Cystinosis Research Foundation

What it reported doing in 2024

EDUCATIONThe Cystinosis Research Foundation is dedicated to educating the cystinosis community, the public and the medical community about cystinosis to ensure early diagnosis and immediate and proper treatment.NATALIES WISHNatalies wish, To have my disease go away forever, was the driving force and inspiration that led to the establishment of the Cystinosis Research Foundation (CRF) in 2003. We knew at that moment that we needed to make every effort to make Natalies wish and the wish of others with cystinosis a reality.HISTORY AND RESEARCHCystinosis is a rare, metabolic, genetic disease that afflicts approximately 2,500 children and adults worldwide. CRF is increasingly focused on funding clinical and translational research which is crucial to finding new treatments and a cure for cystinosis. CRF is committed to prioritizing and aggressively supporting research that has the potential to significantly improve the quality of life for children and adults with cystinosis. RESEARCH GIVES US HOPEWe firmly believe that funding and supporting research gives our community hope for a brighter future for those living with cystinosis. Hope allows us to live with cystinosis until the day a cure is found. Since 2003, the Cystinosis Research Foundation (CRF) has raised over $73 million and has become the worlds leading fund provider of cystinosis research. CRF has strategically and aggressively invested millions of dollars into the CRF research program creating a thriving and collaborative global research community that has changed the course of cystinosis. From the beginning, all CRF operating costs have been privately underwritten, so 100 percent of all donations received by CRF go to support cystinosis research. CRF HAS CREATED A CONTINUOUS CYCLE OF RESEARCH STUDIES BY SEEKING NEW RESEARCH APPLICATIONS TWICE A YEAR. We are pleased to announce that during the 12-month period ending June 30, 2025, CRF announced seven new grantS, AND GRANT PAYMENTS totaling $2,326,589, TO bring us that much closer to better treatments and a cure. RESEARCH PROGRESSFROM THE START OF THE FOUNDATION, WE HAVE UNDERSTOOD THAT FUNDING BASIC AND BENCH RESEARCH WAS important to understanding the cystinosis. Over the years, as discoveries were made in the lab, CRF began funding clinical research, and now we are translating the DATA FROM the clinical studies To the patients. Our strategy of providing seed money to talented researchers has been successful. Our first project involved funding researchers who were focused on a new treatment. CRF provided grants that funded every bench and clinical trial that led to the discovery of a delayed-release form of the life-saving medication for cystinosis. That medication, Procysbi was approved by the FDA on April 30, 2013, and is considered the most significant advancement in the treatment of cystinosis in 30 years. In 2007, CRF began funding Dr. Stphanie Cherqui, at UC San Diego who was focused on stem cell and gene therapy research. As a direct result of CRFs early and continuous funding, in December 2018, the FDA approved a clinical trial to assess the efficacy and safety of a stem cell and gene therapy treatment for cystinosis patients. In October 2019, the first cystinosis patient received the stem cell gene therapy treatment. In 2020, two patients received the therapy. In November 2021, the fourth patient received the treatment and in March 2022, the fifth patient received the treatment. The sixth and final patient in Phase I/II of the clinical trial was treated in October 2022. THE GRANTS AWARDED TO DR. CHERQUI FOR HER STEM CELL WORK HAVE BEEN LEVERAGED BY MULTI-MILLION-DOLLAR GRANTS FROM OTHER FUNDING AGENCIES. IF THIS TREATMENT WORKS, IT COULD STOP THE PROGRESSION OF CYSTINOSIS OR BE THE CURE FOR CYSTINOSIS.Since cystinosis is a systemic disease and affects every cell, CRF has targeted multiple areas of research including kidney disease, muscle wasting, neurological issues, corneal cystinosis, and stem cell and gene th

Financial Efficiency

• Mission deployment rate: 96.7%; rating input score 97
• Program expense ratio: 89.4%; rating input score 89
• Fundraising efficiency: $14.27 raised per $1 spent on fundraising; rating input score 49; 49th percentile among comparable filings

Transparency & Controls

• Consistency checks: 93.8%; rating input score 94; 20th percentile among comparable filings
• Pay-setting controls: Limited
• Foreign activity oversight: 0 of 8 controls in place; rating input score 0
  • Claims monitoring procedures: Fail
  • Requires narrative reports: Fail
  • Conducts field audits: Fail
  • May suspend future funding: Fail
  • Requires financial reports: Fail
  • Conducts site visits: Fail
  • Requires grant agreements: Fail
  • May require repayment: Fail
• Grant itemization coverage: 50%; rating input score 50; 15th percentile among comparable filings
• Major donor itemization: Limited
• Related-party transparency: Limited

Accountability

• Independent voting board: 100%; rating input score 100; 63rd percentile among comparable filings
• Governance controls: 7 of 7 controls in place; rating input score 100
  • Conflict-of-interest policy: Pass
  • Whistleblower policy: Pass
  • Document-retention policy: Pass
  • CEO compensation review process: NotApplicable
  • Other executive compensation review process: NotApplicable
  • Form 990 shared with the board: Pass
  • Board meeting minutes documented: Pass
  • Audit committee oversight: Pass
  • Independent financial-statement audit: Pass

Sustainability

• Operating margin: -8.2%; rating input score 15; 15th percentile among comparable filings
• Total-asset coverage ratio: 2.92; rating input score 73; 73rd percentile among comparable filings
• Liabilities to assets ratio: 0.25; rating input score 39; 39th percentile among comparable filings
• Officer compensation ratio: 0%; rating input score 91; 91st percentile among comparable filings

Growth

• Program expense growth: 2.9% per year; rating input score 100; 27th percentile among comparable filings
• Revenue growth: 0.1% per year; rating input score 100; 12th percentile among comparable filings
• Asset growth: 8.5% per year; rating input score 100; 52nd percentile among comparable filings