CA3005131A1 - Antisense oligomers for treatment of tuberous sclerosis complex - Google Patents

Antisense oligomers for treatment of tuberous sclerosis complex Download PDF

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Publication number
CA3005131A1
CA3005131A1 CA3005131A CA3005131A CA3005131A1 CA 3005131 A1 CA3005131 A1 CA 3005131A1 CA 3005131 A CA3005131 A CA 3005131A CA 3005131 A CA3005131 A CA 3005131A CA 3005131 A1 CA3005131 A1 CA 3005131A1
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CA
Canada
Prior art keywords
mrna
nucleobases
intron
ric pre
fold
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
CA3005131A
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English (en)
French (fr)
Inventor
Isabel AZNAREZ
Huw M. Nash
Adrian Krainer
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.)
Cold Spring Harbor Laboratory
Stoke Therapeutics Inc
Original Assignee
Cold Spring Harbor Laboratory
Stoke Therapeutics 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 Cold Spring Harbor Laboratory, Stoke Therapeutics Inc filed Critical Cold Spring Harbor Laboratory
Publication of CA3005131A1 publication Critical patent/CA3005131A1/en
Pending legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • 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
    • 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
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic or mutational markers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/178Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Analytical Chemistry (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Pathology (AREA)
  • Oncology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA3005131A 2015-12-14 2016-12-14 Antisense oligomers for treatment of tuberous sclerosis complex Pending CA3005131A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267212P 2015-12-14 2015-12-14
US62/267,212 2015-12-14
PCT/US2016/066705 WO2017106375A1 (en) 2015-12-14 2016-12-14 Antisense oligomers for treatment of tuberous sclerosis complex

Publications (1)

Publication Number Publication Date
CA3005131A1 true CA3005131A1 (en) 2017-06-22

Family

ID=59057534

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3005131A Pending CA3005131A1 (en) 2015-12-14 2016-12-14 Antisense oligomers for treatment of tuberous sclerosis complex

Country Status (4)

Country Link
EP (1) EP3390635A4 (enrdf_load_stackoverflow)
JP (2) JP7051683B2 (enrdf_load_stackoverflow)
CA (1) CA3005131A1 (enrdf_load_stackoverflow)
WO (1) WO2017106375A1 (enrdf_load_stackoverflow)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CA2963288A1 (en) 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2017060731A1 (en) 2015-10-09 2017-04-13 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
CA3005256A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CN110446785B (zh) 2017-01-23 2024-04-26 瑞泽恩制药公司 Hsd17b13变体及其应用
MX2019012169A (es) 2017-04-11 2019-12-11 Regeneron Pharma Ensayos para evaluar la actividad de moduladores de miembros de la familia de hidroxiesteroide (17-beta) deshidrogenasa (hsd17b).
ES2965696T3 (es) 2017-08-25 2024-04-16 Stoke Therapeutics Inc Oligómeros antisentido para el tratamiento de afecciones y enfermedades
SG11202003254SA (en) 2017-10-11 2020-05-28 Regeneron Pharma Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation
CN112020556A (zh) 2018-03-21 2020-12-01 瑞泽恩制药公司 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス,インク. コレステリルエステル蓄積症の処置のための方法及び組成物
WO2021034985A1 (en) * 2019-08-19 2021-02-25 Stoke Therapeutics, Inc. Compositions and methods for modulating splicing and protein expression
IL298063A (en) 2020-05-11 2023-01-01 Stoke Therapeutics Inc Opa1 antisense oligomers for treatment of conditions and diseases
EP4560019A1 (en) * 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions
EP4560020A1 (en) * 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2666185A1 (en) * 2006-10-11 2008-04-17 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Influenza targets
US20120252877A1 (en) * 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
SG179038A1 (en) * 2009-09-08 2012-04-27 Lab Corp America Holdings Compositions and methods for diagnosing autism spectrum disorders
WO2015035091A1 (en) * 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CA2963288A1 (en) * 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2017106382A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases

Also Published As

Publication number Publication date
JP2022088533A (ja) 2022-06-14
JP2019500350A (ja) 2019-01-10
WO2017106375A1 (en) 2017-06-22
EP3390635A4 (en) 2019-05-01
JP7051683B2 (ja) 2022-04-11
EP3390635A1 (en) 2018-10-24

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