BRINGING CRISPR TO THE MASSES

Thanks to intensive research, the promise of CRISPR is clear. Now comes the next daunting challenge: delivering this intricate treatment to the mass market.

The Genome Surgery Initiative — not yet its official name, because the concept is still in development — offers an intriguing pathway. The collaboration between UCSF and UC-Berkeley, with future partners at UC-Davis and Stanford, aims to build assembly line-like operations that bring standardization and industrial efficiency to the new field.

The goal is to create a pipeline of gene therapies, using shared tools, techniques, templates and insights. While initial CRISPR work is being done at separate labs, the goal is to have a central brick-and-mortar facility. Part of CRISPR — the enzyme Cas9, which acts like a scalpel — can be standardized. But the other part — the “guide RNA” molecule, which homes in on the target gene sequence — must be personalized. These components, together with the cell’s existing repair machinery, carry out precise “surgery” to create a genetic change. The Initiative hopes to be for this sort of gene surgery what St. Jude Children’s Research Hospital is for children’s cancer — not just a hospital but also a charity and pioneering research institution with every patient in an FDA-approved clinical trial. While the surgery itself would not earn profits for the universities, the offshoot technologies would.

“We already have a lot of momentum, with all the people in different places and doing different things,” said Dr. Bruce Conklin, scientific director of IGI who coconceived of the Center with IGI’s Scientific Director for Biomedicine Dr. Alex Marson.

“It could be a lot more efficient if it were more coordinated,” he said.