DK3782639T3 - Sammensætninger og fremgangsmåder til internalisering af enzymer - Google Patents

Sammensætninger og fremgangsmåder til internalisering af enzymer Download PDF

Info

Publication number
DK3782639T3
DK3782639T3 DK20197224.7T DK20197224T DK3782639T3 DK 3782639 T3 DK3782639 T3 DK 3782639T3 DK 20197224 T DK20197224 T DK 20197224T DK 3782639 T3 DK3782639 T3 DK 3782639T3
Authority
DK
Denmark
Prior art keywords
internalizing
enzymes
compositions
methods
internalizing enzymes
Prior art date
Application number
DK20197224.7T
Other languages
English (en)
Inventor
Andrew Baik
Katherine Cygnar
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Application granted granted Critical
Publication of DK3782639T3 publication Critical patent/DK3782639T3/da

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)
    • C12N9/24—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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6811—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
    • A61K47/6815—Enzymes
    • 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
    • A61K47/6835—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
    • 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
    • A61K47/6835—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6875—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody being a hybrid immunoglobulin
    • A61K47/6879—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody being a hybrid immunoglobulin the immunoglobulin having two or more different antigen-binding sites, e.g. bispecific or multispecific immunoglobulin
    • 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
    • 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
    • A61P3/06—Antihyperlipidemics
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07K16/2896—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against molecules with a "CD"-designation, not provided for elsewhere
    • 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
    • C12N9/2445—Beta-glucosidase (3.2.1.21)
    • 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)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • 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)
    • 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)
    • C12Y301/04041—Sphingomyelin phosphodiesterase D (3.1.4.41)
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • 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)
    • C12Y302/01021—Beta-glucosidase (3.2.1.21)
    • 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)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • 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)
    • C12Y302/01023—Beta-galactosidase (3.2.1.23), i.e. exo-(1-->4)-beta-D-galactanase
    • 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)
    • C12Y302/01031—Beta-glucuronidase (3.2.1.31)
    • 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)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77—Internalization into the cell
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
DK20197224.7T 2015-12-08 2016-12-08 Sammensætninger og fremgangsmåder til internalisering af enzymer DK3782639T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562264702P 2015-12-08 2015-12-08
US201662379629P 2016-08-25 2016-08-25
EP16829343.9A EP3386534B1 (en) 2015-12-08 2016-12-08 Compositions and methods for internalizing enzymes

Publications (1)

Publication Number Publication Date
DK3782639T3 true DK3782639T3 (da) 2022-08-29

Family

ID=57868326

Family Applications (2)

Application Number Title Priority Date Filing Date
DK16829343.9T DK3386534T3 (da) 2015-12-08 2016-12-08 Sammensætninger og fremgangsmåder til internalisering af enzymer
DK20197224.7T DK3782639T3 (da) 2015-12-08 2016-12-08 Sammensætninger og fremgangsmåder til internalisering af enzymer

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DK16829343.9T DK3386534T3 (da) 2015-12-08 2016-12-08 Sammensætninger og fremgangsmåder til internalisering af enzymer

Country Status (22)

Country Link
US (1) US20190112588A1 (da)
EP (3) EP3386534B1 (da)
JP (3) JP7065770B2 (da)
KR (1) KR20180084895A (da)
CN (2) CN108367056B (da)
AU (2) AU2016365834B2 (da)
BR (1) BR112018011474A2 (da)
CA (1) CA3006460A1 (da)
CY (1) CY1123676T1 (da)
DK (2) DK3386534T3 (da)
EA (1) EA201891277A1 (da)
ES (2) ES2926280T3 (da)
HU (2) HUE052155T2 (da)
IL (2) IL259714A (da)
MX (3) MX2018007061A (da)
MY (1) MY187933A (da)
NZ (1) NZ743008A (da)
PH (1) PH12018501074A1 (da)
PL (2) PL3386534T3 (da)
PT (2) PT3782639T (da)
SG (1) SG11201804375WA (da)
WO (1) WO2017100467A2 (da)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2825553T3 (da) 2012-03-14 2018-09-24 Regeneron Pharma : Multispecifikke antigenbindende molekyler og anvendelser deraf
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
DK3386534T3 (da) * 2015-12-08 2020-11-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
CN110325210A (zh) 2017-01-11 2019-10-11 小利兰·斯坦福大学托管委员会 Rspo替代物分子
CA3050133A1 (en) 2017-01-26 2018-08-02 Surrozen, Inc. Tissue-specific wnt signal enhancing molecules and uses thereof
AU2018281280A1 (en) * 2017-06-07 2020-01-16 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
CA3066945A1 (en) * 2017-06-27 2019-01-03 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized asgr1 locus
IL271837B2 (en) * 2017-07-07 2024-01-01 Hanmi Pharm Ind Co Ltd A new medical fusion protein with an enzyme and its uses
TW201925236A (zh) 2017-10-02 2019-07-01 美商戴納立製藥公司 包含酶替代療法酶之融合蛋白
AR113430A1 (es) * 2017-12-22 2020-04-29 Hanmi Pharm Ind Co Ltd Proteína de fusión enzimática terapéutica que tiene una nueva estructura y su uso
AU2019215782A1 (en) * 2018-02-05 2020-07-16 Jcr Pharmaceuticals Co., Ltd. Method for delivering drug to muscle
EP3749373A1 (en) * 2018-02-07 2020-12-16 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
JP2021530223A (ja) * 2018-07-09 2021-11-11 スロゼン， インコーポレイテッド 組織特異性Ｗｎｔシグナル増強分子及びその使用
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
KR20210054513A (ko) * 2018-08-02 2021-05-13 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 폼페병을 치료하기 위한 그의 용도
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2020028864A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US20210322562A1 (en) * 2018-08-02 2021-10-21 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating centronuclear myopathy
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CN113301925A (zh) 2018-12-19 2021-08-24 小利兰·斯坦福大学理事会 用于溶酶体靶向的双官能分子以及相关的组合物和方法
IL302029A (en) 2020-10-14 2023-06-01 Denali Therapeutics Inc Fusion proteins containing sulfoglucosamine sulfohydrolase enzymes and methods thereof
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4377352A2 (en) * 2021-07-30 2024-06-05 Vib Vzw Cation-independent mannose-6-phosphate receptor binders for targeted protein degradation
IL314482A (en) 2022-02-02 2024-09-01 Regeneron Pharma Anti-TFR:GAA and anti-CD63:GAA insertions for the treatment of Pompe disease
US11931421B2 (en) 2022-04-15 2024-03-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
US20240182561A1 (en) 2022-11-04 2024-06-06 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
US20240173426A1 (en) 2022-11-14 2024-05-30 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20050142141A1 (en) * 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
EP1877099B1 (en) 2005-04-06 2012-09-19 Genzyme Corporation Therapeutic conjugates comprising a lysosomal enzyme, polysialic acid and a targeting moiety
CA2641070C (en) * 2006-02-06 2017-01-03 Ramot At Tel-Aviv University Ltd. Enzyme replacement therapy for treating lysosomal storage diseases
US8569032B2 (en) 2007-05-18 2013-10-29 Tokyo Metropolitan Institute Of Medical Science Proteins having acquired A-galactosidase activity
CA2812870C (en) * 2010-09-29 2020-06-09 Oxyrane Uk Limited Mannosidases capable of uncapping mannose-1-phospho-6-mannose linkages and demannosylating phosphorylated n-glycans and methods of facilitating mammalian cellular uptake of glycoproteins
WO2012166653A2 (en) 2011-05-27 2012-12-06 Callidus Biopharma, Inc. Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
DK2818478T3 (da) 2011-10-28 2017-05-22 Regeneron Pharma Humaniseret il-6 og il-6-receptor
DK2825553T3 (da) 2012-03-14 2018-09-24 Regeneron Pharma : Multispecifikke antigenbindende molekyler og anvendelser deraf
EP3315514A1 (en) * 2012-08-29 2018-05-02 F. Hoffmann-La Roche AG Blood brain barrier shuttle
TWI641620B (zh) 2013-08-21 2018-11-21 再生元醫藥公司 抗－ｐｒｌｒ抗體及其用途
CA2962081A1 (en) * 2014-09-26 2016-03-31 Exerkine Corporation Exosomes useful to treat lysosomal storage disease
MA40791A (fr) * 2014-10-24 2017-09-27 Shire Human Genetic Therapies Ciblage lysosomal d'enzymes et utilisations associées
MA41022A (fr) * 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
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.
DK3386534T3 (da) * 2015-12-08 2020-11-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
AU2018281280A1 (en) * 2017-06-07 2020-01-16 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
EP3749373A1 (en) * 2018-02-07 2020-12-16 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery

Also Published As

Publication number Publication date
ES2833937T3 (es) 2021-06-16
MX2022010490A (es) 2022-09-21
AU2016365834B2 (en) 2023-09-28
EA201891277A1 (ru) 2018-11-30
CA3006460A1 (en) 2017-06-15
BR112018011474A2 (pt) 2018-12-04
JP2019505480A (ja) 2019-02-28
CN116196400A (zh) 2023-06-02
US20190112588A1 (en) 2019-04-18
SG11201804375WA (en) 2018-06-28
EP3386534A2 (en) 2018-10-17
EP3386534B1 (en) 2020-09-23
MY187933A (en) 2021-10-29
WO2017100467A4 (en) 2017-09-21
JP2024116182A (ja) 2024-08-27
ES2926280T3 (es) 2022-10-25
PL3782639T3 (pl) 2022-11-28
AU2023285891A1 (en) 2024-01-18
AU2016365834A1 (en) 2018-06-21
WO2017100467A3 (en) 2017-07-20
PL3386534T3 (pl) 2021-03-08
HUE060136T2 (hu) 2023-02-28
EP4074331A1 (en) 2022-10-19
CY1123676T1 (el) 2022-03-24
CN108367056B (zh) 2022-12-23
JP7524236B2 (ja) 2024-07-29
MX2018007061A (es) 2018-08-15
HUE052155T2 (hu) 2021-04-28
PH12018501074A1 (en) 2019-01-28
WO2017100467A2 (en) 2017-06-15
PT3386534T (pt) 2020-11-24
DK3386534T3 (da) 2020-11-30
NZ743008A (en) 2023-05-26
KR20180084895A (ko) 2018-07-25
PT3782639T (pt) 2022-08-10
CN108367056A (zh) 2018-08-03
IL287019A (en) 2021-12-01
JP2022058799A (ja) 2022-04-12
EP3782639B1 (en) 2022-07-20
MX2023000731A (es) 2023-02-13
JP7065770B2 (ja) 2022-05-12
EP3782639A1 (en) 2021-02-24
IL259714A (en) 2018-07-31

Similar Documents

Publication Publication Date Title
DK3386534T3 (da) Sammensætninger og fremgangsmåder til internalisering af enzymer
DK3842442T3 (da) Sammensætninger og fremgangsmåder til hæmning af arginaseaktivitet
DK3368541T3 (da) Sammensætninger og metoder til inhibiering af arginaseaktivitet
DK3325669T3 (da) Sammensætninger og fremgangsmåder til RNA-analyse
DK3390631T3 (da) Fremgangsmåder og sammensætninger til t-rna-baseret guide-rna-ekspression
DK3137605T3 (da) Sammensætninger og fremgangsmåder til modulering af angiopoietin-lignende-3-ekspression
DK3316909T3 (da) Anti-ntb-a-antistoffer og relaterede sammensætninger og fremgangsmåder
DK3125927T3 (da) Fremgangsmåder og sammensætninger til immunmodulering
DK3212778T3 (da) Fremgangsmåder og sammensætninger til stabilisering af trans-splejsning af intein-modificerede proteaser
DK3108009T3 (da) Fremgangsmåder og sammensætninger til DNA-profilering
DK3102673T3 (da) Fremgangsmåder og sammensætninger til behandling af beta-talassæmi
DK3092256T3 (da) Forbindelser og sammensætninger til immunterapi
KR20180084891A (ko) 구조 조성물 및 방법
DK3134130T3 (da) Sammensætninger og fremgangsmåder til behandling af hæmoglobinopatier
DK3393655T3 (da) Fenfluramin-sammensætninger og fremgangsmåder til fremstilling af samme
DK3402483T3 (da) Fremgangsmåder og sammensætninger til aktivering af gamma-delta-T-celler
DK3262008T3 (da) Partikelformede sammensætninger til dannelse af geopolymerer, deres anvendelse og fremgangsmåder til dannelse af geopolymere dermed
DK3319948T3 (da) Acesulfam-kaliumsammensætninger og fremgangsmåder til fremstilling af disse
DK3172233T3 (da) Anti-cd74-antistoffer, sammensætninger omfattende anti-cd74- antistoffer og fremgangsmåder til anvendelse af anti-cd74-antistoffer
DK2924093T3 (da) Multicarboxylat-sammensætninger og fremgangsmåde til fremstilling af disse
DK3322695T3 (da) Acesulfam-kalium-sammensætninger og fremgangsmåder til fremstilling af disse
DK3394259T3 (da) Sammensætninger og fremgangsmåder til reducering af tau-ekspression
DK3234160T3 (da) Sammensætninger og fremgangsmåder til hjælper-stamme-medieret svampegenommodifikation
DK3233192T3 (da) Sammensætninger og fremgangsmåder til målrettet cytokinindgivelse
DK3394272T3 (da) Sammensætninger og fremgangsmåder til effektiv målretning af transgener