RNAi: Paving the Way to a New Class of Treatments

By Staff Reports

(DGIwire) – These are exciting times for gene therapy. As Business Insider recently reported, a biopharmaceutical company announced positive results from a late-stage clinical study of a drug for a rare nerve disorder, familial amyloid polyneuropathy. This drug, notes a report in STAT, harnesses a phenomenon called RNA interference (RNAi). This 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 a range of human diseases.

The STAT article profiles various attempts to overcome a major challenge: figuring out how to deliver RNAi therapies to the right cells safely and effectively. The delivery systems remain highly complex. While success to date has been found with delivery to the liver, work is ongoing to validate the use of RNAi in other organs. As STAT reports, one company exploring the field, RXi Pharmaceuticals, is pursuing RNAi in a broad range of diseases, from fibrosis to cancer.

“If we can expand the role of RNAi to other organ systems beyond the liver, the likelihood that RNAi could overtake antibodies in terms of importance for diseases of man, animals and even plants, is certainly there,” says Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. “It’ll just take work, like anything else.”

The company has created a “self-delivering RNAi” (sd-rxRNA®) therapeutic platform. For the last few years, the company has focused on scarring in the skin and eye. With recent positive clinical results from its platform in dermatology, combined with promising preclinical work from the company’s ongoing immuno-oncology program, the company’s development will now exclusively be focused on novel immuno-oncology therapies. The success of the studies has laid the foundation for the development of a new class of therapeutics and in the near term, RXi will focus on adoptive cell therapy (ACT) using sd-rxRNA therapeutic compounds to treat cancer.

“With a much-improved regulatory landscape for the development of cancer therapeutics, we believe that there is great potential for RNAi-based therapeutics to be a major player in the advancement of novel cancer treatments,” Dr. Geert Cauwenbergh adds.

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