California Institute for Regenerative Medicine
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# CIRM invests $25 million for discovery and preclinical research, Patient Support Program
**FOR IMMEDIATE RELEASE**
Contact:
Koren Temple-Perry
Sr. Director, Marketing & Communications
press@cirm.ca.gov
**South San Francisco, CA, March 29, 2024** – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has awarded to fund numerous projects across the Agency’s discovery, clinical, and infrastructure programs.
The awards will support 11 projects in the Agency’s Foundation Awards (DISC 0) Program, which supports rigorous studies addressing critical basic knowledge gaps in the biology of stem cells and regenerative medicine approaches and to advance stem cell-based tools.
“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.
In addition, CIRM awarded $2.5 million to support the establishment of a Patient Support Program (PSP) to enhance patient access to CIRM-funded clinical trials, an important 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/Institution** | **Amount** |
| --- | --- | --- | --- |
| DISC0-15949 | Neuroimmune interactions in the developing human brain | Nowakowski, Tomasz – UCSF | $1,626,000 |
| DISC0-15737 | Village-based identification of human risk factors for viral neuropathogenesis | Wells, Michael F. – UCLA | $1,577,448 |
| DISC0-15921 | Interrogating Satellite Cell and Myofiber Defects and Repair in Human DMD using Single Nuclei/Single Cell RNA Sequencing of Muscle Resident Cells | Miceli, M. Carrie – UCLA | $1,578,000 |
| DISC0-16039 | 39 Lewy body dementia *α*-synuclein, and cell-specific mechanisms of neurodegeneration | Finkbeiner, Steven M. – Gladstone | $1,739,760 |
| DISC0-16122 | Mapping and modeling endothelial cell fate decisions for pulmonary arterial hypertension | Qiu, Xiaojie – Stanford | $1,540,798 |
| DISC0-15654 | 4 Modeling and understanding alveolar hypoplasia in Down syndrome using iPSCs-derived alveolar type II cells | Al Alam, Denise – Lundquist Institute | $1,524,196 |
| DISC0-15816 | Investigating the SGF29/SAGA complex in regulation of normal and cancer stem cells | Deshpande, Aniruddha – Sanford-Burnham | $1,647,600 |
| DISC0-15774 | Modeling of GATAD2B-associated neurodevelopmental disorder and NuRDopathies: Investigation of cellular & molecular anomalies altering neurodevelopment | Pierson, Tyler Mark – Cedars-Sinai | $1,318,441 |
| DISC0-15972 | Immune cloaking of human stem cell-derived insulin producing cells for curative cell therapy without immunosuppression | Digovich, Katy – Minutia, Inc. | $1,192,586 |
| DISC0-15920 | Harnessing the rejuvenating capacity of pregnancy-associated factors to restore aged stem cell function | Alperin, Marianna – UCSD | $1,539,520 |
| DISC0-15689 | Utilizing Age-Specific Adipocyte Progenitor Cells for Cell Therapy in Older Patients | Wang, Qiong Annabel – City of Hope | $1,508,997 |
## **Advancing Antiviral Research Through Stem Cell Analysis**
A $1.5 million award to Michael Wells, PhD, of UCLA, will support research aiming to identify the genetic and molecular risk factors underlying disease-causing viral infections in multiple diverse ancestral populations.
Through a unique stem cell-based “cell village” platform, researchers will analyze factors underlying differences in viral susceptibility across 150 donors. These cell villages, composed of cells that capture the immense diversity of racial and ethnic groups across California, create 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.
By studying the biological factors that influence differences in immune response to viruses, this research has the potential to advance future antiviral drug development.
“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.
## **Exploring Genetic Defects of Lung Disease in Down Syndrome**
CIRM also awarded $1.5 million to Denise Al Alam, PhD, of the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center to support research that aims to understand lung disease in individuals with Trisomy 21 (also known as Down Syndrome). Although Trisomy 21 impacts multiple organ systems, respiratory complications are a major cause of death in kids and adults with this genetic condition.
This groundbreaking project utilizes patient-derived pluripotent stem cells iPSCs from ethnically diverse backgrounds to model alveolar defects inherent to Trisomy 21. Due to the uniqueness of human Trisomy 21, which cannot be fully replicated in rodent models, this study is poised to generate crucial new Trisomy 21 cell lines to study defects specific to Trisomy 21. Researchers hope to uncover the genes and pathways associated with these defects, paving the way for the development of targeted therapeutic approaches.
In California, about 667 babies are born with Down Syndrome every year, with the highest rate among Latinx infants.
“Respiratory complications are a significant cause of mortality in both children and adults with Down Syndrome. We are thrilled to support this research that aims to deepen our understanding of lung disease in individuals with Down Syndrome. This knowledge holds immense potential to intervene early and improve outcomes for those with this condition,” added Dr. Canet-Avilés.
## **Encouraging Trial Participation in Underserved Communities**
CIRM Board also 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.
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 PSP will be particularly important for providing equal access to California clinical trial participants.
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. 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 Geoff Lomax, CIRM Associate Director of Patient Access. “The Patient Support Program is just one initiative designed to get us a step closer to that vision.”
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**About the California Institute for Regenerative Medicine (CIRM)**
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.
With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.
For more information, go to .
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