"Preclinical data as well as clinical observations from our studies of SB-509 in diabetic neuropathy demonstrated that this ZFP Therapeutic has an effect on muscle function and we are very pleased to see evidence of a similar effect in subjects with ALS," stated Dale Ando, M.D., Sangamo's Vice President of Therapeutic Development and Chief Medical Officer. "Our primary goal for this study was to assess the safety and tolerability of SB-509 in subjects with ALS and to evaluate a dosing schedule and pattern of drug administration as well as to look for indications of efficacy of the drug. At this point we have full data sets from only a handful of the subjects enrolled in the trial and thus have not completed a full analysis of all of the endpoints. Significant improvements in muscular function have not been seen in previous ALS trials. Any progress in this endpoint may translate into an important benefit in the quality of life of these patients. The drug continues to have an excellent safety profile and these initial results are encouraging not only for ALS but potentially for application in other neuromuscular diseases such as dystrophies and myopathies."
SOURCE Sangamo BioSciences, Inc.