JP2019535645A - ラフォラ病の処置のための方法及び組成物 - Google Patents

ラフォラ病の処置のための方法及び組成物 Download PDF

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JP2019535645A
JP2019535645A JP2019513898A JP2019513898A JP2019535645A JP 2019535645 A JP2019535645 A JP 2019535645A JP 2019513898 A JP2019513898 A JP 2019513898A JP 2019513898 A JP2019513898 A JP 2019513898A JP 2019535645 A JP2019535645 A JP 2019535645A
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chimeric polypeptide
antibody
amino acid
seq
cell
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JP2019535645A5 (enExample
Inventor
ダスティン ディー． アームストロング，
ダスティン ディー． アームストロング，
Original Assignee
バレリオン セラピューティクス， エルエルシー
バレリオン セラピューティクス， エルエルシー
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Publication of JP2019535645A publication Critical patent/JP2019535645A/ja
Publication of JP2019535645A5 publication Critical patent/JP2019535645A5/ja
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    • 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
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • C12N9/2411—Amylases
    • C12N9/2414—Alpha-amylase (3.2.1.1.)
    • 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
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/44—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material not provided for elsewhere, e.g. haptens, metals, DNA, RNA, amino acids
    • 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)
    • 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
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55—Fab or Fab'
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hematology (AREA)
  • Psychiatry (AREA)
  • Diabetes (AREA)
  • Hospice & Palliative Care (AREA)
  • Obesity (AREA)
  • Epidemiology (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2019513898A 2016-09-09 2017-09-08 ラフォラ病の処置のための方法及び組成物 Pending JP2019535645A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662385656P 2016-09-09 2016-09-09
US62/385,656 2016-09-09
US201762553048P 2017-08-31 2017-08-31
US62/553,048 2017-08-31
PCT/US2017/050787 WO2018049237A1 (en) 2016-09-09 2017-09-08 Methods and compositions for treatment of lafora disease

Publications (2)

Publication Number Publication Date
JP2019535645A true JP2019535645A (ja) 2019-12-12
JP2019535645A5 JP2019535645A5 (enExample) 2020-10-22

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JP2019513898A Pending JP2019535645A (ja) 2016-09-09 2017-09-08 ラフォラ病の処置のための方法及び組成物

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US (1) US10781433B2 (enExample)
EP (1) EP3509614A4 (enExample)
JP (1) JP2019535645A (enExample)
CN (1) CN109963576A (enExample)
AU (1) AU2017322483A1 (enExample)
CA (1) CA3036524A1 (enExample)
WO (1) WO2018049237A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20250018363A (ko) 2023-07-20 2025-02-05 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물
KR20250080845A (ko) 2023-07-20 2025-06-05 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6178785B2 (ja) 2011-04-01 2017-08-09 イェール ユニバーシティーＹａｌｅ Ｕｎｉｖｅｒｓｉｔｙ 細胞浸透性抗ｄｎａ抗体およびｄｎａ修復を阻害するためのその使用
EP3471777A1 (en) 2016-06-15 2019-04-24 Yale University Antibody-mediated autocatalytic, targeted delivery of nanocarriers to tumors
US12486318B2 (en) 2016-06-15 2025-12-02 Yale University Anti-guanosine antibody as a molecular delivery vehicle
AU2020334924A1 (en) * 2019-08-22 2022-04-07 The Regents Of The University Of California UBE3A for the treatment of Angelman syndrome

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015106290A1 (en) * 2014-01-13 2015-07-16 Valerion Therapeutics, Llc Internalizing moieties
WO2015192092A1 (en) * 2014-06-13 2015-12-17 Valerion Therapeutics, Llc Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
WO2016033324A1 (en) * 2014-08-27 2016-03-03 Valerion Therapeutics, Llc Internalizing moieties for treatment of cancer
JP2016513114A (ja) * 2013-02-20 2016-05-12 バレリオン セラピューティクス， エルエルシー フォーズス・コリ病の処置のための方法および組成物
JP2016514096A (ja) * 2013-02-20 2016-05-19 バレリオン セラピューティクス， エルエルシー ポンぺ病の処置のための方法および組成物

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2010260145B2 (en) * 2009-06-15 2015-08-20 Valerion Therapeutics, Llc Methods and compositions for treatment of Myotubular Myopathy using chimeric polypeptides comprising myotubularin 1 (MTM1) polypeptides
US20130273026A1 (en) * 2012-04-12 2013-10-17 Integrative Enzymatics, Inc. Composition and method for modulating inflammatory molecules with amylase

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016513114A (ja) * 2013-02-20 2016-05-12 バレリオン セラピューティクス， エルエルシー フォーズス・コリ病の処置のための方法および組成物
JP2016514096A (ja) * 2013-02-20 2016-05-19 バレリオン セラピューティクス， エルエルシー ポンぺ病の処置のための方法および組成物
WO2015106290A1 (en) * 2014-01-13 2015-07-16 Valerion Therapeutics, Llc Internalizing moieties
WO2015192092A1 (en) * 2014-06-13 2015-12-17 Valerion Therapeutics, Llc Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
WO2016033324A1 (en) * 2014-08-27 2016-03-03 Valerion Therapeutics, Llc Internalizing moieties for treatment of cancer

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20250018363A (ko) 2023-07-20 2025-02-05 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물
KR20250080845A (ko) 2023-07-20 2025-06-05 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물

Also Published As

Publication number Publication date
EP3509614A4 (en) 2020-04-22
US20190316104A1 (en) 2019-10-17
US10781433B2 (en) 2020-09-22
AU2017322483A1 (en) 2019-04-04
EP3509614A1 (en) 2019-07-17
WO2018049237A1 (en) 2018-03-15
CA3036524A1 (en) 2018-03-15
CN109963576A (zh) 2019-07-02

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