The Choroideremia Research Foundation

What The Choroideremia Research Foundation does

The Choroideremia Research Foundation (CRF), founded in 2000 and headquartered in Springfield, Massachusetts, is a US 501(c)(3) nonprofit patient-advocacy and research-funding organization dedicated to choroideremia (CHM), a rare X-linked inherited retinal disease causing progressive vision loss and total blindness. CRF operates as a small central team of 5 staff supported by a 20+ person volunteer board, and is governed as an independent nonprofit with no parent organization. It is the largest organization in the world solely focused on choroideremia, with 4,375+ CHM family members connected, 160+ International Choroideremia Research Network (ICRN) members from 25 countries, and more than $6 million invested across 100+ research grants since inception.

CRF's core offerings are research funding programs (Randy Wheelock Research Award, General Research Grants, John Oster Leadership Award), research infrastructure (a CHM Biobank with iPSC, fibroblast, DNA, and LCL lines housed at WiCell and Coriell; CHM mouse models; a natural history data repository), patient resources (Eye Donation Program via Eversight, clinical trial information), and community programs (ICRN, Candle resource hub, biennial international conference, Team CHM athletic fundraising). A pivotal 2025 move was the acquisition of all Spark Therapeutics assets for SPK-7001 (AAV2-hCHM-101), including intellectual property, orphan drug designations, and Phase I/II clinical trial data, marking CRF's expansion from pure grantmaker into therapeutic-asset steward. The foundation's partnerships with 4D Therapeutics (intravitreal gene therapy), Biogen and RDCA-DAP (natural history data sharing), and Kiora Pharmaceuticals (KIO-301) extend its role across the translational pipeline.

CRF's revenue model is donation-dependent: individual/family contributions, monthly giving, planned/legacy gifts (including QCDs and bequests), event fundraising, membership programs, and merchandise. Grantmaking is the primary outflow — $335,000 in new 2026 grants, $50,000 Randy Wheelock Award in October 2025, plus six new 2025 grants including a $61,273 deep-learning/molecular-glue grant and a $65,000 CRISPR gene-editing grant. The organization holds a Charity Navigator 100/100 rating and a 2026 Candid Platinum Transparency seal, reflecting high operational accountability. Distribution is community-led: curechm.org, multi-platform social (YouTube, LinkedIn, Instagram, Facebook, X, Bluesky), email, the biennial conference, and Rare Disease Week advocacy.

Name

The Choroideremia Research Foundation

Legal name

The Choroideremia Research Foundation Inc.

Website

https://curechm.org

Company type

Private

Founded year

2000

Operating status

Operating

Headcount range

1–10 employees

Short description

The Choroideremia Research Foundation is a US 501(c)(3) nonprofit that funds choroideremia (CHM) research, supports CHM patients and carrier families, and stewards acquired gene therapy assets, serving 4,375+ community members and 160+ researchers across 25 countries.

Ownership category

akta.pro rank

Product category

Patient Advocacy and Rare Disease Research Foundation

NAICS

Scientific Research and Development Services (5417), Research and Development in Biotechnology (except Nanobiotechnology) (541714), Research and Development in the Physical, Engineering, and Life Sciences (54171)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Patient Recruitment, Site Feasibility & Trial Enablement CROs (HLAGAAAI)

akta.pro secondary industries

Preclinical CROs (Discovery/Pharmacology/DMPK) (HLAGAAAB), Clinical Data Management, Biostatistics & Statistical Programming CROs (HLAGAAAF)

Keywords

• Rare disease research
• Patient advocacy services
• Research grant funding
• Vision research foundation
• Gene therapy research

Headquarters

HQ city

Springfield

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel

Revenue model

1. Donations and Charitable Contributions: CRF relies primarily on donations from individuals, families, and supporters affected by CHM. This includes one-time donations, monthly giving programs, and major gifts.
2. Research Grants: CRF funds research through grants awarded to researchers worldwide. In 2026, CRF announced four new global research grants totaling approximately $335,000.
3. Membership Programs: CRF offers membership programs that provide community connection and support for CHM patients and families.
4. Event Fundraising: Team CHM participates in athletic events like marathons, with fundraisers receiving swag packs. The 2025 International Conference in Minnesota had nearly 200 attendees.
5. Planned Giving and Estate Gifts: CRF accepts bequests, life insurance beneficiary designations, and Qualified Charitable Distributions (QCDs) from IRAs for donors aged 70½ and older.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels12 records

Core offering

The Choroideremia Research Foundation (CRF) is a 501(c)(3) nonprofit patient advocacy organization that funds choroideremia (CHM) research through grants, connects CHM patients and families globally through conferences and support programs, provides educational resources and clinical trial information, and advocates for CHM awareness. The organization has invested over $6 million in research since 2000 across more than 100 grants, supports a biobank of CHM cellular resources, and acquired Spark Therapeutics' SPK-7001 gene therapy assets in 2025 to advance treatment development.

Product overview

The Choroideremia Research Foundation (CRF) operates as a nonprofit patient advocacy organization rather than a traditional product company. Its offerings consist of research funding programs (Randy Wheelock Research Award, General Research Grants, John Oster Leadership Award), research infrastructure (CHM Biobank with iPSC models at WiCell/Coriell, CHM Mouse Models, Natural History Data Repository), patient resources (Eye Donation Program, Clinical Trial Information), and community programs (International Choroideremia Research Network with 160+ members, Candle CHM Resource Hub, Annual International Conference, Team CHM athletic fundraising). The Foundation also supports its mission through merchandise sales and planned giving options. The core value proposition centers on accelerating choroideremia research through strategic funding, global collaboration, and open data sharing.

Differentiator

Problem solved

Functional benefit

Quantifiable outcome

• $6+ million invested in research since 2000
• +3 more outcomes

Named customers6 records

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Partnerships

Eleven partnerships are on record, tiered core, major and minor.

• Spark Therapeutics, Inc.coreStrategic or Co-development Partner · 23 January 2025In 2025, CRF acquired all Spark Therapeutics assets related to SPK-7001 gene therapy for Choroideremia, including intellectual property rights, biological materials, orphan drug designations, and Phase I/II clinical trial data. CRF is now analyzing five-year follow-up data from the trial alongside natural history data from 125+ CHM patients to identify ophthalmic endpoints for regulatory approval.
• BiogenmajorStrategic or Co-development Partner · 1 January 2024CRF partnered with Biogen and the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) to make depersonalized CHM natural history data available to qualified researchers worldwide. This includes data from STAR trial (NCT03496012), NightstaRx/Biogen natural history studies, and Biogen-sponsored trials.
• 4D TherapeuticscoreStrategic or Co-development PartnerCRF partnered with 4D Therapeutics to develop a novel CHM gene therapy vector designed for delivery through intravitreal injection (front of the eye), potentially allowing it to reach more retinal cells than traditional subretinal delivery. This trial is in early stages.
• Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP)majorStrategic or Co-development PartnerCRF works with C-Path's RDCA-DAP to provide access to CHM natural history and clinical trial data, strengthening global collaboration and helping propel progress toward future treatments.
• EversightcoreStrategic or Co-development PartnerCRF partners with Eversight, a nonprofit dedicated to restoring sight and preventing blindness through donation, transplantation, and research, for the CHM Eye Donation Program. Eversight facilitates eye donation from CHM patients and carriers in Michigan, Illinois, Ohio, New Jersey, and Connecticut.
• University of VirginiacoreStrategic or Co-development PartnerCRF collaborates with University of Virginia faculty members Dr. Tom Barker and Dr. Shannon Barker, along with CRF Chief Science Officer Dr. Mike McConnell, on a data analysis project evaluating clinical data from Spark's CHM trial in partnership with UVa Biomedical Engineering students.
• University of Pennsylvania / Children's Hospital of PhiladelphiaminorStrategic or Co-development PartnerCRF thanks collaborators at University of Pennsylvania and Children's Hospital of Philadelphia for their support in the SPK-7001 asset transfer and data review.
• RocheminorStrategic or Co-development PartnerCRF thanks collaborators at Roche for their support in the first-of-its-kind SPK-7001 asset transfer and data review. Roche acquired Spark Therapeutics.
• WiCellcoreOthersCRF's BioBank resources including CHM iPSC lines are housed at WiCell, making cellular resources accessible to scientists for CHM research.
• Coriell InstitutecoreOthersCRF's BioBank resources including CHM iPSC, fibroblasts, DNA, and LCL are housed at Coriell, providing key cellular resources for CHM research internationally.
• Kiora PharmaceuticalscoreStrategic or Co-development PartnerCRF partnered with Kiora Pharmaceuticals to advance novel CHM treatment. Kiora is developing KIO-301 in Phase 2 clinical trials. CRF provided a grant to fund novel clinical trial endpoints for inherited retinal diseases.

Scale indicators8 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Cystinosis Research Network: Ultra-rare disease patient advocacy nonprofit that funds research grants and connects an international patient/researcher community. Operates the same small-team, donation-funded, multi-modality R&D model.
• Foundation Fighting Blindness: The largest US nonprofit funding inherited retinal disease research, including choroideremia. Operates the same model of patient advocacy, research grants, and clinical trial enablement, making it the closest structural peer.
• Research to Prevent Blindness: US nonprofit funding vision research at academic institutions, including inherited retinal diseases. Comparable grantmaking and clinical-translation focus but at larger scale.
• American Macular Degeneration Foundation: Patient advocacy nonprofit funding research and supporting patients with another major retinal disease. Closely comparable funding/education/community model.

Broad incumbents

• Prevent Blindness: Established US eye-health nonprofit with a broader prevention/screening mandate. Adjacent in mission and advocacy scale but less focused on inherited retinal disease research funding.
• Retina International: Global umbrella for retinal disease patient organizations including CRF's own membership. Comparable in advocacy function but covers the full spectrum of inherited and age-related retinal diseases.

Others

• National Organization for Rare Disorders (NORD): Umbrella organization for rare disease patient advocacy groups of which CRF is a member. Comparable in advocacy/governance support role but covers all rare diseases rather than retinal disease specifically.
• Global Genes: Alliance of rare disease patient advocacy organizations of which CRF is a member. Comparable peer in advocacy tooling, education, and rare-disease community coalition building.
• Genetic Alliance: US nonprofit umbrella supporting rare and genetic disease advocacy organizations. Comparable in capacity-building and advocacy infrastructure for disease-specific foundations.

Regional players

• Fighting Blindness Canada: Canadian counterpart to Foundation Fighting Blindness, funding inherited retinal disease research and serving patients. Operates a near-identical nonprofit model in a separate geography.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat6 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xx.com/curechm
• YouTubeyoutube.com/@curechm
• Facebookfacebook.com/CureCHM
• LinkedInlinkedin.com/company/curechm
• Instagraminstagram.com/curechm

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles13 records

Funding overview

Funding rounds

Investors

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M&A1 record

Investments

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Frequently asked questions about The Choroideremia Research Foundation