The California Institute for Regenerative Medicine (CIRM), the world’s largest regenerative medicine organization, awarded $43.8 million to advance stem cell and gene therapy research in California.
Infrastructure & ReMIND
The awards will support projects in CIRM’s Clinical, Discovery, and Infrastructure programs.
Twelve projects received more than $24.1 million through CIRM’s Quest Awards Program.
The program supports promising stem cell and gene therapy technologies. These technologies could eventually improve patient care and reach a broad patient population.
CIRM also approved a new plan for the ReMIND program.
ReMIND stands for Research using Multi-disciplinary, Innovative Approaches in Neuro Diseases.
The program will support basic research into neuropsychiatric diseases.
CIRM expects to award up to $110 million for California research studies between 2024 and 2028.
Awards approved
| 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 |
Adult-onset leukoencephalopathy
A $1.4 million award to Dr. Sunil P. Gandhi, PhD, will support NGL-101 development.
Gandhi serves as Novoglia, Inc.’s founding chief technology officer.
NGL-101 is a cell therapy for adult-onset leukoencephalopathy with axonal spheroids and pigmented glia, or ALSP.
A mutation in the CSF1R gene causes this rare neurological condition.
The gene appears in the outer membrane of certain cells.
ALSP can reduce cognitive function and mobility. The condition affects between 10,000 and 25,000 Americans.
Patients have very few treatment options.
Novoglia researchers will conduct preclinical studies to evaluate a gene-corrected, stem cell-derived microglia therapy.
They will assess the therapy’s safety and effectiveness.
Microglia support brain development, maintain neuronal networks, and repair injuries.
Successful results could improve treatment options for people with ALSP.
The research could also support similar cell-replacement strategies for other central nervous system diseases.
“This award advances CIRM’s mission to support treatments for brain and central nervous system diseases,” said Maria T. Millan, MD, CIRM’s president and CEO.
“These conditions affect millions of people.”
“This follow-on award also reflects our commitment to addressing the unmet medical needs of people with degenerative diseases.”
Investing in a clinical trial to fight brain tumors
In addition to the Quest awards, CIRM awarded nearly $12 million to Dr. Reena Thomas, MD, PhD, of Stanford Medicine.
The funding will support a Phase 1 clinical trial. The trial will evaluate a CAR T-cell therapy for glioblastoma multiforme, or GBM.
GBM is the most common malignant primary brain tumor in adults.
About 12,000 Americans receive a GBM diagnosis each year. Fewer than 10% survive for five years.
CAR T-cell therapy modifies a patient’s T cells, which are immune cells.
Researchers add a protein called a chimeric antigen receptor, or CAR, to the cells.
They then return the modified CAR T cells to the patient.
The cells help the immune system identify and destroy cancer cells.
If successful, this therapy could transform treatment for this deadly brain tumor.
“This project introduces a new target and expands CIRM’s CAR T-cell therapy portfolio,” said Maria T. Millan, MD.
The project targets deadly cancers, including glioblastoma, that resist treatment.
The clinical award comes from CIRM’s Clinical, or CLIN2, program.
CLIN2 awards support clinical trials for regenerative medicine therapies. These therapies include stem cell and gene therapies that address unmet medical needs.
This award brings the total number 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).