EP4728074A2 - Systèmes, procédés et compositions pour déréprimer pkd1 - Google Patents

Systèmes, procédés et compositions pour déréprimer pkd1

Info

Publication number
EP4728074A2
EP4728074A2 EP24824193.7A EP24824193A EP4728074A2 EP 4728074 A2 EP4728074 A2 EP 4728074A2 EP 24824193 A EP24824193 A EP 24824193A EP 4728074 A2 EP4728074 A2 EP 4728074A2
Authority
EP
European Patent Office
Prior art keywords
polynucleotide
binding domain
sequence
seq
grna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP24824193.7A
Other languages
German (de)
English (en)
Inventor
Demetrios Samuel MAXIM
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nephrogen Inc
Original Assignee
Nephrogen Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Nephrogen Inc filed Critical Nephrogen Inc
Publication of EP4728074A2 publication Critical patent/EP4728074A2/fr
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/11—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
    • 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
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

L'invention concerne un système pour empêcher un miARN de la famille des miARN-17 de se lier à la 3'UTR de PKD1, le système comprenant : un ARNg ; et un domaine de liaison aux nucléotides programmable par un polynucléotide, le système modifiant un site de liaison d'un miARN de la famille des miARN-17 dans la 3'UTR de PKD1, empêchant ainsi la liaison du miARN de la famille des miARN-17 et déréprimant l'ARNm de PKD1.
EP24824193.7A 2023-06-13 2024-06-13 Systèmes, procédés et compositions pour déréprimer pkd1 Pending EP4728074A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363472794P 2023-06-13 2023-06-13
PCT/US2024/033918 WO2024259175A2 (fr) 2023-06-13 2024-06-13 Systèmes, procédés et compositions pour déréprimer pkd1

Publications (1)

Publication Number Publication Date
EP4728074A2 true EP4728074A2 (fr) 2026-04-22

Family

ID=93852799

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24824193.7A Pending EP4728074A2 (fr) 2023-06-13 2024-06-13 Systèmes, procédés et compositions pour déréprimer pkd1

Country Status (2)

Country Link
EP (1) EP4728074A2 (fr)
WO (1) WO2024259175A2 (fr)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4680746A1 (fr) * 2023-06-16 2026-01-21 Pyc Therapeutics Limited Compositions et méthodes de traitement d'une maladie rénale
WO2025196338A1 (fr) * 2024-03-22 2025-09-25 Helex Inc. Méthodes et compositions pour le traitement d'une polykystose rénale autosomique dominante
WO2025208171A1 (fr) * 2024-04-04 2025-10-09 PYC Therapeutics Limited Compositions et méthodes avancées de traitement d'une maladie rénale

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2395781C (fr) * 2000-07-13 2010-04-13 The Johns Hopkins University School Of Medicine Detection et traitement de polykystose renale
US7217807B2 (en) * 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
EP4176434A4 (fr) * 2020-05-29 2024-09-11 The General Hospital Corporation Systèmes et procédés de modification stable et héréditaire par édition de précision (shape)

Also Published As

Publication number Publication date
WO2024259175A2 (fr) 2024-12-19
WO2024259175A3 (fr) 2025-01-16

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