Panel Discussion: Scaling CNS Oligonucleotides into Larger Indications & What It Means for Delivery, Manufacturing & Competition
- Assessing whether intrathecal administration is operationally viable for high prevalence CNS populations given procedural burden, treatment infrastructure, and long-term dosing frequency, and whether certain disorders are inherently better suited to intrathecal versus systemic BBB shuttle-based delivery approaches
- Evaluating whether systemic brain shuttle platforms can achieve therapeutically meaningful CNS exposure particularly as programs move from rare to common disease settings
- Examining how expansion into larger indications reshapes manufacturing and competitive dynamics, including oligonucleotide drug substance capacity, cost of goods at commercial scale, multi-vendor CMC complexity for antibody or ligand conjugates, raw material supply constraints for shuttles, and whether manufacturing readiness will ultimately accelerate or hinder competition in the CNS oligo space