This past Thursday, the CIRM Board approved four new clinical trials and ten new discovery awards. These awards bring the total number of CIRM‑funded clinical trials to 68 and allow the agency to surpass its strategic goal of launching 50 new trials.

CIRM awarded $8,970,732 to Dr. Steven Deeks at UCSF to run a clinical trial that modifies a patient’s immune cells to treat—and potentially cure—HIV.

HIV clinical trial

Current HIV treatment relies on long‑term antiretroviral therapy, but many people cannot access or adhere to it.

Dr. Deeks and his team will extract a patient’s T cells and genetically modify them to express two chimeric antigen receptors. These duoCAR‑T cells can recognize and destroy HIV‑infected cells. Researchers then return the modified cells to the patient.

The goal of this one‑time therapy is long‑term HIV control without ART—a functional cure. It may also help the roughly 50 percent of people worldwide who do not respond to current treatments.

Rocket Pharmaceuticals

Rocket Pharmaceuticals awarded Dr. Gayatri Rao $3,728,485 to conduct a clinical trial of a gene therapy for infantile malignant osteopetrosis (IMO), a rare, life-threatening disorder that develops in infancy.  IMO is caused by defective bone cell function, which results in blindness, deafness, bone marrow failure, and death very early in life. 

The trial will use a gene therapy that targets IMO caused by mutations in the TCIRG1 gene.  The team will take a young child’s own blood stem cells and insert a functional version of the TCIRG1 gene.  The team will then introduce the newly corrected blood stem cells back into the child, hoping to halt or prevent IMO progression before much damage occurs. 

Rocket Pharmaceuticals has used the same gene therapy approach to modify blood stem cells in a separate CIRM-funded trial for a rare pediatric disease, with promising results.

Severe form of spina bifida

CIRM awarded $8,996,474 to Dr. Diana Farmer at UC Davis to run a clinical trial testing in utero repair for myelomeningocele, the most severe form of spina bifida. MMC occurs when the developing spinal cord fails to close, leaving it exposed and causing paralysis and bladder and bowel dysfunction.

Dr. Farmer’s team will generate mesenchymal stem cells from placental tissue and seed them onto an FDA‑approved dural graft. Surgeons will place the graft over the exposed spinal cord while the fetus is still developing. The goal is to support proper spinal cord formation and improve motor, bladder, and bowel function.

The trial builds on CIRM‑funded preclinical research.

Sickle cell

CIRM awarded $8,333,581 to Dr. David Williams at Boston Children’s Hospital to run a gene therapy clinical trial for sickle cell disease. This is the second project co‑funded by CIRM and the NHLBI under the Cure Sickle Cell Initiative, which aims to speed development of curative cell and gene therapies.

SCD is caused by a single gene mutation that produces abnormal hemoglobin. Sickled red blood cells block vessels, causing organ damage, pain crises, reduced quality of life, and early death.

The team will modify a patient’s blood stem cells by inserting an engineered gene that silences abnormal hemoglobin and restores fetal hemoglobin. The team then returns the corrected cells to generate normally shaped red blood cells and reduce disease severity.

“Today is a momentous occasion as CIRM reaches 51 clinical trials, surpassing a major goal in our five‑year plan,” said Dr. Maria T. Millan, CIRM President and CEO. “These new trials use innovative regenerative approaches and expand our diverse clinical portfolio.”

The Board also approved ten Quest Awards (DISC2) to advance promising early‑stage technologies that could broaden clinical use and improve patient care.

Awards

APPLICATION	TITLE	INSTITUTION	AWARD AMOUNT
DISC2-12169	Human-induced pluripotent stem cell-derived glial enriched progenitors to treat white matter stroke and vascular dementia.	UCLA	$250,000
DISC2-12170	Development of COVID-19 Antiviral Therapy Using Human iPSC-Derived Lung Organoids	UC San Diego	$250,000
DISC2-12111	Hematopoietic Stem Cell Gene Therapy for X-linked Agammaglobulinemia	UCLA	$250,000
DISC2-12158	Development of a SYF2 antisense oligonucleotide (ASO) treatment for ALS	University of Southern California	$249,997
DISC2-12124	Dual angiogenic and immunomodulating nanotechnology for subcutaneous stem cell derived islet transplantation for the treatment of diabetes	Lundquist Institute	$250,000
DISC2-12105	Human iPSC-derived chimeric antigen receptor-expressing macrophages for cancer treatment	UC San Diego	$250,000
DISC2-12164	Optimization of a human interneuron cell therapy for traumatic brain injury	UC Irvine	$250,000
DISC2-12172	Combating COVID-19 using human PSC-derived NK cells	City of Hope	$249,998
DISC2-12126	The First Orally Delivered Cell Therapy for the Treatment of Inflammatory Bowel Disease	Vitabolus Inc.	$249,000
DISC2-12130	Transplantation of Pluripotent Stem Cell Derived Microglia for the Treatment of Adult-onset Leukoencephalopathy (HDLS/ALSP)	UC Irvine	$249,968