Moe Alsumidaie’s Post

Oral, brain-penetrant therapies for GBA-linked diseases have been a promise for a decade — Sharp is stepping back into the arena. Sharp Therapeutics is preparing a Phase 1–ready package for its small-molecule ‘901, aiming to restore GBA biology with a simple oral regimen that reaches both the periphery and CNS. In a space defined by enzyme replacement and substrate reduction therapies that struggle with neuronopathic disease, this approach could streamline development and expand reach to GBA-associated Parkinson’s disease. But after inconsistent outcomes from prior small-molecule programs, the bar for translational proof — CSF exposure, biomarker shifts, and functional alignment — is high. Early trial design choices around PK/PD, lumbar puncture capability, and add-on versus switch strategy will determine whether Sharp can convert differentiation claims into real clinical momentum. What will be the decisive evidence to shift confidence back toward small-molecule restoration in lysosomal neurodegeneration? 𝐑𝐞𝐚𝐝 𝐌𝐨𝐫𝐞: https://lnkd.in/gtJJQrPH #RareDisease #GaucherDisease #ParkinsonsDisease #Neurodegeneration #Biotech Scott Sneddon Josh Lorenz-Guertin, Ph.D.