Setback for rare diseases as Taysha Gene Therapies pulls back

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I was wrong about Geron.

For years, I’ve believed — and declared publicly — that Geron’s effort to develop a blood cancer drug called imetelstat would end in disappointment. The drug was never getting approved, I said.

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On Thursday, a group of outside experts convened by the Food and Drug Administration voted 12-2 in imetelstat’s favor, concluding the drug’s benefits outweighed its risks for the treatment of patients with myelodysplastic syndrome. The strong endorsement makes an FDA approval highly likely. The decision is expected on or before June 16.

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