Deaths Raise Safety Concerns Around Gene Therapy - (The Wall Street Journal via NewsPoints Desk)

  • Safety scares, including deaths of several study subjects, have set back efforts to bring more gene therapies to market, The Wall Street Journal reported on Friday.

  • Last month, a young boy with a rare neuromuscular disease died after receiving a gene therapy in a clinical trial run by Astellas Pharma, while regulators halted a BioMarin Pharmaceutical gene therapy study after discovering cancer in mice that received the treatment. The death in the Astellas study came after the trial was allowed to resume following previous deaths of three boys treated with the same therapy.

  • "Investor sentiment is probably at all-time lows for gene therapy," said RBC Capital Markets analyst Luca Issi. A few years ago, "the skies were blue, and now that is not the case."

  • However, some researchers say the negative headlines is a symptom of the field’s success, which has led to an increase in gene therapy treatments being tested in patients. "One of the reasons you’re hearing about all these [setbacks] is that the scale of these trials is going up exponentially because the therapies are all working so well," said Nicole Paulk, assistant professor at the University of California, San Francisco. "With that increase in numbers, there will be increases in growing pains."

  • Astellas is still investigating the latest patient death, but think certain X-Linked myotubular myopathy patients are susceptible to liver damage due to their underlying disease and that there is no evidence that an immune reaction to the virus delivering the healthy gene is causing the problem, commented Nathan Bachtell, head of Astellas Gene Therapies.

  • The company hopes to find a way to safely restart the clinical trial, possibly by excluding enrollment of patients with a high risk of liver dysfunction, Bachtell said.

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