SG11202006142PA - Alpha-synuclein antisense oligonucleotides and uses thereof - Google Patents

Alpha-synuclein antisense oligonucleotides and uses thereof

Info

Publication number
SG11202006142PA
SG11202006142PA SG11202006142PA SG11202006142PA SG11202006142PA SG 11202006142P A SG11202006142P A SG 11202006142PA SG 11202006142P A SG11202006142P A SG 11202006142PA SG 11202006142P A SG11202006142P A SG 11202006142PA SG 11202006142P A SG11202006142P A SG 11202006142PA
Authority
SG
Singapore
Prior art keywords
alpha
antisense oligonucleotides
synuclein
synuclein antisense
oligonucleotides
Prior art date
Application number
SG11202006142PA
Inventor
Peter Hagedorn
Richard E Olson
Angela M Cacace
Marianne Lerbech Jensen
Jeffrey M Brown
Meredith, Jr
Annapurna Pendri
Ivar M Mcdonald
Martin Gill
Original Assignee
Roche Innovation Ct 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 Ct Copenhagen As filed Critical Roche Innovation Ct Copenhagen As
Publication of SG11202006142PA publication Critical patent/SG11202006142PA/en

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/3513—Protein; Peptide

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
SG11202006142PA 2018-01-12 2019-01-11 Alpha-synuclein antisense oligonucleotides and uses thereof SG11202006142PA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862616944P 2018-01-12 2018-01-12
PCT/EP2019/050661 WO2019138057A1 (en) 2018-01-12 2019-01-11 Alpha-synuclein antisense oligonucleotides and uses thereof

Publications (1)

Publication Number Publication Date
SG11202006142PA true SG11202006142PA (en) 2020-07-29

Family

ID=67219416

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202006142PA SG11202006142PA (en) 2018-01-12 2019-01-11 Alpha-synuclein antisense oligonucleotides and uses thereof

Country Status (18)

Country Link
US (1) US20220119811A1 (en)
EP (1) EP3737759A1 (en)
JP (2) JP2021511027A (en)
KR (1) KR20200109338A (en)
CN (1) CN112424353A (en)
AU (2) AU2019207859A1 (en)
BR (1) BR112020012921A2 (en)
CA (1) CA3085964A1 (en)
CL (1) CL2020001810A1 (en)
CO (1) CO2020008988A2 (en)
CR (1) CR20200301A (en)
IL (1) IL275950A (en)
MA (1) MA51634A (en)
MX (1) MX2020006973A (en)
PE (1) PE20210172A1 (en)
PH (1) PH12020500570A1 (en)
SG (1) SG11202006142PA (en)
WO (1) WO2019138057A1 (en)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3051839A1 (en) 2017-02-17 2018-08-23 Bristol-Myers Squibb Company Antibodies to alpha-synuclein and uses thereof
WO2019140231A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
JP7317029B2 (en) 2018-02-12 2023-07-28 アイオーニス ファーマシューティカルズ， インコーポレーテッド Modified compounds and uses thereof
CN114829603A (en) * 2019-12-20 2022-07-29 豪夫迈·罗氏有限公司 Enhancer oligonucleotides for inhibiting expression of SCN9A
AU2021206182A1 (en) * 2020-01-06 2022-07-28 AUM LifeTech, Inc. Antisense oligonucleotides for treatment of neurological disorders
IL301712A (en) * 2020-10-01 2023-05-01 Alnylam Pharmaceuticals Inc Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases
MX2023010535A (en) * 2021-03-08 2023-09-19 Servier Lab Antisense oligonucleotides for inhibiting alpha-synuclein expression.

Family Cites Families (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1331011A3 (en) 1991-10-24 2003-12-17 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
JP3756313B2 (en) 1997-03-07 2006-03-15 武 今西 Novel bicyclonucleosides and oligonucleotide analogues
IL135000A0 (en) 1997-09-12 2001-05-20 Exiqon As Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues
TR200604211T1 (en) 1999-02-12 2007-02-21 Daiichi Sankyo Company Limiteddaiichi Sankyo Company Limited New nucleoside and oligonucleotide analoguesNew nucleoside and oligonucleotide analogues
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US7511131B2 (en) * 2002-11-13 2009-03-31 Genzyme Corporation Antisense modulation of apolipoprotein B expression
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
DK3222722T3 (en) 2002-11-18 2019-06-17 Roche Innovation Ct Copenhagen As Antisense design
AU2004255557B2 (en) 2003-06-09 2010-06-10 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
WO2005045034A2 (en) 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA)
CA2580189C (en) 2004-09-29 2013-05-21 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
US20080003570A1 (en) 2004-12-22 2008-01-03 The General Hospital Corporation Translation enhancer elements of genes encoding human Tau protein and human alpha-synuclein protein
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
PL1984381T3 (en) 2006-01-27 2011-03-31 Isis Pharmaceuticals Inc 6-modified bicyclic nucleic acid analogs
EP2019692B1 (en) 2006-05-05 2014-06-04 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of gccr
JP5441688B2 (en) 2006-05-11 2014-03-12 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 5 'modified bicyclic nucleic acid analogs
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
GB0610183D0 (en) 2006-05-23 2006-06-28 Isis Innovation Treatment of neurodegenerative diseases
WO2008109509A1 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting snca gene expression and uses thereof
US8580756B2 (en) 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
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
EP2176280B2 (en) 2007-07-05 2015-06-24 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
WO2009079399A2 (en) 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
DK2285819T3 (en) 2008-04-04 2013-12-02 Isis Pharmaceuticals Inc OLIGOMER COMPOUNDS INCLUDING NEUTRAL BONDED, TERMINAL BICYCLIC NUCLEOSIDES
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
CN102781965A (en) 2009-10-06 2012-11-14 安吉奥开米公司 Compositions and methods for the transport of therapeutic agents
WO2011048125A1 (en) 2009-10-20 2011-04-28 Santaris Pharma A/S Oral delivery of therapeutically effective lna oligonucleotides
EP3231446A1 (en) 2010-04-19 2017-10-18 Nlife Therapeutics S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
US8846637B2 (en) 2010-06-08 2014-09-30 Isis Pharmaceuticals, Inc. Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2012027713A2 (en) 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
AU2011329777B2 (en) 2010-11-17 2016-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CA2828662A1 (en) 2011-04-20 2012-10-26 Roche Glycart Ag Method and constructs for the ph dependent passage of the blood-brain-barrier
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CN104136451A (en) 2011-09-07 2014-11-05 玛瑞纳生物技术有限公司 Synthesis and uses of nucleic acid compounds with conformationally restricted monomers
WO2013154798A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
JP6386461B2 (en) 2012-10-26 2018-09-05 エヌライフ、セラピューティックス、ソシエダッド、リミターダＮｌｉｆｅ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｓ．Ｌ． Compositions and methods for the treatment of Parkinson's disease by selective delivery of oligonucleotide molecules to specific neuronal species
AU2013346767B2 (en) 2012-11-15 2019-04-11 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
AU2014259755B2 (en) 2013-05-01 2018-08-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating apolipoprotein (a) expression
CN105358692B (en) 2013-06-27 2020-08-21 罗氏创新中心哥本哈根有限公司 Antisense oligomers and conjugates targeting PCSK9
WO2016061263A1 (en) * 2014-10-14 2016-04-21 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof

Also Published As

Publication number Publication date
EP3737759A1 (en) 2020-11-18
PH12020500570A1 (en) 2021-05-10
AU2022224819A1 (en) 2022-09-29
US20220119811A1 (en) 2022-04-21
BR112020012921A2 (en) 2020-12-08
CN112424353A (en) 2021-02-26
MA51634A (en) 2020-11-18
JP2022130597A (en) 2022-09-06
CL2020001810A1 (en) 2020-11-27
PE20210172A1 (en) 2021-01-29
JP2021511027A (en) 2021-05-06
CR20200301A (en) 2020-10-26
MX2020006973A (en) 2020-09-09
KR20200109338A (en) 2020-09-22
CO2020008988A2 (en) 2020-08-31
RU2020126575A3 (en) 2022-02-14
IL275950A (en) 2020-08-31
RU2020126575A (en) 2022-02-14
CA3085964A1 (en) 2019-07-18
AU2019207859A1 (en) 2020-07-02
WO2019138057A1 (en) 2019-07-18

Similar Documents

Publication Publication Date Title
IL276549A (en) Camk2d antisense oligonucleotides and uses thereof
IL275950A (en) Alpha-synuclein antisense oligonucleotides and uses thereof
IL268422A (en) Targeted oligonucleotides
IL272524A (en) Clec9a binding agents and use thereof
SG11202105626TA (en) Oligonucleotide compositions and methods thereof
GB201711809D0 (en) Antisense oligonucleotide
IL275903A (en) Antisense oligonucleotides targeting alpha-synuclein and uses thereof
EP3353306A4 (en) Conjugated antisense compounds and their use
IL269714A (en) Oligonucleotide probes and uses thereof
EP3353305A4 (en) Conjugated antisense compounds and their use
IL275902A (en) Antisense oligonucleotides targeting alpha-synuclein and uses thereof
GB201904709D0 (en) Chemically modified oligonucleotides
IL280581A (en) Variant rnai against alpha-synuclein
IL275217A (en) Immunostimulatory oligonucleotides
EP3724206A4 (en) Conjugated antisense compounds and their use
SG10201913477UA (en) G-quadruplex-containing antisense oligonucleotides
SG10201609048RA (en) Antisense oligonucleotides
EP4041248A4 (en) Modified oligonucleotides
GB2569611B (en) Improvements in hair-curling devices
SG10202008771WA (en) Combination therapy with liposomal antisense oligonucleotides
IL286826A (en) Angptl2 antisense oligonucleotides and uses thereof
GB201714409D0 (en) Oligonucleotides and analogues thereof
EP4069844A4 (en) Anti-slc6a1 oligonucleotides and related methods
GB2603720B (en) Hair clip
PL3341481T3 (en) Microrna-328 anti-sense composition and therapeutic use