en) 2012-05-30 2013-12-05 Biostrategies LC Plant lectins as carriers of associated drug substances into animal and human cells
US20150104468A1 (en) 2012-06-04 2015-04-16 Irm Llc Site-specific labeling methods and molecules produced thereby
US20150147310A1 (en) 2012-06-15 2015-05-28 Angiochem Inc. Targeted enzyme compounds and uses thereof
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
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
SG11201500583PA (en) 2012-08-29 2015-04-29 Hoffmann La Roche Blood brain barrier 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
KR102385392B1 (ko) 2012-11-27 2022-04-11 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들
WO2014082184A1 (en) 2012-11-30 2014-06-05 Angiochem Inc. Targeted iduronate-2-sulfatase compounds
EP2939176A4 (en) 2012-12-27 2016-07-27 Schneider Electric It Corp SYSTEMS AND METHOD FOR VISUALIZING AN AIRFLOW
CN105263958B (zh) 2013-03-13 2019-09-27 比奥阿赛斯技术有限公司 p97片段及其应用
WO2014152940A1 (en) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
MX2015015970A (es) 2013-05-20 2016-09-08 Genentech Inc Anticuerpos receptores de antitransferina y metodos de uso.
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
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
CA2918579C (en) 2013-07-22 2023-01-03 Armagen Technologies, Inc. Fusion antibody for delivering n-sulfoglucosamine sulfohydrolase (sgsh) across the blood brain barrier
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
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
BR112016015589A2 (pt) 2014-01-06 2017-10-31 Hoffmann La Roche módulos de trânsito monovalentes para a barreira hematoencefálica
JP6605482B2 (ja) 2014-02-19 2019-11-13 バイオアシス テクノロジーズ インコーポレイテッド Ｐ９７−ｉｄｓ融合タンパク質
MA38816B1 (fr) 2014-04-01 2018-10-31 Swedish Orphan Biovitrum Ab Publ Sulfamidase modifiée et sa production
CA2943890A1 (en) 2014-05-01 2015-11-05 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
US10722559B2 (en) 2014-08-11 2020-07-28 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
RU2714116C2 (ru) 2014-11-06 2020-02-11 Ф. Хоффманн-Ля Рош Аг ВАРИАНТЫ Fc-ОБЛАСТИ С МОДИФИЦИРОВАННЫМ СВЯЗЫВАНИЕМ FcRn И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
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
WO2016077840A2 (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
CN107001473B (zh) 2014-11-19 2021-07-09 豪夫迈·罗氏有限公司 抗-运铁蛋白受体抗体及使用方法
JP6993228B2 (ja) 2014-11-19 2022-03-03 ジェネンテック， インコーポレイテッド 抗トランスフェリン受容体／抗ｂａｃｅ１多重特異性抗体および使用方法
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
WO2016191869A1 (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
PE20180602A1 (es) * 2015-06-24 2018-04-09 Hoffmann La Roche Anticuerpos anti-receptor de transferrina con afinidad disenada
PL3313757T3 (pl) 2015-06-24 2021-05-31 Beumer Group A/S Sortownik liniowy
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.
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
WO2017055570A1 (en) 2015-10-01 2017-04-06 Swedish Orphan Biovitrum Ab (Publ) Modified lysosomal protein and production thereof
US20180264089A1 (en) 2015-10-01 2018-09-20 Swedish Orphan Biovitrum Ab (Publ) Modified iduronate 2-sulfatase 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
DK3782639T3 (da) 2015-12-08 2022-08-29 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
GB201522243D0 (en) 2015-12-16 2016-01-27 Ucl Business Plc Treatment
KR102272399B1 (ko) 2015-12-30 2021-07-05 주식회사 녹십자 헌터증후군을 치료하기 위한 방법 및 조성물
AR107483A1 (es) 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
CN109069595A (zh) 2016-02-24 2018-12-21 生物马林药物股份有限公司 标靶医疗性溶酶体酵素融合蛋白质、其相关调配物与用途
US20190046637A1 (en) 2016-03-01 2019-02-14 Biostrategies LC Materials and methods for mitigating immune-sensitization
EP3452104A1 (en) 2016-04-15 2019-03-13 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
JP6993814B2 (ja) 2016-08-25 2022-01-14 Ｊｃｒファーマ株式会社 抗体融合蛋白質の製造方法
EP3556399A1 (en) 2016-12-19 2019-10-23 Hanmi Pharm. Co., Ltd. Brain targeting long-acting protein conjugate
NZ752703A (en) * 2016-12-26 2022-09-30 Japan Chem Res Novel anti-human transferrin receptor antibody capable of penetrating blood-brain barrier
JP7042220B2 (ja) 2016-12-28 2022-03-25 Ｊｃｒファーマ株式会社 凍結乾燥製剤
GB201701968D0 (en) 2017-02-07 2017-03-22 Univ Manchester Gene therapy
CA3053379A1 (en) * 2017-02-17 2018-08-23 Denali Therapeutics Inc. Anti-tau antibodies and methods of use thereof
US10457717B2 (en) 2017-02-17 2019-10-29 Denali Therapeutics Inc. Engineered polypeptides
ES2932759T3 (es) 2017-02-17 2023-01-25 Denali Therapeutics Inc Polipéptidos de unión al receptor de transferrina diseñados
JP2020510418A (ja) 2017-02-17 2020-04-09 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． トランスフェリン受容体トランスジェニックモデル
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
TWI832818B (zh) 2017-07-07 2024-02-21 南韓商韓美藥品股份有限公司 新穎的治療性酵素融合蛋白及其用途
WO2019022563A2 (ko) 2017-07-28 2019-01-31 한미약품 주식회사 이두로네이트 2-설파타제 결합체
RS64584B1 (sr) 2017-08-10 2023-10-31 Denali Therapeutics Inc Konstruisani polipeptidi koji se vezuju za transferinski receptor
WO2019049967A1 (ja) 2017-09-07 2019-03-14 Ｊｃｒファーマ株式会社 水性医薬組成物
KR20200058510A (ko) 2017-10-02 2020-05-27 데날리 테라퓨틱스 인크. 효소 대체 요법 효소를 포함하는 융합 단백질
WO2019094608A1 (en) 2017-11-08 2019-05-16 Denali Therapeutics Inc. Anti-bace1 antibodies and methods of use thereof
SG11202006420TA (en) 2018-01-10 2020-08-28 Denali Therapeutics Inc Transferrin receptor-binding polypeptides and uses thereof
KR20210027377A (ko) 2018-06-18 2021-03-10 데날리 테라퓨틱스 인크. 프로그래뉼린을 포함하는 융합 단백질
MA53616A (fr) 2018-08-16 2022-05-04 Denali Therapeutics Inc Protéines bispécifiques modifiées
AU2019326545A1 (en) 2018-08-22 2021-03-11 Denali Therapeutics Inc. Anti-HER2 polypeptides and methods of use thereof
KR20220004651A (ko) 2019-04-03 2022-01-11 데날리 테라퓨틱스 인크. 이두로네이트 2-설파타제를 포함하는 단백질 분자의 제제
AU2020411480B2 (en) 2019-12-23 2023-12-21 Denali Therapeutics Inc. Progranulin variants
MX2022008582A (es) 2020-01-13 2022-08-10 Denali Therapeutics Inc Anticuerpos anti-trem2 y metodos para utilizarlos.
US20230092681A1 (en) 2020-02-07 2023-03-23 Denali Therapeutics Inc. Methods for the treatment of hunter syndrome
AU2021362209A1 (en) 2020-10-14 2023-05-18 Denali Therapeutics Inc. Fusion proteins comprising sulfoglucosamine sulfohydrolase enzymes and methods thereof

Also Published As

Publication number Publication date
CR20200129A (es) 2020-08-22
US10870837B2 (en) 2020-12-22
EP3904389A1 (en) 2021-11-03
IL273439A (en) 2020-05-31
SA520411662B1 (ar) 2023-12-03
BR112020005271A2 (pt) 2020-09-15
AU2018345303A1 (en) 2020-04-09
TW201925236A (zh) 2019-07-01
US20200277584A1 (en) 2020-09-03
MX2020002918A (es) 2020-07-22
CN111278859A (zh) 2020-06-12
MX2023009913A (es) 2024-05-21
CA3076369A1 (en) 2019-04-11
CO2020003128A2 (es) 2020-06-19
JP2023179600A (ja) 2023-12-19
US20210198640A1 (en) 2021-07-01
CL2020000810A1 (es) 2020-08-14
MA50746A (fr) 2020-08-12
US20190249155A1 (en) 2019-08-15
EP3692070A1 (en) 2020-08-12
WO2019070577A1 (en) 2019-04-11
JP2021500857A (ja) 2021-01-14
US11866742B2 (en) 2024-01-09
PE20210347A1 (es) 2021-02-25
PH12020550510A1 (en) 2021-04-19
KR20200058510A (ko) 2020-05-27

Similar Documents

Publication Publication Date Title
IL273439A (en) Conjugated proteins involving enzyme replacement in enzyme therapy
EP3621443A4 (en) LIPASE ENZYMS
IL251194A0 (en) Fusion proteins, recombinant bacteria, and methods for using recombinant bacteria
EP3454832A4 (en) GDF15 FUSION PROTEINS AND USES THEREOF
EP3287470A4 (en) Novel recombinant bi-functional fusion protein and preparation and application therefor
IL279541A (en) A novel binding protein specific for CD137 and PD-L1
EP3225690A4 (en) Method for preparing bacterial polysaccharide-modified recombinant fusion protein and use thereof
IL275248A (en) Fusion protein of a therapeutic enzyme with a new structure and its use
EP3409771A4 (en) COUPLING ASSEMBLY FOR THERAPEUTIC ENZYMES
EP3757218A4 (en) MERGER PROTEIN
EP3717652A4 (en) GENE THERAPY FOR MUCOPOLY SACCHARIDOSIS IIIB
ZA202000595B (en) Novel therapeutic enzyme fusion protein and use thereof
IL290675A (en) Medical protein concentrates
AU2018285351A8 (en) Enzymatic replacement therapy and antisense therapy for Pompe disease
ZA201805648B (en) Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
EP3512871A4 (en) LONG UNIFORM RECOMBINANT PROTEIN FIBERS
ZA202003845B (en) Fusion proteins
EP3722305A4 (en) HM-3 FUSION PROTEIN AND USES THEREOF
EP3572507A4 (en) EXPRESSION METHOD FOR RECOMBINANT HAEMOCOAGULASE ACUTUS PROTEIN
IL271813A (en) Recombinant uricase enzyme
EP3668551A4 (en) APOM FC FUSION PROTEINS AND USES THEREOF
SG11202101904UA (en) Spd-1 variant - fc fusion proteins
ZA202102533B (en) Fusion protein
EP3362468A4 (en) Cleavable fusion tag for protein overexpression and purification
IL259868A (en) Enzyme replacement therapy and antisense therapy for Pompe disease