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公开(公告)号：US09617540B2
公开(公告)日：2017-04-11
申请号：US14698554
申请日：2015-04-28
发明人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewicz
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3515 , C12Y203/0102 , C12N2310/3521
摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
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公开(公告)号：US20190136233A1
公开(公告)日：2019-05-09
申请号：US15772649
申请日：2016-11-07
IPC分类号： C12N15/113 , A61K9/00 , A61K9/08 , A61J1/05
摘要： Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a) [apo(a)]. In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.
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公开(公告)号：US11319536B2
公开(公告)日：2022-05-03
申请号：US16729980
申请日：2019-12-30
IPC分类号： C12N15/113 , A61K9/00 , C07K14/775 , A61K31/7125 , A61J1/05 , A61K9/08
摘要： Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a). In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.
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公开(公告)号：US20210169917A1
公开(公告)日：2021-06-10
申请号：US16947310
申请日：2020-07-28
IPC分类号： A61K31/712 , C12N15/113 , C07H19/20 , A61K48/00 , C07H21/02 , A61P3/06 , C07H21/00 , A61K9/00 , C07H21/04
摘要： Provided herein are methods of administering gapmer oligomeric compounds with GalNAc conjugate groups to a human.
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公开(公告)号：US20190046555A1
公开(公告)日：2019-02-14
申请号：US15771598
申请日：2016-11-07
IPC分类号： A61K31/712 , C07H19/20 , C07H21/02 , C07H21/04 , C12N15/113 , A61P3/06 , A61K9/00 , A61K48/00
摘要： Provided herein are methods of administering gapmer oligomeric compounds with GalNAc conjugate groups to a human.
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公开(公告)号：US10557137B2
公开(公告)日：2020-02-11
申请号：US15772649
申请日：2016-11-07
IPC分类号： C12N15/113 , A61K9/08 , A61J1/05 , A61K9/00
摘要： Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a) [apo(a)]. In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.
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