KR101857402B1 - 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료 - Google Patents

헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료 Download PDF

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KR101857402B1
KR101857402B1 KR1020177011705A KR20177011705A KR101857402B1 KR 101857402 B1 KR101857402 B1 KR 101857402B1 KR 1020177011705 A KR1020177011705 A KR 1020177011705A KR 20177011705 A KR20177011705 A KR 20177011705A KR 101857402 B1 KR101857402 B1 KR 101857402B1
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oligonucleotide
rna
antisense
nucleic acid
target
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KR20170054530A (ko
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조셉 콜라드
올가 크로코바 셔만
칼로스 코이토
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큐알엔에이, 인크.
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
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    • 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
    • A61K2121/00—Preparations for use in 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense

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KR1020177011705A 2009-05-01 2010-07-12 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료 Expired - Fee Related KR101857402B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US17471909P 2009-05-01 2009-05-01
US61/174,719 2009-05-01
PCT/US2010/033078 WO2010127195A2 (en) 2009-05-01 2010-04-30 Antisense oligonucleotides of hemoglobins

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KR1020117027996A Division KR20130018395A (ko) 2009-05-01 2010-07-12 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료

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KR20170054530A KR20170054530A (ko) 2017-05-17
KR101857402B1 true KR101857402B1 (ko) 2018-05-11

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KR1020177011705A Expired - Fee Related KR101857402B1 (ko) 2009-05-01 2010-07-12 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료
KR1020117027996A Ceased KR20130018395A (ko) 2009-05-01 2010-07-12 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료

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US (1) US8318690B2 (https=)
EP (1) EP2424987B1 (https=)
JP (1) JP2013524769A (https=)
KR (2) KR101857402B1 (https=)
CN (1) CN102639151B (https=)
CA (1) CA2760589C (https=)
DK (1) DK2424987T3 (https=)
ES (1) ES2661787T3 (https=)
NO (1) NO2424987T3 (https=)
WO (1) WO2010127195A2 (https=)

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SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
EA201492122A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии utrn
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
BR112014028631A2 (pt) * 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
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US12312584B2 (en) 2018-10-02 2025-05-27 Exosome Therapeutics, Inc. cGMP exosome loaded therapeutics for treating sickle cell disease
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