Question · Q3 2019
Dae Gon of SVB Leerink inquired about the specifics of the FDA discussion on the Duchenne Muscular Dystrophy (DMD) platform strategy, the competitive landscape for the AT845 Pompe program, and potential dose-limiting toxicities in preclinical studies for DMD.
Answer
CEO Matt Patterson and COO Natalie Holles responded. Patterson highlighted the company's muscle-directed approach for Pompe as superior and confirmed preclinical data would be shared in February. Holles detailed the productive FDA meeting on DMD, which covered streamlined nonclinical, manufacturing, and clinical development pathways. She also explained that their DMD approach of repairing endogenous transcripts, rather than over-expressing a synthetic protein, may offer a better safety profile.