Two separate CNS-focused approaches are advancing with translational emphasis on reaching the brain and altering disease biology. First, a University of Cincinnati and UC Davis team reported AAV gene therapy targeting cerebrospinal fluid-producing cells reduced hydrocephalus-related brain swelling in animal models, positioning gene delivery as a potential minimally invasive alternative to shunt surgery. Separately, a Lyon-based biotech Axoltis described topline Phase II SEALS trial results for the BBB-repair peptide NX210c in ALS. While the trial did not reach primary endpoints, the company said other readouts support BBB repair biology alongside a favorable safety profile. Together, the updates show continued emphasis on mechanism-first CNS strategies—either through delivery to relevant compartments or via BBB modulation—while reaffirming that clinical endpoints remain the gating factor for success.