[go: up one dir, main page]

MX2022010490A - Composiciones utiles para enzimas de internalizacion. - Google Patents

Composiciones utiles para enzimas de internalizacion.

Info

Publication number
MX2022010490A
MX2022010490A MX2022010490A MX2022010490A MX2022010490A MX 2022010490 A MX2022010490 A MX 2022010490A MX 2022010490 A MX2022010490 A MX 2022010490A MX 2022010490 A MX2022010490 A MX 2022010490A MX 2022010490 A MX2022010490 A MX 2022010490A
Authority
MX
Mexico
Prior art keywords
compositions
methods
internalizing
enzymes
biotherapeutic
Prior art date
Application number
MX2022010490A
Other languages
English (en)
Inventor
Katherine Cygnar
Andrew Baik
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
Publication of MX2022010490A publication Critical patent/MX2022010490A/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6811Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
    • A61K47/6815Enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835Medicinal 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
    • A61K47/6849Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835Medicinal 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
    • A61K47/6875Medicinal 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
    • A61K47/6879Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2896Immunoglobulins [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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405Glucanases
    • C12N9/2434Glucanases acting on beta-1,4-glucosidic bonds
    • C12N9/2445Beta-glucosidase (3.2.1.21)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465Hydrolases (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)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric diester hydrolases (3.1.4)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04041Sphingomyelin phosphodiesterase D (3.1.4.41)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102Alpha-glucosidase (3.2.1.20)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01021Beta-glucosidase (3.2.1.21)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-galactosidase (3.2.1.22)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01023Beta-galactosidase (3.2.1.23), i.e. exo-(1-->4)-beta-D-galactanase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01031Beta-glucuronidase (3.2.1.31)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076L-Iduronidase (3.2.1.76)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77Internalization into the cell
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/70Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74Fusion 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)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Obesity (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)

Abstract

Las enzimas de reemplazo pueden suministrarse eficazmente al lisosoma de una célula diana específica cuando se asocian con una entidad de direccionamiento a la superficie celular. La presente invención proporciona una combinación de la enzima y la entidad de direccionamiento que se denomina complejo bioterapéutico. Por tanto, se proporciona una composición, es decir, un complejo bioterapéutico que comprende una enzima y una proteína de unión a antígeno. La enzima estÔ asociada con una enfermedad de almacenamiento lisosomal (LSD) y la proteína de unión a antígeno se une a un efector de internalización. El efector de internalización media la unión celular y la captación a un compartimento lisosómico. Los complejos bioterapéuticos son capaces de ingresar a las células, segregarse en el lisosoma y suministrar la actividad enzimÔtica de reemplazo al lisosoma que reemplaza la actividad enzimÔtica asociada con la LSD ("enzima endógena") y una proteína de unión a antígeno que se une a un efector de internalización, en donde el efector de internalización es CD63.
MX2022010490A 2015-12-08 2018-06-08 Composiciones utiles para enzimas de internalizacion. MX2022010490A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562264702P 2015-12-08 2015-12-08
US201662379629P 2016-08-25 2016-08-25

Publications (1)

Publication Number Publication Date
MX2022010490A true MX2022010490A (es) 2022-09-21

Family

ID=57868326

Family Applications (3)

Application Number Title Priority Date Filing Date
MX2018007061A MX2018007061A (es) 2015-12-08 2016-12-08 Composiciones y metodos para enzimas de internalizacion.
MX2022010490A MX2022010490A (es) 2015-12-08 2018-06-08 Composiciones utiles para enzimas de internalizacion.
MX2023000731A MX2023000731A (es) 2015-12-08 2018-06-08 Composiciones utiles para enzimas de internalizacion.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2018007061A MX2018007061A (es) 2015-12-08 2016-12-08 Composiciones y metodos para enzimas de internalizacion.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2023000731A MX2023000731A (es) 2015-12-08 2018-06-08 Composiciones utiles para enzimas de internalizacion.

Country Status (23)

Country Link
US (2) US20190112588A1 (es)
EP (3) EP3386534B1 (es)
JP (3) JP7065770B2 (es)
KR (2) KR102851747B1 (es)
CN (2) CN108367056B (es)
AU (2) AU2016365834B2 (es)
BR (1) BR112018011474A2 (es)
CA (1) CA3006460A1 (es)
CY (1) CY1123676T1 (es)
DK (3) DK3782639T3 (es)
EA (1) EA201891277A1 (es)
ES (3) ES2926280T3 (es)
FI (1) FI4074331T3 (es)
HU (2) HUE052155T2 (es)
IL (3) IL287019B2 (es)
MX (3) MX2018007061A (es)
MY (2) MY207684A (es)
NZ (2) NZ783252A (es)
PH (1) PH12018501074A1 (es)
PL (2) PL3782639T3 (es)
PT (3) PT4074331T (es)
SG (1) SG11201804375WA (es)
WO (1) WO2017100467A2 (es)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2825553T3 (pl) 2012-03-14 2018-12-31 Regeneron Pharmaceuticals, Inc. Wieloswoiste cząsteczki wiążące antygen i ich zastosowanie
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CA2987641A1 (en) 2015-07-06 2017-01-12 Regeneron Pharmaceuticals, Inc. Multispecific antigen-binding molecules and uses thereof
KR102851747B1 (ko) * 2015-12-08 2025-08-28 ė¦¬ģ œė„ˆė”  ķŒŒė§ˆģŠˆķ‹°ģ¹¼ģŠ¤ ģøģ½”ķ¬ė ˆģ“ķ‹°ė“œ ķšØģ†Œ ė‚“ģž¬ķ™” 씰성물 ė° 방법
CA3049661A1 (en) 2017-01-11 2018-07-19 The Board Of Trustees Of The Leland Stanford Junior University R-spondin (rspo) surrogate molecules
CN110234662A (zh) 2017-01-26 2019-09-13 ē‘Ÿē½—ę³½ę©å…¬åø 组织特异性wntäæ”å·å¢žå¼ŗåˆ†å­å’Œå…¶ē”Øé€”
BR112019025888A2 (pt) 2017-06-07 2020-06-30 Regeneron Pharmaceuticals, Inc. polinucleotídeo que codifica uma proteína terapêutica multidomínio, vetor de terapia gênica, proteína terapêutica multidomínio recombinante, método de expressão, métodos para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade, para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade epara tratar a deficiência enzimÔtica em um paciente em necessidade e/ou tolerar o paciente à enzima para a qual é deficiente, anticorpo anti-cd63 ou fragmento de ligação a antígeno, e, composição farmacêutica
IL271561B2 (en) 2017-06-27 2025-08-01 Regeneron Pharma Non-human animals comprising a humanized asgr1 locus
BR112020000273A2 (pt) * 2017-07-07 2020-07-14 Hanmi Pharm. Co., Ltd. proteína de fusão de enzima e seu método de preparação, composição farmacêutica, polinucleotídeo que codifica a proteína de fusão de enzima, vetor de expressão, transformante
CA3076369A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
EP3708661A4 (en) 2017-12-22 2021-12-01 Hanmi Pharm. Co., Ltd. THERAPEUTIC ENZYMFUSION PROTEIN WITH A NEW STRUCTURE AND ITS USE
US20210038739A1 (en) * 2018-02-05 2021-02-11 Jcr Pharmaceuticals Co., Ltd. Method for Delivering Drug to Muscle
JP7779653B2 (ja) * 2018-02-07 2025-12-03 ćƒŖć‚øć‚§ćƒćƒ­ćƒ³ćƒ»ćƒ•ć‚”ćƒ¼ćƒžć‚·ćƒ„ćƒ¼ćƒ†ć‚£ć‚«ćƒ«ć‚ŗćƒ»ć‚¤ćƒ³ć‚³ćƒ¼ćƒćƒ¬ć‚¤ćƒ†ćƒƒćƒ‰ ę²»ē™‚ē”Øć‚æćƒ³ćƒ‘ć‚Æč³Ŗé€é”ć®ćŸć‚ć®ę–¹ę³•ćŠć‚ˆć³ēµ„ęˆē‰©
MY206284A (en) 2018-05-17 2024-12-06 Regeneron Pharma Anti-cd63 antibodies, conjugates, and uses thereof
EP3820499A4 (en) 2018-07-09 2022-07-13 Surrozen Operating, Inc. TISSUE SPECIFIC WNT SIGNAL ENHANCEMENT MOLECULES AND USES
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
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
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
KR20210054513A (ko) * 2018-08-02 2021-05-13 ė‹¤ģø ģ„øė¼ķ“Øķ‹±ģŠ¤, ģøķ¬. 근윔 ķ‘œģ ķ™” 복합첓 ė° ķ¼ķŽ˜ė³‘ģ„ ģ¹˜ė£Œķ•˜źø° ģœ„ķ•œ ź·øģ˜ ģš©ė„
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP3830130A4 (en) * 2018-08-02 2022-05-18 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating centronuclear myopathy
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN113301925A (zh) 2018-12-19 2021-08-24 å°åˆ©å…°Ā·ę–Æå¦ē¦å¤§å­¦ē†äŗ‹ä¼š ē”ØäŗŽęŗ¶é…¶ä½“é¶å‘ēš„åŒå®˜čƒ½åˆ†å­ä»„åŠē›øå…³ēš„ē»„åˆē‰©å’Œę–¹ę³•
CA3145672A1 (en) * 2019-07-03 2021-01-07 Palleon Pharmaceuticals Inc. Sialidase-cd20-antibody fusion proteins and methods of use thereof
MA71661A (fr) 2020-10-14 2025-05-30 Denali Therapeutics Inc. ProtƩines de fusion comprenant des enzymes sulfoglucosamine sulfohydrolase et mƩthodes associƩes
TW202233656A (zh) 2020-11-16 2022-09-01 ē¾Žå•†č˜‡ē¾…ę£®ē‡Ÿé‹å…¬åø č‚ē‰¹ē•°ę€§wntčØŠč™Ÿå¢žå¼·åˆ†å­åŠå…¶ē”Øé€”
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
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
JP2024524614A (ja) 2021-07-14 2024-07-05 ćƒ©ć‚¤ć‚·ć‚¢ ć‚»ćƒ©ćƒ”ćƒ„ćƒ¼ćƒ†ć‚£ć‚Æć‚¹ļ¼Œ ć‚¤ćƒ³ć‚³ćƒ¼ćƒćƒ¬ć‚¤ćƒ†ćƒƒćƒ‰ ļ¼”ļ½“ļ½‡ļ½ļ½’ē“°čƒžč”Øé¢å—å®¹ä½“ēµåˆåŒ–åˆē‰©åŠć³ć‚³ćƒ³ć‚øćƒ„ć‚²ćƒ¼ćƒˆ
WO2023016828A2 (en) * 2021-07-30 2023-02-16 Vib Vzw Cation-independent mannose-6-phosphate receptor binders for targeted protein degradation
EP4473103A2 (en) 2022-02-02 2024-12-11 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
AU2023254846A1 (en) 2022-04-15 2024-10-10 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
JP2025538220A (ja) 2022-11-14 2025-11-26 ćƒŖć‚øć‚§ćƒćƒ­ćƒ³ćƒ»ćƒ•ć‚”ćƒ¼ćƒžć‚·ćƒ„ćƒ¼ćƒ†ć‚£ć‚«ćƒ«ć‚ŗćƒ»ć‚¤ćƒ³ć‚³ćƒ¼ćƒćƒ¬ć‚¤ćƒ†ćƒƒćƒ‰ ć‚¢ć‚¹ćƒˆćƒ­ć‚µć‚¤ćƒˆćøć®ē·šē¶­čŠ½ē“°čƒžå¢—ę®–å› å­å—å®¹ä½“ļ¼“åŖ’ä»‹é€é”ć®ćŸć‚ć®ēµ„ęˆē‰©ćŠć‚ˆć³ę–¹ę³•
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
WO2025137213A1 (en) 2023-12-20 2025-06-26 Denali Therapeutics Inc. Fusion proteins comprising acid alpha-glucosidase enzymes and methods thereof
US20250242056A1 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for using plasma cell depleting agents and/or b cell depleting agents to suppress host anti-aav antibody response and enable aav transduction and re-dosing
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response

Family Cites Families (20)

* 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
WO2006108052A2 (en) * 2005-04-06 2006-10-12 Genzyme Corporation Peg and polysialic lysosomal enzyme conjugates via acid labile linkers for therapeutic targeting
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
US8569032B2 (en) * 2007-05-18 2013-10-29 Tokyo Metropolitan Institute Of Medical Science Proteins having acquired A-galactosidase activity
JP6151183B2 (ja) * 2010-09-29 2017-06-21 ć‚Ŗć‚­ć‚·ćƒ¬ć‚¤ćƒ³ ćƒ¦ćƒ¼ć‚±ćƒ¼ ćƒŖćƒŸćƒ†ćƒƒćƒ‰ ćƒžćƒ³ćƒŽćƒ¼ć‚¹āˆ’ļ¼‘āˆ’ćƒ›ć‚¹ćƒ›āˆ’ļ¼–āˆ’ćƒžćƒ³ćƒŽćƒ¼ć‚¹ēµåˆć‹ć‚‰ć‚­ćƒ£ćƒƒćƒ—ć‚’å¤–ć™ć“ćØćŠć‚ˆć³ćƒŖćƒ³é…øåŒ–ļ½Žāˆ’ć‚°ćƒŖć‚«ćƒ³ć‚’č„±ćƒžćƒ³ćƒŽć‚·ćƒ«åŒ–ć™ć‚‹ć“ćØćŒć§ćć‚‹ćƒžćƒ³ćƒŽć‚·ćƒ€ćƒ¼ć‚¼ćŖć‚‰ć³ć«å“ŗä¹³å‹•ē‰©ē“°čƒžć«ć‚ˆć‚‹ē³–ć‚æćƒ³ćƒ‘ć‚Æč³Ŗć®å–ć‚Šč¾¼ćæć‚’äæƒé€²ć™ć‚‹ę–¹ę³•
CA2836318C (en) * 2011-05-27 2018-11-27 Callidus Biopharma, Inc. Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
DK2739649T3 (en) * 2011-08-05 2018-01-08 Bioasis Technologies Inc P97 FRAGMENTS WITH TRANSFER ACTIVITY
PL2663575T3 (pl) 2011-10-28 2015-03-31 Regeneron Pharma Humanizowane IL-6 i receptor IL-6
PL2825553T3 (pl) * 2012-03-14 2018-12-31 Regeneron Pharmaceuticals, Inc. Wieloswoiste cząsteczki wiążące antygen i ich zastosowanie
HUE045144T2 (hu) * 2012-08-29 2019-12-30 Hoffmann La Roche VƩragygƔt-shuttle
AU2014218854B2 (en) * 2013-02-20 2019-01-24 Valerion Therapeutics, Llc Methods and compositions for treatment of Pompe disease
TWI641620B (zh) 2013-08-21 2018-11-21 å†ē”Ÿå…ƒé†«č—„å…¬åø ęŠ—ļ¼ļ½ļ½’ļ½Œļ½’ęŠ—é«”åŠå…¶ē”Øé€”
CA2962081A1 (en) * 2014-09-26 2016-03-31 Exerkine Corporation Exosomes useful to treat lysosomal storage disease
EP3220958A1 (en) * 2014-10-24 2017-09-27 Shire Human Genetic Therapies, Inc. Lysosomal targeting of enzymes, and uses thereof
MA41022A (fr) * 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
TWI769982B (zh) * 2015-06-24 2022-07-11 ę—„å•†ļ¼Ŗļ½ƒļ½’č£½č—„č‚”ä»½ęœ‰é™å…¬åø é€šéŽč”€č…¦éšœå£ä¹‹ęŠ—äŗŗé”žé‹éµč›‹ē™½å—é«”ęŠ—é«”
KR102851747B1 (ko) * 2015-12-08 2025-08-28 ė¦¬ģ œė„ˆė”  ķŒŒė§ˆģŠˆķ‹°ģ¹¼ģŠ¤ ģøģ½”ķ¬ė ˆģ“ķ‹°ė“œ ķšØģ†Œ ė‚“ģž¬ķ™” 씰성물 ė° 방법
BR112019025888A2 (pt) * 2017-06-07 2020-06-30 Regeneron Pharmaceuticals, Inc. polinucleotídeo que codifica uma proteína terapêutica multidomínio, vetor de terapia gênica, proteína terapêutica multidomínio recombinante, método de expressão, métodos para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade, para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade epara tratar a deficiência enzimÔtica em um paciente em necessidade e/ou tolerar o paciente à enzima para a qual é deficiente, anticorpo anti-cd63 ou fragmento de ligação a antígeno, e, composição farmacêutica
JP7779653B2 (ja) * 2018-02-07 2025-12-03 ćƒŖć‚øć‚§ćƒćƒ­ćƒ³ćƒ»ćƒ•ć‚”ćƒ¼ćƒžć‚·ćƒ„ćƒ¼ćƒ†ć‚£ć‚«ćƒ«ć‚ŗćƒ»ć‚¤ćƒ³ć‚³ćƒ¼ćƒćƒ¬ć‚¤ćƒ†ćƒƒćƒ‰ ę²»ē™‚ē”Øć‚æćƒ³ćƒ‘ć‚Æč³Ŗé€é”ć®ćŸć‚ć®ę–¹ę³•ćŠć‚ˆć³ēµ„ęˆē‰©

Also Published As

Publication number Publication date
WO2017100467A4 (en) 2017-09-21
EP3782639A1 (en) 2021-02-24
IL287019A (en) 2021-12-01
IL320861A (en) 2025-07-01
DK3782639T3 (da) 2022-08-29
WO2017100467A2 (en) 2017-06-15
KR20180084895A (ko) 2018-07-25
PT3782639T (pt) 2022-08-10
CY1123676T1 (el) 2022-03-24
SG11201804375WA (en) 2018-06-28
EA201891277A1 (ru) 2018-11-30
PT3386534T (pt) 2020-11-24
US20190112588A1 (en) 2019-04-18
ES3045508T3 (en) 2025-11-28
AU2023285891A1 (en) 2024-01-18
AU2023285891B2 (en) 2024-10-03
JP2024116182A (ja) 2024-08-27
CA3006460A1 (en) 2017-06-15
NZ783252A (en) 2024-12-20
MX2018007061A (es) 2018-08-15
MY187933A (en) 2021-10-29
AU2016365834B2 (en) 2023-09-28
EP4074331B1 (en) 2025-09-03
WO2017100467A3 (en) 2017-07-20
AU2024278420A1 (en) 2025-01-09
HUE052155T2 (hu) 2021-04-28
NZ743008A (en) 2023-05-26
JP7524236B2 (ja) 2024-07-29
CN116196400A (zh) 2023-06-02
JP7065770B2 (ja) 2022-05-12
IL287019B1 (en) 2025-06-01
EP3386534A2 (en) 2018-10-17
ES2926280T3 (es) 2022-10-25
PH12018501074A1 (en) 2019-01-28
DK4074331T3 (da) 2025-10-20
NZ783256A (en) 2025-05-02
ES2833937T3 (es) 2021-06-16
AU2016365834A1 (en) 2018-06-21
PL3782639T3 (pl) 2022-11-28
MX2023000731A (es) 2023-02-13
MY207684A (en) 2025-03-12
HUE060136T2 (hu) 2023-02-28
FI4074331T3 (fi) 2025-10-22
JP2019505480A (ja) 2019-02-28
DK3386534T3 (da) 2020-11-30
IL259714A (en) 2018-07-31
US20250188433A1 (en) 2025-06-12
EP4074331A1 (en) 2022-10-19
PT4074331T (pt) 2025-11-04
IL287019B2 (en) 2025-10-01
BR112018011474A2 (en) 2018-12-04
CN108367056A (zh) 2018-08-03
CN108367056B (zh) 2022-12-23
PL3386534T3 (pl) 2021-03-08
EP3386534B1 (en) 2020-09-23
KR20250133801A (ko) 2025-09-08
EP3782639B1 (en) 2022-07-20
JP2022058799A (ja) 2022-04-12
KR102851747B1 (ko) 2025-08-28

Similar Documents

Publication Publication Date Title
MX2022010490A (es) Composiciones utiles para enzimas de internalizacion.
PH12019550261A1 (en) Compositions and methods for internalizing enzymes
PH12020550510A1 (en) Fusion proteins comprising enzyme replacement therapy enzymes
MX2017004691A (es) Bloqueo de cd73.
EP4559918A3 (en) Glucosylceramide synthase inhibitors
PH12018500810A1 (en) Antibody agents specific for human cd19 and uses thereof
MX2018014890A (es) Composiciones de limpieza que comprenden enzimas.
MX2019007143A (es) Procesamiento de materiales de biomasa.
AU2015311681A8 (en) Globin gene therapy for treating hemoglobinopathies
EP2596122A4 (en) MODULATORS OF SIRT5 AND TESTS FOR SCREENING
MX2019009229A (es) Novedosas variantes enzimaticas que degradan la nicotina.
MX2018010196A (es) Proteinas de fusion de enzimas lisosomales terapeuticas dirigidas, formulaciones asociadas y usos de las mismas.
SG10201804952QA (en) Glucosylceramide synthase inhibitors
MX2015012842A (es) Prodecimiento para preparar inhibidores de la glucosilceramida sintasa.
CA2986376C (en) Poxvirus-derived promoter, and vector comprising same
MX2015009949A (es) Supresor de mimicos de lesion 1 (slm1) suspresor de fenotipos mimicos de lesion.
MX2016008716A (es) Expresion aumentada de proteinas.
MX2017014828A (es) Procesos para incrementar la extraccion de enzimas de alimento animal y medir la actividad de las mismas.
MX2016008595A (es) Expresion mejorada de proteĆ­nas.
TN2013000368A1 (en) Glucosylceramide synthase inhibitors