JP2024505197A - 焦点てんかんおよび神経因性疼痛を処置するためのアデノ随伴ウイルスカプシドおよび操作されたリガンド依存性イオンチャネル - Google Patents

焦点てんかんおよび神経因性疼痛を処置するためのアデノ随伴ウイルスカプシドおよび操作されたリガンド依存性イオンチャネル Download PDF

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JP2024505197A
JP2024505197A JP2023544528A JP2023544528A JP2024505197A JP 2024505197 A JP2024505197 A JP 2024505197A JP 2023544528 A JP2023544528 A JP 2023544528A JP 2023544528 A JP2023544528 A JP 2023544528A JP 2024505197 A JP2024505197 A JP 2024505197A
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seq
neuron
amino acid
aav
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アナヒタ ケラバラ，
エドワード イェー，
アルベナ カンタージエバ，
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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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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • A—HUMAN NECESSITIES
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    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • 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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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)
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  • Wood Science & Technology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pain & Pain Management (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023544528A 2021-01-25 2022-01-25 焦点てんかんおよび神経因性疼痛を処置するためのアデノ随伴ウイルスカプシドおよび操作されたリガンド依存性イオンチャネル Pending JP2024505197A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163141124P 2021-01-25 2021-01-25
US202163141121P 2021-01-25 2021-01-25
US63/141,124 2021-01-25
US63/141,121 2021-01-25
US202163285929P 2021-12-03 2021-12-03
US63/285,929 2021-12-03
PCT/US2022/013658 WO2022159871A1 (en) 2021-01-25 2022-01-25 Adeno-associated virus capsids and engineered ligand-gated ion channels for treating focal epilepsy and neuropathic pain

Publications (3)

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JP2024505197A true JP2024505197A (ja) 2024-02-05
JP2024505197A5 JP2024505197A5 (https=) 2025-02-03
JPWO2022159871A5 JPWO2022159871A5 (https=) 2025-02-03

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JP2023544528A Pending JP2024505197A (ja) 2021-01-25 2022-01-25 焦点てんかんおよび神経因性疼痛を処置するためのアデノ随伴ウイルスカプシドおよび操作されたリガンド依存性イオンチャネル

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US (1) US20240108760A1 (https=)
EP (1) EP4281551A4 (https=)
JP (1) JP2024505197A (https=)
KR (1) KR20230149299A (https=)
AU (1) AU2022211055A1 (https=)
CA (1) CA3205877A1 (https=)
IL (1) IL304511A (https=)
MX (1) MX2023008738A (https=)
WO (1) WO2022159871A1 (https=)

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WO2025188847A1 (en) * 2024-03-07 2025-09-12 The Texas A&M University System Method for treating epilepsy
WO2025230969A1 (en) * 2024-05-02 2025-11-06 4D Molecular Therapeutics Inc. Aav variants with improved cns tropism and uses thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018531926A (ja) * 2015-09-17 2018-11-01 コーダ バイオセラピューティクス， インコーポレイテッド 神経学的障害を処置するための組成物および方法

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1856576B (zh) * 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
WO2015126972A1 (en) * 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
EP3468545A4 (en) * 2016-06-08 2020-07-22 President and Fellows of Harvard College METHODS AND COMPOSITIONS FOR REDUCING TACTILE DYSFUNCTION AND ANXIETY ASSOCIATED WITH AUTISM SPECTRUM DISORDER, RETT SYNDROME AND FRAGILE X SYNDROME
MX2020005470A (es) * 2017-11-27 2020-11-09 Coda Biotherapeutics Inc Composiciones y metodos para enfermedades neurologicas.
US20210261962A1 (en) * 2018-06-21 2021-08-26 The Board Of Regents Of The University Of Texas System Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018531926A (ja) * 2015-09-17 2018-11-01 コーダ バイオセラピューティクス， インコーポレイテッド 神経学的障害を処置するための組成物および方法

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AU2022211055A1 (en) 2023-08-17
US20240108760A1 (en) 2024-04-04
MX2023008738A (es) 2023-09-12
EP4281551A4 (en) 2025-06-04
KR20230149299A (ko) 2023-10-26
CA3205877A1 (en) 2022-07-28
AU2022211055A9 (en) 2024-07-11
WO2022159871A1 (en) 2022-07-28
EP4281551A1 (en) 2023-11-29
IL304511A (en) 2023-09-01

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