AU2021345266A1 - Therapeutic compositions for treating pain via multiple targets - Google Patents

Therapeutic compositions for treating pain via multiple targets Download PDF

Info

Publication number
AU2021345266A1
AU2021345266A1 AU2021345266A AU2021345266A AU2021345266A1 AU 2021345266 A1 AU2021345266 A1 AU 2021345266A1 AU 2021345266 A AU2021345266 A AU 2021345266A AU 2021345266 A AU2021345266 A AU 2021345266A AU 2021345266 A1 AU2021345266 A1 AU 2021345266A1
Authority
AU
Australia
Prior art keywords
composition
oligonucleotide
rna
bases
seq
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
AU2021345266A
Other languages
English (en)
Other versions
AU2021345266A9 (en
Inventor
Sudhir Agrawal
Duncan Brown
Graham T. DEMPSEY
David Gerber
Caitlin LEWARCH
Pin LIU
Owen Mcmanus
Dawei Zhang
Hongkang ZHANG
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.)
Q State Biosciences Inc
Original Assignee
Q State Biosciences 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 Q State Biosciences Inc filed Critical Q State Biosciences Inc
Publication of AU2021345266A1 publication Critical patent/AU2021345266A1/en
Publication of AU2021345266A9 publication Critical patent/AU2021345266A9/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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N2310/315—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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • 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
    • 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
    • C12N2320/31—Combination therapy
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Compounds Of Unknown Constitution (AREA)
AU2021345266A 2020-09-17 2021-09-17 Therapeutic compositions for treating pain via multiple targets Pending AU2021345266A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063079912P 2020-09-17 2020-09-17
US63/079,912 2020-09-17
US202163180875P 2021-04-28 2021-04-28
US63/180,875 2021-04-28
PCT/US2021/050866 WO2022061108A2 (en) 2020-09-17 2021-09-17 Therapeutic compositions for treating pain via multiple targets

Publications (2)

Publication Number Publication Date
AU2021345266A1 true AU2021345266A1 (en) 2023-04-27
AU2021345266A9 AU2021345266A9 (en) 2024-09-05

Family

ID=80775585

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021345266A Pending AU2021345266A1 (en) 2020-09-17 2021-09-17 Therapeutic compositions for treating pain via multiple targets

Country Status (6)

Country Link
US (2) US12152243B2 (https=)
EP (1) EP4214319A4 (https=)
JP (1) JP2023542889A (https=)
AU (1) AU2021345266A1 (https=)
CA (1) CA3195722A1 (https=)
WO (1) WO2022061108A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023542889A (ja) 2020-09-17 2023-10-12 キュー－ステート バイオサイエンシーズ， インコーポレイテッド 複数の標的を介して疼痛を処置するための治療用組成物
WO2025006508A2 (en) * 2023-06-26 2025-01-02 Navega Therapeutics, Inc. Compositions and methods for reducing pain and inflammation through epigenetic modulation
WO2026080897A1 (en) 2024-10-11 2026-04-16 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of chronic pain

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
AU2002255478A1 (en) 2001-01-10 2002-09-12 Pe Corporation (Ny) Kits, such as nucleic acid arrays, comprising a majority of human exons or transcripts, for detecting expression and other uses thereof
GB0120238D0 (en) 2001-08-20 2001-10-10 Univ College Of London Sodium channel regulators and modulators
ATE556714T1 (de) 2002-02-01 2012-05-15 Life Technologies Corp Doppelsträngige oligonukleotide
US7659082B2 (en) 2002-02-19 2010-02-09 Xenon Pharmaceuticals Inc. Methods for identifying analgesic agents
US20040097440A1 (en) 2002-11-11 2004-05-20 Isis Pharmaceuticals Inc. Modulation of jumonji expression
WO2004016754A2 (en) * 2002-08-14 2004-02-26 Pharmacia Corporation ANTISENSE MODULATION OF Nav1.3 EXPRESSION
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
WO2004046160A2 (en) 2002-11-18 2004-06-03 Santaris Pharma A/S Amino-lna, thio-lna and alpha-l-oxy-ln
US7341835B2 (en) 2004-01-13 2008-03-11 Affymetrix, Inc. Methods of analysis of alternative splicing in mouse
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
JP5697297B2 (ja) 2004-05-14 2015-04-08 ロゼッタ ジノミクス リミテッド マイクロｎａｓおよびその使用
AR051829A1 (es) 2004-10-27 2007-02-14 Schering Corp Composiciones y metodos para inhibicion de nav 1 mediante arn corto de interferencia
US20070154896A1 (en) 2005-02-11 2007-07-05 International Business Machines Corporation System and method for identification of MicroRNA target sites and corresponding targeting MicroRNA sequences
CA2626584A1 (en) 2005-11-04 2007-05-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of nav1.8 gene
US8178503B2 (en) 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
US7901882B2 (en) 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
US20100105134A1 (en) 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
WO2009033027A2 (en) 2007-09-05 2009-03-12 Medtronic, Inc. Suppression of scn9a gene expression and/or function for the treatment of pain
FR2929292A1 (fr) 2008-03-28 2009-10-02 Exonhit Therapeutics S A Sa Procede et methodes de diagnostic de la maladie d'alzheimer
WO2009143277A2 (en) * 2008-05-20 2009-11-26 Intradigm Corporation Compositions comprising hscn9a sirna and methods of use thereof
CN109112126B (zh) 2010-06-23 2024-06-14 库尔纳公司 通过抑制电压门控钠通道α亚基(SCNA)的天然反义转录物而治疗SCNA相关疾病
LT2841578T (lt) 2012-04-23 2017-09-25 Biomarin Technologies B.V. Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
MX2015010749A (es) 2013-03-14 2015-11-30 Regeneron Pharma Anticuerpos humanos para nav1.7.
FR3007656B1 (fr) 2013-06-28 2015-06-26 Galderma Res & Dev Modulateurs du canal sodique nav1.9 pour le traitement d'une maladie cutanee inflammatoire et methodes diagnostiques
CA2954791C (en) 2014-07-14 2025-11-18 The Regents Of The University Of California CRISPR/CAS TRANSCRIPTIONAL MODULATION
CA2958524A1 (en) 2014-08-20 2016-02-25 Lifesplice Pharma Llc Splice modulating oligonucleotides and methods of use thereof
WO2016027168A2 (en) 2014-08-20 2016-02-25 Lifesplice Pharma Llc Splice modulating oligonucleotides and methods of use thereof
MX2017012426A (es) 2015-04-03 2018-01-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tmprss6.
WO2017139413A1 (en) 2016-02-08 2017-08-17 Circuit Therapeutics, Inc. System and method to modulate pain and itch through cutaneous transfer of genetic information
KR102189358B1 (ko) 2016-06-03 2020-12-09 주식회사 씨젠 올리고뉴클레오타이드의 특이성 평가
JP7305534B2 (ja) 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
CN109843914B (zh) 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
US11504391B1 (en) * 2016-11-23 2022-11-22 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect
SG11201905601VA (en) * 2017-01-24 2019-08-27 Olipass Corp Scn9a antisense pain killer
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3810776A1 (en) 2018-06-22 2021-04-28 F. Hoffmann-La Roche AG Oligonucleotides for modulating scn9a expression
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
JP2023542889A (ja) 2020-09-17 2023-10-12 キュー－ステート バイオサイエンシーズ， インコーポレイテッド 複数の標的を介して疼痛を処置するための治療用組成物
WO2022232395A2 (en) 2021-04-28 2022-11-03 Q-State Biosciences, Inc. Therapeutic compositions for treating pain via multiple targets

Also Published As

Publication number Publication date
EP4214319A2 (en) 2023-07-26
EP4214319A4 (en) 2025-12-03
CA3195722A1 (en) 2022-03-24
US12152243B2 (en) 2024-11-26
US20250163419A1 (en) 2025-05-22
AU2021345266A9 (en) 2024-09-05
WO2022061108A3 (en) 2022-04-28
JP2023542889A (ja) 2023-10-12
US20220112496A1 (en) 2022-04-14
WO2022061108A2 (en) 2022-03-24

Similar Documents

Publication Publication Date Title
US12344841B2 (en) Chimeric double-stranded nucleic acid
US20260069625A1 (en) Organic compositions to treat kras-related diseases
US20250163419A1 (en) Therapeutic compositions for treating pain via multiple targets
ES2567132T3 (es) Modulación de la expresión del receptor de la hormona de crecimiento y de IGF-I
JP2021527437A (ja) Ｓｃｎ９ａ発現を調節するためのオリゴヌクレオチド
US20220259601A1 (en) Ube3a antisense therapeutics
US20240209377A1 (en) Therapeutic compositions for treating pain via multiple targets
CN116670276A (zh) 用于通过多个靶标治疗疼痛的治疗组合物
US20250277222A1 (en) Therapeutic compositions for treating pain via multiple targets
JP7288052B2 (ja) Ｓｃｎ９ａ発現を阻害するための増強されたオリゴヌクレオチド
US20220370487A1 (en) Compositions targeting sodium channel 1.6
US20250304968A1 (en) Ube3a antisense therapeutics
WO2020007702A1 (en) Antisense oligonucleotides targeting bcl2l11
WO2024187102A2 (en) Compositions and methods for modulating c3
CN121773207A (zh) 用于癌症的tgfb2-irf5治疗剂

Legal Events

Date Code Title Description
SREP Specification republished