Marina Biotech Reports Positive Results from In Vivo Studies on miRNA Delivery

Marina Biotech, Inc., a leading RNA-based drug discovery and development company, has announced results from in vivo studies on the effective delivery of a microRNA (miRNA) mimetic using the company's proprietary Di-Alkylated Amino Acid (DiLA2) delivery system. The studies demonstrated a significant increase in miRNA copies per tumor cell, as well as in other organs and tissues after systemic administration of the mimetic formulated in DiLA2-based liposomes. These results highlight the potential of Marina Biotech's DiLA2 delivery system for RNA-based therapeutics and support the company's plans to expand its therapeutic reach beyond its proprietary UsiRNA-based therapies.

Key Takeaways:

• Organ and tumor distribution studies showed up to a 100-fold increase in miRNA copies per tumor cell compared to baseline levels.
• Similar increases in miRNA levels were noted in liver, lung, and heart after systemic administration of the mimetic formulated in DiLA2-based liposomes.
• Delivery of the miRNA mimetic in DiLA2 liposomes demonstrated approximately 60% knockdown of mRNA for two genes whose down-regulation is the intended target of the miRNA mimetic.
• Marina Biotech's DiLA2 delivery system is capable of efficiently delivering small RNAs into cells and releasing their cargo to affect miRNA-mediated pathways or siRNA-mediated knockdown of a gene target.
• Effective delivery is a well-recognized challenge in the development of RNA-based therapeutics, and the versatility of the DiLA2 system provides a rapid and scientifically robust process for improving the delivery characteristics of novel formulations.
• Marina Biotech's proprietary DiLA2 RNA delivery platform enables the company to tailor the charge, linker, and acyl chains of amino acids to configure liposomes for delivery to target tissues of interest.

Statistics:

• 100: The fold increase in miRNA copies per tumor cell observed in organ and tumor distribution studies.
• 60: The percentage of knockdown of mRNA for two genes whose down-regulation is the intended target of the miRNA mimetic.
• 2: The number of genes whose down-regulation is the intended target of the miRNA mimetic.
• 3: The number of organs and tissues (liver, lung, and heart) where similar increases in miRNA levels were noted after systemic administration of the mimetic formulated in DiLA2-based liposomes.

Sources:

• MARINA BIOTECH, INC., Press Release, "Marina Biotech Announces Positive Results from In Vivo Studies on miRNA Delivery" (September 13, 2010).
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