CIRM leadership briefs CFAOC on strategic reallocation, DEI work and launch of patient-assistance pilot

The California Institute for Regenerative Medicine (CIRM) briefed the Citizens Financial Accountability Oversight Committee on Dec. 18, 2024 on progress implementing its strategic plan and program updates, including a newly adopted Strategic Allocation Framework designed to guide deployment of remaining Prop 14 funds.

Jonathan Thomas, CIRM president and CEO, gave the committee an overview of the agency's mission and structure, noting that CIRM's work began under Proposition 71 and expanded under Proposition 14. He described CIRM's program pillars (discovery/basic research, translational, clinical, infrastructure and education) and said the agency has funded basic research, clinical trials, manufacturing capacity and education programs that aim to build a California workforce in stem cell and gene therapy.

Thomas told the committee CIRM has spent heavily on discovery research (he cited roughly $1.3 billion since inception) and about $650 million on education programs. The Alpha Clinics network supports clinical trials across nine major institutions and, historically, CIRM has funded more than 1,400 grants. Thomas highlighted an updated, data-driven Strategic Allocation Framework that reprioritizes funding across six goals: advance basic research; invest in tools and technologies; move 4—7 rare-disease projects through biologics license application (BLA) readiness; move 15'0 prevalent-condition programs to later-stage trials; pursue accessibility and affordability; and grow workforce development. He said the board adopted the framework in September and staff will translate it into concept plans and program announcements over the coming year.

On equity and access, Thomas and CIRM Vice Chair Maria Monville described multiple efforts to embed diversity, equity and inclusion (DEI) into program design. Thomas said patient outreach and enrollment representation are reviewed and scored during peer review for clinical awards; Monville and other CIRM leaders said a separate Accessibility and Affordability Working Group established by Proposition 14 has its own budget and staff to address cost and access issues for therapies.

The committee heard that the licensing and royalties fund had little or no activity in 2023; CIRM staff explained that a licensing-derived patient assistance program and a separate grant to operate that program were finalized after board approval in fiscal year 2023/24 and the pilot will begin in the coming fiscal year. CIRM said the patient-assistance fund initially received a lump-sum distribution of approximately $15.6 million from a realized transaction and that the organization has contracted Eversana to administer the program.

CIRM also described plans for ‘‘community care centers of excellence'''satellite clinical sites intended to increase access to trials in underrepresented regions. The first awards under that program were expected to be announced in January following community outreach and program design work.

Rafael Aguirre Sacasa, CIRM general counsel, reported on performance-audit follow-ups and internal reforms. He told the committee the organization has tightened IP and revenue-reporting follow-up (noting a 22% non-response rate for certain disclosures that CIRM is actively addressing), migrated document management to Microsoft 365/SharePoint, selected Salesforce for customer relationship management, and is building a research-data dashboard and clinical-trials information dashboard for improved transparency. He said human-resources changes are underway to speed hiring and clarify compensation; the board approved an updated compensation structure in June. Aguirre Sacasa described the agency's efforts to strengthen cybersecurity policy and other risk-management systems.

Committee members asked questions about measurement of DEI outcomes, whether Prop 14's inclusion of gene-therapy projects had raised legislative objections (it had not), the pace at which patient-assistance funds would flow to participants, and the role of AI and data tools in accelerating research. CIRM leaders described ongoing monitoring and said pilot programs will produce further implementation detail for the committee next year.

The presentation was informational; no committee action was required.