Enabling Targeted Oligonucleotide Delivery to CNS for the Treatment of Neurological Pathologies

• 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

Speakers:

Susana Correia

Director, Neuromuscular Research

Dyne Therapeutics