Brittney L Gurda, PhD, VMD

By training, I am a veterinary scientist with expertise in large animal models of human genetic disease and drug development with near 20 years of experience in the field of AAV gene therapy. My graduate work was among the first of its kind uniting X-ray crystallography and cryo-electron microscopy to identify antigenic regions on the capsid surface of adeno-associated viruses (AAV) under Dr. Mavis Agbandje-McKenna at UF. The desire to study this virus further in the translational setting led me to a postdoctoral position with Dr. James M. Wilson at the University of Pennsylvania Gene Therapy Center. At University of Pennsylvania (UPenn), my translational work in feline and canine models of lysosomal storage diseases generated preclinical data for the advancement of AAV gene therapies into the clinical setting. My involvement with these novel large animal models shifted my focus to translational medicine and I completed a degree in veterinary medicine at UPenn. During veterinary school I continued research and focused on the feline model of Niemann-Pick type C1 (NPC1). Here I further characterized the cerebellar pathology in feline NPC1 disease while leading AAV gene therapy studies in the cat model. A second postdoctoral position under a T32 in Neurodevelopmental Disabilities (NINDS T32 NS007413) allowed me to advance my knowledge of the CNS pathways, learn new techniques in neuroscience, and further evaluate how AAV vectors can be exploited to deliver therapeutic genes to the diseased nervous system. Following my post-graduate training, I spent several years in biotech at Spark and SwanBio Therapeutics where I was promoted to Director of Research before rejoining academia. This experience allowed me to focus on drug development giving me valuable insight into the types of studies necessary to generate data for transition into the clinic and strengthened many of my personal skills in leadership, communication, and program management. I am excited to apply my skills in the academic setting where my research currently focuses on elucidating the role of dysfunctional lipid metabolism in storage diseases that lead to neurodegeneration and exploit newly identified pathways to generate novel therapies.

• Adeno-Associated Viral Gene Therapy
• Lysosomal Storage Diseases
• Neurodegenerative diseases
• Translational drug development
• large animal models of genetic disease