friedreich's ataxia

24-Year-Old Researches Treatment for Her Own Crippling Disease: ‘I’m in a Race Against Time’

Shandra Trantham has Friedreich's ataxia (FA), a rare genetic disease that is slowly robbing her of the ability to walk and talk, and can also affect her heart. FA is caused by an inability in the body to make frataxin, a protein that is necessary for normal cell function. To date, there is no cure and no approved treatments — but she's working on one. She's a 4th-year Ph.D. candidate in genetics and genomics at the University of Florida Powell Gene Therapy Center. Trantham works in a lab where gene therapy for FA is being fine-tuned.

AavantiBio and Resilience Announce Strategic Collaboration for Gene Therapy Development and Manufacturing

UF startup and UF Innovate | The Hub resident AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, and National Resilience, Inc. (Resilience), a company building the world’s most advanced biopharmaceutical manufacturing ecosystem and who recently acquired UF startup and UF Innovate | Sid Martin Biotech alum Ology Bioservices, announced a strategic collaboration to support the development and manufacturing of AavantiBio’s pipeline of therapies, including its lead program in Friedreich’s Ataxia (FA).