AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, has announced it is partnering with the University of Florida to research and develop next generation gene therapy capsids with the goal of creating safer, more effective, and tissue specific gene therapies. The expansion of this partnership will enable AavantiBio to further build out its platform focused on advancing innovative gene therapies in areas of significant unmet medical need.
The Adeno-associated virus (AAV) is the “delivery vehicle” for gene therapy, but the AAV’s capsid, or protein shell, is what steers the virus to the target cells and tissues, such as the heart, kidneys, lungs, and brain. As part of the strategic research collaboration focused on optimized genetic capsid codes, AavantiBio and the University of Florida will implement advanced cellular, molecular, and computational tools to develop next generation AAV gene therapies. By utilizing machine learning and UF’s HiPerGator 3.0 supercomputer, the Company is seeking to optimize genetic codes to create a pipeline of improved capsids for more effective and safer delivery for gene therapy.
“We are thrilled to build upon our partnership with the University of Florida and strengthen our commitment to developing the safest and most effective gene therapies to help patients with rare genetic diseases,” said Bo Cumbo, President and Chief Executive Officer of AavantiBio. “The smallest changes in the genetic code of gene therapy capsids have the potential to dramatically improve biodistribution and efficacy to targeted tissues. We look forward to pursuing this exciting research as we aim to build a sustainable platform approach beginning with our lead program in Friedreich’s Ataxia, and currently extending into other complex disease areas where there is high unmet need.”
AavantiBio has an existing partnership with the University of Florida’s Powell Gene Therapy Center and the MDA Care Center at UF Health where co-founders and gene therapy researchers Barry Byrne, M.D., Ph.D., and Manuela Corti, P.T., Ph.D., maintain their research and clinical practices. The Company also has R&D, discovery, process, and analytical development laboratory facilities in Gainesville, FL.
“It has been exciting to watch AavantiBio grow as a company in its offices and lab space at UF Innovate | The Hub,” said Jim O’Connell, Assistant Vice President of Commercialization at UF. “AavantiBio’s use of UF’s powerful AI to develop a pipeline of gene therapy capsids will raise the bar for safe and effective gene therapy and gives the Company the potential to find therapy for diseases that currently have no cure.”
AavantiBio, launched in October 2020 with foundational financial backing from some of the life science industry’s leading investors, has announced strategic partnerships and collaborations with Aldveron, National Resilience, Inc., and Catalent in recent months to support the development and manufacturing of a diversified pipeline of gene therapies.
