Collaborators will evaluate AAV-based gene therapy in canine disease model.

France’s Généthon is partnering with Wake Forest School of Medicine in North Carolina to carry out preclinical evaluation of an AAV vector-based gene therapy for the congenital disease myotubular myopathy. The candidate gene therapy has been developed by Généthon/INSERM researcher Anna Buj Bellow, Ph.D., who has specialized in research on myotubular myopathy using murine models of the disease.

The collaboration with Wake Forest aims to test the ability of the AAV vector to deliver a healthy copy of the myotubularin gene to muscle cells in a canine disease model characterized by Wake Forest researcher Martin Childers, Ph.D. The collaborators hope the work will not only generate new insights into the disease mechanism that build on previous murine research, but potentially lead to clinical trials in humans.

“Généthon has developed unique skills and know-how in the field of gene therapy for rare disases in general and neuromuscular diseases in particular,” remarks Dr. Childers, a professor at Wake Forest’s department of neurology and Institute for Regenerative Medicine. “For our research group, this collaboration with Généthon represents an opportunity to turn academic work into therapeutic reality.”

Généthon is a nonprofit laboratory that was created by the French Muscular Dystrophy Association (AFM) and is almost exclusively funded by the AFM through donations from France’s annual Telethon. Specializing in the preclinical and clinical development of gene therapies for rare diseases, Généthon is currently constructing a 5,000 m2 bioproduction facility, the Généthon BioProd, at Genopole (Evry, France), which is expected to open during early 2011. The new site will have the capacity to produce cGMP gene therapy vectors for Phase II clinical trials, and so address the manufacturing bottleneck which Généthon suggests is currently holding up the clinical development of innovative gene therapies.

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