Biotech Values

[iwfal]

AGTC abandoned the Leber Congenital Amaurosis program for business reasons, according to the S-1/A filing prior to the Mar 2014 IPO:

I am suspicious that that is a convenient excuse:

1) As best I can tell in a quick search the academic gene therapies still do not appear to be widely used even now (7 years later). But it may just be hard to find?

2) The S1 indicates that they had the same problems as others - e.g. primarily off fovea improvement, sporadic, ... . And, worse, the long term followup from other trials indicates it isn't permanent. See here: http://genetics.med.harvard.edu/cepko/wp-content/uploads/publications/Hum.%20Gene%20Ther.%202013%20Cepko.pdf

BTW - the link I gave below is, I would suggest, a substantial concern for gene therapies in general that want to rescue dying cells.