AU2011311344B2 - Short RNA molecules - Google Patents

Short RNA molecules Download PDF

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Publication number
AU2011311344B2
AU2011311344B2 AU2011311344A AU2011311344A AU2011311344B2 AU 2011311344 B2 AU2011311344 B2 AU 2011311344B2 AU 2011311344 A AU2011311344 A AU 2011311344A AU 2011311344 A AU2011311344 A AU 2011311344A AU 2011311344 B2 AU2011311344 B2 AU 2011311344B2
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AU
Australia
Prior art keywords
rna
cells
cell
insulin
short
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Ceased
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AU2011311344A
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English (en)
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AU2011311344A1 (en
Inventor
Pal Saetrom
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Mina Therapeutics Ltd
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Mina Therapeutics Ltd
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Publication date
Priority claimed from GBGB1016989.4A external-priority patent/GB201016989D0/en
Priority claimed from GBGB1103745.4A external-priority patent/GB201103745D0/en
Application filed by Mina Therapeutics Ltd filed Critical Mina Therapeutics Ltd
Publication of AU2011311344A1 publication Critical patent/AU2011311344A1/en
Application granted granted Critical
Publication of AU2011311344B2 publication Critical patent/AU2011311344B2/en
Ceased legal-status Critical Current
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/48—Drugs for disorders of the endocrine system of the pancreatic hormones
    • A61P5/50—Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
    • 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
    • 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
    • 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • 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
    • 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
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/35—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)
  • 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)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Endocrinology (AREA)
  • Oncology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Emergency Medicine (AREA)
  • Child & Adolescent Psychology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2011311344A 2010-10-08 2011-10-10 Short RNA molecules Ceased AU2011311344B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB1016989.4 2010-10-08
GBGB1016989.4A GB201016989D0 (en) 2010-10-08 2010-10-08 Cell induction
GBGB1103745.4A GB201103745D0 (en) 2011-03-07 2011-03-07 Cell induction
GB1103745.4 2011-03-07
PCT/GB2011/051940 WO2012046084A2 (en) 2010-10-08 2011-10-10 Short rna molecules

Publications (2)

Publication Number Publication Date
AU2011311344A1 AU2011311344A1 (en) 2013-05-30
AU2011311344B2 true AU2011311344B2 (en) 2016-09-08

Family

ID=44860439

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2011311344A Ceased AU2011311344B2 (en) 2010-10-08 2011-10-10 Short RNA molecules

Country Status (8)

Country Link
US (6) US8835400B2 (https=)
EP (2) EP2625273B1 (https=)
JP (1) JP5681955B2 (https=)
KR (1) KR20130132795A (https=)
CN (1) CN103314108B (https=)
AU (1) AU2011311344B2 (https=)
CA (1) CA2817191A1 (https=)
WO (2) WO2012046084A2 (https=)

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KR20130132795A (ko) 2010-10-08 2013-12-05 미나 테라퓨틱스 리미티드 짧은 rna 분자
PT2880167T (pt) 2012-07-31 2018-07-23 Univ Texas Métodos e composições para a indução in vivo da formação de células beta pancreáticas
CA3236835A1 (en) 2013-11-22 2015-05-28 Mina Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
US20170044540A1 (en) * 2014-04-22 2017-02-16 Mina Therapeutics Limited Sarna compositions and methods of use
EP3286318A2 (en) * 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
CN106480028B (zh) * 2016-10-12 2019-04-05 上海市第七人民医院 TPO基因的saRNA分子及其应用
BR112019027086A2 (pt) * 2017-06-21 2020-07-07 Timothy A. Bertram células renais bioativas imunoprivilegiadas para o tratamento de doença renal
KR102007457B1 (ko) 2017-06-30 2019-08-05 동국대학교 산학협력단 Cas9 단백질, 가이드 RNA 및 양친매성 펩타이드를 포함하는 나노복합체 및 이의 용도
WO2019033051A1 (en) * 2017-08-11 2019-02-14 City Of Hope RNA APTAMERS AGAINST THE TRANSFERRIN RECEPTOR (TFR)
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
JP7432521B2 (ja) * 2018-04-10 2024-02-16 ラクティゲン セラピューティクス 新規小分子活性化ｒｎａ
WO2019217571A1 (en) * 2018-05-08 2019-11-14 University Of Miami Materials and methods for the delivery of therapeutic nucleic acids to tissues
AU2019285344A1 (en) 2018-06-15 2020-12-10 Mina Therapeutics Limited Combination therapies comprising C/EBP alpha saRNA
CN111849968A (zh) 2019-04-30 2020-10-30 中美瑞康核酸技术(南通)研究院有限公司 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用
CN114585633A (zh) 2019-08-19 2022-06-03 米纳治疗有限公司 寡核苷酸缀合物组合物和使用方法
EP4329777A1 (en) 2021-04-28 2024-03-06 MiNA Therapeutics Limited Combination therapies comprising c/ebp alpha sarna

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WO2009046397A2 (en) * 2007-10-04 2009-04-09 Board Of Regents, The University Of Texas System Modulating gene expression with agrna and gapmers targeting antisense transcripts
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WO2006113246A2 (en) * 2005-04-15 2006-10-26 The Regents Of The University Of California Small activating rna molecules and methods of use
WO2009046397A2 (en) * 2007-10-04 2009-04-09 Board Of Regents, The University Of Texas System Modulating gene expression with agrna and gapmers targeting antisense transcripts
WO2010108126A2 (en) * 2009-03-19 2010-09-23 Fate Therapeutics, Inc. Reprogramming compositions and methods of using the same

Also Published As

Publication number Publication date
EP2625272A2 (en) 2013-08-14
EP2625273A2 (en) 2013-08-14
US20130289258A1 (en) 2013-10-31
US8835400B2 (en) 2014-09-16
US10774331B2 (en) 2020-09-15
WO2012046085A2 (en) 2012-04-12
JP5681955B2 (ja) 2015-03-11
AU2011311344A1 (en) 2013-05-30
CN103314108B (zh) 2015-08-05
WO2012046084A3 (en) 2012-09-27
EP2625273B1 (en) 2015-01-07
US20200362352A1 (en) 2020-11-19
US9885046B2 (en) 2018-02-06
US20220282256A1 (en) 2022-09-08
WO2012046085A3 (en) 2012-07-19
US11365414B2 (en) 2022-06-21
KR20130132795A (ko) 2013-12-05
CA2817191A1 (en) 2012-04-12
US20180112222A1 (en) 2018-04-26
JP2014501492A (ja) 2014-01-23
US8916534B2 (en) 2014-12-23
WO2012046084A2 (en) 2012-04-12
US20130323211A1 (en) 2013-12-05
US20150176008A1 (en) 2015-06-25
EP2625272B1 (en) 2015-09-16
CN103314108A (zh) 2013-09-18

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