This is an illustrative example program used to demonstrate the pipeline layout. It does not represent an approved or actual Peptone program.
Lead Optimization
Alpha-synuclein aggregation modulator
A small molecule that binds monomeric alpha-synuclein and biases its conformational ensemble away from the aggregation pathway implicated in Parkinson's disease.
- Target
- Alpha-synuclein
- Modality
- Small molecule
- Indication
- Parkinson's disease and related synucleinopathies
- Stage
- Lead Optimization
Target
Alpha-synuclein is a small intrinsically disordered protein whose misfolding and aggregation into oligomers and fibrils is a hallmark of Parkinson's disease and other synucleinopathies. Its lack of a stable fold has made it a classic undruggable target for conventional structure-based design.
Approach
Rather than chasing a fixed pocket, Peptone measures the transient contacts that precede aggregation and designs binders that stabilise non-amyloidogenic states of the monomer, lowering the population of aggregation-prone conformers.
Preclinical
Illustrative biophysical and cellular assays show reduced aggregation and preserved physiological behaviour of the target, supporting progression of the chemical series.
Development Plan
This example program sits in lead optimization, where potency, selectivity, and central nervous system exposure are being refined ahead of candidate selection.