Friedreich's Ataxia Research Alliance


533 W Uwchlan Ave
Downingtown, PA 19335
(484) 879-6160
http://www.curefa.org


   

TRAIN Inventory Profile

Mission
Strategy/Approach
Research Portfolio
Financials
Key Accomplishments
Leadership
Ask Us About
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Mission

The Friedreich’s Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), non-profit, tax-exempt organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich’s ataxia.

FARA's Mission is to marshal and focus the resources and relationships needed to cure FA by raising funds for research, promoting public awareness, and aligning scientists, patients, clinicians, government agencies, pharmaceutical companies and other organizations dedicated to curing FA and related diseases.

Strategy/Approach

Strategy Plan
Diverse and deep treatment pipeline.
Sustain and strengthen the Clinical Research Infrastructure to expedite and promote clinical research (Patient Registry, Clinical Research Network, Natural history studies, etc...).
Support and optimize the number of industry partners with a strategic interest in FA, with the goal of funding and advancing our therapeutic pipeline.
Grow and nurture the scientific community.
Promote academic and industry partnerships.
Address regulatory issues to facilitate approval of drugs in development.

Programs
Research grant program - FARA's commitment to advancing FA research towards treatments and a cure is executed through funding high quality research projects. FARA receives more than 50 grant applications/requests per year which go through a peer-review and internal scientific review process. In 2014, FARA funded 29 research grants.

Research conferences - FARA organizes and supports a number of scientific conferences to accelerate and promote sharing of knowledge, insights and build collaborations and synergistic connections between FA researchers.

Education and Awareness - FARA utilizes both traditional and social media strategies to bring greater disease awareness to the general public and to engage and educate the FA community.

Research Portfolio

FARA supports research through funding competitive grants, promoting collaboration among scientists, advocating for public-private partnerships that support drug discovery, drug development and clinical research and hosting open forums for leading scientists to share their insights, ideas and challenges to advancing treatments for FA.

  1. Advance understanding of neuroscience / neuro systems – understanding the neurodegeneration of FA and implications for therapies.
  2. Advance drug discovery-Highest priority in this category will be given to genetic, epigenetic and protein approaches that target increasing frataxin levels
  3. Facilitate the drug development process and translational research so that the most promising discoveries are rapidly brought to treatment trials. Highest priority in this category will be given to IND-enabling studies, biomarker discovery and validation, early phase or pilot clinical studies
  4. Advance clinical research – natural history, discovery and validation of clinical outcome measures and/or biomarkers, identification of early (including pre-symptomatic) quantifiable clinical features, patient reported outcomes, investigator-initiated clinical trials, or evidence-based clinical treatment guidelines. Highest priority in this category will be given to clinical research that utilizes or expands resources of the Collaborative Clinical Research Network in FA
  5. Reduce the morbidity and mortality caused by cardiac disease in FA

Financials

Fiscal year 2014

  • Revenue: $5,847,572
  • Assets: $4,011,934
  • Grants: $4,113,902
  • Gifts Received: $2,005,642
  • Total Expenditures: $5,426,973

Key Accomplishments

  • >$35 million in research funding
  • >820 individuals enrolled in Natural History study that is part of Collaborative Clinical Research Network, entering 11th year
  • >2400 individuals in Patient Registry which has successfully recruited more than 7 clinical trials
  • Treatment pipeline with 9 therapeutic candidates in clinical development and 7 candidates in pre-clinical development
  • 2013 Launched an Ambassador program to train individuals with the disease to be spokespersons and promote patient engagement in research
  • 2014 The Friedreich's Ataxia Center of Excellence was established at the University of Pennsylvania and Children's Hospital of Philadelphia with a gift of $3.25 million (over 3 yrs). 
  • 2014 First Concensus Clinical Management Guidelines for Friedreich's Ataxia published.
  • 2015 launched a Biomarker Consortium with 3 active studies that is partnership with advocacy, academic and industry organizations.
  • 2015 co-hosted largest scientific conference (>350 attendees) with international advocacy partners

Leadership

FARA has a volunteer board of directors made up of individuals who represent different stakeholders (patient families, researchers, industry, and donors) as well as core competency areas related to fiduciary responsibility, communications, and legal. FARA also has a volunteer scientific advisory board with both academic and industry representation.
FARA has an executive staff to administer programs.

Executive Director, Jennifer Farmer, jen.farmer@curefa.org, 484-879-6160

Ask Us About

  • Building research collaborations
  • Clinical trial design and recruitment
  • Maintaining a patient registry or biobank