The FORCE™ Platform for Transferrin Receptor 1-Mediated Delivery of Oligonucleotides to Muscle and CNS
Time: 8:30 am
day: Conference Day One
Details:
- Dyne’s FORCE Platform was designed to deliver oligonucleotides to muscle cells via conjugation to a transferrin receptor 1 (TfR1)-targeting Fab, and is currently in clinical development for the treatment of myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD).
- Consistent with TfR1-mediated uptake, we observe robust delivery of conjugated ASO to the brain of cynomolgus monkeys using FORCE, with broad uptake across different regions.
- In a mouse model of DM1, FORCE delivers oligonucleotides to the brain, and reduces human toxic DMPK RNA expression and nuclear foci, indicating the potential to address the central nervous system manifestations of DM1.
