CIRM funds clinical research in heart failure and cystic fibrosis, supports 10 new translational projects
FOR IMMEDIATE RELEASE
Contact:
Koren Temple-Perry
Sr. Director, Marketing & Communications
press@cirm.ca.gov
South San Francisco, CA, May 30, 2024 – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has awarded $53 million to fund various projects from its Clinical and Translation programs aimed at preventing heart failure to the advancement of cell therapy for Parkinson’s disease and the targeting of numerous cancers.
Among the awards are 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.
Stem Cell-Based Gene Correction Therapy for Cystic Fibrosis Sinusitis
One of two preclinical awards this month is a $6 million grant to Dr. Matthew Porteus and his team at Stanford University to develop an innovative stem cell-based gene correction therapy for chronic sinusitis in Cystic Fibrosis (CF) patients.
CF, a common genetic disease, currently has no curative treatment, leading patients to spend their lives managing symptoms. Despite significant advancements in CF therapies over the past two decades, there are limited effective treatment options for serious sinus disease.
The proposed new therapy may potentially provide treatment options to CF patients with mutations unresponsive to current treatments or those who cannot tolerate existing therapies. While sinus disease in CF does not directly affect mortality, it significantly impacts quality of life and may have implications for lower respiratory tract disease. The proposed approach aims to permanently correct CF transmembrane conductance regulator (CFTR) mutations (the protein that helps to maintain the balance of salt and water on many surfaces in the body), offering a potentially transformative improvement over current standards of care with longer-lasting effects.
“CIRM is delighted to support this pioneering gene corrected sinus airway-based stem cell therapy for chronic sinusitis in CF patients. This innovative approach holds the potential of improving CF patients’ quality of life above and beyond current treatments,” said Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM.
“We are pleased to have put together such an outstanding multi-disciplinary team through the Stanford CDCM to bring this stem-cell-based product to people with cystic fibrosis. And we are excited to partner with CIRM in addressing the large unmet medical need for this population. Our experience has been that such partnerships with CIRM have been highly synergistic,” said Dr. Porteus.
Preventing Heart Failure After Attack
CIRM will provide a $6 million award to Arjun Deb, MD, of UCLA to advance the development of a therapeutic humanized monoclonal antibody for the treatment of myocardial infarction (also known as a heart attack) and subsequent heart failure.
This therapy targets ENPP1, a protein that exacerbates heart damage through inflammation and fibrosis after a cardiovascular event. By inhibiting ENPP1, this one-time treatment aims to curtail inflammation, reduce fibrosis, and prevent the development of heart failure after a heart attack.
In the United States alone, close to 7 million people live with heart failure, and the survival rate post-diagnosis is only about 50 percent over five years. Cardiovascular disease remains a leading cause of death in California and accounts for nearly one-third of all deaths.
“We are very grateful for Agencies such as CIRM who have supported this project from its very beginning,” said Dr. Deb. “We hope to deliver this new therapeutic agent to the millions of people with heart failure in California and beyond.”
The need for a therapy that halts the progression to heart failure after a heart attack is significant, as heart attacks contribute between 40 and 70 percent of all heart failure in the Western world.
“The prevention of heart failure following a heart attack is a high unmet medical need. It is intriguing to know that heart failure from heart attacks could potentially be prevented by a one time-treatment with this drug” added Dr. Creasey.
Dedicating $41 million to Advance Translational Stage Research
CIRM also awarded over $41 million to support 10 projects in its Translational program.
The goal of CIRM’s Translational program is to support promising stem cell-based or gene projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use. Those 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:
| Application # | Program Title | Principal Investigator/Institution | Amount |
| TRAN1-16050 | Combating Ovarian Cancer with Stem Cell-Engineered Off-The-Shelf CAR-NKT Cell Therapy | Yang, Lili – UCLA | $6,312,000 |
| TRAN1-16225 | Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug-resistant focal epilepsy | Nicholas, Cory R. – Neurona Therapeutics | $3,828,714 |
| TRAN1-16011 | Development of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND | Lipshutz, Gerald – UCLA | $5,145,825 |
| TRAN1-16026 | Toward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells | Gomez-Ospina, Natalia – Stanford | $4,997,237 |
| TRAN2-16061 | A Rapid Clinical Digital Patient-derived Organoid to Guide Breast Cancer Treatment Decision | Yuan, Yuan – Cedars-Sinai | $1,535,375 |
| TRAN1-16023 | Hypoimmunogenic iPSC-derived TCR-NK cells for oncology | Conway, Anthony – Replay Holdings, Inc. | $4,107,571 |
| TRAN1-16012 | A high-quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation | Joshi, Abhay – Axent Biosciences Inc. | $3,999,241 |
| TRAN1-16013 | Development of an UNC13A Targeting Antisense Oligonucleotide (ASO) Treatment for ALS, for IND-enabling Studies | Alworth, Samuel V. – AcuraStem Inc. | $4,012,325 |
| TRAN1-16192 | Targeting pancreatic cancer with Allogeneic Off-the-Shelf PSCA-CAR NK cells | Tian, Lei – Beckman Research Institute of City of Hope | $6,036,000 |
| TRAN4-16091 | Purification of Human Hematopoietic Stem Cells (HSCs) for Clinical Stem Cell Transplantation | Weissman, Irving L. – Stanford | $1,499,683 |
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.