AU2012272815B2 - Treatment of proteinopathies - Google Patents

Treatment of proteinopathies Download PDF

Info

Publication number
AU2012272815B2
AU2012272815B2 AU2012272815A AU2012272815A AU2012272815B2 AU 2012272815 B2 AU2012272815 B2 AU 2012272815B2 AU 2012272815 A AU2012272815 A AU 2012272815A AU 2012272815 A AU2012272815 A AU 2012272815A AU 2012272815 B2 AU2012272815 B2 AU 2012272815B2
Authority
AU
Australia
Prior art keywords
lysosomal
polypeptide
disease
synuclein
diseases
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.)
Active
Application number
AU2012272815A
Other languages
English (en)
Other versions
AU2012272815A1 (en
Inventor
Dmitri KRAINC
Joseph R. Mazzulli
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.)
General Hospital Corp
Original Assignee
General Hospital 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
Application filed by General Hospital Corp filed Critical General Hospital Corp
Publication of AU2012272815A1 publication Critical patent/AU2012272815A1/en
Application granted granted Critical
Publication of AU2012272815B2 publication Critical patent/AU2012272815B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • 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
    • A61P25/24—Antidepressants
    • 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
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P3/02—Nutrients, e.g. vitamins, minerals
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • A61P3/12—Drugs for disorders of the metabolism for electrolyte homeostasis
    • A61P3/14—Drugs for disorders of the metabolism for electrolyte homeostasis for calcium homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/06—Antiarrhythmics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • C12Q1/44—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase involving esterase
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/573—Immunoassay; Biospecific binding assay; Materials therefor for enzymes or isoenzymes
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00—Screening for compounds of potential therapeutic value
    • G01N2500/10—Screening for compounds of potential therapeutic value involving cells
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Urology & Nephrology (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Analytical Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
  • Cell Biology (AREA)
  • Cardiology (AREA)
  • Endocrinology (AREA)
  • Obesity (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pain & Pain Management (AREA)
AU2012272815A 2011-06-22 2012-06-22 Treatment of proteinopathies Active AU2012272815B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161499930P 2011-06-22 2011-06-22
US61/499,930 2011-06-22
PCT/US2012/043732 WO2012177997A1 (en) 2011-06-22 2012-06-22 Treatment of proteinopathies

Publications (2)

Publication Number Publication Date
AU2012272815A1 AU2012272815A1 (en) 2014-01-16
AU2012272815B2 true AU2012272815B2 (en) 2017-09-07

Family

ID=47422968

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2012272815A Active AU2012272815B2 (en) 2011-06-22 2012-06-22 Treatment of proteinopathies

Country Status (7)

Country Link
US (1) US9845327B2 (enExample)
EP (1) EP2723384B1 (enExample)
JP (1) JP6061922B2 (enExample)
CN (1) CN103764166B (enExample)
AU (1) AU2012272815B2 (enExample)
CA (1) CA2840224C (enExample)
WO (1) WO2012177997A1 (enExample)

Families Citing this family (98)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5959795B2 (ja) 2006-08-18 2016-08-02 アーメイゲン・テクノロジーズ・インコーポレイテッドＡｒｍａｇｅｎ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． 血液脳関門送達のための物質
EP2182980A4 (en) 2007-07-27 2012-04-18 Armagen Technologies Inc METHOD AND COMPOSITIONS FOR INCREASED ALPHA IDURONIDASE ACTIVITY IN THE CNS
HRP20190690T1 (hr) 2009-10-09 2019-06-28 Armagen, Inc. Postupci i pripravci za povećanje aktiviteta iduronat-2-sulfataze u centralnom nervnom sustavu
CA2834049C (en) 2011-04-25 2018-05-22 Usher Iii Initiative, Inc. Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome
JP2014517079A (ja) 2011-06-22 2014-07-17 バーテックス ファーマシューティカルズ インコーポレイテッド Ａｔｒキナーゼ阻害剤として有用な化合物
EP3378535B1 (en) 2011-10-28 2023-01-04 Prothena Biosciences Limited Humanized antibodies that recognize alpha-synuclein
KR101998818B1 (ko) 2011-11-03 2019-07-10 한민규 무선 통신 장치의 장치 식별 정보를 이용한 컨텐츠 표시 방법, 컨텐츠 제공 방법, 상기 컨텐츠 표시 방법을 수행하는 사용자 단말 및 상기 컨텐츠 제공 방법을 수행하는 컨텐츠 제공 시스템
US20140323581A1 (en) * 2011-12-05 2014-10-30 The Trustees Of Columbia University In The City Of New York Transcriptome wiring analysis in parkinson's disease and uses thereof
KR102086061B1 (ko) 2012-01-27 2020-03-11 프로테나 바이오사이언시즈 리미티드 알파-시누클레인을 인식하는 인간화된 항체
US8940742B2 (en) 2012-04-10 2015-01-27 Infinity Pharmaceuticals, Inc. Heterocyclic compounds and uses thereof
AU2013273473B2 (en) * 2012-06-06 2016-05-26 Consiglio Nazionale Delle Ricerche Allosteric chaperones and uses thereof
JOP20130273B1 (ar) 2012-09-11 2021-08-17 Genzyme Corp مثبطات انزيم (سينثاز) غلوكوسيل سيراميد
UA118441C2 (uk) 2012-10-08 2019-01-25 Протена Біосаєнсиз Лімітед Антитіло, що розпізнає альфа-синуклеїн
US9227976B2 (en) 2012-10-25 2016-01-05 Usher Iii Initiative, Inc. Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome
MA38144A1 (fr) 2012-11-05 2018-08-31 Genzyme Corp Compositions et procédés pour le traitement de protéinopathies
SMT202000713T1 (it) 2012-12-07 2021-03-15 Vertex Pharma Pirazolo [1,5-a] pirimidine utili come inibitori dell'atr chinasi per il trattamento di malattie del cancro
US8969360B2 (en) 2013-03-15 2015-03-03 Vertex Pharmaceuticals Incorporated Compounds useful as inhibitors of ATR kinase
US9663519B2 (en) 2013-03-15 2017-05-30 Vertex Pharmaceuticals Incorporated Compounds useful as inhibitors of ATR kinase
EP2970289A1 (en) 2013-03-15 2016-01-20 Vertex Pharmaceuticals Inc. Compounds useful as inhibitors of atr kinase
WO2014204956A1 (en) * 2013-06-18 2014-12-24 University Of North Carolina At Pembroke Materials for positive cathepsin b modulation and methods of use for treating mild cognitive impairment (mci), early dementia, a-synucleinopathy, traumatic brain injury, cardiomyopathy, eye disease and skin damage
US10513555B2 (en) 2013-07-04 2019-12-24 Prothena Biosciences Limited Antibody formulations and methods
SG10201902074UA (en) 2013-10-04 2019-04-29 Infinity Pharmaceuticals Inc Heterocyclic compounds and uses thereof
WO2015051241A1 (en) 2013-10-04 2015-04-09 Infinity Pharmaceuticals, Inc. Heterocyclic compounds and uses thereof
ES2768678T3 (es) 2013-12-06 2020-06-23 Vertex Pharma Compuesto de 2-amino-6-fluoro-N-[5-fluoro-piridin-3-il]pirazolo[1,5-a]pirimidin-3-carboxamida útil como inhibidor de la ATR quinasa, su preparación, diferentes formas sólidas y derivados radiomarcados de las mismas
EP3105589B1 (en) 2014-02-14 2021-08-11 betaSENSE GmbH Biosensor for conformation and secondary structure analysis
CN113616656B (zh) 2014-03-19 2023-02-17 无限药品股份有限公司 用于治疗PI3K-γ介导的障碍的杂环化合物
WO2015155694A1 (en) 2014-04-08 2015-10-15 Prothena Biosciences Limited Blood-brain barrier shuttles containing antibodies recognizing alpha-synuclein
SG10201902206QA (en) 2014-06-05 2019-04-29 Vertex Pharma Radiolabelled derivatives of a 2-amino-6-fluoro-n-[5-fluoro-pyridin-3-yl]- pyrazolo[1,5-a]pyrimidin-3-carboxamide compound useful as atr kinase inhibitor, the preparation of said compound and different solid forms thereof
AU2015277212B2 (en) 2014-06-17 2020-07-02 Vertex Pharmaceuticals Incorporated Method for treating cancer using a combination of Chk1 and ATR inhibitors
CN105330579B (zh) * 2014-08-08 2019-08-30 杭州雷索药业有限公司 依泽替米贝及其衍生物在治疗或预防癫痫中的应用
WO2016054491A1 (en) 2014-10-03 2016-04-07 Infinity Pharmaceuticals, Inc. Heterocyclic compounds and uses thereof
PT3215511T (pt) 2014-11-06 2024-05-22 Bial R&D Invest S A Pirazolo(1,5-a)pirimidinas substituídas e a sua utilização no tratamento de distúrbios médicos
US20170333435A1 (en) 2014-11-06 2017-11-23 Lysosomal Therapeutics Inc. Substituted imidazo[1,5-a]pyrimidines and their use in the treatment of medical disorders
EP3215509B1 (en) 2014-11-06 2020-02-26 Lysosomal Therapeutics Inc. Substituted pyrrolo[1,2-a]pyrimidines and their use in the treatment of medical disorders
US10441574B2 (en) 2014-11-13 2019-10-15 Washington University Treatment for wolfram syndrome and other endoplasmic reticulum stress disorders
US20170354666A1 (en) * 2014-11-19 2017-12-14 Rush University Medical Center Compositions and methods for treating lysosomal disorders
TW201642855A (zh) * 2015-03-10 2016-12-16 健臻公司 用於治療蛋白質病變之方法
US10016514B2 (en) * 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
JP6924155B2 (ja) 2015-06-11 2021-08-25 アッシャー・サード・イニシアティブ・インコーポレイテッド プロテオパシーを治療又は予防する方法
TWI617573B (zh) * 2015-08-06 2018-03-11 中央研究院 用於酵素置換療法的改造酵素
WO2017040971A1 (en) * 2015-09-03 2017-03-09 Biomarin Pharmaceutical Inc. Methods of using inhibitors of pikfyve for the treatment of lysosomal storage disorders and neurodegenerative diseases
AU2016322552B2 (en) 2015-09-14 2021-03-25 Infinity Pharmaceuticals, Inc. Solid forms of isoquinolinone derivatives, process of making, compositions comprising, and methods of using the same
RU2768621C1 (ru) 2015-09-30 2022-03-24 Вертекс Фармасьютикалз Инкорпорейтед Способ лечения рака с использованием комбинации повреждающих днк средств и ингибиторов atr
US20190018000A1 (en) 2016-01-12 2019-01-17 Cedars-Sinai Medical Center A method of non destructive monitoring of biological processes in microfluidic tissue culture systems
CN105534977A (zh) * 2016-01-14 2016-05-04 山东大学 T型钙离子通道抑制剂nnc55-0396在制备抗神经退行性疾病药物中的应用
EP3411470A4 (en) 2016-02-01 2019-10-09 Emulate, Inc. SYSTEMS AND METHOD FOR BREEDING DARM CELLS IN MICROFLUIDIC DEVICES
WO2017161116A1 (en) 2016-03-17 2017-09-21 Infinity Pharmaceuticals, Inc. Isotopologues of isoquinolinone and quinazolinone compounds and uses thereof as pi3k kinase inhibitors
EP3433623A2 (en) * 2016-03-25 2019-01-30 Genzyme Corporation Biomarkers of proteopathies and uses thereof
MX2018012208A (es) 2016-04-06 2019-03-28 Lysosomal Therapeutics Inc Compuestos a base de imidazo [1,5-a] pirimidinil carboxamida y su uso en el tratamiento de trastornos médicos.
WO2017176962A1 (en) * 2016-04-06 2017-10-12 Lysosomal Therapeutics, Inc. Pyrrolo[1,2-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders
EP3440080A4 (en) 2016-04-06 2020-01-22 Lysosomal Therapeutics Inc. PYRAZOLO [1,5-A] PYRIMIDINYL CARBOXAMIDE COMPOUNDS AND THEIR USE IN THE TREATMENT OF MEDICAL DISORDERS
JP7164774B2 (ja) 2016-05-05 2022-11-02 ビアル－アール・アンド・ディ・インベストメンツ・ソシエダーデ・アノニマ 置換イミダゾ［１，２－ｂ］ピリダジン、置換イミダゾ［１，５－ｂ］ピリダジン、関連化合物、および医学的障害の治療におけるその使用
US11345698B2 (en) 2016-05-05 2022-05-31 Bial—R&D Investments, S.A. Substituted imidazo[1,2-a]pyridines, substituted imidazo[1,2-a]pyrazines, related compounds, and their use in the treatment of medical disorders
WO2017214269A1 (en) 2016-06-08 2017-12-14 Infinity Pharmaceuticals, Inc. Heterocyclic compounds and uses thereof
EP3318277A1 (en) * 2016-11-04 2018-05-09 Institut du Cerveau et de la Moelle Epiniere-ICM Inhibitors of glucosylceramide synthase for the treatment of motor neuron diseases
CA3043709A1 (en) 2016-11-14 2018-05-17 Berg Llc Methods for treating parkinson's disease
ES2834484T3 (es) 2016-11-21 2021-06-17 Univ Ruhr Bochum Método para la preselección de fármacos para enfermedades de plegamiento incorrecto de proteínas
CA3048357A1 (en) 2016-12-29 2018-07-05 Minoryx Therapeutics S.L. Heteroaryl compounds and their use
US11913022B2 (en) 2017-01-25 2024-02-27 Cedars-Sinai Medical Center In vitro induction of mammary-like differentiation from human pluripotent stem cells
US11767513B2 (en) 2017-03-14 2023-09-26 Cedars-Sinai Medical Center Neuromuscular junction
US11414648B2 (en) 2017-03-24 2022-08-16 Cedars-Sinai Medical Center Methods and compositions for production of fallopian tube epithelium
AU2018312441B2 (en) * 2017-08-02 2024-10-17 Stressmarq Biosciences Inc. Antibody binding active alpha-synuclein
JP7437855B2 (ja) * 2017-09-07 2024-02-26 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア 遺伝性シスタチンｃアミロイド血管症（ｈｃｃａａ）および異常なアミロイド沈着を伴う他の神経変性障害の治療のための組成物および方法
WO2019070577A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. FUSION PROTEINS COMPRISING SUBSTITUTE ENZYM THERAPY ENZYMES
US11247987B2 (en) 2017-10-06 2022-02-15 Forma Therapeutics, Inc. Inhibiting ubiquitin specific peptidase 30
MX2020007501A (es) * 2018-02-02 2020-10-16 Enzyvant Therapeutics Gmbh Metodos para tratar la enfermedad de farber.
US12161676B2 (en) 2018-03-23 2024-12-10 Cedars-Sinai Medical Center Methods of use of islet cells
WO2019195800A1 (en) 2018-04-06 2019-10-10 Cedars-Sinai Medical Center Novel differentiation technique to generate dopaminergic neurons from induced pluripotent stem cells
WO2019195798A1 (en) 2018-04-06 2019-10-10 Cedars-Sinai Medical Center Human pluripotent stem cell derived neurodegenerative disease models on a microfluidic chip
WO2019200286A1 (en) 2018-04-13 2019-10-17 University Of Massachusetts Bicistronic aav vectors encoding hexosaminidase alpha and beta-subunits and uses thereof
WO2019212691A1 (en) * 2018-04-30 2019-11-07 Cedars-Sinai Medical Center Diagnostic and drug screening for molecular signatures of early onset sporadic parkinson's disease
IL278291B2 (en) 2018-05-17 2023-10-01 Forma Therapeutics Inc Fused bicyclic compounds useful as ubiquitin-specific peptidase 30 inhibitors
US20210252175A1 (en) * 2018-06-08 2021-08-19 Ac Immune Sa Novel compounds for diagnosis
WO2020023390A1 (en) 2018-07-25 2020-01-30 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
WO2020072873A1 (en) * 2018-10-05 2020-04-09 University Of Massachusetts Raav vectors for the treatment of gm1 and gm2 gangliosidosis
LT3860989T (lt) 2018-10-05 2023-06-12 Forma Therapeutics, Inc. Sulieti pirolinai, kurie veikia kaip ubikvitinui specifinės proteazės 30 (ups30) inhibitoriai
WO2020118056A1 (en) * 2018-12-05 2020-06-11 Washington University Methods of detecting, preventing, reversing, and treating neurological diseases
WO2020146359A1 (en) * 2019-01-08 2020-07-16 Armagen, Inc. Methods and compositions for increasing galactosidase beta-1 activity in the cns
US20220016092A1 (en) * 2019-02-04 2022-01-20 Genzyme Corporation Methods for treating symptoms and disorders associated with lysosomal storage diseases
CN113573708A (zh) * 2019-02-07 2021-10-29 阿尔萨泰克公司 艾莫西平多价衍生物
AU2020245415B2 (en) * 2019-03-22 2025-06-26 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal stoarge disorders
CN114761021A (zh) * 2019-04-29 2022-07-15 托马斯杰斐逊大学 用于治疗神经变性障碍的方法
EP3986412A4 (en) 2019-06-21 2023-11-15 The Broad Institute, Inc. AGENTS TO REVERSE TOXIC PROTEINOPATHIES
US20220354827A1 (en) * 2019-06-28 2022-11-10 The Trustees Of The University Of Pennsylvania Ntranasal dantrolene administration for treatment of alzheimer's disease
JP7539382B2 (ja) * 2019-07-29 2024-08-23 武田薬品工業株式会社 複素環化合物
CN110579457B (zh) * 2019-09-20 2021-11-02 郑州大学第一附属医院 波形蛋白特异响应性荧光探针及其制备方法和应用
EP4093263A4 (en) * 2020-01-23 2024-03-27 Retispec Inc. SYSTEMS AND METHODS FOR DISEASE DIAGNOSIS
BR112022014553A2 (pt) 2020-02-03 2022-09-20 Genzyme Corp Métodos para tratamento de sintomas neurológicos associados a doenças do armazenamento lisossômic
US20240261250A1 (en) * 2020-03-31 2024-08-08 Nuo-Beta Pharmaceutical Technology (Shanghai) Co., Ltd. Applications of p14k inhibitor in intracellular protein misfolding-related diseases and lysosomal storage diseases
CN113720812A (zh) * 2020-05-26 2021-11-30 重庆福莱鲨生物技术有限公司 一种测定蛋白溶解度的方法
MX2023001014A (es) 2020-07-24 2023-03-01 Genzyme Corp Composiciones farmaceuticas que comprenden venglustat.
US20230416224A1 (en) 2020-11-19 2023-12-28 Zevra Denmark A/S Processes for preparing arimoclomol citrate and intermediates thereof
US20240148722A1 (en) * 2021-02-20 2024-05-09 Biohaven Therapeutics Ltd. Small molecules that bind to tdp-43 for the treatment of als and related disorders
CN113332267A (zh) * 2021-03-31 2021-09-03 中山大学孙逸仙纪念医院 苯丁酸钠在制备预防或治疗帕金森病的药物中的应用
WO2022255893A1 (en) * 2021-06-02 2022-12-08 Qatar Foundation For Education, Science And Community Development Methods for diagnosing and differentiating synucleinopathies
CA3232577A1 (en) * 2021-09-20 2023-03-23 Alexander SHTILBANS Combination therapy for neurodegenerative diseases
CN116392490A (zh) * 2021-12-27 2023-07-07 中国科学院广州生物医药与健康研究院 氟桂利嗪的用途以及控制细胞内线粒体数量的方法
CN119236096A (zh) * 2024-10-11 2025-01-03 苏州大学 Grp94蛋白作为递药靶点在帕金森病药物载体构建中的应用

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007140212A2 (en) * 2006-05-24 2007-12-06 Amicus Therapeutics, Inc. Tartrate salt of isofagomine and methods of use
EA200700427A1 (ru) * 2004-08-09 2008-02-28 Элан Фармасьютикалз, Инк. Предупреждение и лечение синуклеинопатических и амилоидогенных заболеваний
WO2012078855A1 (en) * 2010-12-08 2012-06-14 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Substituted pyrazolopyrimidines as glucocerebrosidase activators

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4270537A (en) 1979-11-19 1981-06-02 Romaine Richard A Automatic hypodermic syringe
US4596556A (en) 1985-03-25 1986-06-24 Bioject, Inc. Hypodermic injection apparatus
US4902505A (en) 1986-07-30 1990-02-20 Alkermes Chimeric peptides for neuropeptide delivery through the blood-brain barrier
CA1283827C (en) 1986-12-18 1991-05-07 Giorgio Cirelli Appliance for injection of liquid formulations
GB8704027D0 (en) 1987-02-20 1987-03-25 Owen Mumford Ltd Syringe needle combination
US4940460A (en) 1987-06-19 1990-07-10 Bioject, Inc. Patient-fillable and non-invasive hypodermic injection device assembly
US4941880A (en) 1987-06-19 1990-07-17 Bioject, Inc. Pre-filled ampule and non-invasive hypodermic injection device assembly
US4790824A (en) 1987-06-19 1988-12-13 Bioject, Inc. Non-invasive hypodermic injection device
US5004697A (en) 1987-08-17 1991-04-02 Univ. Of Ca Cationized antibodies for delivery through the blood-brain barrier
US5994392A (en) 1988-02-26 1999-11-30 Neuromedica, Inc. Antipsychotic prodrugs comprising an antipsychotic agent coupled to an unsaturated fatty acid
US6107499A (en) 1988-02-26 2000-08-22 Neuromedica, Inc. Dopamine analog amide
US4933324A (en) 1988-02-26 1990-06-12 Shashoua Victor E Fatty acid-neuroactive drug conjugate as a prodrug
US4939174A (en) 1988-02-26 1990-07-03 Shashoua Victor E Appetite suppression with dopamine-fatty acid conjugates
US5339163A (en) 1988-03-16 1994-08-16 Canon Kabushiki Kaisha Automatic exposure control device using plural image plane detection areas
FR2638359A1 (fr) 1988-11-03 1990-05-04 Tino Dalto Guide de seringue avec reglage de la profondeur de penetration de l'aiguille dans la peau
US5527527A (en) 1989-09-07 1996-06-18 Alkermes, Inc. Transferrin receptor specific antibody-neuropharmaceutical agent conjugates
US5977307A (en) 1989-09-07 1999-11-02 Alkermes, Inc. Transferrin receptor specific ligand-neuropharmaceutical agent fusion proteins
US5312335A (en) 1989-11-09 1994-05-17 Bioject Inc. Needleless hypodermic injection device
US5064413A (en) 1989-11-09 1991-11-12 Bioject, Inc. Needleless hypodermic injection device
US5112596A (en) 1990-04-23 1992-05-12 Alkermes, Inc. Method for increasing blood-brain barrier permeability by administering a bradykinin agonist of blood-brain barrier permeability
US5268164A (en) 1990-04-23 1993-12-07 Alkermes, Inc. Increasing blood-brain barrier permeability with permeabilizer peptides
US5190521A (en) 1990-08-22 1993-03-02 Tecnol Medical Products, Inc. Apparatus and method for raising a skin wheal and anesthetizing skin
US5527288A (en) 1990-12-13 1996-06-18 Elan Medical Technologies Limited Intradermal drug delivery device and method for intradermal delivery of drugs
GB9118204D0 (en) 1991-08-23 1991-10-09 Weston Terence E Needle-less injector
SE9102652D0 (sv) 1991-09-13 1991-09-13 Kabi Pharmacia Ab Injection needle arrangement
US5328483A (en) 1992-02-27 1994-07-12 Jacoby Richard M Intradermal injection device with medication and needle guard
US5383851A (en) 1992-07-24 1995-01-24 Bioject Inc. Needleless hypodermic injection device
US5569189A (en) 1992-09-28 1996-10-29 Equidyne Systems, Inc. hypodermic jet injector
US5334144A (en) 1992-10-30 1994-08-02 Becton, Dickinson And Company Single use disposable needleless injector
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
WO1995024176A1 (en) 1994-03-07 1995-09-14 Bioject, Inc. Ampule filling device
US5466220A (en) 1994-03-08 1995-11-14 Bioject, Inc. Drug vial mixing and transfer device
WO1996000295A1 (en) 1994-06-27 1996-01-04 The Johns Hopkins University Targeted gene delivery system
US6383814B1 (en) 1994-12-09 2002-05-07 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US5599302A (en) 1995-01-09 1997-02-04 Medi-Ject Corporation Medical injection system and method, gas spring thereof and launching device using gas spring
US5730723A (en) 1995-10-10 1998-03-24 Visionary Medical Products Corporation, Inc. Gas pressured needle-less injection device and method
US5893397A (en) 1996-01-12 1999-04-13 Bioject Inc. Medication vial/syringe liquid-transfer apparatus
GB9607549D0 (en) 1996-04-11 1996-06-12 Weston Medical Ltd Spring-powered dispensing device
JPH10215175A (ja) 1996-11-29 1998-08-11 Sony Corp Ｐｌｌ回路及び信号再生装置
US5925628A (en) 1997-03-31 1999-07-20 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US5993412A (en) 1997-05-19 1999-11-30 Bioject, Inc. Injection apparatus
IT1298087B1 (it) 1998-01-08 1999-12-20 Fiderm S R L Dispositivo per il controllo della profondita' di penetrazione di un ago, in particolare applicabile ad una siringa per iniezioni
WO2002032396A2 (en) 2000-10-16 2002-04-25 Massachusetts Institute Of Technology Lipid-protein-sugar particles for delivery of nucleic acids
US7101995B2 (en) 2001-08-27 2006-09-05 Mirus Bio Corporation Compositions and processes using siRNA, amphipathic compounds and polycations
US20040242518A1 (en) 2002-09-28 2004-12-02 Massachusetts Institute Of Technology Influenza therapeutic
US8506959B2 (en) 2002-11-01 2013-08-13 Neotope Biosciences Limited Prevention and treatment of synucleinopathic and amyloidogenic disease
WO2004089471A2 (en) 2003-04-11 2004-10-21 Novo Nordisk A/S NEW PYRAZOLO[1,5-a] PYRIMIDINES DERIVATIVES AND PHARMACEUTICAL USE THEREOF
US7109173B1 (en) 2004-03-16 2006-09-19 Wilbert Gamble Transport of nucleotides, oligonucleotides and polynucleotides into the cytoplasm and nucleus of cells by peptides
AU2006254796B2 (en) 2005-06-08 2012-08-16 Amicus Therapeutics, Inc. Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes
US8716220B2 (en) * 2005-09-07 2014-05-06 Nikolaos Tezapsidis Leptin compositions and methods for treating progressive cognitive function disorders resulting from accumulation of neurofibrillary tangles and amyloid beta
ES2364586T3 (es) 2006-05-24 2011-09-07 Amicus Therapeutics, Inc. Sal tartrato de isofagomina y métodos de uso.
KR101402554B1 (ko) * 2006-06-23 2014-06-19 아미쿠스 세라퓨틱스, 인코포레이티드 β-글루코세레브로시다제의 활성 증진에 의한 신경계 장애 치료 방법
KR101597499B1 (ko) 2007-05-22 2016-02-24 아미쿠스 세라퓨틱스, 인코포레이티드 이소파고민과 이의 유도체를 제조하는 새로운 방법
WO2012061597A1 (en) 2010-11-03 2012-05-10 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer, National Institutes Of Health Alpha-glucosidase binders and methods of their use

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA200700427A1 (ru) * 2004-08-09 2008-02-28 Элан Фармасьютикалз, Инк. Предупреждение и лечение синуклеинопатических и амилоидогенных заболеваний
WO2007140212A2 (en) * 2006-05-24 2007-12-06 Amicus Therapeutics, Inc. Tartrate salt of isofagomine and methods of use
WO2012078855A1 (en) * 2010-12-08 2012-06-14 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Substituted pyrazolopyrimidines as glucocerebrosidase activators

Also Published As

Publication number Publication date
CA2840224A1 (en) 2012-12-27
JP6061922B2 (ja) 2017-01-18
EP2723384A4 (en) 2015-08-12
WO2012177997A1 (en) 2012-12-27
EP2723384A1 (en) 2014-04-30
CN103764166A (zh) 2014-04-30
CA2840224C (en) 2019-08-13
EP2723384B1 (en) 2018-08-29
US9845327B2 (en) 2017-12-19
US20140288093A1 (en) 2014-09-25
AU2012272815A1 (en) 2014-01-16
JP2014523881A (ja) 2014-09-18
CN103764166B (zh) 2017-10-24

Similar Documents

Publication Publication Date Title
AU2012272815B2 (en) Treatment of proteinopathies
US20110081428A1 (en) Use of thioflavin-like compounds to increase life span and/or health span
JP6204961B2 (ja) 脳アミロイド血管症の治療のための薬理シャペロン
JP2009521408A (ja) Ｃｄｃ２様キナーゼ（ＣＬＫ）のモジュレータおよびその使用方法
ES2897712T3 (es) Procedimientos para seleccionar inhibidores selectivos de fosfatasa y no selectivos de fosfatasa
US10022357B2 (en) Amyloid precursor protein MRNA blockers for treating Down syndrome and Alzheimer's disease
WO2009100037A1 (en) Methods for treating a condition characterized by dysfunction in protein homeostasis
WO2017015660A1 (en) Prevention and treatment of aging and neurodegenerative diseases
JP2019504113A (ja) 痛覚過敏を治療する方法
EP2664326A1 (en) Combined therapy for cystic fibrosis
JP2011516486A (ja) 骨質量疾患の診断、予防、及び治療方法
US20250161238A1 (en) NMDAR Antagonists Prevent Ageing and Aging-Associated Conditions and Diseases Through Increasing 20S Proteasome Activity
WO2021197389A1 (zh) Pi4k抑制剂在细胞内蛋白错误折叠相关疾病和溶酶体贮积病中的应用
JP2018510904A (ja) ニーマン・ピック病および他のリソソーム蓄積障害のためのｎｐｃ１のベンゼンスルホンアミド上方制御剤
CN107148270A (zh) 用于治疗和/或预防脑淀粉样变性的给药方案
US20220265605A1 (en) Methods of inducing autophagy using coumarin derivatives and related compounds
Wallace The Role of Tepsin in Membrane Trafficking and Autophagy
Rosarda The Many Mechanisms of AA147 Induced Cytoprotection
HK1215534B (en) Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases
HK1175400B (en) Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)