What We Do

Emily’s Entourage (EE) is the only non-profit organization dedicated to fast-tracking research and drug development for rare, nonsense mutations of Cystic Fibrosis (CF). Here’s why your support is making this Entourage unstoppable:

1. Shattering Research Silos

EE has funded seventeen multi-disciplinary teams across the globe and provided various cells — the lifeline of rare disease research — to countless institutions. By bringing together the key players, we’ve developed a unique strategy that is looking for a major clinical breakthrough in the near future. Ambitious? Yes, but that’s the name of our game! We leverage our world-renowned Scientific Advisory Board to identify the most impactful research projects and provide high impact funding to the most qualified teams.

2. Fast Tracking Breakthroughs

We’re changing the pace of critical drug development by crafting unique partnerships between public, private and nonprofit organizations — from biotech to pharmaceuticals to universities. EE launched Spirovant Sciences (formerly Talee Bio), a biotech company focused on gene therapy for CF, by teaming up with researchers at the University of Iowa and the Philadelphia-based venture firm, Militia Hill Ventures. This unique collaboration harnesses the power of venture capital to expedite advances in gene therapy, moving us closer to a cure for everyone with CF each day.

3. Starting Small but Dreaming Big

Nonsense mutations are relatively rare in the CF population but account for approximately 12% of all genetic diseases, impacting roughly 30 million people worldwide. This includes types of muscular dystrophy, inherited blood disorders, and cancer just to name a few. Gifts have an outsized impact by not only impacting the world of CF, but powering breakthroughs that can have a far-reaching effect on other genetic diseases.

4. Putting Patients in the Driver’s Seat

We’ve become the face of disruption, creating a roadmap for advancing research in the often underfunded area of rare diseases. Our unique twist to this roadmap? The patient is in the driver’s seat and works side-by-side with the scientific community to power research that impacts patients’ lives today. Patients like Emily are taking charge of their disease and affecting real change by becoming tireless advocates who are the face of the precision medicine era.   

5. People Powering the Entourage

Started in 2011 on Emily’s family’s living room floor, EE has raised over $6 million to support life-saving breakthroughs — and it’s all because of the endless passion and power of our Entourage. Made up of thousands of supporters, followers, and volunteers from around the globe, this Entourage is bound by a steadfast tenacity, commitment and the shared belief that Emily and everyone like her deserve hope for a future.