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What happened
Actions of Sangamo Therapeutics (NASDAQ: SGMO) fell 24.7% today on large volumes. The company's actions are in response to a somewhat disappointing interim update for its zinc finger nuclease gene therapy, SB-913, as a possible treatment for the rare metabolic syndrome known as Hunter's Syndrome (or Mucopolysaccharidosis). type II). The update took place at the 2018 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism, which was held in Athens, Greece.
Hunter syndrome is a hereditary disorder of lysosomal storage resulting from an insufficient level of an enzyme called iduronate-2-sulfatase (IDS). This key catabolic enzyme breaks down glycosaminoglycans (GAGs). Low levels of IDS therefore result in GAG accumulation in the body, leading to hypertrophy of organs that can not function properly.
So what
This trial marks the first time that a gene editing technique has been evaluated in a patient. As such, investors were clearly banking on stellar results in terms of safety and efficiency. Sangamo, however, noted that the test manipulators were unable to quantify plasma SDI activity after treatment, as the level of quantification of the current assay was inadequate. Thus, while the marked reductions in GAGs observed in the study are certainly encouraging, this essay simply can not provide direct evidence of the effectiveness of the SB-913 in the current state of affairs. .
Now what
Sangamo hopes to address this blatant weakness by conducting another study in which CountyThe replacement enzyme treatment, Elaprase, is gradually being replaced by SB-913 treatment. In this way, researchers will have a better understanding of the therapeutic effect of SB-913 in this devastating genetic disease. Sangamo also noted that he is working to improve the sensitivity of the IDS test to enable more accurate measurements.
Unfortunately, these two efforts will take a little time to bear fruit. Investors may therefore have to wait around a year to truly understand the real prospects of in vivo editing of the genome.
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