Rare Diseases Receive Preliminary Research Boost From California's Gene Therapy/Stem Cell Agency

$3.9 billion worth of new priorities under review, only one more public session before full CIRM board action

The CEO of the FoxG1 Foundation told CIRM last week that it is ‘brutally hard” to raise money for rare disease research. CIRM is “perhaps the only avenue” for realistic funding, Nasha Fitter said. She tells a personal story about her daughter in the video above.

California’s $12 billion stem cell and gene therapy agency moved forward last week with a plan to tackle rare diseases via a “platform” research approach aimed at helping to commercialize revolutionary therapies and ease their astronomical costs. 

The effort is part of a sweeping re-evaluation of the priorities of the California Institute for Regenerative Medicine (CIRM) as it determines how to spend its last $3.9 billion. Under review not only are rare diseases but such things as stroke, which affects 892,000 persons in California and has a $65.1 billion economic cost, and heart ailments, which affect 1.4 million persons in the state and cost $68 billion. 

Abla Creasey, CIRM vice president for therapeutics development, outlined the platform approach for a key group of CIRM board members during a nearly two-hour meeting Friday. She said it would be a pilot project targeting conditions that were caused by a single gene.