Latus Bio reports AAV capsids reaching 80% of kidney glomeruli and up to 60% of cortical neurons, with human safety and efficacy unproven.
eXmoor Pharma and Evox complete an exosome gene-editing transfer, advancing the CMC path for Evox’s Huntington’s disease programme.
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.
Explore why the FDA reopened uniQure’s AMT-130 approval path and what evidence, surgical and confirmatory trial risks remain before review.