Inside EE’s New Research Roadmap for the Final 10%

At Emily’s Entourage’s inaugural State of the Science: EE Edition webinar, we unveiled a bold new Research Roadmap—and shared why years of scientific progress have brought us to a pivotal moment for the final 10% of people with cystic fibrosis (CF).

For the final 10% of people with cystic fibrosis (CF) who do not benefit from existing CFTR modulators, the need for lifesaving breakthroughs remains more urgent as ever.

That urgency has driven Emily’s Entourage (EE) to fill a critical void—building the scientific foundation that didn’t exist for the final 10% and pushing promising ideas forward.

But this moment is different.

The science is maturing. Therapies are ready to move into the clinic. The regulatory landscape is evolving. And possibilities that once felt out of reach are becoming increasingly tangible.

The job now is to turn that momentum into therapies—and get them to people with CF as quickly as possible.

At our inaugural State of the Science: EE Edition webinar, we unveiled EE’s new Research Roadmap: a bold blueprint for doing exactly that.

From building the science to moving therapies forward

When EE was founded in 2011, the scientific infrastructure needed to develop therapies for the final 10% simply didn’t exist.

So we built it.

EE invested in foundational science, backed high-risk ideas others weren’t ready to fund, and brought new researchers and approaches into the space.

Today, EE has awarded 48 research grants across six countries, catalyzing $73.1 million in follow-on funding—evidence that the ideas we backed early showed enough promise for larger funders and industry partners to take them further.

Now, that science is maturing. Promising approaches are advancing toward clinical development, with a growing pipeline of therapies ready to be tested in the clinic.

EE’s role is shifting, too: from building the pipeline to accelerating the most promising therapies through it.

A new Research Roadmap for the final 10%

Grounded in EE’s focus on translational science, our new Research Roadmap identifies five priority areas where we believe the science is most promising—and where EE can accelerate progress toward the clinic.

• Gene editing to directly correct CF-causing genetic variants 
• Antisense oligonucleotides (ASOs), including exon-skipping approaches with the potential to address multiple rare variants.
• Phage therapy to combat difficult-to-treat, antibiotic-resistant bacterial infections.
• New antibiotic approaches for MRSA and other serious CF pathogens.
• Small molecules aimed at restoring functional CFTR protein.

Science is unpredictable. That’s why EE is pursuing multiple shots on goal—advancing the most promising approaches in parallel.

Why now?

The regulatory landscape is evolving.

Last year, “Baby KJ” received a personalized gene-editing therapy just seven months after birth, demonstrating that a fully bespoke gene editing therapy can be designed, manufactured, validated and delivered to a person in 7 months—a timeline that would have been unimaginable even a year ago. This has paved the way for a new “plausible mechanism pathway” to dramatically accelerate the development of genetic medicines.

While Baby KJ did not have CF, CF happens to be particularly well suited to this approach: its cause is well understood, potential therapies can be tested in the lab, and there are established ways to assess their safety and potential benefit.

With urgency as a design principle, EE is working with researchers, biotech companies, the CF community, and regulators to explore this pathway and other opportunities to compress development timelines and get therapies to people with CF faster.

From the clinic to the community

Getting therapies into clinical trials is a major milestone. But trials can only move as fast as they can find and enroll eligible participants.

EE saw a critical gap in connecting people who don’t benefit from existing CFTR modulators—whether due to their genetic mutations, side effects, or suboptimal response—with relevant trial opportunities. So we created CF Clinical Trial Connect (CTC).

The CTC builds a ready, global community of potential participants and, when new trials emerge, allows EE to quickly identify and connect potentially eligible individuals with study sites. The goal: accelerate enrollment, complete trials faster, and ultimately get new therapies to people sooner.

JOIN CF CLINICAL TRIAL CONNECT

You asked. Our scientists answered.

State of the Science sparked thoughtful questions from across the CF community—more than we could address in the program. Below, EE’s scientific and clinical team answers additional questions we received.

Q: What research is currently being funded or pursued around gene therapy, including viral and non-viral approaches? 

A: EE is funding a diverse pipeline of both viral and non-viral gene therapy. On the non-viral side, we’re investing heavily in lipid nanoparticles (LNPs) designed for aerosol delivery, extracellular vesicles, and polymeric delivery vehicles. For viral approaches, we’re supporting research into novel AAV capsid variants and virus-like particles for safe, efficient gene delivery. 

Q: Is there a definite timeline for when a treatment might be available for all?

A: While we can’t predict when a treatment will be available for all, our goal is to get a new therapy for the final 10% into clinical trials as quickly as is safely possible. The time to approval will then depend on interactions with regulators to determine the evidence needed for approval. 

Q: What research coming forward could be applicable to young children, especially those without modulators?

A: Much of the research coming forward will eventually be applicable to young children, but typically the therapies need to be proven safe and effective in adults and older children before being studied in, and made available to, young children.

Q: How can biotech companies and nonprofits effectively structure partnerships to advance research and therapeutic development?

A: We have established relationships with many biotech companies, and we use those relationships to emphasize the importance of urgency, share insights, advocate for the final 10%, and suggest ways to speed development, such as advance planning and meeting early with regulators. Additionally, our unique venture philanthropic model funds early stage companies and research projects when they’re still risky. We help de-risk these ideas by providing seed funding, which can then get scaled up. 

Q: Could successful gene therapy trials in other progressive diseases create opportunities to accelerate clinical trial access for children with CF?

A: Yes, and the Plausible Mechanism Pathway recently announced by the FDA provides a roadmap for this acceleration.

Missed State of the Science: EE Edition? 

Watch the webinar recording below: