Where others have failed will Atl1102 succeed ?, page-87

It serves to demonstrate the naivety of the gene therapy approach that if we simply induce dystrophin production everything will normalise for boys with Duchenne @waynesworld

Lets draw the analysis out just a little more by referencing the disappointing interim results from the SRP-9001 clinical trial. Just to recap, the micro-dystrophin gene is being delivered into an already damaged muscle matrix and inflamed microenvironment. My understanding of the research by Dr Nagaraju is that gene therapy does result in newly formed better performing muscle. That is the good news. The bad news is that the therapy elicits an immune response that may potentially damage the new muscle. Nagaraju makes the observation that even if you correct dystrophin you dial up an immune response because the body has not seen it before. Could this explain the reason why SRP-9001 failed to demonstrate an improvement in muscle function despite a 28% increase in dystrophin?

All of this analysis is pointing towards the necessity to manage inflammation prior to and/or concurrently with gene therapy. Undoubtedly, corticosteroids have a role but the cell mediated response from circulating lymphocytes require a secondary treatment which is why ATL1102 is shouting from the rooftops, 'have you seen the results from the Phase II trial in DMD at the Royal Children's Hospital in Melbourne'.