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Bit More Cautious

Safety concerns are tempering investor enthusiasm for gene therapies, the Wall Street Journal reports.

It writes that young boy with a neuromuscular condition in a gene-therapy clinical trial run by Astellas Pharma died shortly after the trial resumed — a pause that occurred due to the deaths of three other boys — while regulators stopped another study run by BioMarin Pharmaceutical after finding that mice that received the treatment developed cancer.

The concerns, analysts tell the Journal, have made investors a little wary. A few years ago, "the skies were blue, and now that is not the case," Luca Issi, senior biotechnology analyst at RBC Capital Markets, tells it.

However, some researchers tell the Journal that this increase in safety concerns also reflects the increased interest in gene therapies and the number of gene-therapy trials.

Further, BioMarin's Henry Fuchs tells the Journal that the liver cancer seen in the animal models hasn't been observed in any person who received a gene therapy. And Astellas' Nathan Bachtell notes that certain X-Linked myotubular myopathy patients appear to be more susceptible to liver damage because of their underlying disease, the Journal says, adding that the company hopes to restart its trial by excluding those at high risk.