BEBT-758
BEBT-758 selectively silences the SNCA gene to reduce alpha-synuclein expression. Preclinical studies in mice demonstrate significant suppression of SNCA in critical brain regions, including the cerebral cortex, brainstem, hypothalamus, and cervical spine. Unlike SNCA antibodies that target late-stage pathological aggregates, BEBT-758 siRNA acts upstream by reducing SNCA mRNA, decreasing alpha-synuclein production at its source. This upstream intervention prevents misfolding, oligomerization, and aggregation, aiming to halt the disease's pathological cascade.
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