Bethesda’s Gain Therapeutics Presents New GBA1 Parkinson’s Disease Program Pre-clinical Data

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Compounds reverse the neurodegenerative process observed in a Parkinson’s disease in vivo model

Compounds are novel allosteric regulators that bind, stabilize and restore activity of mutated GCase

Compounds decrease both phosphorylated and aggregated α-synuclein levels in vitro and in vivo