HRP20181712T1 - Pripravci i postupci za modulaciju izrezivanja smn2 kod subjekta - Google Patents

Pripravci i postupci za modulaciju izrezivanja smn2 kod subjekta

Info

Publication number
HRP20181712T1
HRP20181712T1 HRP20181712TT HRP20181712T HRP20181712T1 HR P20181712 T1 HRP20181712 T1 HR P20181712T1 HR P20181712T T HRP20181712T T HR P20181712TT HR P20181712 T HRP20181712 T HR P20181712T HR P20181712 T1 HRP20181712 T1 HR P20181712T1
Authority
HR
Croatia
Prior art keywords
smn2
preparations
procedures
subject
cutting modulation
Prior art date
Application number
HRP20181712TT
Other languages
English (en)
Croatian (hr)
Inventor
C. Frank Bennett
Gene Hung
Frank Rigo
Adrian R. Krainer
Yimin Hua
Marco A. Passini
Lamya Shihabuddin
Seng H. Cheng
Katherine W. Klinger
Original Assignee
Biogen Ma Inc.
Cold Spring Harbor Laboratory
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=43356760&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HRP20181712(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Biogen Ma Inc., Cold Spring Harbor Laboratory filed Critical Biogen Ma Inc.
Publication of HRP20181712T1 publication Critical patent/HRP20181712T1/hr

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • Pulmonology (AREA)
  • Pain & Pain Management (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
HRP20181712TT 2009-06-17 2018-10-19 Pripravci i postupci za modulaciju izrezivanja smn2 kod subjekta HRP20181712T1 (hr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US21803109P 2009-06-17 2009-06-17
EP17203913.3A EP3305302B1 (en) 2009-06-17 2010-06-17 Compositions and methods for modulation of smn2 splicing in a subject

Publications (1)

Publication Number Publication Date
HRP20181712T1 true HRP20181712T1 (hr) 2018-12-28

Family

ID=43356760

Family Applications (2)

Application Number Title Priority Date Filing Date
HRP20181712TT HRP20181712T1 (hr) 2009-06-17 2018-10-19 Pripravci i postupci za modulaciju izrezivanja smn2 kod subjekta
HRP20191978TT HRP20191978T1 (hr) 2009-06-17 2019-10-31 Pripravci i postupci za modulaciju smn2 srastanja u subjektu

Family Applications After (1)

Application Number Title Priority Date Filing Date
HRP20191978TT HRP20191978T1 (hr) 2009-06-17 2019-10-31 Pripravci i postupci za modulaciju smn2 srastanja u subjektu

Country Status (25)

Country Link
US (5) US8980853B2 (enExample)
EP (4) EP3449926B1 (enExample)
JP (8) JP5707396B2 (enExample)
KR (9) KR101965868B1 (enExample)
CN (5) CN116440150A (enExample)
AU (2) AU2010262862C1 (enExample)
CA (1) CA2765396C (enExample)
CY (2) CY1120807T1 (enExample)
DK (2) DK3305302T3 (enExample)
ES (2) ES2761614T3 (enExample)
HK (1) HK1246648B (enExample)
HR (2) HRP20181712T1 (enExample)
HU (2) HUE039741T2 (enExample)
IL (2) IL217014A (enExample)
LT (2) LT3305302T (enExample)
MX (3) MX2011014004A (enExample)
NO (1) NO2020008I1 (enExample)
NZ (2) NZ624712A (enExample)
PL (2) PL3305302T3 (enExample)
PT (2) PT3449926T (enExample)
RU (2) RU2566724C9 (enExample)
SI (2) SI3449926T1 (enExample)
SM (2) SMT202000020T1 (enExample)
TR (1) TR201816256T4 (enExample)
WO (1) WO2010148249A1 (enExample)

Families Citing this family (118)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
US8361977B2 (en) * 2005-06-23 2013-01-29 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of SMN2 splicing
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
PT3449926T (pt) 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
JP2013530154A (ja) 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, 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
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
WO2012150960A1 (en) 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
US9833439B2 (en) 2011-05-25 2017-12-05 Universite Paris Descartes ERK inhibitors for use in treating spinal muscular atrophy
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
US20140343127A1 (en) 2011-11-11 2014-11-20 Santaris Pharma A/S a corporation Compounds for the modulation of smn2 splicing
BR112014011875B1 (pt) 2011-11-18 2022-01-04 Sarepta Therapeutics, Inc Oligonucleotídeos funcionalmente modificados e subunidades dos mesmos
AU2012345638C1 (en) 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
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
US10273474B2 (en) 2012-03-30 2019-04-30 Washington University Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
KR20240148947A (ko) 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
WO2014110291A1 (en) * 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
US9856474B2 (en) 2013-01-16 2018-01-02 Iowa State University Research Foundation, Inc. Deep intronic target for splicing correction on spinal muscular atrophy gene
RU2745324C2 (ru) 2013-03-14 2021-03-23 Ионис Фармасьютикалз, Инк. Композиции и способы модулирования экспрессии tau
EP3560502B1 (en) * 2013-04-12 2025-09-24 The Curators of the University of Missouri Smn2 element 1 antisense compositions and methods and uses thereof
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
WO2015013673A1 (en) 2013-07-25 2015-01-29 Aurasense Therapeutics, Llc Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
US10568898B2 (en) 2013-08-13 2020-02-25 Northwestern University Lipophilic nanoparticles for drug delivery
JP2016531570A (ja) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド
DK3041958T3 (da) 2013-09-04 2020-03-09 Cold Spring Harbor Laboratory Reducering af nonsense-medieret mrna-degradering
IL282239B2 (en) 2013-09-05 2023-10-01 Sarepta Therapeutics Inc Antisense-induced exon2 inclusion in acid alpha-glucosidase
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
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
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
CN104163871A (zh) * 2014-08-12 2014-11-26 大连医科大学 抗肿瘤的特异性剪接因子及制备方法
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10517924B2 (en) 2014-11-24 2019-12-31 Northwestern University High density lipoprotein nanoparticles for inflammation
EP3271460A4 (en) 2015-03-17 2019-03-13 The General Hospital Corporation RNA INTERACTOM 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
WO2016164896A2 (en) 2015-04-10 2016-10-13 Ionis Pharmaceuticals, Inc. Modulation of smn expression
WO2016196670A1 (en) 2015-06-01 2016-12-08 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type vii collagen
WO2016196897A1 (en) 2015-06-04 2016-12-08 Sarepta Therapeutics, Inc. Methods and compounds for treatment of lymphocyte-related diseases and conditions
MA42695A (fr) * 2015-08-28 2018-07-04 Sarepta Therapeutics Inc Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
WO2017062835A2 (en) 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
CA3005434C (en) 2015-11-16 2024-09-10 Ohio State Innovation Foundation METHODS AND COMPOSITIONS FOR TREATING DISORDERS AND DISEASES USING MOTOR NERVE SURVIVAL PROTEIN (MNS)
JP2019500345A (ja) 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー 肝臓病の処置のための組成物および方法
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
ES2903394T3 (es) 2015-12-14 2022-04-01 Cold Spring Harbor Laboratory Composiciones para el tratamiento de la retinitis pigmentosa 13
CA3005246A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
CA3023451A1 (en) 2016-05-06 2017-11-09 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs presenting antisense oligonucleotides (aso) for specific knockdown of interleukin 17 receptor mrna
BR112018075667B1 (pt) 2016-06-14 2024-02-20 Biogen Ma, Inc Métodos para separar um oligonucleotídeo alvo de uma mistura contendo o oligonucleotídeo alvo e uma impureza relacionada com o produto
US11198867B2 (en) 2016-06-16 2021-12-14 Ionis Pharmaceuticals, Inc. Combinations for the modulation of SMN expression
ES2999138T3 (en) 2016-06-24 2025-02-24 Biogen Ma Inc Synthesis of thiolated oligonucleotides without a capping step
WO2018039629A2 (en) 2016-08-25 2018-03-01 Northwestern University Micellar spherical nucleic acids from thermoresponsive, traceless templates
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
WO2018102397A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SI3589730T1 (sl) 2017-02-28 2024-04-30 The Trustees Of The University Of Pennsylvania Adeno-povezan virus (AAV) clade F vektor in njihova uporaba
CN110536964A (zh) 2017-03-10 2019-12-03 国立研究开发法人国立成育医疗研究中心 反义寡核苷酸和糖原贮积病Ia型预防或治疗用组合物
ES2994451T3 (en) 2017-04-20 2025-01-24 Atyr Pharma Inc Compositions for treating lung inflammation
WO2018201090A1 (en) 2017-04-28 2018-11-01 Exicure, Inc. Synthesis of spherical nucleic acids using lipophilic moieties
WO2018235829A1 (ja) 2017-06-19 2018-12-27 住友金属鉱山株式会社 近赤外線硬化型インク組成物とその製造方法、近赤外線硬化膜、および光造形法
JP7199410B2 (ja) 2017-07-10 2023-01-05 ジェロン・コーポレーション 改善されたイメテルスタットの調製方法
RU2020105929A (ru) 2017-08-04 2021-09-06 Скайхоук Терапьютикс, Инк. Способы и композиции для модулирования сплайсинга
EP3673080B1 (en) 2017-08-25 2023-10-18 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
WO2019075357A1 (en) * 2017-10-12 2019-04-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
EP3697910A4 (en) 2017-10-18 2021-07-14 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMER COMPOUNDS
RS66633B1 (sr) 2017-10-23 2025-08-29 Stoke Therapeutics Inc Antisens oligomeri za lečenje bolesti i stanja na bazi besmisleno posredovanog raspadanja rnk
IL274430B2 (en) 2017-11-08 2025-01-01 Novartis Gene Therapies Inc Means and method for preparing viral vectors and their uses
US11667941B2 (en) * 2018-01-12 2023-06-06 Camena Bioscience Limited Compositions and methods for template-free geometric enzymatic nucleic acid synthesis
MX2020007433A (es) 2018-01-12 2020-09-14 Bristol Myers Squibb Co Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos.
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
AU2019211446B2 (en) 2018-01-25 2025-07-03 Biogen Ma Inc. Methods of treating spinal muscular atrophy
WO2019168558A1 (en) * 2018-02-28 2019-09-06 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors
US20210002640A1 (en) * 2018-02-28 2021-01-07 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors
CN111511915A (zh) 2018-03-09 2020-08-07 第一三共株式会社 糖原病Ia型治疗药
JP7333079B2 (ja) 2018-03-19 2023-08-24 国立大学法人 東京医科歯科大学 毒性が軽減した核酸
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
WO2019236949A1 (en) 2018-06-08 2019-12-12 Avexis Inc. Cell-based assay for measuring drug product potency
CA3105174A1 (en) 2018-06-27 2020-01-02 Reborna Biosciences, Inc. Prophylactic or therapeutic agent for spinal muscular atrophy
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
KR20210111242A (ko) 2018-10-29 2021-09-10 바이오젠 엠에이 인코포레이티드 혈액 뇌 장벽 수송을 향상시키기 위한 인간화 및 안정화된 fc5 변이체
EP3886919A1 (en) 2018-11-30 2021-10-06 Novartis AG Aav viral vectors and uses thereof
MA54383A (fr) 2018-12-06 2021-10-13 Biogen Ma Inc Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique
EP3918069A1 (en) 2019-01-31 2021-12-08 Bar Ilan University Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy
JP7603592B2 (ja) 2019-02-05 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
EP3920926A4 (en) 2019-02-05 2022-10-19 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN119638696A (zh) 2019-02-06 2025-03-18 斯基霍克疗法公司 用于调节剪接的方法和组合物
CN113748209A (zh) 2019-02-27 2021-12-03 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
EP3942049A4 (en) 2019-03-20 2023-10-18 President And Fellows Of Harvard College ANTISENSE OLIGONUCLEOTIDE BASED PROGRANULIN AUGMENTATION THERAPY FOR NEURODEGENERATIVE DISEASES
US12458657B2 (en) 2019-04-18 2025-11-04 Brown University Neurogenesis
AU2020268415A1 (en) 2019-05-08 2021-12-23 Biogen Ma Inc. Convergent liquid phase syntheses of oligonucleotides
RS64879B1 (sr) * 2019-07-19 2023-12-29 Biogen Ma Inc Postupci za lečenje ili prevenciju spinalne mišićne atrofije
US20220280548A1 (en) * 2019-08-15 2022-09-08 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
EP4054618A1 (en) * 2019-11-06 2022-09-14 Association Institut de Myologie Combined therapy for muscular diseases
RU2738093C9 (ru) * 2019-12-23 2020-12-29 Общество с ограниченной ответственностью "Гелеспон" Способ синтеза 2'-о-(2-метоксиэтил) тиофосфатного олигонуклеотида
CN120505310A (zh) * 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
CN113444722B (zh) * 2020-03-24 2024-09-24 中国科学院脑科学与智能技术卓越创新中心 单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
AU2021318560A1 (en) * 2020-07-31 2023-03-30 Ractigen Therapeutics Combinatory treatment of SMA with saRNA and mRNA modulators
AR125239A1 (es) 2021-04-02 2023-06-28 Kumiai Chemical Industry Co Compuesto heterocíclico y uso del mismo
US20250235554A1 (en) 2021-10-25 2025-07-24 Novartis Ag Methods for improving adeno-associated virus (aav) delivery
KR20240123838A (ko) 2021-12-20 2024-08-14 프라이 유니베르시탯 베를린 Rbm3 발현을 증가시키는 방법 및 제제
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
US20240150759A1 (en) * 2022-11-04 2024-05-09 The Board Of Trustees Of The Leland Stanford Junior University Splicing Modulators for the Treatment of Timothy Syndrome
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 (125)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
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
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
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
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
EP0497875B1 (en) 1989-10-24 2000-03-22 Isis Pharmaceuticals, Inc. 2' modified oligonucleotides
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
WO1991013080A1 (en) 1990-02-20 1991-09-05 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
DE69032425T2 (de) 1990-05-11 1998-11-26 Microprobe Corp., Bothell, Wash. Teststreifen zum Eintauchen für Nukleinsäure-Hybridisierungsassays und Verfahren zur kovalenten Immobilisierung von Oligonucleotiden
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
CA2095212A1 (en) 1990-11-08 1992-05-09 Hybridon, Inc. Incorporation of multiple reporter groups on synthetic oligonucleotides
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
AU6449394A (en) 1993-03-30 1994-10-24 Sterling Winthrop Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
DE69435005T2 (de) 1993-05-11 2008-04-17 The University Of North Carolina At Chapel Hill Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
FI963325L (fi) 1994-02-28 1996-10-21 Agritech Technologies Ltd Inhibiinikoostumukset ja menetelmät niiden käyttämiseksi
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
EP0708178A1 (en) * 1994-10-19 1996-04-24 Institut National De La Sante Et De La Recherche Medicale (Inserm) Survival motor neuron (SMN) gene: a gene for spinal muscular atrophy
US5792747A (en) 1995-01-24 1998-08-11 The Administrators Of The Tulane Educational Fund Highly potent agonists of growth hormone releasing hormone
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)
AU1430097A (en) 1996-01-16 1997-08-11 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
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
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
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
US20050020525A1 (en) 2002-02-20 2005-01-27 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US6962906B2 (en) 2000-03-14 2005-11-08 Active Motif Oligonucleotide analogues, methods of synthesis and methods of use
AU2001259706A1 (en) 2000-05-09 2001-11-20 Reliable Biopharmaceutical, Inc. Polymeric compounds useful as prodrugs
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
CA2452458A1 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
GB0219143D0 (en) 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
EP1562971B1 (en) 2002-11-05 2014-02-12 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8090542B2 (en) 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
JP2004344072A (ja) * 2003-05-22 2004-12-09 Japan Science & Technology Agency Ｓｍｎ遺伝子のスプライシング調節エレメント
WO2004113867A2 (en) 2003-06-16 2004-12-29 University Of Massachusetts Exon analysis
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
AU2004274021B2 (en) 2003-09-18 2009-08-13 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
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
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
US8361977B2 (en) * 2005-06-23 2013-01-29 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of SMN2 splicing
JP4223027B2 (ja) 2005-06-30 2009-02-12 シャープ株式会社 画像形成装置及び秘匿データ送信方法
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
US20070105807A1 (en) 2005-11-10 2007-05-10 Sazani Peter L Splice switch oligomers for TNF superfamily receptors and their use in treatment of disease
JP5425474B2 (ja) * 2006-01-26 2014-02-26 アイシス ファーマシューティカルズ， インコーポレーテッド ハンチンチン対する、組成物及びその使用
JP5213723B2 (ja) 2006-01-27 2013-06-19 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 マイクロｒｎａの調節に使用するためのオリゴマー化合物及び組成物
CA2640171C (en) 2006-01-27 2014-10-28 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
JP5441688B2 (ja) 2006-05-11 2014-03-12 アイシス ファーマシューティカルズ， インコーポレーテッド ５’修飾二環式核酸類似体
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
CN101796062B (zh) 2007-07-05 2014-07-30 Isis制药公司 6-双取代双环核酸类似物
WO2009120700A2 (en) 2008-03-26 2009-10-01 Families Of Spinal Muscular Atrophy Inhibition of dcps
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US9156037B2 (en) 2009-01-15 2015-10-13 Children's Medical Center Corporation Microfluidic device and uses thereof
US20120021515A1 (en) 2009-02-06 2012-01-26 Swayze Eric E Oligomeric compounds and methods
WO2010120820A1 (en) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
PT3449926T (pt) 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
WO2011032109A1 (en) 2009-09-11 2011-03-17 Sma Foundation Biomarkers for spinal muscular atrophy
US8183002B2 (en) 2009-12-03 2012-05-22 Abbott Laboratories Autoantibody enhanced immunoassays and kits
US10653713B2 (en) 2010-10-06 2020-05-19 Medtronic, Inc. Methods for distributing agents to areas of brain
US20130052670A1 (en) 2011-07-13 2013-02-28 Merck Method for detection of amyloid beta oligomers in a fluid sample and uses thereof
WO2014110291A1 (en) 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
US20140367278A1 (en) 2013-06-12 2014-12-18 PharmOptima, LLC Methods for Detecting Survival Motor Neuron (SMN) Protein in Whole Blood or Cerebral Spinal Fluid

Also Published As

Publication number Publication date
EP3449926B1 (en) 2019-10-09
IL245096B (en) 2018-04-30
NO2020008I1 (no) 2020-04-01
MX361732B (es) 2018-12-14
EP2442816A4 (en) 2013-10-02
CN102665731A (zh) 2012-09-12
ES2761614T3 (es) 2020-05-20
IL245096A0 (en) 2016-06-30
CY1120807T1 (el) 2019-12-11
RU2566724C9 (ru) 2019-03-12
RU2012101491A (ru) 2013-07-27
PT3305302T (pt) 2018-12-14
SMT202000020T1 (it) 2020-03-13
HRP20191978T1 (hr) 2020-05-15
KR20170054537A (ko) 2017-05-17
TR201816256T4 (tr) 2018-11-21
KR20240036132A (ko) 2024-03-19
KR20190017063A (ko) 2019-02-19
JP2024012416A (ja) 2024-01-30
JP5707396B2 (ja) 2015-04-30
US20170015995A1 (en) 2017-01-19
KR20190037378A (ko) 2019-04-05
DK3305302T3 (en) 2018-11-19
US20190030058A1 (en) 2019-01-31
LT3305302T (lt) 2018-12-10
IL217014A0 (en) 2012-02-29
JP6637121B2 (ja) 2020-01-29
EP3643783A1 (en) 2020-04-29
US20200376018A1 (en) 2020-12-03
JP2015131828A (ja) 2015-07-23
AU2010262862A1 (en) 2012-01-19
NZ597071A (en) 2014-05-30
EP3305302B1 (en) 2018-09-19
JP2012530715A (ja) 2012-12-06
MX2018015635A (es) 2020-11-12
KR101965868B1 (ko) 2019-04-05
AU2010262862C1 (en) 2020-04-30
SI3305302T1 (sl) 2018-12-31
US20120190728A1 (en) 2012-07-26
RU2566724C2 (ru) 2015-10-27
JP2026004402A (ja) 2026-01-14
KR20200047790A (ko) 2020-05-07
CN115227709A (zh) 2022-10-25
CY1122687T1 (el) 2021-03-12
RU2683772C2 (ru) 2019-04-02
NZ624712A (en) 2015-10-30
WO2010148249A1 (en) 2010-12-23
CA2765396C (en) 2019-08-20
ES2699827T3 (es) 2019-02-13
AU2016200344B2 (en) 2017-12-21
SMT201800587T1 (it) 2019-01-11
MX2011014004A (es) 2012-06-19
KR20120093138A (ko) 2012-08-22
CN116440150A (zh) 2023-07-18
JP2017061497A (ja) 2017-03-30
US20240238326A1 (en) 2024-07-18
DK3449926T3 (da) 2019-11-11
CN115227710A (zh) 2022-10-25
HUE039741T2 (hu) 2019-01-28
PT3449926T (pt) 2019-11-12
JP6064139B2 (ja) 2017-01-25
LT3449926T (lt) 2020-01-27
JP2020073498A (ja) 2020-05-14
RU2015139553A3 (enExample) 2018-12-25
JP6370860B2 (ja) 2018-08-08
IL217014A (en) 2016-04-21
SI3449926T1 (sl) 2020-04-30
CN106983768A (zh) 2017-07-28
AU2016200344A1 (en) 2016-02-11
US9717750B2 (en) 2017-08-01
EP2442816A1 (en) 2012-04-25
RU2015139553A (ru) 2018-12-25
PL3305302T3 (pl) 2019-02-28
AU2010262862B2 (en) 2015-10-29
KR20260021813A (ko) 2026-02-13
JP2022025131A (ja) 2022-02-09
EP3449926A1 (en) 2019-03-06
HUE046966T2 (hu) 2020-04-28
CA2765396A1 (en) 2010-12-23
CN106983768B (zh) 2023-04-07
EP3305302A1 (en) 2018-04-11
US8980853B2 (en) 2015-03-17
KR20220084437A (ko) 2022-06-21
JP2018184423A (ja) 2018-11-22
PL3449926T3 (pl) 2020-06-01
HK1246648B (en) 2019-09-06
KR20210057223A (ko) 2021-05-20

Similar Documents

Publication Publication Date Title
HRP20181712T1 (hr) Pripravci i postupci za modulaciju izrezivanja smn2 kod subjekta
HRP20180497T1 (hr) Antimikrobni pripravci i odgovarajući postupci njihove upotrebe
BR302012001342S1 (pt) Configuração aplicada em instrumento médico
BR112015002090A2 (pt) placa de laser e amplificador e método de uso
BR112012031446A2 (pt) componentes e métodos para implantes
BRDI7104784S (pt) Configuração aplicada em instrumento médico
BRDI7106721S (pt) Configuração aplicada em instrumento médico
BRDI7104782S (pt) Configuração aplicada em instrumento médico
BRDI7103743S (pt) Configuração aplicada em instrumento médico
IL214782A (en) Cutting insert and milling tool
BR112012031492A2 (pt) campo cirúrgico intervencional,e, kit para procedimentos intervencionais
BR112012010242A2 (pt) derivados de heteroarilpiperidina e heteroarilpiperazina
BR302012001336S1 (pt) Configuração aplicada em instrumento médico
HRP20160879T1 (hr) Pripravci i postupci za moduliranje kinaze
BR302012001341S1 (pt) Configuração aplicada em instrumento médico
BRDI7106720S (pt) Configuração aplicada em instrumento médico
EP2590207A4 (en) Dicing film
BRDI7104785S (pt) Configuração aplicada em instrumento médico
HRP20190060T1 (hr) Postupci i sastavi za sprečavanje pogrešne ugradnje norleukina u proteine
BR302012003127S1 (pt) Configuração aplicada em instrumento médico
BRDI7104906S (pt) Configuração aplicada em instrumento médico
FI20106220A0 (fi) Menetelmä ja järjestely rullainkäytön yhteydessä
BRDI7106698S (pt) Configuração aplicada em instrumento médico
BR302012001339S1 (pt) Configuração aplicada em instrumento médico
BRDI7102651S (pt) Configuração aplicada em instrumento médico