MDRNA, Inc, a leading RNAi-based drug discovery and development company, reported substantial advances in the activity of UsiRNA constructs delivered by its DiLA2 delivery technology resulting in 50 per cent knockdown of Factor VII (FVII) protein at a 80 microgram per kilogram dose via systemic delivery. The company also announced an additional early collaborative effort with a RNA-based therapeutics company focused on microRNA (miRNA)-directed oncology therapies. This adds to MDRNA's existing on-going collaborations and represents the fifth early effort announced in the last nine months. This was reported by J Michael French, president and CEO of MDRNA, at the 2010 BIO International Conference in Chicago, Illinois.
Effective delivery is a well recognized hurdle in the development of RNAi-based therapeutics, and MDRNA has been a leader in this area with its proprietary Di-Alkylated Amino Acid (DiLA2) delivery systems. The versatility of the DiLA2 system provides for a rapid and scientifically robust process for improving the delivery characteristics of its novel formulations to meet the specific requirements of a particular therapeutic application, i.e., administration via systemic versus local delivery or cellular uptake in liver hepatocytes versus lung epithelial cells. In addition, the team at MDRNA is constantly focused on the long term commercial needs of each DiLA2 formulation such as scale-up, manufacturing and cost-of-goods. In continuing to support on-going early collaborative efforts and the advancement of the DiLA2 delivery system, researchers at MDRNA identified novel DiLA2 formulations and process procedures that have improved the systemic delivery of UsiRNA at least five-fold. Using a mouse model and a UsiRNA targeting FVII mRNA in liver (hepatocytes), a single systemically delivered dose of 0.08 mg/kg (80 micrograms per kilogram) provided 50% inhibition of FVII protein activity.
"These results represent rapid advancement in our DiLA2 technology and demonstrate the tremendous potential of this platform for systemic delivery," stated Barry Polisky, chief scientific officer of MDRNA, Inc. "Given the modular nature of DiLA2, the extensive chemical space for this platform, and our robust research efforts into manufacturing procedures, we expect significant near-term advances in the development of our proprietary delivery formulations for RNAi-based therapeutics."
The new collaborative effort announced will utilize the broad capabilities of MDRNA's proprietary discovery engine for RNAi therapeutics and its world-class research team and will focus on MDRNA's proprietary DiLA2 technology for delivery of proprietary miRNAs in experimental oncology models. Other details of the collaborations were not disclosed.
"The level of interest in our technology and the work of our world-class research team is a clear demonstration of the progress MDRNA has made in the past year. With a total of five on-going early collaborative efforts, our team is gaining tremendous experience in working with large international pharmaceutical companies," said Michael French, president and CEO of MDRNA. "In addition, our team is receiving significant exposure within the larger RNAi research community which we see as a huge opportunity for the company. We expect to be able to transition at least two of these early collaborative efforts into major R&D collaborations this year."
MDRNA has a broad intellectual property estate that encompasses four key RNAi technology platforms: siRNA constructs, chemistry, nucleic acid delivery, and gene targets.