There are several key opportunities for philanthropic investment that can bring the Gene Therapy Initiative to life, and unlock new insights that may save lives. These opportunities include:

• Equipment: The caliber of research the GTI will pursue requires leading-edge equipment, some of which is still in final development. Having the most advanced equipment now and having the resources to keep pace with new inventions is critical to the GTI’s future success.

• Collaboration: One of the most important ways that philanthropy contributes to breakthroughs is by empowering emerging research that holds promise but may not have the track record to attract funding from traditional sources. Whether this is on campus or points beyond, supporting collaborative funding for pilot projects and other research can mean the difference between a good idea and transformational therapy.

• Sustainability: Endowed funds allow our faculty leaders to sustain their research efforts without having to allot significant amounts of time to pursuing extramural funding from sources such as the National Institutes of Health and others. We propose establishing key endowed positions to support Drs. Cherqui and Muotri and relieve them of the time-consuming work of pursuing grants so they can, instead, pursue cures.

• Administration: Oversight—whether in the form of regulatory and research compliance or day-to-day operations—is critical to the success of a project such as this. The GTI will require a central administration body to oversee operations and research cores and support to our administrative needs is every bit as essential as support of any other area.