Replies to post #126 on Sangamo Biosciences Inc (SGMO)

[surf1944]

7:04AM Sangamo BioSci announces publication in nature of gene correction strategy for Alpha 1-Antitrypsin deficiency (SGMO) 3.38 : The study, published in Nature, further highlights the precision and broad applicability of ZFN-based genome-editing for the development of ZFP Therapeutics for the treatment of monogenic diseases. "These data demonstrate the potential of combining human iPSCs with ZFN-driven gene correction to generate differentiated cell-based therapies," stated Philip Gregory, D. Phil., Sangamo's vice president of research and chief scientific officer. "Importantly, analysis of the entire coding sequence of a ZFN-corrected iPSC line revealed that the only modification attributable to ZFN activity was the intended repair of the A1AT gene. This demonstrates the singular specificity that can be achieved using Sangamo's ZFP technology

[ZincFinger]

The SB-509 trial was questionable from the start IMHO.

Diabetic Neuropathy is affected by many variables of which VEGF is only one. So there was always the chance that the ZF treatment would have the expected direct result intended (increase of VEGF) but that that increase would not have the expected results for it. And that, unfortunately, was exactly what happened. "The treatment was a success but the patient died."

The zinc finger therapy did indeed result in a higher production of VEGF, but that did not have the expected results. (Another variable affecting the condition was more important)

Thus the trial was, in a very real sense, a success for zinc fingers but a failure for their use to increase VEGF as a therapy for DN. (There are other conditions that increasing VEGF might be therapeutically useful but they have a similar risk as the DN trial. So IMHO SGMO was wise to drop SB-501 for now (later when it has more money it could do trials for its use in other conditions.)

Time to go after the "low hanging fruit" (monogenic diseases, etc.)