BRPI0418104A - compostos oligoméricos para a modulação de bcl-2 - Google Patents

compostos oligoméricos para a modulação de bcl-2

Info

Publication number
BRPI0418104A
BRPI0418104A BRPI0418104-2A BRPI0418104A BRPI0418104A BR PI0418104 A BRPI0418104 A BR PI0418104A BR PI0418104 A BRPI0418104 A BR PI0418104A BR PI0418104 A BRPI0418104 A BR PI0418104A
Authority
BR
Brazil
Prior art keywords
lna
bcl
rna
dna
target binding
Prior art date
Application number
BRPI0418104-2A
Other languages
English (en)
Inventor
Miriam Frieden
Jens Bo Hansen
Henrik Orum
Majken Westergaard
Charlotte Albaeck Thrue
Original Assignee
Santaris Pharma As
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Santaris Pharma As filed Critical Santaris Pharma As
Publication of BRPI0418104A publication Critical patent/BRPI0418104A/pt

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Oncology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Compositions Of Macromolecular Compounds (AREA)

Abstract

"COMPOSTOS OLIGOMéRICOS PARA A MODULAçãO DE BCL-2". A presente invenção fornece composto oligomérico melhorado, em particular compostos de oligonucleotídeo, e métodos para modular a expressão do gene de Bcl-2 em seres humanos. Em particular, esta invenção refere-se a compostos oligoméricos de 10 a 30 nucleobases de comprimento que compreendem um domínio de ligação alvo que é especificamente hibridizável para uma região variando da posição de base n<0> 1459 (5,<39>) n<198> 1476 (3<39>) do mRNA de Bcl-2 humano, o referido domínio e ligação alvo tendo a fórmula: 5<39>-¢(DNA/RNA)~ 0-1~-(LNA/LNA*)~ 2-7~-(DNA/RNA/LNA*)~ 4-14~-(LNA/LNA*)~ 2-7~-(DNA/RNA)~ 0-1~!-3<39> e o referido domínio de ligação alvo compreendendo pelo menos dois nucleotídeos de LNA ou nucleotídeos análogos de LNA ligados por um grupo de fosforotioato (-O-P(O,S)-O-). Em particular o oligo é predominantemente ou totalmente tiolado. A invenção também fornece o uso de tais oligómeros ou conjugados ou quimera para o tratamento de várias doenças associadas com a expressão o gene de Bcl-2, tal como câncer.
BRPI0418104-2A 2003-12-23 2004-12-23 compostos oligoméricos para a modulação de bcl-2 BRPI0418104A (pt)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US53284403P 2003-12-23 2003-12-23
DKPA200301929 2003-12-23
US55839204P 2004-03-31 2004-03-31
DKPA200400517 2004-03-31
US58634004P 2004-07-07 2004-07-07
DKPA200401069 2004-07-07
US62159404P 2004-10-22 2004-10-22
DKPA200401629 2004-10-22
PCT/DK2004/000917 WO2005061710A1 (en) 2003-12-23 2004-12-23 Oligomeric compounds for the modulation of bcl-2

Publications (1)

Publication Number Publication Date
BRPI0418104A true BRPI0418104A (pt) 2007-04-17

Family

ID=42727309

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0418104-2A BRPI0418104A (pt) 2003-12-23 2004-12-23 compostos oligoméricos para a modulação de bcl-2

Country Status (16)

Country Link
US (1) US7622453B2 (pt)
EP (1) EP1706489B9 (pt)
JP (1) JP4642775B2 (pt)
KR (1) KR20070006709A (pt)
CN (1) CN100569945C (pt)
AT (1) ATE467679T1 (pt)
AU (1) AU2004303464B2 (pt)
BR (1) BRPI0418104A (pt)
CA (1) CA2550258A1 (pt)
DE (1) DE602004027163D1 (pt)
DK (1) DK1706489T3 (pt)
ES (1) ES2344566T3 (pt)
IL (1) IL176320A0 (pt)
NZ (1) NZ548254A (pt)
RU (1) RU2377301C2 (pt)
WO (1) WO2005061710A1 (pt)

Families Citing this family (124)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2141233B1 (en) 2002-11-18 2016-10-19 Roche Innovation Center Copenhagen A/S Antisense design
US7399853B2 (en) 2003-04-28 2008-07-15 Isis Pharmaceuticals Modulation of glucagon receptor expression
US20050287667A1 (en) * 2004-06-01 2005-12-29 Pronai Therapeutics, Inc. Methods and compositions for the inhibition of gene expression
US8815599B2 (en) 2004-06-01 2014-08-26 Pronai Therapeutics, Inc. Methods and compositions for the inhibition of gene expression
US7807647B2 (en) * 2004-06-01 2010-10-05 Pronai Therapeutics, Inc. Methods and compositions for cancer therapy
AU2006292217A1 (en) * 2005-09-19 2007-03-29 Isis Pharmaceuticals, Inc. Modulation of glucagon receptor expression
AU2006292293B2 (en) * 2005-09-19 2012-09-13 Isis Pharmaceuticals, Inc. Modulation of glucocorticoid receptor expression
WO2007064853A2 (en) * 2005-12-01 2007-06-07 Pronai Therapeutics, Inc. Locked nucleic acid oligonucleotides
CA2631677C (en) 2005-12-01 2014-08-12 Pronai Therapeutics, Inc. Amphoteric liposome formulation
EP3000480A1 (en) 2005-12-01 2016-03-30 ProNAi Therapeutics, Inc. Cancer therapies and pharmaceutical compositions used therein
CN101437933B (zh) 2005-12-28 2013-11-06 斯克里普斯研究所 作为药物靶标的天然反义和非编码的rna转录物
AU2012216487B2 (en) * 2006-04-03 2015-05-14 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-miRNA antisense oligonucleotides
EP2007889A2 (en) * 2006-04-03 2008-12-31 Santaris Pharma A/S Pharmaceutical compositions comprising anti-mirna antisense oligonucleotides
EA201100813A1 (ru) 2006-04-03 2012-06-29 Сантарис Фарма А/С Фармацевтическая композиция
WO2007143317A2 (en) 2006-05-05 2007-12-13 Isis Pharmaceuticals, Inc Compounds and methods for modulating expression of crp
US20090023221A1 (en) * 2006-05-19 2009-01-22 Exigon A/S Oligonucleotide probes useful for detection and analysis of microrna precursors
EP3916095A1 (en) * 2006-10-18 2021-12-01 Ionis Pharmaceuticals, Inc. Antisense compounds
US8580756B2 (en) 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
US8470791B2 (en) 2007-03-22 2013-06-25 Santaris Pharma A/S RNA antagonist compounds for the inhibition of Apo-B100 expression
DK2548962T3 (en) 2007-09-19 2016-04-11 Applied Biosystems Llc Sirna sequence-independent modification formats to reduce off-target phenotype effects in RNAI and stabilized forms thereof
CN101821391B (zh) 2007-10-04 2016-04-27 桑塔里斯制药公司 微小聚体
BRPI0820508A2 (pt) * 2007-11-26 2017-05-23 Enzon Pharmaceuticals Inc antagonistas de lna cujo alvo é o receptor de andrógeno
WO2009071681A2 (en) * 2007-12-07 2009-06-11 Santaris Pharma A/S Rna antagonist compounds for the modulation of bcl-2
CA2717792A1 (en) 2008-03-07 2009-09-11 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
US8492357B2 (en) 2008-08-01 2013-07-23 Santaris Pharma A/S Micro-RNA mediated modulation of colony stimulating factors
US8153606B2 (en) 2008-10-03 2012-04-10 Opko Curna, Llc Treatment of apolipoprotein-A1 related diseases by inhibition of natural antisense transcript to apolipoprotein-A1
EP3401684B1 (en) * 2008-10-10 2022-03-16 Dana Farber Cancer Institute, Inc. Chemical modulators of pro-apoptotic bax and bcl-2 polypeptides
WO2010043582A1 (en) * 2008-10-17 2010-04-22 Santaris Pharma A/S Method for the treatment of cancer
US8927511B2 (en) 2008-12-04 2015-01-06 Curna, Inc. Treatment of vascular endothelial growth factor (VEGF) related diseases by inhibition of natural antisense transcript to VEGF
KR20110091796A (ko) 2008-12-04 2011-08-12 오피케이오 큐알엔에이, 엘엘씨 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료
JP6091752B2 (ja) 2008-12-04 2017-03-08 クルナ・インコーポレーテッド Ｅｐｏに対する天然アンチセンス転写物の抑制によるエリスロポエチン（ｅｐｏ）関連疾患の治療
ES2560107T3 (es) 2009-02-12 2016-02-17 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado de cerebro (BDNF) por inhibición de transcrito antisentido natural para BDNF
EP2408919B1 (en) 2009-03-16 2017-10-18 CuRNA, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
MX2011009752A (es) 2009-03-17 2011-09-29 Opko Curna Llc Tratamiento de enfermedades relacionadas a homologo tipo delta 1(dlk1) por inhibicion de transcrito antisentido natural a homologo tipo delta (dlk1).
ES2599979T3 (es) 2009-04-24 2017-02-06 Roche Innovation Center Copenhagen A/S Composiciones farmacéuticas para el tratamiento de pacientes de VHC que no responden al interferón
WO2010129799A2 (en) 2009-05-06 2010-11-11 Curna, Inc. Treatment of lipid transport and metabolism gene related diseases by inhibition of natural antisense transcript to a lipid transport and metabolism gene
JP6250930B2 (ja) 2009-05-06 2017-12-20 クルナ・インコーポレーテッド トリステトラプロリン（ｔｔｐ）に対する天然アンチセンス転写物の抑制によるｔｔｐ関連疾患の治療
JP5931720B2 (ja) 2009-05-08 2016-06-08 クルナ・インコーポレーテッド Ｄｍｄファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療
US8957037B2 (en) 2009-05-18 2015-02-17 Curna, Inc. Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor
KR101703695B1 (ko) 2009-05-22 2017-02-08 큐알엔에이, 인크. 전사 인자 ｅ3(ｔｆｅ3)에 대한 천연 안티센스 전사체의 억제에 의해 ｔｆｅ3 및 인슐린 수용체 기질 2(ｉｒｓ2)의 치료
CN103221541B (zh) 2009-05-28 2017-03-01 库尔纳公司 通过抑制抗病毒基因的天然反义转录物来治疗抗病毒基因相关疾病
JP5944311B2 (ja) 2009-06-16 2016-07-05 クルナ・インコーポレーテッド コラーゲン遺伝子に対する天然アンチセンス転写物の抑制によるコラーゲン遺伝子関連疾患の治療
US8951981B2 (en) 2009-06-16 2015-02-10 Curna, Inc. Treatment of paraoxonase 1 (PON1) related diseases by inhibition of natural antisense transcript to PON1
ES2618894T3 (es) 2009-06-24 2017-06-22 Curna, Inc. Tratamiento de enfermedades relacionadas con el receptor del factor de necrosis tumoral 2 (tnfr2) por inhibición del transcrito natural antisentido para tnfr2
JP5907866B2 (ja) 2009-06-26 2016-04-26 クルナ・インコーポレーテッド ダウン症候群遺伝子に対する天然アンチセンス転写物の抑制によるダウン症候群遺伝子関連疾患の治療
EP2456870A1 (en) 2009-07-21 2012-05-30 Santaris Pharma A/S Antisense oligomers targeting pcsk9
CN102712925B (zh) 2009-07-24 2017-10-27 库尔纳公司 通过抑制sirtuin(sirt)的天然反义转录物来治疗sirtuin(sirt)相关性疾病
WO2011017516A2 (en) 2009-08-05 2011-02-10 Curna, Inc. Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
CN104313027B (zh) 2009-08-11 2018-11-20 库尔纳公司 通过抑制脂连蛋白(adipoq)的天然反义转录物治疗脂连蛋白(adipoq)相关疾病
EP2467482A4 (en) 2009-08-21 2013-12-11 Curna Inc TREATMENT OF DISEASES RELATED TO "CIP PROTEIN CHIP EXTREME (PROTEIN INTERACTING WITH HSP70)" BY INHIBITION OF CHIP NATURAL ANTISENSE TRANSCRIPT
JP5964232B2 (ja) 2009-08-25 2016-08-03 カッパーアールエヌエー，インコーポレイテッド ‘ｉｑモチーフ含有ｇｔｐアーゼ活性化タンパク質’（ｉｑｇａｐ）に対する天然アンチセンス転写産物の阻害によるｉｑｇａｐ関連疾患の治療
ES2664591T3 (es) 2009-09-25 2018-04-20 Curna, Inc. Tratamiento de enfermedades relacionadas con la filagrina (flg) mediante la modulación de la expresión y actividad del gen FLG
DK2513310T3 (en) 2009-12-16 2018-02-05 Curna Inc TREATMENT OF DISEASES CONNECTED WITH MEMBRANE-BONDED TRANSCRIPTION FACTOR Peptidase, site 1 (MBTPS1), BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPTION TO MBTPS1
CA2782375C (en) 2009-12-23 2023-10-31 Opko Curna, Llc Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
ES2661387T3 (es) 2009-12-23 2018-03-28 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor de crecimiento de hepatocitos (hgf) mediante inhibición del transcrito antisentido natural a hgf
EP2519634B1 (en) 2009-12-29 2016-06-01 CuRNA, Inc. TREATMENT OF TUMOR PROTEIN 63 (p63) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO p63
JP5993744B2 (ja) 2009-12-29 2016-09-14 カッパーアールエヌエー，インコーポレイテッド 核呼吸因子１（ｎｒｆ１）に対する天然アンチセンス転写物の阻害による核呼吸因子１関連疾患の治療
DK2519632T3 (en) * 2009-12-31 2018-07-23 Curna Inc TREATMENT OF INSULIN RECEPTOR SUBSTRATE 2- (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPTION TO IRS2 AND TRANSCRIPTION FACTOR E3 (TFE3)
DK2521784T3 (en) 2010-01-04 2018-03-12 Curna Inc TREATMENT OF INTERFERON REGULATORY FACTOR 8- (IRF8) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPT TO IRF8
KR101853509B1 (ko) 2010-01-06 2018-04-30 큐알엔에이, 인크. 췌장 발달 유전자에 대한 천연 안티센스 전사체의 억제에 의한 췌장 발달 유전자와 관련된 질환의 치료
KR101854926B1 (ko) 2010-01-11 2018-05-04 큐알엔에이, 인크. 성 호르몬 결합 글로불린 (ｓｈｂｇ)에 대한 자연 안티센스 전사체의 저해에 의한 성 호르몬 결합 글로불린 (ｓｈｂｇ) 관련된 질환의 치료
ES2671877T3 (es) 2010-01-25 2018-06-11 Curna, Inc. Tratamiento de enfermedades relacionadas con la RNASA (H1) mediante inhibición del transcrito antisentido natural a RNASA H1
ES2600356T3 (es) 2010-02-22 2017-02-08 Curna, Inc. Tratamiento de enfermedades relacionadas con pirrolin-5-carboxilato reductasa 1 (PYCR1) por inhibición de transcrito antisentido natural para PYCR1
US8980856B2 (en) 2010-04-02 2015-03-17 Curna, Inc. Treatment of colony-stimulating factor 3 (CSF3) related diseases by inhibition of natural antisense transcript to CSF3
JP5978203B2 (ja) 2010-04-09 2016-08-24 カッパーアールエヌエー，インコーポレイテッド Ｆｇｆ２１に対する天然アンチセンス転写物の阻害による線維芽細胞増殖因子（ｆｇｆ２１）線維芽細胞増殖因子ｆｇｆ２１）関連疾患の治療
EP2957636B1 (en) 2010-05-03 2020-04-01 CuRNA, Inc. Treatment of sirtuin 3 (sirt3) related diseases by inhibition of natural antisense transcript to sirt3
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制ｐａｒ４天然反股轉錄本治療ｐａｒ４相關疾病
US8895528B2 (en) 2010-05-26 2014-11-25 Curna, Inc. Treatment of atonal homolog 1 (ATOH1) related diseases by inhibition of natural antisense transcript to ATOH1
DK2576784T3 (en) 2010-05-26 2018-02-26 Curna Inc TREATMENT OF METHIONIN SULPHOXIDE REDUCTASE A (MSRA) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPTION TO MSRA
DK2585596T3 (da) 2010-06-23 2021-04-06 Curna Inc Behandling af spændingsreguleret natriumkanal-alpha-underenhed (scna)-relaterede sygdomme ved inhibering af naturligt antisense-transkript til scna
JP5998131B2 (ja) 2010-07-14 2016-09-28 カッパーアールエヌエー，インコーポレイテッド Ｄｉｓｃｓｌａｒｇｅｈｏｍｏｌｏｇ（ｄｌｇ）ｄｌｇ１への天然アンチセンス転写物の阻害によるｄｌｇ関連疾患の治療
US8993533B2 (en) 2010-10-06 2015-03-31 Curna, Inc. Treatment of sialidase 4 (NEU4) related diseases by inhibition of natural antisense transcript to NEU4
US9222088B2 (en) 2010-10-22 2015-12-29 Curna, Inc. Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
WO2012068340A2 (en) 2010-11-18 2012-05-24 Opko Curna Llc Antagonat compositions and methods of use
JP6071893B2 (ja) 2010-11-23 2017-02-01 カッパーアールエヌエー，インコーポレイテッド Ｎａｎｏｇへの天然アンチセンス転写物の阻害によるｎａｎｏｇ関連疾患の治療
EP2673361B1 (en) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
ES2653247T3 (es) 2011-06-09 2018-02-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la frataxina (FXN) mediante inhibición del transcrito antisentido natural al gen FXN
EP2742136B1 (en) 2011-08-11 2017-09-27 Ionis Pharmaceuticals, Inc. Gapped oligomeric compounds comprising 5'-modified deoxyribonucleosides in the gap and uses thereof
MX365525B (es) 2011-09-06 2019-06-06 Curna Inc Compuestos que regulan la expresión de subunidades alfa de canales de sodio regulados por voltaje en enfermedades relacionadas con epilepsia mioclónica severa de la infancia.
CA2848753C (en) 2011-09-14 2022-07-26 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
EP3401401B1 (en) 2011-09-20 2020-04-15 Ionis Pharmaceuticals, Inc. Antisense modulation of gcgr expression
WO2013055865A1 (en) 2011-10-11 2013-04-18 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
AU2012328680A1 (en) 2011-10-25 2014-05-01 Ionis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
US20140323557A1 (en) * 2011-11-11 2014-10-30 Santaris Pharma A/S Compounds for the modulation of beta-catenin expression and uses thereof
HUE040179T2 (hu) 2012-03-15 2019-02-28 Curna Inc Agyi eredetû neutrotróf faktorral (Brain-derived neurotrophic factor, BDNF) összefüggõ betegségek kezelése a BDNF-fel kapcsolatos természetes antiszensz transzkriptumok gátlása révén
WO2013159108A2 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
EP2850182A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc COMPOSITIONS AND METHODS FOR MODULATING ATP2A2 GENE EXPRESSION
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EP2850188A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING THE EXPRESSION OF THE HEMOGLOBIN GENE FAMILIES
WO2013173638A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating smn gene family expression
KR20160074368A (ko) 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
CN102827056B (zh) * 2012-09-03 2014-07-23 华东理工大学 N-芳基取代吡咯烷酮衍生物及其用途
CA2884608C (en) * 2012-09-14 2025-05-20 Translate Bio Ma, Inc. MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS
AU2013324716B2 (en) 2012-09-26 2019-06-13 Guandong Mijinjia Biotechnology Co., Ltd Oligomers with improved off-target profile
WO2014059353A2 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
WO2014071406A1 (en) 2012-11-05 2014-05-08 Pronai Therapeutics, Inc. Methods of using biomarkers for the treatment of cancer by modulation of bcl2|expression
HK1214297A1 (zh) 2012-11-15 2016-07-22 Roche Innovation Center Copenhagen A/S 寡核苷酸缀合物
RU2018108405A (ru) 2013-01-30 2019-02-26 Ф. Хоффманн-Ля Рош Аг Конъюгаты углевода и lna-олигонуклеотида
JP6492053B2 (ja) 2013-03-27 2019-03-27 イサルナ セラピューティクス ゲゼルシャフト ミット ベシュレンクテル ハフツング 修飾ｔｇｆ−ベータオリゴヌクレオチド
CN105164261B (zh) 2013-05-01 2022-03-18 莱古路斯治疗法股份有限公司 用于增强的细胞摄取的化合物和方法
EP2992095B1 (en) 2013-05-01 2019-01-09 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-122
MX391977B (es) 2013-06-27 2025-03-21 Roche Innovation Ct Copenhagen As Oligómeros antisentido y conjugados con diana en la proteína-convertasa subtilisina/kexina tipo 9(pcsk9).
WO2015023937A1 (en) * 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Heterochromatin forming non-coding rnas
WO2015075166A1 (en) 2013-11-22 2015-05-28 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for treatment of a bacterial infection
WO2015118407A2 (en) 2014-01-29 2015-08-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Oligonucleotides and methods for inhibiting or reducing bacterial biofilms
EP3341479B1 (en) * 2015-08-24 2019-12-18 Roche Innovation Center Copenhagen A/S Lna-g process
WO2017067970A1 (en) 2015-10-22 2017-04-27 Roche Innovation Center Copenhagen A/S In vitro toxicity screening assay
CN107574243B (zh) * 2016-06-30 2021-06-29 博奥生物集团有限公司 分子标志物、内参基因及其应用、检测试剂盒以及检测模型的构建方法
KR102519171B1 (ko) 2016-10-07 2023-04-07 세카나 파머씨티컬스 지엠비에이치 엔 씨오. 케이지 암 치료를 위한 새로운 접근법
KR20190133173A (ko) * 2017-03-29 2019-12-02 로슈 이노베이션 센터 코펜하겐 에이/에스 유니링커 신속 절단
DK3669193T3 (da) * 2017-08-18 2022-07-11 European Molecular Biology Laboratory Forbedret rna-interaktom-registrering (eric)
WO2019076919A1 (en) 2017-10-17 2019-04-25 INSERM (Institut National de la Santé et de la Recherche Médicale) COMBINED TREATMENT OF CYSTIC FIBROSIS
TWI854980B (zh) 2018-05-08 2024-09-11 美商雷格勒斯治療公司 用於調節mir-122之微小rna化合物及方法
EP3997225A1 (en) 2019-07-10 2022-05-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
WO2021074657A1 (en) 2019-10-17 2021-04-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Combination treatment for cystic fibrosis
EP4061943A1 (en) 2019-11-19 2022-09-28 Institut National de la Santé et de la Recherche Médicale (INSERM) Antisense oligonucleotides and their use for the treatment of cancer
CN115996935B (zh) * 2020-07-09 2025-11-14 豪夫迈·罗氏有限公司 使用改进的氧化方案制备寡核苷酸的方法
WO2022011262A1 (en) 2020-07-10 2022-01-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treating epilepsy
WO2023152369A1 (en) 2022-02-14 2023-08-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Nucleic acid mir-9 inhibitor for the treatment of cystic fibrosis
WO2024017990A1 (en) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
CN120752339A (zh) 2023-01-06 2025-10-03 国家医疗保健研究所 静脉内施用用于治疗疼痛的反义寡核苷酸
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2601676B1 (fr) 1986-07-17 1988-09-23 Rhone Poulenc Sante Procede de preparation du taxol et du desacetyl-10 taxol
US5831066A (en) * 1988-12-22 1998-11-03 The Trustees Of The University Of Pennsylvania Regulation of bcl-2 gene expression
US5108921A (en) 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
US5278324A (en) 1990-08-28 1994-01-11 Virginia Tech Intellectual Properties, Inc. Water soluble derivatives of taxol
MX9102128A (es) 1990-11-23 1992-07-08 Rhone Poulenc Rorer Sa Derivados de taxano,procedimiento para su preparacion y composicion farmaceutica que los contiene
US5250683A (en) 1991-09-23 1993-10-05 Florida State University Certain substituted taxanes and pharmaceutical compositions containing them
US5227400A (en) 1991-09-23 1993-07-13 Florida State University Furyl and thienyl substituted taxanes and pharmaceutical compositions containing them
US5272171A (en) 1992-02-13 1993-12-21 Bristol-Myers Squibb Company Phosphonooxy and carbonate derivatives of taxol
FR2688518B1 (fr) 1992-03-13 1994-05-06 Rhone Poulenc Rorer Sa Procede de preparation de derives du taxane.
US5248796A (en) 1992-06-18 1993-09-28 Bristol-Myers Squibb Company Taxol derivatives
US5254580A (en) 1993-01-19 1993-10-19 Bristol-Myers Squibb Company 7,8-cyclopropataxanes
JP2001505401A (ja) 1993-09-20 2001-04-24 シー． リード，ジョン ｂｃ１−２遺伝子発現の調節
US6133246A (en) * 1997-08-13 2000-10-17 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the modulation of JNK proteins
RU2243231C2 (ru) * 1997-09-12 2004-12-27 Эксикон А/С Бициклические аналоги нуклеозидов, нуклеотидов и олигонуклеотидов
US6794499B2 (en) * 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
EP2341057A3 (en) * 1997-09-12 2011-11-23 Exiqon A/S Oligonucleotide Analogues
ATE423789T1 (de) 1997-09-16 2009-03-15 Univ Oregon Health & Science Rekombinante mhc-moleküle welche nützlich sind für die manipulation von antigen-spezifischen t- zellen
CN1350542A (zh) * 1999-03-18 2002-05-22 埃克西库恩公司 木－lna类似物
ES2269113T3 (es) * 1999-03-24 2007-04-01 Exiqon A/S Sintesis mejorada de -2.2.1 / biciclo-nucleosidos.
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
JP2003511016A (ja) 1999-10-04 2003-03-25 エクシコン エ／エス オリゴヌクレオチドを補充する高親和性ｒｎアーゼｈの設計
DE60119562T2 (de) 2000-10-04 2007-05-10 Santaris Pharma A/S Verbesserte synthese von purin-blockierten nukleinsäure-analoga
AU2002317437A1 (en) 2001-05-18 2002-12-03 Cureon A/S Therapeutic uses of lna-modified oligonucleotides in infectious diseases
AU2002328792A1 (en) 2001-07-12 2003-01-29 Santaris Pharma A/S Method for preparation of lna phosphoramidites
EP1442143A4 (en) * 2002-02-20 2005-02-16 Sirna Therapeutics Inc INHIBITION OF BCL2 GENE EXPRESSION MEDIATED BY RNA INTERFERENCE USING siNA (SHORT INTERFERING NUCLEIC ACID)
DE60315444T2 (de) 2002-05-08 2008-04-30 Santaris Pharma A/S Synthese von locked nucleic acid-derivaten
EP2141233B1 (en) 2002-11-18 2016-10-19 Roche Innovation Center Copenhagen A/S Antisense design

Also Published As

Publication number Publication date
EP1706489A1 (en) 2006-10-04
AU2004303464B2 (en) 2009-10-01
EP1706489B9 (en) 2011-01-05
JP4642775B2 (ja) 2011-03-02
CA2550258A1 (en) 2005-07-07
KR20070006709A (ko) 2007-01-11
DE602004027163D1 (de) 2010-06-24
CN1918292A (zh) 2007-02-21
ES2344566T3 (es) 2010-08-31
WO2005061710A1 (en) 2005-07-07
AU2004303464A1 (en) 2005-07-07
JP2007515175A (ja) 2007-06-14
EP1706489B1 (en) 2010-05-12
ATE467679T1 (de) 2010-05-15
US7622453B2 (en) 2009-11-24
CN100569945C (zh) 2009-12-16
RU2377301C2 (ru) 2009-12-27
DK1706489T3 (da) 2010-09-13
NZ548254A (en) 2008-09-26
RU2006126643A (ru) 2008-01-27
US20050203042A1 (en) 2005-09-15
IL176320A0 (en) 2006-10-05

Similar Documents

Publication Publication Date Title
BRPI0418104A (pt) compostos oligoméricos para a modulação de bcl-2
BRPI0912923A8 (pt) Composto que consiste de um oligonucleotídeo modificadpo composto de 12-30 nucleosídeos ligados, compostos que compreende um oligonucleotídeo modificado que compreende pelo menos 12 nucleosídeos ligados, composto que compreende um oligonucleotídeo modificado que compreende nucleosídeos ligados, e, uso de um composto
ATE460481T1 (de) Verfahren zur hemmung der expression eines zielgens und medikament zur therapie einer tumorerkrankung
EP1325019A4 (en) ANTISENSE MODULATION OF CLUSTERINEXPRESSION
MX2010003606A (es) Modulacion antisentido de la expresion del receptor del factor de crecimiento fibroblastico 4.
ATE540118T1 (de) Antisense-verbindungen
EP1250455A4 (en) ANTISENSE INHIBITION OF PTP1B EXPRESSION.
WO2005049630A3 (en) Antisense modulation of kinesin-like 1 expression
WO2003048315A3 (en) Antisense modulation of mdm2 expression
EP1190099A4 (en) ANTISENSE MODULATION OF PI3K P85 EXPRESSION
EP2527444A3 (en) Antisense oligonucleotide modulation of stat3 expression
EP1248794A4 (en) ANTISENS MODULATION OF THE SMAD7 EXPRESSION
SI2056845T1 (en) STRUCTURE AND USE 5 &#39;PHOSPHATE OF OLIGONUCLEOTES
EP1165594A4 (en) ANTISENS OLIGONUCLEOTIDE MODULATION OF THE STAT3 EXPRESSION
BRPI0514391A (pt) compostos de triazolopiridina para o tratamento de inflamação
EP1417216A4 (en) ANTISENSE MODULATION OF BCL2 ASSOCIATED X-PROTEIN EXPRESSION
WO2001032832A3 (en) Antisense inhibition of nucleolin expression
PT1175489E (pt) Oligonucleotidos contendo uma sequencia antisense estabilizada por uma estrutura secundaria e composicoes farmaceuticas contendo as mesmas
EP1406915A4 (en) ANTI-SENSE MODULATION OF BETA TYPE II TRANSFORMING GROWTH FACTOR RECEPTOR EXPRESSION
EP1648910A4 (en) ANTISENSE MODULATION OF THE EXPRESSION OF STEAROYL COA DESATURASE
BRPI0417988A (pt) análogos de fosfonato antivirais
WO2002095053A3 (en) Antisense modulation of src-c expression
EP1248633A4 (en) ANTISENSE MODULATION OF THE EXPRESSION OF GLYCOGEN SYNTHASE KINASE 3 BETA
BR0210401A (pt) Composto, composição farmacêutica, uso de um composto e método para o tratamento de um distúrbio ou de uma doença de um corpo de animal vivo, incluindo um humano
EP1322658A4 (en) OLIGONUCLEOTIDE MODULATION OF HER-1 RECEPTOR EXPRESSION

Legal Events

Date Code Title Description
B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

Free format text: REFERENTE A 9A ANUIDADE.

B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: REFERENTE AO DESPACHO 8.6 PUBLICADO NA RPI 2260 DE 29/04/2014.