Clinical Trial to Treat Rare Skin Disorder with siRNA Technology
News Jan 18, 2008
PC Project has began a Phase 1b clinical trial for treatment of pachyonychia congenita (PC) with a short interfering RNA (siRNA)-based therapeutic. PC is a dominant genetic disorder resulting from mutations in genes that encode keratins, which give structural integrity to skin cells.
Dr. Sancy Leachman, a University of Utah Dermatologist, is leading the study using a newly developed siRNA called TD101. This new drug has received the orphan drug designation (drug for rare diseases) by the US FDA under the Orphan Drug Act and was developed by TransDerm (Santa Cruz, CA) in collaboration with the International PC Consortium (IPCC), a group of physicians and scientists who agreed in 2004 to work together to develop therapeutics for this rare skin disorder.
Dr. Leachman, who heads the IPCC says, "Our siRNA formulation is designed to inhibit production of the mutated keratin so the normal gene can function correctly."
Treatments based on the powerful new technology are just now coming to clinic. Recent siRNA clinical trials include treatment of age-related macular degeneration (AMD) and respiratory syncytial virus (RSV). TD101 represents the first use of siRNA technology in skin as well as the first siRNA which targets a mutant gene.
Dr. Roger Kaspar, CEO of TransDerm, says, "We feel fortunate to be part of an unprecedented effort involving a group of dedicated physicians, scientists, consultants, advocates and patients in pursuit of a common goal. We hope this drug can help the few PC patients that have the targeted mutation and that we can extend what we have learned to larger numbers of patients suffering from other untreatable dominant genetic disorders."
As genome editing technologies advance toward clinical therapies, they are raising hopes of a completely new way to treat disease. However, challenges need to be addressed before potential treatments can be widely used in patients. To tackle these challenges, the National Institutes of Health has launched the Somatic Cell Genome Editing program, which has awarded multiple grants including more than $3.6 million to assess the safety of genome editing in human cells and tissues.