MX2015005722A - Composiciones y metodos para el tratamiento de proteinopatias. - Google Patents

Composiciones y metodos para el tratamiento de proteinopatias.

Info

Publication number
MX2015005722A
MX2015005722A MX2015005722A MX2015005722A MX2015005722A MX 2015005722 A MX2015005722 A MX 2015005722A MX 2015005722 A MX2015005722 A MX 2015005722A MX 2015005722 A MX2015005722 A MX 2015005722A MX 2015005722 A MX2015005722 A MX 2015005722A
Authority
MX
Mexico
Prior art keywords
methods
mammal
compositions
proteinopathy
administering
Prior art date
Application number
MX2015005722A
Other languages
English (en)
Spanish (es)
Inventor
Seng Cheng
Sergio Pablo Sardi
Lamya Shihabuddin
Original Assignee
Genzyme Corp
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=MX2015005722(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 MX2015005722A publication Critical patent/MX2015005722A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use 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)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Psychiatry (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MX2015005722A 2012-11-05 2013-11-04 Composiciones y metodos para el tratamiento de proteinopatias. MX2015005722A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261722434P 2012-11-05 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
MX2015005722A true MX2015005722A (es) 2016-01-12

Family

ID=49578599

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2015005722A MX2015005722A (es) 2012-11-05 2013-11-04 Composiciones y metodos para el tratamiento de proteinopatias.

Country Status (22)

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

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2484371B1 (en) 2008-06-26 2014-12-17 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
WO2016179497A1 (en) * 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. 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
EP3442530A1 (en) 2016-04-13 2019-02-20 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
SI3448382T1 (sl) 2016-04-29 2021-03-31 Orphazyme A/S, c/o COBIS A/S Arimoclomol za zdravljenje motenj, povezanih z glukocerebrozidazo
EP3264092A1 (en) * 2016-07-01 2018-01-03 Centogene AG Use of lyso-gb1 as druggable target
CN109715194B (zh) * 2016-08-03 2024-06-11 南佛罗里达大学 用于治疗神经障碍的颤蛋白组合物
BR112020006633A2 (pt) * 2017-10-03 2020-10-06 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
CN112553210B (zh) * 2017-10-03 2024-09-24 普利维尔治疗公司 用于溶酶体障碍的基因疗法
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN111819281B (zh) * 2017-10-23 2024-12-31 普利维尔治疗公司 用于神经变性疾病的基因疗法
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
KR20220006527A (ko) * 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
US11999974B2 (en) 2019-04-10 2024-06-04 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
EP3986412A4 (en) 2019-06-21 2023-11-15 The Broad Institute, Inc. Agents for reversing toxic proteinopathies
TW202214864A (zh) 2020-08-06 2022-04-16 應用醫學研究基金會 用於藉基因療法治療諸如阿茲海默症之Tau蛋白病變的病毒顆粒
WO2022066911A1 (en) * 2020-09-23 2022-03-31 Seelos Therapeutics, Inc. Compositions and methods for inhibiting alpha-synuclein aggregation
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
WO2022106614A1 (en) 2020-11-19 2022-05-27 Orphazyme A/S Processes for preparing arimoclomol citrate and intermediates thereof
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
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
EP4448762A1 (en) * 2021-12-17 2024-10-23 F. Hoffmann-La Roche AG Oligonucleotides capable of increasing glucocerebrosidase expression

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
GB9015198D0 (en) 1990-07-10 1990-08-29 Brien Caroline J O Binding substance
US6172197B1 (en) 1991-07-10 2001-01-09 Medical Research Council Methods for producing members of specific binding pairs
US5633425A (en) 1990-08-29 1997-05-27 Genpharm International, Inc. Transgenic non-human animals capable of producing heterologous antibodies
AU8507191A (en) 1990-08-29 1992-03-30 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
US5545806A (en) 1990-08-29 1996-08-13 Genpharm International, Inc. Ransgenic non-human animals for producing heterologous antibodies
US5661016A (en) 1990-08-29 1997-08-26 Genpharm International Inc. Transgenic non-human animals capable of producing heterologous antibodies of various isotypes
ES2113940T3 (es) 1990-12-03 1998-05-16 Genentech Inc Metodo de enriquecimiento para variantes de proteinas con propiedades de union alteradas.
PT1696031E (pt) 1991-12-02 2010-06-25 Medical Res Council Produção de auto-anticorpos a partir de reportórios de segmentos de anticorpo e exibidos em fagos
ATE304604T1 (de) 1993-06-24 2005-09-15 Frank L Graham Adenovirus vektoren für gentherapie
DE69434594T2 (de) 1993-10-25 2006-09-21 Canji, Inc., San Diego Rekombinante adenoviren-vektor und verfahren zur verwendung
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
ATE219517T1 (de) 1995-08-18 2002-07-15 Morphosys Ag Protein-/(poly)peptidbibliotheken
US6696248B1 (en) 1995-08-18 2004-02-24 Morphosys Ag Protein/(poly)peptide libraries
US6924123B2 (en) 1996-10-29 2005-08-02 Oxford Biomedica (Uk) Limited Lentiviral LTR-deleted vector
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ATE482275T1 (de) 1999-07-02 2010-10-15 Morphosys Ag Erzeugung spezifischer bindungspartner die an von genomischen dns-fragmenten oder ests kodierten (poly)peptiden binden
EP2369002A1 (en) 1999-08-09 2011-09-28 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 intrastrand 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
ATE555788T1 (de) * 2006-06-23 2012-05-15 Amicus Therapeutics Inc Verfahren zur behandlung neurologischer erkrankungen mittels verstärkung der beta - glucocerebrosidaseaktivität
US8454954B2 (en) * 2007-05-16 2013-06-04 The Brigham And Women's Hospital, Inc. Treatment of synucleinopathies
US9845327B2 (en) 2011-06-22 2017-12-19 The General Hospital Corporation Treatment of proteinopathies
AU2013240187A1 (en) 2012-03-28 2014-10-30 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Salicylic acid derivatives useful as glucocerebrosidase activators

Also Published As

Publication number Publication date
US20150284472A1 (en) 2015-10-08
KR20150079751A (ko) 2015-07-08
WO2014071282A1 (en) 2014-05-08
MA38144A1 (fr) 2018-08-31
IL238416A0 (en) 2015-06-30
AU2013337354A1 (en) 2015-05-21
JP2016503405A (ja) 2016-02-04
EP2914281A1 (en) 2015-09-09
PH12015500879A1 (en) 2015-06-29
SG11201502989XA (en) 2015-05-28
CL2015001157A1 (es) 2015-10-16
ZA201502618B (en) 2016-01-27
CA2889990A1 (en) 2014-05-08
TW201427695A (zh) 2014-07-16
BR112015009746A2 (pt) 2017-08-15
HK1214521A1 (zh) 2016-07-29
EA201590880A1 (ru) 2015-09-30
CN104902923A (zh) 2015-09-09
TN2015000171A1 (fr) 2016-10-03
CR20150216A (es) 2015-05-29
AR093355A1 (es) 2015-06-03

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