CIRM Announces Recommendations from Strategic Allocation Framework

CIRM Announces Recommendations from Strategic Allocation Framework

FOR IMMEDIATE RELEASE

Contact:
Koren Temple-Perry
Sr. Director, Marketing & Communications
[email protected]

South San Francisco, CA, September 27, 2024 - The California Institute for Regenerative Medicine (CIRM), one of the world's largest institutes dedicated to regenerative medicine, announced key recommendations from its Strategic Allocation Framework (SAF), a structured and data-driven effort designed to prioritize resources and maximize the impact of CIRM's funding.

These recommendations mark a significant step forward in aligning CIRM's initiatives with the Agency's overall mission to accelerate the development and delivery of innovative regenerative medicine treatments.

The SAF will be instrumental in guiding CIRM's remaining $3.86 billion in funding-$1.14 billion of which must go to neurological research as mandated by Prop 14 (2020). The effort kicked off in September 2023 following a prioritization discussion that identified a need for a more structured approach to resource allocation. Over the past six months, CIRM has presented recommendations at joint Science Subcommittee and Neuro Task Force meetings of areas where CIRM's funding can have the greatest impact.

"The regenerative medicine landscape has evolved dramatically since CIRM's inception. With advances in cell and gene therapies, including promising breakthroughs for both rare and prevalent diseases, we must ensure our focus is meeting the demands of this rapidly expanding field," said Jonathan Thomas, PhD, JD, President and CEO of CIRM. "The Strategic Allocation Framework (SAF) is crucial for CIRM to sustain our role as a global leader in regenerative medicine and stem cell research. As such, we must ensure that these remaining resources are distributed wisely to drive the most significant impact possible."

Design questions served as the foundation for guiding the SAF in addition to extensive internal and external key datasets. Data were compiled and analyzed, playing a key role in informing recommendations.

Strategic Recommendations from the SAF Process

The SAF was designed to focus CIRM's resources on measurable impact goals framed within four key categories: Accelerating Discovery & Translation, Cell & Gene Therapy Approvals, Accessibility & Affordability, and Diverse Workforce Development.

The result was a set of six recommendations designed to accelerate the discovery and translation of therapies, advance critical approvals for cell and gene therapies, improve accessibility and affordability, and ensure a diverse and skilled workforce capable of sustaining advancements in regenerative medicine.

The following are goals and recommendations from the SAF process:

Accelerating Discovery & Translation
Goals	Recommendations
1. Catalyze the identification and validation of at least 4 novel targets and biomarkers, ensuring integration into preclinical or clinical research for diseases in California.	Support comprehensive discovery research through DISC4 & DISC5 funding structures Encourage collaborative, multidisciplinary innovation in stem cell and genetic research across diverse disciplines & disease indications with early engagement of industry to address reproducibility & scalability issues
Establish a Data Coordinating and Management Center (DCMC) to streamline data management and enhance the utility of cross-disease data Fund and develop a central hub for data coordination, facilitating better integration with consortia & research initiatives and enabling data science collaborative efforts via dedicated grant
2. Accelerate development and utilization of 5-8 technologies that have the potential to improve safety, efficacy, and/or quality of cell and gene therapies.	Pilot INFR Technology Platform Program to bridge the gap between research and commercialization Foster partnerships between academic researchers & industry professionals to support multi-stakeholder technology incubation programs that achieve defined technology readiness levels thereby facilitating rapid application in cell & gene therapy development
Cell & Gene Therapy Approvals
3. Advance 4-7 rare disease projects to Biologics License Application (BLA)	Accelerate Current rare disease therapy pipeline Increase and scale CLIN4 funding to comprehensively address BLA readiness gaps in manufacturing clinical/non-clinical research and pre-commercialization
Pilot Platform-Based Therapy Development Implement pilot platform-based approach for gene therapy development using life-threatening monogenic neurological disorders as a test case
4. Propel 15-20 therapies targeting diseases affecting Californians to late-stage trials.	Streamline Preclinical Development Programs Consolidate DISC2, TRAN 1-4, and CLIN1 to accelerate the preclinical development incentivizing multidisciplinary collaborations and rapid progression to IND. Incorporate prioritization of innovative therapies for diseases that affect Californians
Update CLIN2 Allow for support of emerging novel clinical trial designs in CLIN2 program Incentivize stage-appropriate market access strategy development and pre-commercialization activities in the CLIN2 program Incorporate prioritization of innovative therapies for diseases that affect Californians
Accessibility & Affordability of CIRM-Funded Cell & Gene Therapies
5. Ensure that every BLA-ready program has a strategy for access and affordability.	Strengthen Clinical Infrastructure Connectivity Build interconnectivity & performance metrics between CIRM Clinical Infrastructure (Alpha Clinics, CCCEs, PSP) to ensure enhanced referral enrollment & retention of California patients in clinical trials
Support Development of Market Access and Reimbursement Strategies Resource clinical programs to support stage appropriate planning & evidence generation to inform robust market access & reimbursement strategies
Influence Policy Deploy AAWG resources to advocate for policies that advance access & reimbursement for regenerative medicines
Enhance Partnerships Engage state & national partners to align initiatives that expand sustainable access to regenerative medicines
Diverse Workforce Development
6. Bolster CIRM's workforce development programs to address gaps and meet evolving demands in regenerative medicine.	Provide high-demand technical training via Bridges & COMPASS program updates Increase training offerings diversify internship types & increase integration with CIRM R&D grants
Create new EDUC program to develop hybrid skillsets Implement new program structure to focus on cross-disciplinary internships
Launch outreach campaigns to educate the public & increase diversity of California's regenerative medicine workforce Develop programming to support outreach education efforts for K-12, teachers, & community members via collaboration with key stakeholders
Additional Recommendations	Restart Grantee Conference to Report SAF Goal Progress Restart recurring grantee conference (timing TBD) with main objective of reporting progress on SAF goals
Keep Conference Grants for Specific CIRM Needs (EDUC1 Mechanism 2) Grantee retains the primary responsibility for planning directing and executing the proposed event. CIRM team will work closely with the grantee to design and implement an event responsive to a specific CIRM needs

"CIRM has made a significant impact on the research ecosystem in California. Our new priorities will refine that process, making sure that every dollar is strategically allocated through impact goals and data-driven analysis. This ensures resources are spent on projects that have the highest potential to make a significant difference. Additionally, our new priorities are shifting toward overcoming translational bottlenecks and clearly defined success metrics. This ensures that the work being funded is moving closer to commercialization and benefiting patients sooner," said Rosa Canet-Avilés, PhD, who spearheaded the SAF effort.

Internal Transitions to Support Prioritization Efforts

To successfully implement the SAF, CIRM has undergone a major restructuring to better align its operations with its strategic priorities:

Dr. Canet-Avilés has transitioned into the role of Chief Science Officer (CSO), overseeing all scientific programs and strategies, ensuring they are closely aligned with CIRM's SAF objectives. In addition to the introduction of the CSO role, several internal transitions have been announced.

Abla Creasey, PhD, has transitioned from her position as Vice President of Therapeutics Development to Executive Strategy Officer - Rare Diseases, where she will oversee the newly established rare diseases platform, a key recommendation outlined in the SAF strategy.

Shyam Patel, PhD, has transitioned from Senior Director of Business Development to the role of Associate Vice President of Preclinical Development and Infrastructure, focusing on advancing preclinical programs.

Janie Byrum, PhD, has become the Senior Science Officer of the CIRM Data Infrastructure for Research and Development (R&D) Programs, transitioning from her current role as Science Officer within the Scientific Programs and Education division.

Additionally, Sara Taylor, PhD, has been promoted to Program Manager in Strategy and Programs, supporting the CSO in implementing CIRM's strategic priorities.

Advancing Promising New Research

Along with approving key recommendations from the SAF, CIRM awarded $6 million to Mark Humayun, MD, PhD, from the USC Keck School of Medicine, to support a pre-clinical project designed to slow the progression of age-related macular degeneration.

CIRM also granted over $22.5 million to nine projects through the Quest Awards Program, which fosters the discovery of innovative stem cell-based and gene therapy technologies.

These awards include the following:

Application #	Program Title	Principal Investigator/Institution	Amount
DISC2-16738	Developing a universal CRISPR gene therapy approach to treat C9orf72 ALS	Clelland, Claire - UCSF	$2,740,529
DISC2-16715	Drug discovery for Charcot Marie Tooth Disease using hPSC-derived Schwann cells	Fattahi, Faranak - UCSF	$2,471,664
DISC2-16589	Genome editing of human Tregs to enable combinational tolerogenic therapy with T cell targeted biologics for T1D	Tang, Qizhi - UCSF	$2,485,549
DISC2-16638	Development of a Trifunctional Self-Renewing Memory CAR T Cell Therapy to Overcome the Heterogeneity and Suppressive Microenvironment of Glioblastoma	Brown, Christine E. - City of Hope	$2,700,810
DISC2-16774	Therapeutic targeting of Glioblastoma Stem Cell survival and self-renewal signaling	Anderson, Aileen J - UC Irvine	$2,567,379
DISC2-16725	B cell receptor-mediated lentiviral expression of anti-HIV antibody	Morizono, Koki - UCLA	$2,751,504
DISC2-16562	Human induced pluripotent stem cells-derived glial enriched progenitors for the treatment of mild traumatic brain injury	Luo, Jian - PAVIR	$2,255,189
DISC2-16590	Modulating cardiac myosin heavy chain isoform expression for treating cardiomyopathies	Chi, Neil Carson - UCSD	$2,772,000
DISC2-16772	Chemically engineered photoreceptors for vision restoration in retinal degeneration associated blindness	Mahato, Biraj - Children's Hospital Los Angeles	$1,711,000

About the California Institute for Regenerative Medicine (CIRM)

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast-track the development of today's most promising stem cell technologies.

CIRM is one of the world's largest institutions dedicated to helping people by bringing the future of regenerative medicine closer to reality.

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