other material
confidence high
sentiment negative
materiality 0.90
Neurogene patient dies after high-dose NGN-401; FDA allows trial to continue at lower dose
Neurogene Inc.
- Patient dosed Nov 5 with 3E15 vg NGN-401 died from hyperinflammatory syndrome linked to high AAV exposure.
- FDA permitted Phase 1/2 trial to continue using 1E15 vg dose for pediatric and adolescent/adult cohorts.
- Neurogene will incorporate the 1E15 vg dose into future registrational trial design.
- This is the same patient previously reported as critical; hyperinflammatory syndrome is rare and life-threatening.