JP2016503405A - タンパク質症を処置するための組成物および方法 - Google Patents

タンパク質症を処置するための組成物および方法 Download PDF

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Publication number
JP2016503405A
JP2016503405A JP2015540845A JP2015540845A JP2016503405A JP 2016503405 A JP2016503405 A JP 2016503405A JP 2015540845 A JP2015540845 A JP 2015540845A JP 2015540845 A JP2015540845 A JP 2015540845A JP 2016503405 A JP2016503405 A JP 2016503405A
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Prior art keywords
gba1
aav
disease
protein
agent
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Abandoned
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JP2015540845A
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Japanese (ja)
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JP2016503405A5 (https=
Inventor
セルジオ・パブロ・サルディ
ラミヤ・シハブッディン
セン・チェン
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Genzyme Corp
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Genzyme Corp
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=49578599&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP2016503405(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of JP2016503405A publication Critical patent/JP2016503405A/ja
Publication of JP2016503405A5 publication Critical patent/JP2016503405A5/ja
Abandoned legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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 [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Psychiatry (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2015540845A 2012-11-05 2013-11-04 タンパク質症を処置するための組成物および方法 Abandoned JP2016503405A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261722434P 2012-11-05 2012-11-05
US61/722,434 2012-11-05
PCT/US2013/068242 WO2014071282A1 (en) 2012-11-05 2013-11-04 Compositions and methods for treating proteinopathies

Publications (2)

Publication Number Publication Date
JP2016503405A true JP2016503405A (ja) 2016-02-04
JP2016503405A5 JP2016503405A5 (https=) 2016-12-22

Family

ID=49578599

Family Applications (1)

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JP2015540845A Abandoned JP2016503405A (ja) 2012-11-05 2013-11-04 タンパク質症を処置するための組成物および方法

Country Status (22)

Country Link
US (1) US20150284472A1 (https=)
EP (1) EP2914281A1 (https=)
JP (1) JP2016503405A (https=)
KR (1) KR20150079751A (https=)
CN (1) CN104902923A (https=)
AR (1) AR093355A1 (https=)
AU (1) AU2013337354A1 (https=)
BR (1) BR112015009746A2 (https=)
CA (1) CA2889990A1 (https=)
CL (1) CL2015001157A1 (https=)
CR (1) CR20150216A (https=)
EA (1) EA201590880A1 (https=)
HK (1) HK1214521A1 (https=)
IL (1) IL238416A0 (https=)
MA (1) MA38144A1 (https=)
MX (1) MX2015005722A (https=)
PH (1) PH12015500879A1 (https=)
SG (1) SG11201502989XA (https=)
TN (1) TN2015000171A1 (https=)
TW (1) TW201427695A (https=)
WO (1) WO2014071282A1 (https=)
ZA (1) ZA201502618B (https=)

Cited By (9)

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JP2019524775A (ja) * 2016-08-03 2019-09-05 ユニヴァーシティ オブ サウス フロリダ 神経障害治療用のリーリン組成物
JP2019527821A (ja) * 2016-07-01 2019-10-03 セントジーン アーゲー ドラッガブルターゲットとしてのリゾＧｂ１の使用
JP2020537543A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
JP2020537544A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
JP2021500049A (ja) * 2017-10-23 2021-01-07 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. 神経変性疾患の遺伝子治療
JP2021501135A (ja) * 2017-10-26 2021-01-14 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 グルコセレブロシダーゼ及びイソファゴミンを含む製剤
JP2021059586A (ja) * 2016-04-29 2021-04-15 オーファザイム エー／エス グルコセレブロシダーゼ関連障害を治療するためのアリモクロモル
JP2022526021A (ja) * 2019-04-10 2022-05-20 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
JP2023545462A (ja) * 2020-10-14 2023-10-30 デナリ セラピューティクス インコーポレイテッド 前頭側頭型認知症を治療及びモニタリングするための方法

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AU2015317447B2 (en) 2014-09-15 2021-02-25 Zevra Denmark A/S Arimoclomol formulation
EP3292206B8 (en) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
SG10202100652VA (en) * 2015-07-21 2021-03-30 Univ Jefferson Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways
BR112018014870A2 (pt) * 2016-01-21 2018-12-11 Protein Dynamic Solutions Llc método e sistema para análise de dados espectrais
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
EP3765855A1 (en) * 2018-03-14 2021-01-20 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd Detection of phospho-serine 129 alpha-synuclein in blood cells as a biomarker for synucleinopathies
PL3953377T3 (pl) 2019-04-10 2026-02-16 Prevail Therapeutics, Inc. Terapie genowe zaburzeń lizosomalnych
EP3986412A4 (en) 2019-06-21 2023-11-15 The Broad Institute, Inc. Agents for reversing toxic proteinopathies
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WO2022066911A1 (en) * 2020-09-23 2022-03-31 Seelos Therapeutics, Inc. Compositions and methods for inhibiting alpha-synuclein aggregation
KR20230128462A (ko) 2020-11-19 2023-09-05 제브라 덴마크 에이/에스 아리모클로몰 시트레이트 및 이의 중간체를 제조하기 위한 공정
CN112569354B (zh) * 2020-12-29 2022-06-10 四川大学华西医院 tau蛋白及其基因作为药物靶点在制备治疗糖尿病药物中的应用
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Cited By (16)

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Publication number Priority date Publication date Assignee Title
JP2021059586A (ja) * 2016-04-29 2021-04-15 オーファザイム エー／エス グルコセレブロシダーゼ関連障害を治療するためのアリモクロモル
JP2019527821A (ja) * 2016-07-01 2019-10-03 セントジーン アーゲー ドラッガブルターゲットとしてのリゾＧｂ１の使用
JP2019524775A (ja) * 2016-08-03 2019-09-05 ユニヴァーシティ オブ サウス フロリダ 神経障害治療用のリーリン組成物
US12516091B2 (en) 2016-08-03 2026-01-06 University Of South Florida Reelin compositions for treatment of neurological disorders
JP7253800B2 (ja) 2016-08-03 2023-04-07 ユニヴァーシティ オブ サウス フロリダ 神経障害治療用のリーリン組成物
JP2023086740A (ja) * 2017-10-03 2023-06-22 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
JP2020537543A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
JP2020537544A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
JP7361037B2 (ja) 2017-10-03 2023-10-13 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
JP7254815B2 (ja) 2017-10-03 2023-04-10 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
JP2021500049A (ja) * 2017-10-23 2021-01-07 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. 神経変性疾患の遺伝子治療
JP7413256B2 (ja) 2017-10-23 2024-01-15 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患の遺伝子治療
JP2021501135A (ja) * 2017-10-26 2021-01-14 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 グルコセレブロシダーゼ及びイソファゴミンを含む製剤
JP2022526021A (ja) * 2019-04-10 2022-05-20 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
JP7664848B2 (ja) 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
JP2023545462A (ja) * 2020-10-14 2023-10-30 デナリ セラピューティクス インコーポレイテッド 前頭側頭型認知症を治療及びモニタリングするための方法

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EP2914281A1 (en) 2015-09-09
SG11201502989XA (en) 2015-05-28
CR20150216A (es) 2015-05-29
BR112015009746A2 (pt) 2017-08-15
CN104902923A (zh) 2015-09-09
CL2015001157A1 (es) 2015-10-16
TW201427695A (zh) 2014-07-16
WO2014071282A1 (en) 2014-05-08
ZA201502618B (en) 2016-01-27
AR093355A1 (es) 2015-06-03
CA2889990A1 (en) 2014-05-08
MX2015005722A (es) 2016-01-12
KR20150079751A (ko) 2015-07-08
IL238416A0 (en) 2015-06-30
EA201590880A1 (ru) 2015-09-30
US20150284472A1 (en) 2015-10-08
MA38144A1 (fr) 2018-08-31
PH12015500879A1 (en) 2015-06-29
AU2013337354A1 (en) 2015-05-21
HK1214521A1 (zh) 2016-07-29
TN2015000171A1 (fr) 2016-10-03

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