RARE Daily

CIRM Issues $43.8 Million in Grants to Advance Stem Cell and Gene Therapy

October 2, 2023

Rare Daily Staff

The California Institute for Regenerative Medicine awarded $43.8 million to fund projects aimed at advancing stem cell and gene therapy research in California.

The awards will support projects in the Agency’s Clinical, Discovery, and Infrastructure programs. They include 12 projects totaling more than $24.1 million from CIRM’s Quest Awards Program, which promotes the discovery of promising new stem cell-based and gene therapy technologies that could be translated to enable broad use and ultimately improve patient care.

CIRM also awarded $7.7 million to four additional facilities as part of the first phase to build a California Cell and Gene Therapy Manufacturing Network, bringing the total of funded facilities to nine. The statewide network is designed to overcome manufacturing bottlenecks that have delayed or stalled development and approval of regenerative medicines.

Phase one of the awards will fund California non-profit GMP manufacturing facilities for two years. Each facility will support accelerating and de-risking the path to commercialization for cell and gene therapies, advancing industry standards and quality by design, and building a diverse and highly skilled manufacturing workforce in California.

Additionally, CIRM approved a new plan for the ReMIND (Research using Multi-disciplinary, Innovative Approaches in Neuro Diseases) program which will support basic research in neuropsychiatric diseases. The program will award up to an estimated $110 million to fund research studies in the state of California from 2024 to 2028.

Among the projects funded was a $1.4 million awarded to Novoglia to support the development of NGL-101, a cell therapy for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), a rare neurological condition caused by a genetic mutation.

Novoglia will conduct preclinical studies to test the efficacy and safety of a gene corrected stem cell-derived microglia. Microglia are cells of the brain that regulate development, maintenance of neuronal networks, and injury repair. If the study is successful, it may have the potential to improve the health of ALSP patients by opening the door to future therapeutic possibilities. This research may also aid in the development of similar cell replacement strategies for other diseases and conditions affecting the central nervous system.

Photo: Novoglia Co-Founders Sunil Gandhi, Robert Spitale, and Mathew Blurton-Jones

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