Gangliosidoses
The Gangliosidoses are a family of lysosomal storage diseases including Tay Sachs, Sandhoff, and GM-1 Gangliosidoses. These diseases have a combined incidence of 1 in 55,000 and are characterized by deficiencies in the lysosomal enzymes which degrade gangliosides, a lipid-linked glycan. Patients experience dramatic morbidity and premature death primarily resulting from lysosomal accumulation of gangliosides in the brain.
A substantial need exists for effective treatment options for patients with gangliosidoses. Recombinant enzyme therapy has limited utility as the infused enzymes do not cross the blood-brain barrier, and other treatment strategies have not proven effective.
To address these needs, Zacharon is developing a novel, brain-penetrant small molecule therapy which selectively inhibits gangliosides thus preventing lysosomal accumulation. Proof of concept has been demonstrated in human cellular models of Tay Sachs and Sandhoff disease. Zacharon is currently completing the preclinical development activities required to advance this exciting new strategy for reducing the devastating symptoms associated with gangliosidoses.