Therapeutic Platform
RXi’s comprehensive therapeutic platform, which includes both RNAi compounds and delivery methods, has the potential to be broadly applicable to multiple therapeutic areas and indications. This novel, proprietary platform consists of:
- Novel RNAi compounds, referred to as rxRNA™ compounds, that are distinct from, and we believe convey significant advantages over, classic siRNA (conventionally-designed “small interfering RNA” compounds), and offer many of the properties that we believe are important to the clinical development of RNAi-based drugs. We have developed a number of unique forms of rxRNA compounds, all of which have been shown to be highly potent. These RNAi compounds include rxRNAori™, rxRNAsolo™ and sd-rxRNA™, or “self delivering” RNA. Based on our research, we believe that these different, novel siRNA configurations have various advantages for therapeutic use. These advantages include high potency, increased resistance to nucleases and off-target effects, and in the case of the sd-rxRNA compounds, access to cells and tissues with no additional formulation required.
- Advanced delivery technologies that may enable the delivery of our rxRNA compounds to treat a variety of acute and chronic diseases using both local and systemic approaches, potentially providing a competitive advantage in the development of many RNAi therapeutic compounds. RXi's suite of delivery technologies is comprised of delivery vehicles which can be combined with various rxRNA compounds, as well as sd-rxRNA compounds, which are chemically modified and have the unique property of entering cells and tissues to effect silencing without the need for any additional vehicle. This suite of delivery technologies has broad potential applications for multiple therapeutic areas targeting both local and systemic applications.
RXi intends to utilize its comprehensive RNAi therapeutic platform and expertise in RNAi to identify lead compounds against multiple target genes, and progress them towards pre-clinical and clinical development in therapeutic areas that address broad unmet medical needs, in both acute and chronic settings. RXi’s therapeutic strategy is to advance a number of these product opportunities internally, while developing others through external collaborations and partnerships.
