MA38144A1 - Compositions et procédés pour le traitement de protéinopathies - Google Patents

Compositions et procédés pour le traitement de protéinopathies

Info

Publication number
MA38144A1
MA38144A1 MA38144A MA38144A MA38144A1 MA 38144 A1 MA38144 A1 MA 38144A1 MA 38144 A MA38144 A MA 38144A MA 38144 A MA38144 A MA 38144A MA 38144 A1 MA38144 A1 MA 38144A1
Authority
MA
Morocco
Prior art keywords
methods
mammal
proteinopathies
compositions
treatment
Prior art date
Application number
MA38144A
Other languages
English (en)
French (fr)
Inventor
Sergio Pablo Sardi
Lamya Shihabuddin
Seng Cheng
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=MA38144(A1) "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 MA38144A1 publication Critical patent/MA38144A1/fr

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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA38144A 2012-11-05 2013-11-04 Compositions et procédés pour le traitement de protéinopathies MA38144A1 (fr)

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
MA38144A1 true MA38144A1 (fr) 2018-08-31

Family

ID=49578599

Family Applications (1)

Application Number Title Priority Date Filing Date
MA38144A MA38144A1 (fr) 2012-11-05 2013-11-04 Compositions et procédés pour le traitement de protéinopathies

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 (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3578195T5 (da) 2008-06-26 2024-06-10 Zevra Denmark As Anvendelse af Hsp70 som regulator af enzymatisk aktivitet
WO2012072082A1 (en) 2010-11-30 2012-06-07 Orphazyme Aps Methods for increasing intracellular activity of hsp70
HUE054957T2 (hu) 2014-09-15 2021-10-28 Orphazyme As Arimoklomol készítése
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
WO2017127679A1 (en) * 2016-01-21 2017-07-27 Protein Dynamic Solutions Llc Method and system for spectral data analysis
WO2017178029A1 (en) 2016-04-13 2017-10-19 Orphazyme Aps Heat shock proteins and cholesterol homeostasis
CA3287846A1 (en) * 2016-04-29 2025-11-29 Zevra Denmark A/S Arimoclomol for treating glucocerebrosidase associated disorders
EP3264092A1 (en) * 2016-07-01 2018-01-03 Centogene AG Use of lyso-gb1 as druggable target
KR102523237B1 (ko) * 2016-08-03 2023-04-18 유니버시티 오브 사우스 플로리다 신경 장애의 치료를 위한 리일린 조성물
WO2019070891A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISORDERS
KR102700963B1 (ko) 2017-10-03 2024-09-02 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
IL278868B2 (en) * 2017-10-03 2025-06-01 Prevail Therapeutics Inc Gene therapies for lysosomal disorders
EP3701030A4 (en) * 2017-10-23 2022-04-20 Prevail Therapeutics, Inc. GENE THERAPIES FOR NEURODEGENERATIVE DISEASES
AR113797A1 (es) * 2017-10-26 2020-06-10 Shire Human Genetic Therapies Formulaciones que comprenden glucocerebrosidasa e isofagomina
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
US11999974B2 (en) 2019-04-10 2024-06-04 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
MX2021012184A (es) * 2019-04-10 2022-01-24 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.
EP3986412A4 (en) 2019-06-21 2023-11-15 The Broad Institute, Inc. Agents for reversing toxic proteinopathies
CA3181024A1 (en) 2020-08-06 2022-02-10 Fundacion Para La Investigacion Medica Aplicada Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy
CN116420078A (zh) * 2020-10-14 2023-07-11 戴纳立制药公司 用于治疗和监测额颞痴呆的方法
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
JP2024546925A (ja) * 2021-12-17 2024-12-26 エフ． ホフマン－ラ ロシュ アーゲー グルコセレブロシダーゼ発現を増加させることができるオリゴヌクレオチド
EP4448763A1 (en) * 2021-12-17 2024-10-23 F. Hoffmann-La Roche AG Oligonucleotide gba agonists

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
ES2108048T3 (es) 1990-08-29 1997-12-16 Genpharm Int Produccion y utilizacion de animales inferiores transgenicos capaces de producir anticuerpos heterologos.
US5633425A (en) 1990-08-29 1997-05-27 Genpharm International, Inc. Transgenic non-human animals capable of 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
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
DE69129154T2 (de) 1990-12-03 1998-08-20 Genentech, Inc., South San Francisco, Calif. Verfahren zur anreicherung von proteinvarianten mit geänderten bindungseigenschaften
ATE463573T1 (de) 1991-12-02 2010-04-15 Medimmune Ltd Herstellung von autoantikörpern auf phagenoberflächen ausgehend von antikörpersegmentbibliotheken
ES2249761T3 (es) 1993-06-24 2006-04-01 Advec Inc. Vectores de adenovirus para terapia genica.
SK283703B6 (sk) 1993-10-25 2003-12-02 Canji, Inc. Rekombinantný adenovírusový vektor a jeho použitie
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
US6828422B1 (en) 1995-08-18 2004-12-07 Morphosys Ag Protein/(poly)peptide libraries
WO1997008320A1 (en) 1995-08-18 1997-03-06 Morphosys Gesellschaft Für Proteinoptimierung Mbh Protein/(poly)peptide libraries
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
ATE403715T1 (de) 1999-08-09 2008-08-15 Targeted Genetics Corp Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
WO2001097829A2 (en) 2000-06-19 2001-12-27 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
JP5303458B2 (ja) * 2006-06-23 2013-10-02 アミークス セラピューティックス インコーポレイテッド β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法
ES2559859T3 (es) * 2007-05-16 2016-02-16 The Brigham And Women's Hospital, Inc. Tratamiento de sinucleinopatías
CN103764166B (zh) 2011-06-22 2017-10-24 通用医疗公司 蛋白质病的治疗
WO2013148333A1 (en) 2012-03-28 2013-10-03 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
WO2014071282A1 (en) 2014-05-08
SG11201502989XA (en) 2015-05-28
IL238416A0 (en) 2015-06-30
PH12015500879A1 (en) 2015-06-29
KR20150079751A (ko) 2015-07-08
CA2889990A1 (en) 2014-05-08
JP2016503405A (ja) 2016-02-04
AR093355A1 (es) 2015-06-03
TW201427695A (zh) 2014-07-16
HK1214521A1 (zh) 2016-07-29
ZA201502618B (en) 2016-01-27
EA201590880A1 (ru) 2015-09-30
BR112015009746A2 (pt) 2017-08-15
CN104902923A (zh) 2015-09-09
CR20150216A (es) 2015-05-29
CL2015001157A1 (es) 2015-10-16
AU2013337354A1 (en) 2015-05-21
TN2015000171A1 (fr) 2016-10-03
EP2914281A1 (en) 2015-09-09
US20150284472A1 (en) 2015-10-08
MX2015005722A (es) 2016-01-12

Similar Documents

Publication Publication Date Title
MA38144A1 (fr) Compositions et procédés pour le traitement de protéinopathies
TN2019000099A1 (en) Anti-lag-3 antibodies and compositions
SG10201804552WA (en) Nanoparticle compositions, formulations thereof, and uses therefor
MA32397B1 (fr) Agonistes mixtes basés sur gip destinés au traitement de troubles métaboliques et de l'obésité
MA41759A (fr) Inclusion de l'exon 2, induite par antisens, dans une alpha-glucosidase acide
MA32132B1 (fr) Utilisation de ranolazine pour traiter la douleur
MA39094A1 (fr) Polythérapie comprenant un inhibiteur de mdm2 et un ou plusieurs principes pharmaceutiquement actifs supplémentaires pour le traitement de cancers
MA40306A1 (fr) Méthodes et formulations pour le traitement de pathologies oculaires vasculaires
MA33743B1 (fr) Compositions comprenant du tramadol et du célécoxib dans le traitement de la douleur
MA33295B1 (fr) Nouvelle formulation de diclofénac
MA35873B1 (fr) Variants polypeptidiques du récepteur de l'activine, seuls ou en combinaison avec une chimiothérapie, et leurs utilisations
MA43283B1 (fr) Composition pour le traitement du cancer
MA38478A1 (fr) Anticorps anti-pac1 humains
MA37946A1 (fr) Traitement de la polyarthrite rhumatoïde
NZ702087A (en) Pharmaceutical compositions and treatment of mastitis
WO2020072126A3 (en) Modulating ptpn2 to increase immune responses and perturbing gene expression in hematopoietic stem cell lineages
MA37777B1 (fr) Préparation pharmaceutique
EP3309555A3 (en) Methods for preventing or treating disorders by increasing bioavailability of iron and related pharmaceutical formulation
IN2014DN08199A (enExample)
MX2018005352A (es) Metodos y composiciones para el tratamiento de amiloidosis.
MA34815B1 (fr) Compositions comprenant un inhibiteur de pi3k et un inhibiteur de mek et leur utilisation pour le traitement du cancer
FR2972330B1 (fr) Composition nutraceutique pour limiter l'absorption de lipides alimentaires et pour induire une perte de poids comprenant comme agent actif au moins un extrait de carotte.
MA38369B1 (fr) Peptides et compositions pour le traitement d'une lesion de l'articulation
WO2008073466A3 (en) Alpha b-crystallin as a therapy for inflammation
MA39228A1 (fr) Compositions à utiliser pour le traitement d'états allergiques