FOR IMMEDIATE RELEASE
Contact:
Esteban Cortez
Director, Marketing & Communications
press@cirm.ca.gov
South San Francisco, CA, March 27, 2025 — The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, approved new and amended funding program concepts which set the stage for providing up to $425 million in scientific research funding across discovery, preclinical and clinical research funding pillars.
The CIRM governing board also approved awarding $26.5 million to fund three projects aimed at addressing various conditions, including Angelman syndrome, a drug-resistant form of epilepsy, and graft-versus-host disease prevention.
The awards will support projects in the Agency’s late-stage preclinical and clinical programs, which provide funding for eligible stem cell and gene therapy-based projects through Investigational New Drug (IND)-clearance and all stages of clinical trial development. These awards bring the number of CIRM-funded clinical trials to 115.
The successful applicants in CIRM’s Clinical program this month are:
| Application # | Program Title | Principal Investigator/Institution | Amount |
| CLIN1-17103 | Expression of Ube3a by the hematopoietic system for the treatment of Angelman syndrome | Anderson, Joseph — UC Davis | $4,487,656 |
| CLIN2-17080 | TRX103 for prevention of GvHD (graft-versus-host disease) in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies | Roncarolo, Maria — Tr1X Inc. | $8,000,000 |
| CLIN2-17135 | Inhibitory Interneuron Cell Therapy for the Treatment of Drug-resistant Bilateral
Temporal Lobe Epilepsy |
Nicholas, Cory — Neurona Therapeutics | $13,999,983 |
“These funding decisions exemplify CIRM’s dedication to driving impactful, patient-focused research that addresses critical unmet medical needs,” said Rosa Canet-Avilés, PhD, Chief Science Officer at CIRM.
“We are particularly excited about these new awards because they underscore our strategic commitment to accelerating groundbreaking therapies from laboratory to patient care. These commitments not only reflect the innovative strength and collaborative spirit of California’s scientific community but also advance our mission to deliver transformative regenerative medicine solutions to those in need. We are eager to support these promising projects as they progress through clinical development and look forward to continued engagement and collaboration on cutting-edge research initiatives in the coming months.”
CIRM board approves new and amended funding program concepts
The CIRM governing board has approved several new funding initiatives aimed at advancing regenerative medicine research in California. These include:
- DISC4: Part of CIRM’s Discovery program, this will fund large, collaborative teams using diverse technologies to address gaps in understanding human diseases.
- DISC5: Also part of the Discovery program, this will support innovative research by pairs of investigators focused on stem cell biology or regenerative medicine.
- PDEV: Part of CIRM’s Preclinical Development program, this will support completion of preclinical development, FDA IND clearance, and clinical trial startup for stem cell-based and genetic therapies.
- CLIN2: Part of CIRM’s Clinical Development program, this will fund clinical trials for stem cell-based and genetic therapies, promoting innovative trial designs, market access strategies, and pre-commercialization activities.
Additionally, the Board approved an updated funding concept for Community Care Centers of Excellence (CCCE), which aims to expand access to regenerative medicine treatments across California.
Jon Thomas, PhD, JD, CIRM President & CEO, stated, “With the adoption of these concept plans, CIRM remains fully committed to funding discovery, preclinical, and clinical research, with up to $425 million allocated to these recurrent programs. At a time of federal funding uncertainty, CIRM is resolute in its support for cell and gene therapy research, as made possible by California voters through Proposition 14.”
CIRM will post the final program announcements and details on its website throughout the spring and fall. Researchers interested in applying should visit our funding opportunities page and are encouraged to sign up for email alerts.
These efforts align with recommendations from CIRM’s Strategic Allocation Framework (SAF), a structured and data-driven effort designed to prioritize resources and maximize the impact of CIRM’s funding.
Dr. Canet-Avilés said, “I’m incredibly proud of the teamwork that made these concepts and awards possible in such a short time. This has been a true collective effort, from our dedicated CIRM staff across programs, grants management, review, legal, and governance to the invaluable leadership of our Science Subcommittee and Neuro Task Force. It’s a testament to our shared commitment and what we can achieve together as we continue driving innovation and advancing therapies for patients.”
A $4.5 Million Award to Advance Research for Angelman Syndrome
A $4.5 million award will fund an IND-enabling research project led by Joseph Anderson, PhD, MAS, of UC Davis, aimed at advancing a gene-modified stem cell therapy for Angelman syndrome.
This rare neurodevelopmental disorder, which manifests in early childhood, causes seizures, movement and balance issues, and intellectual disabilities. Angelman syndrome occurs in one in 15,000 live births or 500,000 people worldwide. Currently, there is no effective treatment, and care is limited to symptom management.
The proposed therapy involves modifying a patient’s own hematopoietic stem cells with a corrected Ube3a gene, which provides instructions for making a protein called ubiquitin protein ligase E3A. Studies suggest that the ubiquitin protein ligase E3A plays a role in the normal development and function of the nervous system.
In this study, the transplanted cells would deliver functional Ube3a protein, potentially preventing, halting, or reversing the symptoms of Angelman syndrome.
“Our team is very grateful to receive this funding from CIRM to further progress our therapy for AS,” said Dr. Anderson. “From our original concept of using a modified yet functional form of Ube3a in a hematopoietic stem cell setting, we look forward to bringing this closer to helping patients.”
$8 Million to Fund a Trial for Graft-Versus-Host Disease Prevention in Stem Cell Transplants
The CIRM governing board has approved an $8 million award to support a Phase I clinical trial led by Maria Grazia Roncarolo, MD, of Tr1X Inc. The trial will assess the safety and tolerability of TRX103, a cell therapy aimed at preventing graft-versus-host disease (GvHD) in blood cancer patients undergoing mismatched stem cell transplants.
GvHD occurs when donor cells attack the recipient’s tissue and organs, often causing life-threatening complications. Current treatments for GvHD, which suppress the immune system, can lead to infections and hinder cancer treatment effectiveness. TRX103, an allogenic engineered regulatory T cell product, may improve transplant access for patients without a matched donor while reducing GvHD risks.
“With CIRM’s support, TRX103 has rapidly advanced from promising preclinical findings into clinical trials, bringing us closer to transforming mismatched stem cell transplants into safer, life-saving options,” said Dr. Roncarolo. “By harnessing the unique power of engineered Tr1 cells, our goal is to prevent graft-versus-host disease, improve immune reconstitution and induce tolerance to host cells—therefore expanding access to curative therapies for patients who urgently need them.”
“This trial represents a hope for all of us transplanters—not only for better outcomes but also for a future where effective, personalized treatments like hematopoietic stem cell transplant become accessible to all patients, regardless of donor match.”
This award approval follows a $4 million CIRM grant received by Tr1X Inc. in early 2024 for late-stage preclinical research, helping advance the therapy to human trials. If successful, TRX103 could increase access to curative stem cell transplants while mitigating GvHD risks.
Advancing an Innovative Cell Therapy for Drug-Resistant Epilepsy
A $14 million award will fund a Phase 1/2 clinical trial led by Cory R. Nicholas, PhD, CEO and co-founder of Neurona Therapeutics, to advance NRTX-1001, the first human cell therapy candidate for drug-resistant bilateral (both sides of the brain) temporal lobe epilepsy (TLE).
Epilepsy, a neurological disorder causing recurring seizures, affects over 3 million people in the U.S., including more than 500,000 in California, and the temporal lobe is the most common region of seizure-onset. While anti-seizure medications are the primary treatment, approximately one-third of patients experience drug-resistant seizures, which severely impact their quality of life. Current treatments for drug-resistant epilepsy often involve invasive lobectomy surgeries that destroy brain tissue and carry serious, irreversible risks. Moreover, patients with bilateral TLE are typically not eligible for resection or ablation surgeries due to the risk of profound and permanent memory loss.
NRTX-1001 could potentially provide a novel, non-destructive alternative to control seizures and avoid neurocognitive impairment. This investigational therapy uses inhibitory neurons to restore balance in the brain’s circuits and suppress seizures. “By administering these inhibitory neurons into targeted parts of the brain, we aim to provide the necessary ‘brake’ that is missing in patients with epilepsy,” explained Dr. Nicholas.
Supported by CIRM from its early discovery stage to the current first-in-human trial, NRTX-1001 has shown potential to treat this form of epilepsy, offering new hope for patients with limited treatment options. In December 2024, the company reported encouraging results from its ongoing Phase 1/2 trial of NRTX-1001 in patients with drug-resistant unilateral (one side of the brain) TLE that indicate the cell therapy has been well-tolerated at both high and low doses and may significantly reduce seizures. It was subsequently announced that the FDA had agreed to the design of a Phase 3 trial planned to start enrolling later this year in patients with unilateral TLE.
“We are sincerely grateful for the continued support and guidance from CIRM,” said Dr. Nicholas. “NRTX-1001’s progress is a testament to CIRM’s vision and commitment to funding innovative science from bench to bedside. With this new award, we now have the opportunity to expand potential indications for NRTX-1001 to include the drug-refractory bilateral TLE population, which is of highest unmet medical need in epilepsy. This grant is a big win for the epilepsy community of patients and families in need of alternative treatment options.”
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.
