In Future CRISPR Gene Editing To Treat Diseases, Says Intellia Co-Founder: CNBC

Gene-editing technology CRISPR reached a significant milestone this week, completing its first systemic delivery as a medicine to the human body.

What Happened: On Monday, Intellia Therapeutics Inc NTLA and Regeneron Pharmaceuticals Inc REGN reported positive data from CRISPR/Cas9 genome editing candidate in transthyretin (ATTR) amyloidosis. 

Previously, other applications of the CRISPR technology had been limited to ex vivo therapy.

Jennifer Doudna, a co-founder of Intellia, was awarded the 2020 Nobel Prize in chemistry for her work on CRISPR gene editing. She told CNBC the evolution of the technology and highlighted that CRISPR delivery remains a significant challenge.

“This is especially an issue in clinical medicine where being able to edit brain cells, heart cells or muscle cells has incredible potential, but right now, we don’t have the tools to introduce the editors into those cells,” Doudna said. “We have the editors; we just don’t know how to get them where they need to go.”

Sickle cell anemia has been an early focus. Genetic diseases of the eye have also been a focus for CRISPR applications, as Doudna said, “it’s easier certainly to deliver to the eye than to other parts of the body.”

Delivering the edited cells to the liver has also proven to be easier thus far. “A liver is an organ that naturally takes up molecules in the body,” she said.

Why It Matters: Doudna said the next step for innovation around CRISPR would be getting those cells to other parts of the body, such as the brain, the heart, and muscles.

The cost of treatment is a concern. Treating sickle cell disease with CRISPR therapy, Doudna said, costs about $2 million a patient.

While addressing delivery challenges may also help lower costs, Doudna said that the medical field needs to figure out how to “scale the molecule production so that we reduce costs.”

Wednesday, Intellia Therapeutics raised $600 million in a public offering.

Price Action: NTLA shares are up 6.47% at $161.42 during the market session on the last check Wednesday.