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news.MDRNA's delivery system formulation has demonstrated stability over a one year period under a variety of conditions. The DiLA2 formulation maintained in-vivo knockdown activity with no observed loss in potency over the course of the year-long study when stored under conditions ranging from -80 oC to 4 oC.
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In the study, neither change in particle characteristics such as size or charge nor loss in siRNA integrity was observed. The company’s proprietary UsiRNA constructs in the DiLA2 formulations have demonstrated positive activity for delivery to hepatocytes in rodent and non-human primates.
In addition, these compounds have demonstrated inhibition of mRNA via RNAi and subsequent reduction in tumor burden in the company’s oncology programs in liver and bladder cancer. Long-term stability of the DiLA2 formulation/UsiRNA cargo is an integral part of the development of the company’s drug products and represents further progress in these programs.
DiLA2 delivery platform is MDRNA’s proprietary platform for creating new liposomal delivery systems based on di-alkylated amino acids (DiLA2). The DiLA2 platform enables MDRNA to tailor the charge, linker length, and acyl chain characteristics to improve delivery of the liposomes to target tissue of interest.
In-vivo studies have demonstrated effective delivery in models of metabolic disorders, cancer, and other diseases. DiLA2 based liposomes are well tolerated for repeat dose, and systemic and local administration. MDRNA is also utilising condensing peptides to form peptide-siRNA nanoparticles to further increase the delivery efficiency of its DiLA2 delivery systems.
Barry Polisky, chief scientific officer at MDRNA, said: “The ability of our novel DiLA2 formulation to be stored frozen or at refrigerated conditions is unique. We have the flexibility to tailor storage conditions to meet the needs of our internal programs, pharma partners and ultimately the commercial requirements of a marketed RNAi-based therapeutic.”