Clinical TrialsEnrollment is complete in the Phase 1/2 trial of NGN-101 in CLN5 Batten disease with interim clinical data expected and a regulatory update that will likely determine the future of this program.
Financial StabilityNeurogene ended 2Q24 with $154M in cash, which the company continues to expect to be sufficient into 2H26.
Regulatory ApprovalRecent RMAT designation, along with FDA's START designation, gives the opportunity for accelerated approval and suggests preliminary clinical data appears promising.