SALT LAKE CITY, Jan. 17 /PRNewswire-USNewswire/ -- PC Project, a public
charity, announced that a Phase 1b clinical trial began today 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.
"We are pleased to be part of an effort to develop treatments based on
our discovery in 1995 of the defective genes underlying PC. Although many of
the gene mutations causing skin disorders have been identified, until now
there has been a lack of effective therapy based on the genetic discoveries
that we and others have made," says Professor Irwin McLean (University of
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."
PC Project is a 501(c)(3) public charity dedicated to helping patients
suffering from pachyonychia congenita. Says Ms. Schwartz, "This project is a
remarkable collaboration between a lay public charity, industry, and academia
working hand in hand." Contributors include the University of Utah (Sancy
Leachman), University of Dundee (W.H. Irwin McLean and Frances Smith),
Stanford University (Christopher Contag), Yale University (Leonard Milstone),
Baylor College of Medicine (Dennis Roop now at University of Colorado),
University of Saskatchewan (Peter Hull), BIOPOLIS/Singapore (E. Birgitte Lane)
and University of Iowa (Rolland Poust). Industry partners include TransDerm
(Roger Kaspar and Robyn Hickerson), Thermo Fisher Scientific/Dharmacon (Devin
Leake), Elixin Pharma (Susan Srivatsa), Preclinsight (Doug Kornbrust),
Visionary Therapeutics Corporation (Steve Hutcherson), Arena Pharmaceuticals
(Ajit Simh) , Agilent Technologies, Nucleic Acid Solutions Division (James
Powell), Accugent (Ming Fai Chan) and GeneDx (Sherri Bale).
For further information:
For further information: Mary Schwartz, Pachyonychia Project Director,
+1-877 628-7300, firstname.lastname@example.org Web Site: