Chan Zuckerberg Biohub to open new rare disease funding round

Biohub, a Chan Zuckerberg Initiative-funded non-profit research organization, announced Thursday that it will open another round of funding through its Rare As One Network this fall. It marks the fourth round of grants for the rare disease community from Rare As One, which has committed more than $150 million to rare disease initiatives through the four funding cycles.

“When I was a pediatric resident, I would sit across from families who had done everything right — researched, advocated, fought for answers — and I had to tell them that medicine just didn’t know yet. Those families never left me. We built Rare As One because we believe patients aren’t bystanders to scientific progress — they are among its most powerful drivers,” Priscilla Chan said in a statement to CNBC.

The application window for the fourth cycle will likely open in October, though an official date has not yet been announced. Awards will be targeted towards groups focused primarily on rare pulmonary and immune diseases, as well as rare cancers.

The announcement also expands Biohub’s partnership with Every Cure, the nonprofit founded by Dr. David Fajgenbaum that uses artificial intelligence to identify opportunities to repurpose existing medicines for diseases with few or no treatment options. Biohub already serves as a foundational philanthropic backer of Every Cure. Through the new collaboration, select patient organizations will partner with Every Cure to advance promising drug repurposing opportunities identified from its AI-driven platform, with the goal of taking those findings and turning them into patient-centered research programs.

“I’m thrilled about our expanded partnership — from an initial grant to get started with building our AI platform in 2023, to where we are now,” Fajgenbaum said. “There’s no better organization or team to work with on this shared mission.”

It’s all part of a broader push from Chan and Mark Zuckerberg to further incorporate AI in their ambitious quest to eliminate disease. Biohub recently launched its Virtual Biology Initiative — a $500 million effort to build a predictive model of the cell. As part of that announcement, Biohub also introduced its own AI model designed to accelerate drug discovery.

“In the seven years since we launched, 94 patient-led organizations have built research networks, launched clinical trials, and in some cases are now sponsoring those trials themselves,” Chan said. “What once felt impossible is happening. And now, with AI accelerating what’s scientifically possible, every day we are lifting the ceiling of what’s possible for families,” she added.

For Biohub’s Rare As One program, the announcement marks another milestone in what has been a transformational relationship with the rare disease advocacy community. To date, Biohub says the organizations it supports have engaged more than 320,000 patients and community members, along with 26,000 researchers. Those organizations have shown how advanced and sophisticated patient-led advocacy groups have become over the past decade, with two-thirds of the groups that received funding from Rare As One building research assets and tools that will help accelerate understanding of rare diseases, and more than half contributing to the development and launch of clinical trials.

“We founded the Rare As One Project because we saw that patients were playing a critical role in scientific discovery,” Tania Simoncelli, Biohub vice president of translational science, said in a blog post. “What we have witnessed across three cycles is something even more powerful than we imagined. Patients, researchers, and clinicians working together aren’t just accelerating timelines: they are reshaping the biomedical research paradigm.”

Sunitha Malepati, a CNBC Cures Advisory Board member and the vice president of the CACNA1A Foundation — a group that was awarded a grant from Biohub — said being selected to the program completely transformed what her group was able to accomplish. “When we joined the network, we were a young organization with a bold vision but limited infrastructure. Through the program’s funding, training, and peer community, we were able to build the organizational capacity needed to actively driving research,” she said.

Malepati said the money helped establish research partnerships and organize the patient and scientific communities around CACANA1A-related disorders, a group of rare, lifelong neurological and genetic conditions. “Rare As One recognized early on that patient-led organizations can be powerful catalysts for scientific advancement, and their belief in our community has helped accelerate hope toward tangible treatments,” she added.

By combining its growing presence in the rare disease community along with its growing investment in AI-based medical technologies, Biohub hopes that those tangible treatments start to come faster than ever before — and that the patients desperate for answers can start getting the help that they need.

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Correction: This story has been corrected to show that Rare As One Network has committed more than $150 million to rare disease initiatives through a total of four funding cycles.