AGENDA : Systemic delivery of AAV for neuro-muscular gene therapy Meeting, December 15th 2010, Evry, France


Jean-Philippe François

A workshop on the systemic delivery of AAV for neuromuscular gene therapy will take place in Evry, France on 15th December 2010.
This one-day workshop begins with introductory talks covering intravascular AAV delivery in the context of neuromuscular diseases, an overview on practical aspects of preclinical studies, and IND filing for loco-regional muscular gene transfer.

Four round-table workshops will then follow, covering non-regulatory preclinical studies, regulatory toxicology and biodistribution, surgical potential side-effects and local toxicity, and immune response and immunotoxicology.

The final workshop presentation will be delivered by Thomas Voit and is entitled “The MD’s point of view: Is the preclinical file supportive enough in terms of safety, risk-benefit and efficacy. Are the animal models and designs the best for supporting the drug development (phase I and further)?”

Representatives from Italian, French and European regulatory agencies will also be present.

The workshop starts at 9:15 am and will end by 6:15 pm at Genocentre, Evry, France.

  • If you would like to participate, you are invited to contact O.-W. Merten from Généthon – Tel: 00 33 1 69 47 25 90