AU2023277730A1 - Oligonucleotides conjugated to oleic acid and uses thereof - Google Patents

Oligonucleotides conjugated to oleic acid and uses thereof Download PDF

Info

Publication number
AU2023277730A1
AU2023277730A1 AU2023277730A AU2023277730A AU2023277730A1 AU 2023277730 A1 AU2023277730 A1 AU 2023277730A1 AU 2023277730 A AU2023277730 A AU 2023277730A AU 2023277730 A AU2023277730 A AU 2023277730A AU 2023277730 A1 AU2023277730 A1 AU 2023277730A1
Authority
AU
Australia
Prior art keywords
oligonucleotide
seq
molecule
analogue
mir
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
AU2023277730A
Other languages
English (en)
Inventor
Rubén Artero Allepuz
Estefanía CERRO HERREROS
Irene GONZÁLEZ MARTÍNEZ
María Beatriz LLAMUSÍ TROISI
Eric G. Marcusson
Nerea MORENO CERVERA
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.)
Arthex Biotech SL
Universitat de Valencia
Original Assignee
Arthex Biotech S L
Universitat de Valencia
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 Arthex Biotech S L, Universitat de Valencia filed Critical Arthex Biotech S L
Publication of AU2023277730A1 publication Critical patent/AU2023277730A1/en
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/542Carboxylic acids, e.g. a fatty acid or an amino acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/113Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
AU2023277730A 2022-05-23 2023-05-23 Oligonucleotides conjugated to oleic acid and uses thereof Pending AU2023277730A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP22382493.9 2022-05-23
EP22382493.9A EP4282964A1 (en) 2022-05-23 2022-05-23 Oligonucleotides conjugated to oleic acid and uses thereof
PCT/EP2023/063829 WO2023227622A1 (en) 2022-05-23 2023-05-23 Oligonucleotides conjugated to oleic acid and uses thereof

Publications (1)

Publication Number Publication Date
AU2023277730A1 true AU2023277730A1 (en) 2025-01-02

Family

ID=81941129

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2023277730A Pending AU2023277730A1 (en) 2022-05-23 2023-05-23 Oligonucleotides conjugated to oleic acid and uses thereof

Country Status (9)

Country Link
US (1) US12116575B2 (https=)
EP (3) EP4282964A1 (https=)
JP (2) JP2025522282A (https=)
CN (1) CN119816594A (https=)
AU (1) AU2023277730A1 (https=)
CA (1) CA3255781A1 (https=)
IL (1) IL317141A (https=)
TW (1) TW202411424A (https=)
WO (1) WO2023227622A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4282964A1 (en) * 2022-05-23 2023-11-29 Universitat de Valéncia Oligonucleotides conjugated to oleic acid and uses thereof
WO2025114442A1 (en) * 2023-11-28 2025-06-05 Arthex Biotech S.L. Oligonucleotides conjugated to oleic acid and uses thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7683036B2 (en) * 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
US10077443B2 (en) * 2012-11-15 2018-09-18 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
ES3047792T3 (en) * 2014-07-14 2025-12-04 Univ California Crispr/cas transcriptional modulation
WO2016201272A1 (en) * 2015-06-12 2016-12-15 King Abdulaziz City For Science And Technology Method of diagnosing patients with conditions caused by mendelian mutations
JP7511326B2 (ja) * 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
ES2659845B1 (es) 2016-09-19 2019-01-04 Univ Valencia Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello
TW202019441A (zh) * 2018-07-20 2020-06-01 美商雷格勒斯治療公司 用於經口遞送寡核苷酸之方法
EP4081302A4 (en) 2019-12-23 2025-01-29 University Of Massachusetts OLIGONUCLEOTIDES FOR TISSUE-SPECIFIC GENE EXPRESSION MODULATION
EP4282964A1 (en) * 2022-05-23 2023-11-29 Universitat de Valéncia Oligonucleotides conjugated to oleic acid and uses thereof

Also Published As

Publication number Publication date
US12116575B2 (en) 2024-10-15
EP4282966A1 (en) 2023-11-29
US20230407305A1 (en) 2023-12-21
TW202411424A (zh) 2024-03-16
JP2025522282A (ja) 2025-07-15
JP7663873B2 (ja) 2025-04-17
CN119816594A (zh) 2025-04-11
EP4282964A1 (en) 2023-11-29
JP2023172954A (ja) 2023-12-06
IL317141A (en) 2025-01-01
CA3255781A1 (en) 2023-11-30
EP4486891A1 (en) 2025-01-08
WO2023227622A1 (en) 2023-11-30

Similar Documents

Publication Publication Date Title
CA2862628C (en) Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
CN109072238B (zh) 使用靶向结缔组织生长因子的rna复合物治疗特发性肺纤维化
JP2011510678A (ja) Dna反復不安定性関連遺伝性障害を治療するための方法及び手段
EP3128008A2 (en) Novel double-stranded oligo rna and pharmaceutical composition comprising same for preventing or treating fibrosis or respiratory diseases
US20230348906A1 (en) Dux4 inhibitors and methods of use thereof
US12116575B2 (en) Oligonucleotides conjugated to oleic acid and uses thereof
JP2019525918A (ja) Smn2の調節のための化合物及び方法
CA3022874A1 (en) Treatment of atopic dermatitis and asthma using rna complexes that target il4ra, trpa1, or f2rl1
WO2022031237A1 (en) Modulation of signal transducer and activator of transcription 3 (stat3) expression
TW201827596A (zh) 抑制補體b因子之表現之核酸
TW201730341A (zh) 抑制補體b因子之表現之反義寡核苷酸
JP2019527549A (ja) 転写プロセシングの調節のための化合物及び方法
KR20200014320A (ko) Apcs의 발현을 억제하는 핵산
WO2025114442A1 (en) Oligonucleotides conjugated to oleic acid and uses thereof
CN115698290A (zh) 用于治疗神经系统疾病的补体组分4抑制剂以及使用它们的相关组合物、系统和方法
EP4149486A1 (en) Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
EP4046631A1 (en) Msi2 as a therapeutic target for the treatment of myotonic dystrophy
EP4446414A1 (en) Antisense oligonucleotide complex
HK40084486A (en) Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
WO2026078062A1 (en) Antisense oligonucleotides targeting tert and uses thereof in the treatment of cancers
CN115605592A (zh) 用于治疗神经系统疾病的补体组分c1r抑制剂以及相关的组合物、系统和使用它们的方法
WO2023168202A2 (en) Certain dux4 inhibitors and methods of use thereof
HK40085370A (en) Complement component 4 inhibitors for treating neurological diseases, and related compositons, systems and methods of using same