Marina, Debiopharm team selects lead DiLA2 formulation for RNAi-based therapy to treat bladder cancer
The Marina/Debiopharm Research and Development (R&D) team has selected and confirmed the lead DiLA2 formulation for a RNAi-based therapy for the treatment of non-muscle invasive bladder cancer. This was announced by Marina Biotech, Inc., a leading oligonucleotide-based drug discovery and development company.
The team has also advanced the lead UsiRNA candidates towards in vivo evaluation to identify a candidate drug product for continued development. The joint R&D Team expects to select the lead candidate in early 2012. Including lead candidate selection, the company anticipates achieving several near-term research milestones in the first half of 2012.
“This is significant progress for our bladder cancer programme with Debiopharm SA,” stated Richard Ho, executive vice president, R&D, Marina Biotech. “As evidenced by the decision of the joint R&D team, the lead DiLA2 formulation that Marina Biotech brought to the collaboration does not only provide for efficient delivery but is appropriate for advancement into development. Furthermore, our UsiRNA construct provided highly active sequences against molecular targets selected for evaluation. We are pleased that the programme is going well and progressing rapidly towards development of a novel and much needed treatment for bladder cancer.”
Marina and Debiopharm SA announced the development and commercialization agreement in February of this year. Debiopharm SA has full responsibility for development and commercialization of any products and is paying up to $24 million based on predefined research and development milestones as well as royalties on the sale of products. Additionally, all Marina research costs for the bladder cancer program have been funded by Debiopharm SA since February 2011.
DiLA2 is a Marina Biotech proprietary delivery platform of novel synthetic di-alklylated amino acid compounds for liposomal delivery formulations. The versatility of the DiLA2 platform provides for a rapid and scientifically robust process for improving the delivery characteristics of novel formulations to meet specific requirements for a particular therapeutic application, i.e., administration via systemic or local delivery. In addition to research advancements, the development teams at Marina Biotech are focused on the long term commercial requirements for delivery of nucleic acid-based therapeutics such as scale-up, manufacturing, and cost-of-goods.
UNA are non-nucleotide, acyclic monomers which provide greater structural flexibility in an oligonucleotide strand. UsiRNA constructs, which are proprietary to Marina Biotech, are siRNA incorporating at least one UNA and are distinct from standard siRNA constructs. UsiRNAs are specifically designed to provide greater specificity for RNAi-based therapeutics. Substitution with UNA in the passenger strand (non-targeting strand) is intended to eliminate its participation in the RNAi process.
Marina Biotech is a biotechnology company focused on the development and commercialization of oligonucleotide-based therapeutics utilizing multiple mechanisms of action including RNA interference (RNAi) and messenger RNA translational blocking.