SMA gene therapy

Based upon initial talks with the FDA, the FDA believed the proof-of concept studies were adequate from Dr. Kaspar and Team's proposal that was sent to them and they did not require a pig model of the disease for testing to move into clinical trials.  What would be required are a few additional studies to determine how much virus for dosing, as the FDA requested a dose escalation trial.  Then, Dr. Kaspar and Team would have to perform a toxicology and biodistribution study in mice to answer whether the doses given are truly safe in mice, and where the virus goes after delivery.  I think we all agree that additional models, including the pig model are good things to test a therapeutic in, but the simple answer is, It is not necessary for Dr. Kaspar to get his gene therapy into clinic based on the first call he and his team had with FDA.  The Sophia's Cure Foundation will continue to fund the SMA gene therapy program from Nationwide Children's hospital/OSU based upon guidance from the  Food and Drug Administration and the requirements they  deem necessary to move 
towards application for human clinical trials.


I spoke with Dr Kasper a little more then a year ago and the work they are doing is so promising, he took Skys name and our information to hold on to, for when they would be ready to do trials, this does not mean he will be picked when the time comes, but he is on the list.