Dyne announces new Pompe disease data showing potential of FORCE platform

Dyne Therapeutics, announced new preclinical data in a Pompe disease model demonstrating the potential of the FORCE platform to deliver enzyme replacement therapy to cardiac and skeletal muscle and the central nervous system or CNS. The data were presented at the New Directions in Biology and Disease of Skeletal Muscle Conference, being held June 23-26 in Fort Lauderdale, FL. “These preclinical data show for the first time the ability of FORCE to deliver enzymes to skeletal and cardiac muscle as well as the CNS, expanding the modularity of our platform beyond oligonucleotides. The data support the potential of FORCE to enable effective enzyme replacement therapy with the opportunity to substantially improve upon available treatments for Pompe disease,” Oxana Beskrovnaya, Ph.D., chief scientific officer of Dyne. “We look forward to continuing to explore this application of our platform as part of our mission to deliver life-transforming therapies for people with serious muscle diseases.” Pompe disease is a rare, severe neuromuscular disorder caused by deficiency of the lysosomal enzyme, acid alpha glucosidase or GAA. Lack of GAA leads to glycogen accumulation and increase in lysosomal size in muscle and subsequent weakness, cardiomyopathy and respiratory failure. Enzyme replacement therapy with GAA is the standard of care and increases survival but has inadequate efficacy in skeletal muscle. Pompe is also characterized by CNS manifestations, including behavioral and cognitive deficits due to glycogen accumulation in CNS cells, which are not addressed by the standard of care therapy.