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 | 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
FUNDED GRANT | Sep 2024

Deliverable in vitro transcribed mRNAs of Frataxin as a therapeutic approach for Friedreich ataxia

Award for Innovative Mindset (AIM), Gene & Stem Cell Therapy Lefkothea Papadopoulou, PhD,
Aristotle University of Thessaloniki
FUNDED GRANT | Sep 2024

Deriving Functional MRI Equivalent Biomarkers in Friedreich Ataxia solely using Brain Structure Data

Award for Innovative Mindset (AIM), Outcome Measures & Biomarkers Susmita Saha, PhD ,
Monash University
FUNDED GRANT | Jul 2024

Characterization of FA iPSC-derived DRG organoid to decipher neurodevelopmental and neurodegenerative mechanisms

Postdoctoral Fellowship, Cell & Animal Models Valentine Mosbach, PhD,
Institut NeuroMyogène, INSERM and Université Claude Bernard Lyon
FUNDED GRANT | Jul 2024

Skeletal Muscle Fatigue and Abnormal Ca2+ Signaling in Friedreich's Ataxia

Postdoctoral Research Award, Mechanism or Pathway of Disease Chen Liang, PhD,
University of Rochester, NY
FUNDED GRANT | Jun 2024

Investigating microglial phenotypes and functions in the pathogenesis of Friedreich’s ataxia

General Research Grant, Mechanism or Pathway of Disease Nadia D’Ambrosi, PhD ,
University of Rome Tor Vergata
FUNDED GRANT | Jun 2024

Multimodal peripheral nervous system imaging to quantify, monitor, and understand the neuropathy of Friedreich ataxia

General Research Grant, Outcome Measures & Biomarkers Roderick Maas, MD, Nens van Alfen, MD, PhD, Bart P. van de Warrenburg, MD, PhD,
Radboud University Medical Center, Nijmegen
FUNDED GRANT | Apr 2024

Targeting Novel Mitochondrial Pathways for Friedreich's Ataxia Therapy

Postdoctoral Research Award, Mechanism or Pathway of Disease Sujyoti Chandra, PhD,
Stanford University
FUNDED GRANT | Apr 2024

Dysregulated microglial Ca2+ homeostasis as a pathogenic contributor and therapeutic target in Friedreich’s ataxia

Postdoctoral Fellowship, Mechanism or Pathway of Disease Carla Pernaci, PhD ,
University of California, San Diego
FUNDED GRANT | Mar 2024

Paired Prime Editors to treat Friedreich’s Ataxia

General Research Grant, Gene & Stem Cell Therapy Jonathan Watts, PhD, Erik Sontheimer, PhD, Scot Wolfe, PhD and Wen Xue, PhD ,
UMass Chan Medical School
FUNDED GRANT | Mar 2024

Treatment of Friedreich’s ataxia using novel ferroptosis inhibitors

General Research Grant, Lead Candidates Brent R. Stockwell, PhD,
Columbia University
FUNDED GRANT | Mar 2024

Developing and delivering of novel gene editing technologies to contract GAA repeats in Friedreich's ataxia

Postdoctoral Fellowship, Gene & Stem Cell Therapy David Rufino-Ramos, PhD,
Harvard Medical School
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