pachyonychia congenita

TransDerm, a small biotech focused on developing a treatment for pachyonychia congenita (PC), has just kicked off a phase I trial of a new drug candidate designed to treat the rare skin condition.

TransDerm is preparing to begin the toxicology studies needed to launch a phase I trial of an improved version of its siRNA-based treatment for pachyonychia congenita, or PC, and expects the study to begin next year, Gene Silencing News has learned.

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.

In addition to evaluating RXi's proprietary RNAi compounds, TransDerm will test delivery technologies from Traversa Therapeutics, Thermo Fisher Scientific, and Life Technologies.

Additional clinical trials will likely use an experimental topical delivery vehicle after intradermal injections proved more painful to the patient than expected.

The US Food and Drug Administration has new guidelines that enable some gene and cell therapies to undergo expedited review, according to the New York Times.

Using gene drives to control invasive species might be too risky, an initial advocate of the approach says.

In Science this week: intellectual property experts argue patent battles such as the one over CRISPR are wasteful, and more.

Researchers have grown tumors in 3D cell cultures to better understand cancer, the Economist reports.