Cystinosis Research Foundation
Cystinosis Research Foundation
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About this organization
Mission
The Cystinosis Research Foundation is dedicated to finding better treatments to improve the quality of life for those with cystinosis and to ultimately find a cure for this devastating disease. CRF is committed to finding a cure through an aggressively funded research agenda. CRF issues research grants bi-annually to accelerate the research process and to ensure that there is never a gap in funding new cutting-edge research ideas. We are focused and determined to improve the quality of life for our patients and to find better treatments, including a cure, for our adults and children living with cystinosis.
About
NATALIE'S WISH: 'TO HAVE MY DISEASE GO AWAY FOREVER' - THOSE SEVEN WORDS PROVIDED THE INSPIRATION TO ESTABLISH THE CYSTINOSIS RESEARCH FOUNDATION (CRF) IN 2003. WE KNEW AT THAT MOMENT THAT WE NEEDED TO MAKE EVERY EFFORT TO MAKE NATALIE'S WISH - AND THE WISHES OF OTHERS WITH CYSTINOSIS - A REALITY.RESEARCHCRF IS DEDICATED TO FINDING BETTER TREATMENTS TO IMPROVE THE QUALITY OF LIFE FOR THOSE WITH CYSTINOSIS AND TO ULTIMATELY FIND A CURE FOR THIS DEVASTATING DESEASE. CRF HAS AN AGGRESSIVE RESEARCH AGENDA. CRF ISSUES GRANTS FOR BENCH AND CLINICAL RESEARCH STUDIES BI-ANNUALLY IN AN EFFORT TO ACCELERATE THE RESEARCH PROCESS AND ENSURE THAT CYSTINOSIS RESEARCH IS ONGOING AND FOCUSED ON NOVEL TREATMENTS AND A CURE. HISTORY AND RESEARCH Cystinosis is a rare, metabolic and genetic disease that afflicts approximately 2,000 children and adults worldwide. CRFs focus is increasingly on the clinical and translational research crucial to making strides towards a cure for cystinosis. CRF is committed to prioritizing and aggressively supporting research which has the potential to significantly improve the quality of life for patients with cystinosis.CRF announces two global calls for research proposals each year. After careful evaluation of every application by the CRF Scientific Review Board, research grants are awarded. In 2006, CRF established the first Cystinosis Research Fellowship Program designed to support scientists and new researchers who have an interest in cystinosis. RESEARCH GIVES US HOPE We firmly believe that research gives us HOPE and hope allows us to live with cystinosis until the day a cure is found. Since 2003, the Cystinosis Research Foundation (CRF) has become the largest fund provider of cystinosis research in the world raising more than $50 million. CRF has changed the course of cystinosis by investing donors gifts strategically and aggressively to create a thriving research community. From the beginning, all CRF operating costs are privately underwritten so that 100 percent of the funds donated goes to support cystinosis research. We are pleased to announce that DURING THE 12 MONTHS PERIOD ENDING JUNE 30, 2019, CRF AWARDED 13 new grants totaling more than $3.3 million for cystinosis research. The new grants bring us that much closer to better treatments and a cure.CRF has issued grants FUNDED AND ONGOING STUDIES to the best and brightest researchers in the world. To date, we have funded 187 multi-year research studies at leading research institutions in 12 countries. CRF-funded researchers have published more than 77 articles in prestigious, top-rated journals. Those articles, available to everyone in the world, have added to the understanding of the pathogenesis of cystinosis.FDA APPROVALS From the beginning, we have understood that funding basic and bench research was important to understanding 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. One of our earliest milestones, and the one of which we are most proud, is that CRF funded every bench and clinical trial that led to the discovery of a delayed-release form of the life-saving medication cystinosis patients must take. The delayed-release medication, approved by the FDA in 2013, is considered the most significant advancement in the treatment of cystinosis in 30 years. IN DECMBER, 2018 THE FDA APPROVED THE FIRST STEM CELL AND GENE THERAPY TRANSPLANT FOR CYSTINOSIS.THE CLINICAL TRIAL, LED BY DR. STEPHANIE CHERQUI, WILL TAKE PLACE AT UC SAN DIEGO. THE CRF FUNDED DR. CHERQUI SINCE 2006. CRF FUNDS HAVE SUBSEQUENTLY BEEN LEVERAGED BY GRANTS FROM OTHER FUNDING AGENCIES WHICH HAVE HELPED ACCERLERATE HER RESERACH. IF THIS TREATMENT WORKS, IT COULD BE THE CURE FOR CYSTINOSIS,Moving forward, we have targeted several areas of research including muscle wasting, neurological issues, corneal cystinosis, and stem cell and gene therapy, all with the goal of finding better treatments and a cure for cystinosis.NANOTECHNOLOGY AND CORNEAL CYSTINOSISWe are dedicated to finding better treatments for corneal cystinosis. Corneal cystinosis is the build-up of cystine crystals in the eyes that causes photophobia (extreme sensitivity to light) severe eye pain, and sometimes, blindness. There is an existing treatment but it is rigorous and requires hourly dosing of medicated eye drops. For many, compliance is difficult. In 2016, CRF formed Nanowafer, Inc., FORMALLY KNOWN AS CORNEAL CYSTINOSIS,INC.,for the purpose of research and development of a NOVEL treatment for corneal cystinosis.THE TREATMENT PROPOSES TO USE A NANOWAFER LOADED WITH CYSTEAMINE MEDICATION TO TREAT CORNEAL CYSTINOSIS. CRF OWNS THE LICENSE FOR THE CYSTEAMINE NANOWAFER WHICH WAS DISCOVERED BY A SCIENTIST AT BAYLOR COLLEGE OF MEDICINE. CYSTINOSIS RESEARCH HELPS OTHERSMany of the discoveries made by CRF researchers are currently being applied to other more prevalent and well-known disorders and diseases including other corneal diseases, kidney diseases and genetic and systemic diseases similar to cystinosis. Support for cystinosis research has reached far beyond the cystinosis community. A cure for cystinosis will help find cures for other diseases potentially helping millions of people. CRF FAMILIES AND PARTNERSThe Cystinosis Research Foundation has witnessed tremendous growth as other cystinosis families have joined our efforts. Their friends and families have embraced the cystinosis cause and enthusiastically raise funds to support cystinosis research through CRF. We have found strength in numbers and our joint effort gives us a renewed sense of community and purpose.DAY OF HOPE CONFERENCEEach year the Cystinosis Research Foundation hosts the Day of Hope Family Conference. In 2019, 66 families from around the world gathered along with CRF funded researchers to share stories, hopes and dreams and build life-long friendships. Leading cystinosis researchers shared their progress on gene and stem cell therapies, novel eye research and neurological challenges related to cystinosis. The conference allows families to learn more about the ongoing cystinosis research and the hope it brings to our community. CURE CYSTINOSIS INTERNATIONAL REGISTRYThe Cure Cystinosis International Registry (CCIR) was launched in 2010 and now has approximately 576 cystinosis patients registered from 44 countries. The Cystinosis Research Foundation in partnership and collaboration with the Cystinosis Foundation and twelve other cystinosis family foundations from around the world, established this comprehensive, global patient registry for cystinosis. The CCIR questionnaire was developed by medical and scientific experts specifically for the cystinosis community. To improve accessibility, the CCIR has been translated into Spanish, Portuguese, Dutch and French. It is our mission to register every person with cystinosis, thus connecting them to the research community in an effort to find a cure for cystinosis. The CCIR is a central hub of information and is an essential tool for the global cystinosis research community. We are certain that the information provided by the patients will accelerate the research process and unite us in our quest to find a cure for cystinosis. CRF INTERNATIONAL RESEARCH SYMPOSIUMCRFs International Research Symposium is held every two years and is only open to invited researchers and scientists. Approximately 65 cystinosis experts from leading international universities and research institutions gather to share their research data and to discuss cutting edge research ideas in an effort to accelerate research. The symposium is known for the openness of the attendees, and for their willingness to work together and form collaborations. The symposium has created a synergistic research community working in partnership with CRF.
Interesting data from their 2020 990 filing
The mission of the non-profit, as stated in the filing, is “Crf's mission is to support bench, clinical and translational research to find better treatments and a cure for cystinosis”.
When referring to its tasks, they were described as: “Crf's mission is to support bench, clinical and translational research to find better treatments and a cure for cystinosis”.
- The non-profit is operating legally in the state of CA.
- The non-profit's address for the year 2020 is listed as 19200 VON KARMAN AVENUE 920, IRVINE, CA, 92612 in the filing.
- The form submitted by the non-profit organization for 2020 reports 1 employees.
- Does not operate a hospital.
- Does not operate a school.
- Does not collect art.
- Does not provide credit counseling.
- Has foreign activities.
- Is not a donor advised fund.
- Is not a private foundation.
- Expenses are greater than $1,000,000.
- Revenue is greater than $1,000,000.
- Revenue less expenses is -$467,694.
- The CEO's salary plan within the organization is not subject to review and approval from a neutral entity.
- The organization has a written policy that describes how long it will retain documents.
- The organization has 20 independent voting members.
- The organization was formed in 2003.
- The organization has a written policy that addresses conflicts of interest.
- The organization is required to file Schedule B.
- The organization is required to file Schedule O.
- The organization pays $63,625 in salary, compensation, and benefits to its employees.
- The organization pays $494,747 in fundraising expenses.
- The organization provides Form 990 to its governing body.
- The organization has minutes of its meetings.
- The organization has a written whistleblower policy.
- The organization has fundraising events.
- The organization has grants to organizations.
- The organization's financial statements were reviewed by an accountant.
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