Skin Fibrosis: Could RNAi Therapy Fill an Unmet Need?


By Staff Reports

(DGIwire) – Imagine skin around a healed wound that feels so tight that flexing it becomes a painful chore. Sounds like a nightmare? It is a reality for those with skin fibrosis—a serious condition characterized by the inflammation and hardening of skin tissue, and ultimately tissue dysfunction, reports Scleroderma News. In the most severe cases, skin fibrosis can lead to debilitating pathologies of the skin, according to research published in the journal Endocrine, Metabolic & Immune Disorders – Drug Targets. Fibrotic tissue is like a scar tissue, thick and rigid, due to excess accumulation of protein below the skin, according to

“We have developed a therapeutic compound that targets connective tissue growth factor (CTGF), a key regulator of fibrosis,” says Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. “There are currently no FDA-approved therapies to treat hypertrophic scarring. Our groundbreaking research may lead to a treatment for this large underserved patient population but will also pave the way for potential treatment options for other fibrotic conditions.”

CTGF plays a key role in tissue regeneration and repair and in part controls scar tissue formation. It is involved in the differentiation of the main cells responsible for the deposition of collagen, a major structural protein of a scar. Elevated levels of CTGF can prolong the tissue repair process and lead to pathological scarring and fibrosis.

RXi Pharmaceuticals’ clinical compound, called RXI-109, is initially being developed to reduce fibrosis or scarring of the skin at a post-surgical wound site. Such scarring represents a high unmet medical need. If approved, RXI-109 would be a “first-in-class” RNAi treatment for the prevention or reduction of dermal scarring.

The development of RXI-109—specifically designed to reduce the expression of CTGF—builds on the research and discovery work of Nobel laureate Dr. Craig Mello, who serves as RXi Pharmaceuticals’ Scientific Advisory Board Chairman. Dr. Mello helped discover the phenomenon of RNA interference (RNAi for short) that works by destroying a gene’s messenger RNA before it can produce a protein. RNAi is the foundation of RXi’s novel therapeutic self-delivering RNAi platform, called sd-rxRNA®.

“RXI-109 is currently being evaluated in Phase 2 clinical trials for the treatment of hypertrophic scars after scar revision surgery,” adds Cauwenbergh. “It 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. We believe that success in the area of skin fibrosis may indicate major potential against fibrosis in the eye and possibly also internal organs such as the liver.”

Harnessing the power of RNAi to reduce fibrosis could offer new hope to many patients in the future.

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