Engineering Biology: From a Concept Idea to Developing a New Drug

RNA based approaches have greatly contributed to better understanding of gene expression and function in vitro. The capability to apply these strategies in vivo in order to validate the role of specific genes in normal or pathological conditions, and to induce therapeutic gene silencing or upregulate a specific protein expression, opened new avenues for utilizing RNA as a novel therapeutic modality. However, the translation of RNA from an effective genomic tool into a novel therapeutic modality has been hindered by the difficulty to deliver RNA molecules into specific target tissues by systemic administration, especially to hematopoietic cells and highly metastatic tumors such as brain tumors and ovarian cancers. Here, I will describe some of the challenges and opportunities in modulating leukocytes response using RNA molecules and discuss adverse effects such as immuno-toxicity. In addition, I will detail the challenges of targeting lipid-based nanoparticles directly into specific cells. Special emphasize will be made on delivery strategies that target lymphocytes in inflammatory bowel diseases and iin several types of B cell hematological malignancies such as Mantle Cell Lymphoma (MCL) with novel therapeutic targets that were recently identified.

Finally, if time permits I will describe the translation of some of these strategies into clinical testing including CMC, process development and scale up.