Federico Mingozzi Ph.D.

At-Large Director


The goal of my career is the development of safe and effective gene-based treatments for genetic and acquired diseases. Large parts of my work have been centered on understanding the interactions between gene therapy vectors and host immune system. The balance between tolerance and immunity in gene transfer is in fact crucial to successful gene transfer, as engraftment of a donated therapeutic transgene is highly dependent on the induction of immunological tolerance to the transgene product itself. Similarly, the modulation of vector immunogenicity is key to the long-term safety and efficacy of gene transfer. My work has been mainly focused on the adeno-associated virus (AAV) vector gene transfer platform, although with my research I also explored other gene transfer modalities. In my career, I contributed to the development and clinical translation of a number of gene therapy approaches for hemophilia, Leber’s congenital amaurosis type 2 (Luxturna), liver metabolic diseases, lysosomal storage diseases, and acquired diseases, among others. Because of the translational nature of this research, manufacturing, regulatory aspects, and clinical trial design also fall within my areas of interest. Currently, I am responsible for a team of scientists working on a pipeline of gene therapies targeting the eye, the liver, and the central nervous system as well as focused on platform development and immunology of gene transfer. My career, across academia and industry, is inspired by my passion for science, the goal of bringing life-changing therapies to patients, and my commitment to training and mentoring the future generations of researchers.

2021

ASGCT 24th Annual Meeting

May 12-15, 2021