CIRM News

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

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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

CIRM approves $2.5 million to establish a Patient Support Program 

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Hataalii Tiisyatonii Begay (HT) participated in a CIRM-funded clinical trial at UCSF for Artemis-SCID, a condition that can be life-threatening or fatal. Photo courtesy Barbara Ries / UCSF. 

The California Institute for Regenerative Medicine (CIRM) funds innovative cell and gene therapy research and educational programs to advance regenerative medicine in the Golden State. Just as important is CIRM’s commitment to ensuring that Californians from diverse backgrounds have access to groundbreaking clinical trials for a variety of diseases and condition resulting from the research.  

That’s why CIRM has approved awarding $2.5 million to EVERSANA—a leading provider of global commercial services to the life sciences industry—to establish a Patient Support Program (PSP) to assist patients enrolled in CIRM-funded clinical trials. 

Addressing Barriers to Clinical Trials 

For many patients battling diseases and chronic health conditions, getting access to a clinical trial can be lifesaving, but it can also be very challenging. Clinical trial patients often face financial challenges, long-distance travels, and require family commitments that can make it difficult to maintain participation.  

Through this award, CIRM and EVERSANA will address informational, financial and logistical bottlenecks experienced by clinical trial patients and their family members. The Patient Support Program will be particularly important for providing equal access to California clinical trial participants.  

Evie Junior (left) participated in a CIRM-funded clinical trial at UCLA for sickle cell disease. Photo courtesy UCLA Broad Stem Cell Research Center.  

Evidence shows that support programs positively impact clinical trial accrual rates, patient diversity, trial adherence, humanistic outcomes, reduced healthcare utilization costs, and quality of life. 

Encouraging Trial Participation in Underserved Communities 

Services offered by the PSP will include maintaining a Patient Support Center to refer patients to clinical trials, verifying participation and financial support eligibility, as well as administering Patient Assistance Fund (PAF) reimbursements to cover travel expenses, meals, accommodations, childcare, and other out of pocket expenses. 

Funds for the PSP are set aside under Proposition 14, the voter-approved initiative that renewed CIRM’s funding in 2020.  

Under Prop 14, royalty revenues that CIRM grantees earn from licensing, inventions or technologies are to be spent “offsetting the costs of providing treatments and cures arising from institute-funded research to California patients who have insufficient means to purchase such treatment or cure, including the reimbursement of patient-qualified costs for research participants.” 

Currently, CIRM has been appropriated $15.6 million from the Patient Assistance Fund to support patients. 

“CIRM is committed to supporting patients through the clinical trial process to continue advancing transformative regenerative medicine therapies to the benefit of all Californians,” said Jonathan Thomas, PhD, JD, CIRM’s Interim President and CEO. “The Patient Support Program is just one initiative designed to get us a step closer to that vision.” 

EVERSANA is expected to initiate work on the approved project plan within 120 days of the final contract. 

CIRM celebrates landmark 100+ clinical trial awards 

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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”.

CIRM awards more than $56 million for clinical research aimed at treating Parkinson’s disease, autoimmune conditions and a variety of cancers

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The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, awarded $56 million to fund various clinical research aimed at treating Parkinson’s disease, autoimmune conditions and a variety of cancers.

The awards will support seven projects—five clinical trials and two preclinical projects—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 approved clinical awards include:

Application # Program Title Principal Investigator/InstitutionAmount 
CLIN1-14845Neural stem cell delivered oncolytic viro-immunotherapy for ovarian cancerAboody, Karen – City of Hope$5,314,547
CLIN1-15337Chimeric TGFB Signaling Receptor (CTSR) Enabled Anti-B7H3 CAR T-cell Therapy in Children and Adolescents and Young Adults (AYA) with Recurrent Solid TumorsAsgharzadeh, Shahab – Children’s Hospital Los Angeles$6,000,000
CLIN2-14801Stem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid TumorsKalbasi, Anusha – Stanford$10,211,085
CLIN2-15547Phase 1/2a Dose Escalation Study of Autologous Neuron Replacement in Sporadic Parkinson’s Disease (PD)Wirth, Edward – Aspen Neuroscience$8,000,000
CLIN2-15562Phase 1 Study of Autologous E-SYNC T Cells in Adult Participants with EGFRvIII+ GlioblastomaOkada, Hideho – UCSF$10,927,618
CLIN2-16063A phase 1/2 study to evaluate a bi-specific CD19/CD20-directed CAR T-cell, in refractory lupus nephritis (LN) and systemic lupus erythematosus (SLE)Benjamin, Jonathan – ImmPACT-Bio, Inc.$8,000,000
CLIN2-16303A Phase 1 Study in Participants with Moderate to Severe Active Systemic Lupus ErythematosusHickingbottom, Barbara – Fate Therapeutics$7,934,448

Included in the awards is an $8 million award to Aspen Neuroscience to advance an investigational stem cell-derived dopaminergic neuron replacement therapy for Parkinson’s disease (PD). This individualized potential therapy is being explored in a First in Human Phase 1/2a clinical trial for patients with moderate to advanced PD.

Another award is a $7.9 million award 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.

Another project added to CIRM’s clinical program includes a $5.3 million award to Karen Aboody, MD of City of Hope for late-stage preclinical research to develop a neural stem cell mediated treatment for a chemo-resistant, metastatic ovarian cancer. CIRM has previously supported Aboody and the City of Hope research team with an award for earlier-stage translational research.

To learn more about CIRM-funded clinical trials, click here. To explore more CIRM awards, visit our grants database.

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

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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 invests $26 million in clinical-stage research, including Phase 2b trial for bipolar depression 

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Stock image of a dual tone brain by Getty Images

South San Francisco, CA, Jan. 29, 2024 – The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, awarded almost $26 million to fund various clinical research — from treating bipolar depression to advancing a therapy for spinal cord injury. 

The awards will support four projects 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.  

In addition, CIRM approved a concept proposal for the Community Care Centers of Excellence (CCCE), a new model for increasing clinical trial access to diverse communities throughout California.  

The clinical awards approved at the CIRM January Independent Citizens’ Oversight Committee (ICOC) meeting include: 

Application #  Program Title  Principal Investigator/Institution Amount  
CLIN1-14840 Prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies de Vries, David – Tr1X Inc $4,000,000 
CLIN2-15085 Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: 
SCN2A 		Kim-McManus, Olivia – UCSD $985,713 
CLIN2-15395 A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach Etkin, Amit – Alto Neuroscience $15,000,000 
CLIN1-15450 Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI) Tuszynski, Mark – UCSD $6,000,000 

Improving the Treatment Landscape for Bipolar Depression  

A $15 million award to Alto Neuroscience and Amit Etkin, MD, PhD, President and CEO of Alto Neuroscience will advance a Phase 2b trial to test a novel therapeutic treatment for bipolar depression (I or II), a condition that impacts 4.4% of U.S. adults at some point in their lives. 

The drug is designed to act on neural progenitor cells by enhancing the brain’s ability to create new neurons and improve connections in the hippocampus, a specific region of the brain implicated in bipolar depression. 

Bipolar depression is a severe, life-long psychiatric condition associated with a significant burden of illness and risk of suicide. 

“Given the prevalence of bipolar depression and the high unmet medical needs of those facing this challenging disorder, this potential new therapy could expand treatment options to the hundreds of thousands of Californians impacted by this chronic neuropsychiatric disease,” said Abla Creasey, PhD, Vice President of Therapeutic Development at CIRM.  

Unlike current treatment options which consist of a regimen of antipsychotics and mood-stabilizing medications, this small molecule drug has the potential to offer better tolerability, and includes a diagnostic approach designed to identify patients who are most likely to benefit. 

Using Stem Cells to Restore Function after Spinal Cord Injury  

Another project added to CIRM’s clinical program includes a $6 million award to Mark Tuszynski, MD, PhD, from the University of California San Diego (UCSD) to explore the use of human embryonic stem cells to treat severe spinal cord injury (SCI). 

The proposed therapy works by implanting neural stem cells at the injury site to regenerate and repair damaged axons associated with SCI. 

SCI impacts more than half a million Americans and is associated with significant morbidity, including long-term and severe disability, chronic neuropathic pain as well as high healthcare costs. 

“The potential therapy represents a unique and forward-looking approach in the treatment landscape of severe spinal cord injury and underscores CIRM’s commitment to advancing groundbreaking solutions to transform patients’ lives,” said Dr. Creasey. 

Improving Access to Treatments Across California  

Additionally, CIRM approved a plan for the Community Care Centers of Excellence (CCCEs). The program is projected to award up to $60 million in CCCE awards. 

The California Stem Cell Research, Treatments, and Cures Initiative of 2020 (Proposition 14) mandates the establishment of the CCCE Program to promote access across the state to clinical trials, treatments, and therapies arising from CIRM-funded research. 

The CCCEs are designed to serve as a hub for expanding access to clinical trials, regenerative medicine treatments, and CIRM education and training programs. The program will seek to enlist community-based partnerships to engage populations that can benefit from these treatments. 

A strategic aim is to provide equitable access to communities and populations that would otherwise have more limited opportunities in the absence of these centers. 

Please direct any inquiries to Koren Temple-Perry, Senior Director of Marketing & Communications at press@cirm.ca.gov.

CIRM Joins World AIDS Day Campaign, Commits $104 Million for HIV Research

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John A. Zaia, MD, of Beckman Research Institute of City of Hope

This year marks the 35th commemoration of World AIDS Day, a global event that aims to end human immunodeficiency virus (HIV) related stigma, honor people lost to the disease, and reaffirm the commitment to ensuring HIV is no longer a public health threat.  

This year, the theme of the global event is World AIDS Day 35: Remember and Commit. 

The California Institute for Regenerative Medicine (CIRM) has joined this global awareness event and remains committed to funding research to find therapies and treatments to fight HIV. 

About HIV & AIDS 

HIV is a virus that attacks the body’s immune system. If HIV is not treated, it can lead to acquired immunodeficiency syndrome (AIDS). There is currently no effective cure, though much progress has been made to reduce HIV transmissions and make the virus undetectable in people. 

Globally, an estimated 38 million people live with HIV. More than 35 million people have died of HIV or AIDS-related illnesses over the past 40 years, making it one of the most devastating pandemics in history.  

In California, there are more than 141,000 people living with HIV as of 2021. That same year, there were 4,444 new HIV diagnoses.  

While the rate of new diagnoses is on the decline in the state, the number of people living with HIV continues to grow. It is estimated that Californians living with HIV will face an average lifetime cost of $510,000 to treat their infection. 

Investing in HIV Research and Therapies at CIRM 

CIRM has invested more than $104 million in the search for stem cell and gene therapy treatments for HIV, ranging from discovery research to clinical trials. 

Recent CIRM investments include a study at UC Davis health, in which researchers take a patient’s own white blood cells, also called T cells, and modify them so they can identify and target HIV cells to control the virus without medication. 

CIRM also funded a clinical trial at UCSF to develop a treatment for HIV. In the study, the UCSF team lead by Steven Deeks, MD will modify a patient’s own immune cells to treat HIV.  

The goal of this one-time therapy is to act as a long-term control of HIV so patients no longer need to take antiretroviral therapy (ART), which involves taking a combination of HIV medicines every day. 

Recent HIV Research Awards 

Other recent CIRM awards include an $11.3 million grant to John A. Zaia, MD, of Beckman Research Institute of City of Hope for a clinical trial that will enroll up to 12 healthy people with HIV to be treated in a first-in-human study.   

Zaia’s City of Hope team, in partnership with the University of San Diego, will test a novel approach developed by City of Hope scientists in which a patient’s T cells are engineered to target both the HIV antigen gp120 and a common virus that infects nearly all persons living with HIV called cytomegalovirus.   

The goal is for these CAR-T cells to persist and eliminate HIV-infected cells so that antiviral drugs are no longer needed. 

World AIDS Day Events

CIRM will be co-sponsoring the upcoming World AIDS Day event in Palm Springs, hosted by HIV+ Aging Research Project and RID HIV. The event will be held at the Camelot Theater on December 1st, with a reception to follow.  

To learn more about CIRM-funded research to find treatments for HIV, visit this web page. 

CIRM invests $50 million in clinical research, including trial for vision loss

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The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, today awarded $50.1 million to fund clinical-stage research projects aimed at advancing stem cell and gene therapy treatments for a variety conditions ranging from neurodegenerative diseases and blood cancers to HIV/AIDS. 

The awards will support six projects in the Agency’s clinical program which provides funding for eligible stem cell and gene therapy-based projects through any stage of clinical trial activity.

The awards include:

Application Title Principal Investigator – Institution Amount 
CLIN1-14607 Cancer Stem Cell Interception with Rebecsinib: A First-in-Class ADAR1 Inhibitor Catriona Jamieson – Aspera Biomedicines, Inc. $3,200,000 
CLIN1-14933 Manufacturing of AS-202, an Antisense oligonucleotides  for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis  
 
 		Samuel Alworth – AcuraStem Inc.  $2,199,782 
CLIN2-14068 Treatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant  Ryotaro Nakamura – Beckman Research Institute of City of Hope $9,054,216 
CLIN2-14748 Evaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV John Zaia – Beckman Research Institute of City of Hope $11,299,976 
CLIN2-14787 A Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy Jane Lebkowski – Regenerative Patch Technologies LLC $12,373,748  
CLIN2-15087 Phase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia Elizabeth Budde – Beckman Research Institute of City of Hope $11,983,547 

Among the awards is a $12.4 million grant to support Regenerative Patch Technologies LLC in a Phase 2b clinical trial to evaluate the safety and efficacy of a retinal pigmented epithelial (RPE) implant. The implant will be evaluated in patients with geographic atrophy, a late-stage form of age-related macular degeneration (AMD), a common condition that can lead to vision loss in older adults.  

The RPE is an important cell layer that supports the retina and plays a critical role in maintaining vision. In geographic atrophy, RPE cells break down over time, leading to impaired vision and a loss of independence.  

The stem cell-based implant aims to promote the survival and function of the retina, protecting the eye from disease progression and potentially improving vision. 

“This award supplies critical funding to support a Phase 2b clinical trial to achieve our goal of improving vision in patients with geographic atrophy”, said Jane Lebkowski, PhD, President of Regenerative Patch Technologies. “We want to thank CIRM for their support of this program.”

Geographic atrophy affects more than 8 million people worldwide and an estimated 1 million people in the United States. There are currently no approved therapies that are effective in improving vision in patients with geographic atrophy.  

 “CIRM is proud to continue to fund this groundbreaking stem cell therapy that has the potential to improve outcomes for the millions of people suffering from geographic atrophy,” said Maria T. Millan, M.D., President and CEO of CIRM. “This investment is follow-on funding to CIRM’s previous support to develop this therapy. It reflects our commitment to advancing cutting-edge science and underscores our dedication to addressing the unmet medical needs of those affected by degenerative diseases.” 

This month’s clinical awards include two preclinical projects and four clinical-stage projects. That brings the number of CIRM-funded clinical trials to 95. For more information on CIRM’s clinical stage program, please visit our Funding Opportunities page.

For media requests and inquiries about these awards, please contact press@cirm.ca.gov.

Novartis acquisition validates CIRM’s impact on cystinosis treatment

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Dr. Stephanie Cherqui

The future looks brighter for cystinosis patients and their families.  

As one of 7,000 rare diseases, cystinosis causes an abnormal build-up of the amino acid cystine that can lead to organ failure and premature death. With little to no treatment options, children and young adults primarily affected by the condition often have a poor prognosis – until now.  

Thanks to $17 million in early funding from the California Institute for Regenerative Medicine (CIRM) and the unwavering dedication of researchers like Dr. Stephanie Cherqui at UC San Diego (UCSD), a gene therapy to address the genetic defects of cystinosis is making its way through the field.

Cherqui’s stem cell gene therapy offers a potential breakthrough by modifying a patient’s own blood stem cells with a functional version of the defective CTNS gene to address the underlying cause of the disease.  

Last month, Novartis, a pharmaceutical company with extensive expertise in clinical-stage gene therapy and access to infrastructure, purchased the gene therapy for $87.5 million from AVROBIO. CIRM funded the preclinical work for this study, which involved completing the testing needed to apply to the Food and Drug Administration (FDA) as well as a subsequent clinical trial.  

“CIRM played a significant role in advancing the stem cell gene therapy program for cystinosis from bed-to-bedside. Through its funding and support, CIRM has allowed me to perform the pre-clinical studies required for an investigational new drug application, and then to conduct the first phase of the clinical trial in an academic setting,” said Dr. Cherqui.  

Not only will the Novartis acquisition expedite the global availability of this therapy for people with cystinosis, but it also affirms CIRM’s pivotal role in the development of early-stage research that can turn into tangible treatments. 

“The Novartis acquisition has the potential to accelerate clinical development of novel hematopoietic gene therapy for cystinosis patients. We’re excited for the cystinosis patient community as well as the UCSD, UCLA and AVROBIO teams that led this advancement,” said Dr. Shyam Patel, Senior Director of Business Development and Alliance Management at CIRM. “Furthermore, the Novartis acquisition validates CIRM’s Clinical funding model, which has supported this program since 2016 through preclinical studies as well as through the ongoing first-in-human clinical trial that was coordinated through the UCSD CIRM Alpha Clinic.” 

The impact of this acquisition goes far beyond the walls of research laboratories. For those with cystinosis, it brings a renewed sense of hope and possibility.  

CIRM invests $10 million to build California Cell and Gene Therapy Manufacturing Network

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A laboratory at UCLA’s current good manufacturing practices facility | Photo credit: Elena Zhukova

The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, awarded $10 million to five facilities as part of the first phase to build a California Cell and Gene Therapy Manufacturing Network.

The awards include:

Application # Program Title Principal Investigator/Institution Amount 
INFR5-14663  Laboratory for Cell and Gene Medicine: A partner in the California Cell and Gene Therapy Manufacturing Network Steven Feldman — Stanford University $2,000,000 
INFR5-14739   Enhancing California’s Manufacturing of Leading-Edge Cell & Gene Therapies Dawn Ward — University of California, Los Angeles $2,000,000 
INFR5-14756  Scalable Expansion for Stem Cell-Derived Therapies Taby Ahsan — Beckman Research Institute of City of Hope $1,998,174 
INFR5-14636  A comprehensive biomanufacturing center solving bottlenecks in cell and gene therapy manufacturing to accelerate new therapies for California patients Dhruv Sareen — Cedars-Sinai Medical Center $2,000,000 
INFR5-14719  Open Manufacturing Network for Cell and Gene Therapies Brian Rudolph Shy — University of California, San Francisco $1,999,933 

The statewide network is designed to overcome manufacturing bottlenecks that have delayed or stalled development and approval of regenerative medicines. 

“These awards are a critical first step in formation of the manufacturing network and will help California academic GMP manufacturing facilities raise the quality and competency of cell and gene therapy manufacturing across the state,” says CIRM Senior Director of Business Development & Alliance Management Dr. Shyam Patel. “Plus, these awards will also provide manufacturing career training opportunities for Californians, which align perfectly with CIRM’s mission.”  

Phase one of the awards will fund California non-profit GMP manufacturing facilities for two years. Each facility will support in 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.  

Phase two of the awards will fund collaborative partnerships that will scale efforts from the first phase across the network. 

To learn more about CIRM’s Infrastructure programs, including future funding opportunities, visit our website.