JP2025503910A - 神経疾患を処置するための組成物 - Google Patents

神経疾患を処置するための組成物 Download PDF

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JP2025503910A
JP2025503910A JP2024543337A JP2024543337A JP2025503910A JP 2025503910 A JP2025503910 A JP 2025503910A JP 2024543337 A JP2024543337 A JP 2024543337A JP 2024543337 A JP2024543337 A JP 2024543337A JP 2025503910 A JP2025503910 A JP 2025503910A
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disease
cns
composition
vector
promoter
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JP2025503910A5 (https=
Inventor
アンナ トレチャコヴァ，
レスター スアレス，
アン ブラーイ，
マイケル エル． ロバーツ，
キャロライン ペドル，
イレアナ ゲリーニ，
フアン マヌエル イグレシアス，
ジョージ オマル ヤネス－クーナ，
フィリップ モリエ，
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アスクレピオス バイオファーマシューティカル， インコーポレイテッド
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Publication of JP2025503910A publication Critical patent/JP2025503910A/ja
Publication of JP2025503910A5 publication Critical patent/JP2025503910A5/ja
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    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
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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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/14—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced flavin or flavoprotein as one donor, and incorporation of one atom of oxygen (1.14.14)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Immunology (AREA)
  • Psychiatry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2024543337A 2022-01-27 2023-01-27 神経疾患を処置するための組成物 Pending JP2025503910A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263303673P 2022-01-27 2022-01-27
US63/303,673 2022-01-27
US202263321335P 2022-03-18 2022-03-18
US63/321,335 2022-03-18
PCT/US2023/011746 WO2023147058A2 (en) 2022-01-27 2023-01-27 Compositions for treating neurological disease

Publications (2)

Publication Number Publication Date
JP2025503910A true JP2025503910A (ja) 2025-02-06
JP2025503910A5 JP2025503910A5 (https=) 2026-01-19

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JP2024543337A Pending JP2025503910A (ja) 2022-01-27 2023-01-27 神経疾患を処置するための組成物

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US (1) US20250109398A1 (https=)
EP (1) EP4469156A4 (https=)
JP (1) JP2025503910A (https=)
CN (1) CN119677545A (https=)
AU (1) AU2023211606A1 (https=)
CA (1) CA3248890A1 (https=)
WO (1) WO2023147058A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118599984B (zh) * 2024-04-22 2025-12-09 暨南大学附属第一医院（广州华侨医院） Chchd2在制备促进神经干细胞增殖与分化产品中的应用

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20190992T1 (hr) * 2014-12-24 2019-09-20 Uniqure Ip B.V. Suprimiranje gena za huntingtin inducirano s rnai
JP2018500037A (ja) * 2014-12-31 2018-01-11 シンセティック ジェノミクス インコーポレーテッド 高効率なインビボゲノム編集のための組成物及び方法
KR102427379B1 (ko) * 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
US10457940B2 (en) * 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
SG11202000840YA (en) * 2017-07-31 2020-02-27 Reflection Biotechnologies Ltd Cellular models of and therapies for ocular diseases
US11364309B2 (en) * 2017-11-10 2022-06-21 Norwegian University Of Science And Technology (Ntnu) Neuronal enhancers
AU2019382824A1 (en) * 2018-11-19 2021-06-03 Uniqure Ip B.V. Method and means to deliver miRNA to target cells
CN115443339A (zh) * 2019-12-20 2022-12-06 阿斯克肋匹奥生物制药公司 用于治疗亨廷顿病的方法
GB202005732D0 (en) * 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
EP4213891A4 (en) * 2020-09-21 2025-07-23 Askbio Inc METHODS OF TREATING NEUROLOGICAL DISEASE

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Publication number Publication date
WO2023147058A2 (en) 2023-08-03
CN119677545A (zh) 2025-03-21
WO2023147058A3 (en) 2023-10-12
CA3248890A1 (en) 2023-08-03
EP4469156A2 (en) 2024-12-04
EP4469156A4 (en) 2026-04-08
AU2023211606A1 (en) 2024-07-11
US20250109398A1 (en) 2025-04-03

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