SG11201804375WA - Compositions and methods for internalizing enzymes - Google Patents

Compositions and methods for internalizing enzymes

Info

Publication number
SG11201804375WA
SG11201804375WA SG11201804375WA SG11201804375WA SG11201804375WA SG 11201804375W A SG11201804375W A SG 11201804375WA SG 11201804375W A SG11201804375W A SG 11201804375WA SG 11201804375W A SG11201804375W A SG 11201804375WA SG 11201804375W A SG11201804375W A SG 11201804375WA
Authority
SG
Singapore
Prior art keywords
international
publication
date
tarrytown
pct
Prior art date
Application number
SG11201804375WA
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
Publication of SG11201804375WA publication Critical patent/SG11201804375WA/en

Links

Classifications

    • 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
    • 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
    • A61K47/6835—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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Cell Biology (AREA)
  • Biophysics (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (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

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -' Organization International Bureau (43) International Publication Date .... ....r .„,„1 15 June 2017(15.06.2017) WIPO I PCT (10) WO International 111111111111311111111111111111111111111111111111111111111111111111111111111111111111111111 2017/100467 Publication A4 Number (51) International Patent Classification: DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, A61K 38/47 (2006.01) C12N 9/42 (2006.01) HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, A61K 38/46 (2006.01) A61P 43/00 (2006.01) KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, C12N 9/40 (2006.01) MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, (21) International Application Number: RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, PCT/US2016/065647 TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, (22) International Filing Date: ZA, ZM, ZW. 8 December 2016 (08.12.2016) (84) Designated States (unless otherwise indicated, for every (25) Filing Language: English kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, (26) Publication Language: English TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, (30) Priority Data: TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, 62/264,702 8 December 2015 (08.12.2015) US DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, 62/379,629 25 August 2016 (25.08.2016) US LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, (71) Applicant: REGENERON PHARMACEUTICALS, GW, KM, ML, MR, NE, SN, TD, TG). INC. [US/US]; 777 Old Saw Mill River Road, Tarrytown, New York 10591 (US). Declarations under Rule 4.17: (72) Inventors: BAIK, Andrew; 777 Old Saw Mill River of inventorship (Rule 4.17(iv)) Road, Tarrytown, New York 10591 (US). CYGNAR, Published: Katherine; 777 Old Saw Mill River Road, Tarrytown, — with international search report (Art 21(3)) New York 10591 (US). — with amended claims (Art 19(1)) (74) Agent: ZAHNER, Joseph; Kutak Rock LLP, 2300 Main Street, Suite 800, Kansas City, Missouri 64108 (US). — with sequence listing part of description (Rule 5.2(a)) (81) Designated States (unless otherwise indicated, for every (88) Date of publication of the international search report: 20 July 2017 kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, Date of publication of the amended claims: 21 September 2017 BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, ll• .4t IN C ) i t C:: , C:: , l'I IN l'I 0 (54) Title: COMPOSITIONS AND METHODS FOR INTERNALIZING ENZYMES el 0 (57) : Compositions and methods for treating lysosomal storage diseases are disclosed. Biotherapeutic complexes contain- ing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The biotherapeutic com - plexes are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.
SG11201804375WA 2015-12-08 2016-12-08 Compositions and methods for internalizing enzymes SG11201804375WA (en)

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
PCT/US2016/065647 WO2017100467A2 (en) 2015-12-08 2016-12-08 Compositions and methods for internalizing enzymes

Publications (1)

Publication Number Publication Date
SG11201804375WA true SG11201804375WA (en) 2018-06-28

Family

ID=57868326

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201804375WA SG11201804375WA (en) 2015-12-08 2016-12-08 Compositions and methods for internalizing enzymes

Country Status (22)

Country Link
US (1) US20190112588A1 (en)
EP (3) EP3782639B1 (en)
JP (2) JP7065770B2 (en)
KR (1) KR20180084895A (en)
CN (2) CN116196400A (en)
AU (2) AU2016365834B2 (en)
BR (1) BR112018011474A2 (en)
CA (1) CA3006460A1 (en)
CY (1) CY1123676T1 (en)
DK (2) DK3386534T3 (en)
EA (1) EA201891277A1 (en)
ES (2) ES2833937T3 (en)
HU (2) HUE060136T2 (en)
IL (2) IL259714A (en)
MX (3) MX2018007061A (en)
MY (1) MY187933A (en)
NZ (1) NZ743008A (en)
PH (1) PH12018501074A1 (en)
PL (2) PL3386534T3 (en)
PT (2) PT3386534T (en)
SG (1) SG11201804375WA (en)
WO (1) WO2017100467A2 (en)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112014022692A8 (en) 2012-03-14 2021-07-20 Regeneron Pharma multispecific antigen binding molecule
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP3574019A4 (en) 2017-01-26 2021-03-03 Surrozen, Inc. Tissue-specific wnt signal enhancing molecules and uses thereof
NZ760232A (en) * 2017-06-07 2023-05-26 Regeneron Pharma Compositions and methods for internalizing enzymes
WO2019006034A1 (en) * 2017-06-27 2019-01-03 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized asgr1 locus
TWI832818B (en) * 2017-07-07 2024-02-21 南韓商韓美藥品股份有限公司 Novel therapeutic enzyme fusion protein and use thereof
MA50746A (en) 2017-10-02 2020-08-12 Denali Therapeutics Inc FUSION PROTEINS INCLUDING SUBSTITUTIVE ENZYMOTHERAPY ENZYMES
NZ765453A (en) * 2017-12-22 2024-03-22 Hanmi Pharmaceutical Co Ltd Therapeutic enzyme fusion protein having novel structure and use thereof
SG11202006433PA (en) * 2018-02-05 2020-08-28 Jcr Pharmaceuticals Co Ltd Method for delivering drug to muscle
BR112020016005A2 (en) * 2018-02-07 2020-12-15 Regeneron Pharmaceuticals, Inc. METHOD OF ADMINISTERING A THERAPEUTIC PROTEIN TO THE CENTRAL NERVOUS SYSTEM, MULTIDOMINIUM THERAPEUTIC PROTEIN, POLYNUCLEOTIDE, GENE THERAPY VECTOR, AND USE OF A NUCLEOTIDE THAT CODES THE THERAPEUTIC PROTEIN.
CA3104868A1 (en) * 2018-07-09 2020-01-16 Surrozen, Inc. Tissue-specific wnt signal enhancing molecules and uses thereof
WO2020028844A1 (en) * 2018-08-02 2020-02-06 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
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EA202190417A1 (en) * 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. MUSCLE-SPECIFIC COMPLEXES AND THEIR APPLICATION FOR TREATMENT OF PUMP'S DISEASE
KR20210081324A (en) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. Muscle targeting complexes and their use for treating facioscapulohumeral muscular dystrophy
CN113301925A (en) 2018-12-19 2021-08-24 小利兰·斯坦福大学理事会 Bifunctional molecules for lysosomal targeting and related compositions and methods
PE20231931A1 (en) 2020-10-14 2023-12-01 Denali Therapeutics Inc FUSION PROTEINS COMPRISING SULFOGLUCOSAMINE SULFOHYDROLASE ENZYMES AND METHODS THEREOF
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11638761B2 (en) 2021-07-09 2023-05-02 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
WO2023016828A2 (en) * 2021-07-30 2023-02-16 Vib Vzw Cation-independent mannose-6-phosphate receptor binders for targeted protein degradation
WO2023150623A2 (en) 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
US11931421B2 (en) 2022-04-15 2024-03-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2472937C (en) * 2002-01-11 2014-06-17 Biomarin Pharmaceutical, Inc. 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
US7341720B2 (en) * 2005-04-06 2008-03-11 Genzyme Corporation Targeting of glycoprotein therapeutics
US10265412B2 (en) * 2006-02-06 2019-04-23 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
US9347050B2 (en) * 2010-09-29 2016-05-24 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
US9545450B2 (en) 2011-05-27 2017-01-17 Amicus Therapeutics Inc. Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
MX358390B (en) 2011-10-28 2018-08-17 Regeneron Pharma Humanized il-6 and il-6 receptor.
BR112014022692A8 (en) * 2012-03-14 2021-07-20 Regeneron Pharma multispecific antigen binding molecule
RS58928B1 (en) * 2012-08-29 2019-08-30 Hoffmann La Roche Blood brain barrier shuttle
TWI641620B (en) 2013-08-21 2018-11-21 再生元醫藥公司 Anti-prlr antibodies and uses thereof
CA2962081A1 (en) * 2014-09-26 2016-03-31 Exerkine Corporation Exosomes useful to treat lysosomal storage disease
MA40791A (en) * 2014-10-24 2017-09-27 Shire Human Genetic Therapies LYSOSOMAL TARGETING OF ENZYMES AND RELATED USES
MA41022A (en) * 2014-11-24 2017-10-03 Shire Human Genetic Therapies LYSOSOMIAL TARGETING AND CORRESPONDING USES
CN114409783A (en) * 2015-06-24 2022-04-29 Jcr制药股份有限公司 Anti-human transferrin receptor antibodies across the blood brain barrier
NZ760232A (en) * 2017-06-07 2023-05-26 Regeneron Pharma Compositions and methods for internalizing enzymes
BR112020016005A2 (en) * 2018-02-07 2020-12-15 Regeneron Pharmaceuticals, Inc. METHOD OF ADMINISTERING A THERAPEUTIC PROTEIN TO THE CENTRAL NERVOUS SYSTEM, MULTIDOMINIUM THERAPEUTIC PROTEIN, POLYNUCLEOTIDE, GENE THERAPY VECTOR, AND USE OF A NUCLEOTIDE THAT CODES THE THERAPEUTIC PROTEIN.

Also Published As

Publication number Publication date
EP3782639B1 (en) 2022-07-20
EP3782639A1 (en) 2021-02-24
PT3782639T (en) 2022-08-10
JP2022058799A (en) 2022-04-12
JP2019505480A (en) 2019-02-28
PL3386534T3 (en) 2021-03-08
PH12018501074A1 (en) 2019-01-28
NZ743008A (en) 2023-05-26
DK3386534T3 (en) 2020-11-30
CN116196400A (en) 2023-06-02
PT3386534T (en) 2020-11-24
PL3782639T3 (en) 2022-11-28
EP3386534A2 (en) 2018-10-17
HUE052155T2 (en) 2021-04-28
HUE060136T2 (en) 2023-02-28
CN108367056A (en) 2018-08-03
IL259714A (en) 2018-07-31
ES2926280T3 (en) 2022-10-25
JP7065770B2 (en) 2022-05-12
MY187933A (en) 2021-10-29
CY1123676T1 (en) 2022-03-24
IL287019A (en) 2021-12-01
WO2017100467A3 (en) 2017-07-20
AU2016365834A1 (en) 2018-06-21
WO2017100467A2 (en) 2017-06-15
EP3386534B1 (en) 2020-09-23
DK3782639T3 (en) 2022-08-29
MX2023000731A (en) 2023-02-13
MX2022010490A (en) 2022-09-21
CA3006460A1 (en) 2017-06-15
BR112018011474A2 (en) 2018-12-04
EP4074331A1 (en) 2022-10-19
WO2017100467A4 (en) 2017-09-21
AU2023285891A1 (en) 2024-01-18
AU2016365834B2 (en) 2023-09-28
US20190112588A1 (en) 2019-04-18
ES2833937T3 (en) 2021-06-16
MX2018007061A (en) 2018-08-15
CN108367056B (en) 2022-12-23
KR20180084895A (en) 2018-07-25
EA201891277A1 (en) 2018-11-30

Similar Documents

Publication Publication Date Title
SG11201804375WA (en) Compositions and methods for internalizing enzymes
SG11201805320XA (en) Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
SG11201908719QA (en) Biomarkers and car t cell therapies with enhanced efficacy
SG11201909949XA (en) Targeted immunotolerance
SG11201809912UA (en) Hybrid carriers for nucleic acid cargo
SG11201900772YA (en) Gene editing of car-t cells for the treatment of t cell malignancies with chimeric antigen receptors
SG11201907870VA (en) Compositions and methods for inhibition of lineage specific proteins
SG11201808990QA (en) Compositions for topical application of compounds
SG11201806322QA (en) Maytansinoid derivatives, conjugates thereof, and methods of use
SG11201807301SA (en) Spiro-condensed pyrrolidine derivatives as deubiquitylating enzymes (dub) inhibitors
SG11201804134YA (en) Fgfr2 inhibitors alone or in combination with immune stimulating agents in cancer treatment
SG11201805709RA (en) Anti-pro/latent myostatin antibodies and methods of use thereof
SG11201810003UA (en) Using programmable dna binding proteins to enhance targeted genome modification
SG11201803701YA (en) Methods and compositions for gene editing in hematopoietic stem cells
SG11201907299XA (en) Multi-specific binding proteins for activation of natural killer cells and therapeutic uses thereof to treat cancer
SG11201407486PA (en) Compositions and methods for modulating utrn expression
SG11201407819UA (en) Improved methods of cell culture for adoptive cell therapy
SG11201804934PA (en) Novel Compounds
SG11201407859YA (en) Methods of treating cancer using pd-l1 axis binding antagonists and vegf antagonists
SG11201803906PA (en) Control of cellular redox levels
SG11201901959YA (en) Modified stem cell memory t cells, methods of making and methods of using same
SG11201407669QA (en) Methods for improving safety of blood-brain barrier transport
SG11201907646YA (en) Proteins binding her2, nkg2d and cd16
SG11201406903SA (en) Cell culture compositions and methods for polypeptide production
SG11201900554YA (en) Spiro-lactam nmda modulators and methods of using same