Evox Therapeutics advances MSH3 Huntington’s disease programme through eXmoor Pharma transfer
eXmoor Pharma and Evox complete an exosome gene-editing transfer, advancing the CMC path for Evox’s Huntington’s disease programme.
Pharma & Biotech
Pharma & Biotech
Can uniQure turn the FDA’s AMT-130 reversal into a Huntington’s disease approval?
The Food and Drug Administration will accept uniQure’s three-year AMT-130 data as the primary basis for an accelerated approval filing, but external controls, irreversible brain delivery and confirmatory evidence remain decisive risks.
Pharma & Biotech
FDA accepts uniQure’s AMT-130 filing strategy after months of regulatory uncertainty
Explore why the FDA reopened uniQure’s AMT-130 approval path and what evidence, surgical and confirmatory trial risks remain before review.