Susana Correia
Director, Neuromuscular Research Dyne Therapeutics
Seminars
Friday 21st August 2026
Enabling Targeted Oligonucleotide Delivery to CNS for the Treatment of Neurological Pathologies
11:30 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
