News that genomics research has produced a revolutionary cure for sickle cell disease is being hailed around the world. But Fyodor Urnov urges caution when measuring the impact of altering DNA to treat complicated, deadly medical conditions.

“We have a responsibility to pursue CRISPR’s enormous potential to achieve previously impossible solutions to some of the world’s big challenges, solutions that will be available to anyone,” Urnov told international fellows in the National Press Foundation’s Covering Rare Diseases program. As co-director of the Innovative Genomics Institute at the University of California Berkely, Urnov works closely with Jennifer Doudna, the Institute’s Nobel Prize-winning founder who believes that the CRISPR technology she invented must be made available to all who need it regardless of the barriers of geography and income that are persistent challenges toward the goal of health equity.

Urnov explained the basics of CRISPR technology and explored a pathway toward equity in the genomics and biotech realms for National Press Foundation Covering Rare Diseases fellows on Nov. 14, 2023. The following quotes are highlights from his session.

The ABCs of Genomics

“The promise of genome editing is computer-like, word-processor-like control over human DNA. And we represented this a long time ago with this keyboard, where instead of the normal letters of the English alphabet, we have just the four letters of human DNA.

I want to very, very strongly and upfront apologize and acknowledge the egregious bias towards diseases and healthcare narratives that are focused on middle to upper-income countries. And this is a serious failing of healthcare, broadly, of biomedicine, of education.”

Where You Live Shouldn’t Rule You Out

“Nearly the entirety of clinical development of CRISPR is happening in the United States, New Zealand, and Europe. Our central goal is to change that. But I just want to be very clear and upfront to state to you, in the strongest possible terms, that I’m not oblivious to the fact that I’m a professor in California, and the immediate focus of the work is the world around us. But we are very mindful of the fact that we live in a much, much larger world which has needs that our current effort doesn’t address.”

Progress is Strong Yet Measured

“So for the first flavor of disease where you have one gene, one mutation, clear disease, our field has made major progress for sickle cell disease and beta-thalassemia, which together, the two most prevalent genetic diseases on birth that, I will describe to you where we are with that. There are diseases, such as Alzheimer’s, where a specific variant predisposes. So for neurodegenerative disease risk, I think we’re three to five years away from a clinical trial for this. Remarkably, the last category, which is cardiovascular disease, which I explained to you is a complex condition, has recently been the focus of gene editors. And in fact, a clinical trial was just described two days ago where the first 10 subjects were dosed with gene editing for cardiovascular disease. So scientifically and clinically, really good progress.”

Battling Misinformation

“I need to now explain to you how gene editing works. I think it’s important because it’s a technology that has a lot of misunderstanding about it. And the very first thing I want to say, and I want to be abundantly clear as I can, 100% of what I’m going to say has to do with editing existing people with existing diseases, children or adults. Zero percent of what I’m discussing is so-called embryo editing or germline engineering for reproductive purposes. There is an astonishing amount of disinformation about this, and I can summarize for you the truth about this germline editing in one sentence: it has zero ethically justifiable medical use. Zero. And all the people who state otherwise simply don’t know what they’re talking about. In the next decade, human embryo editing for reproductive purposes will have zero impact, zero, on public health. Instead, it’ll just be the topic of a lot of poorly informed public debate.”

Building Capacity Where It’s Needed Most

“To make this sickle medicine, you need a specialized hospital, you need a specialized facility, you need all of this stuff, and that doesn’t really exist, nor does it exist in Africa where the public health burden is enormous. And for beta thalassemia, which is incredibly prevalent in Vietnam, in Thailand, in parts of India, again, the infrastructure doesn’t exist. So just these medicines cannot be delivered there. And last, most of the world’s genetic diseases are not currently the focus of biotech companies or pharma because they just don’t see how they’re going to make a profit on a rare disease. So wrapping up… the central goal of CRISPR, and us in academia and nonprofit, is really build a nonprofit path. And what does that mean? Well, first of all, that means focusing on diseases that are neglected.”

Access the full transcript here.

This training is sponsored by Fondation Ipsen. NPF is solely responsible for the content.