Regulatory Reversal Restores Hope for Huntington's Gene Therapy

Huntington's disease currently has no effective treatments and typically kills patients in their 50s and 60s, making this gene therapy's potential 75% disease-slowing capability potentially transformative. The regulatory reversal highlights how FDA personnel and political shifts can dramatically impact patient access to breakthrough therapies. This case could establish important precedent for how gene therapies—particularly those requiring invasive procedures—navigate FDA approval pathways.