EJP RD’s innovative Rare Diseases Research (RDR) Challenges call in partnership with Fondation Maladies Rares was aimed at facilitating and funding collaboration between industry, academia, SMEs, and patient organisations to solve specific research challenges in rare diseases.
The fourth challenge issued under the RDR call was for the development of a pre-clinical assay to detect instability of microsatellite repeat expansions. We are glad to announce the launch of the TRXassay, a novel pre-clinical assay to detect triplet repeat expansions, as a response to this challenge. The project is co-funded by the EJP RD and by LoQus23 Therapeutics Ltd of Cambridge, UK.
In this project, three teams from universities in Ireland, Scotland and Wales will work together to develop novel technologies to monitor how the number of repeats changes in cells grown in the laboratory. This system will then be used to identify new drugs that slow the rate at which the repeat grows. The hope is that one such drug could then be used to treat multiple inherited disorders, including Huntington’s disease and myotonic dystrophy.
Speaking on behalf of the EJP RD, Dr Christine Fetro of the Fondation Maladies Rares said: “The TRXassay was one of three projects selected for funding within the Rare Diseases Research (RDR) Challenges call led by Fondation Maladies Rares and EJP RD. This collaborative project brings together academic innovators with an industry partner to drive rare disease research towards effective treatments, which is at the very heart of this European funding initiative.”
Press release from National University of Ireland Galway: https://www.nuigalway.ie/about-us/news-and-events/news-archive/2022/may/nui-galway-leads-new-research-project-on-neurological-diseases.html