Scientific Strategy

Steadfast in our mission to foster and accelerate innovative treatments and a cure for PolG disorders, The PolG Foundation aims to be a conduit for scientific collaboration by connecting academic research, drug discovery and clinical development together.

To achieve our PolG research goals, we work in several different ways:

We support PolG academic researchers around the world by funding a diversity of preclinical, translational and clinical initiatives.

We support investigators with an ‘extraordinary idea’ for PolG research.

We have funded the development of two PolG Transgenic Mice. Characterisation is underway.

A Systematic Review of Animal and Cell Models of PolG-related Disease is awaiting publication.

We actively invite biotech and pharmaceutical companies to reach out and partner with us to develop PolG therapeutics.

Defined Areas of Research

Basic science: understanding mechanisms and modelling PolG disease

  • Understanding the pathophysiology and how this can be manipulated for therapeutic intervention
  • Generating models that mimic human disease. Special focus on mouse and stem cell models
    • Using models to understand disease mechanisms
    • Using models to test drugs/compounds

Getting ready for clinical trials

  • Register/cohort studies
  • Identification of genetic/environmental triggers
  • Understanding the clinical diversity of this monogenic disease

Clinical trials including drug repurposing initiatives

  • Pilot studies
  • Full trials