The 2025 World Medical Innovation Forum (WMIF) brought together leaders from across medicine, biotech, government, research, investors and other industries, to discuss a wide range of topics at the cutting-edge of healthcare. If you missed it, not to worry – there's plenty of event news and highlights to catch up on.
One particular panel that drew interest was on a topic on a new modality in medicine: the use of gene editing technologies to treat disease. The panel, called “Gene Editing: Precision Medicine in Practice” was moderated by Benjamin Kleinstiver, PhD, an investigator in the Center for Genomic Medicine at Mass General Brigham and the Kayden-Lambert MGH Research Scholar. Other panelists included:
- Omar Abudayyeh, PhD, Director of Gene Editing, Gene and Cell Therapy Institute, Mass General Brigham; Assistant Professor of Medicine
- Steve Favaloro, Chairman & CEO, Genezen
- David Liu, PhD, Richard Merkin Professor; Director, Merkin Institute of Transformative Technologies in Healthcare, Broad Institute
- Trevor Martin, PhD, Co-founder & CEO, Mammoth Biosciences
- Laura Sepp-Lorenzino, PhD, Senior Advisor, Former CSO, Intellia Therapeutics
You can watch the full panel here.
Three patients. Three devastating diseases. One technology that helped rewrite their stories.
At the 2025 World Medical Innovation Forum, six gene editing experts shared examples of the power of these innovative therapies and the challenges that remain in scaling up, accelerating progress, and applying this technology to more common diseases.
“It would be an understatement to say that it’s been a phenomenal year for the gene editing field,” the panel’s moderator Ben Kleinstiver, PhD, an investigator at Mass General Brigham, told the audience. “There’s been tremendous successes both in academia and industry in terms of developing gene editing drugs for patients.”
Kleinstiver and David Liu, PhD, of the Broad Institute of MIT and Harvard, showed photos of three patients who are among the first treated with gene editing therapies and shared their stories:
Alyssa Tapley
In May of 2021, 13-year-old Alyssa Tapley was diagnosed with an aggressive form of T-cell acute lymphoblastic leukemia. When standard treatments failed, she enrolled in a clinical trial of a base-editing technology that used engineered T-cells from a donor to attack her cancer cells. The treatment was successful, and she remains in remission.
Baby KJ
When KJ was born in August 2024, he was diagnosed with a severe metabolic disease. Just seven months later, KJ was treated with a bespoke base editing medicine and, today, he’s showing signs of tremendous benefit. KJ can now eat a roughly normal protein diet and has gone home with his family.
Tracy Atteberry
Tracy Atteberry, who has a rare, inherited immune system disorder, received a gene-editing treatment known as prime editing in May 2025. The editing was performed on his own hematopoietic stem cells to correct a mutation, and the cells were then infused back into his body.
“These are three out of hundreds of patients who have been treated with gene editing in dozens of clinical trials,” said Kleinstiver.
Successes and Setbacks
Despite this promise of gene editing and three successes detailed above, there are plenty of challenges in the field with companies contracting their development pipelines and focusing on very specific programs.
In September 2025, just days after the World Medical Innovation Forum, in fact, The New York Times reported that the treatment that Atteberry received is no longer being offered by the company that made it.
Trevor Martin, Mammoth Biosciences CEO, and a panelist, acknowledged the paradox the field is facing as scientific progress meets economic pressures.
“It's never been more exciting in terms of the results we're seeing in gene editing,” said Martin. "But if I'm being honest, it's never been more depressing in terms of the level of investment and excitement around it from an economic side."
Breaking the Bottleneck
Omar Abudayyeh, PhD, director of gene editing at the Mass General Brigham Gene and Cell Therapy Institute, sees both the challenges that face the field, but also the opportunities for broader gene-editing treatments if academia and industry can work together.
“It’s been an incredible year, especially seeing baby KJ get treated,” said Abudayyeh, who works on developing new gene editing approaches that may to scale to a wider range of patients.
“There’s a long tail of mutations and thousands of potential pathogenic variants. The idea of treating all of them is daunting. But the idea that you can go from concept to deploying a therapy in a person within six months or so is incredible,” he said. “Thinking through how to reproduce that at scale is an exciting prospect.”
David Liu, PhD, runs a lab that has pioneered the development of many technologies including the base editing and prime editing approaches that underlie the treatments that Alyssa, KJ, and Atteberry received.
“The capabilities of the field have never been more rich in opportunities, and the field has never felt more obligated to connect the science with the people,” he said.
On the Shoulders of Giants
Delivering genetic therapies to patients in need requires speed—clinicians are often racing against a progressive disease to find a treatment.
In KJ’s case, which Kleinstiver refers to as a “scientific whirlwind,” researchers and clinicians were able to move quickly because the right technology had already been developed.
“To me, the most important learning is that no new science had to be invented,” said Liu. “That’s the only reason this could take seven months for something that would usually take roughly seven years.”
While acknowledging the regulatory and financial challenges that face biotechnology companies, experts on the panel remained optimistic about the future of gene editing, and its potential to be applied not only to rare diseases but to more common conditions as well.
To date, the liver remains one of the most accessible targets for gene editing therapies, and researchers are working on ways to deliver therapies to other sites.
“I don't see why anyone will have to live with a genetic liver disease five to 10 years from now,” said Martin. “I think that is an incredible statement to be able to make, but I think you can genuinely say it today.
“What I'm most excited about beyond that, though, is how do we move beyond the liver?”
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