WO2023215321A3 - Polynucleotide compositions for the treatment of premature termination diseases - Google Patents

Polynucleotide compositions for the treatment of premature termination diseases Download PDF

Info

Publication number
WO2023215321A3
WO2023215321A3 PCT/US2023/020739 US2023020739W WO2023215321A3 WO 2023215321 A3 WO2023215321 A3 WO 2023215321A3 US 2023020739 W US2023020739 W US 2023020739W WO 2023215321 A3 WO2023215321 A3 WO 2023215321A3
Authority
WO
WIPO (PCT)
Prior art keywords
diseases
polynucleotide compositions
treatment
premature termination
oligonucleotide
Prior art date
Application number
PCT/US2023/020739
Other languages
French (fr)
Other versions
WO2023215321A2 (en
Inventor
Yan Shuen POON
Anurag Agarwal
Racquel Noh BRACKEN
Eliot Leo COFFEY
Anna Maria BADNER
Original Assignee
Confluence Therapeutics, 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 Confluence Therapeutics, Inc. filed Critical Confluence Therapeutics, Inc.
Publication of WO2023215321A2 publication Critical patent/WO2023215321A2/en
Publication of WO2023215321A3 publication Critical patent/WO2023215321A3/en

Links

Classifications

    • 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
    • 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
    • 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
    • C12N2310/11—Antisense

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Polynucleotide compositions comprising (a) a complementary oligonucleotide comprising at least 10 consecutive nucleobases having at least 80% complementarity to a target gene having a premature UGA stop codon; (b) an optional linker selected from the group consisting of (i) an oligonucleotide and (ii) a chemical linker; and (c) an oligonucleotide comprising a SECIS element, or pharmaceutically acceptable salts thereof are disclosed herein.
PCT/US2023/020739 2022-05-02 2023-05-02 Polynucleotide compositions for the treatment of premature termination diseases WO2023215321A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263337523P 2022-05-02 2022-05-02
US63/337,523 2022-05-02

Publications (2)

Publication Number Publication Date
WO2023215321A2 WO2023215321A2 (en) 2023-11-09
WO2023215321A3 true WO2023215321A3 (en) 2024-05-16

Family

ID=88646955

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/020739 WO2023215321A2 (en) 2022-05-02 2023-05-02 Polynucleotide compositions for the treatment of premature termination diseases

Country Status (1)

Country Link
WO (1) WO2023215321A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060099673A1 (en) * 2001-08-02 2006-05-11 Hansjorg Hauser Novel recombinant gene expression method by stop codon suppression
US20090269807A1 (en) * 2008-04-29 2009-10-29 Board Of Regents Of The University Of Nebraska- Lincoln Compositions and Methods for the Expression of Selenoproteins in Eukaryotic Cells
US20200283762A1 (en) * 2013-09-04 2020-09-10 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060099673A1 (en) * 2001-08-02 2006-05-11 Hansjorg Hauser Novel recombinant gene expression method by stop codon suppression
US20090269807A1 (en) * 2008-04-29 2009-10-29 Board Of Regents Of The University Of Nebraska- Lincoln Compositions and Methods for the Expression of Selenoproteins in Eukaryotic Cells
US20200283762A1 (en) * 2013-09-04 2020-09-10 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

Also Published As

Publication number Publication date
WO2023215321A2 (en) 2023-11-09

Similar Documents

Publication Publication Date Title
US10767174B2 (en) Method for RGEN RNP delivery using 5′-phosphate removed RNA
MX2023004383A (en) Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste).
WO2008116860A3 (en) Dsrna compositions and methods for treating hpv infections
WO2004065601A3 (en) Lipophilic derivatives of double-stranded ribonucleic acid
WO2005116002A3 (en) Inhibitors of 11-beta-hydroxy steroid dehydrogenase type 1
MX2007008065A (en) Compositions and methods for modulating gene expression using self-protected oligonucleotides.
WO2008132234A4 (en) Rna antagonist compounds for the modulation of beta-catenin
MX2021011921A (en) Compound, method and pharmaceutical composition for dux4 expression adjustment.
WO2004039957A3 (en) Inhibition of gene expression using rna interfering agents
MX2023003810A (en) Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases.
EP4357334A3 (en) Conjugated oligonucleotide compounds, methods of making and uses thereof
EP1594885B8 (en) Medicament for inhibiting tumour growth
CA3155921A1 (en) Huntingtin (htt) irna agent compositions and methods of use thereof
MX2023008469A (en) Modified double stranded oligonucleotides.
WO2004001013A3 (en) Inhibition of gene expression in vertebrates using double-stranded rna (rnai)
WO2020099482A3 (en) Angptl 3 oligonucleotides influencing the regulation of the fatty acid metabolism
WO2007111998A3 (en) Dsrna compositions and methods for treating hpv infection
WO2023215321A3 (en) Polynucleotide compositions for the treatment of premature termination diseases
WO2024011203A3 (en) Ocular vectors and uses thereof
WO2023178144A3 (en) Galnac compositions for improving sirna bioavailability
WO2023091644A3 (en) Hsd17b13-related double stranded oligonucleotide compositions and methods relating thereto
WO2009044294A3 (en) Means and methods for the treatment of cataract and presbyopia
WO2022271836A3 (en) Pikfyve antisense oligonucleotides
WO2023009817A3 (en) Phosphate prodrugs of cannabinoids
WO2023004422A8 (en) Crispr-associated transposon systems and methods of using same

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 23799946

Country of ref document: EP

Kind code of ref document: A2