Artificial Intelligence has potential to transform gene therapy

The research, Comprehensive AAV capsid fitness landscape reveals a viral gene and enables machine-guided design, published in the journal Science, was conducted by Dyno Therapeutics, a biotechnology company pioneering use of Artificial Intelligence in gene therapy. AAV capsids are presently the most commonly used vector for gene therapy because of their established ability to deliver genetic material to patient organs with a proven safety profile. However, there are only a few naturally occurring AAV capsids, and they are deficient in essential properties for optimal gene therapy, such as targeted delivery, evasion of the immune system, higher levels of viral production, and greater transduction efficiency. Starting at Harvard in 2015, the authors set out to overcome the limitations of current capsids by developing new machine-guided technologies to rapidly and systematically engineer a suite of new, improved capsids for widespread therapeutic use. In the research the authors demonstrate the advance of their unique machine-guided approach to AAV engineering.