Rajiv Prasad's questions to Boundless Bio (BOLD) leadership • Q3 2019
Question
Rajiv Prasad of William Blair asked for a comparison of the current Pompe construct versus the previous one, particularly regarding potency and biodistribution, and questioned if the muscle-directed approach was also being applied to the DMD program.
Answer
CEO Matt Patterson explained that the only change to the Pompe product was the promoter, which was fine-tuned to optimize expression levels, and that the preclinical data is very strong. He emphasized the approach is focused on muscle expression. He clarified that the DMD program utilizes a different scientific approach, vectorized exon skipping, which involves a different promoter situation than the direct protein expression used in Pompe.