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Rare Disease

Sanofi to license Maze’s Pompe disease compound for $150 million

by Shi En Kim
May 7, 2023 | A version of this story appeared in Volume 101, Issue 15

 

Sanofi will license Maze Therapeutics’ developmental compound for Pompe disease, a rare genetic disorder characterized by the weakening of skeletal muscles and the heart. The compound, MZE001, is a small molecule that limits the enzyme responsible for glycogen production. Maze will receive cash and future equity investment totaling $150 million. It’s also eligible for royalty payments and $600 million in milestones.

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