KR102686857B1 - 변이체 RNAi - Google Patents

변이체 RNAi Download PDF

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KR102686857B1
KR102686857B1 KR1020207011227A KR20207011227A KR102686857B1 KR 102686857 B1 KR102686857 B1 KR 102686857B1 KR 1020207011227 A KR1020207011227 A KR 1020207011227A KR 20207011227 A KR20207011227 A KR 20207011227A KR 102686857 B1 KR102686857 B1 KR 102686857B1
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delete delete
vector
aav
nucleic acid
promoter
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KR20200056428A (ko
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캐서린 알. 오리어던
애덤 팔레르모
브렌다 리처즈
리사 엠. 스타넥
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젠자임 코포레이션
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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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
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    • C12N2310/531Stem-loop; Hairpin
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    • C12N2710/16011Herpesviridae
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    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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KR1020207011227A 2017-09-22 2018-09-21 변이체 RNAi Active KR102686857B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762561843P 2017-09-22 2017-09-22
US62/561,843 2017-09-22
PCT/US2018/052221 WO2019060726A1 (en) 2017-09-22 2018-09-21 Variant rnai

Publications (2)

Publication Number Publication Date
KR20200056428A KR20200056428A (ko) 2020-05-22
KR102686857B1 true KR102686857B1 (ko) 2024-07-18

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US (3) US11603529B2 (https=)
EP (1) EP3684932A1 (https=)
JP (1) JP7248658B2 (https=)
KR (1) KR102686857B1 (https=)
CN (1) CN111356763B (https=)
AR (1) AR113134A1 (https=)
AU (2) AU2018335410B2 (https=)
BR (1) BR112020005569A2 (https=)
CA (1) CA3076191A1 (https=)
CO (1) CO2020003187A2 (https=)
IL (1) IL273394B2 (https=)
MX (2) MX2020003095A (https=)
MY (1) MY205041A (https=)
PH (1) PH12020550117A1 (https=)
SG (1) SG11202002488UA (https=)
TW (1) TWI902656B (https=)
UY (1) UY37897A (https=)
WO (1) WO2019060726A1 (https=)
ZA (1) ZA202001586B (https=)

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JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. ハンチントン病治療組成物および方法
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US20140335054A1 (en) 2010-04-23 2014-11-13 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US20150099793A1 (en) 2013-10-03 2015-04-09 Washington University RATIONAL DESIGN OF microRNA-siRNA CHIMERAS FOR MULTI-FUNCTIONAL TARGET SUPPRESSION

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AU2018335410B2 (en) 2025-04-03
JP2020535803A (ja) 2020-12-10
AU2018335410A1 (en) 2020-05-07
KR20200056428A (ko) 2020-05-22
US12331296B2 (en) 2025-06-17
AR113134A1 (es) 2020-01-29
US20230392149A1 (en) 2023-12-07
CO2020003187A2 (es) 2020-04-13
TW201923079A (zh) 2019-06-16
CN111356763B (zh) 2024-03-12
US20200216848A1 (en) 2020-07-09
IL273394B2 (en) 2025-04-01
SG11202002488UA (en) 2020-04-29
MX2020003095A (es) 2020-10-15
CA3076191A1 (en) 2019-03-28
MY205041A (en) 2024-09-29
WO2019060726A1 (en) 2019-03-28
IL273394B1 (en) 2024-12-01
CN111356763A (zh) 2020-06-30
RU2020114229A3 (https=) 2022-02-01
BR112020005569A2 (pt) 2020-10-06
US11603529B2 (en) 2023-03-14
AU2025205089A1 (en) 2025-07-24
RU2020114229A (ru) 2021-10-22
IL273394A (en) 2020-05-31
JP7248658B2 (ja) 2023-03-29
ZA202001586B (en) 2024-01-31
EP3684932A1 (en) 2020-07-29
MX2025008949A (es) 2025-09-02
UY37897A (es) 2019-04-30
TWI902656B (zh) 2025-11-01
PH12020550117A1 (en) 2020-12-07
US20250290075A1 (en) 2025-09-18

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