WO2025019295A3 - Injection pelvienne rénale pour administrer un médicament au rein - Google Patents

Injection pelvienne rénale pour administrer un médicament au rein Download PDF

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Publication number
WO2025019295A3
WO2025019295A3 PCT/US2024/037749 US2024037749W WO2025019295A3 WO 2025019295 A3 WO2025019295 A3 WO 2025019295A3 US 2024037749 W US2024037749 W US 2024037749W WO 2025019295 A3 WO2025019295 A3 WO 2025019295A3
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WIPO (PCT)
Prior art keywords
injection
aav
renal pelvic
kidney
subject
Prior art date
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Pending
Application number
PCT/US2024/037749
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English (en)
Other versions
WO2025019295A2 (fr
Inventor
Hiroyuki Nakai NAKAI
Masahiro HORIKAWA
Taisuke FURUSHO
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Oregon Health and Science University
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Oregon Health and Science University
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Application filed by Oregon Health and Science University filed Critical Oregon Health and Science University
Publication of WO2025019295A2 publication Critical patent/WO2025019295A2/fr
Publication of WO2025019295A3 publication Critical patent/WO2025019295A3/fr
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • 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
    • 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
    • C12N2750/14122—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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

L'invention concerne des méthodes d'administration d'une composition contenant un virus adéno-associé (AAV) à un ou plusieurs types de cellules rénales chez un sujet avec ou sans immunité contre l'AAV, les méthodes consistant à administrer localement au rein du sujet un virus adéno-associé (AAV) comprenant un génome et une protéine de capside d'AAV par injection pelvienne rénale, l'injection pelvienne rénale consistant en une injection pelvienne rénale directe ou en une injection pelvienne rénale transurétérale rétrograde. L'invention concerne également des méthodes de traitement d'un trouble rénal chez un sujet avec ou sans immunité contre l'AAV, les méthodes consistant à administrer localement au rein du sujet par injection pelvienne rénale, celle-ci pouvant être une injection pelvienne rénale directe ou une injection pelvienne rénale transurétérale rétrograde, un virus adéno-associé (AAV) comprenant un génome et une protéine de capside d'AAV, le génome codant pour un agent thérapeutique qui traite le trouble rénal chez le sujet. L'AAV peut être AAVKP1, AAVKP2, AAVKP3, AAVDJ, AAV2G9, AAV2.7m8, AAV3, AAVLK03, ou AAVShH10.
PCT/US2024/037749 2023-07-17 2024-07-12 Injection pelvienne rénale pour administrer un médicament au rein Pending WO2025019295A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363527248P 2023-07-17 2023-07-17
US63/527,248 2023-07-17

Publications (2)

Publication Number Publication Date
WO2025019295A2 WO2025019295A2 (fr) 2025-01-23
WO2025019295A3 true WO2025019295A3 (fr) 2025-03-13

Family

ID=94282517

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/037749 Pending WO2025019295A2 (fr) 2023-07-17 2024-07-12 Injection pelvienne rénale pour administrer un médicament au rein

Country Status (1)

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WO (1) WO2025019295A2 (fr)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025193938A1 (fr) * 2024-03-13 2025-09-18 Oregon Health & Science University Administration de chaîne alpha de collagène de type iv par l'utilisation d'un vecteur aav à double inteine split
GB202414499D0 (en) * 2024-10-02 2024-11-13 Purespring Therapeutics Ltd Methods

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013159036A1 (fr) * 2012-04-19 2013-10-24 Oregon Health & Science University Plasmides et vecteurs de virus associés aux adénovirus
US20200024616A1 (en) * 2018-03-30 2020-01-23 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
US20220354969A1 (en) * 2021-05-10 2022-11-10 Oregon Health & Science University Enhancing viral vector-based gene therapy

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013159036A1 (fr) * 2012-04-19 2013-10-24 Oregon Health & Science University Plasmides et vecteurs de virus associés aux adénovirus
US20200024616A1 (en) * 2018-03-30 2020-01-23 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
US20220354969A1 (en) * 2021-05-10 2022-11-10 Oregon Health & Science University Enhancing viral vector-based gene therapy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
SCHREIBER CLAIRE A., HOLDITCH SARA, IKEDA YASUHIRO: "307. Hitting the Kidney from All Angles: Using Direct Intrarenal Pelvis Injection Combined with Serotype and Promoter Targeting to Achieve Renal AAV Gene Delivery", MOLECULAR THERAPY, ELSEVIER INC, UNITED STATES, vol. 24, 1 May 2016 (2016-05-01), United States, pages S124, XP093291526, ISSN: 1525-0016, DOI: 10.1016/S1525-0016(16)33116-1 *

Also Published As

Publication number Publication date
WO2025019295A2 (fr) 2025-01-23

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