CA2817191A1 - Short rna molecules - Google Patents

Short rna molecules Download PDF

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Publication number
CA2817191A1
CA2817191A1 CA2817191A CA2817191A CA2817191A1 CA 2817191 A1 CA2817191 A1 CA 2817191A1 CA 2817191 A CA2817191 A CA 2817191A CA 2817191 A CA2817191 A CA 2817191A CA 2817191 A1 CA2817191 A1 CA 2817191A1
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CA
Canada
Prior art keywords
rna
cells
cell
insulin
short
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.)
Abandoned
Application number
CA2817191A
Other languages
English (en)
French (fr)
Inventor
Pal Saetrom
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.)
Mina Therapeutics Ltd
Original Assignee
Mina Therapeutics Ltd
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
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 CA2817191A1 publication Critical patent/CA2817191A1/en
Abandoned legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1136Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/04Anorexiants; Antiobesity agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/48Drugs for disorders of the endocrine system of the pancreatic hormones
    • A61P5/50Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1135Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/113Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion 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)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Emergency Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Child & Adolescent Psychology (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)
CA2817191A 2010-10-08 2011-10-10 Short rna molecules Abandoned CA2817191A1 (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 (1)

Publication Number Publication Date
CA2817191A1 true CA2817191A1 (en) 2012-04-12

Family

ID=44860439

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2817191A Abandoned CA2817191A1 (en) 2010-10-08 2011-10-10 Short rna molecules

Country Status (8)

Country Link
US (6) US8916534B2 (enExample)
EP (2) EP2625273B1 (enExample)
JP (1) JP5681955B2 (enExample)
KR (1) KR20130132795A (enExample)
CN (1) CN103314108B (enExample)
AU (1) AU2011311344B2 (enExample)
CA (1) CA2817191A1 (enExample)
WO (2) WO2012046084A2 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20130132795A (ko) * 2010-10-08 2013-12-05 미나 테라퓨틱스 리미티드 짧은 rna 분자
EP2880167B1 (en) 2012-07-31 2018-04-18 The Board of Regents of The University of Texas System Methods and compositions for in vivo induction of pancreatic beta cell formation
DK3071696T3 (da) 2013-11-22 2019-10-07 Mina Therapeutics Ltd C/ebp alfa kort aktiverings-rna-sammensætninger og fremgangsmåder til anvendelse
US20170044540A1 (en) * 2014-04-22 2017-02-16 Mina Therapeutics Limited Sarna compositions and methods of use
WO2016170348A2 (en) * 2015-04-22 2016-10-27 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
EP3641811B1 (en) * 2017-06-21 2025-02-26 ProKidney Immunoprivileged bioactive renal cells for the treatment of kidney disease
KR102007457B1 (ko) 2017-06-30 2019-08-05 동국대학교 산학협력단 Cas9 단백질, 가이드 RNA 및 양친매성 펩타이드를 포함하는 나노복합체 및 이의 용도
CN118931907A (zh) * 2017-08-11 2024-11-12 希望之城 转铁蛋白受体(TfR)的RNA适体
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
US20200208152A1 (en) 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
AU2019252204A1 (en) * 2018-04-10 2020-11-26 Ractigen Therapeutics Novel small activating 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
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
US20240207304A1 (en) 2021-04-28 2024-06-27 Mina Therapeutics Limited Combination Therapies Comprising C/EBP Alpha saRNA

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US8193330B2 (en) 1999-04-06 2012-06-05 The Regents Of The University Of California Polynucleotides comprising Neurogenin3 promoter and bHLH encoding domains
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
GB0329449D0 (en) 2003-12-19 2004-01-28 Omnicyte Ltd Stem cells
EP1871426B1 (en) * 2005-04-15 2017-06-07 The Regents of The University of California Small activating rna molecules and their use
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JP5066095B2 (ja) 2005-11-17 2012-11-07 ボード・オブ・リージエンツ,ザ・ユニバーシテイ・オブ・テキサス・システム 染色体dnaに標的化されるオリゴマーによる遺伝子発現の調節
CN103301475B (zh) 2005-12-28 2016-08-03 斯克里普斯研究所 药物组合物和表达载体以及调节基因表达的方法和核酸分子的应用
JP2010519910A (ja) 2007-03-02 2010-06-10 エムディーアールエヌエー,インコーポレイテッド Bcl2遺伝子の発現を抑制するための核酸化合物およびその使用
WO2008109556A1 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting telomerase gene expression and uses thereof
EP3128015A3 (en) 2007-05-29 2017-05-03 Christopher B. Reid A method for providing a desired cell population capable of further differentiation in vivo
CA2701639A1 (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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KR20110006672A (ko) 2008-04-07 2011-01-20 뉴포텐셜, 인크. Hdac 조절제의 사용을 통한 다능 유전자의 유도에 의한 세포 재프로그래밍
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JP4936343B6 (ja) 2008-10-23 2018-06-27 国立大学法人 東京大学 microRNAの機能阻害法
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US20120207744A1 (en) * 2009-03-19 2012-08-16 Mendlein John D Reprogramming compositions and methods of using the same
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KR101853509B1 (ko) 2010-01-06 2018-04-30 큐알엔에이, 인크. 췌장 발달 유전자에 대한 천연 안티센스 전사체의 억제에 의한 췌장 발달 유전자와 관련된 질환의 치료
GB201010557D0 (en) * 2010-06-23 2010-08-11 Mina Therapeutics Ltd RNA molecules and uses thereof
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KR20130132795A (ko) * 2010-10-08 2013-12-05 미나 테라퓨틱스 리미티드 짧은 rna 분자

Also Published As

Publication number Publication date
JP5681955B2 (ja) 2015-03-11
EP2625272A2 (en) 2013-08-14
US20150176008A1 (en) 2015-06-25
CN103314108A (zh) 2013-09-18
WO2012046085A3 (en) 2012-07-19
WO2012046084A2 (en) 2012-04-12
WO2012046085A2 (en) 2012-04-12
WO2012046084A3 (en) 2012-09-27
EP2625273B1 (en) 2015-01-07
US11365414B2 (en) 2022-06-21
KR20130132795A (ko) 2013-12-05
JP2014501492A (ja) 2014-01-23
AU2011311344B2 (en) 2016-09-08
US9885046B2 (en) 2018-02-06
CN103314108B (zh) 2015-08-05
EP2625273A2 (en) 2013-08-14
US8835400B2 (en) 2014-09-16
US8916534B2 (en) 2014-12-23
US20130323211A1 (en) 2013-12-05
US20180112222A1 (en) 2018-04-26
US20220282256A1 (en) 2022-09-08
AU2011311344A1 (en) 2013-05-30
EP2625272B1 (en) 2015-09-16
US20200362352A1 (en) 2020-11-19
US20130289258A1 (en) 2013-10-31
US10774331B2 (en) 2020-09-15

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Effective date: 20171011