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Clinical trials for IMO, spina bifida and more among those approved by CIRM board

OAKLAND, CA (US), November 2020 – The governing board of the California Institute for Regenerative Medicine (CIRM) has approved several new clinical trials in addition to 10 new discovery research awards. These new awards bring the total number of CIRM-funded clinical trials to 68. 

Among the approved trials is one by Gayatri Rao, M.D., J.D., from Rocket Pharmaceuticals. She received a $3,728,485 award to investigate a gene therapy for infantile malignant osteopetrosis (IMO), a rare and 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 newly corrected blood stem cells will then be introduced back into the child, with the hope of halting or preventing the progression of IMO in young children before much damage can occur. 

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

Diana Farmer, M.D., at UC Davis also received funding ($8,996,474) to conduct a clinical trial of in utero repair of myelomeningocele (MMC), the most severe form of spina bifida. MMC is a birth defect that occurs due to incomplete closure of the developing spinal cord, resulting in neurological damage to the exposed cord. This damage leads to lifelong lower body paralysis, and bladder and bowel dysfunction.

Dr. Farmer and her team will use placenta tissue to generate mesenchymal stem cells (MSCs). The newly generated MSCs will be seeded onto an FDA-approved dural graft and the product will be applied to the spinal cord while the infant is still developing in the womb. The goal of this therapy is to help promote proper spinal cord formation and improve motor function, bladder function, and bowel function. 

The clinical trial builds upon the work of CIRM-funded preclinical research.

David Williams, M.D., at Boston Children’s Hospital was awarded $8,333,581 to conduct a gene therapy clinical trial for sickle cell disease (SCD). This is the second project that is part of an agreement between CIRM and the National Heart, Lung and Blood Institute (NHLBI), part of the National Institutes of Health, to co-fund cell and gene therapy programs under the NHLBI’s “Cure Sickle Cell” Initiative. The goal of this agreement is to markedly accelerate clinical development of cell and gene therapies to cure SCD.

SCD is an inherited disease caused by a single gene mutation resulting in abnormal hemoglobin, which causes red blood cells to “sickle” in shape. Sickling of red blood cells clogs blood vessels and leads to progressive organ damage, pain crises, reduced quality of life and early death. 

The team will take a patient’s own blood stem cells and insert a novel engineered gene to silence abnormal hemoglobin and induce normal fetal hemoglobin expression. The modified blood stem cells will then be reintroduced back into the patient. The goal of this therapy is to aid in the production of normal shaped red blood cells, thereby reducing the severity of the disease.

The board also approved 10 awards that are part of CIRM’s Quest Awards Program (DISC2), which promote promising new technologies that could be translated to enable broad use and improve patient care.

In other CIRM news, California voters recently approved Proposition 14, the California Stem Cell Research, Treatments and Cures Initiative of 2020. The ballot measure provides $5.5 billion in general obligation bond funding to CIRM to support stem cell and regenerative medicine research in California.

Learn more:
https://www.cirm.ca.gov/about-cirm/newsroom/press-releases/11122020/cirm-board-approves-four-new-clinical-trials
https://www.cirm.ca.gov/about-cirm/newsroom/press-releases/11132020/statement-approval-proposition-14