SG11202006528XA - Antisense oligonucleotides targeting alpha-synuclein and uses thereof - Google Patents

Antisense oligonucleotides targeting alpha-synuclein and uses thereof

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Publication number
SG11202006528XA
SG11202006528XA SG11202006528XA SG11202006528XA SG11202006528XA SG 11202006528X A SG11202006528X A SG 11202006528XA SG 11202006528X A SG11202006528X A SG 11202006528XA SG 11202006528X A SG11202006528X A SG 11202006528XA SG 11202006528X A SG11202006528X A SG 11202006528XA
Authority
SG
Singapore
Prior art keywords
synuclein
antisense oligonucleotides
oligonucleotides targeting
targeting alpha
alpha
Prior art date
Application number
SG11202006528XA
Other languages
English (en)
Inventor
Richard Olson
Angela Cacace
Jr Jere Meredith
Nino Devidze
James Loy
Carl Baldick
Annapurna Pendri
Ivar Mcdonald
Peter Hagedorn
Marianne Jensen
Original Assignee
Bristol Myers Squibb Co
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 Bristol Myers Squibb Co, Roche Innovation Ct Copenhagen As filed Critical Bristol Myers Squibb Co
Publication of SG11202006528XA publication Critical patent/SG11202006528XA/en

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    • 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
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    • 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
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    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
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    • C12N2310/323—Chemical structure of the sugar modified ring structure
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SG11202006528XA 2018-01-12 2019-01-11 Antisense oligonucleotides targeting alpha-synuclein and uses thereof SG11202006528XA (en)

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
SG11202006528XA true SG11202006528XA (en) 2020-08-28

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SG11202006528XA SG11202006528XA (en) 2018-01-12 2019-01-11 Antisense oligonucleotides targeting alpha-synuclein and uses thereof

Country Status (15)

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

Families Citing this family (10)

* 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
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
SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CN120310791A (zh) 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
WO2019157531A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
JP2023509477A (ja) * 2020-01-06 2023-03-08 オーム ライフテック インコーポレイテッド 神経障害の処置のためのアンチセンスオリゴヌクレオチド
WO2022189363A1 (en) * 2021-03-08 2022-09-15 Les Laboratoires Servier Antisense oligonucleotides for inhibiting alpha-synuclein expression
KR102680316B1 (ko) * 2021-11-01 2024-07-01 오토텔릭바이오 주식회사 안티센스 올리고뉴클레오타이드
JP2026507572A (ja) * 2023-02-17 2026-03-04 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 アルファ－シヌクレイン発現を調節するための化合物および方法
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
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
JPH0874B2 (ja) 1990-07-27 1996-01-10 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 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド
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
DE69233046T2 (de) 1991-10-24 2004-03-04 Isis Pharmaceuticals, Inc., Carlsfeld 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
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
AU3222793A (en) 1991-11-26 1993-06-28 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5840708A (en) 1992-12-14 1998-11-24 Allegheny University Of The Health Sciences 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
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
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
US6913745B1 (en) 1997-12-02 2005-07-05 Neuralab Limited Passive immunization of Alzheimer's disease
JP2002516256A (ja) 1998-05-26 2002-06-04 アイ・シー・エヌ・フアーマシユーテイカルズ・インコーポレイテツド 二環式糖成分を有する新規ヌクレオシド
US6833361B2 (en) 1998-05-26 2004-12-21 Ribapharm, Inc. Nucleosides having bicyclic sugar moiety
WO1999061066A2 (en) 1998-05-27 1999-12-02 Avigen, Inc. Convection-enhanced delivery of aav vectors
WO2000002919A1 (en) 1998-07-13 2000-01-20 Merck & Co., Inc. Growth hormone secretagogue related receptors and nucleic acids
DE69939663D1 (de) 1998-11-05 2008-11-13 Univ Jefferson Behandlung von morbus parkinson mit oligonukleotiden
US20040226056A1 (en) 1998-12-22 2004-11-11 Myriad Genetics, Incorporated Compositions and methods for treating neurological disorders and diseases
EP1140973A4 (en) 1998-12-22 2002-10-30 Myriad Genetics Inc PROTEIN-PROTEIN INTERACTIONS IN NEURODEGENERATIVE DISORDERS
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
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
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
EP1238069A2 (en) 1999-07-15 2002-09-11 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
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
EP1268856A2 (de) 2000-04-07 2003-01-02 Epigenomics AG Detektion von snp's und cytosin-methylierungen
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
US20020119155A1 (en) 2000-10-17 2002-08-29 Myriad Genetics, Inc. Protein-protein interactions in neurodegenerative diseases
US20020124273A1 (en) 2000-10-17 2002-09-05 Jean-Marc Roch 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
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
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
US20050019320A1 (en) 2001-12-27 2005-01-27 Eiji Sugaru Remedies for anorexia or lifestyle-related diseases and method of screening the same
WO2003101927A1 (en) 2002-05-31 2003-12-11 Proteotech, Inc. Compounds, compositions and methods for the treatment of amyloid diseases and synucleinopathies such as alzheimer's disease, type 2 diabetes, and parkinson's disease
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
AU2003291755A1 (en) 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004046160A2 (en) 2002-11-18 2004-06-03 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
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
CA2522497A1 (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
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
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
US7595306B2 (en) 2003-06-09 2009-09-29 Alnylam Pharmaceuticals Inc Method of treating neurodegenerative disease
WO2005004794A2 (en) 2003-06-09 2005-01-20 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
CA2538252C (en) 2003-09-18 2014-02-25 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
PL1716235T3 (pl) 2004-02-09 2012-11-30 Ulrich Bogdahn Inhibitory przekazywania sygnałów TGF-R do leczenia zaburzeń ośrodkowego układu nerwowego
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
US7919472B2 (en) 2004-09-17 2011-04-05 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
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
EP3470072A1 (en) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
WO2007011907A2 (en) 2005-07-19 2007-01-25 University Of Rochester Alpha-synuclein antibodies and methods related thereto
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
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
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
KR20080068019A (ko) 2005-09-15 2008-07-22 산타리스 팔마 에이/에스 아포지단백질-ｂ100 발현 억제용 ｒｎａ 길항제 화합물
CA2634598A1 (en) 2005-12-23 2007-07-05 Link Medicine Corporation Treatment of synucleinopathies
EP1991677A2 (en) 2006-01-26 2008-11-19 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
JP2009536222A (ja) 2006-05-05 2009-10-08 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 Ｐｃｓｋ９の発現を調節するための化合物および方法
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
GB0610183D0 (en) 2006-05-23 2006-06-28 Isis Innovation Treatment of neurodegenerative diseases
WO2008003943A2 (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
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
PL2583978T3 (pl) 2007-02-23 2016-07-29 Prothena Biosciences Ltd Co Profilaktyka i leczenie chorób synukleinopatycznych i amyloidogennych
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
GB0715809D0 (en) 2007-08-14 2007-09-26 Univ Leuven Kath Alpha synuclein toxicity
KR101654007B1 (ko) 2007-08-15 2016-09-05 아이오니스 파마수티컬즈, 인코포레이티드 테트라하이드로피란 핵산 유사체
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
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
US9290534B2 (en) 2008-04-04 2016-03-22 Ionis Pharmaceuticals, Inc. Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside
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
CN102317316B (zh) 2008-12-19 2014-08-13 帕尼玛制药股份公司 人抗α突触核蛋白自身抗体
BRPI1015472A2 (pt) 2009-04-09 2015-11-24 Amicus Therapeutics Inc métodos para prevenção e/ou tratamento de doenças degenerativas do sistema nervoso central
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
US9012421B2 (en) 2009-08-06 2015-04-21 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
CA2796722C (en) 2010-04-19 2021-06-01 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
US9290759B2 (en) 2010-08-25 2016-03-22 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
CA2817960C (en) 2010-11-17 2020-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2753631A1 (en) 2011-09-07 2014-07-16 Marina Biotech, Inc. Synthesis and uses of nucleic acid compounds with conformationally restricted monomers
WO2013045652A1 (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
WO2013086207A1 (en) 2011-12-06 2013-06-13 The Ohio State University 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
US20140155816A1 (en) 2012-10-09 2014-06-05 Eu Sol Biotech Co., Ltd. Method for treating brain injury or stroke
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
EP2911695B1 (en) 2012-10-26 2023-11-29 Palomo Limited Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
CN104837996A (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
WO2014110291A1 (en) 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
DK2951191T3 (en) 2013-01-31 2019-01-14 Ionis Pharmaceuticals Inc PROCEDURE FOR MANUFACTURING OLIGOMERIC COMPOUNDS USING MODIFIED CLUTCH PROTOCOLS
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
HUE048738T2 (hu) 2013-06-27 2020-08-28 Roche Innovation Ct Copenhagen As Antiszensz oligomerek és konjugátumok, amelyek a PCK9-t célozzák meg
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
EP3041934A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2015038892A1 (en) 2013-09-13 2015-03-19 Moderna Therapeutics, Inc. Polynucleotide compositions containing amino acids
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
US10221414B2 (en) 2013-10-11 2019-03-05 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
CN105917005B (zh) 2013-11-18 2020-09-11 迪阿米尔有限责任公司 使用来自体液的miRNA检测和监控帕金森病(PD)的方法
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
PL4023249T3 (pl) 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
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
WO2016079183A1 (en) * 2014-11-19 2016-05-26 Roche Innovation Center Copenhagen A/S Lna chiral phosphorothioates
WO2016127002A1 (en) 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
US11077132B2 (en) * 2015-02-04 2021-08-03 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
CN107709560A (zh) 2015-04-15 2018-02-16 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
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
JP2018528783A (ja) 2015-09-25 2018-10-04 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． コンジュゲートアンチセンス化合物及びその使用
US20210052631A1 (en) 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2017119463A1 (ja) 2016-01-07 2017-07-13 国立大学法人大阪大学 α－シヌクレイン発現抑制剤
RU2747822C2 (ru) * 2016-03-14 2021-05-14 Ф. Хоффманн-Ля Рош Аг Олигонуклеотиды для понижения экспрессии pd-l1
US20190224339A1 (en) 2016-04-29 2019-07-25 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
AR109750A1 (es) 2016-09-29 2019-01-23 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de tau
HUE065170T2 (hu) 2016-09-29 2024-05-28 Biogen Ma Inc Vegyületek és módszerek a TAU expresszió csökkentésére
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
SG11202006142PA (en) 2018-01-12 2020-07-29 Roche Innovation Ct Copenhagen As Alpha-synuclein antisense oligonucleotides and uses thereof
CN120310791A (zh) * 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
US20210130826A1 (en) 2018-04-06 2021-05-06 Ionis Pharmaceuticals, Inc. Methods of modulating antisense activity
EP4663758A3 (en) 2018-05-09 2026-04-29 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
AR115847A1 (es) 2018-07-25 2021-03-03 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de la atxn2
WO2020055917A1 (en) 2018-09-10 2020-03-19 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating cln3 expression
WO2020061497A1 (en) 2018-09-20 2020-03-26 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of lmna expression
US12281305B2 (en) 2018-11-21 2025-04-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing prion expression
JP7561129B2 (ja) 2018-12-21 2024-10-03 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 Ｐｍｐ２２の発現を低減するための化合物及び方法
WO2020160163A1 (en) 2019-01-29 2020-08-06 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression

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