CIRM invests $1.5 million to UCLA for research identifying risk factors for viral infections

Image of Michael Wells, PhD from UCLA

The California Institute for Regenerative Medicine (CIRM) recently awarded $1.5 million to a new research project led by Michael Wells, PhD, of UCLA, that aims to identify the genetic and molecular risk factors underlying disease-causing viral infections in multiple diverse ancestral populations.

Dr. Wells and his team of researchers will be using a stem cell-based “cell village” platform to analyze factors underlying differences in viral susceptibility across 150 donors. These cell villages are composed of cells that capture the immense diversity of racial and ethnic groups across California, creating an environment for comprehensive analysis of genetic variants and cellular characteristics.

“As the personalized medicine revolution begins to take shape, it’s imperative that we ensure our findings are relevant to all people. Cell villages could play a significant role in addressing societal issues like the health disparities experienced by women and ethnic minorities in the United States,” said Dr. Wells.

This research has the potential to impact health disparities and advance future antiviral drug development. By studying the biological factors that influence differences in immune response to viruses, the project could provide valuable insights into how to develop more effective treatments.  

“CIRM is dedicated to promoting diversity, equity, and inclusion in the research we fund. Projects such as this one, harnessing knowledge utilizing stem cell lines from a wide array of ancestral backgrounds, underlines our commitment to research that is reflective of our diverse population and is a cornerstone of our overarching objectives,” said Rosa Canet-Avilés, PhD, Vice President of Scientific Programs at CIRM.


Images of cell-villages above were provided by Dr. Michael Wells’ Lab and UCLA.

Equipping California nurses with skills to support cell and gene therapy patients 

Regenerative medicine is a rapidly evolving field. As more therapies reach commercialization and clinical trial stages, there remains an immediate need to equip direct patient care nurses with skills to support patients on complex clinical trials as well as novel standard-of-care therapies. 

To address this need and continue efforts to build a regenerative medicine workforce in California, the California Institute for Regenerative Medicine (CIRM) partnered with City of Hope and the Alpha Clinics Network to host the Regenerative Medicine Nurse Training Symposium in April. Nearly 200 people joined the event virtually and in person at City of Hope’s Duarte campus near Los Angeles.  

The CIRM-funded Alpha Clinics are a network of top California medical centers specializing in delivering stem cell and gene therapy clinical trials to patients. Each Alpha Clinic site contributed educational materials for the symposium.  

A Dynamic Learning Experience for California Nurses

The two-day program provided a dynamic learning experience to direct care registered nurses, clinical research nurses, advanced practice providers and others seeking to enhance their knowledge in regenerative medicine. The event aimed to empower nurses to disseminate accurate, evidence-based information to their students and colleagues, ultimately advancing the field of regenerative medicine nursing in California.  

CIRM Board Chair Vito Imbasciani, PhD, MD, kicked off the event and provided an overview of CIRM’s history and the important role nurses play in advancing the state agency’s mission to “accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world.” 

“You are pioneers,” said Imbasciani. “You are at the threshold of a dawning era where cell and gene therapies, as well as regenerative medicine as a whole, will become more and more commonplace and a part of routine clinical care.” 

Throughout the event, attendees were immersed in engaging and practical sessions, emphasizing nurses’ pivotal roles in the realm of cell and gene therapy, particularly through CIRM’s Alpha Clinics. Attendees were eligible to receive up to 14 Continuing Education Units (CEUs) for both days of attendance. 

Highlighting the Patient Experience

Numerous presenters described the patient journey in clinical trials, highlighting that patients often encounter financial and logistical challenges participating in cell and gene therapy trials because they require many visits to a medical center. CIRM’s Associate Director of Patient Access Geoff Lomax underscored the patient journey and the value of CIRM’s Patient Support Program and Community Care Centers of Excellence, emerging CIRM programs aimed at providing financial, logistical and social support for patients in clinical trials. 

Symposium speakers also highlighted the critical importance of nurses in healthcare, especially in the administration of cutting-edge treatments like CAR-T Cell therapies. A nurse participant recognized that many nursing educational courses don’t include regenerative medicine-focused trainings and emphasized the need for training in such therapies to provide the best care to patients. 

Other topics discussed at the event included apheresis—a technology that separates donated blood components to treat certain illnesses—and Good Manufacturing Practices (GMP). Because nurses play an integral role in these processes, several event sessions were dedicated to practical considerations.  

Representatives from bluebird bio, a biomedical company and event sponsor, presented exciting developments in gene therapy, highlighting the recent FDA approval of LYFGENIA, a gene therapy treatment for sickle cell disease. 

The symposium fostered networking opportunities among attendees, enabling them to exchange ideas, experiences, and best practices with peers and staff throughout the state.  

“Symposiums like these are so important for nurses because we get to hear about all these different therapies that we don’t typically work with,” said Emma King, an attendee from Children’s Hospital Los Angeles. “You can see what’s coming in the future and what different hospitals around California do for regenerative medicine and hear about patient experiences. It makes you a better, well-rounded nurse.”  

Kelly Tomlinson, DNP, RN, NEA-BC, is the administrative director of Immune Effector Cell Therapy at host hospital City of Hope and was both a moderator and speaker at the symposium. She said regenerative medicine “is the future.”  

“This symposium helped to educate and engage our nursing workforce by giving them new tools and knowledge within this emerging field,” Tomlinson said. “The expertise at this event was astounding. I was honored to not only be a participant but a moderator and speaker. I left with a wealth of knowledge and a renewed excitement for what the future holds for our patients and communities.”  


Thank you to the City of Hope Alpha Clinic for organizing the event and to each CIRM Alpha Clinic for contributing speakers and materials for the program. The event was sponsored by Johnson & Johnson, bluebird bio, Kite Pharma, and AbelZeta. Senior Advisor, Community Outreach and Advocacy Jacqueline Hantgan contributed to this article. 

CIRM awards $25 million for discovery and clinical research, infrastructure programs

Stock image of a researcher

The California Institute for Regenerative Medicine (CIRM) has recently approved awarding $25 million to fund multiple projects across discovery research, clinical research, and infrastructure programs.

This funding will support 11 projects under CIRM’s Foundation Awards Program, which aims to drive rigorous studies addressing critical basic knowledge gaps in the biology of stem cells and regenerative medicine approaches and advance stem cell-based tools.

Additionally, CIRM awarded $6 million to Eugenio Cingolani, PhD, of Cedars Sinai, to support a preclinical project in the Agency’s clinical awards (CLIN 1). The project is designed to advance a noninvasive cell-derived therapy for ventricular tachycardia, a condition in which the heart rhythm is irregular.

“CIRM is dedicated to the advancement of early-stage transformative therapies for prevalent health conditions such as heart disease,” said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM.

CIRM also awarded $2.5 million to support establishing a Patient Support Program (PSP) to enhance patient access to CIRM-funded clinical trials, an essential component of CIRM’s mission and Infrastructure Program.

The discovery awards approved at CIRM’s March Independent Citizens’ Oversight Committee (ICOC) meeting include:

Application #  Program Title  Principal Investigator/InstitutionAmount  
DISC0-15949Neuroimmune interactions in the developing human brainNowakowski, Tomasz – UCSF$1,626,000
DISC0-15737Village-based identification of human risk factors for viral neuropathogenesisWells, Michael F. – UCLA$1,577,448
DISC0-15921Interrogating Satellite Cell and Myofiber Defects and Repair in Human DMD using Single Nuclei/Single Cell RNA Sequencing of Muscle Resident CellsMiceli, M. Carrie – UCLA$1,578,000
DISC0-1603939 Lewy body dementia α-synuclein, and cell-specific mechanisms of neurodegenerationFinkbeiner, Steven M. – Gladstone$1,739,760
DISC0-16122Mapping and modeling endothelial cell fate decisions for pulmonary arterial hypertensionQiu, Xiaojie – Stanford$1,540,798
DISC0-156544 Modeling and understanding alveolar hypoplasia in Down syndrome using iPSCs-derived alveolar type II cellsAl Alam, Denise – Lundquist Institute$1,524,196
DISC0-15816Investigating the SGF29/SAGA complex in regulation of normal and cancer stem cellsDeshpande, Aniruddha – Sanford-Burnham$1,647,600
DISC0-15774Modeling of GATAD2B-associated neurodevelopmental disorder and NuRDopathies: Investigation of cellular & molecular anomalies altering neurodevelopmentPierson, Tyler Mark – Cedars-Sinai$1,318,441
DISC0-15972Immune cloaking of human stem cell-derived insulin producing cells for curative cell therapy without immunosuppressionDigovich, Katy – Minutia, Inc.$1,192,586
DISC0-15920Harnessing the rejuvenating capacity of pregnancy-associated factors to restore aged stem cell functionAlperin, Marianna – UCSD$1,539,520
DISC0-15689Utilizing Age-Specific Adipocyte Progenitor Cells for Cell Therapy in Older PatientsWang, Qiong Annabel – City of Hope$1,508,997

State agency launches ‘Championing Change for Sickle Cell in California’ campaign

The California Institute for Regenerative Medicine (CIRM), established by the voters of California, is accelerating research and awareness of Sickle Cell Disease.

The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, is proud to announce the launch of “Championing Change for Sickle Cell in California,” a pioneering campaign aimed at increasing awareness of Sickle Cell Disease (SCD) and research aimed at improving the treatment landscape for the condition.

With more than 100,000 Americans and 20 million people worldwide affected by this debilitating condition, CIRM is committed to funding cutting-edge cell and gene therapy programs that have the potential to transform the lives of SCD patients.

Sickle cell disease is a genetic disorder that causes red blood cells to assume a sickle shape under stress, leading to clogged blood vessels, excruciating pain crises, and organ damage.

This disease disproportionately affects people of African American and Latinx heritage. It is estimated that SCD occurs among about 1 out of every 365 Black or African-American births. Latinx Californians make up 7.3 percent of sickle cell disease cases—and Latinx newborns make up 11 percent of new cases.

In collaboration with key organizations such as sickle cell patient advocacy group Axis Advocacy, the National Heart, Lung, and Blood Institute’s (NHLBI) Cure Sickle Cell Initiative, and the UCSF Sickle Cell Center of Excellence, CIRM is working to champion change and drive progress for sickle cell treatments.

Community partners and patient advocates play a crucial role in the development and success of these treatments, and CIRM recognizes their importance. Among the key calls to action of the campaign include becoming a sickle cell disease advocate, spreading awareness of clinical trial options for sickle cell, downloading a fact sheet and learning more about the NHLBI Cure Sickle Cell Initiative.

In addition to spreading awareness of this condition, the campaign highlights how CIRM is funding research into stem cell, gene therapy, and other regenerative medicine procedures for sickle cell disease. To date, CIRM has invested over $65 million towards research for sickle cell, ranging from basic discovery to clinical trials.

Evie Junior participated in a CIRM-funded clinical trial for sickle cell disease at UCLA. Photo: Jaquell Chandler

CIRM and the NHLBI are co-funding two clinical trials that are investigating different gene therapy approaches for patients with severe sickle cell disease. These trials include a Phase 1/2 trial led by Dr. Mark Walters of UCSF Benioff Children’s Hospital Oakland and a Phase 2 trial led by Dr. David A. Williams of Boston Children’s Hospital. These clinical trials are essential in the search for new therapies to treat sickle cell disease.

CIRM’s new campaign comes at the heels of two recently FDA-approved gene therapy treatments for sickle cell disease. Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia have brought forth a new era of treatment options for people living with the condition.

CIRM introduced the “Championing Change for Sickle Cell in California” campaign at Oakland’s Black Joy Parade in February 2023. Pictured is CIRM’s Community Outreach Manager Aditi Desai.

“As the first regulatory authorization for a CRISPR-based gene-editing therapy in the world, this approval not only marks a milestone in the advancement of cell and gene therapies, but it also marks a transformative step in the treatment landscape for the millions of people impacted by sickle cell disease,” said CIRM Vice President of Therapeutics Development, Abla Creasey, PhD.


For more information on CIRM’s Sickle Cell Disease campaign, ongoing research projects, and ways to champion change, please visit cirm.ca.gov/sickle-cell-disease. To get involved in the campaign, please contact CIRM’s Community Outreach Manager Aditi Desai at adesai@cirm.ca.gov.

[VIDEO] Igniting innovation: How CIRM is bringing new and diverse perspectives in regenerative medicine 

CIRM has produced videos highlighting the experiences of participants from our educational and training programs.

At the California Institute for Regenerative Medicine (CIRM), we are passionate about catalyzing growth in regenerative medicine while championing inclusivity. One of CIRM’s strategic goals is to build a diverse and skilled workforce that represents the rich tapestry of California’s population.  
 
That’s why the CIRM—spearheaded by the Scientific Programs & Education team—is launching a set of videos to showcase the unique perspectives of CIRM’s educational and training program participants from across the state, including students, trainees, program directors, and CIRM leadership. Watch both videos on CIRM’s YouTube channel here and here.

Funding Training Programs for a Diverse California 

CIRM’s education and training programs support the next generation of regenerative medicine scientists, ranging from high school and undergrad students to doctoral candidates and clinical fellows.  
 
CIRM funds four unique internship programs, each with its own set of requirements: SPARK (high school), Bridges (Undergraduate and Masters), COMPASS (Undergraduate), and Scholars (Pre-Doctoral, Post-Doctoral, and Clinical Fellows). 

Each program offers hands-on experience to foster a workforce adept in stem cell science and the technical skills that are crucial for present and future scientific and medical advancements.  
 
Partnering with community colleges and universities, as well as research institutions and biotechnology companies, each program aims to attract diverse talent to regenerative medicine by offering varied career paths and accessibility across a broader educational spectrum.  

Aileen Nava is doctoral student at UCLA and a CSU Fullerton Bridges alumna from 2017

To date, CIRM’s educational and training programs have supported 3,800 interns and fellows, the majority of whom have achieved employment or chose to pursue further education in regenerative medicine or related fields.  
 
Alumni from these programs participate in a broad spectrum of rewarding careers. These range from traditional scientific disciplines to in-demand, complementary and/or ancillary pressions within education, community and patient advocacy, business development, and regulatory affairs. Over 70 institutions have joined CIRM in its mission to advance world class science, deliver real world solutions, and provide opportunities to all.  

Opportunities for People with Various Educational Backgrounds 

In addition to highlighting experiences of program participants, the videos emphasize that careers in regenerative medicine aren’t limited to those with advanced degrees like PhDs or MDs. There are diverse roles and job opportunities accessible to individuals with various educational backgrounds and skill levels.  

David Mendoza Bautista is a masters student intern at Novo Nordisk and a Cal Poly Bridges Trainee.

“Often [students] are first gen, [from] underrepresented groups, these are an amazing group of students who are getting their first opportunity both at the higher education and also the first in their families to do research in labs,” said Bridges Program mentor Samantha Butler, PhD. 
 
At CIRM, diversity fosters innovation. In addition to academic and industry research opportunities, CIRM aims to grow the entire regenerative medicine workforce pipeline, including opportunities in therapeutics manufacturing and clinical trials.  
 
To learn more about CIRM’s educational programs, including information on how to apply, visit our website at cirm.ca.gov/education


CIRM thanks all program participants, program directors, and CIRM leadership who made these videos possible. Video produced by CIRM’s Scientific Programs & Education team and Hartlove-Goodyear. 

Advancing Clinical Research for a CAR T-Cell Therapy for Systemic Lupus Erythematosus (SLE), Autoimmune Diseases

Symptoms of Lupus may include pain or swelling in the joints. Image is of swollen hands.
Symptoms of lupus may include pain or swelling in the joints, extreme fatigue, a butterfly rash on the cheeks and nose, and more.

The California Institute for Regenerative Medicine (CIRM) has awarded $7.9 million to Barbara Hickingbottom, MD, of Fate Therapeutics to advance clinical research for FT819, an induced pluripotent stem cell (iPSC)-derived CD19 CAR T-cell therapy for Systemic Lupus Erythematosus (SLE).

SLE is a debilitating autoimmune disease and affects more than 200,000 Americans, particularly women of color.

FT819 targets B cells with the aim to reset the immune system and provide drug-free remission for patients with autoimmune diseases. Fate manufactures FT819 using a clonal master iPSC line as a renewal cell source, providing a uniform cell therapy product that is mass produced and delivered off-the-shelf to patients.

As a result, FT819 is designed to bring the curative potential of cell therapy to large numbers of patients with SLE and other autoimmune diseases. 

Anatomical diagram of Lupus Erythematosus symptoms.

“This innovative approach shows great promise in transforming clinical practice for Systemic Lupus Erythematosus, providing a new potential treatment option for individuals and families affected by this challenging disease,” added Dr. Creasey.

“CD19 CAR T cell therapy has demonstrated tremendous potential for patients with autoimmune diseases,” said Dr. Hickingbottom. “We look forward to partnering with CIRM to broadly realize this potential with FT819, the industry’s first CAR T-cell therapy manufactured from a clonal master iPSC line to reach clinical investigation.” 

Former CIRM Bridges Intern Receives Rising Black Scientist Award

Image of Kevin Brown Jr. in a lab coat.
Photo Credit: California State University, San Marcos.

Cal State San Marcos cellular and molecular biology student and former California Institute for Regenerative Medicine (CIRM) Bridges intern Kevin Brown Jr. has been awarded the Rising Black Scientist Award from the journal Cell Press, Cell Signaling Technology (CST), and the Elsevier Foundation.

Brown was selected from a pool of more than 350 applicants in various sciences. In the application, Cell Press asked emerging black scientists to share their scientific vision, experiences that sparked their interest in science, how they want to contribute to a more inclusive scientific community, and how these all fit together on their journey.

The awards were created in 2020 to break down barriers and provide visibility and funds to support talented black scientists in the life or medical sciences on their career journey. The awards have since then been expanded to include the physical, earth and environmental, and data sciences.

In addition to the publication of their essays in the prominent journal Cell, Brown and three other winners will receive $10,000 to support their research and a $500 travel grant.

How an Operating Table Experience Inspired Brown’s Passion for Science 

Brown’s essay “From the operating table to global science: How a near-death experience sparked my passion for life” details his experience receiving surgery for a heart condition as a child.

“The surgeon, a tall man with a cascade of dark hair framing his sharp facial features, not only put my anxiety to rest but also planted a seed of curiosity in my mind as he gracefully explained how he intended to ‘fix my heart,’” Brown wrote in his winning essay.

“The day I left the hospital, I told myself I’d leave my positive impact on the world through science and medicine, and I haven’t stopped striving for that since.”

Image of Kevin Brown Jr.

He further explains his goal toward developing a robust stem cell model to explore personalized approaches into modern medical practice.

“My personal exploration into how stem cells can help us grapple with Alzheimer’s disease has sparked my interest in a blended neuroscience-regenerative medicine path. However, as someone who has had a lifelong battle with personal and familial cardiac complications, I’m deeply inspired by the possibility of understanding how to utilize stem cells to face the growing number of diverse patients with cardiovascular issues.” 

Sparking Curiosity in CIRM’s Bridges Internship Program 

In his Cell essay, Brown also details his experience joining the CIRM Bridges to Stem Cell Research and Therapy Program, which prepares California’s undergraduate and master’s graduate students for careers in stem cell research and therapy development.

In the program, he studied for one year in Dr. Hollis Cline’s neuroscience lab at The Scripps Research Institute, which he says sparked his curiosity even further.

Image of Kevin Brown Jr. in the lab.

“Surprisingly, I left her office with a fresh slate of objectives and a newfound confidence to allow my imagination to be the precursor for innovation,” Brown writes. “It was at that moment that my goal of blending science and medicine, in hopes of creating change, was finally materializing.”

Brown has been very forthcoming in sharing his story. Last year, he detailed his Bridges experience in a blog for The Stem Cellar and CIRM’s 2023 annual report, in which he highlighted his passion for medicine in neuroscience and the challenges he faced in science communication.


To read the official press release about the Rising Black Scientists Awards, click here. Learn more about Kevin Brown Jr. in a feature from Cal State San Marcos.  

CIRM celebrates landmark 100+ clinical trial awards 

Photo credit: Barbara Ries for UCSF, Jaquell Chandler, and OHSU/Christine Torres Hicks 

The California Institute for Regenerative Medicine (CIRM), California’s Stem Cell Agency and the world’s largest institution dedicated to regenerative medicine reached a milestone when it approved funding for its 100th clinical trial.

At the February Independent Citizens’ Oversight Committee (ICOC) meeting, the board approved five new clinical trials for Parkinson’s disease, autoimmune conditions, cancers, and other conditions, which brought CIRM’s awards from 98 to 103.

“CIRM has proven how much can be achieved if we invest in cutting-edge research. As we celebrate our 100 clinical-trial funding milestone, we want to thank the people of California for their continued trust as we advance the field and deliver on our mission,” said Jon Thomas, PhD, JD, Interim President and CEO of CIRM.

The voters of California believed in this vision when they passed Proposition 71 in 2004 to establish CIRM as the first ever state-funded institute to advance science for the people. This vote of confidence renewed CIRM’s funding through Prop 14, ensuring the Agency’s important work could continue. 

Making an Impact through Investment

CIRM funded its first ever clinical trial in 2011, which was also the first clinical trial to use cells derived from embryonic stem cells. Since then, billions in grants have been deployed towards research projects to address a wide range of health issues, from arthritis to Alzheimer’s disease, cancer, rare diseases in children, heart disease, and stroke. The Agency has funded clinical trials for a variety of conditions that affect millions, including vision loss, Amyotrophic Lateral Sclerosis (ALS), HIV, brain cancers, and COVID-19.

“CIRM has supported the development of NRTX-1001, our human pluripotent stem cell (hPSC)-derived inhibitory interneuron cell therapy for the potential treatment of epilepsy, from discovery through preclinical translation and into clinical development—truly a bench to bedside initiative,” noted Cory R. Nicholas, cofounder, and chief executive officer of Neurona Therapeutics, which is evaluating NRTX-1001 in an ongoing Phase 1/2 clinical trial in adults with drug-resistant temporal lobe epilepsy.

“CIRM’s support of the NRTX-1001 program has been a great example of the fulfillment of its original mission to fund the development of regenerative hPSC-derived cell therapies that can address unmet medical needs and make a transformative, disease-modifying impact on the lives of patients and their families.” 

Neurona clinical trial patient Annette Adkins, after receiving the single dose of NRTX-1001.
Photo Courtesy of OHSU/Christine Torres Hicks

CIRM continues to leave an increasing, indelible mark on the fields of stem cell and gene therapy research, actively integrating Diversity, Equity, and Inclusion (DEI) values into clinical trial applications. All applicants for clinical trial funding at CIRM must submit a DEI plan showing how they will engage and work with underserved populations to include clinical trial participants from these communities. The plans also must outline the applicant’s strategies to foster cultural sensitivity within their teams and partner institutions. The requirement is designed to improve the enrollment of participants from underrepresented racial and ethnic populations in clinical trials.

Beyond funding clinical trials, CIRM continues to push the boundaries of the regenerative medicine field, implementing new programs in manufacturing, data sharing, Community Care Centers of Excellence, and the ReMIND initiative, a vital new program that aims to accelerate discoveries that advance our understanding of Central Nervous System (CNS) disorders.


To learn more about CIRM’s clinical trial research funding, explore this page on our website. For more about diverse representation in clinical trials, see our blog, “Why diversity in clinical trials is essential”.

Neurona Therapeutics Raises $120M Financing, Cell Therapy Shows Positive Outcomes in CIRM-Funded Trial

Photo Courtesy of Neurona Therapeutics/Curtis Myers

Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders, recently announced the successful completion of a $120 million financing.  

Neurona’s financial success is an encouraging sign for the stem cell and gene therapy sector, highlighting partnerships with investors and public organizations like the California Institute for Regenerative Medicine (CIRM). 

CIRM recently awarded Neurona an $8 million clinical project grant to test NRTX-1001—a neural cell therapy derived from human stem cells—for drug-resistant epilepsy. CIRM has supported this research from the initial discovery research stage to the ongoing first-in-human clinical trial. 

NRTX-1001 is being evaluated in an ongoing clinical trial and has potential application in Alzheimer’s disease and other disorders of the nervous system. 

“This financing is a testament to the hard work and dedication of the Neurona team, commitment of our collaborators, and encouraging preliminary data from the first patients in the ongoing clinical trial of NRTX-1001 cell therapy,” said Cory R. Nicholas, PhD, Neurona’s chief executive officer and co-founder. 

The Potential for Suppressing Seizures in a Single Dose

One patient in the CIRM-funded clinical trial is Annette Adkins, who has experienced a remarkable transformation in her life since receiving the single dose of the neuronal cell therapy NRTX-1001.  

In the past, Annette endured frequent seizures, but thanks to this groundbreaking single-dose therapy, she is now hiking again and doing other activities she loves.

Neurona clinical trial patient Annette Adkins, after receiving the single dose of NRTX-1001.
Photo Courtesy of OHSU/Christine Torres Hicks

In a recent announcement, the Neurona research team reported that Annette had undergone the procedure safely and had a greater than 90% reduction in seizure frequency. 

Annette’s story underscores CIRM’s dedication to investing in meaningful research that provides real-world solutions for unmet medical needs.

Seizing Life episode, featuring Annette Adkins, who discusses the impacts of epilepsy on her professional and personal life, and how it led her to participate in the CIRM-funded Neurona clinical trial.

Updates from the Trial

Data from the first cohort of five participants in Neurona’s ongoing clinical trial were shared in December 2023.  

The five participants entered the study with a history of seizure activity that was not controlled by anti-seizure medications. These subjects received a one-time administration of NRTX-1001 as well as temporary immunosuppression to promote the long-term persistence of cell therapy.  

The first two subjects, who experienced 32 and 14 seizures per month, respectively, during the six-month baseline, have continued to report a reduction of more than 95% in overall seizure counts more than one year after taking the dose of NRTX-1001. 

The remaining three subjects were treated with NRTX-1001 more recently and have been followed for three months after the dose. Two of the three subjects demonstrated reduced monthly seizure frequencies of 76% and 87% from baseline levels (26 and 30 seizures per month, respectively) since the first month, with elimination of their more severe focal impaired-awareness seizures.


To read the full release and more updates from the trials, visit the Neurona website.

CIRM Announces Interim CEO

Vito Imbasciani, PhD, MD, and Jonathan Thomas, PhD, JD
Vito Imbasciani, PhD, MD, Board Chair and Jonathan Thomas, PhD, JD, Interim President and CEO

South San Francisco, CA, Dec. 15, 2023 – The Independent Citizens Oversight Committee (ICOC), the governing board of the California Institute for Regenerative Medicine (CIRM), named Jonathan Thomas, PhD, JD as CIRM’s Interim President and CEO at the Dec. 14 meeting. Dr. Thomas previously served as the ICOC Board Chair, a position he held for 12 years before retiring in early 2023.

The ICOC formed a presidential search committee to identity a new CEO and President, following the departure of Dr. Maria Millan in November of this year. Dr. Thomas will serve as Interim CEO throughout the executive search process. 

“We are pleased to confirm JT (Dr. Thomas) as the interim President and CEO for CIRM, an appointment that brings valuable support to the agency’s momentum during the ongoing search for a new permanent President and CEO,” said Vito Imbasciani, PhD, MD, Board Chair of CIRM. “The board is dedicated to recruiting a permanent leader with strong strategic leadership experience who will help the organization in its next phase of growth.”

JT was elected as Chair of the Board in 2011 after he was nominated by then-Governor Jerry Brown, Treasurer Bill Lockyer, and Lieutenant Governor Gavin Newsom. In that time, he led CIRM in granting $2.5 billion to support groundbreaking research to accelerate stem cell treatments to patients with unmet medical needs.

He chaired the agency as CIRM expanded its work with industry, revamped its award processes, prepared for the expiration of bond funding, supported the drafting of Proposition 14, and planned for the next phase of CIRM’s programs after the voters approved $5.5 billion in additional funding.

Before joining CIRM, he was a Co-Founding Partner at Saybrook Capital, an investment banking and private equity firm, where he led an early round of financing for Advanced Cell Technology (now Astellas Pharma). JT’s legal experience includes clerking for White House Counsel Lloyd Cutler in the last year of the Carter Administration and clerking for the Honorable George Mackinnon of the United States Circuit Court of Appeals for the District of Columbia Circuit. JT holds a bachelor’s degree from Yale University, a JD from Yale, and a PhD from Oxford University, where he was a George C. Marshall Scholar.

 “I’m excited and honored to rejoin CIRM’s team and Board in this new capacity. My goal during this interim period is to work with CIRM’s incredible leadership team to continue to deliver on our mission and to provide steady guidance throughout this transitional period,” said Dr. Thomas. 

For more information go to www.cirm.ca.gov