[go: up one dir, main page]

WO2020011744A3 - Antisense oligonucleotides targeting cers5 - Google Patents

Antisense oligonucleotides targeting cers5 Download PDF

Info

Publication number
WO2020011744A3
WO2020011744A3 PCT/EP2019/068322 EP2019068322W WO2020011744A3 WO 2020011744 A3 WO2020011744 A3 WO 2020011744A3 EP 2019068322 W EP2019068322 W EP 2019068322W WO 2020011744 A3 WO2020011744 A3 WO 2020011744A3
Authority
WO
WIPO (PCT)
Prior art keywords
cers5
antisense oligonucleotides
oligonucleotides targeting
diabetes
expression
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.)
Ceased
Application number
PCT/EP2019/068322
Other languages
French (fr)
Other versions
WO2020011744A2 (en
Inventor
Marie Wirkström LINDHOLM
Steffen Schmidt
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.)
Roche Innovation Center Copenhagen AS
Original Assignee
Roche Innovation Center Copenhagen AS
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 Roche Innovation Center Copenhagen AS filed Critical Roche Innovation Center Copenhagen AS
Publication of WO2020011744A2 publication Critical patent/WO2020011744A2/en
Publication of WO2020011744A3 publication Critical patent/WO2020011744A3/en
Anticipated expiration legal-status Critical
Ceased 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to CERS5 pre-mRNA exon and junction sequences, which are capable of inhibiting the expression of CERS5 protein. Inhibition of CERS5 expression is beneficial for a range of medical disorders including obesity, insulin resistance, diabetes such as type 2 diabetes, atherosclerosis, cardiomyopathy, opioid drug addiction and colorectal cancer.
PCT/EP2019/068322 2018-07-11 2019-07-09 Antisense oligonucleotides targeting cers5 Ceased WO2020011744A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18182921.9 2018-07-11
EP18182921 2018-07-11

Publications (2)

Publication Number Publication Date
WO2020011744A2 WO2020011744A2 (en) 2020-01-16
WO2020011744A3 true WO2020011744A3 (en) 2020-02-27

Family

ID=62916545

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2019/068322 Ceased WO2020011744A2 (en) 2018-07-11 2019-07-09 Antisense oligonucleotides targeting cers5

Country Status (1)

Country Link
WO (1) WO2020011744A2 (en)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005116204A1 (en) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
WO2008006007A2 (en) * 2006-07-03 2008-01-10 Columbia University Defective cystic fibrosis transduction regulator (cftr) causes increased sphingolipid synthesis
EP2213738A2 (en) * 2002-11-14 2010-08-04 Dharmacon, Inc. siRNA molecules targeting Bcl-2
WO2014205449A2 (en) * 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0862439A4 (en) 1995-11-22 2001-01-10 O Paul O P Ts Ligands to enhance cellular uptake of biomolecules
JP3756313B2 (en) 1997-03-07 2006-03-15 武 今西 Novel bicyclonucleosides and oligonucleotide analogues
KR100414936B1 (en) 1997-09-12 2004-01-13 엑시콘 에이/에스 Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues
NZ513402A (en) 1999-02-12 2003-06-30 Sankyo Co Novel nucleosides and oligonucleotide analogues
PT1178999E (en) 1999-05-04 2007-06-26 Santaris Pharma As L-ribo-lna analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
DK2752488T3 (en) 2002-11-18 2020-04-20 Roche Innovation Ct Copenhagen As Antisense design
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
ES2516815T3 (en) 2006-01-27 2014-10-31 Isis Pharmaceuticals, Inc. Analogs of bicyclic nucleic acids modified at position 6
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
JP5665317B2 (en) 2006-10-18 2015-02-04 アイシス ファーマシューティカルズ， インコーポレーテッド Antisense compound
US20100086543A1 (en) 2007-04-02 2010-04-08 Saint Louis University Compositions and methods for treating conditions associated with ceramide biosynthesis
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
AU2009234266B2 (en) 2008-04-11 2015-08-06 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
US9012421B2 (en) 2009-08-06 2015-04-21 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US8846637B2 (en) 2010-06-08 2014-09-30 Isis Pharmaceuticals, Inc. Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
KR20130132475A (en) 2010-12-17 2013-12-04 애로우헤드 리서치 코오포레이션 Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
RU2582235C2 (en) 2010-12-29 2016-04-20 Ф.Хоффманн-Ля Рош Аг Low-molecular conjugates for intracellular delivery of nucleic acids
EP2673361B1 (en) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
WO2013022967A1 (en) 2011-08-11 2013-02-14 Isis Pharmaceuticals, Inc. Gapped oligomeric compounds comprising 5'-modified deoxyribonucleosides in the gap and uses thereof
US9221864B2 (en) 2012-04-09 2015-12-29 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
SG11201503821YA (en) 2012-11-15 2015-06-29 Roche Innovation Ct Copenhagen As Oligonucleotide conjugates
RU2649367C2 (en) 2013-01-30 2018-04-02 Ф. Хоффманн-Ля Рош Аг Lna oligonucleotide carbohydrate conjugates
KR102482890B1 (en) 2013-05-01 2022-12-30 아이오니스 파마수티컬즈, 인코포레이티드 COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN (a) EXPRESSION
CA2909868C (en) 2013-05-01 2021-10-19 Regulus Therapeutics Inc. Compounds and methods for enhanced cellular uptake
CA2935426C (en) 2014-01-30 2023-07-25 F. Hoffmann-La Roche Ag Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2213738A2 (en) * 2002-11-14 2010-08-04 Dharmacon, Inc. siRNA molecules targeting Bcl-2
WO2005116204A1 (en) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
WO2008006007A2 (en) * 2006-07-03 2008-01-10 Columbia University Defective cystic fibrosis transduction regulator (cftr) causes increased sphingolipid synthesis
WO2014205449A2 (en) * 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
MESICEK J ET AL: "Ceramide synthases 2, 5, and 6 confer distinct roles in radiation-induced apoptosis in HeLa cells", CELLULAR SIGNALLING, ELSEVIER SCIENCE LTD, GB, vol. 22, no. 9, 1 September 2010 (2010-09-01), pages 1300 - 1307, XP027087695, ISSN: 0898-6568, [retrieved on 20100614] *
WEGNER MARTHE-SUSANNA ET AL: "The enigma of ceramide synthase regulation in mammalian cells", PROGRESS IN LIPID RESEARCH, PERGAMON PRESS, PARIS, FR, vol. 63, 13 May 2016 (2016-05-13), pages 93 - 119, XP029672393, ISSN: 0163-7827, DOI: 10.1016/J.PLIPRES.2016.03.006 *

Also Published As

Publication number Publication date
WO2020011744A2 (en) 2020-01-16

Similar Documents

Publication Publication Date Title
CY1121037T1 (en) ANTO-SENSORY POLYNUCLEOTIDS TO STILE EXTRACTION AND METHODS OF DYSTROPHY TREATMENT
MX2020006999A (en) Improved peptide pharmaceuticals for treatment of nash and other disorders.
MX2018005340A (en) Proteolysis targeting chimera compounds and methods of preparing and using same.
EP4527459A3 (en) Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
CY1118157T1 (en) GLUCOSE RESIDUES EXTENSIVE INSPECTORS AND THEIR USES
WO2010135695A3 (en) TREATMENT OF TRANSCRIPTION FACTOR E3 (TFE3) and INSULIN RECEPTOR SUBSTRATE 2 (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO TFE3
NZ631245A (en) Exon skipping compositions for treating muscular dystrophy
NZ598724A (en) Soybean transgenic event mon 87708 and methods of use thereof
PH12015501953A1 (en) Substituted nucleotide analogs
MX2017008184A (en) Substituted nucleosides, nucleotides and analogs thereof.
TN2010000170A1 (en) Use of a beta blocker for the manufacture of a medicament for the treatment of hemangiomas
SG10201903290YA (en) Modified double-stranded rna agents
MX2018002665A (en) Combination comprising immunostimulatory oligonucleotides.
NZ631512A (en) Compositions and methods for modulating apolipoprotein (a) expression
WO2011085066A3 (en) Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene
JOP20180094A1 (en) Heterocyclic compound as a protein kinase inhibito
MX2018015082A (en) Phenyl propanamide derivative, and manufacturing method and pharmaceutical application thereof.
UA99633C2 (en) Inhibition of macrophage-stimulating protein receptor (ron) and methods of treatment thereof
ZA202204666B (en) 2-isoindol-1,3,4-oxadiazole derivatives useful as hdac6 inhibitors
WO2011017516A3 (en) Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
WO2019217397A3 (en) Compositions and methods for improving strand biased
MX2011011348A (en) Treatment of osteoarthritis pain.
MX388737B (en) THERAPEUTIC OLIGONUCLEOTIDES.
EA201791986A1 (en) Oligonucleotides, corresponding to exon 73 of the COL7A1 gene, for the therapy of bulletic epidermolysis
BR112014018657A8 (en) 1H-INDAZOL-3-CARBOXAMIDE COMPOUNDS

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 19735586

Country of ref document: EP

Kind code of ref document: A2

NENP Non-entry into the national phase

Ref country code: DE

122 Ep: pct application non-entry in european phase

Ref document number: 19735586

Country of ref document: EP

Kind code of ref document: A2