Jennifer Doudna: A Multi-Billion Dollar Market For CRISPR Therapies Is Possible

Forbes

29-05-2025

In a recent conversation with Nobel Prize winning CRISPR pioneer Jennifer Doudna, we explored the transformative potential of gene editing for medicine, agriculture, and society.
The power of CRISPR cannot be overstated. The first CRISPR therapy, approved by the Food and Drug Administration at the end of 2023, can effectively cure a disease that was once untreatable.
'Having spoken with Victoria Gray, who was the first US patient in the first sickle cell trial with the CRISPR therapy, I can tell you it's transformative for patients. It completely changes the trajectory of their lives in a positive way, which is amazing,' Doudna told me.
And earlier this month, news broke of a baby born with a rare metabolic disorder who became the world's first patient to receive a personalized CRISPR therapy. Incredibly, the therapy was developed in only six months, the result of a collaboration that included scientists from Doudna's Innovative Genomics Institute. Today, the baby has safely received three doses of the therapy and is reportedly thriving.
That said, the first approved CRISPR medicine has been plagued by a slow rollout. By the end of last year, only a handful of patients had received it, due to the complexities of accrediting centers to offer the drug, manufacturing and administering the bespoke therapy, and screening and prepping patients, which requires them to endure chemotherapy. These issues have led some observers to question the technology's commercial future.
'Can gene editing deliver on its promise?' read one recent headline. For Doudna, the answer is yes – as long as certain hurdles are overcome.
First, the good news.
Teams of scientists in the U.S., Europe, and China are making real strides toward effective genetic medicines. Among the highlights is Cambridge, Mass-based Intellia Therapeutics, which Doudna co-founded, and which is running a phase III trial for liver disease using CRISPR.
'The remarkable thing there is that they were the first team to show that you could get effective in vivo editing with a one-time injection of a CRISPR therapy, which is a real milestone in the field' Doudna said. 'I see that as definitely an important future direction for probably most CRISPR therapies going forward.'
And just reported this month, early data from CRISPR Therapeutics demonstrated in a small trial that its gene editing therapy reduces bad cholesterol and triglycerides by as much as 80 percent.
The initial CRISPR research in medicine began in rare diseases, like sickle cell anemia, in which a single gene mutation is responsible for causing human illness, and therefore, is an ideal target for a tool like gene editing. But many groups are working to develop such therapies to address common diseases, like heart disease, as a form of preventative medicine.
The largest technological barrier today, Doudna said, is not with CRISPR itself, but with the delivery of the CRISPR enzymes into the right molecules and tissues in the patient – or animal or plant, depending on the application.
'What's great is many people are awake to this problem,' Doudna said. 'They realize that this is gating for cell and gene therapies, and they're working on it. So what I'm seeing now is a lot of clever ideas out there, a lot of interesting advances that are being made in academic groups primarily. And I think companies are trying to quickly capitalize on that, which they should. So I'm pretty bullish that, again, it's not going to happen overnight, but I think over the next few years, we will see increasingly the ability to do targeted in vivo editing in ways that will be impactful.'
But that's not the only barrier that must be solved for CRISPR to reach its full potential.
Doudna hopes to see rare diseases treated as groups, with CRISPR as a programmable therapy that can be tailored to different mutations. There are more than 7,000 rare genetic diseases that mostly affect children and lack treatment options, but such small markets mean that biotechs and pharma companies are often reluctant to invest in developing therapies.
A platform-based approach to bringing CRISPR medicines to the market could lessen the costs and provide an accelerated path to market – if regulators embrace it.
Doudna's team at the Innovative Genomics Institute is pushing hard to accomplish exactly that.
'I hope that by really working on the regulatory landscape to get platform designation for CRISPR, so that you could streamline clinical testing, I think that would be certainly helpful, because we have to figure out how to open up the market for these types of therapies,' Doudna said.
Today, the market caps for gene editing companies are far too low, reflecting a need to win back investor confidence. But once a large market opportunity emerges that solves many patients' needs, the attitude of investors should rebound.
Doudna likened the current market sentiment toward CRISPR to a common cycle with biotech breakthroughs: At first, there is a great deal of excitement, creating hype. Investments flood the space. Then reality sets in; new science is hard, and people realize it's going to take more time to develop, leading to a pullback.
'I think that's where we are right now with gene editing and maybe cell therapies in general, and then at some point it's going to swing back,' Doudna said. 'The question is when.'
A similar cycle plagued the development of another emerging technology, RNAi, but the leading company, Alnylam, eventually developed a robust pipeline and a blockbuster drug for a serious disease of the heart muscle.
So where might the first CRISPR blockbuster drug come from?
Doudna speculates it might be an in vivo T-cell therapy. Multiple teams are working in this area with the aim of creating therapies for cancer and autoimmune diseases.
'I think that is going to happen,' Doudna said. 'It's just a question of when because the data look great.' The companies 'won't all be successful, but I think some of them will. And when they do, then you're going to see that multi-billion dollar market.'
Human medicine is not the only arena where gene editing will be transformative. Doudna is bullish on CRISPR in agriculture, an industry where she believes we will see the biggest global impact from gene editing in the near-term.
At the Innovative Genomics Institute, Doudna's team is working on gene editing the metabolism of cows to reduce their methane emissions. It's a project that would also help farmers by enhancing milk and meat production. The team is also working on making rice less dependent on water, which is important in regions struggling with droughts.
'We're just at the beginning of what's going to be incredibly exciting over the next five to 10 years in agriculture, where increasingly plant breeders are going to be able to use CRISPR to make five or 10 changes at once in plants that will bring about the kinds of phenotypic changes that are valuable, commercially important globally, and also much easier than if you were to try to do that with traditional plant breeding,' Doudna said.
It's essential to note that a gene-edited crop is not a GMO, which involves adding the genetic material of one species into another to confer an advantageous trait. A gene-edited crop, by contrast, has very precise changes made to its own genome to confer an advantage, rather than the insertion of any foreign material. Such changes could theoretically occur via traditional cross breeding but would take decades longer. That's why the U.S. Department of Agriculture has ruled that edits in plants that could have occurred naturally can be classified as a non-regulated product, unlike GMOs.
'I do think it's very important, again, to work with regulators and to ensure that regulation is really based on science and safety, but you also don't want to disallow things that are completely safe, and that give us an important advantage as we move forward in the world,' Doudna said.
Finally, Doudna stressed the importance of curiosity-driven science and international collaborations in discovering breakthroughs and translating them into commercial products that improve our lives.
CRISPR came out of an international partnership with Doudna's fellow Nobel Prize winner, the French microbiologist Emmanuel Charpentier. Other government-funded researchers in Spain and around Europe contributed knowledge that converged in the eventual development of CRISPR.
'People say, 'What are you doing to cure cancer, solve heart disease, cure Alzheimer's?'' Doudna told me. 'Those kinds of targeted projects are important, but they can't be done in isolation…. Innovation comes a lot of times from just studying things that initially don't have a connection to what you want to do, but in the end, you find out they are connected. And that's absolutely the story of CRISPR.'
That remarkable story is still just getting started.
Thank you to Kira Peikoff for additional research and reporting on this article.