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.

Customers might want to consider what they might learn about their risk of diseases like Alzheimer's before snagging the genetic testing kits that are on many gift guides this year, NJ.com writes.

The Wall Street Journal reports there is uncertainty surrounding whether He Jiankui's embryo editing did what he said it did.

Stat News reports that the pause on procuring fetal tissue for intramural US National Institutes of Health research will soon affect additional labs there.

In Nature this week: genomic analysis of the invasive fall webworm, amp of constrained coding regions within the human genome, and more.