Meghan Soustek-Kramer

DTD functional lead supporting SGT-501 (Catecholaminergic Polymorphic Ventricular Tachycardia) clinical program and both early internal and external AAV cardiac programs.

• Served as the R&D functional lead for the SGT-501 (CPVT) team supporting key activities such as assay development and disease/MOA understanding. • Managed both external and internal functional potency efforts to support SGT-003 (Duchenne Muscular Dystrophy) clinical program. • Managed and trained one associate scientist and multiple undergraduate co-ops, to support ongoing potency assay efforts for both SGT-501 (CPVT) and SGT-003 (DMD).

• Led in vitro functional potency assay development efforts for SGT-001/SGT-003 for DMD. • Managed relationships and oversee experimental planning and testing with external vendors • Designed and performed an in vitro chemical screen that identified topoisomerase II inhibitors as potent compounds capable of significantly enhancing AAV transduction in multiple muscle cell lines.

Completed the development and transfer of a cell-based expression assay for SGT-001.

• Developed and performed a high-throughput chemical screen to identify small molecules that can rescue mitochondrial deficient cells from energetic stress-induced cell death • Mechanistically described a novel pathway defining how cells cope with an increase in nutrient and ER stress by increasing mitochondrial supercomplex formation

• Developed, optimized, and tested rAAV vectors for neuromuscular disorders (Pompe and Barth Syndrome)