Shire Adds to Genetic Disorder Drugs with U.S. Deal

08.01.2013 -

British drugmaker Shire has agreed to buy Lotus Tissue Repair, a U.S. biotechnology company developing the first treatment for a rare genetic disorder that causes extremely fragile skin and recurrent blister formation.

Shire said on Tuesday the purchase, for an undisclosed sum, would boost the pipeline of its Human Genetic Therapies unit, which makes protein therapies for rare genetic conditions like Hunter's Syndrome and Fabry and Gaucher diseases.

Cambridge, MA-based Lotus Tissue Repair's lead product candidate, which is in late pre-clinical development, is a protein replacement therapy for the treatment of dystrophic epidermolysis bullosa (DEB), Shire said.

"DEB ... (impacts) the lives of patients and their families, many of whom have few or no treatment options other than palliative care," said Philip J. Vickers, head of R&D at Shire Human Genetic Therapies.

He said protein replacement therapy had the potential to provide a first-in-class disease-modifying treatment for children with DEB.