Nobel Laureate and CRISPR Co-Inventor Jennifer Doudna Discussed Gene-Editing Breakthroughs at HAS 24

At the Healthcare Analytics Summit™ 2024 (HAS^® 24) this February, Biochemist Jennifer Doudna, PhD, of the University of California, Berkeley, sat down with Melissa Welch, MD, MPH, president of Welch Perspectives Healthcare Consulting, to discuss the ground-breaking discovery of CRISPR technology and its many applications in medical science, agriculture, and the environment.

During her discussion with Welch, Doudna recounted her childhood in Hilo, Hawaii, where the natural world inspired her. As a preteen, her dad, a literature professor at the University of Hawaii, gave her a book entitled The Double Helix: A Personal Account of the Discovery of the Structure of DNA, which sparked a childhood fascination with DNA and genetic materials.

She told a room of nearly 1,000 HAS 24 attendees that her future felt uncertain as she struggled in a first-year chemistry class. However, she had a strong passion for science and chemistry, especially the exploration of the chemical intricacies of life, which fueled her ambition to embark on a career in that field despite battling feelings of self-doubt.

Doudna rose to prominence for co-developing the CRISPR-Cas9 genome engineering technology and earning the 2020 Nobel Prize in Chemistry. She founded the Innovative Genomics Institute (IGI) and is a vocal advocate for responsible CRISPR use.

In addition to the future of this cutting-edge technology, Doudna touched on the merits of genetic editing to address climate change and rare diseases and its utility in preventative medicine.

Below is an edited excerpt of Doudna and Welch’s conversation.

Welch: What is CRISPR? And why is it so important?

Doudna: It came from a bacterial immune system- how bacteria fight viral infection. That’s how we got interested in studying it, and we had no idea in the beginning that it would become a powerful technology. But it works by recognizing DNA sequences and cutting them precisely. So, by understanding the biochemistry and the chemistry of that reaction, it became clear to me, my lab members, and our collaborator, Emmanuelle Charpentier, that this could be used as a powerful tool to induce targeted changes in genome editing, precise alterations to the genetic code in cells that could do things like how to help researchers understand gene function, but also correct genetic mutations like the ones that cause sickle cell disease.

Welch: There are lots of applications of CRISPR in the medical field. Can you talk more about those applications since you made the discovery in 2012?

Doudna: It’s been 12 years since we published our work showing how CRISPR can be used for genome editing. What’s been amazing is that in the first decade of CRISPR, there was extraordinary adoption of the technology, expansion into all areas of biology, and demonstrations that CRISPR can be useful as a clinical application but also for manipulating plants and the organisms that support agriculture in ways that will be very, very impactful going forward.

CRISPR today is being used in the clinic ex vivo, meaning the blood stem cells are removed from the patient, edited in a lab, and transplanted back into the patient with a bone marrow transplant, as in the case of treating sickle cell disease.

What I imagine in the next decade of CRISPR and all the new technologies associated with genome editing is that we are going to see increasing applications in other disease areas and perhaps even for preventive medicine, which I think is something very interesting to think about, especially as we’re here at this conference speaking about innovation and where things are going in the future. Then, in the agricultural space, we are looking at opportunities to use genome editing to make the kinds of targeting changes in plants and microbes that will help us address the rapidly approaching challenges of climate change.

Welch: Let’s talk about some of the medical applications. What is the potential? We’ve heard about sickle cell and thalassemia. Alzheimer’s, perhaps?

Doudna:  We’re in a really exciting moment in time, and I really want to communicate to this audience because many of you will bring this technology to your patients. I envision a day when CRISPR is a standard of care for many diseases. How do we get there? That is a question I ask myself daily.

We started the Institute in the Bay area about seven years ago with this goal in mind: how can we take a very exciting and powerful technology in this whole area of science that was expanding so rapidly and ensure that ultimately everyone can benefit from it – not just a few, not just the rich, but everybody.

We’re seeing an interesting moment in healthcare right now where people are appreciating [the technology], and I think we’re right on the cusp of seeing a big expansion in the use of genome editing. It really can provide a one-and-done cure for people who suffer from a genetic disorder.

At the Institute, we’re working on expanding into other areas, including how to better deliver these genome editing molecules into particular cell types of the body so that editing can be done safely and effectively.

Welch: This question gets more into the medical affordability and accessibility of treatments from genome editing

developments or other aspects where we develop cutting-edge therapies. Is a cure a cure if it’s not accessible? What are your thoughts on that?

Doudna: I’d say, “No, it’s not.” If you can’t get access to it, most patients aren’t benefiting from it, so it’s not that useful. It may be an interesting academic exercise. It may lead to publications. It may even lead to companies and investors making money. But it’s not going to have a widespread impact. And that’s what I want to see us achieve.

Welch: How do you think you, as a biochemist, can play a role in influencing healthcare affordability, accessibility, and the price of drugs?

Doudna: As a biochemist, and for me personally and the work that I’m leading in my laboratory, we’re focused on the technical and scientific advances that need to happen to get us there, such as meeting the delivery challenges, making genome editing molecules as efficient and effective as possible so you don’t need a high dose.

These are all things that are very important to do. But beyond that, we have to be partnering with companies and clinicians. We need to be working with regulatory agencies. And we need to be working with patients and stakeholders, helping them understand what CRISPR is, how it works, how it can impact them, and what the risks and benefits are.

We have a great team at the Innovative Genomics Institute looking deeply at how we will overcome this barrier to access. We’ve prepared reports on this. We’re about to publish in a scientific journal with a roadmap with specific recommendations for what can be done to accelerate the affordability and accessibility of these kinds of treatments.

Welch: When you think about AI, what can we expect of AI in the context of gene editing?

Doudna: In our sphere, I see an extraordinary opportunity to use machine learning and AI to tell us which genes to edit. CRISPR is a great technology, but you must know which genes to edit and the outcomes. That is one area where I think we will increasingly have the kind of patient data and outcome information that can allow us to predict the effects of editing.

We also need to know how genes interact with each other. That’s a big area of biology where we need a better handle and an area where AI will have a big impact. It will also increasingly allow us to analyze patient data, including a comprehensive set of information about the disease, including the whole genome sequences and how a person has responded to a particular therapy. That allows us to couple that information with modifications that could accelerate their ability to benefit from therapies.

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