09/25/2025 | Press release | Distributed by Public on 09/25/2025 17:39
FOR IMMEDIATE RELEASE
Contact: Amy Adams Senior Director of Communications [email protected]
South San Francisco, CA, September 25, 2025 - The California Institute for Regenerative Medicine (CIRM) has approved awarding more than $73 million across 23 grants to support discovery research projects in regenerative medicine at institutions throughout California.
These awards-approved at the September meeting of the Independent Citizen's Oversight Committee ("the Board')-will fund early-stage research intended to spark the new ideas and tools that are the first step in developing novel disease therapies. CIRM increased funding allocated to this round of grants from $37 million to $74 million in June in response to research funding cuts affecting California institutions.
CIRM was created by voters in 2004 through California's Proposition 71, and extended in 2020 through Proposition 14, to accelerate therapies for unmet medical needs. The agency funds stem cell and gene therapy research (collectively called regenerative medicine) from the earliest stages through clinical trials with the goal of advancing therapies for people in California and the world.
The recently approved awards, known as DISC0 Foundation Awards, are part of CIRM's Discovery Stage Research programs. Within three years, funded projects are expected to advance the field's understanding of stem cells and genetics in human biology and disease, develop stem cell-based tools for biomedical innovation, or unlock new applications in regenerative medicine.
"Discovery research is critical for uncovering novel disease targets and biomarkers that we can translate into therapies for clinical use," said Kelly Shepard, PhD, Director of Discovery & Education programs at CIRM. "The DISC0 projects have the potential to address gaps in our current knowledge, advance our understanding of the origins and mechanisms of disease, and expand existing applications of stem cell and gene therapy-based treatments."
The DISC0 awards include two funding tracks: one for individual principal investigators, and a second that supports collaborative teams led by a principal investigator with one or more co-investigators.
Groundbreaking treatments for disease do not arise out of thin air. Each of the 116 clinical trials CIRM has funded to date began with an idea or discovery that came out of research much like those proposed in the DISC0 projects, including trials for epilepsy and blindness that originated from CIRM-funded discovery research. The DISC0 projects funded today lay the groundwork for therapies tomorrow.
"Discovery stage research creates the foundation for future preclinical research, clinical trials and ultimately cures and treatments for people with unmet medical needs," said Rosa Canet-Avilés, PhD, Chief Science Officer at CIRM. "Through our Strategic Allocation Framework, CIRM's funding pipeline from discovery research through preclinical development to clinical trials is coordinated to ensure our research portfolio advances toward patients as quickly as possible."
The research funded in this round of DISC0 grants includes projects that seek to identify new ways to modify the human genome for treating disease, novel stem cell transplant techniques for neurodegenerative disorders in infants and young children, and investigation into the genetic basis for the higher risk of leukemia in people with Down syndrome, among many others.
The 23 approved grants are:
Application # | Program Title | Principal Investigator/Institution | Amount |
DISC0-17363 | Allele Prospector: Leveraging human genetic variation to enable therapeutic genome editing in hundreds of disease genes | Bruce R. Conklin - The J. David Gladstone Institutes | $5,112,209 |
DISC0-17394 | Harnessing developmental biology to achieve safe and efficient in vivo genome editing of HSCs | Tippi C. MacKenzie - University of California, San Francisco | $2,316,683 |
DISC0-17652 | Next generation stem cell transplantation approaches for pediatric neurodegenerative disorders | Nicole G Coufal - University of California, San Diego | $4,628,762 |
DISC0-17515 | Hearing the Silence: Genome-wide Mapping of Cell-Type-Specific Silencers in the Developing Human Brain | Jingjing Li - University of California, San Francisco | $4,244,432 |
DISC0-17608 | Interrogation of tandem repeat variants contributing to neurodevelopmental and psychiatric traits using stem cell models | Melissa Gymrek - University of California, San Diego | $2,405,997 |
DISC0-17610 | Unraveling the developmental path from altered hematopoietic stem cells to leukemia in Down syndrome | Hanna Mikkola - University of California, Los Angeles | $2,337,847 |
DISC0-17674 | Identifying and Overcoming Roadblocks to Hearing Restoration Using Human Induced Pluripotent Stem Cells | Konstantina Stankovic - Stanford University | $4,608,000 |
DISC0-17626 | Development of in vitro and in vivo functional human synthetic kidney organoid (hSKO) model as a platform technology for kidney research | Zhongwei Li - University of Southern California | $2,287,926 |
DISC0-17364 | Mechanisms of Transcription Factor Haploinsufficiency in Human Congenital Heart Disease | Benoit Bruneau - The J. David Gladstone Institutes | $2,444,376 |
DISC0-17635 | Genetic and Epigenetic Regulation of XIST and X-chromosome silencing in hiPSCs: Overcoming Barriers in Stem Cell-Based Therapies for Women's Health | Kathrin Plath - University of California, Los Angeles | $2,358,742 |
DISC0-17421 | Developing replacement islet cells for diabetes using human stem cells | Seung K. Kim - Stanford University | $3,943,364 |
DISC0-17487 | Mechanisms underlying dosage sensitivity in developmental disorders | Joanna Wysocka - Stanford University | $2,304,000 |
DISC0-17685 | Dissecting the cellular and molecular interactions between embryo and endometrium during human implantation | Matteo Amitaba Mole' - Stanford University | $2,290,157 |
DISC0-17946 | High-Throughput Discovery of Embryo Formation Factors Using Stem Cell-Based Human Embryo Models | Magdalena Zernicka-Goetz - California Institute of Technology | $2,872,697 |
DISC0-17976 | Enhancing clinical predictability with novel models of iPSC-derived nociceptor for chronic pain | Fernando Aleman - Navega Therapeutics | $1,498,623 |
DISC0-18130 | Unlocking the regenerative potential of hepatocyte plasticity for diseases of the biliary system | Holger F Willenbring - University of California, San Francisco | $4,871,000 |
DISC0-17315 | IFN-γ suppresses AT2 cell regeneration to promote lung fibrosis | Peter Chen - Cedars-Sinai Medical Center | $4,525,190 |
DISC0-17998 | Global profiling of miRNA-based gene activation to enable a new category of genetic medicine | Chi Zhang - Iris Medicine, Inc. | $2,997,574 |
DISC0-17566 | In neurons and beyond: how protein interactions shape the cellular response to Huntington's Disease | Leslie M Thompson - University of California, Irvine | $2,056,195 |
DISC0-17276 | Modeling Rett syndrome neurological disorder with human pluripotent stem cells to develop in cellulo screening platforms | Robert Tjian - University of California, Berkeley | $2,393,281 |
DISC0-17488 | A novel platform to rescue neurodevelopmental disorders caused by haploinsufficiency | Giordano Lippi - Calibr | $4,086,486 |
DISC0-18038 | Base Editing, Single-Cell Multiomics, and Cardiac Organoids to Decode Genetic Variants | Joseph C. Wu - Stanford University | $4,606,248 |
DISC0-17507 | Unraveling nuclear Tau functions using age-equivalent human induced neurons from healthy aging donors and tauopathy patients | Jerome Mertens - University of California, San Diego | $2,137,778 |
Also at the September meeting, CIRM President and CEO Jonathan Thomas, PhD, JD, gave a presentation updating the Board on the progress of implementing the Strategic Allocation Framework (SAF). CIRM developed the SAF to ensure that the agency's remaining funds are translated into therapies, and to that end the SAF includes measurable goals that act as milestones for driving new disease cures and treatments. Funding preferences within new program announcements are intended to select for the applications most likely to enable CIRM to reach those goals.
Thomas showed a budget forecast for how CIRM will spend roughly $3.5 million over the next six years across the agency's five funding pillars: discovery, preclinical, clinical, education, and infrastructure. He also updated the Board on the progress of developing and implementing new funding programs.
"I want to remind you that the SAF and the preferences that will help us achieve the impact goals are not intended to be static," Thomas told the Board. "They are designed to adapt based on CIRM's internal portfolio and developments in the field."
At the same meeting, two new board members were sworn in. They are Marguerite Casillas, a patient advocate for multiple sclerosis, and Shannon Dahl, PhD, Founder and CEO, Carve Bio. Both were appointed by the California Lieutenant Governor. Read more about the CIRM Independent Citizen's Oversight Committee.
"I am so proud to be part of an organization that supports cutting-edge science to help improve the lives of Californians living with serious diseases. I've been impressed by the talented and passionate staff members of CIRM, and I look forward to meeting my fellow board members and contributing my voice and lived experiences to our discussions," Casillas said.
"I look forward bringing my experience in advancing therapeutics from research and development to reimbursement, while partnering with fellow board members who bring rich perspectives from biotech, patient advocacy, and university leadership," Dahl said. "Together with the CIRM team, we will support CIRM's mission of bringing meaningful treatments to patients in California and beyond, and shaping best practices for access in healthcare."
CIRM was created by the people of California to fund stem cell and gene therapy research with the goal of accelerating treatments for patients with unmet medical needs. With $5.5 billion in funding allocated through both Proposition 71 in 2004 and Proposition 14 in 2020, CIRM supports stem cell and gene therapy discoveries from inception through clinical trials, trains a workforce in California to fill jobs in the state's thriving biotech and biomedical research industry, and creates infrastructure to make clinical trials accessible for people throughout California. All of CIRM's research, workforce development, and infrastructure programs are designed to benefit the people of California, whose vision created the agency.