Here you can read a selection of notable comments about the landmark paper on in vivo genome-editing published in the New England Journal of Medicine on 26 June. The trial, conducted in the UK and New Zealand, produced the first-ever clinical data supporting the safety and efficacy of intravenous infusion of a single-dose CRISPR treatment. The treatment, developed by two US-based companies (Intellia Therapeutics and Regeneron Pharmaceuticals) targets a rare and fatal condition called transthyretin amyloidosis.
Jennifer Doudna (CRISPR co-inventor and co-founder of Intellia): “It’s a critical first step in being able to inactivate, repair, or replace any gene that causes disease, anywhere in the body” (source Science).
Jennifer Doudna, again: “This is a major milestone for patients. While these are early data, they show us that we can overcome one of the biggest challenges with applying CRISPR clinically so far, which is being able to deliver it systemically and get it to the right place” (source NPR)
Fyodor Urnov (UC Berkeley): “The joy of CRISPR-Cas9 is moving from editing gene 1 to editing gene 2 is simple. Intellia is putting that to smart use in advancing its clinical efforts from one disease (TTR) to the next one (HAE= hereditary angioedema). Keep everything the same, change 20 nucleotides in the guide RNA! (source Twitter)
Kenneth Chien (Karolinska Institute, co-founder of Moderna, which makes one of the mRNA-based COVID-19 vaccines): The new treatment, which includes an mRNA encoding one of CRISPR’s two components, “begins the convergence of the fields of CRISPR and mRNA” (source Science)
David Liu (Harvard University and the Broad Institute): “These impressive results from Gilmore et al. show that gene editing technologies, mRNA engineering & manufacturing, and in vivo delivery can already come together into one-time treatments that offer new hope for patients suffering from genetic diseases” (source Twitter)
Julian Gillmore (University College London, Royal Free Hospital, first author of the NEJM paper): “It really is exciting. This has the potential to completely revolutionize the outcome for these patients who have lived with this disease in their family for many generations. It’s decimated some families that I’ve been looking after. So this is amazing” (source NPR)
Patrick Doherty (patient-volunteer in the trial): “I definitely feel better. I’m speaking to you from upstairs in our house. I climbed stairs to get up here. I would have been feeling breathless. I’m thrilled” (source NPR)
John Leonard (Intellia President and chief executive officer): “The interim results support our belief that the treatment has the potential to halt and reverse the devastating complications of ATTR amyloidosis with a single dose. Solving the challenge of targeted delivery of CRISPR/Cas9 to the liver also unlocks the door to treating a wide array of other genetic diseases with our modular platform, and we intend to move quickly to advance and expand our pipeline. With these data, we believe we are truly opening a new era of medicine” (source press release)