JP7781052B2 - 神経学的疾患のための組成物および方法 - Google Patents

神経学的疾患のための組成物および方法

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JP7781052B2
JP7781052B2 JP2022510971A JP2022510971A JP7781052B2 JP 7781052 B2 JP7781052 B2 JP 7781052B2 JP 2022510971 A JP2022510971 A JP 2022510971A JP 2022510971 A JP2022510971 A JP 2022510971A JP 7781052 B2 JP7781052 B2 JP 7781052B2
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receptor
ligand
pain
vector
engineered
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JP2022545226A5 (https=
JP2022545226A (ja
Inventor
アンソニー ジュニア ラウ,
オリオン ピー. ジュニア キーファー,
ステファニー マキンソン,
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トレイムス バイオ, インコーポレイテッド
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1787Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/04Centrally acting analgesics, e.g. opioids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/06Antimigraine agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/08Antiepileptics; Anticonvulsants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/22Anxiolytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/24Antidepressants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pain & Pain Management (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Biotechnology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
JP2022510971A 2019-08-21 2020-08-21 神経学的疾患のための組成物および方法 Active JP7781052B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024180241A JP2025004243A (ja) 2019-08-21 2024-10-15 神経学的疾患のための組成物および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962889963P 2019-08-21 2019-08-21
US62/889,963 2019-08-21
PCT/US2020/047503 WO2021035179A1 (en) 2019-08-21 2020-08-21 Compositions and methods for neurological diseases

Related Child Applications (1)

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JP2024180241A Division JP2025004243A (ja) 2019-08-21 2024-10-15 神経学的疾患のための組成物および方法

Publications (3)

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JP2022545226A JP2022545226A (ja) 2022-10-26
JP2022545226A5 JP2022545226A5 (https=) 2023-08-29
JP7781052B2 true JP7781052B2 (ja) 2025-12-05

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JP2022510971A Active JP7781052B2 (ja) 2019-08-21 2020-08-21 神経学的疾患のための組成物および方法
JP2024180241A Pending JP2025004243A (ja) 2019-08-21 2024-10-15 神経学的疾患のための組成物および方法

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Country Status (7)

Country Link
US (1) US20220348635A1 (https=)
EP (1) EP4017874A1 (https=)
JP (2) JP7781052B2 (https=)
CN (1) CN114746450A (https=)
AU (1) AU2020334147A1 (https=)
CA (1) CA3151920A1 (https=)
WO (1) WO2021035179A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL304040B2 (en) 2016-07-07 2024-09-01 Hughes Howard Med Inst Modified ligand-gated ion channels and methods of use
SG11202004246YA (en) 2017-11-10 2020-06-29 Hughes Howard Med Inst Modified ligand-gated ion channels and methods of use
IL300816A (en) * 2020-08-21 2023-04-01 Trames Bio Inc Preparations and methods for neurological diseases
US20250009906A1 (en) * 2021-11-19 2025-01-09 President And Fellows Of Harvard College Nociceptor-specific gene regulatory elements for the treatment of pain
EP4473002A4 (en) * 2022-01-31 2026-04-08 Hughes Howard Med Inst Modified ion channels sensitive to a ligand and methods of use
KR20240161957A (ko) * 2022-02-18 2024-11-13 트라메스 바이오 인코포레이티드 리간드 개폐 이온 통로 및 사용 방법
EP4514831A1 (en) * 2022-04-27 2025-03-05 Howard Hughes Medical Institute Chemogenetic receptors and methods of making and using
WO2025117698A1 (en) * 2023-12-01 2025-06-05 Massachusetts Institute Of Technology Ribozyme-enhanced rna trans-splicing

Citations (8)

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US20040009554A1 (en) 2002-05-09 2004-01-15 Memory Pharmaceuticals Corp. QM-7 and QT-6 cells transfected with mutant cell surface expressed channel receptors and assays using the transfected cells
US20060142188A1 (en) 2003-02-14 2006-06-29 Memory Pharmaceuticals Corporation Monkey alpha-7 nicotinic acetylcholine receptor and methods of use thereof
US20090100532A1 (en) 2007-09-25 2009-04-16 California Institute Of Technology Transgenic mice expressing hypersensitive nicotinic receptors
JP2009189261A (ja) 2008-02-12 2009-08-27 Univ Fukuoka てんかんモデル非ヒト哺乳動物
JP2012504966A (ja) 2008-10-09 2012-03-01 ハワード ヒューズ メディカル インスティチュート 新規なキメラリガンド開口型イオンチャネルおよびその使用方法
WO2018175443A1 (en) 2017-03-20 2018-09-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Targeted gene therapies for pain and other neuro-related disorders
WO2019094778A1 (en) 2017-11-10 2019-05-16 Scott Sternson Modified ligand-gated ion channels and methods of use
WO2019104307A1 (en) 2017-11-27 2019-05-31 Coda Biotherapeutics, Inc. Compositions and methods for neurological diseases

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WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
AU8672198A (en) 1997-07-31 1999-02-22 Chiron Corporation Method enabling readministration of aav vector via immunosuppression of host
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
JP2003523320A (ja) 1999-09-29 2003-08-05 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ウイルスベクターの迅速なpeg改変方法、高められた遺伝子形質導入のための組成物、高められた物理的安定性を伴う組成物、およびそのための用途
TW200412942A (en) 2002-08-06 2004-08-01 Abbott Lab Appetite control method
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040009554A1 (en) 2002-05-09 2004-01-15 Memory Pharmaceuticals Corp. QM-7 and QT-6 cells transfected with mutant cell surface expressed channel receptors and assays using the transfected cells
US20060142188A1 (en) 2003-02-14 2006-06-29 Memory Pharmaceuticals Corporation Monkey alpha-7 nicotinic acetylcholine receptor and methods of use thereof
US20090100532A1 (en) 2007-09-25 2009-04-16 California Institute Of Technology Transgenic mice expressing hypersensitive nicotinic receptors
JP2009189261A (ja) 2008-02-12 2009-08-27 Univ Fukuoka てんかんモデル非ヒト哺乳動物
JP2012504966A (ja) 2008-10-09 2012-03-01 ハワード ヒューズ メディカル インスティチュート 新規なキメラリガンド開口型イオンチャネルおよびその使用方法
WO2018175443A1 (en) 2017-03-20 2018-09-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Targeted gene therapies for pain and other neuro-related disorders
WO2019094778A1 (en) 2017-11-10 2019-05-16 Scott Sternson Modified ligand-gated ion channels and methods of use
WO2019104307A1 (en) 2017-11-27 2019-05-31 Coda Biotherapeutics, Inc. Compositions and methods for neurological diseases

Also Published As

Publication number Publication date
US20220348635A1 (en) 2022-11-03
CN114746450A (zh) 2022-07-12
EP4017874A1 (en) 2022-06-29
AU2020334147A1 (en) 2022-03-31
WO2021035179A1 (en) 2021-02-25
JP2025004243A (ja) 2025-01-14
JP2022545226A (ja) 2022-10-26
CA3151920A1 (en) 2021-02-25

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