TfR1-Mediated Delivery of Oligonucleotides and Enzymes to Address Neuromuscular Diseases: Translating Research into Functional Improvement in the Clinic
Time: 12:00 pm
day: Day Two - Morning - Track A
Details:
- The FORCE™ platform leverages Transferrin Receptor 1 (TfR1) to enable delivery of therapeutic oligonucleotides and enzymes to muscle and the CNS
- Preclinical data in models of DM1, DMD and Pompe diseases demonstrate that the FORCE platform delivers broad functional improvement in muscle and CNS
- The FORCE platform achieved POC in the ACHIEVE and DELIVER trials in DM1 and DMD, demonstrating translation of preclinical research into functional improvement across multiple clinical measures
