Accelerating Research in Drug Repurposing for paediatric OTCD Patients

Abstract

Drug repurposing can play a critical role in treating rare diseases. To accelerate drug repurposing research for a rare genetic condition called, Ornithine Transcarbamylase Deficiency (OTCD), our team has engaged in cross-collaboration with different stakeholders - including patient advocates, scientists, and clinicians - early on in the research journey.

Our collaborative network consists of research partners in the US, Switzerland, Australia, as well as the Netherlands. This was accomplished in less than two years. Although research can be very costly and time-consuming, early engagement is likely to shorten the amount of time it takes to conduct research by mobilizing resources (ie. expertise, equipment and/or funding) and by creating further opportunities for meaningful collaboration.

The poster will be divided into three sections: (1) What our team is currently working on as part of a drug repurposing initiative/research (building a liver organoid model), (2) how this research stemmed from early collaboration efforts (partnering with scientists from different research fields), and (3) what we hope to achieve with this model, and by when (confirm mechanism of action for the specific OTCD mutation, c.77G>A and conduct a comprehensive drug screen on this model).