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Gene Therapy: What’s Up With AAV?


Aug 12, 2020 02:47 PM

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Nicole Paulk, PhD
Assistant Adjunct Professor @UCSF
Dr. Nicole Paulk is an Assistant Adjunct Professor of AAV Gene Therapy at UCSF in San Francisco. Dr. Paulk has a BS in Medical Microbiology, a PhD in Viral Gene Therapy and Regenerative Hepatology, and completed her Postdoctoral Fellowship at Stanford University in human gene therapy. She is a leader in engineering next-generation AAV gene delivery platforms and comparative AAV ‘omics. Her translational AAV gene therapy lab is species/organ/pathway/disease agnostic; instead, she forges new paths for the field by engineering disruptive technologies to make AAV therapies cheaper, easier and faster to get these critical drugs to the rare disease and cancer patients who need them.
Federico Mingozzi, PhD
Chief Scientific Officer @Spark Therapeutics
Dr. Federico Mingozzi is the chief scientific officer at Spark Therapeutics, joining the company in 2017 from INSERM in France, where he was Research Director, and the non-profit Généthon. There he led the development of in vivo liver-targeted gene therapies for rare inherited diseases. Federico began his scientific career studying the genetic basis of bleeding disorders. Previously as director of translational research the Center for Cellular and Molecular Therapeutics at the Children’s Hospital Philadelphia (CHOP), he was involved in several first-in-human clinical studies. He also led studies to characterize immune responses to AAV vectors. Dr. Mingozzi received his bachelor’s degree in biology and his Ph.D. in biochemistry and molecular biology from the University of Ferrara in Italy.