RARE Daily

CIRM Issues $17.5 Million in Clinical Stage Research Funding

December 7, 2023

Rare Daily Staff

The California Institute for Regenerative Medicine has awarded $17.5 million to fund clinical-stage research projects aimed at advancing stem cell and gene therapy treatments 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. To date, CIRM has funded 96 clinical trials.

The awards include a $3.9 million award to Terry Pirovolakis of Elpida Therapeutics to support pre-clinical to clinical gene therapy development for Charcot-Marie-Tooth disease type 4J, a rare, debilitating, progressive hereditary motor and sensory neurological disease; a $4.6 million grant to Thomas Martin of the University of California, San Francisco to support development of an enhanced and fully non-viral BCMA CAR-T cell therapy for relapsed and refractory multiple myeloma; and  an $8.9 million award to Diana Farmer of the University of California, Davis to support a clinical trial of a cellular therapy for in utero repair of myelomeningocele, a severe form of spina bifida.

Myelomeningocele is a birth defect that occurs when the spine and spinal cord aren’t formed properly during fetal development, usually resulting in life-long 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 quality of life of these affected children and their families.

“This award supplies critical funding to support a phase 2 clinical trial to achieve our goal of using stem cells before birth to improve the ability to walk and have bowel and bladder control in patients born with spina bifida,” said Farmer, distinguished professor of surgery at UC Davis, chief of pediatric surgery at Shriners Children’s Northern California and principal investigator for the project, also known as the CuRe Trial. UC Davis Children’s Hospital and Shriners Children’s Northern California have one of the largest and most well-known spina bifida programs in the nation.

 

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