RXi Pharmaceuticals is a discovery-stage biopharmaceutical company engaged in the discovery, development and commercialization of proprietary therapeutics based on RNA interference (RNAi) for the treatment of human diseases. RNAi is a naturally occurring mechanism for the regulation of gene expression that has the potential to be harnessed to selectively inhibit the activity of any human gene. It is believed that this inhibition may potentially treat human diseases by “turning-off” genes that lead to disease.
RXi’s founders include world-reknowned researchers in the RNAi field, including Dr. Craig Mello, recipient of the 2006 Nobel Prize for Medicine for his co-discovery of RNAi and Blais University Chair of Molecular Medicine at the University of Massachusetts Medical School. RXi’s management team has extensive experience in the field of RNA interference and in advancing RNA/DNA based drugs into the clinic.
The rxRNA™ compounds developed by RXi are distinct from traditional small interfering RNA (siRNA) compounds widely used by other companies in the RNAi field. RXi’s unique compounds are highly active and potent, demonstrate increased nuclease stability, show reduced toxicity levels and are readily manufactured.
Many genes associated with disease have been identified, but few have been able to be targeted with conventional modalities, such as small molecules and antibodies. RXi believes that RNAi technology may play an important role in targeting these “undruggable” genes and potentially treating a range of diseases that could otherwise remain untreated. With our pioneering work in developing the RXi technology platform, the company believes that it will discover many drug candidates than can be developed as therapeutics. RXi is seeking to advance therapeutic candidates into the clinic using a combination of internal therapeutic development effort, as well as working with partners in the discovery and development process.
The distribution of RNAi compounds into the right tissues of the body is essential for RNAi therapeutics to be effective. RXi is using various technologies to deliver its rxRNA compounds to the appropriate tissues and cells, including the proprietary sd-rxRNA™ (or "self-delivering" RNA) which requires no additional formulation to access cells and tissues.
Our Competitive Strengths
We believe we are well positioned to compete successfully in the RNAi-based therapeutics market due to the following competitive strengths:
- Novel, proprietary therapeutic platform with the potential to generate multiple RNAi therapeutic product opportunities, comprised of:
- Key therapeutic target genes
- Our portfolio of next generation RNAi molecules that include sd-rxRNA compounds, which have the unique property of entering cells and tissues to effect silencing without the need for any additional delivery vehicle, as well as multiple novel RNAi structures, all of which offer distinct approaches to RNAi based therapeutic development
- Accomplished business team with significant experience in RNAi therapeutics and in building and managing emerging life science companies
- Scientific advisors who are recognized leaders in RNAi research, including Dr. Craig Mello, recipient of the 2006 Nobel Prize in Medicine for co-discovering RNAi, and Dr. Victor Ambros, who was awarded the 2008 Albert Lasker Award for Basic Medical Research for his work leading to the groundbreaking discovery of the first microRNA (miRNA)
- Strong, broad and early intellectual property position encompassing:
- Novel approaches to RNAi chemistry and configuration
- Proprietary formulation and delivery of active RNAi compounds
- Key sequences targeting therapeutically important genes
- A focus on unmet medical needs targeting both local and systemic applications
