Clinical Trial MilestonesCompletion of dosing in the pivotal REVEAL Part B cohort and a six-month interim analysis could enable an earlier topline readout and a subsequent regulatory filing for the full Rett population if results are positive.
Label ExpansionFDA confirmation that limited pediatric safety data could support a broader label for TSHA-102 covering patients aged two and up if the pivotal study succeeds would expand the addressable patient population.
Regulatory Risk ReductionBreakthrough Therapy Designation combined with FDA alignment on competitive positioning reduces regulatory uncertainty and clarifies a differentiated approval pathway versus competing gene therapies.