Both Pfizer and Sarepta are starting to perform "sub group...

Both Pfizer and Sarepta are starting to perform "sub group analysis" - that old trick! @Uboy

One of the pillars of gene therapy was its universality. As you have indicated it looks like that particular pillar has been effectively discarded by Pfizer and Sarepta. Safety fears have led Pfizer to scale back their program excluding patients with certain genetic mutations. I notice that Sarepta have similarly inserted a mutation specific exclusion criteria for the upcoming EMBARK protocol for SRP-9001, excluding individuals with mutations within exons 1-17 as well as exon 45.

One of the rabbit holes I have disappeared into is Thrombospondin 1 (TSP-1). Again, I am harking back to the proteomics analysis but I do anticipate there will be a lot of harking coming up and not just to do with the herald angels at Christmas but with potential new applications for ATL1102. For instance did you know that TSP-1 is weakly expressed in the normal heart. However, my understanding is that patients with known cardiovascular conditions have elevated levels of TSP-1 that contributes to inflammation and fibrosis. According to the paper Thrombospondin 1 in Metabolic Diseases, TSP-1 largely contributes to the rapid remodelling of the extracellular matrix. One would think that regulating TSP-1 by a mean reduction of 49% would be a good thing. ATL1102 does it without even blinking because in binding to CD49d a powerfully beneficial chain reaction is set off that could have profound implications for myocarditis-related cardiomyopathy in DMD. And not just DMD. Think of myocarditis in the news. Think of reported incidences of myocarditis as one of the serious adverse events with the Pfizer mRNA vaccine. Did you know that like DMD, and according to the Australian Government guidance on mRNA Covid-19 vaccines, the risk of myocarditis and pericarditis following the vaccine occurs in males under 30 years of age.

Gene therapy in DMD is tottering on one less pillar with the others starting to crumble. Sure looks like it needs a bit of support.