UPDATE ON PFIZER’S PHASE 1B OPEN-LABEL MINI-DYSTROPHIN GENE THERAPY TRIAL FOR DUCHENNE, page-8

Its appropriate to read the recent Research Report on ANP against the backdrop of the Pfizer update. Is the current approach to gene editing or gene therapy treatment simply a mirage?

Heavy on the science. It's what you would expect from a biotech interested in developing a therapeutic that could potentially work to ameliorate clinical symptoms of DMD. There are some big bold claims that will bear scrutiny and none more so than the statement that CD49d T-cells seem to ultimately be responsible for a lot of the clinical symptoms experienced by DMD patients. Think about it, dwell upon it and ponder the implications in regard to not only the mechanism of action for ATL1102 but also the hype around gene therapy. That's right, it says CD49d and not the lack of dystrophin. Are you beginning to see the mirage?

Its well to remember that boys are not struck down by DMD from birth. Its a progressive disease that manifests over time. Its also well to remember that micro-dystrophin or exon skipping for that matter, is not the dystrophin gene. So are we saying that DMD is a disease caused by excessive expression of CD49d. Not at all, the pathogenesis of the disease results from a defect in the dystrophin gene but the debilitating expression of the disease results from a chronic state of inflammation where the off switch malfunctions and fibrosis begins with the destructive process of fat replacing muscle.

The science is laid out. There is the opportunity to refute the science. There is also the opportunity to embrace the science. We stand at the threshold of the commencement for a pivotal trial for ATL1102 that could provide hope for DMD patients and their families.