At BIO 2025’s PACE event, patient advocates and industry leaders pushed for policy incentives to protect development of genetically targeted technologies such as antisense oligonucleotides (ASOs). Luke Rosen, founder of KIF1A.ORG, described his daughter Susannah’s apparent symptom reversal on an ASO; panelists included Holly Kordasiewicz from Ionis and moderator Emily Roberts from Genentech. Speakers cited the MINI Act and other policy tools as ways to sustain investment in rare‑disease GTTs. For readers: GTTs are therapies that target disease at the genetic or RNA level, including ASOs, siRNA, and gene therapies.