CN109963576A - 用于治疗拉福拉病的方法和组合物 - Google Patents

用于治疗拉福拉病的方法和组合物 Download PDF

Info

Publication number
CN109963576A
CN109963576A CN201780069052.6A CN201780069052A CN109963576A CN 109963576 A CN109963576 A CN 109963576A CN 201780069052 A CN201780069052 A CN 201780069052A CN 109963576 A CN109963576 A CN 109963576A
Authority
CN
China
Prior art keywords
chimeric polypeptide
antibody
amino acid
seq
antigen
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
CN201780069052.6A
Other languages
English (en)
Chinese (zh)
Inventor
达斯汀·D·阿姆斯特朗
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.)
Valerion Therapeutics LLC
Original Assignee
Valerion Therapeutics LLC
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 Valerion Therapeutics LLC filed Critical Valerion Therapeutics LLC
Publication of CN109963576A publication Critical patent/CN109963576A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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

Landscapes

  • 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)
CN201780069052.6A 2016-09-09 2017-09-08 用于治疗拉福拉病的方法和组合物 Pending CN109963576A (zh)

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 (1)

Publication Number Publication Date
CN109963576A true CN109963576A (zh) 2019-07-02

Family

ID=61562328

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780069052.6A Pending CN109963576A (zh) 2016-09-09 2017-09-08 用于治疗拉福拉病的方法和组合物

Country Status (7)

Country Link
US (1) US10781433B2 (cg-RX-API-DMAC7.html)
EP (1) EP3509614A4 (cg-RX-API-DMAC7.html)
JP (1) JP2019535645A (cg-RX-API-DMAC7.html)
CN (1) CN109963576A (cg-RX-API-DMAC7.html)
AU (1) AU2017322483A1 (cg-RX-API-DMAC7.html)
CA (1) CA3036524A1 (cg-RX-API-DMAC7.html)
WO (1) WO2018049237A1 (cg-RX-API-DMAC7.html)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114502190A (zh) * 2019-08-22 2022-05-13 加利福尼亚大学董事会 用于治疗天使综合征的ube3a

Families Citing this family (5)

* 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
WO2025018822A1 (ko) 2023-07-20 2025-01-23 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물
KR102814185B1 (ko) 2023-07-20 2025-06-12 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130273026A1 (en) * 2012-04-12 2013-10-17 Integrative Enzymatics, Inc. Composition and method for modulating inflammatory molecules with amylase
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
CN105189543A (zh) * 2013-02-20 2015-12-23 瓦莱里昂治疗有限责任公司 治疗福布斯科里病的方法和组合物

Family Cites Families (3)

* 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
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
CA2995673A1 (en) * 2014-08-27 2016-03-03 Valerion Therapeutics, Llc Internalizing moieties for treatment of cancer

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130273026A1 (en) * 2012-04-12 2013-10-17 Integrative Enzymatics, Inc. Composition and method for modulating inflammatory molecules with amylase
CN105189543A (zh) * 2013-02-20 2015-12-23 瓦莱里昂治疗有限责任公司 治疗福布斯科里病的方法和组合物
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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114502190A (zh) * 2019-08-22 2022-05-13 加利福尼亚大学董事会 用于治疗天使综合征的ube3a

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
JP2019535645A (ja) 2019-12-12
CA3036524A1 (en) 2018-03-15

Similar Documents

Publication Publication Date Title
JP6709733B2 (ja) 内在化部分
US10781433B2 (en) Methods and compositions for treatment of Lafora disease
US20210040464A1 (en) Methods and compositions for treatment of polyglucosan disorders
AU2019236822B2 (en) GIPR antibody and GLP-1 fusion protein thereof, and pharmaceutical composition and application thereof
US20220411520A1 (en) Gipr antibody and fusion protein between same and glp-1, and pharmaceutical composition and application thereof
JP2016513114A (ja) フォーズス・コリ病の処置のための方法および組成物
KR20220093363A (ko) N-말단 scFv 다중특이적 결합 분자
AU2019337394B2 (en) Improved anti-FLT3 antigen binding proteins
JP2023541816A (ja) 自己免疫性疾患および癌を治療するための方法および組成物
TW201706303A (zh) 具有高親和性、結合性及專一性之結合轉形生長因子－β１的ｓｃＦｖ－Ｆｃ二聚體
JP2016027801A (ja) 改良された抗血清アルブミン結合変異体
KR20230154921A (ko) 골격근 질환을 치료하기 위한 조성물 및 방법
US20210395716A1 (en) Methods and compositions for treatment of protein aggregation-based disorders
CA3111462C (en) Improved anti-flt3 antigen binding proteins
WO2025049818A1 (en) Tnfr1 antagonists lacking agonist activity and uses thereof
TW202539735A (zh) 一種包含特異性結合ｄｌｌ３和ｃｄ３的雙特異性抗體的醫藥組成物
CN118119402A (zh) 工程化抗her2双特异性蛋白
WO2021004975A1 (en) MODIFIED IgA ANTIBODY PROTEINS

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20190702