Gene therapies on clinical hold after tumour found in five‑year‑old
The development of a brain tumour in a study participant has led the FDA to temporarily halt two ongoing gene therapy programs. The affected company, Regenxbio, immediately lost nearly one‑fifth of its market value.
The tumour was discovered during a routine MRI scan of an asymptomatic five‑year‑old participant who had been treated four years earlier with the gene therapy candidate RGX‑111 for the rare hereditary metabolic disorder Hurler syndrome.
An investigation is underway to determine whether the tumour is related to the treatment, Regenxbio said in a press release.
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