CA2840224C - Treatment of proteinopathies - Google Patents

Treatment of proteinopathies Download PDF

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Publication number
CA2840224C
CA2840224C CA2840224A CA2840224A CA2840224C CA 2840224 C CA2840224 C CA 2840224C CA 2840224 A CA2840224 A CA 2840224A CA 2840224 A CA2840224 A CA 2840224A CA 2840224 C CA2840224 C CA 2840224C
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Prior art keywords
disease
lysosomal
polypeptide
synuclein
diseases
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CA2840224A
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French (fr)
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CA2840224A1 (en
Inventor
Dmitri KRAINC
Joseph R. Mazzulli
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General Hospital Corp
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General Hospital Corp
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    • 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
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    • 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

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  • 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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Urology & Nephrology (AREA)
  • Neurology (AREA)
  • Diabetes (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)
  • Obesity (AREA)
  • Endocrinology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pain & Pain Management (AREA)
CA2840224A 2011-06-22 2012-06-22 Treatment of proteinopathies Active CA2840224C (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
CA2840224A1 CA2840224A1 (en) 2012-12-27
CA2840224C true CA2840224C (en) 2019-08-13

Family

ID=47422968

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2840224A Active CA2840224C (en) 2011-06-22 2012-06-22 Treatment of proteinopathies

Country Status (7)

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

Families Citing this family (101)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8759297B2 (en) 2006-08-18 2014-06-24 Armagen Technologies, Inc. Genetically encoded multifunctional compositions bidirectionally transported between peripheral blood and the cns
AU2008282496B2 (en) 2007-07-27 2013-04-04 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the CNS
EP2485761B1 (en) 2009-10-09 2019-02-27 Armagen, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
PL2701510T3 (pl) 2011-04-25 2017-07-31 Usher Iii Initiative Inc Pirazolopirydazyny oraz sposoby leczenia zwyrodnieniowych chorób siatkówki i utraty słuchu związanych z zespołem ushera
US9309250B2 (en) 2011-06-22 2016-04-12 Vertex Pharmaceuticals Incorporated Substituted pyrrolo[2,3-b]pyrazines as ATR kinase inhibitors
RS63930B1 (sr) 2011-10-28 2023-02-28 Prothena Biosciences Ltd Humanizovana antitela koja prepoznaju alfa-sinuklein
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
DK2807188T3 (da) 2012-01-27 2019-10-07 Prothena Biosciences Ltd Humaniserede antistoffer, der genkender alpha-synuclein
US8940742B2 (en) 2012-04-10 2015-01-27 Infinity Pharmaceuticals, Inc. Heterocyclic compounds and uses thereof
JP2015518872A (ja) * 2012-06-06 2015-07-06 フォンダッツィオーネ・テレソン アロステリックシャペロンおよびその使用
MA37975B2 (fr) 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide
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
AU2013337354A1 (en) 2012-11-05 2015-05-21 Genzyme Corporation Compositions and methods for treating proteinopathies
SI2941432T1 (en) 2012-12-07 2018-07-31 Vertex Pharmaceuticals Incorporated 2-AMINO-6-FLUORO-N- (5-FLUORO-4- (4- (4- (OXETHAN-3-YL) PIPERAZIN-1-CARBONYL) PIPERIDIN-1-YLIPRIDIN-3-YL) 1,5 ALFA) PYRIMIDINE-3-CARBOXAMIDE AS ATR KINAZE INHIBITOR
EP2970288A1 (en) 2013-03-15 2016-01-20 Vertex Pharmaceuticals Incorporated Compounds useful as inhibitors of atr kinase
US8957078B2 (en) 2013-03-15 2015-02-17 Vertex Pharmaceuticals Incorporated Compounds useful as inhibitors of ATR kinase
WO2014143240A1 (en) 2013-03-15 2014-09-18 Vertex Pharmaceuticals Incorporated Fused pyrazolopyrimidine derivatives useful as inhibitors of atr kinase
EP3010532A4 (en) * 2013-06-18 2017-06-14 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
PT3052485T (pt) 2013-10-04 2021-10-22 Infinity Pharmaceuticals Inc Compostos heterocíclicos e suas utilizações
US9751888B2 (en) 2013-10-04 2017-09-05 Infinity Pharmaceuticals, Inc. Heterocyclic compounds and uses thereof
PT3077397T (pt) 2013-12-06 2020-01-22 Vertex Pharma Composto de 2-amino-6-fluoro-n-[5-fluoro-piridin-3-il]pirazolo[1,5-a]pirimidin-3-carboxamida útil como inibidor da atr quinase, a sua preparação, diferentes formas sólidas e derivados radiomarcados do mesmo
WO2015121339A1 (en) 2014-02-14 2015-08-20 RUHR-UNIVERSITäT BOCHUM Biosensor for conformation and secondary structure analysis
MX382033B (es) 2014-03-19 2025-03-13 Infinity Pharmaceuticals Inc Compuestos heterocíclicos para utilizarlos en el tratamiento de trastornos mediados por pi3k-gamma.
CA2944402A1 (en) 2014-04-08 2015-10-15 Prothena Biosciences Limited Blood-brain barrier shuttles containing antibodies recognizing alpha-synuclein
MX373102B (es) 2014-06-05 2020-04-17 Vertex Pharma Derivados radiomarcados de un compuesto de 2-amino-6-fluoro-n-[5-fluoro-piridin-3-il]-pirazolo[1,5-a]pirimidin-3-carboxamida útil como inhibidor de ataxia telangiectasia mutada y rad3 relacionado (atr) cinasa, preparación de tal compuesto y diferentes formas sólidas del mismo.
SG11201610500WA (en) 2014-06-17 2017-01-27 Vertex Pharma 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
EP4406616A3 (en) 2014-11-06 2024-11-06 Bial-R&D Investments, S.A. Substituted pyrazolo(1,5-a)pyrimidines and their use in the treatment of medical disorders
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
US20180185368A1 (en) 2014-11-06 2018-07-05 Lysosomal Therapeutics Inc. Substituted pyrrolo[1,2-a]pyrimidines and their use in the treatment of medical disorders
WO2016077706A1 (en) 2014-11-13 2016-05-19 Washington University Treatment for wolfram syndrome and other endoplasmic reticulum stress disorders
CN107205976A (zh) * 2014-11-19 2017-09-26 拉什大学医学中心 用于治疗溶酶体病症的组合物和方法
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
WO2016201266A1 (en) 2015-06-11 2016-12-15 Usher Iii Initiative, Inc. Methods of treating or preventing a proteopathy
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
WO2017048702A1 (en) 2015-09-14 2017-03-23 Infinity Pharmaceuticals, Inc. Solid forms of isoquinolinone derivatives, process of making, compositions comprising, and methods of using the same
AU2016331955B2 (en) 2015-09-30 2022-07-21 Vertex Pharmaceuticals Incorporated Method for treating cancer using a combination of DNA damaging agents and ATR inhibitors
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
KR20180124971A (ko) * 2016-03-25 2018-11-21 젠자임 코포레이션 단백질병증의 바이오마커 및 이의 용도
CA3020305A1 (en) 2016-04-06 2017-10-12 Lysosomal Therapeutics Inc. Imidazo [1,5-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders
EP3440081A4 (en) * 2016-04-06 2019-09-18 Lysosomal Therapeutics Inc. PYRROLO [1,2-A] PYRIMIDINYL-CARBOXAMIDE COMPOUNDS AND THEIR USE IN THE TREATMENT OF MEDICAL DISORDER
AU2017246455B2 (en) 2016-04-06 2021-09-30 Bial - R&D Investments, S.A. Pyrazolo[1,5-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders
KR102530515B1 (ko) 2016-05-05 2023-05-09 비알 - 알&디 인베스트먼츠, 에스.에이. 치환된 이미다조[1,2-b]피리다진, 치환된 이미다조[1,5-b]피리다진, 관련 화합물, 및 의학적 장애의 치료에서의 그의 용도
WO2017192931A1 (en) 2016-05-05 2017-11-09 Lysosomal Therapeutics Inc. SUBSTITUTED IMDAZO[1,2-α]PYRIDINES, SUBSTITUTED IMIDAZO[1,2-α]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
WO2018090054A1 (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
CN110325516A (zh) 2016-12-29 2019-10-11 米尼奥尔克斯治疗有限公司 杂芳基化合物和它们的用途
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
WO2018176001A2 (en) 2017-03-24 2018-09-27 Cedars-Sinai Medical Center Methods and compositions for production of fallopian tube epithelium
EP3661961A4 (en) * 2017-08-02 2021-04-14 Stressmarq Biosciences Inc. ANTIBODIES BINDING TO ACTIVE ALPHA-SYNUCLEIN
FI3678739T3 (fi) * 2017-09-07 2024-05-21 Childrens Hospital Philadelphia Koostumuksia ja menetelmiä periytyvän kystatiini c -amyloidiangiopatian (hccaa) ja muiden poikkeaviin amyloidikertymiin liittyvien hermostoa rappeuttavien häiriöiden hoitoon
CR20200129A (es) 2017-10-02 2020-08-22 Denali Therapeutics Inc Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas
AU2018346597B2 (en) 2017-10-06 2023-07-13 Forma Therapeutics, Inc. Inhibiting Ubiquitin Specific Peptidase 30
WO2019150192A1 (en) * 2018-02-02 2019-08-08 Enzyvant Therapeutics Gmbh Methods for treating farber disease
EP4700387A2 (en) 2018-03-23 2026-02-25 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
CN112203697A (zh) 2018-04-13 2021-01-08 马萨诸塞大学 编码氨基己糖苷酶alpha和beta亚基的双顺反子AAV载体及其用途
EP3787613B1 (en) * 2018-04-30 2026-03-11 Cedars-Sinai Medical Center Pkc activation for use in treating early onset sporadic parkinson's disease
JP7449242B2 (ja) 2018-05-17 2024-03-13 フォーマ セラピューティクス，インコーポレイテッド ユビキチン特異的ペプチダーゼ３０（ｕｓｐ３０）阻害剤として有用な縮合二環化合物
WO2019234243A1 (en) * 2018-06-08 2019-12-12 Ac Immune Sa Novel compounds for diagnosis
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
US12448629B2 (en) 2018-10-05 2025-10-21 University Of Massachusetts rAAV vectors for the treatment of GM1 and GM2 gangliosidosis
JP7530889B2 (ja) 2018-10-05 2024-08-08 フォーマ セラピューティクス，インコーポレイテッド ユビキチン特異的プロテアーゼ３０（ｕｓｐ３０）阻害剤として作用する縮合ピロリン
SG11202105606XA (en) 2018-12-05 2021-06-29 Univ Washington Methods of detecting, preventing, reversing, and treating neurological diseases
US20210403584A1 (en) * 2019-01-08 2021-12-30 Armagen, Inc. Methods and compositions for increasing galactosidase beta-1 activity in the cns
CN114040762A (zh) 2019-02-04 2022-02-11 建新公司 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病
CN113645969A (zh) * 2019-02-04 2021-11-12 建新公司 用于治疗与溶酶体贮积症相关的症状和病症的方法
EP3920921A4 (en) * 2019-02-07 2022-11-02 Alsatech, Inc. POLYVALENT EMOXYPIN DERIVATIVES
CA3132379A1 (en) * 2019-03-22 2020-10-01 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal storage disorders
JP2022531177A (ja) * 2019-04-29 2022-07-06 トーマス・ジェファーソン・ユニバーシティ 神経変性障害を治療するための方法
WO2020257736A1 (en) 2019-06-21 2020-12-24 The Broad Institute, Inc. Agents for reversing toxic proteinopathies
AU2020302992A1 (en) * 2019-06-28 2022-02-03 The Trustees Of The University Of Pennsylvania Intranasal 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
AU2021209954A1 (en) * 2020-01-23 2022-09-01 Retispec Inc. Systems and methods for disease diagnosis
JP2023512366A (ja) 2020-02-03 2023-03-27 ジェンザイム・コーポレーション リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法
BR112022019864A2 (pt) * 2020-03-31 2022-11-16 Nuo Beta Pharmaceutical Tech Shanghai Co Ltd Composto de oxofenilarsina deuterada e uso do mesmo
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
EP4178580B1 (en) 2020-07-09 2025-11-05 Usher III Initiative, Inc. Bf 844 for use in the treatment of cancer
AU2021311131B2 (en) 2020-07-24 2026-02-19 Genzyme Corporation Pharmaceutical compositions comprising venglustat
KR20230128462A (ko) 2020-11-19 2023-09-05 제브라 덴마크 에이/에스 아리모클로몰 시트레이트 및 이의 중간체를 제조하기 위한 공정
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
WO2023042178A2 (en) * 2021-09-20 2023-03-23 Aleta Neuroscience, Llc 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
CN119236096B (zh) * 2024-10-11 2026-03-06 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
CN120771139A (zh) * 2025-07-18 2025-10-14 昆明理工大学 维拉帕米在制备治疗Rett综合征药物中的应用

Family Cites Families (57)

* 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
US4790824A (en) 1987-06-19 1988-12-13 Bioject, Inc. Non-invasive hypodermic injection device
US4941880A (en) 1987-06-19 1990-07-17 Bioject, Inc. Pre-filled ampule and non-invasive hypodermic injection device assembly
US4940460A (en) 1987-06-19 1990-07-10 Bioject, Inc. Patient-fillable and non-invasive hypodermic injection device assembly
US5004697A (en) 1987-08-17 1991-04-02 Univ. Of Ca Cationized antibodies for delivery through the blood-brain barrier
US4933324A (en) 1988-02-26 1990-06-12 Shashoua Victor E Fatty acid-neuroactive drug conjugate as a prodrug
US6107499A (en) 1988-02-26 2000-08-22 Neuromedica, Inc. Dopamine analog amide
US4939174A (en) 1988-02-26 1990-07-03 Shashoua Victor E Appetite suppression with dopamine-fatty acid conjugates
US5994392A (en) 1988-02-26 1999-11-30 Neuromedica, Inc. Antipsychotic prodrugs comprising an antipsychotic agent coupled to an unsaturated fatty acid
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
US5268164A (en) 1990-04-23 1993-12-07 Alkermes, Inc. Increasing blood-brain barrier permeability with permeabilizer peptides
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
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
JPH11505505A (ja) 1994-06-27 1999-05-21 ザ・ジョンズ・ホプキンス・ユニバーシティー ターゲッティングされた遺伝子輸送システム
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
EA013752B1 (ru) * 2004-08-09 2010-06-30 Элан Фармасьютикалз, Инк. Предупреждение и лечение синуклеинопатических и амилоидогенных заболеваний
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.
PL1860101T3 (pl) * 2006-05-24 2011-10-31 Amicus Therapeutics Inc Sól winianu izofagominy i sposoby zastosowania
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
CA2688850C (en) * 2007-05-22 2015-08-11 Amicus Therapeutics, Inc. New method for preparing isofagomine and its derivatives
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
EP3366688B1 (en) * 2010-12-08 2022-02-09 The U.S.A. as represented by the Secretary, Department of Health and Human Services Substituted pyrazolopyrimidines as glucocerebrosidase activators

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