CA3176204A1 - Oligonucleotides for msh3 modulation - Google Patents

Oligonucleotides for msh3 modulation Download PDF

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Publication number
CA3176204A1
CA3176204A1 CA3176204A CA3176204A CA3176204A1 CA 3176204 A1 CA3176204 A1 CA 3176204A1 CA 3176204 A CA3176204 A CA 3176204A CA 3176204 A CA3176204 A CA 3176204A CA 3176204 A1 CA3176204 A1 CA 3176204A1
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CA
Canada
Prior art keywords
nucleotides
dsrna
antisense strand
sense strand
strand
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
CA3176204A
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English (en)
French (fr)
Inventor
Anastasia Khvorova
Chantal FERGUSON
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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Publication of CA3176204A1 publication Critical patent/CA3176204A1/en
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    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • 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
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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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)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurosurgery (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3176204A 2020-04-20 2021-04-20 Oligonucleotides for msh3 modulation Pending CA3176204A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063012603P 2020-04-20 2020-04-20
US63/012,603 2020-04-20
PCT/US2021/028166 WO2021216556A2 (en) 2020-04-20 2021-04-20 Oligonucleotides for msh3 modulation

Publications (1)

Publication Number Publication Date
CA3176204A1 true CA3176204A1 (en) 2021-10-28

Family

ID=78270051

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3176204A Pending CA3176204A1 (en) 2020-04-20 2021-04-20 Oligonucleotides for msh3 modulation

Country Status (6)

Country Link
US (1) US20210355491A1 (enExample)
EP (1) EP4138860A4 (enExample)
JP (1) JP2023522701A (enExample)
AU (1) AU2021260581A1 (enExample)
CA (1) CA3176204A1 (enExample)
WO (1) WO2021216556A2 (enExample)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
WO2020041769A1 (en) 2018-08-23 2020-02-27 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
EP4146807A4 (en) * 2020-05-08 2024-09-04 Takeda Pharmaceuticals U.S.A., Inc. METHODS OF TREATING NUCLEOTIDE AMPLIFICATION DISEASES ASSOCIATED WITH MSH3 ACTIVITY
CA3174079A1 (en) 2020-05-26 2021-12-02 Anastasia Khvorova Synthetic oligonucleotides having regions of block and cluster modifications
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
EP4164656A4 (en) * 2020-06-11 2025-10-22 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING MSH3 EXPRESSION
BR112023019981A2 (pt) 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas
EP4423272A2 (en) * 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2023114989A2 (en) * 2021-12-17 2023-06-22 University Of Massachusetts Oligonucleotides for mlh3 modulation
US20240035027A1 (en) * 2022-05-04 2024-02-01 University Of Massachusetts Oligonucleotides for pms2 modulation
WO2023225495A2 (en) * 2022-05-16 2023-11-23 Atalanta Therapeutics, Inc. Compositions and methods for treatment of microsatellite dna expansion disorders
WO2024064954A2 (en) * 2022-09-23 2024-03-28 The Children's Hospital Of Philadelphia Compositions and methods for treating huntington's disease and related disorders
CN122341734A (zh) * 2023-10-11 2026-07-03 优尼科生物制药有限公司 重复扩增障碍中msh3的核酸调节

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AUPO974597A0 (en) * 1997-10-10 1997-11-06 Rhone-Poulenc Agro Methods for obtaining plant varieties
US7361468B2 (en) * 2004-07-02 2008-04-22 Affymetrix, Inc. Methods for genotyping polymorphisms in humans
US20150307876A1 (en) * 2012-02-10 2015-10-29 Regents Of The University Of Minnesota Dna assimilation
WO2015171918A2 (en) * 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
CA3255657A1 (en) * 2015-02-10 2025-12-01 Genzyme Corporation VARIANT RNAi
RS60762B1 (sr) * 2015-04-03 2020-10-30 Univ Massachusetts Oligonukleotidna jedinjenja za ciljane irnk hantingtina
US20170009304A1 (en) * 2015-07-07 2017-01-12 Splicingcodes.Com Method and kit for detecting fusion transcripts
WO2017132669A1 (en) * 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
WO2020117702A1 (en) * 2018-12-03 2020-06-11 Triplet Therapeutics, Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with msh3 activity

Also Published As

Publication number Publication date
JP2023522701A (ja) 2023-05-31
EP4138860A4 (en) 2024-08-07
WO2021216556A2 (en) 2021-10-28
AU2021260581A1 (en) 2022-11-24
WO2021216556A3 (en) 2022-03-03
US20210355491A1 (en) 2021-11-18
EP4138860A2 (en) 2023-03-01

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