Rare Daily Staff

The Chan Zuckerberg Initiative will award $13.5 million to 30 patient-led organizations that are working to find treatments and cures for rare diseases.

The grants are part of the Initiative’s Rare As One Project, which supports work that patient communities are doing to accelerate research and drive progress in the fight against rare diseases.

As a group, rare disease is anything but rare as more than 7,000 rare diseases affect 400 million people worldwide. The vast majority of these diseases are not well known or understood, and fewer than five percent have any FDA-approved therapy. The knowledge and experiences of patients suffering from these diseases are key to driving breakthroughs in research and treatment, but a lack of funding and infrastructure to support such patient-led research is holding back progress.

“No one is more committed to finding cures for rare diseases than the patients and families of those affected by these disorders,” said Priscilla Chan, co-founder and co-CEO of the Chan Zuckerberg Initiative (CZI).

CZI launched the Rare As One Network Request for Applications in June 2019 in the first phase of the Rare As One Project, and invited patient-led, 501(c)(3) organizations to apply, with the goal of funding 10 rare disease groups.

Because of the volume and demonstrated need of so many patient organizations, CZI expanded the funding to 30 different organizations, including groups dedicated to finding cures for a broad range of diseases such as rare cancers and neurodegenerative and autoimmune diseases. The patient advocates who lead these organizations have a variety of backgrounds—many hold dual roles, such as a stay-at-home mom, surgeon, community organizer, author, computer scientist, and more.

CZI is providing these patient organizations with funding, training, community mentorship, and capacity-building services to assist them in establishing a network of researchers and clinicians, convening their community, and developing a research agenda. The hope is that these organizations will share feedback with and learn from one another, and identify how to best address the most pressing needs across a spectrum of rare diseases.

“We’re excited to welcome this group of grantees—led by patients and caregivers with diverse backgrounds, perspectives, and expertise—to work together and learn together,” said Tania Simoncelli, CZI science policy director and Rare As One Project lead. “We hope the support, training, and mentorship provided through CZI’s program will be transformative for these organizations and serve as a model that can be catalytic for other groups battling rare diseases.”

Photo: Tania Simoncelli, CZI science policy director and Rare As One Project lead

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