The Nobel Prize 2025: From basic research in immunology to clinical application
By Alexandra Hoegberg
November 3, 2025
This year’s Nobel Prize in Physiology or Medicine was awarded to discoveries in peripheral immune tolerance, celebrating an area of research that has opened the door to numerous clinical applications in autoimmune diseases and beyond.
In October, the Nobel Assembly at Karolinska Institutet announced that this year’s Nobel Prize in Physiology or Medicine has been awarded to Dr. Fred Ramsdell at Sonoma Biotherapeutics, Dr. Mary E. Brunkow at the Institute for Systems Biology, and Dr. Shimon Sakaguchi at Osaka University for their discoveries concerning peripheral immune tolerance – the mechanisms that prevent the immune system from attacking the body’s own tissues.
In Ventures Accelerated’s interview with Fred Ramsdell, he recounts that his initial reaction to winning the Nobel Prize was a mixture of disbelief and amusement. He and his wife Laura O’Neill had been camping deep in the mountains, completely off-grid with no cell service, when the Nobel announcement was made. He was unaware it was even Nobel week. Upon returning to a small town, his wife’s phone exploded with messages. “You won the Nobel Prize!” she told him, to which he incredulously replied: “No.”
When he finally got reception, Ramsdell discovered over 200 text messages and hundreds of emails. The whole experience was surreal: instead of celebrating with champagne and media attention as you might expect of a Nobel laureate, Fred Ramsdell and Laura O’Neill quietly went to an Irish pub where “no one knew or cared,” he said. He found this low-key moment humorous and fitting to his personality: “It was much more how I’d prefer it to be,” he says.
The Nobel Prize for peripheral immune tolerance
While central tolerance, the deletion of self-reactive immune cells in the thymus, was long considered the primary safeguard against autoimmunity, Shimon Sakaguchi made a discovery in 1995 that challenged this view. He identified a previously unknown class of immune cells, known as regulatory T cells (Tregs), that suppress immune responses in peripheral tissues and protect against autoimmune disease.
In 2001, Mary Brunkow and Fred Ramsdell jointly identified the Foxp3 gene as the key regulator of Treg development. They showed that mutations in this gene caused severe autoimmune disease in mice and in humans, establishing its essential role in immune regulation.
Along with Ramsdell’s team, Alexander Rudensky’s team and Shimon Sakaguchi later demonstrated that Foxp3 governs the development of the same Tregs Sakaguchi had discovered earlier, linking the cellular and genetic mechanisms of peripheral tolerance.
When asked what the Nobel Prize means for the research field of peripheral immune tolerance, Fred Ramsdell emphasizes that the recognition shines a spotlight on decades of work that are finally bearing fruit in clinical settings.
He highlights that Sonoma Biotherapeutics, the company he co-founded with Jeffrey Bluestone and others, has just released early phase I clinical data using Tregs in rheumatoid arthritis patients. This represents a full-circle moment: 25 years ago, their team proposed that harnessing Tregs could treat autoimmune diseases, a concept that was purely theoretical at the time. Now, that idea is being tested in real patients.
Although the data so far involve only six patients, Fred Ramsdell views it as a breakthrough milestone showing how foundational discoveries can eventually translate into therapeutic applications. “The timing of the prize is really good,” he remarks, as the field is now moving from theory to tangible impact.
Ramsdell elaborates that the discovery of Tregs has transformed not only autoimmunity research, but also cancer immunotherapy. While Tregs prevent autoimmunity, they can also suppress beneficial immune responses against tumors. Temporarily blocking these cells in cancer settings may enhance treatment responses – a strategy already being explored in both preclinical and clinical contexts.
Moreover, he notes, the immune system plays a role in diseases not traditionally seen as immune-driven, such as ALS and heart disease, where inflammation contributes to pathology. Understanding Treg biology could therefore lead to therapies across an even broader spectrum of conditions. “It really is this central node,” he says, describing how the immune system must balance between attacking threats like COVID-19 and preventing self-destruction.
Clinical applications of immune system research in Sweden
One example of how the Nobel Prize-awarded basic research can be translated into clinical application is Diamyd Medical’s project portfolio. Diamyd is a Swedish biopharmaceutical company focused on precision medicine for autoimmune diabetes. The company is working to restore peripheral immunological tolerance through antigen-specific immunotherapy targeting an autoantigen, GAD65, in type-1 diabetes.
Its lead investigational therapy, Diamyd®, is an antigen-specific immunotherapy designed to preserve endogenous insulin production in individuals with recent-onset type-1 Diabetes. The therapy has received both Orphan Drug Designation and fast-track status from the U.S. FDA. A confirmatory Phase III trial is currently enrolling patients across eight European countries and the United States. An early readout of the Phase III trial is expected in March 2026. The company has two other phase II candidates in the pipeline.
Dr Karin Rosén, a US-based board member of Diamyd Medical, and a seasoned biotechnology senior executive, who has held senior VP and C-suite roles at Horizon Therapeutics (acquired by Amgen), GSK (GlaxoSmithKline), Genentech, and others, explains:
“We give GAD65 to try to reprogram T cells and the immune response. The importance, from a clinical standpoint, is that you then can continue to preserve the function of the beta cells and the body’s own ability to produce insulin. The best insulin is the insulin that you produce yourself, so the longer you can prevent the body from breaking down all the beta cells and allow them to keep producing insulin, the better off you are to regulate blood sugar and prevent the many severe complications from diabetes, including kidney and cardiovascular disease.”
“To be able to treat and prevent autoimmune type-1 diabetesis an example of a clinical indication that demonstrates the significance of the discovery that these three Nobel Prize laureates are involved in. This is a great example of clinical application of that basic research, which is so crucial for developing new effective medicines,” Karin Rosén says.
Aqilion is another Swedish life science company, which focuses on unmet medical needs within chronic inflammation and autoimmune disorders. At its founding in 2018, the company was established with the specific aim to focus on research around T cells.
“It was a strategic decision we made without having a single asset. Instead, we started looking for targets we could imagine: if we reach this drug target, then we can influence the balance between the different T cell groups and we can affect the T cells so that the inflammation is reduced,” Aqilion’s CEO Sarah Fredriksson explains.
“To start a biotech company and think that somewhere in that biology, that’s where we should be. And we had nothing, we had to start from scratch, and it turned out that the timing was good, because new research findings started to emerge and investors also began to look at the area of inflammation and autoimmune diseases again,” she says.
From this decision, the four candidates that are currently in Aqilion’s pipeline emerged. One of them is AhR (AQ312), for the indication ulcerative colitis – a form of inflammatory bowel disease (IBD) where the immune system mistakenly attacks the lining of the colon, causing chronic inflammation and ulceration. There is at present no similar treatment on the market as current treatments are often immunosuppressants, while Aqilion's AhR candidate is immuno-modulating and boosts Tregs to restore the balance of the T cells in the bowel.
Another one of their newer projects is PKCtheta, whose primary indication is IBD. The aim is to inhibit the activity of the kinase PKCTheta that is expressed in T-cells to reduce the vicious cycles of inflammation. PKCTheta is a key kinase involved in T cell receptor (TCR) signaling and Treg function.
Their other two promising candidates are JAK1 (AQ280) – for the indication eosinophilic esophagitis (EoE), a chronic allergic inflammatory condition of the esophagus – which recently reported positive pharmacokinetic results from its phase II study. And a program based on an inhibitor of TAK1, a central target in the innate immune system.
When reflecting on the importance of the research in peripheral immune tolerance that has now been awarded the Nobel Prize, Sarah Fredriksson muses:
“All science focused on T cell–driven inflammation is extremely important. If you look at all the indications involving chronic inflammation and autoimmune diseases, the unmet need is enormous. These diseases cost society a huge amount of money, sometimes people can’t work, their lives are shortened, and they suffer. It’s great that this kind of research gets attention through a Nobel Prize, because then maybe these diseases will also get a bit more recognition,” she says.
Commercializing innovation
Dr. Patricia Beckmann, who formerly worked with Fred Ramsdell at Immunex Corporation – where he was active in the immunology department and she in the biochemistry department – has put applied science front and center in her career.
At Immunex, she worked on how to stimulate the differentiation of cells in the immune system and to inhibit cellular processes that were detrimental and causing disease. She found that her passion lay in identifying novel immune modulators in many different areas and positioning the discoveries to find a clinical use.
“It is not simple and there are many roadblocks and potholes that you need to avoid when considering commercialization. Immunex was selling off many of the things I was inventing – which I found frustrating – and I needed to learn to speak in more of a business manner, discussing markets, opportunities, and so on. So, from research I took a step away to work in law. It was my transition to the business side of science.”
Patricia Beckmann moved into venture capital, working with diverse interests and a public portfolio of biotech stocks and a private portfolio of early-stage companies. These experiences, combined with her decades-long career in life sciences, provided her with insight into how to examine commercial markets for technologies, where to position technologies for best efficacy, and when to pivot.
“It has served me well to work in biotech research and operations, law and venture. Especially as I work with early-stage companies. I can see where they are from a science perspective and where they want to go and help them see either how to get there from a commercialization perspective or how to pivot to a better application,” Patricia Beckmann says.
Interview with Fred Ramsdell, Nobel Laureate in Medicine, featuring Ventures Accelerated representatives Helena Strigård and Olesia Karvetskaia.