$1.5 BILLION in potential milestones from Shire is no small potatoes (plus double digit royalties)!
The monogenic diseases will be VERY safe bets!: correct the gene and you correct the problem. With one important limitation!:
Depending on the tissues that the gene is expressed in and the delivery method used for the zinc fingers, the correction rate may not be 100%. (and with current knowledge is unlikely to be.) But it doesn't have to be for many monogenic diseases: For hemophilia, for example, it is only necessary for the gen expression to reach high single digits of percent for function to be returned to normal (thanks to the efficiency of the blood clotting cascade). So ZFs will have not problem attaining high levels of efficacy (as demonstrated in the mouse model), which is why four of the genes chosen were involved in the blood clotting pathway.
It will be interesting to see what other three monogenetic diseases Shire intends to go after (my hope is for sickle cell to be one of them. But that's very unlikely: 1) there would be no selection factor in favor of the corrected blood cells because the stem cells producing the defective RBCs would be entirely unaffected by their higher loss rate 2)Killing all of the existing blood forming stem cells would be a serious undertaking (however if it's a life long CURE for the condition it might, nevertheless, be warranted. So ultimately it will be done but I suspect they'll go after easier targets first (plus the FDA would want to see more working ZF treatments before allowing something on the level of knocking out all of the blood forming stem cells).
IMHO this puts a new higher and very stable floor for SGMO at current levels (about 4.50 range). What SGMO now has is financing for four drug candidates and up to three more PLUS some MAJOR validation by a very big player.
Not to mention years of expenditures in cash on hand plus the increasing revenue streams from SIAL and DAS which should start picking up in the near future.
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