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 | Oct 2025

Exploring the Role of Extracellular Traps in the Pathogenesis of Friedreich’s Ataxia

Award for Innovative Mindset (AIM), Mechanism or Pathway of Disease Katia Aquilano, PhD ,
University of Rome Tor Vergata, Italy
FUNDED GRANT | Aug 2025

Histone H3 Enzyme Activity as a Regulator of Iron-Sulfur Cluster Stability in Friedreich’s Ataxia

Award for Innovative Mindset (AIM), Mechanism or Pathway of Disease Siavash Kurdistani, MD,
University of California Los Angeles
FUNDED GRANT | Aug 2025

Investigating new epigenetic repressor complexes involved in silencing the FXN locus in cells from FRDA patients

Award for Innovative Mindset (AIM), Mechanism or Pathway of Disease Patrick Lomonte, PhD and Hélène Puccio, PhD ,
Université Claude Bernard - Lyon I
FUNDED GRANT | Aug 2025

The N-terminus of frataxin and its role in FRDA

Award for Innovative Mindset (AIM), Mechanism or Pathway of Disease David Lynch, MD, PhD, Jill Napierala, PhD, Yina Dong, PhD, Ian Blair, PhD,
Children’s Hospital of Philadelphia, University of Texas Southwestern, University of Pennsylvania
FUNDED GRANT | Jul 2025

Rational design of new CNS neuronal models for selective vulnerability in FRDA

Award for Innovative Mindset (AIM), Cell & Animal Models Pietro Mazzara, PhD ,
Columbia University
FUNDED GRANT | Apr 2025

Molecular and cellular mechanisms of cardiac fibrosis in Friedreich ataxia

General Research Grant, Cardiac Research Giovanni Manfredi, MD, PhD and Mark Payne, MD ,
Weill Cornell Medicine and Indiana University
FUNDED GRANT | May 2025

Enhancing Friedreich Ataxia Research: Data Augmentation and Multimodal Fusion Focusing on the Spine-Brain Axis

Postdoctoral Research Award, Outcome Measures & Biomarkers Ravi Dadsena, PhD,
RWTH Aachen University Hospital, Germany
FUNDED GRANT | Mar 2025

Inhibition of the TLR4 receptor prevents neuro-inflammation and degeneration in frataxin-deficient sensory neurons

Postdoctoral Fellowship, Mechanism or Pathway of Disease Deepika Mokkachamy Chellapandi, PhD,
Université Claude Bernard Lyon 1
FUNDED GRANT | Mar 2025

Characterization of ocular phenotype of the YG8-800 mouse model of FA and retinal-targeted treatment by AAV based CRISPR-Cas9 gene editing

General Research Grant, Gene & Stem Cell Therapy Shannon Boye, PhD,
University of Florida
FUNDED GRANT | Mar 2025

Unraveling Epigenetic Mechanisms and Therapeutic Strategies for FXN Silencing in Friedreich’s Ataxia

Postdoctoral Research Award, Mechanism or Pathway of Disease Xiaonan Guan, PhD ,
New York Genome Center
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
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