RARECast: Delivering Enzyme Replacement Therapies Across the Blood-Brain Barrier

June 16, 2017

Enzyme replacement therapies are available for a number rare, metabolic diseases that collectively are known as lysosomal storage disorders. These therapies have brought great benefits to patients. One problem, however, is that the enzymes don’t cross the blood-brain barrier and don’t address the severe and progressive neurological complications caused by many of these diseases. ArmaGen thinks it has a solution. By connecting the enzyme to a protein that is allowed to cross the blood-brain barrier, ArmaGen hopes to exploit a natural mechanism to address the CNS complications of these diseases. We spoke to Mathias Schmidt, CEO of ArmaGen, about the company’s platform technology, its pipeline of enzyme replacement therapies for lysosomal storage disorders, and its implications for more common neurological diseases.

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