Nicholas Muzyczka

Dr. Muzyczka received his Ph.D. in Biochemistry from Johns Hopkins in 1973 and joined Dan Nathans’ laboratory as a postdoctoral fellow where he began his work on gene therapy. In 1980, his lab initiated the development of AAV vectors for gene therapy. His lab invented a way of doing genetics on AAV, and then invented the first AAV vectors, as well as the AAV vectors that are in use today. In 1994, he founded the Powell Gene Therapy Center at the University of Florida where he served as Director for six years. In 1995, he was awarded the American Cancer Society Edward R. Koger chair. His laboratory went on to develop many of the techniques currently used in AAV technology, including standard methods for vector production, purification, and quality control. His laboratory also participated in experiments that demonstrated the first long-term AAV mediated expression in the eye and CNS. To enable these animal studies, his lab helped humanize the small marker gene GFP and his lab participated in the first demonstration of using rAAV to create an animal disease model. His basic research in AAV biology includes mapping of the TR sequences that are recognized by the Rep protein for replication, the identification of the cellular proteins required for replication and the complete reconstitution of AAV DNA replication in vitro, the identification of the transcription control elements in all three AAV promoters, and the characterization of more than 100 AAV capsid mutants that control capsid assembly, cell entry, and trafficking. His laboratory has also developed novel vectors for targeting AAV to specific tissues. In 1999, he founded Applied Genetic Technologies Corp (AGTC) with the help of four fellow scientists at UF and UNC. He served as the company’s first CEO and COB and served as a Director on the Board until 2012, shortly before the company went public. In 2015, he founded Lacerta Therapeutics, whose mission is to develop therapies for the central nervous system and lysosomal storage diseases, and he currently serves as its COB. In 2017, he was inducted into the National Academy of Inventors.