MX2020007433A - Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos. - Google Patents

Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos.

Info

Publication number
MX2020007433A
MX2020007433A MX2020007433A MX2020007433A MX2020007433A MX 2020007433 A MX2020007433 A MX 2020007433A MX 2020007433 A MX2020007433 A MX 2020007433A MX 2020007433 A MX2020007433 A MX 2020007433A MX 2020007433 A MX2020007433 A MX 2020007433A
Authority
MX
Mexico
Prior art keywords
act
antisense oligonucleotides
synuclein
alpha
protein
Prior art date
Application number
MX2020007433A
Other languages
English (en)
Inventor
Annapurna Pendri
Ivar M Mcdonald
Richard E Olson
Angela M Cacace
Peter Hagedorn
Marianne Lerbech Jensen
Nino Devidze
Jr Jere E Meredith
James K Loy
Carl J Baldick
Original Assignee
Bristol Myers Squibb Co
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 Bristol Myers Squibb Co filed Critical Bristol Myers Squibb Co
Publication of MX2020007433A publication Critical patent/MX2020007433A/es

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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
    • 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
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

La presente descripción se refiere a oligonucleótidos antisentido, que actúan sobre mARN de SNCA (por ejemplo, en una unión intrón-exón) en una célula, que tiene como resultado una expresión reducida de la proteína SNCA. La reducción de la expresión de la proteína SCNA es beneficiosa para el tratamiento de determinados trastornos médicos, por ejemplo, un trastorno neurológico.
MX2020007433A 2018-01-12 2019-01-11 Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos. MX2020007433A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862616937P 2018-01-12 2018-01-12
PCT/US2019/013249 WO2019140231A1 (en) 2018-01-12 2019-01-11 Antisense oligonucleotides targeting alpha-synuclein and uses thereof

Publications (1)

Publication Number Publication Date
MX2020007433A true MX2020007433A (es) 2020-09-14

Family

ID=65269095

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020007433A MX2020007433A (es) 2018-01-12 2019-01-11 Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos.

Country Status (17)

Country Link
US (5) US11447775B2 (es)
EP (1) EP3737387A1 (es)
JP (2) JP7455746B2 (es)
KR (1) KR102840298B1 (es)
CN (2) CN111836624A (es)
AU (1) AU2019208006B2 (es)
BR (1) BR112020013994A2 (es)
CA (1) CA3088112A1 (es)
CO (1) CO2020008425A2 (es)
EA (1) EA202091695A1 (es)
IL (1) IL275902B2 (es)
MX (1) MX2020007433A (es)
MY (1) MY203356A (es)
PE (1) PE20201501A1 (es)
SA (1) SA520412423B1 (es)
SG (1) SG11202006526YA (es)
WO (1) WO2019140231A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
MX2020007433A (es) 2018-01-12 2020-09-14 Bristol Myers Squibb Co Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos.
EA202091693A1 (ru) * 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof

Family Cites Families (180)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5102785A (en) 1987-09-28 1992-04-07 E. I. Du Pont De Nemours And Company Method of gene mapping
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
EP0544824B1 (en) 1990-07-27 1997-06-11 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
ATE239484T1 (de) 1991-10-24 2003-05-15 Isis Pharmaceuticals Inc Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
AU3222793A (en) 1991-11-26 1993-06-28 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
AU685074B2 (en) 1992-12-14 1998-01-15 Start Technology Partnership Administration of oligonucleotides antisense to dopamine receptor MRNA for diagnosis and treatment of neurological pathologies
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5981279A (en) 1995-11-09 1999-11-09 Allegheny University Of The Health Sciences Compositions and methods to regulate calmodulin gene expression, and uses thereof for influencing cell growth and differentiation
WO1997033550A2 (en) 1996-03-15 1997-09-18 Start Technology Partnership Methods and compositions for diagnosis and treatment of pathological conditions related to abnormal dopamine receptor expression
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US5932557A (en) 1997-08-12 1999-08-03 Mustafa; S. Jamal Adenosine A1 receptor antisense oligonucleotide treatment of alcohol and marijuana-induced psychomotor impairments
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
US6913745B1 (en) 1997-12-02 2005-07-05 Neuralab Limited Passive immunization of Alzheimer's disease
WO1999060855A1 (en) 1998-05-26 1999-12-02 Icn Pharmaceuticals, Inc. Novel nucleosides having bicyclic sugar moiety
US6833361B2 (en) 1998-05-26 2004-12-21 Ribapharm, Inc. Nucleosides having bicyclic sugar moiety
PT1080202E (pt) 1998-05-27 2006-05-31 Avigen Inc Distribuicao de vectores aav codificando aadc intensificada por conveccao
CA2333857A1 (en) 1998-07-13 2000-01-20 Merck & Co., Inc. Growth hormone secretagogue related receptors and nucleic acids
JP2002528509A (ja) 1998-11-05 2002-09-03 トーマス・ジェファーソン・ユニバーシティ オリゴヌクレオチドを用いるパーキンソン病の治療
JP2002536963A (ja) 1998-12-22 2002-11-05 ミリアド・ジェネティックス・インコーポレイテッド 神経変性疾患におけるタンパク質−タンパク質相互作用
US20040226056A1 (en) 1998-12-22 2004-11-11 Myriad Genetics, Incorporated Compositions and methods for treating neurological disorders and diseases
KR100573231B1 (ko) 1999-02-12 2006-04-24 상꾜 가부시키가이샤 신규 뉴클레오시드 및 올리고뉴클레오티드 유사체
AU5081200A (en) 1999-05-04 2000-11-17 Aventis Pharma S.A. Use of inducible no-synthase antisense oligonucleotides for preventing and treating cerebral ischemia
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
AU5959900A (en) 1999-07-15 2001-02-05 Mcgill University Oligonucleotides for metabotropic glutamate receptor type 1 (mglur1)
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
AU3087801A (en) 2000-02-04 2001-08-14 Molecular Dynamics Inc Human genome-derived single exon nucleic acid probes useful for analysis of geneexpression in human breast and hbl 100 cells
AU2001250572A1 (en) 2000-04-07 2001-10-23 Epigenomics Ag Detection of single nucleotide polymorphisms (snp's) and cytosine-methylations
US20030032791A1 (en) 2000-06-26 2003-02-13 Alan Robertson Scott Novel melanocortin-4 receptor sequences and screening assays to identify compounds useful in regulating animal appetite and metabolic rate
US6277640B1 (en) 2000-07-31 2001-08-21 Isis Pharmaceuticals, Inc. Antisense modulation of Her-3 expression
US20030060438A1 (en) 2000-08-18 2003-03-27 Henry James L. Oligonucleotides and other modulators of the NK-1 receptor pathway and therapeutic uses thereof
US20020119927A1 (en) 2000-10-17 2002-08-29 Myriad Genetics, Inc. Protein-protein interactions in neurodegenerative diseases
US6653102B2 (en) 2000-10-17 2003-11-25 Myriad Genetics, Inc. Nucleic acid encoding a phosphatase 2C that interacts with Fe 65
US20020151506A1 (en) 2000-12-29 2002-10-17 Castillo Gerardo M. Catechins for the treatment of fibrillogenesis in Alzheimer's disease, Parkinson's disease, systemic AA amyloidosis, and other amyloid disorders
CA2452458A1 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US6455308B1 (en) 2001-08-01 2002-09-24 Isis Pharmaceuticals, Inc. Antisense modulation of serum amyloid A4 expression
US20050014689A1 (en) 2001-10-02 2005-01-20 Eiji Sugaru Remedies for life style-related diseases or cibophobia and method of screening the same
US20030143738A1 (en) 2001-11-08 2003-07-31 Hiroki Yokota Compositions and methods for treating ischemic stroke
EP1468694A1 (en) 2001-12-27 2004-10-20 Sumitomo Pharmaceuticals Company, Limited Remedies for anorexia or lifestyle-related diseases and method of screening the same
US20070276034A1 (en) 2002-05-31 2007-11-29 Luke Esposito Compounds, compositions and methods for the treatment of synucleinopathies
SI1511710T1 (sl) 2002-05-31 2014-04-30 Proteotech, Inc. Spojine, sestavki in postopki za zdravljenje amiloidnih bolezni in sinukleinopatij, kot je Alzheimerjeva bolezen, diabetes tipa 2 in Parkinsova bolezen
TW200509968A (en) 2002-11-01 2005-03-16 Elan Pharm Inc Prevention and treatment of synucleinopathic disease
US8506959B2 (en) 2002-11-01 2013-08-13 Neotope Biosciences Limited Prevention and treatment of synucleinopathic and amyloidogenic disease
EP1562971B1 (en) 2002-11-05 2014-02-12 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
AU2003295387A1 (en) 2002-11-05 2004-06-03 Isis Parmaceuticals, Inc. Modified oligonucleotides for use in rna interference
EP2752488B1 (en) 2002-11-18 2020-02-12 Roche Innovation Center Copenhagen A/S Antisense design
CA2515368A1 (en) 2003-02-13 2004-08-26 Luciano Rossetti Regulation of food intake and glucose production by modulation of long-chain fatty acyl-coa levels in the hypothalamus
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
AU2004231746A1 (en) 2003-04-16 2004-11-04 Whitehead Institute For Biomedical Research Yeast ectopically expressing abnormally processed proteins and uses therefor
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
EP1481680A1 (en) 2003-05-30 2004-12-01 Aventis Pharma Deutschland GmbH Use of S1P
EP1481685A1 (en) 2003-05-30 2004-12-01 Aventis Pharma Deutschland GmbH Use of PAM
US20050032695A1 (en) 2003-05-30 2005-02-10 Aventis Pharma Deutschland Gmbh Method for alleviating pain using protein associated with MYC and related compounds, and assays for identifying such compounds
US7595306B2 (en) 2003-06-09 2009-09-29 Alnylam Pharmaceuticals Inc Method of treating neurodegenerative disease
CA2542232A1 (en) 2003-06-09 2005-01-20 Alnylam Pharmaceuticals, Inc. Method for treating neurodegenerative disease by inhibiting alpha-synuclein
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
AU2004274021B2 (en) 2003-09-18 2009-08-13 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
RU2385933C2 (ru) 2004-02-09 2010-04-10 Реженьон Гмбх Ингибиторы передачи сигнала трансформирующих факторов роста (tgf-r) для лечения расстройств цнс
JP2007531794A (ja) 2004-04-05 2007-11-08 アルニラム ファーマスーティカルズ インコーポレイテッド オリゴヌクレオチドの合成および精製に使用する方法および反応試薬
EP2397563A3 (en) 2004-09-17 2012-07-18 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
CA2580189C (en) 2004-09-29 2013-05-21 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
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
US7776538B2 (en) 2005-03-03 2010-08-17 National University Corporation Nagoya University Method for diagnosing methamphetamine dependence
JP4942044B2 (ja) 2005-03-03 2012-05-30 国立大学法人富山大学 精神障害関連遺伝子及びその利用
US20080206253A1 (en) 2005-03-14 2008-08-28 Xiao-Ying Hua Method For Preventing or Treating Pain in a Mammal
US8361977B2 (en) 2005-06-23 2013-01-29 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of SMN2 splicing
US20080300204A1 (en) 2005-07-19 2008-12-04 University Of Rochester Alpha-Synuclein Antibodies and Methods Related Thereto
US20100130425A1 (en) 2005-09-09 2010-05-27 Oregon Health & Science University Use of toll-like receptor ligands in treating excitotoxic injury, ischemia and/or hypoxia
WO2007030580A2 (en) 2005-09-09 2007-03-15 Oregon Health & Science University Neuroprotectants
EP1940419A2 (en) 2005-09-09 2008-07-09 Oregon Health and Science University Neuroprotectants
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
AU2006291836B2 (en) 2005-09-15 2012-02-23 Santaris Pharma A/S RNA antagonist compounds for the inhibition of Apo-Bl00 expression
US20070213366A1 (en) 2005-12-23 2007-09-13 Justman Craig J Treatment of Synucleinopathies
JP5425474B2 (ja) 2006-01-26 2014-02-26 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 ハンチンチン対する、組成物及びその使用
CA2640171C (en) 2006-01-27 2014-10-28 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
CA3044969A1 (en) 2006-05-05 2007-12-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gene expression
JP5441688B2 (ja) 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’修飾二環式核酸類似体
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
CN101495500A (zh) 2006-07-03 2009-07-29 宰派洛德有限公司 α-突触核蛋白聚集的抑制
KR20080036902A (ko) 2006-10-24 2008-04-29 재단법인서울대학교산학협력재단 아밀로이드 형성 펩타이드 또는 단백질의 가용성 회합체에선택적으로 작용하는 절단제
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
JP5558834B2 (ja) 2007-02-23 2014-07-23 ヤンセン アルツハイマー イミュノセラピー シヌクレイノパチーおよびアミロイド形成疾患（ａｍｙｌｏｉｄｏｇｅｎｉｃｄｉｓｅａｓｅ）の予防および処置
US8580756B2 (en) 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
CN101796062B (zh) 2007-07-05 2014-07-30 Isis制药公司 6-双取代双环核酸类似物
GB0715809D0 (en) * 2007-08-14 2007-09-26 Univ Leuven Kath Alpha synuclein toxicity
JP5572090B2 (ja) 2007-08-15 2014-08-13 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 テトラヒドロピラン核酸類似体
US8012116B2 (en) 2007-08-17 2011-09-06 Marc Ronald Del Bigio Device to reduce brain edema by surface dialysis and cooling
DK2205737T3 (da) 2007-10-04 2013-05-21 Santaris Pharma As Mikromirer
US20090123575A1 (en) 2007-11-12 2009-05-14 Thomas Lake Blended Compositions for Treatment of Alzheimer's Disease and Other Amyloidoses
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
US20090176729A1 (en) 2007-12-14 2009-07-09 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
WO2009124238A1 (en) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
US20100056622A1 (en) 2008-08-27 2010-03-04 Lauterbach Edward C Methods of Using Ramelteon to Treat Patients Suffering from a Variety of Neurodegenerative Diseases
DK2356129T3 (da) 2008-09-24 2013-05-13 Isis Pharmaceuticals Inc Substituerede alpha-L-bicykliske nukleosider
EP3521309A1 (en) * 2008-12-19 2019-08-07 Biogen International Neuroscience GmbH Human anti-alpha-synuclein antibodies
ES2505243T3 (es) 2009-04-09 2014-10-09 Amicus Therapeutics, Inc. Agentes terapéuticos para prevenir y/o tratar trastornos degenerativos del sistema nervioso central
PT3449926T (pt) 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
EP2490699A1 (en) 2009-10-20 2012-08-29 Santaris Pharma A/S Oral delivery of therapeutically effective lna oligonucleotides
KR20130103662A (ko) 2010-04-19 2013-09-24 엔라이프 떼라퓨틱스, 에스.엘. 올리고뉴클레오티드 분자들의 특정 뉴런 유형들로의 선택적인 전달을 위한 조성물들 및 방법
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2012027558A2 (en) 2010-08-25 2012-03-01 The Trustees Of Columbia University In The City Of New York OPTIMIZED miRNA CONSTRUCTS
WO2012027713A2 (en) 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
WO2012068405A2 (en) 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CA2863253A1 (en) 2011-09-07 2013-03-14 Marina Biotech, Inc. Synthesis and uses of nucleic acid compounds with conformationally restricted monomers
EP2761002B1 (en) 2011-09-28 2021-11-17 Royal College of Surgeons in Ireland Inhibition of microrna-134 for the treatment of seizure-related disorders and neurologic injuries
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
EP2719407A1 (en) 2012-10-09 2014-04-16 EU Sol Biotech Co., Ltd. Kit for treating brain injury or stroke
EP4144845B1 (en) 2012-10-12 2024-04-24 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
JP6452614B2 (ja) 2012-11-15 2019-01-16 ロシュ イノベーション センター コペンハーゲン エーエス オリゴヌクレオチドコンジュゲート
WO2014110291A1 (en) 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
US9701708B2 (en) 2013-01-31 2017-07-11 Ionis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified coupling protocols
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
PE20152002A1 (es) 2013-05-01 2016-01-21 Isis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de ttr y vhb
SG10201908122XA (en) 2013-06-27 2019-10-30 Roche Innovation Ct Copenhagen As Antisense oligomers and conjugates targeting pcsk9
EP3041938A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Circular polynucleotides
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US9925277B2 (en) 2013-09-13 2018-03-27 Modernatx, Inc. Polynucleotide compositions containing amino acids
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
MX2016004651A (es) 2013-10-11 2016-08-05 Ionis Pharmaceuticals Inc Composiciones para modular la expresion de c9orf72.
AU2014337156A1 (en) 2013-10-18 2016-05-12 Modernatx, Inc. Compositions and methods for tolerizing cellular systems
EP3076994A4 (en) 2013-12-06 2017-06-07 Modernatx, Inc. Targeted adaptive vaccines
US20170002060A1 (en) 2014-01-08 2017-01-05 Moderna Therapeutics, Inc. Polynucleotides for the in vivo production of antibodies
RU2021109685A (ru) 2014-04-23 2021-04-13 МОДЕРНАТиЭкс, ИНК. Вакцины на основе нуклеиновых кислот
US20180085391A1 (en) 2014-08-08 2018-03-29 Modernatx, Inc. Compositions and methods for the treatment of ophthalmic diseases and conditions
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
JP2017536119A (ja) 2014-11-19 2017-12-07 ロシュ イノベーション センター コペンハーゲン エーエス キラルホスホロチオエート連結を含むｌｎａギャップマーオリゴヌクレオチド
EP3253871A1 (en) 2015-02-04 2017-12-13 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
UY36550A (es) 2015-02-04 2016-08-31 Roche Innovation Ct Copenhagen As Oligómeros antisentido de tau y sus usos
HK1250243A1 (zh) 2015-04-15 2018-12-07 哈德森医学研究所 治疗方法
EP3722424A1 (en) 2015-04-16 2020-10-14 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
CN108025089A (zh) 2015-07-22 2018-05-11 波涛生命科学有限公司 寡核苷酸组合物及其方法
US20210052631A1 (en) 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US11116843B2 (en) 2015-09-25 2021-09-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US11234995B2 (en) 2016-01-07 2022-02-01 Osaka University α-synuclein expression inhibitor
TWI840950B (zh) 2016-03-14 2024-05-01 瑞士商赫孚孟拉羅股份公司 用於降低pd-l1表現之寡核苷酸
EP3448875A4 (en) 2016-04-29 2020-04-08 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
CN109477108A (zh) 2016-05-04 2019-03-15 波涛生命科学有限公司 寡核苷酸组合物和其方法
AR109750A1 (es) 2016-09-29 2019-01-23 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de tau
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
WO2019009298A1 (ja) 2017-07-05 2019-01-10 国立大学法人大阪大学 α－シヌクレイン発現抑制剤
US20200172903A1 (en) 2017-07-05 2020-06-04 Osaka University ENA ANTISENSE OLIGONUCLEOTIDE FOR INHIBITION OF alpha-SYNUCLEIN EXPRESSION
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
MX2020007433A (es) 2018-01-12 2020-09-14 Bristol Myers Squibb Co Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos.
KR20200109338A (ko) 2018-01-12 2020-09-22 로슈 이노베이션 센터 코펜하겐 에이/에스 알파-시누클레인 안티센스 올리고뉴클레오티드 및 이의 용도
WO2019195519A1 (en) 2018-04-06 2019-10-10 Ionis Pharmaceuticals, Inc. Methods of modulating antisense activity
WO2019217708A1 (en) 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
AU2019287635B2 (en) 2018-06-14 2026-02-12 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
CA3106986A1 (en) 2018-07-25 2020-01-30 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn2 expression
US20220280545A1 (en) 2018-09-10 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating cln3 expression
US20220031731A1 (en) 2018-09-20 2022-02-03 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of lmna expression
EP3884053A4 (en) 2018-11-21 2023-02-01 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING PRION EXPRESSION
EP3897837A4 (en) 2018-12-21 2023-08-16 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING PMP22 EXPRESSION
UY38562A (es) 2019-01-29 2020-08-31 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de app

Also Published As

Publication number Publication date
US20250327072A1 (en) 2025-10-23
US20200354720A1 (en) 2020-11-12
EA202091695A1 (ru) 2021-02-08
MY203356A (en) 2024-06-25
JP2024037914A (ja) 2024-03-19
IL275902B1 (en) 2025-06-01
KR20200140793A (ko) 2020-12-16
AU2019208006B2 (en) 2024-11-21
CO2020008425A2 (es) 2020-08-21
KR102840298B1 (ko) 2025-07-31
SA520412423B1 (ar) 2024-03-17
US20210180065A1 (en) 2021-06-17
BR112020013994A2 (pt) 2020-12-08
EP3737387A1 (en) 2020-11-18
CA3088112A1 (en) 2019-07-18
US12180478B2 (en) 2024-12-31
CN120310791A (zh) 2025-07-15
US11447775B2 (en) 2022-09-20
US20250136988A1 (en) 2025-05-01
IL275902A (en) 2020-08-31
SG11202006526YA (en) 2020-08-28
US11359197B2 (en) 2022-06-14
PE20201501A1 (es) 2020-12-29
US20230193260A1 (en) 2023-06-22
JP7455746B2 (ja) 2024-03-26
AU2019208006A1 (en) 2020-07-23
WO2019140231A1 (en) 2019-07-18
IL275902B2 (en) 2025-10-01
JP2021510512A (ja) 2021-04-30
CN111836624A (zh) 2020-10-27

Similar Documents

Publication Publication Date Title
CO2020009715A2 (es) Oligonucleótidos antisentido que actúan sobre alfa-sinucleína, y usos de estos
CO2020008425A2 (es) Oligonucleótidos antisentido que actúan sobre alfa-sinucleína, y usos de estos
CO2020008988A2 (es) Oligonucleótidos antisentido para alfa-sinucleína y usos de los mismos
CL2017001956A1 (es) Oligomeros antisentido de tau y uso de los mismos
MX2020008581A (es) Oligonucleotidos antisentido de la proteina cinasa del tipo ii delta dependiente del calcio/calmodulina (camk2d) y sus usos.
MX2015013948A (es) Composicion farmaceutica, metodos para tratamiento y sus usos.
CL2016002312A1 (es) Modulador del receptor de andrógeno y usos de este.
CL2017001893A1 (es) Uso de prg4 como agente antiinflamatorio
MX391624B (es) Métodos de uso de indazol-3-carboxamidas y su uso como inhibidores de la ruta de señalización de wnt/b-catenina.
GT201400222A (es) Agentes para tratar trastornos que implican la modulación de receptores de rianodina
MX388693B (es) Composiciones de nanoparticulas para terapia sostenida.
MX2018005825A (es) Celulas inmunes modificadas y usos de las mismas.
NI201500150A (es) TRATAMIENTO DE CÁNCER CON DIHIDROPIRAZINO-PIRAZINAS×Se proporcionan en la presente método
MX2017014257A (es) Metodos de tratamiento de trastornos linfoproliferativos asociados al virus de epstein-barr por terapia con celulas t.
MX2017012926A (es) Inhibidores y sus usos.
MX2021003263A (es) Combinacion de un antagonista de proteina de muerte programada 1 (pd-1), un inhibidor de ataxia telangiectasia y proteina relacionada con rad3 (atr) y un agente de platino para el tratamiento de cancer.
BR112018067967A2 (pt) tratamento de pacientes com hipercolesterolemia familiar homozigótica por meio de terapia hipolipemiante
CL2019000846A1 (es) Proteína terapéutica.
CL2022001871A1 (es) Oligonucleótidos antisentido que actúan sobre alfa-sinucleína, y usos de estos
MX343409B (es) Combinaciones farmaceuticas que incluyen conjugados antiinflamatorios y antioxidantes utiles para tratar trastornos metabolicos.
AR119334A1 (es) Inhibidores de la traducción y sus usos
CY1122708T1 (el) Ενωσεις οξα-διαζασπειρο με δραση εναντι του πονου
AR110113A1 (es) Anticuerpo anti-selectina y sus usos
CL2023002644A1 (es) Oligonucleótidos antisentido para inhibir la expresión de la alfa-sinucleína
AR122753A2 (es) Inhibidores selectivos de proteínas cinasas delta pi3k, composiciones farmacéuticas, usos, y métodos de tratamiento y/o prevención de las enfermedades o trastornos mediados por cinasas