CIRM Awards Nearly $100 Million to Boost Discovery, Translational, and Clinical Disease Research
FOR IMMEDIATE RELEASE
Contact:
Esteban Cortez
Director, Marketing & Communications
press@cirm.ca.gov
South San Francisco, CA, January 30, 2025 – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has awarded nearly $100 million to fund various projects from its Discovery, Translational, and Clinical programs.
“CIRM remains deeply committed to accelerating stem cell and gene therapies for patients in need,” said Rosa Canet-Avilés, PhD, Chief Science Officer at CIRM. “We are excited to support these groundbreaking projects, advancing research from discovery to clinical impact across neurological, psychiatric, cardiovascular, blood, and cancer-related diseases, as well as chronic pain.”
Among the awards are two preclinical projects (CLIN1) and two clinical trials (CLIN2) in the Agency’s clinical program, which provides funding for eligible stem cell and gene therapy-based projects through all stages of clinical trial development.
The successful applicants in CIRM’s Clinical program this month are:
| Application # | Program Title | Principal Investigator/Institution | Amount |
| CLIN1-16103 | Targeted DOK7 gene therapy for Congenital Myasthenic Syndromes | Sepulveda, Patricio — Amplo Biotechnology Inc. | $2,894,305 |
| CLIN1-17165 | Advancing a novel antisense oligonucleotide for the treatment of spinocerebellar ataxia type 3 (SCA3), a devastating neurodegenerative disease | Perlman, Susan — Cure Rare Disease | $5,692,538 |
| CLIN2-14796 | The UCLA Delayed Immunological Tolerance after Kidney Transplantation Program | Veale, Jeffrey — University of California, Los Angeles |
$7,343,925 |
| CLIN2-17083 | Phase 1b Study of TN-401 in Adults with PKP2 Mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) | Pollman, Matthew — Tenaya Therapeutics, Inc. | $8,000,000 |
A Potential Regenerative Therapy for Rare Neuromuscular Disorders
A $2.8 million preclinical award to Patricio Sepulveda, PhD, MBA, CEO of Amplo Biotechnology Inc. will advance AMP-101, a gene therapy product for the treatment of DOK7 Congenital Myasthenic Syndrome.
Congenital Myasthenic Syndromes (CMS) are a group of rare genetic neuromuscular disorders characterized by debilitating muscle weakness. People with DOK7 CMS face life-long challenges, including extreme muscular weakness, ventilatory crisis with infections, profound lethargy, and reliance on devices such as breathing aids, feeding tubes, and wheelchairs.
While the current standard of care is suboptimal for most patients, the proposed gene therapy would potentially be a long-term regenerative treatment.
By delivering a functional copy of the DOK7 gene to the patient, AMP-101 aims to correct the underlying genetic defect. This innovative therapy could significantly improve patient outcomes and transform the life for those living with this life-limiting disorder.
“It is with great pride, and an increased sense of responsibility towards the patients we wish to serve that we receive the support from CIRM. We are grateful of this opportunity and confident in our ability to transform the lives of CMS patients,” expressed Dr. Sepulveda.
Advancing a Gene Therapy Clinical Trial for an Inherited Heart Condition
An $8 million clinical award to Matthew Pollman, MD, of Tenaya Therapeutics will advance a clinical-stage gene therapy product, TN-401, for the treatment of Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) caused by plakophilin-2 (PKP2) gene mutations. PKP2 mutations are the most common genetic cause of ARVC, estimated to affect more than 70,000 people in the U.S. alone.
ARVC typically manifests in young adults and results in a high risk of life-threatening ventricular arrhythmias, sudden cardiac death, and progression to heart failure.
TN-401 gene replacement therapy is designed to address the underlying cause of disease by delivering a functional version of the PKP2 gene into heart muscle cells to potentially restore healthy function and slow or reverse disease progression.
“We are honored to receive this grant from the California Institute for Regenerative Medicine, which will support our ongoing RIDGE-1 clinical trial of TN-401, a potential best-in-class gene therapy intended to address symptomatic patients carrying PKP2 gene mutations, the most frequent genetic cause of arrhythmogenic right ventricular cardiomyopathy,” said Dr. Pollman, Senior Vice President of Clinical Development at Tenaya Therapeutics. “Our goal is to harness the power of gene therapy to correct the underlying cause of this severe and progressive disease, offering new hope for an improved quality of life to those the individuals and families affected by this debilitating condition.”
Dedicating $57.5 million to Advance Translational Stage Research
Additionally, CIRM awarded over $57 million to support 13 projects in its Translational program.
The goal of CIRM’s Translational program is to support promising stem cell-based or gene therapy projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use. Projects can include therapeutic candidates, diagnostic methods or devices, and novel tools that address critical bottlenecks in research.
The successful applicants in CIRM’s Translational program this month are:
| TRAN1-16924 | A novel non-genotoxic antibody conditioning therapeutic to expand access to and safety of HSCT and CD34+ gene therapies |
Chhabra, Akanksha — Maro Bio | $4,043,427 |
| TRAN1-16935 | Parkin Gene Therapy for Parkinson’s Disease | Johnston, Jennifer — NysnoBio GT Neurology | $2,450,510 |
| TRAN1-16959 | A Novel Gene Therapy to Target Glioblastoma via Custom-Engineered Adenovirus-Associated Viral Vectors | Tran, David — University of Southern California | $5,927,454 |
| TRAN1-16998 | FM-IL2 CAR T cells for Pancreatic Cancer | Allen, Gregory — University of California, San Francisco | $5,644,776 |
| TRAN1-16919 | Hematopoietic Stem Cell Gene Therapy for Alpha Thalassemia | Kohn, Donald — University of California, Los Angeles | $5,620,230 |
| TRAN1-16965 | Evaluation of an ex vivo lentiviral gene therapy for the treatment of Angelman syndrome | Hollis, Roger — University of California, Los Angeles | $5,843,083 |
| TRAN1-17069 | A targeted antisense oligonucleotide therapeutic strategy for Timothy syndrome | Pasca, Sergiu — Stanford University | $5,596,629 |
| TRAN1-16978 | Development of an AAV Epigenetic Gene Therapy for Gain-of-Function SCN9A Disorders and Chronic Pain | Moreno, Ana — Navega Therapeutics | $3,982,633 |
| TRAN1-16960 | Genetic Therapy Targeting mHTT mRNA and Somatic Expansion to Treat Huntington’s Disease | August, Paul — ReviR Therapeutics | $4,618,687 |
| TRAN1-17000 | GlyTR2 CAR T cell translation: safe pan-cancer killing via velcro-like density-dependent targeting of cancer glycans | Demetriou, Michael — University of California, Irvine | $4,581,144 |
| TRAN1-16994 | A gene therapy for the treatment of congenital lipodystrophy | Lewis, John — Entos Pharmaceuticals | $4,000,000 |
| TRAN1-16986 | Development of an off-the-shelf iPSC derived CAR T cell therapy for the treatment of solid tumors | Hosking, Martin — Fate Therapeutics, Inc. | $4,000,000 |
| TRAN4-17158 | Purpose built cell engineering for rapid manufacturing of stem-like cell therapies | Han, Sewoon — CellFE Biotech | $1,187,250 |
Funding Translational Research for Parkinson’s Disease
A $2.5 million award to Jennifer Johnston, PhD, CEO of NysnoBio GT Neurology will support translational research on an Adeno-associated Virus (AAV)-based gene therapy to deliver the Parkin gene to Parkinson’s Disease patients with mutations in the Parkin gene.
Currently, there are no disease-modifying therapies for Parkinson’s. The proposed gene therapy targets the most common genetic cause of Parkinson’s by restoring Parkin, a neuroprotective factor known to reinstate health to diseased cells.
If successful, the therapy has potential to be a curative therapy for the movement disorder associated with Parkinson’s, dramatically improving quality of life for patients in California and the world.
“NysnoBio is honored to be awarded this prestigious grant from CIRM, whose funding is critical to advance pioneering therapies to cure human disease,” said Dr. Johnston. “CIRM is a uniquely steadfast supporter of cell and gene therapies, removing roadblocks and creating efficiencies to ensure integration of new therapeutic modalities into the healthcare arsenal against human disease and suffering.”
Supporting a Gene Therapy to Treat Painful, Chronic Conditions
A $4 million grant to Ana Moreno, PhD, of Navega Therapeutics will support translational research to develop an AAV epigenetic gene therapy for gain-of-function SCN9A disorders and chronic pain.
The proposed gene therapy targets the gene SCN9A that encodes for Nav1.7, a sodium channel responsible for pain signal transmission, at the DNA level, to treat chronic pain.
If successful, the therapy may offer a potential treatment for patients suffering from debilitating conditions like Primary Erythromelalgia and Small Fiber Neuropathy, where traditional pharmacologic interventions fail or lead to opioid dependency.
This approach not only provides a significant advancement in treating pain without the risks of addiction but also sets a precedent for using gene therapy in other chronic conditions, potentially revolutionizing pain management and enhancing patient quality of life.
The translational award to Navega Therapeutics represents a pipeline progression from a previous CIRM Discovery (DISC2) award.
Supporting Collaborative Research for Neuropsychiatric Disorders
CIRM’s Board approved nearly $17 million to support two additional projects in the Agency’s pilot ReMIND Program. The approved awards will go to two research teams lead by Aparna Bhaduri, PhD, at UCLA and Mercedes Paredes, MD, PhD, at UCSF:
| DISC4-16337 | Defining Neurovascular Metabolism in Neurodevelopmental and Neuropsychiatric Disorders | Bhaduri, Aparna — University of California, Los Angeles | $10,330,000 |
| DISC4-16345 | Multi-gene modulation to rescue CNS-associated microdeletion syndromes | Paredes, Mercedes — University of California, San Francisco | $6,253,897 |
ReMIND–Research using Multidisciplinary, Innovative Approaches in Neuro Diseases–is an initiative designed to accelerate the discovery of mechanisms underlying neuropsychiatric disorders. The goal of this initiative is to provide a strong foundation for future translational and clinical investigations, potentially leading to the identification of novel targets and biomarkers.
“Effective treatments for neuropsychiatric diseases remain a significant unmet medical need in California, the US and the World,” said Dr. Canet-Avilés, Chief Science Officer at CIRM. “The ReMIND program is part of a deliberate drive at CIRM to support multidisciplinary discovery research as a way to catalyze innovation, especially in areas of large potential impact.”
In 2024, CIRM awarded $67.5 million to support five projects under the ReMIND program. These latest approvals bring the final number of funded teams to seven, comprising 44 investigators in total from 10 California research institutions. CIRM will support the network of funded teams by facilitating data and knowledge sharing over the next four years.
“Our hope for the ReMIND program is to catalyze innovative, cross-disciplinary collaborations,” said Senior Science Officer Chan Lek Tan, PhD. “We believe the funded teams represent a diverse set of approaches and questions and will move this goal forward.”
About the California Institute for Regenerative Medicine (CIRM)
The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research to deliver treatments for patients with unmet medical needs.
Established in 2004 through the passage of Proposition 71, CIRM was initially funded with $3 billion from the state of California to support ongoing research, and in 2020, was funded again with another $5.5 billion through Proposition 14 to continue the Agency’s important work.
CIRM has provided billions in funding to support stem cell, genetic research, and development programs in its portfolio. Through the Agency’s research, infrastructure, and education programs, CIRM aims to transform the field of regenerative medicine, stimulate economic growth, and improve the lives of diverse communities throughout the state.
For more information, go to www.cirm.ca.gov.