40 FLORIDA STATE UNIVERSITY / COLLEGE OF MEDICINE
• IPRD’ s Viral Vector and Gene Editing Core, also in the college’ s Biomedical Sciences wing, is fully operational. This is where“ precision medicine” takes place, developing treatments and potential cures for rare diseases. The quality-controlled facility specializes in the design, development and production of viral vectors, which carry“ therapeutic” genes, to effect change. Precise editing of DNA is accomplished through advanced gene-editing tools such as CRISPR – Cas systems, while complementary technologies like optogenetics and engineered proteins or antibodies, help researchers control, visualize and modulate biological processes with exceptional precision. The Viral Vector and Gene Editing Core is slated to move to the FSU Health Academic Health Center on the Tallahassee Memorial HealthCare campus when it is completed late in 2026.
• IPRD’ s Precision Pediatric Clinic is taking shape at an existing facility on Phillips Road. A team of physicians, clinical geneticists, genetic counselors and additional health-care professionals will occupy the building as early as February 2026. The clinic will take a“ genome-first” approach, offering specialized diagnostic care for children with undiagnosed or complex conditions while also providing comprehensive general pediatric care for all children and families. It will also serve as a hub for patient recruitment into research studies
• Still to be determined is the base location for the Master’ s in Genetic Counseling Program, which has been approved by the Florida Board of Governors, and tentatively scheduled to roll out with its first class in the fall 2027. Bhide indicated that IPRD is closing in on the selection of a program director, who will chart the course for securing its accreditation.
The training and preparation of genetic counselors, who will provide guidance to individuals and families navigating the complexities of genetic information in the rare disease space, is critical to IPRD’ s mission. The program, just the second to be offered in-state, will also help fill the growing need for skilled and compassionate experts in the wake of advances in genetics and genomics.
• The Rare Disease Research Program will not require an independent facility. Since 2023, the IPRD Research Advisory Council has solicited investigators, university-wide, to submit research grant applications for projects focused on discovering and developing technologies for the diagnosis and treatment of a broad spectrum of rare diseases.
To date, the council has awarded grants for approximately 20 projects through four rounds of funding cycles( twice annually). While the majority of those grants have gone to College of Medicine researchers, they have also been awarded to investigators in the Department of Chemistry and Biochemistry, and the Department of Health, Nutrition and Food Sciences. All research has been conducted in existing laboratory space.
The Momentum Continues
The mere thought of providing meaningful assistance and hope to children and families in need – through research, clinical expertise, counseling and education – has a huge appeal.
Cynthia Vied, a lifelong research scientist who was charged with setting up and is now leading IPRD’ s Diagnostic Lab, was moved by the story of Adam Anderson’ s son, Andrew.
“ Hearing his story and knowing what happened to his family, it made all of us that became part of the IPRD want to do more and help other families that go through what they call the diagnostic odyssey,” said Vied, adding that she has been energized to learn and grow alongside IPRD’ s development.“ This was the first time I’ d been involved with anything that had direct human impact, and obviously I had not done anything clinically related.
“ Hearing about rare disease and pediatrics really gets people motivated, us included, and also administra-