Discovery & Development Drug Discovery, Small Molecules

Slowing the Pace of Parkinson’s

Researchers explore a small molecule with the potential to tackle disease progression

Maryam Mahdi | 03/02/2020

This article is part of our special focus on "traditional" pharma: The Small Molecule Manufacturer (read more here). You can find more articles from The Small Manufacturer here.

A team, led by Matthew Disney from Scripps Research and M. Maral Mouradian from the Rutgers Robert Wood Johnson Medical School Institute for Neurological Therapeutics, has developed a small molecule drug, dubbed Synucleozid, which targets the messenger RNA (mRNA) of ɑ-synuclein, a protein whose misfolding is genetically and neuropathologically linked to Parkinson’s disease (PD) (1). Though there are several experimental treatments in development that target ɑ-synuclein protein aggregates, the team’s goal is to prevent them from developing entirely by inhibiting its translation at the mRNA stage.

Disney and Mouradian are now optimizing the drug so that it can be applied to other neurodegenerative diseases.

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Discovery & Development Drug Discovery, Small Molecules

MD Disney et al., “Translation of the intrinsically disordered protein α-synuclein is inhibited by a small molecule targeting its structured mRNA.” PNAS, 1457-1467 (2020)

Maryam Mahdi

Deputy Editor

After finishing my degree, I envisioned a career in science communications. However, life took an unexpected turn and I ended up teaching abroad. Though the experience was amazing and I learned a great deal from it, I jumped at the opportunity to work for Texere. I'm excited to see where this new journey takes me!