JP2021525711A - ゴーシェ病の処置 - Google Patents

ゴーシェ病の処置 Download PDF

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Publication number
JP2021525711A
JP2021525711A JP2020565929A JP2020565929A JP2021525711A JP 2021525711 A JP2021525711 A JP 2021525711A JP 2020565929 A JP2020565929 A JP 2020565929A JP 2020565929 A JP2020565929 A JP 2020565929A JP 2021525711 A JP2021525711 A JP 2021525711A
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JP
Japan
Prior art keywords
polypeptide
fragment
activator
amino acids
disease
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.)
Pending
Application number
JP2020565929A
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English (en)
Japanese (ja)
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JP2021525711A5 (https=
JPWO2019231725A5 (https=
Inventor
メイ メイ ティアン，
メイ メイ ティアン，
マーク デイ，
マーク デイ，
Original Assignee
バイオアシス テクノロジーズ インコーポレイテッド
バイオアシス テクノロジーズ インコーポレイテッド
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Application filed by バイオアシス テクノロジーズ インコーポレイテッド, バイオアシス テクノロジーズ インコーポレイテッド filed Critical バイオアシス テクノロジーズ インコーポレイテッド
Publication of JP2021525711A publication Critical patent/JP2021525711A/ja
Publication of JP2021525711A5 publication Critical patent/JP2021525711A5/ja
Publication of JPWO2019231725A5 publication Critical patent/JPWO2019231725A5/ja
Pending legal-status Critical Current

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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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7004—Monosaccharides having only carbon, hydrogen and oxygen atoms
    • 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)
    • A61K38/47—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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/04—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; involved in cellular and subcellular movement (3.6.4)
    • C12Y306/04006—Vesicle-fusing ATPase (3.6.4.6)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2020565929A 2018-05-27 2019-05-18 ゴーシェ病の処置 Pending JP2021525711A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862677013P 2018-05-27 2018-05-27
US62/677,013 2018-05-27
PCT/US2019/033012 WO2019231725A2 (en) 2018-05-27 2019-05-18 Treatment of gaucher disease

Publications (3)

Publication Number Publication Date
JP2021525711A true JP2021525711A (ja) 2021-09-27
JP2021525711A5 JP2021525711A5 (https=) 2022-05-02
JPWO2019231725A5 JPWO2019231725A5 (https=) 2022-05-02

Family

ID=68697612

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020565929A Pending JP2021525711A (ja) 2018-05-27 2019-05-18 ゴーシェ病の処置

Country Status (8)

Country Link
US (1) US20210113703A1 (https=)
EP (1) EP3796928A4 (https=)
JP (1) JP2021525711A (https=)
KR (1) KR20210024492A (https=)
CN (1) CN112512558A (https=)
AU (1) AU2019276882A1 (https=)
CA (1) CA3101097A1 (https=)
WO (1) WO2019231725A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210355468A1 (en) * 2020-05-18 2021-11-18 Bioasis Technologies, Inc. Compositions and methods for treating lewy body dementia
US20210393787A1 (en) * 2020-06-17 2021-12-23 Bioasis Technologies, Inc. Compositions and methods for treating frontotemporal dementia
KR102593930B1 (ko) 2023-04-18 2023-10-25 주식회사 한국스크랩 친환경 폐 알루미늄 박리 자동화 시스템
CN119074730A (zh) * 2024-08-28 2024-12-06 北京大学第一医院(北京大学第一临床医学院) 葡萄糖神经酰胺合酶抑制剂在制备预防和/或治疗代谢应激性肝脏损伤相关疾病产品中的应用

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006506317A (ja) * 2002-01-11 2006-02-23 バイオマリン ファーマシューティカル インコーポレイテッド 治療用リソソーム酵素の送達のための酵素送達系としてのｐ９７の使用
JP2013500976A (ja) * 2009-07-28 2013-01-10 シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド ゴーシェ病を治療するための組成物および方法
JP2013512252A (ja) * 2009-11-27 2013-04-11 ジェンザイム・コーポレーション グルコシルセラミドシンターゼの阻害剤としての非晶質及び結晶型のｇｅｎｚ１１２６３８ヘミ酒石酸塩
US20150023929A1 (en) * 2012-02-19 2015-01-22 Protalix Ltd. Oral unit dosage forms and uses of same for the treatment of gaucher disease
JP2016515121A (ja) * 2013-03-13 2016-05-26 バイオアシス テクノロジーズ インコーポレイテッド ｐ９７のフラグメントおよびその使用

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2582869T3 (es) * 2011-07-05 2016-09-15 Bioasis Technologies Inc Conjugados de P97-anticuerpo
US10058619B2 (en) * 2014-05-01 2018-08-28 Bioasis Technologies, Inc. P97-polynucleotide conjugates
US20180009898A1 (en) * 2016-07-07 2018-01-11 Bioasis Technologies, Inc. Antibodies against p97 and conjugates thereof

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006506317A (ja) * 2002-01-11 2006-02-23 バイオマリン ファーマシューティカル インコーポレイテッド 治療用リソソーム酵素の送達のための酵素送達系としてのｐ９７の使用
JP2013500976A (ja) * 2009-07-28 2013-01-10 シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド ゴーシェ病を治療するための組成物および方法
JP2013512252A (ja) * 2009-11-27 2013-04-11 ジェンザイム・コーポレーション グルコシルセラミドシンターゼの阻害剤としての非晶質及び結晶型のｇｅｎｚ１１２６３８ヘミ酒石酸塩
US20150023929A1 (en) * 2012-02-19 2015-01-22 Protalix Ltd. Oral unit dosage forms and uses of same for the treatment of gaucher disease
JP2016515121A (ja) * 2013-03-13 2016-05-26 バイオアシス テクノロジーズ インコーポレイテッド ｐ９７のフラグメントおよびその使用

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
"The Lipid", VOL．27 NO．1, JPN6023019827, 2016, pages 4 - 8, ISSN: 0005220375 *

Also Published As

Publication number Publication date
WO2019231725A3 (en) 2020-01-09
EP3796928A2 (en) 2021-03-31
CN112512558A (zh) 2021-03-16
CA3101097A1 (en) 2019-12-05
WO2019231725A2 (en) 2019-12-05
US20210113703A1 (en) 2021-04-22
AU2019276882A1 (en) 2021-01-14
EP3796928A4 (en) 2022-03-02
KR20210024492A (ko) 2021-03-05

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