KR20200112811A - 신경퇴행성 질환에 대한 조성물 및 방법 - Google Patents

신경퇴행성 질환에 대한 조성물 및 방법 Download PDF

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KR20200112811A
KR20200112811A KR1020207016109A KR20207016109A KR20200112811A KR 20200112811 A KR20200112811 A KR 20200112811A KR 1020207016109 A KR1020207016109 A KR 1020207016109A KR 20207016109 A KR20207016109 A KR 20207016109A KR 20200112811 A KR20200112811 A KR 20200112811A
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receptor
engineered
vector
pain
ligand
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Korean (ko)
Inventor
케네쓰 피. 그린버그
오리온 주니어 키퍼
스테파니 매킨슨
앤서니 라우
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코다 바이오테라퓨틱스 인코포레이티드
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Priority to KR1020267002196A priority Critical patent/KR20260030128A/ko
Publication of KR20200112811A publication Critical patent/KR20200112811A/ko
Ceased legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • 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)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020207016109A 2017-11-27 2018-11-27 신경퇴행성 질환에 대한 조성물 및 방법 Ceased KR20200112811A (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020267002196A KR20260030128A (ko) 2017-11-27 2018-11-27 신경퇴행성 질환에 대한 조성물 및 방법

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762590911P 2017-11-27 2017-11-27
US62/590,911 2017-11-27
US201862659911P 2018-04-19 2018-04-19
US62/659,911 2018-04-19
PCT/US2018/062575 WO2019104307A1 (en) 2017-11-27 2018-11-27 Compositions and methods for neurological diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020267002196A Division KR20260030128A (ko) 2017-11-27 2018-11-27 신경퇴행성 질환에 대한 조성물 및 방법

Publications (1)

Publication Number Publication Date
KR20200112811A true KR20200112811A (ko) 2020-10-05

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KR1020207016109A Ceased KR20200112811A (ko) 2017-11-27 2018-11-27 신경퇴행성 질환에 대한 조성물 및 방법
KR1020267002196A Pending KR20260030128A (ko) 2017-11-27 2018-11-27 신경퇴행성 질환에 대한 조성물 및 방법

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KR1020267002196A Pending KR20260030128A (ko) 2017-11-27 2018-11-27 신경퇴행성 질환에 대한 조성물 및 방법

Country Status (12)

Country Link
US (2) US10538571B2 (https=)
EP (1) EP3716990A4 (https=)
JP (2) JP2021503960A (https=)
KR (2) KR20200112811A (https=)
CN (1) CN111741760A (https=)
AU (2) AU2018373279B2 (https=)
CA (1) CA3082907A1 (https=)
IL (2) IL274702B2 (https=)
MX (2) MX2020005470A (https=)
SG (1) SG11202004488TA (https=)
WO (1) WO2019104307A1 (https=)
ZA (1) ZA202408862B (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020167624A1 (en) 2019-02-13 2020-08-20 Ptc Therapeutics, Inc. Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia
SG11202108519TA (en) 2019-02-13 2021-09-29 Ptc Therapeutics Inc Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia
AU2020334147A1 (en) * 2019-08-21 2022-03-31 Trames Bio, Inc. Compositions and methods for neurological diseases
WO2021168110A1 (en) * 2020-02-20 2021-08-26 The Regents Of The University Of California Therapies for the treatment of diseases and disorders associated with abnormal expression of a neural-associated gene
IL300816A (en) * 2020-08-21 2023-04-01 Trames Bio Inc Preparations and methods for neurological diseases
WO2022067044A1 (en) * 2020-09-25 2022-03-31 The Regents Of The University Of California Compositions and methods for ameliorating medical conditions
MX2023008738A (es) * 2021-01-25 2023-09-12 Trames Bio Inc Cápsides de virus adenoasociados y canales iónicos controlados por ligandos diseñados para el tratamiento contra epilepsia focal y dolor neuropático.
US11969400B2 (en) * 2021-03-23 2024-04-30 Kumara V. Nibhanipudi Ibuprofen for symptomatic treatment of diarrheas in HIV patients
WO2023278295A1 (en) 2021-06-29 2023-01-05 The Broad Institute, Inc. Compositions and methods for ameliorating anterodorsal thalamus hyperexcitability
CN113730809B (zh) * 2021-10-09 2024-08-16 科悦医疗(苏州)有限公司 一种非侵入式迷走神经联合骶神经刺激装置
EP4473002A4 (en) * 2022-01-31 2026-04-08 Hughes Howard Med Inst Modified ion channels sensitive to a ligand and methods of use
CN119137144A (zh) * 2022-02-18 2024-12-13 特拉梅斯生物股份有限公司 用于神经系统疾病的组合物和方法
KR20240161957A (ko) * 2022-02-18 2024-11-13 트라메스 바이오 인코포레이티드 리간드 개폐 이온 통로 및 사용 방법
WO2025015092A1 (en) * 2023-07-10 2025-01-16 Kriya Therapeutics, Inc. Novel cam-kinase promoter and uses thereof for treating nervous system disorders
CN121866054A (zh) * 2023-08-16 2026-04-14 拜尔吉生物股份有限公司 用于治疗癫痫发作的方法
GB202401492D0 (en) 2024-02-05 2024-03-20 Ucl Business Ltd Chloride channels and uses thereof

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69434067T2 (de) * 1993-03-08 2006-03-02 Merck & Co., Inc. Menschliche neuronale nikotin-acetylcholin-rezeptor-verbindungen und methoden ihres einsatzes
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
EP0893950A4 (en) 1996-03-26 2002-02-27 Univ California SELECTIVE TARGET CELL ACTIVATION BY EXPRESSION OF A RECEPTOR SPECIFICALLY ACTIVATED BY SYNTHETIC LIGANDS
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
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
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
AU4980200A (en) 1999-05-27 2000-12-18 Pharmacia & Upjohn Company Methods and compositions for measuring ion channel conductance
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
EP1280902A2 (en) 2000-04-29 2003-02-05 Millennium Pharmaceuticals, Inc. 23949 and 32391, human ion channels and uses thereof
EP1532452A4 (en) 2002-05-31 2006-11-29 Sloan Kettering Inst Cancer HETEROLOGY STIMULUS-CONTROLLED ION CHANNELS AND METHOD FOR USE THEREOF
SI1633772T1 (sl) 2003-06-19 2016-06-30 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
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
WO2008143875A1 (en) 2007-05-14 2008-11-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Targeted delivery of glycine receptors to excitable cells
EP2344636B1 (en) * 2008-10-09 2017-12-06 Howard Hughes Medical Institute Novel chimeric ligand-gated ion channels and methods of use thereof
WO2014093251A1 (en) * 2012-12-10 2014-06-19 University Of Pittsburgh Of The Commonwealth System Of Higher Education Engineered receptors and their use
GB201404470D0 (en) 2014-03-13 2014-04-30 Ucl Business Plc Therapeutic methods and materials
US10682424B2 (en) 2015-03-31 2020-06-16 New York University Compositions and method for reducing seizures
AU2016324317A1 (en) 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
IL304040B2 (en) 2016-07-07 2024-09-01 Hughes Howard Med Inst Modified ligand-gated ion channels and methods of use
US11713470B2 (en) 2017-03-20 2023-08-01 University of Pittsburgh—of the Commonwealth System of Higher Education Targeted gene therapies for pain and other neuro-related disorders

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Publication number Publication date
SG11202004488TA (en) 2020-06-29
IL274702B2 (en) 2026-04-01
WO2019104307A1 (en) 2019-05-31
EP3716990A1 (en) 2020-10-07
KR20260030128A (ko) 2026-03-05
JP2024160218A (ja) 2024-11-13
AU2018373279B2 (en) 2024-07-25
AU2024227560A1 (en) 2024-11-14
IL324810A (en) 2026-01-01
US20210061873A1 (en) 2021-03-04
JP2021503960A (ja) 2021-02-15
IL274702B1 (en) 2025-12-01
EP3716990A4 (en) 2021-12-08
US20190161529A1 (en) 2019-05-30
CA3082907A1 (en) 2019-05-31
MX2020005470A (es) 2020-11-09
IL274702A (en) 2020-06-30
AU2018373279A1 (en) 2020-06-04
ZA202408862B (en) 2026-01-28
US10538571B2 (en) 2020-01-21
MX2024014448A (es) 2024-12-06
CN111741760A (zh) 2020-10-02

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