EA201491048A1 - Соединения двухцепочечных рнк к casp2 и их применение - Google Patents

Соединения двухцепочечных рнк к casp2 и их применение

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Publication number
EA201491048A1
EA201491048A1 EA201491048A EA201491048A EA201491048A1 EA 201491048 A1 EA201491048 A1 EA 201491048A1 EA 201491048 A EA201491048 A EA 201491048A EA 201491048 A EA201491048 A EA 201491048A EA 201491048 A1 EA201491048 A1 EA 201491048A1
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EA
Eurasian Patent Office
Prior art keywords
casp2
connections
application
cellular rna
inhibits
Prior art date
Application number
EA201491048A
Other languages
English (en)
Other versions
EA036003B1 (ru
Inventor
Шай Эрлих
Джеймс Д. Томпсон
Рабия Озден
Original Assignee
Кварк Фармасьютикалс, Инк.
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.)
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Publication date
Application filed by Кварк Фармасьютикалс, Инк. filed Critical Кварк Фармасьютикалс, Инк.
Publication of EA201491048A1 publication Critical patent/EA201491048A1/ru
Publication of EA036003B1 publication Critical patent/EA036003B1/ru

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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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 enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61M5/00Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/002Packages specially adapted therefor, e.g. for syringes or needles, kits for diabetics
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    • C12N15/1135Non-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
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    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
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Abstract

Данное описание касается способов лечения пациентов, страдающих от или с риском развития глазного заболевания, нарушения или повреждения, и включает схемы лечения с использованием соединения двухцепочечной РНК, угнетающей экспрессию CASP2, или его фармацевтически приемлемой соли.
EA201491048A 2012-01-04 2013-01-03 Способ лечения оптической нейропатии с применением двухцепочечной рнк к casp2 EA036003B1 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261582886P 2012-01-04 2012-01-04
US201261596231P 2012-02-08 2012-02-08
PCT/US2013/020012 WO2013103632A1 (en) 2012-01-04 2013-01-03 Double-stranded rna compounds to casp2 and uses thereof

Publications (2)

Publication Number Publication Date
EA201491048A1 true EA201491048A1 (ru) 2015-09-30
EA036003B1 EA036003B1 (ru) 2020-09-11

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
EA201491048A EA036003B1 (ru) 2012-01-04 2013-01-03 Способ лечения оптической нейропатии с применением двухцепочечной рнк к casp2

Country Status (12)

Country Link
US (2) US9382542B2 (ru)
EP (1) EP2800812A1 (ru)
JP (2) JP2015507478A (ru)
KR (2) KR20140109430A (ru)
CN (1) CN104053776A (ru)
AU (1) AU2013206951B2 (ru)
BR (1) BR112014016640A2 (ru)
CA (1) CA2858627C (ru)
EA (1) EA036003B1 (ru)
IL (1) IL233142B (ru)
NZ (1) NZ625980A (ru)
WO (1) WO2013103632A1 (ru)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015136255A1 (en) * 2014-03-11 2015-09-17 The University Of Birmingham Combined inhibition of caspase-2 and caspase-6 in neuroprotection and axon regeneration
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6251666B1 (en) 1997-03-31 2001-06-26 Ribozyme Pharmaceuticals, Inc. Nucleic acid catalysts comprising L-nucleotide analogs
US6444776B1 (en) 1998-12-21 2002-09-03 Novartis Ag Organic polymers
IT1305306B1 (it) * 1999-03-17 2001-05-04 Farmigea Spa Uso della pirenoxina per la protezione dei tessuti corneali ininterventi di fotocheratectomia.
US6444676B1 (en) * 1999-12-20 2002-09-03 Iok-Hou Pang Use of PARP inhibitors in the treatment of glaucoma
US20030157030A1 (en) * 2001-11-02 2003-08-21 Insert Therapeutics, Inc. Methods and compositions for therapeutic use of rna interference
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
WO2005004794A2 (en) * 2003-06-09 2005-01-20 Alnylam Pharmaceuticals Inc. Method of treating neurodegenerative disease
GB0403041D0 (en) * 2004-02-11 2004-03-17 Milner Anne J Induction of apoptosis
WO2007031091A2 (en) * 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
US8168584B2 (en) * 2005-10-08 2012-05-01 Potentia Pharmaceuticals, Inc. Methods of treating age-related macular degeneration by compstatin and analogs thereof
JP2010507387A (ja) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のsiRNAおよびその使用方法
WO2008094860A2 (en) * 2007-01-30 2008-08-07 Allergan, Inc. Treating ocular diseases using peroxisome proliferator-activated receptor delta antagonists
BRPI0817605A2 (pt) 2007-10-03 2017-05-09 Quark Pharmaceuticals Inc novas estruturas de sirna
CA2738957A1 (en) * 2008-10-22 2010-04-29 Quark Pharmaceuticals, Inc. Methods for treating eye disorders
JP2010260857A (ja) * 2009-04-10 2010-11-18 Santen Pharmaceut Co Ltd シロリムス誘導体を有効成分として含有する網脈絡膜疾患の治療剤
WO2011007209A1 (en) * 2009-07-16 2011-01-20 Institut National De La Sante Et De La Recherche Medicale (Inserm) Cleaved and phosphorylated crmp2 as blood marker of inflammatory diseases of the central nervous system
US8785393B2 (en) * 2009-07-31 2014-07-22 Panmira Pharmaceuticals, Llc Ophthalmic pharmaceutical compositions of DP2 receptor antagonists
EP2510098B1 (en) * 2009-12-09 2015-02-11 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the cns
WO2011084193A1 (en) * 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs

Also Published As

Publication number Publication date
KR20140109430A (ko) 2014-09-15
AU2013206951A1 (en) 2014-07-03
EP2800812A1 (en) 2014-11-12
JP2018099117A (ja) 2018-06-28
CA2858627A1 (en) 2013-07-11
JP6774928B2 (ja) 2020-10-28
NZ625980A (en) 2015-05-29
AU2013206951B2 (en) 2016-11-24
CN104053776A (zh) 2014-09-17
IL233142A0 (en) 2014-07-31
JP2015507478A (ja) 2015-03-12
EA036003B1 (ru) 2020-09-11
US20160304879A1 (en) 2016-10-20
US9382542B2 (en) 2016-07-05
WO2013103632A1 (en) 2013-07-11
KR20190137940A (ko) 2019-12-11
US20140371439A1 (en) 2014-12-18
US10570394B2 (en) 2020-02-25
IL233142B (en) 2019-01-31
KR102202004B1 (ko) 2021-01-12
BR112014016640A2 (pt) 2021-03-16
CA2858627C (en) 2020-12-01

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