NACFC 2026 Recap: Genetic Medicine Takes Center Stage

From first-of-its-kind personalized gene editing to advances in lung delivery, this year’s North American Cystic Fibrosis Conference (NACFC) offered a closer look at the science shaping what’s next for the final 10%.

What happens when the breakthroughs transforming other genetic diseases begin to converge with cystic fibrosis (CF) research?

That question took center stage at the 2026 North American Cystic Fibrosis Conference (NACFC), held October 7–10, 2026, in Atlanta, Georgia. Across plenaries and scientific sessions, researchers explored how advances in gene editing, drug delivery, and clinical trial design could open new possibilities for people with CF who do not benefit from existing CFTR modulators.

For the final 10% of the CF community—those unable to benefit from current mutation-targeted therapies—three developments were particularly relevant and offered reasons for optimism.

A Glimpse of What’s Possible

One of the conference’s most compelling stories came from Kiran Musunuru, MD, PhD, MPH, ML, MRA at the University of Pennsylvania’s Perelman School of Medicine, who shared the case of “Baby KJ,” an infant with a rare liver disease who received a gene-editing therapy custom-designed for his genetic mutation.

The milestone offered a striking example of moving personalized genetic medicine  into clinical practice at an unprecedented pace—and a glimpse of what may one day be possible for people with CF, including those with rare CF mutations.

The exciting takeaway for the final 10%? While personalized gene editing remains an emerging approach, its success in other diseases offers a valuable foundation for CF researchers to build upon, opening new avenues for people with CF who do not benefit from current mutation-targeted therapies. 

Adding to that momentum are recently announced plans for a CF gene-editing program in partnership between the Cystic Fibrosis Foundation and Dr. Musunuru that is poised to bring new expertise and attention to the development of genetic therapies for CF, starting with the R553X mutation.

The Delivery Dilemma

If genetic medicine holds the potential to transform CF treatment, getting it to the right cells remains one of the biggest obstacles.

James Dahlman, PhD, at Emory University’s School of Medicine, and fellow researchers highlighted emerging technologies designed to overcome that challenge, including next-generation lipid nanoparticles (LNPs) and advances in viral capsids that could improve delivery of genetic therapies to the lungs.

Why is this so promising for the final 10%? Some genetic therapies, including gene replacement, have the potential to benefit people across different CF mutations. More effective lung delivery could help bring these mutation-agnostic approaches closer to clinical testing..

In other words, solving the delivery challenge could help unlock the potential of an entirely new class of CF therapies.

Rethinking the Road to Clinical Trials

The scientific progress was accompanied by an equally important conversation: How do we effectively test the next generation of CF treatments?

A dedicated Lunch & Learn session examined innovative clinical trial designs and endpoints, particularly as highly effective modulators reshape the CF landscape.

For a small and genetically diverse population such as the final 10%, these questions carry particular weight. Traditional trial approaches can be difficult to implement when eligible patient populations are so small.

The encouraging takeaway? Researchers are actively exploring how to adapt clinical trials to this changing landscape. More innovative designs and meaningful measures of treatment effectiveness could make it more feasible to evaluate emerging therapies for smaller populations, helping promising science advance toward the clinic for rare mutations of CF.

EE at NACFC: Catalyzing Collaboration, Accelerating Breakthroughs 

For Emily’s Entourage (EE), NACFC was also an opportunity to bring together the people specifically focused on turning the scientific advances into real treatment options for the final 10% of the CF community.

On Thursday, Oct. 8, EE hosted a special dinner convening leading CF experts for an intimate, productive conversation focused on accelerating therapies for the final 10% and identifying opportunities to move promising approaches into the clinic. Discussions explored which genetic therapies are best positioned for success in the next two years, key clinical trial endpoints, the importance of incorporating participants’ lived experiences into efficacy assessments, and priorities for selecting the first clinical trial candidates.

The following evening, Friday, Oct. 9, EE hosted its annual NACFC reception, bringing together more than 110 members of the CF research and clinical communities from around the world to connect and hear updates from EE leadership. A highlight of the evening was the announcement of Margarida Amaral, PhD, as EE’s 2026 Dr. Carolynn Denning Trailblazer Award recipient, honoring her pioneering contributions to CF research and her role in advancing new possibilities for treatments. 

From Momentum to Medicine

With 17 genetic therapies approved across other diseases in recent years, CF researchers have an expanding body of knowledge to build upon. The excitement at NACFC 2026 reflected that progress, alongside a growing focus on the practical challenges ahead.

The question is no longer simply what genetic medicine might make possible. It’s how researchers can adapt those advances to the unique biology of CF, deliver them effectively, and bring them into clinical trials for the people who need them most—and do it quickly.

Now, the challenge is to match this progress with the urgency and determination needed to turn these promising discoveries into lifesaving therapies for the final 10%. It’s a challenge EE is working tirelessly to overcome—and one we won’t stop pursuing until every single person with CF has a breakthrough of their own.