STRIALIS: A platform to mechanistically subtype and treat Parkinson's disease.

Obiettivo

Parkinson’s disease (PD) is a molecularly heterogeneous disorder for which disease-modifying therapies remain elusive. Despite decades of investment, late-stage clinical trials have repeatedly failed, often following promising efficacy in traditional animal models. These failures highlight a critical gap between preclinical testing and clinical outcomes, driven in part by the limited ability of existing models to capture the molecular diversity of PD and thus to inform patient stratification strategies.

STRIALIS is a next-generation physiologically relevant human model designed to improve the predictability of PD drug development by enabling systematic evaluation of therapeutic candidates across a broad spectrum of molecularly defined PD subtypes. By moving beyond one-size-fits-all models, STRIALIS allows for the identification of mechanistically matched responder populations and for the deprioritisation of candidates unlikely to translate to human benefit, thereby reducing costly late-stage attrition.

In our project, we will: 1) assemble a panel of representative molecular causes of PD, which will be integrated into the first version of the model (Atlas v1); 2) benchmark this panel using previously validated reference compounds; 3) increase throughput, shorten assay duration and reduce operational costs; and 4) identify industry adoption barriers and prepare to comply with regulatory requirements in subsequent phases. If successful, we will expand this platform by exploring the mechanistic spectrum of idiopathic PD, accounting for ~90% of cases, update the atlas accordingly (v2) and move forward towards the deployment of our preclinical services in a pilot project with the Johnson&Johnson PD drug portfolio.

This approach will position STRIALIS as a scalable, regulatory-ready New Approach Methodology that bridges preclinical disease biology and clinical decision-making, addressing a critical bottleneck in the development of effective treatments for PD.