Novel viral vectors in gene therapy, Joel Schneider, CEO & Robert Kotin, Founder, Carbon Biosciences

Synopsis: Joel Schneider, Ph.D. and Robert Kotin, Ph.D. are the President & CEO and Founder & Chief Technology Advisor, respectively, of Carbon Biosciences, an emerging leader in the development of novel parvovirus-derived gene therapies. Joel and Robert sit down with host Rahul Chaturvedi to discuss the arc of their careers, how the AAV field has changed over the last decade, and how they approach building out their team at Carbon. They also talk about Carbon’s focus on cystic fibrosis and the implications of The Cystic Fibrosis Foundation investing in their Series A. Finally, they both weigh in on what excites them when thinking about the future of gene therapy. Biographies: Dr. Joel Schneider joined Carbon as CEO in 2022, after serving as Chief Operating Officer at Solid Biosciences. As Solid’s first employee, he played an instrumental role in building the company’s unique disease-focused business model. Dr. Schneider is an accomplished biosciences executive with a track record of achievement in identifying, developing, and financing high potential therapeutic modalities and has diverse leadership experiences across R&D, technical and corporate operations, and corporate development organizations. Dr. Schneider holds a Ph.D. from Rutgers University and an undergraduate degree from Brandeis University and is the author of numerous peer-reviewed articles related to Duchenne and stem cell biology. He completed a postdoctoral fellowship at Harvard University in the Department of Stem Cell and Regenerative Biology, where he characterized and developed the small molecules that enhance skeletal muscle regeneration. Dr. Robert Kotin has been a leader in adeno-associated virus (AAV) research for 35 years, focusing on the molecular biology of the virus’s non-structural proteins and then leveraging this understanding to develop novel AAV vectors for somatic cell gene therapy. Beginning as a postdoctoral fellow at Cornell University Medical Center, Dr. Kotin discovered a common integration site for AAV DNA in human chromosome 19, which he designated AAVS1 locus. He spent most of his career in the Intramural Research Program at the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH), first as a tenure-track investigator and then as a tenured senior investigator and Head of the Laboratory of Molecular Biology and Gene Therapy. While at the NIH, Dr. Kotin’s laboratory invented and developed a scalable recombinant adeno-associated virus (rAAV) production process in Sf9 cells which was licensed by UniQure, ThermoFisher, Voyager, Biomarin, and others and was used to produce Glybera™, the first rAAV product granted regulatory agency approval for sale. Additional research from the Kotin lab resulted in the discovery of an AAV replicative product that has been described as closed-ended linear duplex DNA (ceDNA) and became the basis of the non-viral gene therapy company Generation Bio (NASDAQ: GBIO). Dr. Kotin served as vice president of virology and gene therapy at Voyager Therapeutics from 2014 to 2016. Since 2016, he has served as an adjunct professor at UMass Medical School, where his research interests include vectorizing and characterizing ancestral parvoviruses based on inferred sequences from endogenous virus elements (EVEs) as novel gene therapy vectors. Dr. Kotin earned his B.A. in biology from the University of California, Santa Cruz, and his doctorate in microbiology from Rutgers University and the University of Medicine and Dentistry of New Jersey (now Robert Wood Johnson Medical School).