AU2023232895A1 - Methods of treatment of gnaq and gna11 driven disease - Google Patents

Methods of treatment of gnaq and gna11 driven disease Download PDF

Info

Publication number
AU2023232895A1
AU2023232895A1 AU2023232895A AU2023232895A AU2023232895A1 AU 2023232895 A1 AU2023232895 A1 AU 2023232895A1 AU 2023232895 A AU2023232895 A AU 2023232895A AU 2023232895 A AU2023232895 A AU 2023232895A AU 2023232895 A1 AU2023232895 A1 AU 2023232895A1
Authority
AU
Australia
Prior art keywords
seq
acid molecule
nucleic acid
strand
sequence
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
AU2023232895A
Other languages
English (en)
Inventor
Veronica KINSLER
Davide ZECCHIN
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.)
Francis Crick Institute Ltd
Original Assignee
Francis Crick Institute 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
Priority claimed from GBGB2203164.5A external-priority patent/GB202203164D0/en
Priority claimed from GBGB2203233.8A external-priority patent/GB202203233D0/en
Application filed by Francis Crick Institute Ltd filed Critical Francis Crick Institute Ltd
Publication of AU2023232895A1 publication Critical patent/AU2023232895A1/en
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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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

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 Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Dermatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2023232895A 2022-03-07 2023-03-07 Methods of treatment of gnaq and gna11 driven disease Pending AU2023232895A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB2203164.5 2022-03-07
GBGB2203164.5A GB202203164D0 (en) 2022-03-07 2022-03-07 Methods of treatment of gnaq and gna11 mosaicism
GBGB2203233.8A GB202203233D0 (en) 2022-03-08 2022-03-08 Methods of treatment of GNAQ and GNA11 driven disease
GB2203233.8 2022-03-08
PCT/EP2023/055817 WO2023170109A1 (en) 2022-03-07 2023-03-07 Methods of treatment of gnaq and gna11 driven disease

Publications (1)

Publication Number Publication Date
AU2023232895A1 true AU2023232895A1 (en) 2024-08-29

Family

ID=85556425

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2023232895A Pending AU2023232895A1 (en) 2022-03-07 2023-03-07 Methods of treatment of gnaq and gna11 driven disease

Country Status (5)

Country Link
US (1) US20250230435A1 (https=)
EP (1) EP4490290A1 (https=)
JP (1) JP2025509242A (https=)
AU (1) AU2023232895A1 (https=)
WO (1) WO2023170109A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025245193A1 (en) * 2024-05-21 2025-11-27 The University Of North Carolina At Chapel Hill Methods and compositions for inhibition of mutant gnaq and gna11

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2746514C (en) * 2008-12-10 2018-11-27 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
US10544461B2 (en) * 2013-04-16 2020-01-28 The Johns Hopkins University Diagnostic and prognostic test for sturge-weber syndrome, klippel-trenaunay-weber syndrome, and port-wine stains (PWSS)
CN105218668B (zh) * 2015-10-30 2020-03-24 山东农业大学 马耳他型布氏杆菌的EF-Tu蛋白单克隆抗体MAb及其制备方法与应用
US20210403864A1 (en) * 2018-11-06 2021-12-30 Memorial Sloan Kettering Cancer Center Methods and compositions for treating activated g-alpha q mutant cancers or melanocytic malignancies

Also Published As

Publication number Publication date
JP2025509242A (ja) 2025-04-11
US20250230435A1 (en) 2025-07-17
EP4490290A1 (en) 2025-01-15
WO2023170109A1 (en) 2023-09-14

Similar Documents

Publication Publication Date Title
JP7749645B2 (ja) Ａｎｇｐｔｌ３発現を阻害する組成物及び方法
JP6637121B2 (ja) 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法
ES2543004T3 (es) Composiciones y métodos para inhibir la expresión de transtiretina
ES2984631T3 (es) Composiciones y procedimientos para disminuir la expresión de tau
JP2019500345A (ja) 肝臓病の処置のための組成物および方法
TWI857290B (zh) 用於調節ｐｎｐｌａ３表現之組合物及方法
CN104302768A (zh) 治疗β-联蛋白相关疾病的有机组合物
JP7560616B2 (ja) 膜貫通型セリンプロテアーゼ６（ｔｍｐｒｓｓ６）発現を阻害するための組成物および方法
US20250230435A1 (en) Methods of treatment of gnaq and gna11 driven disease
CN103764173A (zh) 用于治疗骨骼肌病的组合物和方法
CN118843690A (zh) 治疗gnaq和gna11驱动的疾病的方法
JP2024532019A (ja) アルファ－１アンチトリプシン発現を阻害するための組成物及び方法
WO2025088215A1 (en) Methods of treatment of melanocytic disease
EP4608978A2 (en) Methods of treatment of melanocytic disease
WO2024089274A2 (en) Methods of treatment of melanocytic disease
US20090004660A1 (en) Method for detecting neuroblastoma and its malignancy and method for suppressing the same
JP2026513654A (ja) メラノサイト性疾患の治療方法
US20250283086A1 (en) Compositions and methods for treatment of kidney disease
TW202345873A (zh) 調節ｓｃａｐ活性之組合物及方法
WO2026041910A2 (en) Compositions and methods for inhibiting xdh expression