Clinical Development

Delivery Systems for

Clinical Development


We are developing novel delivery systems to effectively deliver nucleic acid therapeutics in a clinical setting. Scaled-up synthesis procedures have been implemented to produce our transfection reagents in large quantities. We are committed to undertaking the required safety and toxicological studies to facilitate clinical testing of our delivery systems.
One area of focus of RJH Biosciences is to implement RNA interference (RNAi) via delivery of short interfering RNA. Our initial therapeutic application is blood cancers, while recognizing that the RNAi activity can be implemented in a large range of human cancers. Another focus is direct administration of plasmid DNA (pDNA) to express therapeutic proteins in situ, with applications in immunotherapy.
The R&D activity is at the preclinical stage and we are committed to undertaking the initial development of our therapeutic agents in select human disorders. We actively seek partners in blood cancer and immunotherapy areas for clinical collaborations.
For disorders beyond the focus of RJH Biosciences, we are open to working with pharmaceutical and biotechnology companies in other clinical indications. Our delivery systems were shown to be effective for a range of nucleic acids and we are confident that we can implement nucleic acid therapies in a range of diseases.
We have been adopting our delivery systems for treatment of patient-derived cells (in blood cancers) as well as preclinical animal models. The following publications provide a glimpse of our scientific studies with patient cells and animal models.
  • Valencia-Serna et al. siRNA-mediated BCR-ABL silencing in primary chronic myeloid leukemia cells with lipopolymers. J. Controlled Release (2019) 310: 141-154.
  • Thapa et al. Breathing new life into TRAIL for breast cancer therapy: co-delivery of pTRAIL and complementary siRNAs for breast cancer therapy using lipopolymers. Human Gene Therapy (2019) 30: 1531-1546
  • Valencia-Serna et al. siRNA/Polymer nanoparticles to arrest growth of chronic myeloid leukemia cells in vitro and in vivo. European J. Pharmaceutics and Biopharmaceutics (2018) 130: 66-70.
  • Landry et al. Targeting CXCR4/SDF-1 axis by lipopolymer complexes of siRNA in acute myeloid leukemia. J. Controlled Release (2016) 224: 8-21.
  • Aliabadi et al. Effect of siRNA pre-exposure on subsequent response to siRNA therapy. Pharm. Res. (2015) 32: 3813-3826.
  • Gul-Uludag et al. Nanoparticle-mediated silencing of CD44 receptor in CD34+ acute myeloid leukemia blasts. Leukemia Research (2014) 38: 1299–1308.