JP7532338B2 - ヘキソサミニダーゼａ、酸スフィンゴミエリナーゼおよびパルミトイル－タンパク質チオエステラーゼ１のｃｎｓにおける活性を増加させるための方法および組成物 - Google Patents

ヘキソサミニダーゼａ、酸スフィンゴミエリナーゼおよびパルミトイル－タンパク質チオエステラーゼ１のｃｎｓにおける活性を増加させるための方法および組成物 Download PDF

Info

Publication number
JP7532338B2
JP7532338B2 JP2021506506A JP2021506506A JP7532338B2 JP 7532338 B2 JP7532338 B2 JP 7532338B2 JP 2021506506 A JP2021506506 A JP 2021506506A JP 2021506506 A JP2021506506 A JP 2021506506A JP 7532338 B2 JP7532338 B2 JP 7532338B2
Authority
JP
Japan
Prior art keywords
amino acid
hir
enzyme
fusion
antibody
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2021506506A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021533167A5 (enExample
JP2021533167A (ja
Inventor
ウィリアム・エム・パードリッジ
ルーベン・ジェイ・ボアド
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Armagen Inc
Original Assignee
Armagen Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Armagen Inc filed Critical Armagen Inc
Publication of JP2021533167A publication Critical patent/JP2021533167A/ja
Publication of JP2021533167A5 publication Critical patent/JP2021533167A5/ja
Priority to JP2024124266A priority Critical patent/JP2024153824A/ja
Application granted granted Critical
Publication of JP7532338B2 publication Critical patent/JP7532338B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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
    • A61K47/6847—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 hormone or a hormone-releasing or -inhibiting factor
    • 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
    • A61K47/6889—Conjugates wherein the antibody being the modifying agent and wherein the linker, binder or spacer confers particular properties to the conjugates, e.g. peptidic enzyme-labile linkers or acid-labile linkers, providing for an acid-labile immuno conjugate wherein the drug may be released from its antibody conjugated part in an acidic, e.g. tumoural or environment
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2869—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against hormone receptors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/02—Thioester hydrolases (3.1.2)
    • C12Y301/02022—Palmitoyl-protein hydrolase (3.1.2.22)
    • 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)
    • C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/545—Medicinal preparations containing antigens or antibodies characterised by the dose, timing or administration schedule
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/52—Constant or Fc region; Isotype
    • C07K2317/53—Hinge
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
    • C07K2317/565—Complementarity determining region [CDR]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • 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)
    • C12Y302/01052—Beta-N-acetylhexosaminidase (3.2.1.52)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Endocrinology (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Obesity (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
JP2021506506A 2018-08-07 2019-08-07 ヘキソサミニダーゼａ、酸スフィンゴミエリナーゼおよびパルミトイル－タンパク質チオエステラーゼ１のｃｎｓにおける活性を増加させるための方法および組成物 Active JP7532338B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024124266A JP2024153824A (ja) 2018-08-07 2024-07-31 ヘキソサミニダーゼａ、酸スフィンゴミエリナーゼおよびパルミトイル－タンパク質チオエステラーゼ１のｃｎｓにおける活性を増加させるための方法および組成物

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862715696P 2018-08-07 2018-08-07
US201862715693P 2018-08-07 2018-08-07
US201862715697P 2018-08-07 2018-08-07
US62/715,697 2018-08-07
US62/715,696 2018-08-07
US62/715,693 2018-08-07
PCT/US2019/045547 WO2020033577A2 (en) 2018-08-07 2019-08-07 Methods and compositions for increasing the activity in the cns of hexosaminidase a, acid sphingomyelinase, and palmitoyl-protein thioesterase 1

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024124266A Division JP2024153824A (ja) 2018-08-07 2024-07-31 ヘキソサミニダーゼａ、酸スフィンゴミエリナーゼおよびパルミトイル－タンパク質チオエステラーゼ１のｃｎｓにおける活性を増加させるための方法および組成物

Publications (3)

Publication Number Publication Date
JP2021533167A JP2021533167A (ja) 2021-12-02
JP2021533167A5 JP2021533167A5 (enExample) 2022-08-10
JP7532338B2 true JP7532338B2 (ja) 2024-08-13

Family

ID=69415150

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021506506A Active JP7532338B2 (ja) 2018-08-07 2019-08-07 ヘキソサミニダーゼａ、酸スフィンゴミエリナーゼおよびパルミトイル－タンパク質チオエステラーゼ１のｃｎｓにおける活性を増加させるための方法および組成物
JP2024124266A Pending JP2024153824A (ja) 2018-08-07 2024-07-31 ヘキソサミニダーゼａ、酸スフィンゴミエリナーゼおよびパルミトイル－タンパク質チオエステラーゼ１のｃｎｓにおける活性を増加させるための方法および組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024124266A Pending JP2024153824A (ja) 2018-08-07 2024-07-31 ヘキソサミニダーゼａ、酸スフィンゴミエリナーゼおよびパルミトイル－タンパク質チオエステラーゼ１のｃｎｓにおける活性を増加させるための方法および組成物

Country Status (6)

Country Link
US (1) US20240252667A1 (enExample)
EP (1) EP3833689A4 (enExample)
JP (2) JP7532338B2 (enExample)
AU (1) AU2019317460A1 (enExample)
CA (1) CA3107749A1 (enExample)
WO (1) WO2020033577A2 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013507131A (ja) 2009-10-09 2013-03-04 アーメイゲン・テクノロジーズ・インコーポレイテッド Ｃｎｓにおけるイズロン酸２−スルファターゼ活性を増加させるための方法および組成物
AU2024241509A1 (en) * 2023-03-20 2025-09-25 Spark Therapeutics, Inc. Ppt1 gene therapy

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009515819A (ja) 2005-10-07 2009-04-16 アーメイゲン・テクノロジーズ・インコーポレイテッド 血液脳関門輸送用融合タンパク質
JP2010534723A (ja) 2007-07-27 2010-11-11 アーメイゲン・テクノロジーズ・インコーポレイテッド 中枢神経系のα−Ｌ−イデュロニダーゼ活性を増加させるための方法および組成物
JP2013507131A (ja) 2009-10-09 2013-03-04 アーメイゲン・テクノロジーズ・インコーポレイテッド Ｃｎｓにおけるイズロン酸２−スルファターゼ活性を増加させるための方法および組成物
JP2015509701A (ja) 2011-12-02 2015-04-02 アーメイゲン・テクノロジーズ・インコーポレイテッドＡｒｍａｇｅｎ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． Ｃｎｓにおけるアリールスルファターゼａ活性を増加するための方法および組成物
JP2016525545A (ja) 2013-07-22 2016-08-25 アーメイゲン・テクノロジーズ・インコーポレイテッドＡｒｍａｇｅｎ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． Ｃｎｓにおける酵素活性を増大するための方法および組成物

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050142141A1 (en) * 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
CA2641070C (en) * 2006-02-06 2017-01-03 Ramot At Tel-Aviv University Ltd. Enzyme replacement therapy for treating lysosomal storage diseases
IL193282A (en) * 2006-02-06 2013-04-30 Yeda Res & Dev Chimeric protein of therapeutic enzyme and granulocyte-stimulating factor for treatment of lysosomal storage diseases
EP2910632B1 (en) * 2012-10-19 2017-07-05 Tokushima University Highly functional enzyme having modified substrate specificity of human beta-hexosaminidase b and exhibiting protease resistance
EP3119892B1 (en) * 2014-03-17 2021-03-10 The Hospital For Sick Children Beta-hexosaminidase protein variants and associated methods for treating gm2 gangliosdoses
US10538589B2 (en) * 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
EP3448874A4 (en) * 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009515819A (ja) 2005-10-07 2009-04-16 アーメイゲン・テクノロジーズ・インコーポレイテッド 血液脳関門輸送用融合タンパク質
JP2010534723A (ja) 2007-07-27 2010-11-11 アーメイゲン・テクノロジーズ・インコーポレイテッド 中枢神経系のα−Ｌ−イデュロニダーゼ活性を増加させるための方法および組成物
JP2013507131A (ja) 2009-10-09 2013-03-04 アーメイゲン・テクノロジーズ・インコーポレイテッド Ｃｎｓにおけるイズロン酸２−スルファターゼ活性を増加させるための方法および組成物
JP2015509701A (ja) 2011-12-02 2015-04-02 アーメイゲン・テクノロジーズ・インコーポレイテッドＡｒｍａｇｅｎ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． Ｃｎｓにおけるアリールスルファターゼａ活性を増加するための方法および組成物
JP2016525545A (ja) 2013-07-22 2016-08-25 アーメイゲン・テクノロジーズ・インコーポレイテッドＡｒｍａｇｅｎ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． Ｃｎｓにおける酵素活性を増大するための方法および組成物

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Biochimica et Biophysica Acta，2015年，Vol.1852，p.2292-2300，http://dx.doi.org/10.1016/j.bbadis.2015.04.026
Mol Genet Metab.，2010年，Vol.99, No.4，p.374-378 (P.1-12)，doi:10.1016/j.ymgme.2009.12.002

Also Published As

Publication number Publication date
AU2019317460A1 (en) 2021-03-18
EP3833689A2 (en) 2021-06-16
JP2024153824A (ja) 2024-10-29
CA3107749A1 (en) 2020-02-13
WO2020033577A2 (en) 2020-02-13
WO2020033577A3 (en) 2020-03-19
US20240252667A1 (en) 2024-08-01
JP2021533167A (ja) 2021-12-02
EP3833689A4 (en) 2022-05-25

Similar Documents

Publication Publication Date Title
US20260078178A1 (en) Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
US20220348672A1 (en) Methods and compositions for increasing enzyme activity in the cns
US20240409650A1 (en) Methods and compositions for increasing n-acetylglucosaminidase activity in the cns
US8715661B2 (en) Methods and compositions for increasing arylsulfatase A activity in the CNS
US20230279156A1 (en) Methods and compositions for increasing alpha-l-iduronidase activity in the cns
US20250179201A1 (en) Methods and compositions for increasing galactosidase beta-1 activity in the cns
JP2024153824A (ja) ヘキソサミニダーゼａ、酸スフィンゴミエリナーゼおよびパルミトイル－タンパク質チオエステラーゼ１のｃｎｓにおける活性を増加させるための方法および組成物

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220802

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220802

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230711

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230920

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231013

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231205

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240126

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20240502

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240604

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20240702

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20240731

R150 Certificate of patent or registration of utility model

Ref document number: 7532338

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150