A Ready Source of Funding to Advance Rare Disease Therapies

When California voters approved Proposition 14 in 2020, it created $5.5 billion in funding for the California Institute for Regenerative Medicine to fund research and development of advanced therapies.

The vote gave new life to CIRM, which California voters funded with $3 billion to advance stem cell research in 2004 in the face of Bush Administration restrictions on stem cell research. The new funding expands the types of research the agency will fund to include a range of genetic medicines.

During the RARE-X Exchange Forum in San Diego on September 14 and 15, representatives of CIRM participated in roundtable discussions to raise awareness about funding available to support the discovery, translational research, and clinical development of therapies to treat rare diseases.

“We always funded stem cell research, including research targeting cancer stem cells. That’s always been our mandate,” said Lila Collins, senior science officer of therapeutics for CIRM, who noted more than half of the institute’s funding has gone to rare disease therapies. “With the passage of Prop 14 in 2020, genetic therapies directly came under our umbrella of fundable technologies. That includes ASOs, siRNAs, gene editing, and all of that. We want to get the word out.”

CIRM’s funding opportunities extend across the movement from basic science to clinical research, with the amounts growing larger as work moves deeper into human clinical testing. Discovery grants range from $500,000 to $1.5 million. Translational research grants, which include pre-IND grants, can run up to $4 million. Clinical grants extend from $4 million to $15 million. Translational and clinical grants require co-funding for for-profit grantees.

To be eligible for a Discovery or Translational grant, the applicant must be considered a California organization (more than half of its employees are employed and paid in the state). While organizations based outside of California are not eligible for CRIM’s Discovery and Translational grants, they can apply for clinical awards to support done within the state of California.

CIRM’s program announcements spell out in detail what the institution will look for in an application. CIRM scientists like Collins are ready to guide investigators through the application process and help them understand what CIRM is looking to fund, including data-sharing, DEI requirements, and help connecting them to resources.

“We fund based on the scientific merits, the unmet medical needs, and the probability of technical success as seen by the reviewers,” said Collins. “It’s about the quality of the applications itself. You can have a huge impact on a small patient population. We can do that as a state funding agency. We don’t have to make those decisions like a Big Pharma.”

Getting an earlier-stage grant doesn’t prevent an applicant from seeking additional funding as a potential therapy advances toward the market. In fact, CIRM likes when that happens, and there are many instances where the institute has provided multiple grants to support the development of a therapy at different stages.

The discovery and translation grants are made twice a year. The clinical grants are made monthly. It takes CIRM about 90 days to act on an application.

“That’s one of the things that we measure our success on. We call it progression events,” said Collins. “We love that. That’s our goal to help progress good science through the pipeline.”