Advances in the generation, purification, and cellular delivery of nucleic acids has enabled an entire platform of new drug modalities. Transient gene therapy in the form of mRNA made headlines with the unprecedented development speed and efficacy of the COVID-19 vaccine. AAV and similar stable gene therapy formats are also entering the market at an increasing rate. Each transgene drug can be recoded in an (almost) infinite number of unique sequences while still encoding the same protein. How to design the 'best' sequence? This presentation will provide case studies on how recoding of genes as drug modalities can be explored using machine learning and synthetic biology to map out the sequence-function correlations and how to apply it for improved drug fitness.
As ATUM’s Co-Founder and Chief Commercial Officer, Dr. Gustafsson coordinates the company's bioengineering efforts and oversees external communications. Prior to co-founding ATUM/DNA2.0 in 2003, Dr. Gustafsson led the bioinformatic team at Maxygen under legendary Pim Stemmer. Before Maxygen, Dr. Gustafsson worked as a scientist at Kosan Biosciences and had research and teaching positions at UC Santa Cruz, UC San Francisco, and University of Umeå. He received his PhD in 1992 from the University of Umeå, Sweden. Dr. Gustafsson has published approximately 50 peer reviewed publications and is the inventor of 45 issued US patents.