Crispr Gene-Editing Drugs Show Promise In Preliminary Study

Intellia Therapeutics reported encouraging early-stage study results for its Crispr gene-editing treatments, the latest sign that the pathbreaking technology could result in commercially available drugs in the coming years. The Wall Street Journal reports: Intellia said Friday that one of its treatments, code-named NTLA-2002, significantly reduced levels of a protein that causes periodic attacks of swelling in six patients with a rare genetic disease called hereditary angioedema, or HAE. In a separate study building on previously released trial data, Intellia's treatment NTLA-2001 reduced a disease-causing protein by more than 90% in 12 people with transthyretin-mediated amyloidosis cardiomyopathy, or ATTR-CM, a genetic disease that can lead to heart failure. Despite the positive results, questions remain about whether therapies based on Crispr will work safely and effectively, analysts said. Intellia's latest studies involved a small number of patients, and were disclosed in news releases and haven't been published in a peer-reviewed journal. The NTLA-2002 study results were presented at the Bradykinin Symposium in Berlin, a medical meeting focused on angioedema. The data came from small, so-called Phase 1 studies conducted in New Zealand and the U.K. that didn't include control groups. Results from such early studies can be unreliable predictors of a drug's safety and effectiveness once the compound is tested in larger numbers of patients. The findings, nevertheless, add to preliminary but promising evidence of the potential for drugs based on the gene-editing technology. Last year, Intellia said that NTLA-2001 reduced the disease-causing protein involved in ATTR patients.

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