Biotech Values

[genisi]

What are your thoughts on substrate reduction therapies? Or do you think ERT is the best approach for the foreseeable future?

ERT, leaves little room for improvement in safety or efficacy and will likely remain the gold-standard treatment for Gaucher.
As for substrate reduction therapies, it depends on the substrate that accumulates and to a greater extent, on its inhibitor, especially its specificity (the higher it is, the fewer off-target side effects). Zavesca, as you know, is a poor drug: it has lower efficacy than Cerezyme and a high rate of SE, particularly GI and neurologic toxicities. Genz-112638 has a higher specificity to the substrate and is a lot more potent than Zavesca and is almost as active as Cerezyme. However, in order to be a competitive threat to ERTs, its tox profile should be clean but its cardiac safety is still unclear.
My thoughts were and still are, that chaperones will not deliver and that long-term, Genz-112638 might change the current gold-standard treatment or at least be used as a maintenance therapy following ERT.