Transkaryotic Therapies files IND to conduct Phase I/II trial of enzyme replacement therapy
As a first step in developing a treatment for Hunter syndrome, TKT will conduct a Phase I/II clinical trial designed to assess the safety, pharmacokinetic profile, and clinical activity of I2S enzyme replacement therapy. Twelve patients with Hunter syndrome will be enrolled in the dose-escalating study and will be randomized to receive I2S or placebo for six months. Joseph Muenzer, associate professor of pediatrics at the University of North Carolina at Chapel Hill, is the principal investigator of the study. TKT expects to complete Phase I/II testing in approximately 1 year.
Hunter syndrome is an inherited rare genetic disorder caused by deficient activity of I2S. I2S is present in the lysosomes of cells, where it helps break down large biomolecules called glycosaminoglycans. Patients affected by Hunter syndrome suffer from a variety of symptoms including skeletal deformities, obstructive airway disease, cardiac failure, and, in severe cases, neurologic decline can occur. TKT estimates that approximately 5,000 patients worldwide are affected by Hunter syndrome. In severe cases, the life expectancy for patients with Hunter syndrome is typically 10-15 years of age, however, in mild cases, patients can survive into the fifth or sixth decade of life. Currently, there is no effective treatment.
Transkaryotic Therapies, Inc. (TKT) is a biopharmaceutical company dedicated to the development and commercialization of products based on its three proprietary development platforms: Gene-Activated proteins, Niche Protein products, and Gene Therapy. TKT's Niche Protein product platform is based on protein replacement for the treatment of rare genetic diseases, a group of disorders characterized by the absence of certain metabolic enzymes.
For more information, contact Justine E. Koenigsberg of Transkaryotic Therapies at 617-349-0271.
With contributions from Jim Pomager
Assistant Editor, Drug Discovery Online