Enabling Targeted Oligonucleotide Delivery to CNS for the Treatment of Neuromuscular & Neurological Diseases: From Concept to Clinic
- 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