Pfizer takeover of CD47 blocking Co., page-39

It doesn't make sense. Clinical trials have demonstrated that gene therapy treatments can successfully produce high levels of micro-dystrophin in DMD patients but are unable to raise the bar on functional outcomes. Now we find the FDA is asking a please explain in relation to the use of high dosage rates of AAV vectors administered in GT clinical trials. Genetic investigational treatments for DMD are certainly in the frame. As previously noted, three of the highest doses of AAV vectors in a table of the seven highest dosages in different clinical trials, are in DMD.

Investors have been interpreting results to date through rose coloured glasses but seismic jolts throughout 2021 may shatter the illusion that gene therapy has come of age. From a parents perspective, you would have to ask the question what's the ****** point.

Meanwhile, Big Pharma who can afford to write off whole programs of non-functioning, struggling or dead-in-the-water clinical assets may soon ask whether its a whole deal safer and meaningful to modulate the expression of a patients own cells rather than augment gene expression through gene therapy. Certainly its too early to write off gene therapy but probes by the FDA into the use of high dosage rates of AAV vectors may send some programs back to the drawing board.