RNA Interference: Nobel-Winning Science Coming to Fruition

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(DGIwire) – When Craig C. Mello and Andrew Z. Fire were jointly awarded the Nobel Prize in Physiology and Medicine in 2006, the phenomenon for which they were honored—RNA interference (RNAi)—had already been making headlines for years since its initial discovery in 1998. RNAi is a naturally occurring process by which a particular “messenger RNA” can be destroyed before it is translated into protein. Since the overexpression of certain proteins plays a role in many disease conditions, the ability to inhibit gene expression with RNAi provides a potentially powerful tool to treat human disease.

A decade later, RNAi research is more robust than ever, with the first drug using the technology to inhibit disease-causing genes potentially being approved as early as 2017, according to STAT. And an overview of RNAi in The Scientist reveals its promise following refinements in RNAi targeting and delivery; in addition to a rising number of early clinical successes, the therapy is showing promise in the treatment of everything from viral infections to cancer.

“The sheer versatility of RNA in the clinical realm means it is likely to make a significant impact on human medicine,” says Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. “It is broadly possible to use RNAi to silence any overexpressed gene known to be responsible for a disease or condition.”

RNAi is the foundation of RXi’s novel therapeutic self-delivering RNAi platform, called sd-rxRNA®. Specifically designed sd-rxRNA therapeutic compounds have been developed to reduce the expression of connective tissue growth factor (CTGF), a critical regulator of hypertrophic scar formation. The company’s first clinical candidate, RXI-109, is currently in a Phase 2 clinical trial to reduce the formation of hypertrophic scars following scar revision surgery. RXI-109 has been shown to be safe and well tolerated in all trials to date, and, preliminary results have indicated an improved scar appearance with RXI-109 treatment.

Additionally, RXi has initiated a Phase 1/2 clinical trial to evaluate treatment with RXI-109 in patients with subretinal scarring associated with advanced wet age-related macular degeneration. The primary objective is to evaluate the safety, tolerability and pharmacokinetics of single and multiple doses of RXI-109 when administered by intravitreal injection. The overarching objective of this clinical research is to evaluate the potential of RXI-109 to block the formation of further subretinal scarring in these patients, with the goal of maintaining vision for a longer period of time than with anti-VEGF treatment alone, the current standard of care.

“What began as a remarkable discovery in 1998 and garnered the Nobel Prize in 2006 has met with repeated successes in clinical studies and is moving ever forward in the quest to help patients who could benefit from a variety of new therapies,” Dr. Cauwenbergh adds.

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