A symposium at ChristianaCare this week focused on the latest gene editing breakthroughs and the challenges of getting these new technologies to market.
Gene editing with CRISPR has been hailed as a potential game-changing treatment for conditions like cystic fibrosis, sickle cell disease and cancer. At least two patients in the U.S. have received treatment with the technology, and one Chinese doctor shocked the world last year when he used it to create genetically modified twins.
ChristianaCare researchers recently made another advancement in the gene editing world when they discovered a unique site in the DNA of tumor cells different from DNA in other cells. They say the discovery could ensure CRISPR is used to only attack cancerous cells without harming neighboring healthy ones.
Dr. Eric Kmiec is Director of the Gene Editing Institute at ChristianaCare’s Helen F. Graham Cancer Center.
He says this discovery distinguishes ChristianaCare in a growing field of institutions seeking CRISPR patents.
“This discovery seems to put us, at least, on our own path,” said Kmiec. “Our feedback from the FDA has been that that particular observation is what they have become very excited about.”
But getting new CRISPR technologies to market is complicated and time consuming with legal and clinical challenges. Experts say there are more than 5,700 CRISPR patents currently up for review and only four therapies have been approved by the FDA.
UC Berkeley is currently in a patent dispute over the use of CRISPR/cas-9 in eukaryotic cells.
Dr. Samantha Maragh is Leader of the Genome Editing Program at the National Institute of Standards and Technology. She was also a panelist at Thursday’s symposium.
She says, with the rapid advance of CRISPR onto the scene, regulatory agencies don’t even know which questions to ask about possible unintended consequences of this new technology that edits human genetic makeup.
“There aren’t any norms right now to say, ‘what is the best way to look for the change you want; what is the best way to look for any changes that you didn’t want?’” said Maragh.
It’s also not clear how the new products will be best used to reach the populations who may need them most.
Kmiec says ethnic diversity is an important consideration as studies continue on the new gene technologies. He points out the underlying genetic sequence among different races and ethnicities is often different, and the vast amount of DNA in the research database is from people of European descent.
“But if we’re going to develop universal therapies, where breakthrough therapies reach minority communities, which is our major objective here, we’re going to have input from minority groups to be able to build those databases so we can devise the tools to actually make them work in a universal way—the whole population,” said Kmiec.
Kmiec says ChristianaCare is testing the new technology on animals and will soon file a clinical trial application with the FDA.
