On the other hand, their stock price just took a solid hit. Derek Lowe goes into why: http://blogs.sciencemag.org/pipeline/archives/2018/09/05/sangamos-gene-th

On the other hand, their stock price just took a solid hit. Derek Lowe goes into why: http://blogs.sciencemag.org/pipeline/archives/2018/09/05/sangamos-gene-therapy-results http://blogs.sciencemag.org/pipeline/archives/2018/09/05/san... There are only two patients that we’re talking about here. But both of them showed significant declines in the heparan sulfate and dermatan sulfate in their urine, which is good news. However, and this is where the arguing really revs up, they could not find any IDS enzyme in the blood of these patients. You’d have kinda thought that you’d see it there (they sure saw it in the animal studies), and a lot of observers/investors sort of lost it at that news, sending SGMO down pretty briskly in the early trading today. The company responded by saying that there had to be new active enzyme in there for the urine numbers to go the way that they did, that their detection method isn’t incredibly sensitive to start with, that there’s another cohort in the trial that’s getting a larger dose of the gene therapy, etc. Of course, they also said that urinary levels of GAG were an approvable biomarker and that’s how the enzyme replacement therapy was approved, and that appears not to be true. The enzyme (Elaprase, from Shire) showed effects in the amount the treated patients could walk and the trials looked at lung function as well. In the end, you’re going to want to see functional improvements in these patients anyway, but everyone’s looking for an earlier read. The selloff in Sangamo’s shares continues as of this writing, and other gene-editing stocks are dropping as well (and since these are all companies using different gene-editing technologies, that seems a bit like panic if you ask me). But this can’t have been the way that Sangamo wanted this to go. When that next cohort reports, maybe we’ll have a better idea of what’s going on – either that, or the shouting will get even louder. . .