gene therapy

A cardiologist and Professor of Medicine at the University of Pennsylvania Perelman School of Medicine, Kiran Musunuru is a clinician and a researcher whose important work has moved the ball forward on gene therapy. As co-founder and scientific advisor to Verve Therapeutics, Kiran has a special perspective – an insider’s view of the business, from someone who is both an academic (MD, PhD, MPH) and a humanist at heart. No one better to discuss the news that broke last month, with early good reports on treatment for ATTR amyloidosis that uses lipid nanoparticles to deliver a CRISPR package for in vivo editing. Is this the breakthrough moment we’ve been waiting for since the Human Genome Project began? Yes, says Kiran Musunuru, going all-in on the next 5 years.

Kiran Musunuru, Associate Professor at the University of Pennsylvania Perelman School of Medicine, is back on The Beagle to talk about Verve Therapeutics, a company that announced its arrival earlier this month, with the modest goal of protecting the world from heart disease (honestly, “protect the world form heart disease" IS the Verve tagline).

Verve’s ambitions are founded upon work by Kiran, among others, that is directed at using gene therapy to fix – or even prevent – high cholesterol levels associated with increased risk of heart disease, the single most likely cause of death in adults. A small number of people carry a naturally-occurring variant that keeps their cholesterol levels low throughout their lives.

Can we use a CRISPR-based therapy to mimic that effect in people with sky-high lipid levels? And what about the rest of us? Kiran and the co-founders of Verve ask us to imagine a world where gene therapy is used like a vaccine.