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Pharmaceutical industry

Marina Biotech Publishes Data on DiLA2-Based Delivery Technology for Systemic siRNA Administration

Strategic Insights

— 1 min read

Marina Biotech, a leading RNAi-based drug discovery and development company, has announced the publication of data highlighting the application of their proprietary DiLA2-based delivery technology for systemic administration of siRNAs. The research, published in Molecular Therapy, describes the development of an amphoteric DiLA2 delivery system that enables efficient knockdown of multiple targets in liver. The technology has significant implications for the advancement of RNAi-based therapeutics into the clinic.

Key Takeaways:

  • The DiLA2 delivery system enables efficient knockdown of multiple targets in liver, including ApoB, TTR, Factor VII, and PCSK9 messenger RNA (mRNA).
  • The amphoteric formulation of DiLA2 resulted in greater than 80% knockdown of the mentioned mRNAs with a single 2 mg/kg dose.
  • The ED50 values for knockdown of these mRNAs ranged from 0.1 to 0.25 mg/kg, indicating the efficacy and specificity of the DiLA2 technology.
  • The DiLA2-based formulations demonstrated physical and chemical stability for greater than one year when stored frozen, a necessary attribute for developing liposomal-based siRNA drug products.
  • Marina Biotech has entered an exclusive agreement with Debiopharm Group for the development and commercialization of the Company's bladder cancer program, which utilizes a DiLA2-based delivery technology.
  • The DiLA2 platform provides a vast and chemically diverse library of compounds that can be tailored to meet particular siRNA delivery objectives for the treatment of various diseases.

Statistics:

  • The DiLA2 delivery system resulted in greater than 80% knockdown of ApoB, TTR, Factor VII, and PCSK9 mRNAs with a single 2 mg/kg dose.
  • The ED50 values for knockdown of these mRNAs ranged from 0.1 to 0.25 mg/kg.
  • The DiLA2-based formulations demonstrated physical and chemical stability for greater than one year when stored frozen.

Sources:

  • Adami et al., "An Amino Acid-based Amphoteric Liposomal Delivery System for Systemic Administration of siRNA", Molecular Therapy advance online publication 19 April 2011 doi: 10.1038/mt.2011.56
  • Marina Biotech Press Release, "Marina Biotech Announces Publication of Data Highlighting the Application of Its Proprietary DiLA2-Based Delivery Technology for Systemic Administration of siRNAs", April 26, 2011

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