The California Institute for Regenerative Medicine (CIRM) has approved over $73 million in funding for 23 discovery research grants to support early-stage regenerative medicine projects at institutions across California.
In June, CIRM increased the funding for this round from $37 million to $74 million in response to recent research funding cuts in the state.
CIRM was established by California voters in 2004 through Proposition 71 and extended in 2020 via Proposition 14. The agency funds stem cell and gene therapy research (collectively referred to as regenerative medicine) at all stages, from early discovery through clinical trials, to advance treatments for unmet medical needs.
The newly approved awards, called DISC0 Foundation Awards, are part of CIRM’s Discovery Stage Research programs. Over the next three years, these projects are expected to:
- Advance the understanding of stem cells and genetics in human biology and disease
- Develop stem cell-based tools for biomedical innovation
- 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 another for collaborative teams led by a principal investigator with one or more co-investigators.
Projects funded in this round cover a range of topics, including genome modification for disease treatment, novel stem cell transplant techniques for neurodegenerative disorders in infants and children, and research into the genetic basis for the higher risk of leukemia in people with Down syndrome.
The 23 approved grants include:
- 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)
- 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)
- Next generation stem cell transplantation approaches for pediatric neurodegenerative disorders — Nicole G Coufal, University of California, San Diego ($4,628,762)
- 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)
- 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)
- Unraveling the developmental path from altered hematopoietic stem cells to leukemia in Down syndrome — Hanna Mikkola, University of California, Los Angeles ($2,337,847)
- Identifying and Overcoming Roadblocks to Hearing Restoration Using Human Induced Pluripotent Stem Cells — Konstantina Stankovic, Stanford University ($4,608,000)
- 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)
- Mechanisms of Transcription Factor Haploinsufficiency in Human Congenital Heart Disease — Benoit Bruneau, The J. David Gladstone Institutes ($2,444,376)
- 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)
- Developing replacement islet cells for diabetes using human stem cells — Seung K. Kim, Stanford University ($3,943,364)
- Mechanisms underlying dosage sensitivity in developmental disorders — Joanna Wysocka, Stanford University ($2,304,000)
- Dissecting the cellular and molecular interactions between embryo and endometrium during human implantation — Matteo Amitaba Mole’, Stanford University ($2,290,157)
- High-Throughput Discovery of Embryo Formation Factors Using Stem Cell-Based Human Embryo Models — Magdalena Zernicka-Goetz, California Institute of Technology ($2,872,697)
- Enhancing clinical predictability with novel models of iPSC-derived nociceptor for chronic pain — Fernando Aleman, Navega Therapeutics ($1,498,623)
- Unlocking the regenerative potential of hepatocyte plasticity for diseases of the biliary system — Holger F Willenbring, University of California, San Francisco ($4,871,000)
- IFN-γ suppresses AT2 cell regeneration to promote lung fibrosis — Peter Chen, Cedars-Sinai Medical Center ($4,525,190)
- Global profiling of miRNA-based gene activation to enable a new category of genetic medicine — Chi Zhang, Iris Medicine, Inc. ($2,997,574)
- 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)
- 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)
- A novel platform to rescue neurodevelopmental disorders caused by haploinsufficiency — Giordano Lippi, Calibr ($4,086,486)
- Base Editing, Single-Cell Multiomics, and Cardiac Organoids to Decode Genetic Variants — Joseph C. Wu, Stanford University ($4,606,248)
- 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)
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