Innovation in therapeutic strategies:
One of the most inspiring aspects of the conference was the breadth of innovative therapeutic approaches on display. Researchers are increasingly thinking beyond traditional drug development models and exploring novel biological systems and mechanisms. Highlights included the use of microglia as drug delivery agents within the brain, as well as engineering live gut bacteria to produce L-dopa, offering a potentially transformative approach to treatment delivery.
Clinical trial momentum:
There was strong momentum across the clinical pipeline, from early-stage programmes to large-scale Phase III trials. A standout session focused on lysosomal biology, highlighting two particularly promising therapeutic candidates now in Phase I trials:
- TTP-34, which activates CREB and is thought to enhance lysosomal function, has shown the ability to rescue Parkinson’s disease pathology in both in vitro and in vivo models.
- VQ-101 (Vanqua Bio), a small molecule allosteric activator of glucocerebrosidase (GCase), demonstrated neuroprotection in models of GBA-associated Parkinson’s disease, alongside strong evidence of target engagement in vivo.
Read the full story on the Department of Physiology, Anatomy and Genetics website.