Federico Mingozzi, PhD

Treasurer (2023-2026)


Federico Mingozzi, PhD, is the chief science and technology officer at Spark Therapeutics, Inc. The goal of Dr. Mingozzi's career is the development of safe and effective gene-based treatments for genetic and acquired diseases. His academic research focused on the understanding the interactions between gene therapy vectors and host immune system, and the development and clinical translation of gene therapies based on adeno-associated virus (AAV) vectors. Key findings derived from this work include the induction of immune tolerance via liver gene transfer, the characterization of immune responses to AAV vectors in humans, and the development of strategies to overcome humoral and cell-mediated immunity to AAV. This work is fundamentally important, as it addressed hurdles of the technology that are key to the long-term safety and efficacy of in vivo gene transfer.

As part of Dr. Mingozzi's translational work, he has been involved in pioneering gene therapy trials for bleeding disorders, inherited blindness (LUXTURNA®), metabolic, neuromuscular diseases, and others. The nature of this research extended my areas of expertise into delivery platform technologies, manufacturing, regulatory aspects, and clinical trial design. The transition from academia to industry broadened his leadership experience and research interests. Currently, he leads a talented organization focused on building and progressing a pipeline of gene therapies directed to the eye, the liver, and the central nervous system. This includes work on novel delivery and therapeutic modalities and manufacturing-related activities in technical development, analytical development, and quality control. He is passionate about science, bringing life-changing therapies to patients, and training and mentoring the future generations of researchers in gene therapy.


Register for the 27th Annual Meeting

May 7-11, 2024 | Baltimore, MD

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