AU2021224729A1 - Methods for reducing HTT expression - Google Patents

Methods for reducing HTT expression Download PDF

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Publication number
AU2021224729A1
AU2021224729A1 AU2021224729A AU2021224729A AU2021224729A1 AU 2021224729 A1 AU2021224729 A1 AU 2021224729A1 AU 2021224729 A AU2021224729 A AU 2021224729A AU 2021224729 A AU2021224729 A AU 2021224729A AU 2021224729 A1 AU2021224729 A1 AU 2021224729A1
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AU
Australia
Prior art keywords
less
weeks
administering
once
modified oligonucleotide
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Pending
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AU2021224729A
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English (en)
Inventor
C. Frank Bennett
Roger Lane
Daniel A. Norris
Anne V. SMITH
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.)
Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of AU2021224729A1 publication Critical patent/AU2021224729A1/en
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • 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
    • C12N2310/31—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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Steroid Compounds (AREA)
  • Saccharide Compounds (AREA)
AU2021224729A 2020-02-21 2021-02-19 Methods for reducing HTT expression Pending AU2021224729A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062980010P 2020-02-21 2020-02-21
US62/980,010 2020-02-21
PCT/US2021/018681 WO2021168183A1 (en) 2020-02-21 2021-02-19 Methods for reducing htt expression

Publications (1)

Publication Number Publication Date
AU2021224729A1 true AU2021224729A1 (en) 2022-09-08

Family

ID=77391689

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021224729A Pending AU2021224729A1 (en) 2020-02-21 2021-02-19 Methods for reducing HTT expression

Country Status (12)

Country Link
US (1) US20230108340A1 (https=)
EP (1) EP4107269A4 (https=)
JP (1) JP2023515466A (https=)
KR (1) KR20220144837A (https=)
CN (1) CN115279903A (https=)
AU (1) AU2021224729A1 (https=)
BR (1) BR112022016370A2 (https=)
CA (1) CA3172336A1 (https=)
IL (1) IL295625A (https=)
MX (1) MX2022010204A (https=)
WO (1) WO2021168183A1 (https=)
ZA (1) ZA202209112B (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025265013A2 (en) 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1991677A2 (en) * 2006-01-26 2008-11-19 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
NZ714887A (en) * 2009-09-11 2019-07-26 Ionis Pharmaceuticals Inc Modulation of huntingtin expression
LT3052628T (lt) * 2013-10-04 2020-09-10 Alnylam Pharmaceuticals, Inc. Kompozicijos ir būdai alas1 geno raiškai nuslopinti
EP3797780B1 (en) * 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
IL320434A (en) * 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
US11268093B2 (en) * 2017-01-03 2022-03-08 Rula Zain-Luqman Therapeutic method for huntington's disease
CN114286710A (zh) * 2019-06-28 2022-04-05 宾州研究基金会 治疗亨廷顿病的方法和材料

Also Published As

Publication number Publication date
WO2021168183A1 (en) 2021-08-26
IL295625A (en) 2022-10-01
CA3172336A1 (en) 2021-08-26
BR112022016370A2 (pt) 2022-10-04
EP4107269A1 (en) 2022-12-28
MX2022010204A (es) 2022-09-19
JP2023515466A (ja) 2023-04-13
US20230108340A1 (en) 2023-04-06
KR20220144837A (ko) 2022-10-27
EP4107269A4 (en) 2024-11-20
CN115279903A (zh) 2022-11-01
ZA202209112B (en) 2024-10-30

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