PLATINUM2024

Cystinosis Research Foundation

Irvine, CA   |  www.cystinosisresearch.org
GuideStar Charity Check

Cystinosis Research Foundation

EIN: 32-0067668


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

Subject area info

Education

Health

Musculoskeletal diseases

Population served info

Children and youth

Adults

NTEE code info

Fund Raising and/or Fund Distribution (H12)

Research Institutes and/or Public Policy Analysis (B05)

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 ongoing and focused on novel treatments and a cure.  CRF has issued 220 multi-year research grants in 12 countries and those researchers have published over 103 articles in prestigious science 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

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

The Cystinosis Research Foundation was formed in 2003, since that time CRF has funded 235 multi-year cystinosis research grants in 13 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 ongoing 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 220 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
Fiscal year: Jul 01 - Jun 30

Revenue vs. expenses:  breakdown

SOURCE: IRS Form 990 info
NET GAIN/LOSS:    in 
Note: When component data are not available, the graph displays the total Revenue and/or Expense values.

Liquidity in 2023 info

SOURCE: IRS Form 990

6.22

Average of 3.49 over 10 years

Months of cash in 2023 info

SOURCE: IRS Form 990

25.3

Average of 18.3 over 10 years

Fringe rate in 2023 info

SOURCE: IRS Form 990

8%

Average of 7% over 10 years

Funding sources info

Source: IRS Form 990

Assets & liabilities info

Source: IRS Form 990

Financial data

SOURCE: IRS Form 990

Cystinosis Research Foundation

Revenue & expenses

Fiscal Year: Jul 01 - Jun 30

SOURCE: IRS Form 990 info

Fiscal year ending: cloud_download Download Data

Cystinosis Research Foundation

Balance sheet

Fiscal Year: Jul 01 - Jun 30

SOURCE: IRS Form 990 info

The balance sheet gives a snapshot of the financial health of an organization at a particular point in time. An organization's total assets should generally exceed its total liabilities, or it cannot survive long, but the types of assets and liabilities must also be considered. For instance, an organization's current assets (cash, receivables, securities, etc.) should be sufficient to cover its current liabilities (payables, deferred revenue, current year loan, and note payments). Otherwise, the organization may face solvency problems. On the other hand, an organization whose cash and equivalents greatly exceed its current liabilities might not be putting its money to best use.

Fiscal year ending: cloud_download Download Data

Cystinosis Research Foundation

Financial trends analysis Glossary & formula definitions

Fiscal Year: Jul 01 - Jun 30

SOURCE: IRS Form 990 info

This snapshot of Cystinosis Research Foundation’s financial trends applies Nonprofit Finance Fund® analysis to data hosted by GuideStar. While it highlights the data that matter most, remember that context is key – numbers only tell part of any story.

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Business model indicators

Profitability info 2019 2020 2021 2022 2023
Unrestricted surplus (deficit) before depreciation -$15,019 $748,924 $950,606 -$238,394 $454,245
As % of expenses -0.3% 22.4% 27.5% -7.8% 15.9%
Unrestricted surplus (deficit) after depreciation -$15,219 $732,442 $932,718 -$256,283 $436,357
As % of expenses -0.3% 21.8% 26.9% -8.3% 15.2%
Revenue composition info
Total revenue (unrestricted & restricted) $5,402,719 $4,308,424 $3,809,513 $3,589,447 $3,244,975
Total revenue, % change over prior year 0.0% -20.3% -11.6% -5.8% -9.6%
Program services revenue 0.0% 0.0% 0.0% 0.0% 0.0%
Membership dues 0.0% 0.0% 0.0% 0.0% 0.0%
Investment income 1.0% 2.1% 3.2% 6.0% 7.5%
Government grants 0.0% 0.0% 0.0% 0.0% 0.0%
All other grants and contributions 99.0% 94.3% 96.0% 81.4% 89.2%
Other revenue 0.0% 3.6% 0.7% 12.6% 3.4%
Expense composition info
Total expenses before depreciation $5,870,213 $3,346,208 $3,452,952 $3,054,824 $2,853,832
Total expenses, % change over prior year 0.0% -43.0% 3.2% -11.5% -6.6%
Personnel 1.1% 2.0% 2.0% 2.3% 2.4%
Professional fees 0.8% 2.9% 2.4% 3.6% 4.2%
Occupancy 0.0% 0.0% 0.0% 0.0% 0.0%
Interest 0.0% 0.0% 0.0% 0.0% 0.0%
Pass-through 63.5% 67.3% 80.9% 70.5% 57.8%
All other expenses 34.5% 27.8% 14.7% 23.7% 35.6%
Full cost components (estimated) info 2019 2020 2021 2022 2023
Total expenses (after depreciation) $5,870,413 $3,362,690 $3,470,840 $3,072,713 $2,871,720
One month of savings $489,184 $278,851 $287,746 $254,569 $237,819
Debt principal payment $0 $0 $0 $0 $0
Fixed asset additions $33,864 $71,495 $0 $0 $0
Total full costs (estimated) $6,393,461 $3,713,036 $3,758,586 $3,327,282 $3,109,539

Capital structure indicators

Liquidity info 2019 2020 2021 2022 2023
Months of cash 15.5 21.5 20.7 24.2 25.3
Months of cash and investments 18.3 31.5 33.3 39.3 43.0
Months of estimated liquid unrestricted net assets 13.9 26.8 29.2 32.1 36.3
Balance sheet composition info 2019 2020 2021 2022 2023
Cash $7,593,080 $5,984,805 $5,968,759 $6,158,524 $6,005,918
Investments $1,382,144 $2,812,223 $3,619,845 $3,836,415 $4,212,457
Receivables $549,803 $912,433 $600,684 $30,933 $0
Gross land, buildings, equipment (LBE) $90,702 $162,197 $162,197 $162,197 $162,197
Accumulated depreciation (as a % of LBE) 62.9% 45.3% 56.4% 67.4% 78.4%
Liabilities (as a % of assets) 29.7% 23.3% 18.0% 19.1% 15.9%
Unrestricted net assets $6,817,282 $7,549,724 $8,482,442 $8,226,159 $8,662,516
Temporarily restricted net assets $0 N/A N/A N/A N/A
Permanently restricted net assets $0 N/A N/A N/A N/A
Total restricted net assets $0 $0 $0 $0 $0
Total net assets $6,817,282 $7,549,724 $8,482,442 $8,226,159 $8,662,516

Key data checks

Key data checks info 2019 2020 2021 2022 2023
Material data errors No No No No No

Operations

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

Documents
Form 1023/1024 is not available for this organization

President and Chair of the Board

Chair of the Board Nancy J. Stack

Nancy has been involved in various fundraising activities including political fundraising, non-profit work and charitable organizations for many years. Her interest in the non-profit world became much more personal in 1991, when second daughter Natalie was born and diagnosed with cystinosis. In 2003 Nancy and her husband Geoffrey established the Cystinosis Research Foundation to improve the quality of life for those living with cystinosis and to find more effective treatments and a ultimately a cure. Nancy holds a BA degree from University of California, Los Angeles, a MA degree from University of Southern California and a JD from Western State College of Law. Nancy, a licensed attorney, has made the CRF her full-time commitment. Jeff and Nancy live in Corona Del Mar, California and have two daughters, Alexandra and Natalie.

Number of employees

Source: IRS Form 990

Cystinosis Research Foundation

Officers, directors, trustees, and key employees

SOURCE: IRS Form 990

Compensation
Other
Related
Show data for fiscal year
Compensation data
Download up to 5 most recent years of officer and director compensation data for this organization

There are no highest paid employees recorded for this organization.

Cystinosis Research Foundation

Board of directors
as of 06/05/2024
SOURCE: Self-reported by organization
Board of directors data
Download the most recent year of board of directors data for this 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

Technology Transformation

Denice Flerchinger

Tina's Hope For A Cure

Stephen Jenkins

Sam's Hope for a Cure

David Mossman

Donahue Schriber Realty Group

Kevin Partington

Cushman & Wakefield

Teresa Partington

Jenna & Patrick's Foundation of Hope

Brian Sturgis

24 Hours for Hank

Jill Emerson

Hope for Brooke

Clay Emerson

Hope for Brooke

Angela Kirchhof

Hope for Hayden

Barbara Kulyk

Kristen Murray

Seth's Circle of Hope

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.

  • 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

Organizational demographics

SOURCE: Self-reported; last updated 6/5/2024

Who works and leads organizations that serve our diverse communities? Candid partnered with CHANGE Philanthropy on this demographic section.

Leadership

The organization's leader identifies as:

Race & ethnicity
White/Caucasian/European
Gender identity
Female

Race & ethnicity

Gender identity

No data

Transgender Identity

No data

Sexual orientation

No data

Disability

No data