Disease, Disorders, Medical Disciplines

CHOROIDEREMIA RESEARCH FOUNDATION INC

Saving Sight is our Vision

aka CRF, CHM Research Foundation, Cure CHM

Springfield, MA

Mission

To raise funds in support of scientific research leading to a treatment or cure of choroideremia, a hereditary retinal-degenerative disease that causes blindness; to educate people affected by the disease; and to inform the public.

Ruling Year

2001

Executive Director

Mrs. Kathi Wagner

Main Address

23 E Brundreth St

Springfield, MA 01109 USA

Keywords

CHM, rare eye disease, genetic eye disease, Choroideremia, retinal eye disease, RPE, research, eye disease, fundraising, REP-1, outreach, support

EIN

04-3511754

 Number

4633923674

Cause Area (NTEE Code)

Eye Diseases, Blindness and Vision Impairments (G41)

Fund Raising and/or Fund Distribution (H12)

Eye (H41)

IRS Filing Requirement

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

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Social Media

Programs + Results

What we aim to solve

Choroideremia is a rare, X-linked recessive form of hereditary retinal degeneration that affects roughly 1 in 50,000 males. The disease causes a gradual loss of vision, starting with childhood night blindness, followed by peripheral vision loss and progressing to loss of central vision later in life. Progression continues throughout the individual's life, but both the rate of change and the degree of visual loss are variable among those affected, even within the same family. Choroideremia is caused by a loss-of-function mutation in the CHM gene which encodes Rab escort protein 1 (REP1), a protein involved in lipid modification of Rab proteins. While the complete mechanism of disease is not fully understood, the lack of a functional protein in the retina results in cell death and the gradual deterioration of the retinal pigment epithelium (RPE), photoreceptors and the choroid. Currently, there is no treatment or cure for choroideremia.

Our programs

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

SOURCE: Self-reported by organization

Research

Where we work

Our Results

How does this organization measure their results? It's a hard question but an important one. These quantitative program results are self-reported by the organization, illustrating their committment to transparency, learning, and interest in helping the whole sector learn and grow.

SOURCE: Self-reported by organization

Number of Facebook followers

TOTALS BY YEAR
Population(s) served

Caregivers,

People with vision impairments

Related program

Research

Type of Metric

Context - describing the issue we work on

Direction of Success

Increasing

Total dollars received in contributions

TOTALS BY YEAR
Population(s) served

Caregivers,

People with vision impairments

Related program

Research

Type of Metric

Input - describing resources we use

Direction of Success

Increasing

Context notes

Donations as reported on IRS Form 990; increased revenue on even years is related to biennial conference.

Number of people on the organization's email list

TOTALS BY YEAR
Population(s) served

Caregivers,

People with vision impairments

Related program

Research

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Number of press releases developed and distributed

TOTALS BY YEAR
Population(s) served

Caregivers,

People with vision impairments

Related program

Research

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context notes

August 2019 - purchased 12 month subscription to EIN Newswire for 12 press releases, first release submitted in August 2019

Total number of conferences held

TOTALS BY YEAR
Population(s) served

Caregivers,

People with vision impairments

Related program

Research

Type of Metric

Outcome - describing the effects on people or issues

Direction of Success

Increasing

Context notes

Biennial conferences held in different US locations for approximately 300 attendees

Charting Impact

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

SOURCE: Self-reported by organization

What is the organization aiming to accomplish?

What are the organization's key strategies for making this happen?

What are the organization's capabilities for doing this?

How will they know if they are making progress?

What have they accomplished so far and what's next?

Gene Replacement Therapy 1. Continue collaboration with pharmaceutical industry partners to assist in clinical trial enrollment, outcome measure selection, and other items to achieve the most efficient and successful clinical trial. 2. Interact with members of regulatory agencies such as the FDA to provide disease-specific information about CHM. 3. Provide education to the CHM community regarding status of clinical trials to ensure that they are properly informed and have realistic expectations on timing and potential success of therapy. 4. Identify potential improvements to gene therapy with the ultimate goal of stopping the progression of retinal degeneration in CHM. Restorative Therapies 1. Identify pre-clinical research projects that have specific relevance to CHM patients in accelerating the potential development of restorative therapies. 2. Monitor the advancement of potential restorative therapies that have relevance to treatment of CHM patients outside of those requiring funding. 3. Advocate for the inclusion of CHM patients in proposed clinical trials for restorative therapies. 4. Develop basic informational materials regarding currently approved retinal implant devices and of relevant active research projects that could be used in CHM patients. Therapies to Stop or Slow Progression 1. Identify high-quality research projects that could lead to treatments which would stop or slow the progression of disease in Choroideremia patients. This will be expanded as a greater knowledge of the molecular mechanisms of disease progression is understood. 2. Interface with leaders in the scientific community to identify projects that could lead to treatments useful for CHM patients. Improved Understanding 1. Improve understanding of molecular mechanisms of CHM by 2020. 2. Clearly define cell type(s) directly affected by REP1 loss of function by 2023. 3. Continue advocacy efforts to pharmaceutical companies involved in current and future natural history studies to promote public access to the data gained from these studies. 4. Define a mechanism to store natural history data when it is made available in a format that could be easily accessed by the research community. Biobank and Registry 1. Establish an inventory of CHM cell models, antibodies, reagents, etc. to make available to entities engaged in CHM research and treatment. 2. Educate the research community on the availability of these scientific materials that could be accessed for use in research projects to make research programs more efficient and cost-effective. 3. Develop and promote a CRF registry with the goal of including > 500 affected individuals internationally. Meetings and Conferences 1. To host conferences, regional meetings, and scientific symposia for the CHM community including patients and families, clinicians, scientists, ad members of industry. 2. To establish an international panel of retinal & CHM experts to collaborate and discuss avenues of research.

Communication is ongoing with entities conducting clinical trials and preclinical research. CRF assists where needed to help encourage enrollment in studies, connect researchers with appropriate preclinical resources and provide funding for projects of interest. We continue to recruit new scientific advisory board members globally and are expanding outreach to multidisciplinary researchers publishing scientific papers. Regular meetings with scientists occur at professional and CRF meetings to facilitate information sharing and collaboration. The CRF Registry now contains contact information for 460 international patients who are interested in providing natural history and other information to help accelerate research. Participation in the registry will be promoted on our website, via email and and on social media channels. The next biennial Conference is scheduled for June 2020 in Rochester, NY. A new dual-track format will be initiated to provide programming for the entire CHM community including patients and families, clinicians, scientists, ad members of industry. A new website is under development for Fall 2019 which will provided a more targeted and customized CHM education for both medical professionals and patients/family members. Medical professionals will be directed to content including an inventory of commercially available CHM cell models, antibodies, reagents, etc. as well as to information regarding the latest in research findings and information on how to become involved with CRF's Scientific Advisory Board. Patients and family members will be directed to new content which will include a "Living with Choroideremia Toolkit", and additional webinar programs which will be developed in 2020. The website will also offer additional fundraising content to better support individuals seeking to make contributions and hold peer-to-peer events. New information about volunteer opportunities will increase engagement by members and leverage skill sets of patients/family members. By increasing fundraising and volunteer programs, we will be able to fund more research and provide additional education and advocacy programs for patients and caregivers. Several social media pages on various channels are also being consolidated to create consistency in branding and new logos and design elements will help to professionalize our image and consistency. A new CRM database has been purchased to automate and integrate transaction processing, accounting, and email systems reducing manual effort and improving efficiency and accuracy. The new database will also improve fundraising effectiveness via better reporting and communication tools.

The CRF historically has been, and continues to be, a Foundation primarily operated by volunteers. The Foundation has endeavored to keep costs and overhead low in an effort to maximize the percentage of donations provided directly to scientific research efforts. The growth of the CHM research platform, additional CRF initiatives, and the associated need for increased fundraising sparked evolution of that thinking. It has become more apparent to the Board of Directors that CRF needed to dedicate funds to its organizational structure to manage the ever-increasing technology driven environment. As a virtual organization, a strong structure becomes increasingly important to keep its processes running efficiently and to stay competitive with other fundraising organizations. In the past several years, CRF has begun to add paid staff positions to assist in the development and management of the organization. Starting initially with an Executive Director and Director of Operations, CRF expanded its team to include positions such as the Directors of Advocacy and Director of Patient Engagement in 2017. CRF will be celebrating its 20th year of service in 2020. The organization currently has 19 board members, 10 board committees, 11 Scientific Advisory Board Members, dozens of volunteers and 5 staff, including one full time, and 4 part time employees. Our budget for the 2019-20 Fiscal Year is approximately $565,000.

The board will annually review and update the Strategic Plan to identify areas of accomplishment, as well as areas still needed development, and new areas of focus. Regular surveys will be conducted to patients and family members to ensure we are meeting the education and advocacy needs of the community. Surveys will also be conducted following the Conference to determine its effectiveness.

Research – Early and ongoing investments and efforts are moving us closer to effective treatments for Choroideremia (CHM). As of 2018, we have awarded over $2.5 million to support research on causes and potential treatments. The CRF-funded biobank created from CHM patient samples is being used by researchers and sponsors in the US, Europe, and Canada. Two key collaborative projects are exploring the retina degeneration process to identify other treatment options, and new approaches for delivery of improved therapeutic agents for CHM. Gene Therapy Clinical trials – There are now clinical trials in several stages. Biogen is now in phase 3 studies; Spark Therapeutics has completed phase 1-2 studies; and 4D Molecular Therapeutics/Genentech expects to begin clinical trials in 2019. The CRF works with companies to connect them with our community; we play an essential role in recruiting study participants and study sites; we provide the patient voice to investigators to aid in study design; and we actively provide family and patient support. Partnering for advocacy and awareness – There are now over 30 entities working on various aspects of CHM studies and trials. We are working to build upon relationships with key figures at the FDA, EMA and the National Eye Institute and will be more than doubling the size of our Scientific Advisory Board. On Capitol Hill, CRF joined in advocacy that resulted in the recent $31 million increase in federal funds for retinal research. Our staff presents at conferences and panels across the U.S. and internationally. We represent patient needs as we address regulatory challenges for ongoing retinal disease research. International Role – CRF’s mission is international in scope. We serve Choroideremia patients worldwide; we convene and sponsor CHM research in many countries; we include international representatives on our Board of Directors; and we travel and partner to share our mission globally. Fiscal Year 2017-18 saw the launch and promotion of CRF’s new international CHM patient registry (CHMregistry.org), as well as plans for presentations at the CHM Frankfurt conference and three major, global summits in the U.S as well as a regional meeting in London in November, 2019. CRF’s International Choroideremia Scientific Symposium - This event was held in June 2019 and advanced discourse and collaborative thinking among international experts in Choroideremia and related inherited retinal dystrophies. Organizational growth – With our recent Strategic Plan, CRF’s Board has set the course to build organizational capacity, focusing on increased mission impact and longer-term sustainability. We’ve engaged additional staffing to further develop infrastructure and maximize the potential for reaching goals established in the Strategic Plan.

External Reviews

Photos

Financials

CHOROIDEREMIA RESEARCH FOUNDATION INC

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Operations

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

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Board Leadership Practices

GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section, which enables organizations and donors to transparently share information about essential board leadership practices.

SOURCE: Self-reported by organization

BOARD ORIENTATION & 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?

Not Applicable

CEO OVERSIGHT

Has the board conducted a formal, written assessment of the chief executive within the past year?

Not Applicable

ETHICS & TRANSPARENCY

Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year?

Not Applicable

BOARD COMPOSITION

Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership?

Not Applicable

BOARD PERFORMANCE

Has the board conducted a formal, written self-assessment of its performance within the past three years?

Not Applicable

Organizational Demographics

Who works and leads organizations that serve our diverse communities? This organization has voluntarily shared information to answer this important question and to support sector-wide learning. GuideStar partnered on this section with CHANGE Philanthropy and Equity in the Center.

SOURCE: Self-reported; last updated 09/04/2019

Leadership

The organization's leader identifies as:

Race & Ethnicity
White/Caucasian/European
Gender Identity
Female, Not Transgender (Cisgender)
Sexual Orientation
Heterosexual or Straight
Disability Status
Person without a disability

Race & Ethnicity

No data

Gender Identity

No data

Sexual Orientation

No data

Disability

No data