The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, has awarded $17.5 million to fund clinical-stage research projects aimed at advancing stem cell and gene therapies for a variety of conditions, ranging from neurodegenerative diseases to blood cancers.  

The awards will support three 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. These awards bring the number of CIRM-funded clinical trials to 96.

The awards include

Application	Program Title	Principal Investigator / Institution	Amount
CLIN1-14945	Pre-Clinical to Clinical Gene Therapy Development for CMT4J	Pirovolakis, Terry – Elpida Therapeutics	$3,930,964
CLIN1-15060	A 1XX-enhanced and fully non-viral BCMA chimeric antigen receptor (CAR) T cell therapy for Relapsed and Refractory Multiple Myeloma	Martin, Thomas – UCSF	$4,585,501
CLIN2-15115	The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele	Farmer, Diana L. – UC Davis	$8,996,477

 This month’s clinical awards include two preclinical projects and one clinical-stage project. Among the awards is a $9 million grant to Diana Farmer, MD, at UC Davis and chief of pediatric surgery at Shriners Children’s Northern California to support the completion of a Phase 2 clinical trial to improve the outcomes for children born with a severe form of spina bifida called myelomeningocele (MMC).  

MMC is a birth defect that occurs when the spine and spinal cord aren’t formed properly during fetal development, usually resulting in lifelong lower body paralysis and bowel and bladder dysfunction.  

The CIRM award will support the advancement of a one-of-a-kind treatment delivered while a fetus is still developing in the mother’s womb via a patch coated with placenta-derived stem cells. The treatment is meant to prevent paralysis and preserve normal bodily function, and ultimately improve the quality of life of these affected children and their families. Learn more about the procedure here.

Image credit: UC Davis Health 

Farmer said the award provides critical support for a Phase 2 trial using stem cells before birth to improve mobility and bowel and bladder control in babies with spina bifida. Farmer is a distinguished professor of surgery at UC Davis and chief of pediatric surgery at Shriners Children’s Northern California. UC Davis Children’s Hospital and Shriners Children’s Northern California run one of the nation’s largest spina bifida programs.

Farmer’s research

CIRM has funded several stages of Farmer’s research, including preclinical studies and a Phase 1 safety trial. The Phase 1 study showed no safety concerns in six patients.

Aijun Wang said the team is grateful for CIRM and Shriners Children’s, which are supporting Phase 2 of the CuRe trial. He said the partnership enables this groundbreaking work and reflects the team’s collaborative approach to solving unmet medical needs.

CIRM is also supporting two preclinical projects: a $4 million grant for a gene therapy for CMT4J, a rare progressive neurological disease, and a $4.6 million grant to develop a CAR‑T cell therapy for multiple myeloma, the second most common blood cancer.

For more details on CIRM’s clinical‑stage program, visit CIRM’s funding opportunities page.