Phoenix Bio




ヒトApoC-3アンチセンス核酸を投与して7日後のapoC-Ⅲ mRNA

ヒトのapoC-Ⅲ mRNAのみ発現が低下し、ヒトとマウスで薬効の種差が確認されました。

(Wada et al., OTS 2019(15th) ポスター発表資料より改変)



学会:15th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) 2019

  • 演題:Evaluation of Antisense Oligonucleotides Targeting Human mRNA Using Chimeric Mice with Humanized Liver
  • 演者:和田 郁人 先生(国立循環器病研究センター)



  • 演題:ヒト肝臓キメラマウスを核酸医薬の評価モデルに応用するための基礎検討と実用性評価
  • 演者:和田 郁人 先生(国立循環器病研究センター)


学会:The Liver Meeting 2017(AASLD)

  • 演題:GalXC Technology Enables Potent and Durable RNAi-Mediated Inhibition of Hepatitis B Virus in Preclinical Models
  • 演者:Dicerna Pharmaceuticals社


学会:The Liver Meeting 2016(AASLD)

  • 演題:Development of Second Generation RNA Interference Therapy for Hepatitis B Virus Infection
  • 演者:Arbutus Biopharma社


学会:The Liver Meeting 2016(AASLD)

  • 演題:LUNAR-HBV, a UNA Oligomer Combination for the Treatment of Chronic Hepatitis B Virus Infection
  • 演者:Arcturus Therapeutics


学会:The Liver Meeting 2015(AASLD)

  • 演題:TKM-HBV, a Novel RNA Interference Treatment for Chronic Hepatitis B, has a Complementary Mode of Action to Current Standard of Care Nucleotide Analog
  • 演者:Arbutus Biopharma社


学会:EASL The International Liver Congress 2015

  • 演題:RG-101, a Novel Galnac-conjugated inhibitor of microRNA-122, demonstratessignificant viral load reduction and reduces liver steatosis in human hepatocyte chimeric mice infected with genotype 1a or hard-to-treat genotype 3a hepatitis C virus(HCV)
  • 演者:Regulus Therapeutics社


学会:10th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) 2014

  • 演題:Update on the preclinical Development of an LNP-Based HBV Therapeutic
  • 演者:Tekmira社


学会:The Liver Meeting 2013(AASLD)

  • 演題:RG-101, a GalNAc-conjugated anti-miR Employing a Unique Mechanism of Action by Targeting Host Factor MicroRNA-122(miR-122), Demonstrated Potent Activity and Reduction of HCV in Preclinical Studies
  • 演者:Regulus Therapeutics社




  • 掲載雑誌名:ACS Infect Dis. 2019 May 10;5(5):725-737. doi: 10.1021/acsinfecdis.8b00191. Epub 2018 Nov 19.
  • 論文名:ARB-1740, a RNA Interference Therapeutic for Chronic Hepatitis B Infection
  • 著者:Thi et al.(Arbutus Biopharma社)
  • URL(外部リンク):


  • 掲載雑誌名:ACS Infect Dis. 2019 May 10;5(5):738-749. doi: 10.1021/acsinfecdis.8b00192. Epub 2018 Nov 20.
  • 論文名:Hepatitis B Virus Therapeutic Agent ARB-1740 Has Inhibitory Effect on Hepatitis Delta Virus in a New Dually-Infected Humanized Mouse Model
  • 著者:Ye et al.(Arbutus Biopharma社)
  • URL(外部リンク):


  • 掲載雑誌名:Nucleic Acid Ther. 2018 Oct;28(5):285-296. doi: 10.1089/nat.2018.0734. Epub 2018 Aug 8.
  • 論文名:MicroRNAs Enable mRNA Therapeutics to Selectively Program Cancer Cells to Self-Destruct
  • 著者:Jain et al.(Moderna Therapeutics社)
  • URL(外部リンク):


  • 掲載雑誌名:Mol Ther. 2018 Aug 1;26(8):1983-1995. doi: 10.1016/j.ymthe.2018.05.016. Epub 2018 Jun 15.
  • 論文名:Specific Inhibition of Hepatic Lactate Dehydrogenase Reduces Oxalate Production in Mouse Models of Primary Hyperoxaluria
  • 著者:Lai et al.(Dicerna Pharmaceuticals社)
  • URL(外部リンク):


  • 掲載雑誌名:J Control Release. 2017 Nov 28;266:216-225. doi: 10.1016/j.jconrel.2017.09.044. Epub 2017 Oct 3.
  • 論文名:Highly specific delivery of siRNA to hepatocytes circumvents endothelial cell-mediated lipid nanoparticle-associated toxicity leading to the safe and efficacious decrease in the hepatitis B virus
  • 著者:Sato et al.(北海道大学)
  • URL(外部リンク):


  • 掲載雑誌名:J Hepatol. 2016 Mar;64(3):547-55. doi: 10.1016/j.jhep.2015.10.014. Epub 2015 Oct 24.
  • 論文名:Novel pH-sensitive multifunctional envelope-type nanodevice for siRNA-based treatments for chronic HBV infection
  • 著者:Yamamoto et al.(東京都医学総合研究所)
  • URL(外部リンク):


  • 掲載雑誌名:J Virol. 2014 May;88(9):4647-56. doi: 10.1128/JVI.00105-14. Epub 2014 Jan 29.
  • 論文名:Inhibition of hepatitis C virus in chimeric mice by short synthetic hairpin RNAs: sequence analysis of surviving virus shows added selective pressure of combination therapy
  • 著者:Dallas et al.(SomaGenics社)
  • URL(外部リンク):


  • 掲載雑誌名:Sci Rep. 2014 Apr 23;4:4750. doi: 10.1038/srep04750.
  • 論文名:In vivo therapeutic potential of Dicer-hunting siRNAs targeting infectious hepatitis C virus
  • 著者:Watanabe et al.(東京都医学総合研究所)
  • URL(外部リンク):


  • 掲載雑誌名:Gastroenterology. 2014 Jan;146(1):63-6.e5. doi: 10.1053/j.gastro.2013.09.049. Epub 2013 Sep 27.
  • 論文名:Formulated minimal-length synthetic small hairpin RNAs are potent inhibitors of hepatitis C virus in mice with humanized livers
  • 著者:Ma et al.(Hoffmann-La Roche社)
  • URL(外部リンク):