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In Vivo Inhibition of MicroRNA to Decrease Tumor Growth in Mice
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In Vivo Inhibition of MicroRNA to Decrease Tumor Growth in Mice

Published on: August 23, 2019

Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression

Manuela Aleku1, Petra Schulz, Oliver Keil

  • 1Silence Therapeutics AG, Berlin, Germany.

Cancer Research
|December 3, 2008
PubMed

Insights

Atu027, a novel siRNA-lipoplex, effectively silences protein kinase N3 (PKN3) in preclinical models. This targeted therapy shows promise for treating advanced solid cancers by inhibiting tumor growth and metastasis.

Area of Science:

  • Oncology
  • Molecular Biology
  • Nanomedicine

Background:

  • Small interfering RNA (siRNA) delivery systems like AtuPLEX enable RNA interference (RNAi) in the vasculature.
  • Protein kinase N3 (PKN3) is a target for cancer therapy.

Purpose of the Study:

  • To report preclinical data for Atu027, a siRNA-lipoplex targeting PKN3 for advanced solid cancer treatment.
  • To evaluate the in vitro and in vivo efficacy and specificity of Atu027.

Main Methods:

  • In vitro studies on primary endothelial cells to assess PKN3 inhibition effects.
  • Systemic administration of Atu027 in mice, rats, and nonhuman primates.
  • Evaluation of Atu027 efficacy in orthotopic prostate and pancreatic cancer mouse models.

Main Results:

  • Atu027 inhibited PKN3 function in endothelial cells, impairing tube formation and migration but not proliferation.
  • Systemic Atu027 administration achieved specific, RNAi-mediated silencing of PKN3 expression across species.
  • Significant inhibition of tumor growth and lymph node metastasis was observed in treated cancer models.
  • Tumor vasculature analysis revealed reduced lymph vessel density without affecting microvascular density.

Conclusions:

  • Atu027 demonstrates potent, specific PKN3 silencing via RNAi in vivo.
  • Atu027 shows significant preclinical efficacy in reducing tumor growth and metastasis in solid cancer models.
  • The findings support the further development of Atu027 as a targeted cancer therapeutic.

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