California is Building a New Path to Rare Disease Cures; First Target is Neuro Afflictions

Fresh details unveiled last week by CIRM

WEST HOLLYWOOD, Ca. -- California’s $12 billion stem cell and gene therapy agency revealed more details last week about its new, embryonic plan to create a scientific platform to streamline the development of therapies that will cut across a variety of rare diseases. 

The proposal emerged from a year-long re-evaluation of the priorities of the California Institute for Regenerative Medicine (CIRM) aimed at maximizing its impact with state voters who created CIRM through a ballot initiative. They will also be the ones who decide whether the agency is refunded in a few years.  

“We're going to streamline developing rare diseases -- not one grant at a time, not one disease at a time,” said Abla Creasey, CIRM’s executive strategy officer for rare diseases. The agency is hoping to create “a conveyor belt type system that will allow us to get 10 and 20 and 100 different rare diseases moving towards approval simultaneously,” she said at a CIRM/Cedars Sinai gathering here.