Updated: Maze reveals positive first look at PhI Pompe disease candidate
The scientists at Maze Therapeutics call themselves “genetic navigators,” and on Thursday, they mapped out some positive Phase I results from their most advanced program.
MZE001 was well-tolerated in healthy volunteers. The drug is designed for patients with Pompe disease, a rare muscle-wasting condition caused by the buildup of a complex sugar called glycogen.
Pompe patients are missing a functioning copy of the enzyme responsible for disposing of glycogen. While some drugmakers have sought to infuse artificial copies of that enzyme or deliver a gene for a healthy one, MZE001 targets GYS1, a gene responsible for making glycogen. The idea is to inhibit GYS1 to prevent the buildup from occurring in the first place.
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