Australian Friedreich Ataxia Stem Cell and Gene Therapy Consortium

Research area: GeneticsGenetic Health (BLC)  | Status: Active

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The Consortium’s vision is to facilitate and undertake Friedreich ataxia cell and gene therapy clinical trials leading to successful outcomes for patients as soon as possible.

The Australian Friedreich Ataxia Stem Cell and Gene Therapy Consortium is a strategic alliance of pre-eminent experts in Friedreich Ataxia, Stem Cells, Gene Therapy, Policy, Advocacy and Regulation.

The Australian Friedreich Ataxia Stem Cell and Gene Therapy Consortium is a strategic alliance of pre-eminent experts in Friedreich Ataxia, Stem Cells, Gene Therapy, Policy, Advocacy and Regulation.

Overview

Friedreich Ataxia Stem Cell logoThe Consortium has been assembled by the Murdoch Children’s Research Institute to develop strategies and undertake programs that will expedite Friedreich Ataxia cell and gene therapy clinical trial readiness and translation in Australia and internationally.

Our purpose

Currently, there are no approved treatments or cure for Friedreich Ataxia.

  • 1 in 30,000 people in Australia are born with Friedreich Ataxia.
  • 1 in 90 unaffected people are carriers of a faulty Friedreich Ataxia gene.

At least seven companies and/or academic institutions are currently developing gene therapies for Friedreich Ataxia. Murdoch Children’s Research Institute is the Australian Collaborative Clinical Research Network (CCRN) in FA site and is preparing to be the Australian site for Australia’s first approved Friedreich Ataxia gene therapy clinical trials.

What’s involved

  • Establishment of FA Gene Therapy Readiness at the Murdoch Children’s Research Institute.
  • A Consortium Advisory Board meets regularly and provides expertise and guidance on accelerating progress towards FA cell and gene therapy clinical trial readiness and translation.
  • The Australian and international FA research community attends an annual FA Stem Cell and Gene Therapy Colloquium and helps advance research and fill key gaps.
  • Consortium-directed research projects include vector development, qualitative study of patient and family views of gene therapy trials and outcome measure selection.
  • An International Consortium for FA Gene Therapy Readiness that includes key stakeholders in clinical research sites, pharma and biotech industry partners, regulatory authorities and patient education and informed consent.

Information for participants

We are committed to fast tracking FA cell and gene therapy clinical trials in Australia for all individuals with FA. Here are some ways you can get involved

Contact us

Murdoch Children's Research Institute
The Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Australia

FA Clinical Research Database
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