MyoDys®

A Gene Therapy Product Candidate for Muscular Dystrophy

Medical Need

Muscular dystrophy encompasses a family of diseases associated with loss of muscle function. The most common of these are Duchenne and Becker muscular dystrophies, affecting roughly 1 of every 3500 male births. Patients with these diseases suffer from a genetic defect in which dystrophin protein is either absent or inadequate in their muscle cells. It is a lethal X-linked inherited disorder, caused by deletions or mutations in the dystrophin gene. Patients with this disease suffer from progressive and diffusive wasting, and death typically occurs by the third decade of life.

Product Description and Rationale

MyoDys® is comprised of plasmid DNA encoding the full-length human dystrophin gene. Mirus’ Pathway IV ™ delivery technology is used to administer the pDNA to a patient’s limb skeletal muscles. The clinical objective is to delay or prevent loss of muscle function, initially in the hand and forearm, thereby enhancing the patient’s quality of life.

Partners

This product is being developed with Transgene S.A. of Strasbourg, France as part of a strategic alliance to develop gene therapies for muscular dystrophy.

In addition, selected aspects of the research have been supported directly or indirectly by the Muscular Dystrophy Association (MDA) and the Association Francaise contre les Myopathies (AFM).

Development Status

A Phase I study has been completed (data released June 5, 2003 by Transgene) which has shown safety and dystrophin expression in skeletal muscle in Duchenne and Becker patients using an alternative delivery approach. A Phase I/II study is now in preparation using Pathway IV ™ that will provide regional delivery to forearm muscles.