The Chan Zuckerberg Initiative has announced grants totaling $13.5 million in support of patient-led organizations working to find effective treatments and cures for rare diseases.
Awarded through CZI's Rare As One Project, the grants will provide funding, training, mentorship, and capacity-building assistance to organizations working to establish networks of researchers and clinicians, convene community members, and develop a research agenda. The project also provides space for organizations to share feedback and best practices with respect to addressing urgent needs across a spectrum of rare diseases.
Launched in June 2019, the project originally sought to fund ten rare disease groups, but in response to the volume and demonstrated need of applicants CZI expanded the funding to thirty organizations, including groups dedicated to finding cures for rare cancers and neurodegenerative and autoimmune diseases. According to CZI, as many as seven thousand rare diseases affect four hundred million people globally. The vast majority of the diseases are not well known or understood, and fewer than 5 percent have any FDA-approved therapy.
Grant recipients include the Association for Creatine Deficiencies, Congenital Hyperinsulinism International, Cure CMD, the DADA2 Foundation, the Glut1 Deficiency Foundation, the Lennox-Gastaut Syndrome Foundation, Project 8p, and the Snyder-Robinson Foundation.
"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."
For a complete list of Rare As One Project recipients, see the CZI website.
(Photo credit: INADcure Foundation)