Hi TB,The ADOA gene affects mitochondrial function. Increasing...

Hi TB,

The ADOA gene affects mitochondrial function. Increasing the production of the protein with our drug (hopefully) increases mitochondrial function. Mitochondria are responsible for energy production in the cell.

PYC wonder if improving mitochondrial function in some other conditions that have poor mitochondrial function and are running out of energy for other reasons may help. A good hypothesis I think but not a direct link like replacing the gene.

Just out of interest there are many conditions not in the eye where poor mitochondrial function exists not related to a direct mutation in the gene. For example Ataxia Telagectasia a brain condition has a complex pathway that results in poor mitochondrial function. I’m not really suggesting PYC will try to specifically target this condition but just a poorly evidenced general opinion there may be other applications for this drug than the ones already touted.

This is potentially a different approach to what we have previously had with one gene one drug one market. This one drug could potentially target multiple conditions. Pretty interesting and hence all the talk about multiple phase II trials. There are some downsides. We would lose our high probability of success approach. We no longer have a single gene mutation, we may not have the organ in a dish, I don’t think we will get orphan status but the glaucoma market is huge and inadequately treated.

Just some late night musings after one too many gin and tonics.

PS did I hear him say there were around 6000 similar single gene mutation diseases somewhere in that talk? Did I hear that wrong? That’s a long list to work through.

Eagle.