The Deep Roots Of Gene Therapy In Florida

By Cynthia Challener, Ph.D.

For over six decades, adeno-associated virus (AAV) has been at the forefront of gene therapy research, with its structure, biology, and applications continually evolving. Florida, particularly the University of Florida (UF) in Gainesville, has played a pivotal role in shaping AAV innovation. Pioneering scientists such as Ken Berns, Nick Muzyczka, and Barry Byrne contributed foundational work in AAV biology and vector development, making UF a global hub for AAV research.

Today, Ascend furthers this legacy from its GMP manufacturing facility in Alachua, FL, just outside Gainesville. Acquiring the Chemistry, Manufacturing, and Controls (CMC) team from Beacon Therapeutics in 2024, Ascend combines state-of-the-art process development with proximity to UF, the world’s leading institution for AAV publications. This strategic location and partnership enable Ascend to drive innovation in AAV gene therapy, offering unparalleled expertise and capabilities to deliver clinical and commercial materials for groundbreaking therapies.