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FARA Research Priorities

The FARA grant program supports basic, translational and clinical research with the goal of advancing therapeutic development in FA. All proposed research must fall within FARA's Grant Program Priorities, which are as follows:
 

FARA Grant Program Priorities
Advancing understanding of neuroscience/ systems Understanding the neurodevelopmental and neurodegenerative changes of FA and their implications for pathogenesis and therapies. Special consideration will be given to proposals addressing non cell-autonomous mechanisms as contributors to the pathophysiology of FA and as in relation to the development of effective treatments.
Advancing understanding of cardiac disease in FA Understanding the pathogenesis of the cardiomyopathy associated with FA, identification and evaluation of potential biomarkers of disease and developing therapies to reduce the morbidity and mortality caused by cardiac disease. Special consideration will be given to proposals addressing the etiology of variable presentation of cardiac disease in FA.
Advancing understanding of the molecular basis of FA Understanding the normal function(s) of frataxin and the consequences of loss of frataxin, as well as the precise mechanisms of FXN gene silencing as they relate to the identification of novel therapeutic targets. Priority will be given to proposals that explore the metabolic basis of cell/tissue-type specific vulnerability, and the role of stress in regulating frataxin expression and function.
Advancing drug discovery Developing effective therapies for FA. Priority will be given to genetic, epigenetic, and protein replacement approaches that increase frataxin levels. FARA will consider high feasibility therapeutic discovery projects focused on pathways likely to affect disease progression and key symptoms.
Facilitating the drug development process and translational research FARA prioritizes funding of in vitro and in vivo preclinical studies aimed at facilitating the rapid translation to the clinic of promising therapeutic approaches. These include studies to evaluate mechanism of action and target engagement, drug efficacy, safety and toxicity profiles in animals, identification of lead candidates, exploration of drug delivery systems (with particular focus on non-viral gene and protein delivery). High priority will be given to the discovery and validation of clinical outcome assessments and biomarkers, such as development of methods to measure frataxin in affected tissues and identification of novel pharmacodynamic markers to evaluate the response to therapeutics in affected tissues, in early stage clinical trials.
Advancing clinical research FARA supports clinical research that informs and creates resources necessary for drug and clinical development, furthers our understanding of the natural history and improves clinical outcomes for those living with FA. These include natural history studies, biomarker and functional outcome measure discovery and validation, and investigator-initiated clinical trials.
Special consideration will be given to proposals addressing:
  • the clinical and metabolic characterization of fatigue in FA;
  • identification of early (including pre-symptomatic) quantifiable functional and clinical outcome measures for pediatric clinical trials;
  • identification of novel, clinically meaningful, functional endpoints with measurable changes detectable within one year, especially those based on the use of digital and at-home monitoring devices.
Priority will be given to proposals that utilize or expand resources of the Collaborative Clinical Research Network in FA

 
 
 

Grant program updates and announcements

View special interest RFPs and other grant program updates and announcements here.

Newly awarded grants

Cell & Animal Models

Paola Costantini, PhD - University of Padova, Italy​ Three-dimensional mature cardiac microtissues from human induced pluripotent stem cells to explore mitochondrial dynamics, cardiac function and therapeutic options in Friedreich Ataxia

Gene & Stem Cell Therapy​

Jill Napierala, PhD - University of Alabama at Birmingham​ Regulation of frataxin expression - implications for Friedreich's ataxia therapy​
Helene Puccio, PhD - Institute NeuroMyoGène, Lyon, France​ Defining the therapeutic window and threshold for neuronal gene therapy in Friedreich Ataxia​

Mechanism or Pathway of Disease​

Arnulf Koeppen, MD - VA Medical Center, Albany​ The pathogenesis of the major neural lesions in Friedreich ataxia: dorsal root ganglion and dentate nucleus​


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