Oxana Beskrovnaya
Chief Innovation Officer Dyne Therapeutics
Seminars
Thursday 20th August 2026
Panel Discussion: Scaling CNS Oligonucleotides into Larger Indications & What It Means for Delivery, Manufacturing & Competition
8:30 am
- 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
Thursday 20th August 2026
Enabling Targeted Oligonucleotide Delivery to CNS for the Treatment of Neuromuscular & Neurological Diseases: From Concept to Clinic
11:00 am
- We have developed a TfR1 mediated delivery platform capable of transporting therapeutic oligonucleotides to both muscle and the CNS
- This delivery platform has translated into clinically meaningful functional improvements in neuromuscular diseases, including DMD and DM1
- In the CNS, our approach achieves broad and homogeneous brain distribution of oligonucleotide payloads, reaching deep brain regions that are inaccessible via intrathecal administration
- We have now extended the TfR1 mediated CNS delivery approach to neurological disease targets, demonstrating robust target modulation in non human primates across multiple brain regions, including deep structures such as the caudate and putamen
- These new data highlight the potential of our CNS delivery platform to enable disease modifying therapies for neurodegenerative and other neurological disorders
