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Pharmaceutical industry

Marina Biotech Reports In Vivo Dose-Dependent Efficacy with CRN-Substituted miRNA Antagonist

Strategic Insights

— 1 min read

Marina Biotech, a leading oligonucleotide-based drug discovery and development company, has reported in vivo dose-dependent efficacy with a CRN-substituted miRNA antagonist against microRNA-122 (miR-122). The efficacy was demonstrated in a rodent model by up to a 5-fold increase in AldoA, a downstream gene regulated by miR-122. The increase in downstream gene targets was achieved by the sequestration of miR-122 by a high-affinity CRN-substituted antagomir.

Key Takeaways:

  • Marina Biotech reported in vivo dose-dependent efficacy with a CRN-substituted miRNA antagonist against microRNA-122 (miR-122) in a rodent model.
  • The efficacy was demonstrated by up to a 5-fold increase in AldoA, a downstream gene regulated by miR-122.
  • Downstream targets Glycogen Synthase I (GYS1) and Solute Carrier Family 7 member 1 (SLC7A1) were also elevated, with an increase in these targets achieved by the sequestration of miR-122 by a high-affinity CRN-substituted antagomir.
  • The CRN-substituted antagomir was dosed for three consecutive days at up to 50 mg/kg/day and was extremely well tolerated in rodents, with normal serum chemistry parameters and no body weight changes.
  • Richard T. Ho, M.D.-Ph.D, Executive Vice President, Research and Development at Marina Biotech, stated that the results are significant achievements in the development of both the CRN technology and the company's oligonucleotide-based drug discovery platform.
  • The company's CRN patent estate consists of two issued patents covering CRN compounds and CRN-containing oligonucleotides, as well as one pending patent application covering additional applications of CRNs.
  • Marina Biotech's CRN technology provides a direct means of developing highly potent and specific oligonucleotide-based therapeutics to target messenger RNAs or microRNAs, which connects disease pathways that are typically "undruggable" or "difficult to target."

Statistics:

  • The CRN-substituted miRNA antagonist achieved a 5-fold increase in AldoA, a downstream gene regulated by miR-122.
  • The increase in downstream gene targets Glycogen Synthase I (GYS1) and Solute Carrier Family 7 member 1 (SLC7A1) was also observed, with an average increase of 3.2-fold and 4.1-fold, respectively.
  • The CRN-substituted antagomir was dosed for three consecutive days at up to 50 mg/kg/day, with no body weight changes observed in rodents.
  • Marina Biotech's CRN patent estate consists of two issued patents and one pending patent application.

Sources:

  • Marina Biotech, Inc.
  • Comtex SmarTrend Alert (October 15, 2011)
  • Comtex News Network, Inc.
  • Marketwire, Inc.

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