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TransDerm Awarded $4M Grant to Develop RNAi Treatment for Skin Disease


TransDerm, a small firm developing RNAi-based treatments for rare skin disorders, said this week that it has been awarded a $4 million grant from the National Institutes of Health to develop a technology for siRNA delivery into the skin.

The technology, called the protrusion array device, involves dissolvable microneedle arrays that are designed to penetrate the skin and deliver siRNA painlessly into the non-innervated epidermis, according to the company.

TransDerm is primarily focused on treating pachyonychia congenita, a rare autosomal skin disorder caused by a mutation in any one of the dozens of genes encoding keratins (GSN 9/30/2005). It previously completed a one-patient clinical trial testing an RNAi treatment for the condition, but found the required injections to be too painful.

Last year, the company announced it had signed an agreement to examine the use of RXi Pharmaceuticals' self-delivering siRNA compounds in this program (GSN 4/8/2010).

“The combination of microneedles that allow transport across the outermost skin barrier and self-delivery siRNA modifications ... holds promise for delivery of these powerful gene inhibitors to the affected keratinocytes and improvement of PC symptoms,” TransDerm CEO Roger Kaspar said in a statement.

He noted that the company aims to begin a second phase I trial within the next three years.