Title:
ALPHA-SYNUCLEIN ANTISENSE OLIGONUCLEOTIDES AND USES THEREOF
Document Type and Number:
WIPO Patent Application WO/2019/138057
Kind Code:
A1
Abstract:
The present disclosure relates to antisense oligonucleotides, which target Alpha-synuclein (SNCA) transcript in a cell, leading to reduced expression of SNCA protein. Reduction of SNCA protein expression is beneficial for the treatment of certain medical disorders, e.g., a neurological disorder such as a synucleinopathy.
Inventors:
HAGEDORN PETER (DK)
OLSON RICHARD E (US)
CACACE ANGELA M (US)
JENSEN MARIANNE LERBECH (DK)
BROWN JEFFREY M (US)
MEREDITH JERE E (US)
PENDRI ANNAPURNA (US)
MCDONALD IVAR M (US)
GILL MARTIN (US)
OLSON RICHARD E (US)
CACACE ANGELA M (US)
JENSEN MARIANNE LERBECH (DK)
BROWN JEFFREY M (US)
MEREDITH JERE E (US)
PENDRI ANNAPURNA (US)
MCDONALD IVAR M (US)
GILL MARTIN (US)
Application Number:
PCT/EP2019/050661
Publication Date:
July 18, 2019
Filing Date:
January 11, 2019
Export Citation:
Assignee:
ROCHE INNOVATION CT COPENHAGEN AS (DK)
International Classes:
C12N15/113; A61K48/00; C07K14/47
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"Manipulating the Mouse Embryo", 1986, COLD SPRING HARBOR LABORATORY PRESS
CROOKE: "Antisense drug Technology: Principles, Strategies and Applications", 2007, CRC PRESS
AUSUBEL ET AL.: "Current Protocols in Molecular Biology", 1989, JOHN WILEY AND SONS
BARCISZEWSKI: "Nucleic Acid and Peptide Aptamers: Methods and Protocols", vol. 535, 2009, article "Locked Nucleic Acid Aptamers"
KUO Y ET AL., HUM MOL GENET, vol. 19, 2010, pages 1633 - 50
"National Research Council: Guide for the Care and Use of Laboratory Animals (The National Academies Collection: Reports funded by National Institutes of Health", NATIONAL ACADEMIES PRESS
DATABASE Geneseq [online] 27 November 2008 (2008-11-27), "Human TNF targeted mdRNA sense strand, SEQ ID 255.", XP002792105, retrieved from EBI accession no. GSN:ATL95109 Database accession no. ATL95109
MARQUES O ET AL., CELL DEATH DIS., vol. 19, 2012, pages e350
GALVIN JE ET AL., ARCHIVES OF NEUROLOGY, vol. 58, 2001, pages 186 - 190
VALERA E ET AL., J NEUROCHEM, vol. 139, no. 1, October 2016 (2016-10-01), pages 346 - 352
RECCHIA A, FASEB J., vol. 18, 2004, pages 617 - 26
"Concise Dictionary of Biomedicine and Molecular Biology", 2002, CRC PRESS
"The Dictionary of Cell and Molecular Biology", 1999, ACADEMIC PRESS
"Oxford Dictionary Of Biochemistry And Molecular Biology, Revised", 2000, OXFORD UNIVERSITY PRESS
KARLIN ET AL., PROC. NATL. ACAD. SCI., vol. 87, 1990, pages 2264 - 2268
KARLIN ET AL., PROC. NATL. ACAD. SCI., vol. 90, 1993, pages 5873 - 5877
ALTSCHUL ET AL., NUCLEIC ACIDS RES., vol. 25, 1991, pages 3389 - 3402
ALTSCHUL, NUCLEIC ACIDS RES., vol. 25, 1997, pages 3389 - 3402
ALTSCHUL ET AL., METHODS IN ENZYMOLOGY, vol. 266, 1996, pages 460 - 480
NEEDLEMAN; WUNSCH, J. MOL. BIOL., vol. 48, 1970, pages 444 - 453
MYERS; MILLER, CABIOS, vol. 4, 1989, pages 11 - 17
STANLEY T CROOKS: "Antisense Drug Technology: Principles, Strategies and Applications", 2007, CRC PRESS
SHASHANK ET AL., BRAIN RESEARCH, vol. 1006, no. 1, 2004, pages 8 - 17
ROSE ET AL., NATURE NEUROSCI, vol. 4, 2001, pages 773 - 774
ZONTA ET AL., J PHYSIOL PARIS., vol. 96, no. 3-4, 2002, pages 193 - 8
PASTI ET AL., J. NEUROSCI., vol. 21, no. 2, 2001, pages 477 - 484
FREIER; ALTMANN, NUCL. ACID RES., vol. 25, 1997, pages 4429 - 4443
UHLMANN, CURR. OPINION IN DRUG DEVELOPMENT, vol. 3, no. 2, 2000, pages 293 - 213
HIRAO ET AL., ACCOUNTS OF CHEMICAL RESEARCH, vol. 45, 2012, pages 2055
BERGSTROM, CURRENT PROTOCOLS IN NUCLEIC ACID CHEMISTRY, 2009, pages 1.4.1
DELEAVEY; DAMHA, CHEMISTRY AND BIOLOGY, vol. 19, 2012, pages 937
MORITA ET AL., BIOORGANIC & MED.CHEM. LETT., vol. 12, pages 73 - 76
SETH, J. ORG. CHEM., vol. 75, no. 5, 2010, pages 1569 - 81
SETH, J. ORG. CHEM, 2010
MITSUOKA ET AL., NUCLEIC ACIDS RESEARCH, vol. 37, no. 4, 2009, pages 1225 - 1238
"Protective Groups in Organic Chemistry", 1999, WILEY
"Protective Groups in Organic Synthesis", 1999, JOHN WILEY & SONS
DASS CR., J PHARM PHARMACOL, vol. 54, no. l, 2002, pages 3 - 27
TOUBOUL, ANTICANCER RES., vol. 22, no. 6A, 2002, pages 3349 - 56
VERJOUT, MUTAT. RES., vol. 640, 2000, pages 127 - 38
STOWE, J. VIROL. METHODS, vol. 75, no. 1, 1998, pages 93 - 91
SAMBROOK ET AL.: "Molecular Cloning A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
"Molecular Cloning: A Laboratory Manual", 1992, COLD SPRINGS HARBOR LABORATORY
"DNA Cloning, Volumes I and II", 1985
"Oligonucleotide Synthesis", 1984
"Nucleic Acid Hybridization", 1984
"Transcription And Translation", 1984
FRESHNEY: "Culture Of Animal Cells", 1987, ALAN R. LISS, INC.
"Immobilized Cells And Enzymes", 1986, IRL PRESS
PERBAL: "Methods In Enzymology", 1984, ACADEMIC PRESS, INC., article "A Practical Guide To Molecular Cloning; the treatise"
"Gene Transfer Vectors For Mammalian Cells", 1987, COLD SPRING HARBOR LABORATORY
"Methods In Enzymology", vol. 154;155
"Immunochemical Methods In Cell And Molecular Biology", 1987, ACADEMIC PRESS
"Handbook Of Experimental Immunology", vol. I-IV, 1986
"Manipulating the Mouse Embryo", 1986, COLD SPRING HARBOR LABORATORY PRESS
CROOKE: "Antisense drug Technology: Principles, Strategies and Applications", 2007, CRC PRESS
AUSUBEL ET AL.: "Current Protocols in Molecular Biology", 1989, JOHN WILEY AND SONS
BARCISZEWSKI: "Nucleic Acid and Peptide Aptamers: Methods and Protocols", vol. 535, 2009, article "Locked Nucleic Acid Aptamers"
KUO Y ET AL., HUM MOL GENET, vol. 19, 2010, pages 1633 - 50
"National Research Council: Guide for the Care and Use of Laboratory Animals (The National Academies Collection: Reports funded by National Institutes of Health", NATIONAL ACADEMIES PRESS
Attorney, Agent or Firm:
RUDOLPH HANSEN, Marianne (CH)
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