MX2020007439A - 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
MX2020007439A
MX2020007439A MX2020007439A MX2020007439A MX2020007439A MX 2020007439 A MX2020007439 A MX 2020007439A MX 2020007439 A MX2020007439 A MX 2020007439A MX 2020007439 A MX2020007439 A MX 2020007439A MX 2020007439 A MX2020007439 A MX 2020007439A
Authority
MX
Mexico
Prior art keywords
act
antisense oligonucleotides
synuclein
alpha
protein
Prior art date
Application number
MX2020007439A
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 MX2020007439A publication Critical patent/MX2020007439A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711Alzheimer's disease; Amyloid plaque core protein
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/395Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/505Medicinal preparations containing antigens or antibodies comprising antibodies
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Mycology (AREA)
  • Endocrinology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (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.
MX2020007439A 2018-01-12 2019-01-11 Oligonucleotidos antisentido que actuan sobre alfa-sinucleina y usos de estos. MX2020007439A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862616994P 2018-01-12 2018-01-12
PCT/US2019/013255 WO2019140236A1 (en) 2018-01-12 2019-01-11 Antisense oligonucleotides targeting alpha-synuclein and uses thereof

Publications (1)

Publication Number Publication Date
MX2020007439A true MX2020007439A (es) 2020-09-14

Family

ID=65269098

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020007439A MX2020007439A (es) 2018-01-12 2019-01-11 Oligonucleotidos antisentido que actuan sobre alfa-sinucleina y usos de estos.

Country Status (15)

Country Link
US (2) US12241065B2 (es)
EP (1) EP3737761A1 (es)
JP (2) JP7547201B2 (es)
KR (1) KR102839166B1 (es)
CN (1) CN111819283A (es)
AU (1) AU2019208011B2 (es)
BR (1) BR112020014011A2 (es)
CA (1) CA3087966A1 (es)
CO (1) CO2020009715A2 (es)
EA (1) EA202091693A1 (es)
IL (1) IL275903B2 (es)
MX (1) MX2020007439A (es)
PE (1) PE20211749A1 (es)
SG (1) SG11202006528XA (es)
WO (1) WO2019140236A1 (es)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110506057B (zh) 2017-02-17 2023-09-29 百时美施贵宝公司 Alpha突触核蛋白抗体及其应用
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
BR112020013994A2 (pt) * 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
CA3163789A1 (en) * 2020-01-06 2021-07-15 Veenu AISHWARYA Antisense oligonucleotides for treatment of neurological disorders
PE20240696A1 (es) * 2021-03-08 2024-04-10 Servier Lab Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina
KR102680316B1 (ko) * 2021-11-01 2024-07-01 오토텔릭바이오 주식회사 안티센스 올리고뉴클레오타이드
WO2024173759A2 (en) * 2023-02-17 2024-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating alpha-synuclein expression
WO2024196752A2 (en) * 2023-03-17 2024-09-26 Duke University Compositions for cell specific snca-targeted gene therapy and methods of making and using same

Family Cites Families (184)

* 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
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
WO1993007883A1 (en) 1991-10-24 1993-04-29 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
BR9106702A (pt) 1990-07-27 1993-06-08 Isis Pharmaceuticals Inc Analogo de oligonucleotideos e processos para modular a producao de uma proteina por um organismo e para tratar um organismo
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
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
CA2151585A1 (en) 1992-12-14 1994-06-23 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
AU2582197A (en) 1996-03-15 1997-10-01 Start Technology Partnership Methods and compositions for diagnosis and treatment of pathological conditions related to abnormal dopamine receptor expression
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
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
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
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6913745B1 (en) 1997-12-02 2005-07-05 Neuralab Limited Passive immunization of Alzheimer's disease
CA2333380A1 (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
DE69929600T2 (de) 1998-05-27 2006-09-07 Avigen Inc., Alameda Konvektion-erhöhte verabreichung aadc-kodierende aav vektoren
JP2002520011A (ja) 1998-07-13 2002-07-09 メルク・アンド・カンパニー・インコーポレーテッド 成長ホルモン分泌促進物質関連受容体および核酸
EP1135113B1 (en) 1998-11-05 2008-10-01 Thomas Jefferson University Treatment of parkinson's disease with oligonucleotides
EP1140973A4 (en) 1998-12-22 2002-10-30 Myriad Genetics Inc PROTEIN-PROTEIN INTERACTIONS IN NEURODEGENERATIVE DISORDERS
US20040226056A1 (en) 1998-12-22 2004-11-11 Myriad Genetics, Incorporated Compositions and methods for treating neurological disorders and diseases
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma A/S L-ribo-lna analogues
WO2000066725A1 (fr) 1999-05-04 2000-11-09 Aventis Pharma S.A. Utilisation d'oligonucleotides antisens de no-synthase inductible dans la prevention et le traitement de l'ischemie cerebrale
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
WO2001005963A2 (en) 1999-07-15 2001-01-25 Mcgill University ANTISENSE 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
WO2001057252A2 (en) 2000-02-04 2001-08-09 Aeomica, Inc. Methods and apparatus for high-throughput detection and characterization of alternatively spliced genes
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
US20020115606A1 (en) 2000-10-17 2002-08-22 Myriad Genetics, Inc. Protein-protein interactions in neurodegenerative diseases
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
JP2005504020A (ja) 2001-07-03 2005-02-10 アイシス・ファーマシューティカルス・インコーポレーテッド ヌクレアーゼ耐性キメラオリゴヌクレオチド
US6455308B1 (en) 2001-08-01 2002-09-24 Isis Pharmaceuticals, Inc. Antisense modulation of serum amyloid A4 expression
JPWO2003030936A1 (ja) 2001-10-02 2005-01-20 住友製薬株式会社 生活習慣病又は拒食症治療薬及びそのスクリーニング方法
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
PT2527315E (pt) 2002-05-31 2014-06-24 Proteotech Inc Compostos, composições e métodos para o tratamento de doenças da amiloide e sinucleínopatias tais como doença de alzheimer, diabetes do tipo 2, e doença de parkinson
US20070276034A1 (en) 2002-05-31 2007-11-29 Luke Esposito Compounds, compositions and methods for the treatment of synucleinopathies
US8506959B2 (en) 2002-11-01 2013-08-13 Neotope Biosciences Limited Prevention and treatment of synucleinopathic and amyloidogenic disease
TW200509968A (en) 2002-11-01 2005-03-16 Elan Pharm Inc Prevention and treatment of synucleinopathic disease
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US9045518B2 (en) 2002-11-18 2015-06-02 Santaris Pharma A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
CN1964630A (zh) 2003-02-13 2007-05-16 耶希瓦大学艾伯塔·爱恩斯坦医学院 通过控制下丘脑的长链脂肪酰基-辅酶A(LC-CoA)的水平来调控食物摄取和葡萄糖产生
WO2004093790A2 (en) 2003-04-16 2004-11-04 Whitehead Institute For Biomedical Research Yeast ectopically expressing abnormally processed proteins and uses therefor
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
EP1481685A1 (en) 2003-05-30 2004-12-01 Aventis Pharma Deutschland GmbH Use of PAM
EP1481680A1 (en) 2003-05-30 2004-12-01 Aventis Pharma Deutschland GmbH Use of S1P
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
EP2508608A1 (en) 2003-06-09 2012-10-10 Alnylam Pharmaceuticals Inc. Method of treating neurodegenerative disease
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
WO2005027962A1 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. 4’-thionucleosides and oligomeric compounds
AU2005210151B2 (en) 2004-02-09 2010-07-01 Aigner, Ludwig Dr. Inhibitors of TGF-R signaling for treatment of CNS disorders
WO2005097817A2 (en) 2004-04-05 2005-10-20 Alnylam Pharmaceuticals, Inc. Process and reagents for oligonucleotide synthesis and purification
EP2397563A3 (en) 2004-09-17 2012-07-18 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
WO2006039253A2 (en) 2004-09-29 2006-04-13 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
WO2006093034A1 (ja) 2005-03-03 2006-09-08 National University Corporation Nagoya University 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
SI2548560T1 (sl) 2005-06-23 2015-12-31 Isis Pharmaceuticals, Inc. Sestavki in postopki za moduliranje izrezovanja smn2
CA2657953A1 (en) 2005-07-19 2007-01-25 University Of Rochester Alpha-synuclein antibodies and methods related thereto
WO2007030581A2 (en) 2005-09-09 2007-03-15 Oregon Health & Science University Neuroprotectants
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
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
EP1968591A4 (en) 2005-12-23 2010-02-17 Link Medicine Corp TREATMENT OF SYNUCLEINOPATHIES
PT2161038E (pt) 2006-01-26 2014-03-10 Isis Pharmaceuticals Inc Composições e suas utilizações dirigidas à huntingtina
JP5342881B2 (ja) 2006-01-27 2013-11-13 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 6−修飾された二環式核酸類似体
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 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
AU2007270916A1 (en) 2006-07-03 2008-01-10 Zapaloid Limited Inhibition of alpha-synuclein aggregation
KR20080036902A (ko) 2006-10-24 2008-04-29 재단법인서울대학교산학협력재단 아밀로이드 형성 펩타이드 또는 단백질의 가용성 회합체에선택적으로 작용하는 절단제
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
PL2118300T3 (pl) 2007-02-23 2015-11-30 Prothena Biosciences Ltd Zapobieganie i leczenie synukleinopatii i amyloidozy
WO2008113832A2 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S SHORT RNA 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
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
GB0715809D0 (en) 2007-08-14 2007-09-26 Univ Leuven Kath Alpha synuclein toxicity
WO2009023855A2 (en) 2007-08-15 2009-02-19 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
US8012116B2 (en) 2007-08-17 2011-09-06 Marc Ronald Del Bigio Device to reduce brain edema by surface dialysis and cooling
EP2623599B1 (en) 2007-10-04 2019-01-02 Roche Innovation Center Copenhagen A/S Micromirs
US20090123575A1 (en) 2007-11-12 2009-05-14 Thomas Lake Blended Compositions for Treatment of Alzheimer's Disease and Other Amyloidoses
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 (da) 2008-04-04 2013-12-02 Isis Pharmaceuticals Inc Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider
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
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
AU2009328505B2 (en) 2008-12-19 2014-11-27 Panima Pharmaceuticals Ag Human anti-alpha-synuclein autoantibodies
CA2758187C (en) 2009-04-09 2017-03-07 Robert Boyd Methods for preventing and/or treating degenerative disorders of the central nervous system
PT3305302T (pt) 2009-06-17 2018-12-14 Biogen Ma Inc Composições e métodos de modulação de excisões de smn2 em um sujeito
EP2462153B1 (en) 2009-08-06 2015-07-29 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
WO2011087804A2 (en) 2009-12-21 2011-07-21 Medtronic, Inc. Peptide-polynucleotide compositions, and methods for transfecting a cell with dna and treatment of neurodegenerative disease
RU2664452C2 (ru) 2010-04-19 2018-08-17 Нлифе Терапеутикс, С.Л. Конъюгат, медицинское средство и способы лечения и/или профилактики депрессии и болезни, связанной с отложением телец Леви
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
CA3077910A1 (en) 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
EP3453761A1 (en) 2011-08-29 2019-03-13 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CN104136451A (zh) 2011-09-07 2014-11-05 玛瑞纳生物技术有限公司 具有构象限制的单体的核酸化合物的合成和用途
CA2850258A1 (en) 2011-09-28 2013-04-04 Royal College Of Surgeons In Ireland Inhibition of microrna-134 for the treatment of seizure-related disorders and neurologic injuries
US9725716B2 (en) 2011-12-06 2017-08-08 Ohio State Innovation Foundation and Research Institute at Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
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
US20160138014A1 (en) 2012-10-12 2016-05-19 Isis Pharmaceuticals, Inc. Antisense compounds and uses thereof
AU2013336581A1 (en) 2012-10-26 2015-06-11 Nlife Therapeutics, S.L. Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
US10077443B2 (en) 2012-11-15 2018-09-18 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
EP2943225A4 (en) 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
WO2014120861A2 (en) 2013-01-31 2014-08-07 Isis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified coupling protocols
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
ES2770667T3 (es) 2013-06-27 2020-07-02 Roche Innovation Ct Copenhagen As Oligómeros antisentido y conjugados que se dirigen a PCSK9
US20160194625A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
EP3043826A4 (en) 2013-09-13 2017-05-24 Moderna Therapeutics, Inc. Polynucleotide compositions containing amino acids
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
JP2016538829A (ja) 2013-10-03 2016-12-15 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. 低密度リポタンパク質受容体をコードするポリヌクレオチド
EP4166667A3 (en) 2013-10-11 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
AU2014337156A1 (en) 2013-10-18 2016-05-12 Modernatx, Inc. Compositions and methods for tolerizing cellular systems
JP6930832B2 (ja) 2013-11-18 2021-09-01 ディアミール, エルエルシーDiamir, Llc パーキンソン病(PD)の検出およびモニタリングのための、体液からのmiRNAを使用する方法
US20170173128A1 (en) 2013-12-06 2017-06-22 Moderna TX, Inc. Targeted adaptive vaccines
US20170002060A1 (en) 2014-01-08 2017-01-05 Moderna Therapeutics, Inc. Polynucleotides for the in vivo production of antibodies
SG10201912038TA (en) 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
WO2016022914A1 (en) 2014-08-08 2016-02-11 Moderna Therapeutics, Inc. Compositions and methods for the treatment of ophthalmic diseases and conditions
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
JP2017536366A (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 Lnaキラルホスホロチオエート
WO2016127002A1 (en) * 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
AU2016215155A1 (en) * 2015-02-04 2017-08-17 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
HK1250243A1 (zh) 2015-04-15 2018-12-07 哈德森医学研究所 治疗方法
RS60230B1 (sr) 2015-04-16 2020-06-30 Ionis Pharmaceuticals Inc Kompozicije za moduliranje ekspresije c9orf72
CN108135921B (zh) 2015-07-22 2023-10-17 波涛生命科学有限公司 寡核苷酸组合物及其方法
EP3353306A4 (en) 2015-09-25 2019-06-05 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US20210052631A1 (en) 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
JP6569920B2 (ja) 2016-01-07 2019-09-04 国立大学法人大阪大学 α−シヌクレイン発現抑制剤
KR20250065735A (ko) 2016-03-14 2025-05-13 에프. 호프만-라 로슈 아게 Pd-l1 발현의 감소를 위한 올리고뉴클레오티드
EP3448875A4 (en) 2016-04-29 2020-04-08 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
JP2019516680A (ja) 2016-05-04 2019-06-20 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. オリゴヌクレオチド組成物およびその方法
ES2963428T3 (es) 2016-09-29 2024-03-27 Biogen Ma Inc Compuestos y métodos para reducir la expresión de Tau
AR109750A1 (es) 2016-09-29 2019-01-23 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de 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
WO2019009299A1 (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するENAアンチセンスオリゴヌクレオチド
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
BR112020013994A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos
AU2019207859A1 (en) 2018-01-12 2020-07-02 Roche Innovation Center Copenhagen A/S Alpha-synuclein antisense oligonucleotides and uses thereof
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
WO2019195519A1 (en) 2018-04-06 2019-10-10 Ionis Pharmaceuticals, Inc. Methods of modulating antisense activity
SG11202010215TA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn3 expression
CA3103429A1 (en) 2018-06-14 2019-12-19 Don W. Cleveland 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
BR112021000308A2 (pt) 2018-07-25 2021-04-13 Ionis Pharmaceuticals, Inc. Compostos e métodos para redução da expressão de atxn2
JP7511563B2 (ja) 2018-09-10 2024-07-05 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 Cln3の発現を調節するための化合物及び方法
US20220031731A1 (en) 2018-09-20 2022-02-03 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of lmna expression
SG11202103794QA (en) 2018-11-21 2021-05-28 Ionis Pharmaceuticals Inc Compounds and methods for reducing prion expression
WO2020132558A1 (en) 2018-12-21 2020-06-25 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
AU2019208011B2 (en) 2025-06-05
US20250188463A1 (en) 2025-06-12
CO2020009715A2 (es) 2020-08-21
IL275903B1 (en) 2025-11-01
CN111819283A (zh) 2020-10-23
BR112020014011A2 (pt) 2020-12-08
SG11202006528XA (en) 2020-08-28
US20200362347A1 (en) 2020-11-19
EP3737761A1 (en) 2020-11-18
JP2024122973A (ja) 2024-09-10
PE20211749A1 (es) 2021-09-06
JP7547201B2 (ja) 2024-09-09
EA202091693A1 (ru) 2021-04-14
JP2021510511A (ja) 2021-04-30
WO2019140236A1 (en) 2019-07-18
CA3087966A1 (en) 2019-07-18
IL275903A (en) 2020-08-31
AU2019208011A1 (en) 2020-07-23
IL275903B2 (en) 2026-03-01
US12241065B2 (en) 2025-03-04
KR20200140240A (ko) 2020-12-15
KR102839166B1 (ko) 2025-07-28

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.
CL2016002312A1 (es) Modulador del receptor de andrógeno y usos de este.
CL2017001893A1 (es) Uso de prg4 como agente antiinflamatorio
GT201400222A (es) Agentes para tratar trastornos que implican la modulación de receptores de rianodina
MX388693B (es) Composiciones de nanoparticulas para terapia sostenida.
BR112013017154A2 (pt) uso de fitocanabinóide canabidiol (cbd) em combinação com um fármaco antiepiléptico padrão (saed) no tratamento de epilepsia
MX2018005825A (es) Celulas inmunes modificadas y usos de las mismas.
NI201500150A (es) TRATAMIENTO DE CÁNCER CON DIHIDROPIRAZINO-PIRAZINAS×Se proporcionan en la presente método
NI201600021A (es) Inhibidores de rorc2 heterobicicloarilo y sus métodos de uso
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.
BR112018067967A2 (pt) tratamento de pacientes com hipercolesterolemia familiar homozigótica por meio de terapia hipolipemiante
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.
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