DK1910395T3 - Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning - Google Patents

Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning

Info

Publication number
DK1910395T3
DK1910395T3 DK06773838.5T DK06773838T DK1910395T3 DK 1910395 T3 DK1910395 T3 DK 1910395T3 DK 06773838 T DK06773838 T DK 06773838T DK 1910395 T3 DK1910395 T3 DK 1910395T3
Authority
DK
Denmark
Prior art keywords
compositions
methods
smn2 splicing
modulating smn2
modulating
Prior art date
Application number
DK06773838.5T
Other languages
English (en)
Inventor
Brenda F Baker
Adrian R Krainer
Yimin Hua
Original Assignee
Isis Pharmaceuticals Inc
Cold Spring Harbor Lab
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 Isis Pharmaceuticals Inc, Cold Spring Harbor Lab filed Critical Isis Pharmaceuticals Inc
Application granted granted Critical
Publication of DK1910395T3 publication Critical patent/DK1910395T3/da

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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
DK06773838.5T 2005-06-23 2006-06-23 Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning DK1910395T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US69354205P 2005-06-23 2005-06-23
PCT/US2006/024469 WO2007002390A2 (en) 2005-06-23 2006-06-23 Compositions and methods for modulation of smn2 splicing

Publications (1)

Publication Number Publication Date
DK1910395T3 true DK1910395T3 (da) 2013-02-04

Family

ID=37595872

Family Applications (3)

Application Number Title Priority Date Filing Date
DK17185196.7T DK3308788T3 (da) 2005-06-23 2006-06-23 Sammensætninger og fremgangsmåder til modulering af smn2-splejsning
DK06773838.5T DK1910395T3 (da) 2005-06-23 2006-06-23 Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning
DK12188625.3T DK2548560T3 (da) 2005-06-23 2006-06-23 Sammensætninger og fremgangsmåder til modulering af SMN2-splejsning

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DK17185196.7T DK3308788T3 (da) 2005-06-23 2006-06-23 Sammensætninger og fremgangsmåder til modulering af smn2-splejsning

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK12188625.3T DK2548560T3 (da) 2005-06-23 2006-06-23 Sammensætninger og fremgangsmåder til modulering af SMN2-splejsning

Country Status (13)

Country Link
US (6) US8361977B2 (da)
EP (5) EP3470072A1 (da)
CY (2) CY2017040I2 (da)
DK (3) DK3308788T3 (da)
ES (3) ES2397113T3 (da)
HK (1) HK1247103B (da)
HU (2) HUE027486T2 (da)
LT (2) LT3308788T (da)
LU (1) LUC00046I2 (da)
PL (3) PL2548560T3 (da)
PT (3) PT1910395E (da)
SI (2) SI3308788T1 (da)
WO (1) WO2007002390A2 (da)

Families Citing this family (102)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
ES2397113T3 (es) 2005-06-23 2013-03-04 Isis Pharmaceuticals, Inc. Composiciones y procedimientos para modular el corte y empalme de SMN2
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
EP2203173B1 (en) * 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2219680A2 (en) 2007-11-13 2010-08-25 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2009151546A2 (en) * 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8986935B2 (en) 2008-08-13 2015-03-24 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
IT1398361B1 (it) * 2009-01-21 2013-02-22 Fond Santa Lucia Uso di mutanti dominanti negativi di sam68 per il trattamento della sma
JP2012523225A (ja) * 2009-04-10 2012-10-04 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 疾患の処置のためのトリシクロ−ｄｎａアンチセンスオリゴヌクレオチド、組成物及び方法
US20120149757A1 (en) * 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
CN115227709A (zh) * 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
KR102182663B1 (ko) 2010-05-28 2020-11-25 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
EP2582397A4 (en) * 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc COMPOUNDS AND METHOD FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US10017763B2 (en) 2010-09-03 2018-07-10 Sarepta Therapeutics, Inc. dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
IT1405793B1 (it) * 2010-10-15 2014-01-24 Univ Ferrara Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
IL273838B (en) 2011-05-05 2022-09-01 Sarepta Therapeutics Inc Peptide and oligonucleotide conjugates, preparations containing them and their uses
US20140298496A1 (en) * 2011-06-23 2014-10-02 Cold Spring Harbor Laboratory Phenocopy model of disease
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
JP2014533944A (ja) 2011-11-11 2014-12-18 サンタリス ファーマ アー／エスＳａｎｔａｒｉｓ Ｐｈａｒｍａ Ａ／Ｓ Ｓｍｎ２スプライシングの調節のための化合物
JP2015500204A (ja) 2011-11-18 2015-01-05 サレプタ セラピューティクス， インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
WO2013082548A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
AU2012345638C1 (en) * 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
EP2788087A4 (en) 2011-12-06 2015-08-26 Ohio State Innovation Foundation Non-ionic, low-osmolar contrast agents for the administration of antisense oligonucleotides and the treatment of diseases
CN118581086A (zh) 2012-01-27 2024-09-03 比奥马林技术公司 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
KR20150030205A (ko) * 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법
SG11201407486PA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating utrn expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CN112007045B (zh) 2012-07-13 2026-01-06 波涛生命科学有限公司 手性控制
DK2872485T3 (da) 2012-07-13 2021-03-08 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
WO2014110291A1 (en) * 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
WO2014113540A1 (en) * 2013-01-16 2014-07-24 Iowa State University Research Foundation, Inc. A deep intronic target for splicing correction on spinal muscular atrophy gene
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
KR102275504B1 (ko) 2013-09-05 2021-07-09 사렙타 쎄러퓨틱스, 인코퍼레이티드 산성 알파-글루코시다제 내 안티센스-유도된 엑손2 포함
JP2016534035A (ja) * 2013-10-04 2016-11-04 ラナ セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症を治療するための組成物及び方法
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
WO2015108048A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
KR102423317B1 (ko) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 키랄 디자인
US9845469B2 (en) 2014-02-10 2017-12-19 Ohio State Innovation Foundation Antisense oligonucleotides for treatment of spinal muscular atrophy
MA39835A (fr) 2014-04-17 2017-02-22 Biogen Ma Inc Compositions et procédés de modulation de l'épissage du smn2 chez un patient
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
US10858650B2 (en) 2014-10-30 2020-12-08 The General Hospital Corporation Methods for modulating ATRX-dependent gene repression
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
US10851371B2 (en) 2015-04-10 2020-12-01 Ionis Pharmaceuticals, Inc. Modulation of SMN expression
US10849917B2 (en) 2015-06-01 2020-12-01 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type VII collagen
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
AU2016317667A1 (en) * 2015-08-28 2018-03-22 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
AU2016334804B2 (en) 2015-10-09 2022-03-31 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
WO2017062835A2 (en) 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
US9669109B1 (en) 2015-11-24 2017-06-06 Universita Di Ferrara Modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
ES2882500T3 (es) 2015-12-14 2021-12-02 Cold Spring Harbor Laboratory Oligómeros antisentido para el tratamiento del síndrome de Dravet
WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
MY200252A (en) 2016-04-18 2023-12-16 Sarepta Therapeutics Inc Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
JP7075359B2 (ja) 2016-06-14 2022-05-25 バイオジェン・エムエイ・インコーポレイテッド オリゴヌクレオチドの精製のための疎水性相互作用クロマトグラフィー
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
WO2017223258A1 (en) 2016-06-24 2017-12-28 Biogen Ma Inc. Synthesis of thiolated oligonucleotides without a capping step
US11655470B2 (en) 2016-07-05 2023-05-23 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing COL6-related disorders and methods for treating same
US20190321387A1 (en) * 2016-07-15 2019-10-24 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
MX2019010275A (es) 2017-02-28 2019-10-15 Univ Pennsylvania Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este.
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CN110536694A (zh) 2017-04-20 2019-12-03 Atyr 医药公司 用于治疗肺部炎症的组合物和方法
US11549125B2 (en) 2017-08-09 2023-01-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof
SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
US11596646B2 (en) 2017-10-12 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2019079637A2 (en) 2017-10-18 2019-04-25 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMERIC COMPOUNDS
CN120505370A (zh) 2017-11-08 2025-08-19 诺华股份有限公司 制备病毒载体的手段和方法及其用途
US12241065B2 (en) 2018-01-12 2025-03-04 Bristol-Myers Squibb Company 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
WO2019147960A1 (en) 2018-01-25 2019-08-01 Biogen Ma Inc. Methods of treating spinal muscular atrophy
WO2019213525A1 (en) * 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US12228571B2 (en) 2018-06-08 2025-02-18 Novartis Ag Cell-based assay for measuring drug product potency
JP2022509372A (ja) 2018-10-29 2022-01-20 バイオジェン・エムエイ・インコーポレイテッド 血液脳関門輸送の促進のためのヒト化され安定化されたｆｃ５バリアント
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
AU2019389047A1 (en) 2018-11-30 2021-05-20 Novartis Ag AAV viral vectors and uses thereof
KR20210102294A (ko) 2018-12-06 2021-08-19 바이오젠 엠에이 인코포레이티드 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
WO2020191212A1 (en) 2019-03-20 2020-09-24 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
MA55891A (fr) 2019-05-08 2022-03-16 Ajinomoto Kk Synthèses en phase liquide convergente d'oligonucléotides
HUE064071T2 (hu) 2019-07-19 2024-02-28 Biogen Ma Inc Eljárások a spinális izomatrófia kezelésére vagy megelõzésére
EP4706689A2 (en) 2019-12-19 2026-03-11 Entrada Therapeutics, Inc. Compositions for delivery of antisense compounds
PH12022552258A1 (en) 2020-02-28 2023-11-20 Ionis Pharmaceuticals Inc Compounds and methods for modulating smn2
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
AU2022271873A1 (en) 2021-05-10 2024-01-04 Entrada Therapeutics, Inc. Compositions and methods for intracellular therapeutics
US20250235554A1 (en) 2021-10-25 2025-07-24 Novartis Ag Methods for improving adeno-associated virus (aav) delivery
FR3128873B1 (fr) 2021-11-10 2026-01-16 Biophytis Phytoecdysones et/ou dérivés de 20-hydroxyecdysone en combinaison avec un principe actif visant à restaurer l’expression SMN pour leur utilisation dans le traitement de l’amyotrophie spinale
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5176996A (en) * 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
FR2645345A1 (fr) * 1989-03-31 1990-10-05 Thomson Csf Procede de modulation dirigee de la composition ou du dopage de semi-conducteurs, notamment pour la realisation de composants electroniques monolithiques de type planar, utilisation et produits correspondants
US5256775A (en) * 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US6582908B2 (en) * 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
FR2692265B1 (fr) 1992-05-25 1996-11-08 Centre Nat Rech Scient Composes biologiquement actifs de type phosphotriesters.
EP1897942A1 (en) 1993-05-11 2008-03-12 The University Of North Carolina At Chapel Hill Antisense oligonucleotides which combat aberrant splicing and methods of using the same
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
PL184202B1 (pl) 1994-02-28 2002-09-30 Agritech Technologies Ltd Nieterapeutyczny sposób zwiększania zdolności produkcyjnej ptaka, fuzyjne białko heterologiczne obejmujące białko ptasiej inhibiny oraz sposób wytwarzania fuzyjnego białka heterologicznego, gen fuzyjny, komórka gospodarza i wektor
US7034009B2 (en) * 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US20030228597A1 (en) * 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
DE19906484A1 (de) * 1999-02-17 2000-09-07 Wegerer Kristina Elektrische Maschine
NZ514348A (en) * 1999-05-04 2004-05-28 Exiqon As L-ribo-LNA analogues
US6649411B2 (en) 1999-07-30 2003-11-18 The United States Of America As Represented By The Department Of Health And Human Services Methods of inhibiting cancer cells with ADNF III antisense oligonucleotides
US6770633B1 (en) 1999-10-26 2004-08-03 Immusol, Inc. Ribozyme therapy for the treatment of proliferative skin and eye diseases
US6962906B2 (en) * 2000-03-14 2005-11-08 Active Motif Oligonucleotide analogues, methods of synthesis and methods of use
WO2002038738A2 (en) 2000-11-09 2002-05-16 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
US6376508B1 (en) * 2000-12-13 2002-04-23 Academia Sinica Treatments for spinal muscular atrophy
GB0219143D0 (en) * 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
JP2006507841A (ja) * 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
WO2004113867A2 (en) 2003-06-16 2004-12-29 University Of Massachusetts Exon analysis
GB0425625D0 (en) * 2004-11-22 2004-12-22 Royal College Of Surgeons Ie Treatment of disease
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
ES2397113T3 (es) 2005-06-23 2013-03-04 Isis Pharmaceuticals, Inc. Composiciones y procedimientos para modular el corte y empalme de SMN2
US20120021515A1 (en) 2009-02-06 2012-01-26 Swayze Eric E Oligomeric compounds and methods
US20120149757A1 (en) * 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
CN115227709A (zh) * 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法

Also Published As

Publication number Publication date
EP3470072A1 (en) 2019-04-17
HK1247103B (en) 2019-09-06
EP1910395A2 (en) 2008-04-16
SI2548560T1 (sl) 2015-12-31
PT3308788T (pt) 2019-01-30
US8946183B2 (en) 2015-02-03
PL3308788T3 (pl) 2019-05-31
LTPA2017037I1 (lt) 2017-11-27
HK1181307A1 (en) 2013-11-08
LUC00046I2 (da) 2018-01-10
WO2007002390A3 (en) 2007-11-15
PT1910395E (pt) 2012-12-03
PL2548560T3 (pl) 2015-11-30
SI3308788T1 (sl) 2019-01-31
US20130109091A1 (en) 2013-05-02
CY1121134T1 (el) 2019-12-11
LT3308788T (lt) 2018-12-10
US20170088835A1 (en) 2017-03-30
EP3308788A1 (en) 2018-04-18
US20100216238A1 (en) 2010-08-26
EP2548560A1 (en) 2013-01-23
US20180291376A1 (en) 2018-10-11
HUS1700042I1 (hu) 2017-12-28
US20150353929A1 (en) 2015-12-10
US8361977B2 (en) 2013-01-29
HUE027486T2 (en) 2016-09-28
PL1910395T3 (pl) 2013-03-29
LTC2548560I2 (lt) 2018-11-12
DK3308788T3 (da) 2019-01-02
WO2007002390A2 (en) 2007-01-04
CY2017040I1 (el) 2018-04-04
LUC00046I1 (da) 2017-11-10
ES2545223T3 (es) 2015-09-09
DK2548560T3 (da) 2015-07-13
ES2397113T3 (es) 2013-03-04
EP2548560B1 (en) 2015-06-03
EP1910395A4 (en) 2010-09-22
PT2548560E (pt) 2015-09-22
EP3308788B1 (en) 2018-10-24
EP2644700A1 (en) 2013-10-02
EP1910395B1 (en) 2012-10-17
US20200181613A1 (en) 2020-06-11
CY2017040I2 (el) 2018-04-04
ES2702531T3 (es) 2019-03-01

Similar Documents

Publication Publication Date Title
EP1910395A4 (en) COMPOSITIONS AND METHOD FOR MODULATING SMN2 SPLICING
DK2178851T3 (da) Forbindelser og fremgangsmåder til modulation af FXR
NO20200670A1 (no) Sammensetninger og fremgangsmåter for modulering av hemostase
DK1677735T3 (da) Fremgangsmåder og sammensætninger til modulering af adipocytfunktion
DK1879573T3 (da) Modulatorer af indolamin 2,3-dioxygenase og fremgangsmåder til anvendelse deraf
DK2041071T3 (da) Biarylsammensætninger og fremgangsmåder til modulering af en kinasekaskade
DK2203478T3 (da) Sammensætninger og fremgangsmåder til anvendelse af antistoffer mod sclerostin
DK2087002T3 (da) Sammensætninger og fremgangsmåder til binding af sphingosin-1-phosphat
DK2460820T3 (da) PCV2-immunogene sammensætninger og fremgangsmåder til fremstilling af sådanne sammensætninger
DK2929891T3 (da) Fremgangsmåde til modulering af appetitten
DK1928454T3 (da) Pyridonderivater til modulering af stress-aktiveret proteinkinasesystem
DK2102398T3 (da) Fremgangsmåde til fremstilling af en ankelsok og ankelsok opnået derved
DK2135603T3 (da) Sammensætninger og fremgangsmåder til øgning af insulinsensitivitet
DK2319448T3 (da) Laserindretning til fjernelse af hår
DK3290513T3 (da) Sammensætninger og fremgangsmåder til forhindring af aav-vektoraggregering
DK1915414T3 (da) Polythioetherpolymerer og hærdedygtige sammensætninger indeholdende disse
DK2066339T3 (da) Sammensætninger og fremgangsmåder til forstærkning af immunreaktioner
DK1858864T3 (da) Stabiliserede sammensætninger af flygtige alkyleringsmidler og fremgangsmåder til anvendelse deraf
DE602007011741D1 (de) Optisches modulationselement
ATE540027T1 (de) Tetrahydroindazol-cannabinoidmodulatoren
EP1633311A4 (en) COMPOSITIONS AND METHOD FOR MODULATING S-NITROSOGLUTATHION REDUCTASE
DK1852430T3 (da) Fremgangsmåde til fremstilling af proanthocyanidinoligomer
DK3417905T3 (da) Applikator til indføring af et implantat
DK2320739T3 (da) Farmaceutiske sammensætninger og fremgangsmåder til stabilisering af disse
DE602006001461D1 (de) Optischer parametrischer Verstärker