FUNDING FA RESEARCH

Grant Program

FARA supports research through funding competitive grants across the spectrum from basic research through drug development and clinical research programs in Friedreich’s ataxia (FA). FARA promotes collaboration among scientists, advocates for public-private partnerships, and hosts open forums for leading scientists to share their insights and ideas to advance therapeutic development for FA.

All investigators interested in FA-related research are invited to submit a Letter of Intent (LOI) through FARA’s grant submission portal. All proposed research must fall within FARA’s Grant Program Priorities.

FARA-Funded Grants

FUNDED GRANT | Nov 2024

Design and synthesis of SynGR library to probe into the role of NSD-2,3 proteins in FXN expression in FRDA

Postdoctoral Fellowship, Mechanism or Pathway of Disease Anushree Achari, PhD,
St. Jude Children’s Research Hospital
FUNDED GRANT | Jan 2025

Rescuing frataxin expression by alternative splicing reprogramming with base editing

General Research Grant, Gene & Stem Cell Therapy Alessandro Quattrone, PhD,
University of Trento
FUNDED GRANT | Dec 2024

Application of RSwitch technology to improve Friedreich's ataxia viral gene therapy safety and efficacy: control of FXN transgene expression through a small molecule regulation

General Research Grant, Gene & Stem Cell Therapy Samuel Hasson, PhD,
Rgenta Therapeutics, Inc
FUNDED GRANT | Dec 2024

Development of Epigenetic Editors for Frataxin Reactivation

Award for Innovative Mindset (AIM), Gene & Stem Cell Therapy Daniel Hart, PhD,
Epicrispr Biotechnologies, Inc.
FUNDED GRANT | Nov 2024

Quantifying sitting balance in children and adults with Friedreich ataxia - development of clinical and instrumented measures

General Research Grant, Outcome Measures & Biomarkers Sarah Milne, PhD,
Murdoch Children’s Research Institute
FUNDED GRANT | Nov 2024

Impact of cerebellar transcranial direct current stimulation on FA brain and cerebellar neurophysiological fingerprints and on clinical symptoms assessed by multimodal evaluations

Bronya J. Keats Research Collaboration Award, Improving Clinical Outcomes Gilles Naeije, MD, PhD,
Université Libre de Bruxelles, Belgium
FUNDED GRANT | Oct 2024

Understanding the Pathogenesis of Friedreich’s Ataxia by Single-Cell Integration of Gene Expression, Epigenetics and Connectivity in the Cerebellar Nuclei

Postdoctoral Fellowship, Mechanism or Pathway of Disease Julian Cheron, MD, PhD,
Johns Hopkins University
FUNDED GRANT | Oct 2024

Enhancing Gene Therapy Delivery to the Brain via Human Blood-Brain Barrier Receptors

Postdoctoral Research Award, Gene & Stem Cell Therapy Changfan Lin, PhD,
California Institute of Technology
FUNDED GRANT | Sep 2024

Shortening GAA repeats in Friedreich's Ataxia

Graduate Research Fellowship, Gene & Stem Cell Therapy David Berryman ,
University of Florida
FUNDED GRANT | Sep 2024

NRF2 and MAO-Ai therapy to improve cardiac health and prevent arrhythmias in Friedreich’s ataxia

Graduate Research Fellowship, Lead Candidates Francisco Figueroa,
University of California, Davis
FUNDED GRANT | Sep 2024

Postnatal development of the cerebellar circuitry in the KIKO mouse model of Friedreich’s ataxia

General Research Grant, Gene & Stem Cell Therapy Jordi Magrane, PhD,
Weill Cornell Medicine
FUNDED GRANT | Sep 2024

Developing the novel investigational mitochondrial therapeutic SBT-589 for Friedreich’s ataxia

Kyle Bryant Translational Research Award, Lead Candidates Laura Kropp, PhD, MPH,
Stealth BioTherapeutics
TOOLS FOR FOR RESEARCHERS & INDUSTRY

Research Resources

FARA provides valuable resources designed to support industry partners and researchers dedicated to advancing research and development for FA. Whether you’re a seasoned researcher or a new entrant to the field, FARA can offer tools to build the foundation you need to make significant contributions to the FA research landscape.

ADVANCING RESEARCH AND THERAPEUTIC DEVELOPMENT

Research Initiatives

FARA’s research initiatives are organized efforts aimed at advancing knowledge about FA through research. These initiatives put the patient voice in the forefront and focus on collaboration between researchers across countries and continents, bridging gaps in knowledge to benefit all stakeholders.

FARA funds FA research through 3 different mechanisms: the grant program, institutional supported programs and FARA directed projects. FARA believes the sum of these initiatives to be most impactful in advancing research and therapeutic development. The goal is to drive the science, facilitate access to resources, promote collaboration among FA investigators and encourage public/private partnerships.

Learn More About FARA's Research Initiatives

FA Global Clinical Consortium

FARA Directed Research

Institutional Supported Programs

PROGRESS OF RESEARCH AND DEVELOPMENT ON LEAD THERAPEUTIC CANDIDATES

Drug Development Pipeline

FARA supports the advancement of treatments with financial resources, advocacy, patient engagement and/or fostered collaboration. FARA believes that there is merit in each of these approaches and that effective treatment of FA will come in the form of a "cocktail approach" – a combination of two or more therapies.

This pipeline focuses on drugs that are already in preclinical development or have advanced to human clinical trials, and descriptions are updated regularly as new information becomes available.

Drug Development Pipeline
drug development pipeline
PLAY A ROLE IN THE SEARCH FOR TREATMENTS AND CURES

Participate in Research

Participation in FA research directly influences the treatments that reach those living with FA, potentially shortening the timeline for access to life-changing advancements.

Clinical trials are the final step before therapies reach the wider community, making them an important step on the journey to slow, stop, reverse, and cure FA.

Young girl in hospital