Firefly Fund Gifts $5 Million to Create Center for Rare Diseases at UT Austin Dell Medical School

Rare Daily Staff

The Firefly Fund unveiled a gift of $5 million as the founding sponsor of the Center for Rare Diseases at the University of Texas Dell Medical School.

Firefly Fund is a nonprofit organization founded in 2017 to fund the research and education necessary to accelerate a cure for all rare neurodegenerative genetic diseases that affect children and currently have no cure — starting with rare, lysosomal storage disorder Niemann-Pick Disease Type C.

The goals and vision of the Center for Rare Diseases include ending the diagnostic odyssey rare disease families often endure.

“Every family we’ve spoken to in the rare disease community has experienced that moment of uncertainty, panic and fear where they truly did not know what steps to take next,” said Chris Andrews, co-founder and member of the board of directors of Firefly Fund.

The Center for Rare Diseases also aims to serve as a nexus of innovation, research, and support for patients and families navigating the complexities of rare diseases. The center will leverage resources across the UT System and UT Dell Medical School in molecular biosciences, medicinal chemistry, biological-based therapies, pharmacy, and engineering to transform the landscape of rare disease research, diagnosis, and treatment.

The establishment of the Center for Rare Diseases will be a significant milestone in the battle against pediatric rare diseases, both locally in Central Texas and worldwide. There is an urgent call for innovative approaches to research and care.

“In the aggregate, rare diseases are not as uncommon as we might think. One in 17 Americans is living with a condition we classify as rare. So, what we perceive as isolated illnesses actually pose a challenge to our entire healthcare system,” said Steve Ekker, associate dean of Innovation at Dell Med.

The Center for Rare Diseases at Dell Med will enable agility and scalability of research in the rare disease space. Further, through strategic collaborations with biotech and pharmaceutical companies, the Center for Rare Diseases aims to swiftly translate discoveries into impactful therapies.

Photo: Steve Ekker, associate dean of Innovation at Dell Med