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Mar 23, 2015
 |  BioCentury  |  Emerging Company Profile

Complementary edge

Abeona's gene therapies could have dosing, efficacy advantage for MPS III

The neurological manifestations of many lysosomal storage disorders are poorly treated because it hasn't been possible to get enough of an enzyme or gene therapy vector across the blood-brain barrier to replace dysfunctional proteins. Abeona Therapeutics LLC aims to improve on more advanced candidates for mucopolysaccharidosis III with preclinical gene therapies that can be delivered systemically to treat both CNS and peripheral manifestations of the disease.

No therapies are approved to treat mucopolysaccharidosis (MPS) III, a rare lysosomal storage disorder also known as Sanfilippo syndrome, which kills 70% of patients by age 18.

The disease is caused by genetic mutations that impair the body's ability to degrade heparan sulfate glycosaminoglycans (HSGAGs). Sanfilippo type A is caused by mutations in N-sulfoglucosamine sulfohydrolase (SGSH; HNS), while type B is caused by mutations in the N-acetylglucosaminidase alpha (NAGLU) gene.

In both types, accumulation of HSGAGs in the brain causes motor, behavioral and other neurological symptoms, and accumulation in the periphery can lead to...

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