Skip to main content
Premium Trial:

Request an Annual Quote

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

Premium

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.

The Scan

Sick Newborns Selected for WGS With Automated Pipeline

Researchers successfully prioritized infants with potential Mendelian conditions for whole-genome sequencing or rapid whole-genome sequencing, as they report in Genome Medicine.

Acne-Linked Loci Found Through GWAS Meta-Analysis

Researchers in the European Journal of Human Genetics find new and known acne vulgaris risk loci with a genome-wide association study and meta-analysis, highlighting hair follicle- and metabolic disease-related genes.

Retina Cell Loss Reversed by Prime Editing in Mouse Model of Retinitis Pigmentosa

A team from China turns to prime editing to correct a retinitis pigmentosa-causing mutation in the PDE6b gene in a mouse model of the progressive photoreceptor loss condition in the Journal of Experimental Medicine.

CRISPR Screens Reveal Heart Attack-Linked Gene

Researchers in PLOS Genetics have used CRISPR screens to home in on variants associated with coronary artery disease that affect vascular endothelial function.