Alex Lim's questions to Boundless Bio (BOLD) leadership • Q1 2019
Question
Alex Lim from Mizuho inquired about the differentiation of an AAV approach versus a lentiviral approach for Pompe disease and asked if other vectorized exon skipping therapies are in development.
Answer
Matt Patterson, Chairman and CEO, differentiated the approaches by describing Audentes' AAV therapy as a single IV infusion for direct tissue transduction, whereas a lentiviral approach is a more complex ex vivo cell extraction, modification, and re-implantation process. He stated the company believes the in vivo AAV approach is superior for Pompe. Regarding other vectorized exon skipping therapies, he was not aware of any specifically but noted other vectorized antisense approaches exist for diseases like Huntington's, which have some scientific similarities.