CZI Awards $13 Million to Patient-Led Organizations Advancing Rare Disease Research

Today, the Chan Zuckerberg Initiative (CZI) announced $13 million in funding for 40 patient-led, rare disease advocacy organizations that are working alongside researchers and clinicians to accelerate research in their disease areas. These grants are part of CZI’s Rare As One (RAO) Project, aimed at supporting and lifting up the work that patient communities are doing to drive progress in the fight against rare diseases.

Rare disease is not rare. As many as 7,000 rare diseases affect 400 million people globally. The Rare As One Project is committed to uniting rare disease patient advocates in their quest for cures.

With today’s announcement, 20 patient-driven rare disease organizations will join the initial 30 grantee organizations awarded in February 2020 as part of the Rare As One Network. Funding will support these organizations to advance the development of an international, patient-led collaborative research network in the organizations’ individual disease area of focus, strengthen their organizational capacity, convene their communities, and align patients and researchers towards shared priorities. Learn more about these organizations. Additionally, 20 separate rare disease groups will receive general operating support or core program support.

“In order for biomedicine and research to advance quickly and efficiently, especially in rare disease, patients must be full partners with researchers and clinicians in research,” said CZI Head of Science Cori Bargmann. “We’re proud to expand our cohort of Rare As One grantees and further support the rare disease ecosystem as we work towards treatments and cures together.”

CZI recognizes that the diagnostic odyssey is a pressing challenge faced by rare disease communities as they seek to build strong, diverse, and inclusive communities, as well as move science forward towards the development of treatments and cures for all those impacted by the disease. As a result, this second cycle placed additional emphasis on the potential to address diagnosis-related research challenges and the importance of addressing diversity and inclusion in those efforts.

“Patients are experts in their own diseases, and their knowledge and commitment to advancing progress in their disease areas has the power to center patient priorities and dramatically accelerate the pace of research,” said Heidi Bjornson-Pennell, CZI Rare As One Program Manager. “The RAO Network is proud to lift up these efforts by offering new tools, funding, and capacity-building support and training to help these organizations grow and scale.”

For this second cycle of funding, in March 2021 CZI launched the Rare As One Network Request for Applications, inviting patient-led, U.S.-based nonprofit 501(c)(3), rare disease advocacy organizations to apply.

The following 20 organizations selected to join the Rare As One Network will each receive $600,000 over the three-year grant period, for a total of $12 million:

• A Cure for Ellie
• A Foundation Building Strength
• Angioma Alliance
• CACNA1A Foundation
• Chelsea’s Hope Lafora Children Research Fund
• Cure VCP Disease
• FamilieSCN2A Foundation
• Foundation for Sarcoidosis Research
• Mission: Cure
• PACS1 Syndrome Research Foundation
• PCD Foundation
• Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network
• Raymond A. Wood Foundation
• Recurrent Respiratory Papillomatosis Foundation
• Shwachman-Diamond Syndrome Alliance
• Smith-Kingsmore Syndrome Foundation
• Tatton Brown Rahman Syndrome Community
• Team Telomere
• The Stiff Person Syndrome Research Foundation
• The TBCK Foundation

The following additional 20 organizations will each receive $50,000 for general operating support or core program support in support of their quest to accelerate research and find treatments and cures for rare disease:

• 17q12 Foundation
• ASXL Rare Research Endowment Foundation
• Autoimmune Hepatitis Association
• CSNK2A1 Foundation
• CureGPX4
• Global DARE Foundation
• Hope for Hypothalamic Hamartomas
• International SCN8A Alliance
• IPPF
• National Foundation for Ectodermal Dysplasias
• Osteogenesis Imperfecta Foundation
• Sickle Cell Transplant Advocacy and Research Alliance
• SLC6A1 Connect
• Soft Bones, Inc., The US Hypophosphatasia Foundation
• Spinal CSF Leak Foundation
• The APS Type 1 Foundation
• The Global Foundation for Peroxisomal Disorders
• The Naa10 Foundation D/B/A Ogden Syndrome Foundation US
• United MSD Foundation
• Vasculitis Foundation

The Rare As One Project is part of CZI’s Science in Society program.

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About the Chan Zuckerberg Initiative

The Chan Zuckerberg Initiative was founded in 2015 to help solve some of society’s toughest challenges — from eradicating disease and improving education, to addressing the needs of our local communities. Our mission is to build a more inclusive, just, and healthy future for everyone. For more information, please visit chanzuckerberg.com.