Dr Douglas Mann on Next Steps to Studying Gene Editing in Heart Failure

Initial data on NTLA-2001, which is a novel investigative intravenous agent that works to prevent development of cardiac transthyretin (ATTR) amyloidosis by targeting the TTR gene and TTR protein levels, were presented at this year’s American Heart Association’s Scientific Sessions in Chicago.

In an interview with The American Journal of Managed Care® at this year’s American Heart Association’s Scientific Sessions in Chicago, Douglas L. Mann, MD, professor of medicine at Washington University School of Medicine in St. Louis and editor-in-chief of JACC: Basic to Translational Science, discussed CRISPR/Cas9 editing findings following administration of NTLA-2001 among patients who have transthyretin amyloidosis with cardiomyopathy—a condition that can often lead to heart failure.


Does the presence of COVID-19 make the need for this therapy more urgent?

What we found with COVID-19, if you have underlying heart disease, COVID makes it worse. So I think for people who do have far advanced disease, such as New York Heart class III and IV heart failure patients, if they got COVID, it would complicate the disease. Perhaps those people would be on ventilators more or have longer hospitalizations, or even have adverse outcomes such as death. I think COVID just takes everything that is bad with heart disease and makes it worse. So I would predict for the amyloid population—as we know in the heart failure population—it makes outcomes worse.

What are the next steps to study this therapy?

I think that the next steps would be to do a properly powered designed clinical trial, a phase 3 trial, to test it in more patients to look for the adverse effects, but also to try to achieve hard end points such as cardiovascular death reduction and hospitalization reduction. I would be stunned if they're not planning that trial now based on the successful results of their early phase 1 studies. I think it's a really encouraging therapy, and the nice part is is that you get sustained reduction of levels with 1 injection every 6 months. So I think from the patient standpoint, it's going to be really a nice therapy.

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