Valerion Therapeutics said today that the company developed a fusion protein, VAL-1221, that combines its antibody delivery system with recombinant human acid alpha-glucosidase (rhGAA) to better deliver rhGAA into affected tissues of patients with Pompe disease.
Patients who suffer from Pompe disease have a deficient supply of the lysosomal enzyme, GAA, which causes glycogen to accumulate in cardiac and skeletal muscles. Although researchers have observed that glycogen accumulates in both lysosomes and cytoplasm of Pompe disease patients, the currently-approved enzyme-replacement therapy only clears glycogen in the lysosome.
Get the full story at our sister site, Drug Delivery Business News.
The post Valerion develops antibody delivery method for Pompe disease therapy appeared first on MassDevice.
from MassDevice http://ift.tt/2lCTfTL
Cap comentari:
Publica un comentari a l'entrada