Pioneering and Delivering the Future of Genomic Medicines

Nav 1.7 Prion Undisclosed C9orf72 Huntington NEUROPATHIC PAIN PRION DISEASE NEUROLOGY ALS HUNTINGTON'S • Selectively repress expression of a mutant allele while allowing for the expression of the healthy allele 13 • Blocking Nav1.7 in the DRG is expected to prevent the transmission of nociceptive pain signals to the brain • Progressive, with no disease modifying therapy Fabry Disease (isaralgagene civaparvovec or ST-920) | --- | --- | --- | --- | |-------|-------|-------|------------------------------------ ...