Cystinosis Research Foundation

Irvine, CA   |  www.cystinosisresearch.org

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.

Ruling year info

2003

President and Chair of the Board

Chair of the Board Nancy J. Stack

Main address

19200 Von Karman Ave Suite 920

Irvine, CA 92612 USA

Show more contact info

EIN

32-0067668

NTEE code info

Fund Raising and/or Fund Distribution (H12)

Research Institutes and/or Public Policy Analysis (B05)

IRS filing requirement

This organization is required to file an IRS Form 990 or 990-EZ.

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Communication

Programs and results

What we aim to solve

SOURCE: Self-reported by organization

Cystinosis - is an extremely rare (striking an estimated 2,000 people worldwide) metabolic disease in which the amino acid cystine gets into the cells, but has no transporter out. Because of the defect in transportation, the cell crystallizes causing early cell death. Cystinosis slowly destroys the organs in the body including the kidneys, liver, eyes, muscles, and brain. With such a rare disease affecting such a small population, money for cystinosis research is scarce to nonexistent. The Cystinosis Research Foundation's mission is to support bench, clinical and translational cystinosis research to find better treatments and a cure for cystinosis.

Our programs

SOURCE: Self-reported by organization

What are the organization's current programs, how do they measure success, and who do the programs serve?

CRF International Cystinosis Research Symposium - Building our Research Community

CRF’s 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.

Population(s) Served
Adults
Children and youth

CRF has an aggressive and strategic research agenda. CRF issues grants for bench, clinical and translational research studies bi-annually in an effort to accelerate the research process and ensure that cystinosis research is on-going and focused on novel treatments and a cure.  CRF has issued 187 multi-year research grants in 12 countries and those researchers have published over 80 articles in prestigious journals. As a result of donor support, we have funded researchers whose work has exponentially increased the breadth and knowledge about cystinosis and as a result of that knowledge, new discoveries have been achieved.

Population(s) Served
Adults
Children and youth

CRF research grants have resulted in two FDA approvals. CRF awarded several grants to improve the treatment of cystinosis and those research studies resulted in the 2013 FDA approval of a new medication that improved the quality of life for our patient community. In 2006, CRF awarded its first grant to Dr. Stephanie Cherqui at UC San Diego that was focused on stem cell therapy as a possible cure for cystinosis. In December 2018, the FDA approved the first stem cell and gene therapy clinical trial for cystinosis based on Dr. Cherqui’s work. We believe that the stem cell treatment will be a cure for cystinosis.

Population(s) Served
Adults
Children and youth

Where we work

Our results

SOURCE: Self-reported by organization

How does this organization measure their results? It's a hard question but an important one.

Number of research studies funded

This metric is no longer tracked.
Totals By Year
Population(s) Served

Children and youth, Adults

Related Program

Progress for Improved Treatment - Research

Type of Metric

Context - describing the issue we work on

Direction of Success

Increasing

Context Notes

The Cystinosis Research Foundation was formed in 2003, since that time CRF has funded 204 multi-year cystinosis research grants in 12 countries around the globe.

Our Sustainable Development Goals

SOURCE: Self-reported by organization

Learn more about Sustainable Development Goals.

Goals & Strategy

SOURCE: Self-reported by organization

Learn about the organization's key goals, strategies, capabilities, and progress.

Charting impact

Four powerful questions that require reflection about what really matters - results.

Through our aggressively funded research agenda, the Cystinosis Research Foundation issues grants for bench and clinical research studies bi-annually in order to accelerate research progress and ensure that cystinosis research is on-going and focused on novel treatments and a cure. The Cystinosis Research Foundation is dedicated to educating the public and the medical community about cystinosis to ensure early diagnosis and proper treatment. CRF is the largest private fund provider of cystinosis research grants in the world. CRF has raised over $60 million for cystinosis research. It has awarded 204 multi-year grants and is funding researchers in 12 countries.
The CRF was established with the sole purpose of raising funds to find better treatments and a cure for cystinosis.

Funding quality cystinosis research studies remains a priority and is an ongoing process. The CRF announces global calls for research proposals bi-annually in the spring and fall of each year. After review and recommendations by our Scientific Review Board, research grants are issued. In 2006, CRF established the first Cystinosis Research Fellowship Program designed to support scientists and new researchers who have an interest in cystinosis research.
In 2020, CRF funded more than $3 million for 23 cystinosis research studies covering muscle wasting, new drug discoveries, stem cell therapy, corneal cystinosis treatment, cognitive dysfunction, and molecular mechanisms in cystinosis.

Today, with the support of our cystinosis family partners, friends and community we have significantly changed the course of cystinosis research. Our funding efforts have allowed talented doctors and researchers in the area of cystinosis to initiate novel research studies and to advance their research efforts. in 2020, CRF funded $3,150,914 for 23 new research studies which included 14 new grants, 8 extension grants, and 1 equipment grant. CRF is the largest private fund provider of cystinosis research in the world.

In 2018 the FDA approved a stem cell and gene therapy clinical trial for cystinosis. In October 2019, the first patient was transplanted, in June 2020 the second patient was transplanted and in November 2020, the third patient was transplanted. We are pleased to report that all three patients are doing well and are off cysteamine treatment. We are optimistic that this treatment will stop the progression of cystinosis or be the cure for cystinosis. We are thankful to Dr. Cherqui of the University of California, San Diego who has dedicated her career to finding a cure for cystinosis and leading the clinical trial.

CRF's dedication to educating the public and the medical community about cystinosis to ensure early diagnosis and proper treatment has made a tremendous difference by improving the quality of life for numerous patients and families around the world. One accomplishment began with the CRF funded research at the University of California, San Diego (UCSD) which resulted in the development of a slow-release form of the drug cysteamine. On April 30, 2013, the FDA approved the new drug Procysbi™. To date, the CRF has funded and committed more than $60 million to cystinosis research and has awarded 204 research studies and fellowships in 12 countries.

Financials

Cystinosis Research Foundation
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Operations

The people, governance practices, and partners that make the organization tick.

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Connect with nonprofit leaders

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  • Compare nonprofit financials to similar organizations

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Cystinosis Research Foundation

Board of directors
as of 1/28/2021
SOURCE: Self-reported by organization
Board co-chair

Founding Trustee Nancy Stack

Cystinosis Research Foundation

Term: 2003 -


Board co-chair

Mr. Geoffrey L. Stack

SARES•REGIS Group

Term: 2003 -

Nancy Stack

Cystinosis Research Foundation

Geoffrey Stack

SARES REGIS Group

Donald Solsby

Compass Direct

Marcu Alexander

Hearts for Hadley

Stephanie Cherqui

University of California, San Diego

Bruce Crair

Boingo Wireless, Inc.

Denice Flerchinger

Tina's Hope For A Cure

Thomas Gendron

Woodward, Inc.

Traci Gendron

Tanner's Time for a Cure

John Hagestad

SARES REGIS Group

Michael Hayde

Western National Group

Stephen Jenkins

Sam's Hope for a Cure

Erin Little

Liv-A-Little Foundation

David Mossman

Donahue Schriber Realty Group

Kevin Partington

Cushman & Wakefield

Teresa Partington

Jenna & Patrick's Foundation of Hope

Brian Sturgis

24 Hours for Hank

Lauren Hartz

Lots of Love for Landon

Natalie Stack

Patient Advocate

Jill Emerson

Hope for Brooke

Board leadership practices

SOURCE: Self-reported by organization

GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.

  • Board orientation and education
    Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations? No
  • CEO oversight
    Has the board conducted a formal, written assessment of the chief executive within the past year ? No
  • Ethics and transparency
    Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year? Yes
  • Board composition
    Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership? Yes
  • Board performance
    Has the board conducted a formal, written self-assessment of its performance within the past three years? No

Organizational demographics

SOURCE: Self-reported; last updated 1/28/2021,

Who works and leads organizations that serve our diverse communities? GuideStar partnered on this section with CHANGE Philanthropy and Equity in the Center.

Leadership

The organization's leader identifies as:

Race & ethnicity
White/Caucasian/European
Gender identity
Female

Race & ethnicity

No data

Gender identity

No data

 

No data

Sexual orientation

No data

Disability

No data