MedCity News July 9, 2024
A uniQure gene therapy for Huntington’s disease has interim clinical data showing an 80% slowing of disease progression. An accelerated approval pathway is one of the topics the company wants to discuss with the FDA.
A uniQure gene therapy for Huntington’s disease has interim data showing the one-time treatment slows progression of this rare neurodegenerative disorder that has no FDA-approved therapies. Based on these encouraging data, the company said Tuesday it now plans to discuss with regulators the potential for a faster clinical and regulatory path forward.
Huntington’s stems from a genetic mutation that leads to abnormal versions of the huntingin protein. The disease leads to motor dysfunction, behavioral changes, and cognitive decline. The uniQure gene therapy, code-named AMT-130, uses...
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