PT3449926T - Composições e métodos de modulação de excisões de smn2 em um sujeito - Google Patents

Composições e métodos de modulação de excisões de smn2 em um sujeito

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Publication number
PT3449926T
PT3449926T PT181858200T PT18185820T PT3449926T PT 3449926 T PT3449926 T PT 3449926T PT 181858200 T PT181858200 T PT 181858200T PT 18185820 T PT18185820 T PT 18185820T PT 3449926 T PT3449926 T PT 3449926T
Authority
PT
Portugal
Prior art keywords
modulation
compositions
subject
methods
smn2 splicing
Prior art date
Application number
PT181858200T
Other languages
English (en)
Portuguese (pt)
Inventor
H Cheng Seng
Frank Bennett C
Rigo Frank
R Krainer Adrian
Hua Yimin
A Passini Marco
Shihabuddin Lamya
W Klinger Katherine
Hung Gene
Original Assignee
Cold Spring Harbor Laboratory
Biogen Ma Inc
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=PT3449926(T) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Cold Spring Harbor Laboratory, Biogen Ma Inc filed Critical Cold Spring Harbor Laboratory
Publication of PT3449926T publication Critical patent/PT3449926T/pt

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    • 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
    • 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
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—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
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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

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  • 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
  • Pulmonology (AREA)
  • Psychiatry (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)
PT181858200T 2009-06-17 2010-06-17 Composições e métodos de modulação de excisões de smn2 em um sujeito PT3449926T (pt)

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US21803109P 2009-06-17 2009-06-17

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PT3449926T true PT3449926T (pt) 2019-11-12

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PT181858200T PT3449926T (pt) 2009-06-17 2010-06-17 Composições e métodos de modulação de excisões de smn2 em um sujeito

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US (5) US8980853B2 (enExample)
EP (4) EP2442816A4 (enExample)
JP (7) JP5707396B2 (enExample)
KR (8) KR20170054537A (enExample)
CN (5) CN115227709A (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) HUE046966T2 (enExample)
IL (2) IL217014A (enExample)
LT (2) LT3305302T (enExample)
MX (3) MX2011014004A (enExample)
NO (1) NO2020008I1 (enExample)
NZ (2) NZ597071A (enExample)
PL (2) PL3449926T3 (enExample)
PT (2) PT3305302T (enExample)
RU (2) RU2683772C2 (enExample)
SI (2) SI3449926T1 (enExample)
SM (2) SMT201800587T1 (enExample)
TR (1) TR201816256T4 (enExample)
WO (1) WO2010148249A1 (enExample)

Families Citing this family (114)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
PT3308788T (pt) 2005-06-23 2019-01-30 Cold Spring Harbor Laboratory Composições e métodos para a modulação do splicing de smn2
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
AU2009323766B2 (en) 2008-12-02 2016-10-06 Wave Life Sciences Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
AU2010262862C1 (en) 2009-06-17 2020-04-30 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject
SG177564A1 (en) 2009-07-06 2012-02-28 Ontorii Inc Novel nucleic acid prodrugs and methods 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
KR101829644B1 (ko) 2010-05-28 2018-02-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
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
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
DK2638163T3 (en) 2010-11-12 2017-07-24 Massachusetts Gen Hospital POLYCOMB-ASSOCIATED NON-CODING RNAs
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
CN103619356B (zh) 2011-05-05 2017-09-12 萨勒普塔医疗公司 肽寡核苷酸缀合物
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
BR112014011018A2 (pt) 2011-11-11 2017-05-02 Santaris Pharma As compostos para modulação de splining de smn2
KR20140097398A (ko) 2011-11-18 2014-08-06 사렙타 쎄러퓨틱스, 인코퍼레이티드 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트
US9944926B2 (en) 2011-11-30 2018-04-17 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
CA2858576A1 (en) * 2011-12-06 2013-06-13 Research Institute At Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
EP2831232A4 (en) 2012-03-30 2015-11-04 Univ Washington METHODS OF MODULATING THE EXPRESSION OF TAU TO REDUCE THE STROKE AND MODIFY A NEURODEGENERATIVE SYMPTOM
JP6268157B2 (ja) 2012-07-13 2018-01-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
SG10201912895PA (en) 2012-07-13 2020-02-27 Wave Life Sciences Ltd Chiral control
EP2943225A4 (en) 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
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
KR20150130430A (ko) 2013-03-14 2015-11-23 아이시스 파마수티컬즈 인코포레이티드 타우 발현을 조절하는 조성물 및 방법
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
AU2014292926B2 (en) 2013-07-25 2020-03-05 Exicure Operating Company Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
WO2015023797A1 (en) 2013-08-13 2015-02-19 Northwestern University Lipophilic nanoparticles for drug delivery
WO2015023941A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Oligonucleotides targeting euchromatin regions of genes
PL3041958T3 (pl) 2013-09-04 2020-11-02 Cold Spring Harbor Laboratory Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi
WO2015035231A1 (en) 2013-09-05 2015-03-12 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 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
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 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
PT3094728T (pt) 2014-01-16 2022-05-19 Wave Life Sciences Ltd Desenho quiral
WO2015161170A2 (en) 2014-04-17 2015-10-22 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
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
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
KR102620328B1 (ko) 2014-10-03 2024-01-02 콜드스프링하버러보러토리 핵 유전자 산출량의 표적화 증강
CA2966044A1 (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
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation 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
MA42695A (fr) 2015-08-28 2018-07-04 Sarepta Therapeutics Inc Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
EP3377073A4 (en) 2015-11-16 2019-06-26 Ohio State Innovation Foundation METHOD AND COMPOSITIONS FOR TREATING DISORDERS AND DISEASES WITH THE SURVIVAL MOTOR NEURON (SMN) PROTEIN
CA3005090A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of liver diseases
ES2903394T3 (es) 2015-12-14 2022-04-01 Cold Spring Harbor Laboratory Composiciones para el tratamiento de la retinitis pigmentosa 13
CN109312343B (zh) 2015-12-14 2022-09-27 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CA3005246A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases
EP3389671A4 (en) 2015-12-14 2019-07-17 Cold Spring Harbor Laboratory ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE SYNDROME
US11060089B2 (en) 2016-04-18 2021-07-13 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
AU2017261360A1 (en) 2016-05-06 2018-11-29 Exicure Operating Company Liposomal Spherical Nucleic Acid (SNA) constructs presenting Antisense Oligonucleotides (ASO) for specific knockdown of interleukin 17 receptor MRNA
CN109641928B (zh) 2016-06-14 2022-08-30 比奥根Ma公司 用于寡核苷酸纯化的疏水性相互作用色谱法
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
US11142545B2 (en) 2016-06-24 2021-10-12 Biogen Ma Inc. Synthesis of thiolated oligonucleotides without a capping step
US11364304B2 (en) 2016-08-25 2022-06-21 Northwestern University Crosslinked micellar spherical nucleic acids
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
JP2019535839A (ja) 2016-11-29 2019-12-12 ピュアテック ヘルス エルエルシー 治療剤の送達のためのエクソソーム
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CN118956781A (zh) 2017-02-28 2024-11-15 宾夕法尼亚州大学信托人 腺相关病毒(aav)进化枝f载体及其用途
EP3594346A4 (en) * 2017-03-10 2020-12-16 National Center For Child Health And Development ANTISENS OLIGONUCLEOTIDE AND COMPOSITION FOR THE PREVENTION OR TREATMENT OF TYPE IA GLYCOGEN STORAGE DISEASE
US11767520B2 (en) 2017-04-20 2023-09-26 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
US11696954B2 (en) 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
JP7247886B2 (ja) 2017-06-19 2023-03-29 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
ES2901099T3 (es) 2017-07-10 2022-03-21 Geron Corp Proceso mejorado para la preparación de imetelstat
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
SMT202400071T1 (it) 2017-08-25 2024-03-13 Stoke Therapeutics Inc Oligomeri antisenso per il trattamento di condizioni e malattie
SG11202001783YA (en) 2017-10-12 2020-03-30 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
DK3700570T3 (da) 2017-10-23 2025-03-31 Stoke Therapeutics Inc Antisense-oligomerer til behandling af ikke-sense medieret rna henfald baserede tilstande og sygdomme
BR112019013268A2 (pt) 2017-11-08 2019-12-17 Avexis Inc. meios e métodos para a preparação de vetores virais e usos dos mesmos
US11447775B2 (en) 2018-01-12 2022-09-20 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CN111819283A (zh) 2018-01-12 2020-10-23 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
WO2019140353A1 (en) * 2018-01-12 2019-07-18 Camena Bioscience Limited Compositions and methods for template-free geometric enzymatic nucleic acid synthesis
MX2020007852A (es) 2018-01-25 2021-01-29 Biogen Ma Inc Metodos para el tratamiento de la atrofia muscular espinal.
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
WO2019181946A1 (ja) 2018-03-19 2019-09-26 国立大学法人東京医科歯科大学 毒性が軽減した核酸
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
EP3801638A1 (en) 2018-06-08 2021-04-14 Novartis AG Cell-based assay for measuring drug product potency
JOP20200338A1 (ar) 2018-06-27 2020-12-24 Reborna Biosciences Inc عامل وقائي أو علاجي للضمور العضلي نخاعي المنشأ
JP2021534154A (ja) * 2018-08-15 2021-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. 脊髄性筋萎縮症のための併用療法
US20220089704A1 (en) 2018-10-29 2022-03-24 Biogen Ma Inc. Humanized and stabilized fc5 variants for enhancement of blood brain barrier transport
TWI844587B (zh) 2018-11-30 2024-06-11 瑞士商諾華公司 Aav病毒載體及其用途
KR20210102294A (ko) 2018-12-06 2021-08-19 바이오젠 엠에이 인코포레이티드 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질
WO2020157760A1 (en) 2019-01-31 2020-08-06 Bar Ilan University Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
JP7603595B2 (ja) 2019-02-06 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
WO2020191212A1 (en) 2019-03-20 2020-09-24 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
EP3955742A4 (en) 2019-04-18 2023-05-24 Brown University Neurogenesis
AU2020268415A1 (en) 2019-05-08 2021-12-23 Biogen Ma Inc. Convergent liquid phase syntheses of oligonucleotides
FI3999643T3 (fi) * 2019-07-19 2023-11-30 Biogen Ma Inc Menetelmiä spinaalisen lihasatrofian hoitamiseksi tai ehkäisemiseksi
JP7689110B2 (ja) * 2019-08-15 2025-06-05 バイオジェン・エムエイ・インコーポレイテッド 脊髄性筋萎縮症のための併用療法
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
AU2021225957A1 (en) * 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2
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
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
US20230287416A1 (en) * 2020-07-31 2023-09-14 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
CN118043039A (zh) 2021-10-25 2024-05-14 诺华股份有限公司 用于改善腺相关病毒（aav）递送的方法
JP2024547028A (ja) 2021-12-20 2024-12-26 フライエ ウニベルシテート ベルリン Ｒｂｍ３発現を増加する方法及び作用薬
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 (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
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
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
JPH05504552A (ja) 1989-10-24 1993-07-15 ギリアド　サイエンシズ，インコーポレイテッド ２’位が改変されたオリゴヌクレオチド
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
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
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
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
DK0455905T3 (da) 1990-05-11 1998-12-07 Microprobe Corp Dipsticks til nukleinsyrehybridiseringsassays og fremgangsmåde til kovalent immobilisering af oligonukleotider
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
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
ATE198598T1 (de) 1990-11-08 2001-01-15 Hybridon Inc Verbindung von mehrfachreportergruppen auf synthetischen oligonukleotiden
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
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
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
DK0691968T3 (da) 1993-03-30 1998-02-23 Sanofi Sa Acykliske nukleosid-analoge og oligonukleotidsekvenser indeholdende disse
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
EP0698092B1 (en) 1993-05-11 2007-07-25 The University Of North Carolina At Chapel Hill Antisense oligonucleotides which combat aberrant splicing and methods of using the same
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
FI963325A7 (fi) 1994-02-28 1996-10-21 Agritech Tech Ltd 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
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
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)
CA2253382A1 (en) 1996-01-16 1997-07-24 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
US7875733B2 (en) 2003-09-18 2011-01-25 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising 4′-thionucleosides for use in gene modulation
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
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
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
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
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
US20020013287A1 (en) 2000-05-09 2002-01-31 Reliable Biopharmaceuticals, Inc. St Louis Missouri Polymeric compounds useful as prodrugs
EP1416972B1 (en) 2000-11-09 2012-02-08 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
WO2003004602A2 (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
EP2314691A3 (en) 2002-11-14 2012-01-18 Dharmacon, Inc. Fuctional 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
US7427672B2 (en) 2003-08-28 2008-09-23 Takeshi Imanishi 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
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
PT3308788T (pt) 2005-06-23 2019-01-30 Cold Spring Harbor Laboratory Composições e métodos para a modulação do splicing de smn2
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
WO2007058894A2 (en) 2005-11-10 2007-05-24 The University Of North Carolina At Chapel Hill Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
PT2161038E (pt) * 2006-01-26 2014-03-10 Isis Pharmaceuticals Inc Composições e suas utilizações dirigidas à huntingtina
CN102908630B (zh) 2006-01-27 2014-11-19 Isis制药公司 6-修饰的双环核酸类似物
EP1984499B1 (en) 2006-01-27 2015-05-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
EP2066684B1 (en) 2006-05-11 2012-07-18 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
EP2125852B1 (en) 2007-02-15 2016-04-06 Ionis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
ES2388590T3 (es) 2007-05-30 2012-10-16 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos con puente aminometileno N-sustituido.
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
WO2009006478A2 (en) 2007-07-05 2009-01-08 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
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
WO2010091308A2 (en) * 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
WO2010120820A1 (en) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
AU2010262862C1 (en) 2009-06-17 2020-04-30 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject
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
MX2014000480A (es) 2011-07-13 2014-06-23 Merck Sharp & Dohme Metodo para la deteccion de oligomeros beta amiloide en una muestra de fluidos y usos de los mismos.
EP2943225A4 (en) 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
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

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SI3305302T1 (sl) 2018-12-31
KR20190017063A (ko) 2019-02-19
IL217014A0 (en) 2012-02-29
CN115227709A (zh) 2022-10-25
JP2018184423A (ja) 2018-11-22
RU2566724C9 (ru) 2019-03-12
MX361732B (es) 2018-12-14
EP3305302A1 (en) 2018-04-11
EP3643783A1 (en) 2020-04-29
LT3449926T (lt) 2020-01-27
KR101965868B1 (ko) 2019-04-05
CN106983768A (zh) 2017-07-28
MX2011014004A (es) 2012-06-19
US20170015995A1 (en) 2017-01-19
CY1120807T1 (el) 2019-12-11
EP2442816A4 (en) 2013-10-02
PL3449926T3 (pl) 2020-06-01
EP3305302B1 (en) 2018-09-19
JP6370860B2 (ja) 2018-08-08
WO2010148249A1 (en) 2010-12-23
JP5707396B2 (ja) 2015-04-30
IL217014A (en) 2016-04-21
AU2010262862A1 (en) 2012-01-19
NZ597071A (en) 2014-05-30
TR201816256T4 (tr) 2018-11-21
DK3305302T3 (en) 2018-11-19
ES2699827T3 (es) 2019-02-13
EP3449926A1 (en) 2019-03-06
US8980853B2 (en) 2015-03-17
KR20190037378A (ko) 2019-04-05
JP6637121B2 (ja) 2020-01-29
HRP20191978T1 (hr) 2020-05-15
SI3449926T1 (sl) 2020-04-30
AU2010262862B2 (en) 2015-10-29
JP2015131828A (ja) 2015-07-23
KR20220084437A (ko) 2022-06-21
RU2012101491A (ru) 2013-07-27
CA2765396C (en) 2019-08-20
NO2020008I1 (no) 2020-04-01
IL245096B (en) 2018-04-30
RU2015139553A (ru) 2018-12-25
RU2683772C2 (ru) 2019-04-02
KR20200047790A (ko) 2020-05-07
MX2018015635A (es) 2020-11-12
SMT201800587T1 (it) 2019-01-11
KR20240036132A (ko) 2024-03-19
IL245096A0 (en) 2016-06-30
RU2015139553A3 (enExample) 2018-12-25
DK3449926T3 (da) 2019-11-11
CN102665731A (zh) 2012-09-12
JP2024012416A (ja) 2024-01-30
CN106983768B (zh) 2023-04-07
JP2022025131A (ja) 2022-02-09
US20190030058A1 (en) 2019-01-31
HK1246648B (en) 2019-09-06
AU2016200344A1 (en) 2016-02-11
JP2020073498A (ja) 2020-05-14
CN115227710A (zh) 2022-10-25
HUE039741T2 (hu) 2019-01-28
AU2016200344B2 (en) 2017-12-21
JP6064139B2 (ja) 2017-01-25
PL3305302T3 (pl) 2019-02-28
HRP20181712T1 (hr) 2018-12-28
US20120190728A1 (en) 2012-07-26
CA2765396A1 (en) 2010-12-23
HUE046966T2 (hu) 2020-04-28
ES2761614T3 (es) 2020-05-20
US20200376018A1 (en) 2020-12-03
CN116440150A (zh) 2023-07-18
LT3305302T (lt) 2018-12-10
KR20170054537A (ko) 2017-05-17
US20240238326A1 (en) 2024-07-18
RU2566724C2 (ru) 2015-10-27
JP2012530715A (ja) 2012-12-06
JP2017061497A (ja) 2017-03-30
US9717750B2 (en) 2017-08-01
KR20210057223A (ko) 2021-05-20
KR20120093138A (ko) 2012-08-22
CY1122687T1 (el) 2021-03-12
PT3305302T (pt) 2018-12-14
AU2010262862C1 (en) 2020-04-30
NZ624712A (en) 2015-10-30
SMT202000020T1 (it) 2020-03-13
EP3449926B1 (en) 2019-10-09

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