JP2024505570A - 神経学的疾患及び疼痛を治療するためのエピジェネティック遺伝子調節 - Google Patents

神経学的疾患及び疼痛を治療するためのエピジェネティック遺伝子調節 Download PDF

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JP2024505570A
JP2024505570A JP2023546482A JP2023546482A JP2024505570A JP 2024505570 A JP2024505570 A JP 2024505570A JP 2023546482 A JP2023546482 A JP 2023546482A JP 2023546482 A JP2023546482 A JP 2023546482A JP 2024505570 A JP2024505570 A JP 2024505570A
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nucleic acid
promoter
disease
acid sequence
target molecules
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JPWO2022165362A5 (https=
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ギレン フェルナンド アレマン，
コラド， アナ マリア モレノ
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ナヴェガ セラピューティクス， インコーポレイテッド
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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
    • A61K48/005—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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K48/0025—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 non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—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 non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
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    • 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
    • C07K14/43504—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates
    • C07K14/43513—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates from arachnidae
    • C07K14/43518—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates from arachnidae from spiders
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Insects & Arthropods (AREA)
  • Immunology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023546482A 2021-02-01 2022-01-31 神経学的疾患及び疼痛を治療するためのエピジェネティック遺伝子調節 Pending JP2024505570A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163144408P 2021-02-01 2021-02-01
US63/144,408 2021-02-01
PCT/US2022/014601 WO2022165362A2 (en) 2021-02-01 2022-01-31 Epigenetic gene regulation to treat neurological diseases and pain

Publications (3)

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JP2024505570A true JP2024505570A (ja) 2024-02-06
JP2024505570A5 JP2024505570A5 (https=) 2025-02-10
JPWO2022165362A5 JPWO2022165362A5 (https=) 2025-02-10

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JP2023546482A Pending JP2024505570A (ja) 2021-02-01 2022-01-31 神経学的疾患及び疼痛を治療するためのエピジェネティック遺伝子調節

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US (1) US20240366795A1 (https=)
EP (1) EP4284927A4 (https=)
JP (1) JP2024505570A (https=)
CN (1) CN117203334A (https=)
AU (1) AU2022212277A1 (https=)
CA (1) CA3206936A1 (https=)
WO (1) WO2022165362A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024258906A1 (en) * 2023-04-17 2025-11-06 Sangamo Therapeutics, Inc. Zinc finger protein transcription factors for repressing nav1.7 expression
US20250057981A1 (en) * 2023-06-26 2025-02-20 Navega Therapeutics, Inc. Compositions and methods for reducing pain and inflammation through epigenetic modulation
WO2025076459A1 (en) * 2023-10-05 2025-04-10 Navega Therapeutics, Inc. Compositions and methods for epigenetic modulation of nav1.7
CN119139498B (zh) * 2024-09-26 2025-11-25 武汉泓宸创新生物科技有限公司 用于治疗与运动神经元损伤相关的疾病的药物

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016533710A (ja) * 2013-10-03 2016-11-04 ヤンセン バイオテツク，インコーポレーテツド プロトキシン−ｉｉ変異体及びその使用方法
JP2019524162A (ja) * 2016-08-18 2019-09-05 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア モジュラーＡＡＶ送達システムによるＣＲＩＳＰＲ−Ｃａｓゲノム編集
JP2020526554A (ja) * 2017-07-13 2020-08-31 ユニバーシティ オブ マイアミ 疼痛を管理するための方法
WO2020210542A1 (en) * 2019-04-09 2020-10-15 The Regents Of The University Of California Long-lasting analgesia via targeted in vivo epigenetic repression

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016533710A (ja) * 2013-10-03 2016-11-04 ヤンセン バイオテツク，インコーポレーテツド プロトキシン−ｉｉ変異体及びその使用方法
JP2019524162A (ja) * 2016-08-18 2019-09-05 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア モジュラーＡＡＶ送達システムによるＣＲＩＳＰＲ−Ｃａｓゲノム編集
JP2020526554A (ja) * 2017-07-13 2020-08-31 ユニバーシティ オブ マイアミ 疼痛を管理するための方法
WO2020210542A1 (en) * 2019-04-09 2020-10-15 The Regents Of The University Of California Long-lasting analgesia via targeted in vivo epigenetic repression

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WO2022165362A2 (en) 2022-08-04
WO2022165362A3 (en) 2022-09-01
CA3206936A1 (en) 2022-08-04
AU2022212277A1 (en) 2023-09-14
EP4284927A2 (en) 2023-12-06
CN117203334A (zh) 2023-12-08
EP4284927A4 (en) 2025-07-02
US20240366795A1 (en) 2024-11-07

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