Innovative Funding, Outsized Impact

As EE has expanded the breadth and depth of its support for drug development over the past ten years, a key part of its evolution has been the creation of new business models and funding mechanisms like venture philanthropy for biomedical research.

One example is cystetic Medicines, a company that began as an EE-funded research project, which has gone on to receive significant investment to advance a new CF treatment using amphotericin B, an already approved antifungal drug, as a molecular “prosthetic” to replace the missing CFTR ion channel. Founded by Martin D. Burke, MD, PhD, University of Illinois at Urbana-Champaign, and Michael J. Welsh, MD, University of Iowa, cystetic Medicines has secured $25 million in financing from Deerfield Management to bring the treatment to clinic.

Additionally, EE played a pivotal role in conceiving of and providing the seed funding to launch Spirovant Sciences, a biopharmaceutical company focused on gene therapy for CF. Spirovant Sciences has since been acquired twice, most recently by Sumitomo Dainippon Pharma of Japan, and is rapidly advancing novel, mutation-agnostic gene therapy for CF using adeno-associated virus (AAV) and lentivirus vectors.

These are examples of how EE’s initial investments resulted in tens of millions of follow-on dollars being invested in a CF therapy, catapulting the development of that therapy. What’s more, they are projects that had been passed over, and, in some cases, rejected by other funding sources, meaning that if not for EE’s initial investment, critical CF research may never have moved forward at all and certainly not as quickly.

The value EE offers the scientific community goes beyond the funding it provides through its grant program and venture philanthropy investments. Given EE’s expertise in mutation-targeted therapies and strong network of researchers and clinicians, EE has continued to develop an array of non-funding, consultative services to facilitate therapeutic development for early-stage companies in the CF space. Companies seek insights from EE on therapeutic development and trial design, access to research resources such as cell and animal models, key opinion leaders in the field, and more. As EE has grown and expanded its network and scope of impact and services, the organization has developed a hard-earned and well-deserved reputation for holistic leadership across the CF landscape. EE serves as a critical connector of people, ideas, technology, and resources.