Expert: How Can ‘RNA Interference’ Enhance Drug Development?

According to recent reports, there is a renewed interest in pursuing therapies based on RNAi. Short for “RNA interference,” RNAi is a naturally occurring phenomenon by which short, double-stranded RNAs interfere with the expression of targeted genes. The development of therapeutics based on RNAi technology takes advantage of this phenomenon and potentially allows pharmaceutical companies to reduce the expression of particular genes within living cells. This, in turn, inhibits the activity of proteins that produce unwanted effects such as excessive scarring on the skin and in the eye.

One of the leading companies at the forefront of RNAi research is RXi Pharmaceuticals. Under its CEO, Dr. Geert Cauwenbergh, RXi Pharmaceuticals has developed a therapeutic platform of “self-delivering” RNAi compounds, called sd-rxRNA®. The first of these compounds, RXI-109, is in development to target connective tissue growth factor (CTGF), a key regulator of fibrosis and scar formation in the eye and skin.

The company believes the therapeutic reduction of CTGF could help reduce loss of vision associated with retinal scarring in conditions such as advanced wet age-related macular degeneration, and is conducting a Phase 1/2 clinical study to evaluate RXI-109 in this indication. Separately, the company is also conducting a Phase 2 clinical study to evaluate the potential of RXI-109 to reduce the production of hypertrophic (raised) scars after scar revision surgery. Hypertrophic scars are a result of too much collagen being produced at the site of a wound, a process that could be inhibited using RNAi.

Dr. Cauwenbergh believes that RNAi offers a novel approach to the drug development process because RNAi compounds can potentially be designed to target any one of the thousands of human genes, many of which are “undruggable” by other modalities.

Recently, RXi announced that it has entered into an option agreement to acquire MirImmune, a cell therapy and immuno-oncology biotech company that has been using RXi’s sd-rxRNA® platform in this exciting and potentially transformational field of medicine. MirImmune has generated evidence that shows the potential of using the sd-rxRNA platform to enhance cell therapy treatments for immuno-oncology.

More About Dr. Geert Cauwenbergh

Dr. Cauwenbergh has had a distinguished career in the pharmaceutical industry. He previously served as Chairman and Chief Executive Officer of Barrier Therapeutics, Inc., a publicly traded biopharmaceutical company he founded in 2001 that focused on dermatology drug development. Barrier was acquired by Stiefel Laboratories, Inc. in 2008. Prior to founding Barrier, Dr. Cauwenbergh held a number of ascending senior management positions at Johnson & Johnson, where he was employed for 23 years. As Vice President, Research and Development for Johnson & Johnson’s Skin Research Center, he was responsible for the worldwide research and development of all skin care products for the Johnson & Johnson consumer companies. He is a member of the board of directors of Phosphagenics and Moberg Pharmaceuticals. In 2005, Dr. Cauwenbergh was inducted into the New Jersey High-Tech Hall of Fame, and, from 2009 to 2010, he served as Chairman of the Board of Trustees of BioNJ. He has authored more than 100 publications and has been a guest editor for a number of books in mycology and infectious diseases. Dr. Cauwenbergh received his Doctorate in Medical Sciences from the Catholic University of Leuven, Faculty of Medicine (Belgium), where he also completed his masters and undergraduate work.


RNA interference, RNAi, drug development, RNA, RXi Pharmaceuticals, Geert Cauwenbergh, sd-rxRNA, RXI-109, connective tissue growth factor, CTGF, fibrosis, scar formation, retinal scarring, age-related macular degeneration, AMD, hypertrophic scar



Laura Radocaj

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