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Sangamo BioSciences Announces Presentation of Preliminary Data From Phase 2 Study of SB-509 at International ALS Symposium
Date:12/9/2009

scle testing (MMT), a widely used method of evaluation of muscle function in subjects with ALS, 32% of SB-509 treated subjects in cohort 1 (6/19) demonstrated an improvement in muscle function as assessed by an overall positive slope in their MMT score from day 0 - day 120 compared to historical controls in which 17% improved (26/153). Of the subjects within the intent to treat group who showed improvement in MMT slope across all visit days, 5 also showed parallel improvement in one or more endpoints (ALS-FRS-R and FVC).

A variety of data supporting the use of SB-509 in ALS were presented. These included preclinical experiments from a rat transgenic SOD1 model of ALS that demonstrated improved motor performance and hindlimb grip strength in SB-509-treated compared to placebo-treated animals as well as clinical observations from Sangamo's clinical study of SB-509 in subjects with diabetic neuropathy (SB-509-601). Data from the SB-509-601 study included previously reported dose-related improvements in the muscle testing component of the neurologic exam in the lower limb, or NIS-LL, as well as statistically significant improvements in intraepidermal nerve fiber density (IENFD) in SB-509 treated subjects. In addition, data were also presented demonstrating retrograde neuronal transport in the sciatic nerve back to the dorsal root ganglia of VEGF-A which had been produced in the muscle after IM administration of SB-509 in a diabetic rat model.

About Amyotrophic Lateral Sclerosis (ALS)

More than 30,000 Americans have ALS, according to the ALS Association, a nonprofit organization that supports ALS research and public and patient education about the disease. 3,000 to 5,000 new cases of the disease are diagnosed every year. Men and women of all ethnic and racial groups are equally affected, usually between the ages of 40 and 70. The disease attacks the motor nerves, nerve cells in the brain and spinal cord that control the body'
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SOURCE Sangamo BioSciences, Inc.
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