Thanks for that, Jack. Something tells me you are WAY more of an expert than you are giving yourself credit for. You naturally have an MD's mindset it seems, IMO.
Maybe Buerger's Disease would have been the ticket. Taking the 'ultra-orphan' route in rare diseases has commanded drug prices of over $1M per patient/annually. Eleprase, etc. in the enzyme replacement market.
In the case of ALXN and PNH, TX cost is well north of 500K/PT/Year...
Coulda, Should, Would, but the 3D bet is yet to be covered.
I do fear the loss of any head-start in this space.