HK1214521A1 - 用於治療蛋白質病的組合物和方法 - Google Patents

用於治療蛋白質病的組合物和方法 Download PDF

Info

Publication number
HK1214521A1
HK1214521A1 HK16102536.9A HK16102536A HK1214521A1 HK 1214521 A1 HK1214521 A1 HK 1214521A1 HK 16102536 A HK16102536 A HK 16102536A HK 1214521 A1 HK1214521 A1 HK 1214521A1
Authority
HK
Hong Kong
Prior art keywords
methods
mammal
compositions
proteinopathy
administering
Prior art date
Application number
HK16102536.9A
Other languages
English (en)
Chinese (zh)
Inventor
Sergio Pablo SARDI
Lamya Shihabuddin
Seng Cheng
Original Assignee
Genzyme Corporation
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
Family has litigation
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=HK1214521(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of HK1214521A1 publication Critical patent/HK1214521A1/zh

Links

Classifications

    • 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

Landscapes

  • 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)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
HK16102536.9A 2012-11-05 2013-11-04 用於治療蛋白質病的組合物和方法 HK1214521A1 (zh)

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 (1)

Publication Number Publication Date
HK1214521A1 true HK1214521A1 (zh) 2016-07-29

Family

ID=49578599

Family Applications (1)

Application Number Title Priority Date Filing Date
HK16102536.9A HK1214521A1 (zh) 2012-11-05 2013-11-04 用於治療蛋白質病的組合物和方法

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=)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8540985B2 (en) 2008-06-26 2013-09-24 Orphazyme Aps Use of Hsp70 as a regulator of enzymatic activity
RU2013125923A (ru) 2010-11-30 2015-01-10 Орфазиме Апс СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70
US10709700B2 (en) 2014-09-15 2020-07-14 Orphazyme A/S Arimoclomol formulation
CA2985235A1 (en) * 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. Glucocerebrosidase gene therapy for parkinson's disease
WO2017015491A1 (en) * 2015-07-21 2017-01-26 Thomas Jefferson University Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways
CA3011719A1 (en) * 2016-01-21 2017-07-27 Protein Dynamic Solutions Llc Method and system for spectral data analysis
EP3442530A1 (en) 2016-04-13 2019-02-20 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
KR20210059796A (ko) * 2016-04-29 2021-05-25 오르파짐 에이/에스 글루코세레브로시다제 연관 장애를 치료하기 위한 아리모클로몰
EP3264092A1 (en) * 2016-07-01 2018-01-03 Centogene AG Use of lyso-gb1 as druggable target
AU2017306558B2 (en) * 2016-08-03 2024-09-19 University Of South Florida Reelin compositions for treatment of neurological disorders
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
AU2018346104B2 (en) * 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
BR112020008033A2 (pt) * 2017-10-23 2020-10-27 Prevail Therapeutics, Inc. terapias gênicas para doença neurodegenerativa
KR20240171186A (ko) * 2017-10-26 2024-12-06 다케다 야쿠힌 고교 가부시키가이샤 글루코세레브로시다제 및 이소파고민을 포함하는 제제
US20210041461A1 (en) * 2018-03-14 2021-02-11 Yissum Research Developmen Company Of The Hebrew University Of Jerusalem Ltd Detection of phospho-serine 129 alpha-synuclein in blood cells as a biomarker for synucleinopathies
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
CA3136004A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
WO2020257736A1 (en) 2019-06-21 2020-12-24 The Broad Institute, Inc. Agents for reversing toxic proteinopathies
CN116390773A (zh) 2020-08-06 2023-07-04 应用医学研究基金会 用于通过基因疗法治疗诸如阿尔茨海默病的Tau蛋白病的病毒颗粒
WO2022066911A1 (en) * 2020-09-23 2022-03-31 Seelos Therapeutics, Inc. Compositions and methods for inhibiting alpha-synuclein aggregation
WO2022082178A2 (en) * 2020-10-14 2022-04-21 Denali Therapeutics Inc. Methods for treating and monitoring frontotemporal dementia
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
CN118382697A (zh) * 2021-12-17 2024-07-23 豪夫迈·罗氏有限公司 能够增加葡糖脑苷脂酶表达的寡核苷酸
JP2024546887A (ja) * 2021-12-17 2024-12-26 ジェネンテック， インコーポレイテッド オリゴヌクレオチドｇｂａアゴニスト

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4676980A (en) 1985-09-23 1987-06-30 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Target specific cross-linked heteroantibodies
US5225539A (en) 1986-03-27 1993-07-06 Medical Research Council Recombinant altered antibodies and methods of making altered antibodies
US5892019A (en) 1987-07-15 1999-04-06 The United States Of America, As Represented By The Department Of Health And Human Services Production of a single-gene-encoded immunoglobulin
GB8823869D0 (en) 1988-10-12 1988-11-16 Medical Res Council Production of antibodies
US6172197B1 (en) 1991-07-10 2001-01-09 Medical Research Council Methods for producing members of specific binding pairs
GB9015198D0 (en) 1990-07-10 1990-08-29 Brien Caroline J O Binding substance
US5545806A (en) 1990-08-29 1996-08-13 Genpharm International, Inc. Ransgenic non-human animals for producing heterologous antibodies
ATE158021T1 (de) 1990-08-29 1997-09-15 Genpharm Int Produktion und nützung nicht-menschliche transgentiere zur produktion heterologe antikörper
US5661016A (en) 1990-08-29 1997-08-26 Genpharm International Inc. Transgenic non-human animals capable of producing heterologous antibodies of various isotypes
US5633425A (en) 1990-08-29 1997-05-27 Genpharm International, Inc. Transgenic non-human animals capable of producing heterologous antibodies
US5625126A (en) 1990-08-29 1997-04-29 Genpharm International, Inc. Transgenic non-human animals for producing heterologous antibodies
CA2095633C (en) 1990-12-03 2003-02-04 Lisa J. Garrard Enrichment method for variant proteins with altered binding properties
PT1024191E (pt) 1991-12-02 2008-12-22 Medical Res Council Produção de auto-anticorpos a partir de reportórios de segmentos de anticorpo e exibidos em fagos
EP0705344B8 (en) 1993-06-24 2006-05-10 Advec Inc. Adenovirus vectors for gene therapy
AU687117B2 (en) 1993-10-25 1998-02-19 Canji, Inc. Recombinant adenoviral vector and methods of use
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
US7264963B1 (en) 1995-08-18 2007-09-04 Morphosys Ag Protein(poly)peptide libraries
ES2176484T3 (es) 1995-08-18 2002-12-01 Morphosys Ag Bancos de proteinas/(poli)peptidos.
US6924123B2 (en) 1996-10-29 2005-08-02 Oxford Biomedica (Uk) Limited Lentiviral LTR-deleted vector
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO2001002588A2 (en) 1999-07-02 2001-01-11 Morphosys Ag Generation of specific binding partners binding to (poly)peptides encoded by genomic dna fragments or ests
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
WO2007150064A2 (en) * 2006-06-23 2007-12-27 Amicus Therapeutics, Inc. METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE
EP2154969B1 (en) * 2007-05-16 2015-11-18 The Brigham and Women's Hospital, Inc. Treatment of synucleinopathies
CA2840224C (en) 2011-06-22 2019-08-13 The General Hospital Corporation Treatment of proteinopathies
JP2015512913A (ja) 2012-03-28 2015-04-30 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ グルコセレブロシダーゼ活性剤として有用なサリチル酸誘導体

Also Published As

Publication number Publication date
JP2016503405A (ja) 2016-02-04
TW201427695A (zh) 2014-07-16
MX2015005722A (es) 2016-01-12
CR20150216A (es) 2015-05-29
EA201590880A1 (ru) 2015-09-30
PH12015500879A1 (en) 2015-06-29
IL238416A0 (en) 2015-06-30
AU2013337354A1 (en) 2015-05-21
US20150284472A1 (en) 2015-10-08
CL2015001157A1 (es) 2015-10-16
SG11201502989XA (en) 2015-05-28
AR093355A1 (es) 2015-06-03
EP2914281A1 (en) 2015-09-09
BR112015009746A2 (pt) 2017-08-15
ZA201502618B (en) 2016-01-27
CA2889990A1 (en) 2014-05-08
KR20150079751A (ko) 2015-07-08
CN104902923A (zh) 2015-09-09
WO2014071282A1 (en) 2014-05-08
TN2015000171A1 (fr) 2016-10-03
MA38144A1 (fr) 2018-08-31

Similar Documents

Publication Publication Date Title
HK1214521A1 (zh) 用於治療蛋白質病的組合物和方法
ZA201402049B (en) Compositions and methods for treating presbyopia, mild hyperopia, and irregular astigmatism
PH12014500767A1 (en) Compositions for the treatment of dry eye
HK1203356A1 (en) Pharmaceutical combinations comprising a thionucleotide analog
WO2012024620A3 (en) Autotaxin inhibitors and uses thereof
WO2009089494A3 (en) Pharmaceutical compositions
HUE047006T2 (hu) Anti-caprin-1 antitesteket tartalmazó gyógyászati kompozíció rák kezelésére és/vagy megelõzésére
WO2013188813A3 (en) Novel therapeutics for brain cancer
HK1220980A1 (zh) 用於治疗庞贝氏症的方法和组合物
HK1210962A1 (en) Methods for treatment of atherosclerosis
WO2014144606A3 (en) Methods of treating muscular dystrophy
NZ702087A (en) Pharmaceutical compositions and treatment of mastitis
EP2667882A1 (en) Methods and compositions for treating, reducing or preventing deterioration of the visual system of animals
EP2713715A4 (en) COMPOSITIONS AND METHODS OF TREATING MATERIALS WITH INSECTICIDES AND POTENTRICULENT AGENTS
ZA201306990B (en) Methods and compositions for treating, reducing, or preventing damage to the nervous system of animals
WO2014080286A3 (en) Methods and compositions for treating multiple sclerosis and related disorders
HK1218251A1 (zh) 用拉喹莫德治疗多发性硬化症
IL228666A (en) Pharmaceuticals for the prevention and / or treatment of HIV in humans
WO2013057592A3 (en) Treatment of sickle cell disease
WO2011097577A9 (en) Compositions and methods for treating or preventing retinal degeneration
EP3103446A4 (en) Pharmaceutical preparation for preventing and treating progressive myopia
WO2013033602A3 (en) Fatty acid amides, compositions and methods of use
WO2012131719A3 (en) A herbal formulation for treatment of silent estrus in animals
PT2793946T (pt) Composição tópica filmogénica e uso da mesma para tratar ou prevenir a onicofagia
PT2686005T (pt) Composições tópicas compreendendo diamina oxidase para o tratamento ou prevenção de doenças associadas com elevados níveis de histamina que envolvem um aumento na dor