WO2020185651A3 - Compositions and methods for treating huntington's disease - Google Patents

Compositions and methods for treating huntington's disease Download PDF

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Publication number
WO2020185651A3
WO2020185651A3 PCT/US2020/021652 US2020021652W WO2020185651A3 WO 2020185651 A3 WO2020185651 A3 WO 2020185651A3 US 2020021652 W US2020021652 W US 2020021652W WO 2020185651 A3 WO2020185651 A3 WO 2020185651A3
Authority
WO
WIPO (PCT)
Prior art keywords
disease
compositions
methods
treating huntington
huntington
Prior art date
Application number
PCT/US2020/021652
Other languages
French (fr)
Other versions
WO2020185651A2 (en
Inventor
Samuel Ian GUNDERSON
Rafal Goraczniak
Original Assignee
Rutgers, The State University Of New Jersey
Silagene, 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 Rutgers, The State University Of New Jersey, Silagene, Inc. filed Critical Rutgers, The State University Of New Jersey
Priority to BR112021017635A priority Critical patent/BR112021017635A2/en
Priority to CN202080019190.5A priority patent/CN113924364A/en
Priority to AU2020238872A priority patent/AU2020238872A1/en
Priority to JP2021553317A priority patent/JP2022524383A/en
Priority to KR1020217032259A priority patent/KR20220002882A/en
Priority to CA3132388A priority patent/CA3132388A1/en
Priority to EP20770560.9A priority patent/EP3935167A2/en
Priority to SG11202109180RA priority patent/SG11202109180RA/en
Publication of WO2020185651A2 publication Critical patent/WO2020185651A2/en
Publication of WO2020185651A3 publication Critical patent/WO2020185651A3/en
Priority to IL286083A priority patent/IL286083A/en

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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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 N.A.
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid

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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)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Compositions and methods are provided for the inhibition, treatment and/or prevention of Huntington's disease.
PCT/US2020/021652 2019-03-08 2020-03-09 Compositions and methods for treating huntington's disease WO2020185651A2 (en)

Priority Applications (9)

Application Number Priority Date Filing Date Title
BR112021017635A BR112021017635A2 (en) 2019-03-08 2020-03-09 Compositions and methods for treating Huntington's disease
CN202080019190.5A CN113924364A (en) 2019-03-08 2020-03-09 Compositions and methods for treating huntington's disease
AU2020238872A AU2020238872A1 (en) 2019-03-08 2020-03-09 Compositions and methods for treating huntington's disease
JP2021553317A JP2022524383A (en) 2019-03-08 2020-03-09 Compositions and Methods for Treating Huntington's Disease
KR1020217032259A KR20220002882A (en) 2019-03-08 2020-03-09 Compositions and methods for treating Huntington's disease
CA3132388A CA3132388A1 (en) 2019-03-08 2020-03-09 Compositions and methods for treating huntington's disease
EP20770560.9A EP3935167A2 (en) 2019-03-08 2020-03-09 Compositions and methods for treating huntington's disease
SG11202109180RA SG11202109180RA (en) 2019-03-08 2020-03-09 Compositions and methods for treating huntington's disease
IL286083A IL286083A (en) 2019-03-08 2021-09-02 Compositions and methods for treating huntington’s disease

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962815647P 2019-03-08 2019-03-08
US62/815,647 2019-03-08

Publications (2)

Publication Number Publication Date
WO2020185651A2 WO2020185651A2 (en) 2020-09-17
WO2020185651A3 true WO2020185651A3 (en) 2020-10-29

Family

ID=72428020

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2020/021652 WO2020185651A2 (en) 2019-03-08 2020-03-09 Compositions and methods for treating huntington's disease

Country Status (10)

Country Link
EP (1) EP3935167A2 (en)
JP (1) JP2022524383A (en)
KR (1) KR20220002882A (en)
CN (1) CN113924364A (en)
AU (1) AU2020238872A1 (en)
BR (1) BR112021017635A2 (en)
CA (1) CA3132388A1 (en)
IL (1) IL286083A (en)
SG (1) SG11202109180RA (en)
WO (1) WO2020185651A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024035952A1 (en) * 2022-08-12 2024-02-15 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6900016B1 (en) * 2000-09-08 2005-05-31 Applera Corporation Polymorphisms in known genes associated with inflammatory autoimmune disease, methods of detection and uses thereof
US20070044171A1 (en) * 2000-12-14 2007-02-22 Kovalic David K Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20110212520A1 (en) * 2002-08-05 2011-09-01 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
US20150099796A1 (en) * 2007-03-30 2015-04-09 Rutgers, The State University Of New Jersey Compositions and Methods for Gene Silencing

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BRPI0906657A2 (en) * 2008-01-29 2015-07-14 Proyecto Biomedicina Cima Sl Methods and compositions capable of synergistically causing post-transcriptional silencing of gene expression
AU2015252117A1 (en) * 2008-04-11 2015-11-26 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
LT3277814T (en) * 2015-04-03 2020-10-26 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6900016B1 (en) * 2000-09-08 2005-05-31 Applera Corporation Polymorphisms in known genes associated with inflammatory autoimmune disease, methods of detection and uses thereof
US20070044171A1 (en) * 2000-12-14 2007-02-22 Kovalic David K Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20110212520A1 (en) * 2002-08-05 2011-09-01 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
US20150099796A1 (en) * 2007-03-30 2015-04-09 Rutgers, The State University Of New Jersey Compositions and Methods for Gene Silencing

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
RICHFIELD, E ET AL.: "259. Targeted Gene Silencing by U1 Adaptor Oligonucleotides in Preclinical Models of Parkinson's Disease and Huntington's Disease", MOLECULAR THERAPY, vol. 24, no. 1, May 2016 (2016-05-01), pages S102, XP055755794 *

Also Published As

Publication number Publication date
CA3132388A1 (en) 2020-09-17
CN113924364A (en) 2022-01-11
WO2020185651A2 (en) 2020-09-17
BR112021017635A2 (en) 2021-11-09
EP3935167A2 (en) 2022-01-12
KR20220002882A (en) 2022-01-07
JP2022524383A (en) 2022-05-02
IL286083A (en) 2021-10-31
SG11202109180RA (en) 2021-09-29
AU2020238872A1 (en) 2021-09-16

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