Intellia and Regeneron Release Positive Data on Investigational CRISPR Drug

Intellia Therapeutics and Regeneron Pharmaceuticals have reported positive interim data from their trial of a CRISPR/Cas9-based in vivo genome-editing treatment agent, indicating that the treatment may halt or even reverse disease progression in patients with transthyretin amyloidosis, a progressive condition characterized by the buildup of abnormal deposits of a specific protein in the body’s organs and tissues.

In the trial of 15 patients, the treatment achieved 86 percent and 93 percent mean reduction in serum transthyretin protein at two different dosages, the companies said, adding that the agent was relatively well-tolerated.

These data suggest that treatment with a one-time, systemically delivered CRISPR-based investigational therapy has the potential to substantially reduce levels of a disease-causing protein, said Intellia CEO John Leonard.

Intellia is planning on completing enrollment in the phase 1 trial later this year, which will include patients with transthyretin amyloidosis with cardiomyopathy.