CIRM invests $44 million to advance regenerative medicine research and manufacturing in California
For immediate release
Sr. Director, Marketing & Communications
South San Francisco, CA – The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, awarded $43.8 million to fund projects aimed at advancing stem cell and gene therapy research in California. The awards will support projects in the Agency’s Clinical, Discovery, and Infrastructure programs.
Among the awards are 12 projects totaling more than $24.1 million from CIRM’s Quest Awards Program, which promotes the discovery of promising new stem cell-based and gene therapy technologies that could be translated
to enable broad use and ultimately improve patient care.
Additionally, CIRM approved a new plan for the ReMIND (Research using Multi-disciplinary, Innovative Approaches in Neuro Diseases) program which will support basic research in neuropsychiatric diseases. The program is estimated to award up to $110 million to fund research studies in the state of California from 2024 to 2028.
Awards approved at the CIRM September Independent Citizens’ Oversight Committee (ICOC) meeting include:
|Application #||Program Title||Principal Investigator/Institution||Amount|
|DISC2-14897||Assessing the Functional, Immunologic and Microbiologic Characteristics of Human Livers Created in Chimeric Pigs||Sher, Linda – University of Southern California||$2,736,590|
|DISC2-14899||RNA-based therapeutics to augment regulatory T cells: a novel approach to treat myocarditis||Liao, Ke – Cedars-Sinai Medical Center||$2,264,509|
|DISC2-14907||A First-in-Class Treatment for Progressive Multifocal Leukoencephalopathy Via Multimodal Immune System Engineering||Wilson, Michael – University of California, San Francisco||$2,182,396|
|DISC2-14935||Orthogonal IL2 Receptor Transduced Regulatory T Cells for Clinical Application||Negrin, Robert – Stanford University||$1,920,652|
|DISC2-14963||Development of an Optogenetic Vision Restoration Gene Therapy Using an Engineered Form of Melanopsin||Baker, Cameron – Adverum Biotechnologies||$1,150,820|
|DISC2-14982||Treatment of Myasthenic Syndrome due to Choline Acetyltransferase Deficiency Using AAV9-mediated Gene Therapy||Maselli, Ricardo – University of California, Davis||$1,500,000|
|DISC2-15010||C9orf72 repeat expansion-tuned allelic suppression by CRISPRi as an ALS therapy||Ho, Ritchie – Cedars-Sinai Medical Center||$2,274,768|
|DISC2-15119||Drug discovery for gastrointestinal motility disorders using hPSC-derived enteric ganglioids||Fattahi, Faranak – University of California, San Francisco||$1,954,367|
|DISC2-15120||Gene-corrected human microglia for the treatment of adult onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)||Gandhi, Sunil – Novoglia, Inc.||$1,417,948|
|DISC2-15137||Inhibitory interneurons derived from human induced pluripotent stem cells to treat stroke||Lorenzo Llorente, Irene – Palo Alto Veterans Institute for Research||$2,140,122|
|DISC2-14900||Developing a breast cancer stem cell drug||Clarke, Michael — Stanford University||$2,293,051|
|DISC2-15114||Development of a VAV2 antisense oligonucleotide (ASO) treatment for ALS||Ichida, Justin — University of Southern California||$2,296,376|
A $1.4 million award to Sunil P. Gandhi, PhD, Founding Chief Technology Officer of Novoglia, Inc. will support the development of NGL-101, a cell therapy for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).
ALSP is a rare neurological condition caused by a genetic mutation in the CSF1R gene found in the outer membrane of certain cells. ALSP is characterized by symptoms of reduced cognitive function and mobility and affects between 10,000 and 25,000 Americans. There are very few treatment options for ALSP.
In the proposed research, investigators at Novoglia, Inc. will conduct preclinical studies to test the efficacy and safety of a gene corrected stem cell-derived microglia. Microglia are cells of the brain that regulate development, maintenance of neuronal networks, and injury repair.
If the study is successful, it may have the potential to greatly improve the health of ALSP patients by opening the door to future therapeutic possibilities. This research may also aid in the development of similar cell replacement strategies for other diseases and conditions affecting the central nervous system.
“This award advances CIRM’s mission to support the research and development of treatments for diseases and conditions of the brain and central nervous system that affect millions,” says Maria T. Millan, MD, President and CEO of CIRM. “This investment is also a follow-on funding from a prior CIRM award, reflecting our commitment to address the unmet medical needs of those impacted by degenerative diseases.”
Investing in a clinical trial to fight brain tumors
In addition to the Quest awards, CIRM awarded nearly $12 million to Reena Thomas, MD, PhD of Stanford Medicine for a phase 1 clinical trial to assess the safety of a CAR T cell therapy for glioblastoma multiforme (GBM), the most common malignant primary brain tumor in adults.
Each year about 12,000 Americans are diagnosed with GBM and the 5-year survival rate is less than 10 percent.
In CAR T cell therapy, a patient’s own T cells (immune cells) are modified with a protein called chimeric antigen receptor (CAR). These newly created CAR T cells are reintroduced back into patients to help the immune system identify and destroy cancer cells. If successful, the therapy could potentially transform the landscape for this lethal type of brain tumor.
“This project represents a new target that expands CIRM’s portfolio of CAR T cell therapies for deadly refractory cancers like glioblastoma,” says Maria T. Millan, MD.
The clinical award is part of CIRM’s Clinical (CLIN2) awards, which support the completion of a clinical trial for a regenerative medicine-based therapy (stem cell-based or gene therapy) that addresses an unmet medical need.
This award brings the total of CIRM-funded clinical trials to 96.
Building a California Cell and Gene Therapy Manufacturing Network
CIRM also awarded $7.7 million to four additional facilities as part of the first phase to build a California Cell and Gene Therapy Manufacturing Network, bringing the total of funded facilities to nine. The awards include:
|Application #||Program Title||Principal Investigator/Institution||Amount|
|INFR5-14562||UCSD Advanced Cell Therapy Laboratory||Kaufman, Dan – University of California, San Diego||$2,000,000|
|INFR5-14574||The UCI GMP Cell and Gene Therapy Manufacturing Facility||Anderson, Aileen – University of California, Irvine||$2,000,000|
|INFR5-14667||Advancing Cell Therapy Manufacturing Through Collaboration||Abou-el-Enein, Mohamed – University of Southern California||$1,999,964|
|INFR5-14779||Creating A Collaborative California Cell and Gene Therapy Manufacturing Network||Fury, Brian – University of California, Davis||$1,719,365|
The statewide network is designed to overcome manufacturing bottlenecks that have delayed or stalled development and approval of regenerative medicines.
Phase one of the awards will fund California non-profit GMP manufacturing facilities for two years. Each facility will support accelerating and de-risking the path to commercialization for cell and gene therapies, advancing industry standards and quality by design, and building a diverse and highly skilled manufacturing workforce in California.
Supporting multidisciplinary discovery-stage research for neuropsychiatric diseases
The CIRM governing board also approved the ReMIND Program, a set of new funding opportunities to support multidisciplinary discovery stage research in neuropsychiatric diseases.
The goal of the new program is to accelerate research innovations that would improve our understanding of the biology of neuropsychiatric diseases. The program includes two distinct and separate funding opportunities: the first, ReMIND-L, supports large collaborative multidisciplinary projects and the second, ReMIND-X, supports exploratory, high risk, high reward projects.
The program was developed in response to Proposition 14’s mandate to dedicate at least $1.5 billion to the support of research and development of treatments for diseases and conditions of the brain and central nervous system (CNS).
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.
With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.
For more information go to www.cirm.ca.gov