California Institute for Regenerative Medicine

The committee discussed the proposed concept plan for data science and software engineering awards, specifically focusing on the infrastructure needed for data integration in regenerative medicine. Key topics included the importance of open-source software, the challenges of managing diverse and siloed biomedical datasets, and the need for scalable software tools. The subcommittee reviewed the eligibility, award structure, and outreach plans for this potential funding program, and held a vote to endorse moving the concept plan forward to the full board for approval.

The Data Science and Software Engineering (INFR9) Concept Plan outlines a strategy to accelerate regenerative medicine research through the development of innovative, open source software for data integration. Key priorities include supporting the creation of broadly applicable, validated tools to solve multimodal data integration bottlenecks in stem cell-based and genetic therapy research. The plan emphasizes robust deployment, adoption, and sustainability, adherence to open source best practices and transparent development, fostering community and contributor engagement, and utilizing pre-existing public datasets. This initiative is designed to catalyze the identification and validation of novel targets and biomarkers and reduce time to therapeutic development.

The meeting focused on informational updates regarding access and affordability for cell and gene therapies. Key topics included a recap of an Access and Affordability Summit, an FDA workshop on pediatric clinical trials, an update on the first CIRM-funded gene therapy approval for Leuko-cyte Adhesion Deficiency 1, and revisions to access plan requirements for preclinical and clinical programs.

The meeting focused on informational updates regarding the broader engagement of the patient access team and the organization of the AWG's strategy for cell and gene therapy access and affordability. Key topics included a recap of a collaborative summit with ISSCR and UCLA on therapy accessibility and payment models, as well as an FDA workshop discussing the design of pediatric rare disease clinical trials and the importance of including parent and patient advocate input in trial design and execution.

The committee meeting focused on several key items, including the appointment and reappointment of scientific members to the Grants Working Group, as well as the evaluation procedures for committee leadership and the CIRM President/CEO. Discussions also covered amendments to funding mechanisms for CIRM conferences and meetings. A major portion of the session was dedicated to the consideration of applications for discovery program announcements and clinical program announcements, along with amendments to the Community Cares Centers of Excellence concept plan. The committee reviewed program preferences, guiding principles, and a co-funding program amendment. Additionally, the committee received updates from the communications team and reports from the Chair and President.