Intellia’s first CRISPR gene editing therapy shows promise in small study

“Intellia Therapeutics has moved one step closer to its ambition of creating one-and-done gene-editing treatments for people with genetic diseases. Last August, the Cambridge company and its partner, Regeneron Pharmaceuticals, published a landmark study showing that even low doses of their one-time treatment based on CRISPR-Cas9 technology could dramatically reduce levels of a disease-causing protein made in the liver of people with a rare condition called transthyretin amyloidosis.

“The takeaway on that day was this stuff can work,” said Intellia chief executive John Leonard. But questions remained, including how consistent the treatment is from person to person, and how long its effect lasts, he added. Since August, Intellia has expanded its small clinical trial from 6 to 15 people and tested a total of four doses of its treatment. New data released Monday suggest the highest dose of the treatment worked even better than the initial doses tested last year, and that the treatments were durable. “Thus far, we have not seen any loss of effect,” Leonard said.

“Dr. Vaishali Sanchorawala, director of the Amyloidosis Center at Boston University School of Medicine and Boston Medical Center, said that longer-term follow up of the patients will be needed to see if the treatment can eventually reverse organ damage caused by the disease, but she is encouraged by the results so far. ”CRISPR gene editing for transthyretin amyloidosis is going to be the revolutionary treatment for this rare disease,” she said. “This is truly an epic moment.”

View Full Article By Ryan Cross of the Boston Globe, Updated February 28, 2022, 3:59 p.m.