Marina Biotech Announces Significant Knockdown of Gene Targets in Tumors with a microRNA Mimetic
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Marina Biotech, Inc. has reported results from in vivo studies in rodent cancer models focused on effective delivery of a microRNA (miRNA) mimetic using Marina Biotech's proprietary Di-Alkylated Amino Acid (DiLA2) delivery system.
Organ and tumor distribution studies demonstrated up to a 100-fold increase in miRNA copies per tumor cell as compared to baseline levels. Similar increases in the miRNA levels were noted in liver, lung, and heart after systemic administration of the mimetic formulated in DiLA2-based liposomes. Moreover, 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.
"These data highlight the significant breadth in the potential uses of Marina Biotech's DiLA2 delivery system for RNA-based therapeutics," said J. Michael French, President and CEO of Marina Biotech at the Rodman & Renshaw 12th Annual Healthcare Conference in New York City. "The results support our plans to expand our therapeutic reach beyond our proprietary UsiRNA-based therapies to include both microRNA mimetics and antagomirs. Our broad delivery capability which includes both the DiLA2 and SMARTICLES(R) technologies combined with our conformationally-restricted nucleotide (CRN) technology for stabilizing single-stranded oligonucleotides, creates a formidable drug discovery engine for the development of miRNA-based therapeutics."
"These experiments further demonstrate the flexibility of our DiLA2-based delivery technology for systemic administration," stated Barry Polisky, Ph.D., Chief Scientific Officer of Marina Biotech, Inc. "The 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. This is very exciting in the context of our ongoing efforts to develop RNA-based molecules that regulate the protein production of specific therapeutic targets involved in a host of diseases, including cancer."
Effective delivery is a well-recognized challenge in the development of RNA-based therapeutics. The versatility of the DiLA2 system provides for a rapid and scientifically robust process for improving the delivery characteristics of novel formulations to meet specific requirements of a particular therapeutic application, i.e., systemic administration for delivery to liver hepatocytes or local delivery to cells, such as the lung epithelium.
Organ and tumor distribution studies demonstrated up to a 100-fold increase in miRNA copies per tumor cell as compared to baseline levels. Similar increases in the miRNA levels were noted in liver, lung, and heart after systemic administration of the mimetic formulated in DiLA2-based liposomes. Moreover, 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.
"These data highlight the significant breadth in the potential uses of Marina Biotech's DiLA2 delivery system for RNA-based therapeutics," said J. Michael French, President and CEO of Marina Biotech at the Rodman & Renshaw 12th Annual Healthcare Conference in New York City. "The results support our plans to expand our therapeutic reach beyond our proprietary UsiRNA-based therapies to include both microRNA mimetics and antagomirs. Our broad delivery capability which includes both the DiLA2 and SMARTICLES(R) technologies combined with our conformationally-restricted nucleotide (CRN) technology for stabilizing single-stranded oligonucleotides, creates a formidable drug discovery engine for the development of miRNA-based therapeutics."
"These experiments further demonstrate the flexibility of our DiLA2-based delivery technology for systemic administration," stated Barry Polisky, Ph.D., Chief Scientific Officer of Marina Biotech, Inc. "The 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. This is very exciting in the context of our ongoing efforts to develop RNA-based molecules that regulate the protein production of specific therapeutic targets involved in a host of diseases, including cancer."
Effective delivery is a well-recognized challenge in the development of RNA-based therapeutics. The versatility of the DiLA2 system provides for a rapid and scientifically robust process for improving the delivery characteristics of novel formulations to meet specific requirements of a particular therapeutic application, i.e., systemic administration for delivery to liver hepatocytes or local delivery to cells, such as the lung epithelium.
