RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced that the Company' Chief Scientific Officer, Anastasia Khvorova, Ph.D., presented new pre-clinical data on novel RNAi therapeutic approaches at IBC' TIDES Oligonucleotide and Peptide® Technology and Product Development Conference in Las Vegas, Nevada. This conference is one of the longest-running, most prestigious conferences on oligonucleotide-based therapeutics, a field of drug development that includes RNAi. The conference was not webcasted.
Regarded as a revolutionary discovery in biology, RNA interference (RNAi) is a naturally occurring mechanism whereby short, double-stranded RNA molecules interfere with the expression of genes in living cells. RXi' world-leading scientists have made significant advancements in this technology and Dr. Khvorova presented new forms of potential RNAi drugs with improved therapeutic properties.
Tod Woolf, Ph.D., President and CEO of RXi Pharmaceuticals commented: “Our research team is making significant progress, and I am very impressed with the amount of robust scientific data generated by our laboratory. The breakthrough technologies presented yesterday by Dr. Khvorova have the potential to address multiple multi-billion dollar therapeutic markets, may lead to improved therapeutics for many currently untreatable diseases and will help RXi realize its goal of becoming the technology leaders in the RNAi therapeutics space.”
RXi presented data on the following therapeutically advanced RNAi compounds and delivery technologies:
- Glucan Encapsulated RNAi Particles (GeRPS) – The GeRP technology delivers RNAi compounds directly to macrophages. RXi has been building on the work published in an April 30th Nature article reporting positive pre-clinical data demonstrating that administration of RNAi therapeutics effectively reduces systemic inflammatory response. RXi has brought the GeRP technology in-house, established manufacturing in its own laboratory and has advanced the technology to the level necessary for further pre-clinical development.
- Self-delivering RNA (sdRNA) – The proprietary self-delivering-rxRNA™ technology promotes spontaneous cellular uptake of the RNAi therapeutic, which is similar to the uptake of traditional small molecule drugs. Without the use of an additional delivery vehicle, this RNAi delivery technology potentially results in greater efficacy along with lower costs and side effects attributed to a delivery formulation. RXi believes that self-delivering rxRNA™ should support direct application or subcutaneous administration, opening the door for many clinical areas of RNAi therapeutic development.
- Advancements to rxRNA™ compounds – Improvements have been made to rxRNA™ compounds allowing them to work in concert with the new delivery technologies. Efficient cellular uptake is critical for an effective therapeutic and advancements have been made to minimize rxRNA™ compounds to improve delivery. These minimized compounds also reduce the immune response and are easier and less costly to manufacture. These advancements are proprietary to RXi and encompass novel and unconventional chemistries when compared to the current siRNA molecules.
The combination of these new advanced technologies may enable RXi to discover and develop drugs for multiple indications and to build a sustainable pipeline of products for unmet medical needs.