Date: 2011-03-08
Type of information: Collaboration agreement
Compound: gene therapy for myotubular myopathy
Company: Genethon (France) Wake Forest University School of Medicine (USA)
Therapeutic area: Rare diseases - Neuromuscular diseases
Type agreement: collaboration
Action mechanism:
Disease: myotubular myopathy
Details: Généthon and Wake Forest University School of Medicine are to collaborate on the preclinical development of a gene therapy for myotubular myopathy. Généthon/INSERM researcher Dr Anna Buj Bello has been working on murine models of myotubular myopathy for many years, in order to understand the disease mechanism and design therapeutic strategies. Her research group has developed an adenoassociated virus (AAV) vector for transferring a healthy copy of the myotubularin gene into muscle cells. The collaborative work will test this approach in a canine disease model characterized by Martin Childers, D.O., Ph.D., a professor in the Department of Neurology and the Institute for Regenerative Medicine at Wake Forest. Généthon will transfer its method for local-regional perfusion of AAV vectors, supply expert advice and produce the batches of vectors to be used in the work in the United States.
This transatlantic collaboration will help the researchers to understand the mechanisms involved in the disease and complement the data generated in mice. If the results are confirmed in dogs, the therapy could then be clinically tested in humans.
Financial terms:
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