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WO2024165876A3 - Compositions and methods of using c/ebp alpha sarna - Google Patents

Compositions and methods of using c/ebp alpha sarna Download PDF

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Publication number
WO2024165876A3
WO2024165876A3 PCT/GR2024/000008 GR2024000008W WO2024165876A3 WO 2024165876 A3 WO2024165876 A3 WO 2024165876A3 GR 2024000008 W GR2024000008 W GR 2024000008W WO 2024165876 A3 WO2024165876 A3 WO 2024165876A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
compositions
ebp alpha
sarna
alpha sarna
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.)
Ceased
Application number
PCT/GR2024/000008
Other languages
French (fr)
Other versions
WO2024165876A2 (en
Inventor
Konstantina SKOURTI-STATHAKI
Vikash REEBYE
Luke James HASLETT
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.)
Mina Therapeutics Ltd
Original Assignee
Mina Therapeutics Ltd
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 Mina Therapeutics Ltd filed Critical Mina Therapeutics Ltd
Publication of WO2024165876A2 publication Critical patent/WO2024165876A2/en
Publication of WO2024165876A3 publication Critical patent/WO2024165876A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The invention relates to methods of using saRNAs targeting C/EBPα and pharmaceutical compositions comprising the saRNAs to treat diseases such as lysosomal storage disorders.
PCT/GR2024/000008 2023-02-10 2024-02-07 Compositions and methods of using c/ebp alpha sarna Ceased WO2024165876A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363484276P 2023-02-10 2023-02-10
US63/484,276 2023-02-10

Publications (2)

Publication Number Publication Date
WO2024165876A2 WO2024165876A2 (en) 2024-08-15
WO2024165876A3 true WO2024165876A3 (en) 2024-09-19

Family

ID=90458331

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GR2024/000008 Ceased WO2024165876A2 (en) 2023-02-10 2024-02-07 Compositions and methods of using c/ebp alpha sarna

Country Status (1)

Country Link
WO (1) WO2024165876A2 (en)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015075557A2 (en) * 2013-11-22 2015-05-28 Mina Alpha Limited C/ebp alpha compositions and methods of use
WO2016170349A1 (en) * 2015-04-22 2016-10-27 Mina Therapeutics Limited C/ebp alpha sarna compositions and methods of use

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015075557A2 (en) * 2013-11-22 2015-05-28 Mina Alpha Limited C/ebp alpha compositions and methods of use
WO2016170349A1 (en) * 2015-04-22 2016-10-27 Mina Therapeutics Limited C/ebp alpha sarna compositions and methods of use

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
ALBERT KWOK ET AL: "Developing small activating RNA as a therapeutic: current challenges and promises", THERAPEUTIC DELIVERY, vol. 10, no. 3, 26 March 2019 (2019-03-26), GB, pages 151 - 164, XP055751654, ISSN: 2041-5990, DOI: 10.4155/tde-2018-0061 *
HABIB NAGY ET AL: "Drugging transcription factors with small activating RNAs: A novel approach for enhancing bone marrow therapy for monogenic rare diseases", MOLECULAR GENETICS AND METABOLISM, ACADEMIC PRESS, AMSTERDAM, NL, vol. 138, no. 2, 8 February 2023 (2023-02-08), XP087263720, ISSN: 1096-7192, [retrieved on 20230208], DOI: 10.1016/J.YMGME.2022.107134 *
JAKUB TOLAR ET AL: "alpha-L-iduronidase therapy for mucopolysaccharidosis type I", BIOLOGICS: TARGETS AND THERAPY, vol. 2, no. 4, 5 December 2008 (2008-12-05), NZ, pages 743 - 751, XP055290615, ISSN: 1177-5475, DOI: 10.2147/BTT.S3180 *
MICHAEL J PRZYBILLA: "352 Transcriptional Upregulation of [alpha]-L-iduronidase Utilizing Small-Activating RNA Following Bone Marrow Transplant in a Murine Model of Hurler Syndrome", MOLECULAR THERAPY, 1 May 2023 (2023-05-01), pages 189 - 190, XP093163952, Retrieved from the Internet <URL:https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(23)00248-4> *
PRZYBILLA MICHAEL J ET AL: "Treating murine Hurler syndrome utilizing small-activating RNA following bone marrow transplant", MOLECULAR GENETICS AND METABOLISM, ACADEMIC PRESS, AMSTERDAM, NL, vol. 138, no. 2, 8 February 2023 (2023-02-08), XP087263091, ISSN: 1096-7192, [retrieved on 20230208], DOI: 10.1016/J.YMGME.2022.107279 *
REEBYE VIKASH ET AL: "Gene activation of CEBPA using saRNA: preclinical studies of the first in human saRNA drug candidate for liver cancer", ONCOGENE, NATURE PUBLISHING GROUP UK, LONDON, vol. 37, no. 24, 7 March 2018 (2018-03-07), pages 3216 - 3228, XP036822767, ISSN: 0950-9232, [retrieved on 20180307], DOI: 10.1038/S41388-018-0126-2 *
RYAN L. SETTEN ET AL: "Development of MTL-CEBPA: Small Activating RNA Drug for Hepatocellular Carcinoma", CURRENT PHARMACEUTICAL BIOTECHNOLOGY, vol. 19, no. 8, 11 October 2018 (2018-10-11), NL, pages 611 - 621, XP055559534, ISSN: 1389-2010, DOI: 10.2174/1389201019666180611093428 *

Also Published As

Publication number Publication date
WO2024165876A2 (en) 2024-08-15

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