Question · Q1 2021
Gbola Amusa from Chardan focused on the noteworthy long-term biopsy data from Patients 4-6, asking for hypotheses on the variability between patients, particularly the lower expression in Patient 4. He also inquired about the biological rationale for the delayed but increased expression kinetics observed over time.
Answer
Chief Scientific Officer Dr. Carl Morris explained that while Patient 4 had lower expression, they still showed positive functional trends, suggesting a potential threshold effect. He hypothesized that increased expression over time could be due to continued protein production stabilizing the muscle. CEO Ilan Ganot highlighted that this long-term data underscores the potential for durability. Dr. Morris further speculated that as muscle health improves, more consistent protein production could occur, and noted patient age could also be a factor.