Global Blood Therapeutics (NASDAQ:GBT)

In Longview CO in the 80's there was a bin of molasses that ruptured and flowed out over the streets in a 3-ft wall of goo that would have made for a good horror flick. That's what I picture after a biotech finally gets FDA approval. They are stuck in a morass, trying to move forward through the goo after the excitement of approval is over. I green-thumbed GBT before approval and patted myself on the back when it came through, but now they have to actually sell an $80,000/yr product over the debate on drug pricing. I'm hanging on to this ticker in real life because I think it is the best product right now for SCD, and my weakness is good science. Even if gene therapy eventually comes to fruition, this product can be used in conjunction for those individuals that are not completely cured. It may be a while before we see sales numbers that mean anything, and a more sensible person might take profits and wait to see sales numbers to buy back in. My rating is in the 20's, and I've not been accuse of being sensible lately. So why are you listening to me? Do your own DD.

potential big winner in treating blood cancers like acute myeloid leukemia (AML). The company is on track to report Phase 2 data from its AML program later this year.

In the meantime, however, the GBT partnership news is sending SYRS shares up sharply over the past month.

Syros’ therapies, like its AML drug called SY-1425, work by controlling how genes are expressed. In cancer, this could be a very valuable approach, since shutting down cancer genes could help create new treatments.

But Syros’ gene expression platform could be broadly applicable to other types of disease including sickle cell disease and beta thalassemia.

Global Blood Therapeutics wants to tap into Syros’ drug discovery engine to find new targets and leads, with a primary interest in finding a way to induce the production of fetal hemoglobin through an oral drug.

Success means GBT will gain exclusive license to develop these leads into commercial therapies, potentially producing a functional cure. For Syros, success is a $20 million upfront payment, access to future milestone payments and three years of preclinical research payments.