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Australian Friedreich Ataxia Stem Cell and Gene Therapy Consortium

Research project

What is the Australian Friedreich Ataxia Stem Cell and Gene Therapy Consortium?

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 Consortium has been assembled by Murdoch Children’s Research Institute (MCRI) 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 vision is to facilitate and undertake Friedreich Ataxia cell and gene therapy clinical trials leading to successful outcomes and treatments for patients as soon as possible.

Key facts:
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 7 companies and/or academic institutions are currently developing gene therapies for Friedreich Ataxia; 2 of these groups hope to file Investigational New Drug (IND) applications with the United States Food and Drug Administration (FDA) in 2021.

MCRI is the Australian Collaborative Clinical Research Network (CCRN) in FA site.

MCRI is preparing to be the Australian site for Australia’s first approved Friedreich Ataxia gene therapy clinical trials.

Our Mission:

The Consortium is an alliance of key experts who seek to identify all Friedreich Ataxia Cell and Gene Therapy clinical trial readiness and translation needs and address them through directed research projects and programs.

The Consortium seeks to facilitate and undertake Friedreich Ataxia gene therapy clinical trials in Australia, leading to successful outcomes for patients as soon as possible.

Who we are:

Consortium Executive Committee

The Consortium Executive Committee’s role is to facilitate, manage and coordinate all projects required to accelerate progress towards Friedreich Ataxia cell and gene therapy clinical trial readiness and translation.

Consortium Advisory Board

The Consortium Advisory Board provides expertise and guidance to the Executive Committee for accelerating progress towards Friedreich Ataxia cell and gene therapy clinical trial readiness and translation. Its responsibility is to develop strategies required from a research, regulatory, industry, hospital, clinical and patient perspective to ensure cell and gene therapy clinical trial readiness and translation in Australia.

Our Partners

We thank and acknowledge the support of FARA (Friedreich’s Ataxia Research Alliance).

Our Research:

The Consortium undertakes directed research projects and programs to advance preparedness and readiness. They include:

Friedreich Ataxia Stem Cell and Gene Therapy Colloquium

The Australian Friedreich Ataxia Stem Cell and Gene Therapy Consortium was launched on Thursday, 12 March 2020 at the inaugural Friedreich Ataxia Stem Cell and Gene Therapy Colloquium at MCRI.

The launch was hosted by Dr Katie Allen, MP (Federal Member for Higgins), Professor Kathryn North (Director, MCRI) and Professor Martin Delatycki (Co-Director, Bruce Lefroy Centre, MCRI and Chair, Australian Friedreich Ataxia Stem Cell and Gene Therapy Consortium).

The Colloquium was attended by more than 30 Australian and international experts who participated in discussions focused on identifying gaps and exploring solutions to accelerate progress towards Friedreich Ataxia Stem Cell and Gene Therapy Clinical Trial Readiness in Australia.

It included scientific presentations on:

  • Progress towards overcoming challenges that face gene therapy trials
  • Stem cell models for validating and optimising gene therapy products
  • Development of robust biomarkers that are essential for initiating clinical trials.

The inaugural Colloquium inspired conversations, connections and collaborations for new Australian stem cell and gene therapy research projects that seek to fast track progress towards successful patient clinical trials.

International Gene Therapy Preparedness Consortium

The first Consortium program with FARA (Friedreich’s Ataxia Research Alliance) was the formation of an International Gene Therapy Preparedness Consortium. It seeks to identify all of the requirements for cell and gene therapy clinical trial preparedness internationally.

The International Consortium works together with key stakeholders including;

  • International FRDA Clinical Research sites in the USA, Canada, Europe, Australia, New Zealand and South America
  • Pharmaceutical and Biotech Industry Partners
  • Clinical Trial sponsors
  • Regulatory authorities

Gene Therapy Patient Education

The development of educational materials for the Patient community is an essential component of Gene Therapy Preparedness. A Qualitative Study exploring attitudes of individuals with Friedreich ataxia and their families towards stem cell and gene therapy is being undertaken by a team led by Associate Professor Louise Corben, Bruce Lefroy Centre, MCRI. The study will assist to inform the development of Patient Education materials by FARA for the patient community.

To learn more about Friedreich Ataxia Stem Cell and Gene Therapy research and clinical trials:
Sign up to our database: email varlli.scott@mcri.edu.au
Join the FA Global Patient Registry

Make a donation

Donate to support the Australian Friedreich Ataxia Stem Cell and Gene Therapy Consortium.