fara continues to raise money to support groundbreaking research to help find new treatments and a cure for Friedreich Ataxia.
Over the past year, fara has expanded its support and guidance of Friedreich Ataxia research projects in partnership with Friedreich Ataxia advocacy groups around the world, particularly the Friedreich’s Ataxia Research Alliance in the United States.
All of the research projects are focused on advancing promising therapeutic approaches and cover strategic Friedreich Ataxia research areas such as drug discovery and investigation,
gene and stem cell therapy, cell and animal models, outcome measures and biomarkers, and improving clinical outcomes through the Collaborative Clinical Research Network in Friedreich Ataxia. Additionally, fara supported the participation of researchers in scientific meetings co-sponsored by fara around the world.
Promising international gene therapy research means that significant progress is being made towards initiating gene therapy clinical trials for Friedreich Ataxia.
Several biotechnology companies are committed to advancing gene therapies for Friedreich Ataxia and one of them, Agilis Biotherapeutics, was recently granted Orphan Drug Designation by the United States Food and Drug Administration for its Friedreich Ataxia gene therapy product candidate, AGIL-FA. This is an important step for advancing this product candidate into clinical trials. fara is committed to facilitating gene therapy ‘Clinical Trial Readiness’ in Australia.