Shionogi & Co., Ltd. and Maze Therapeutics, Inc. Announce Exclusive Worldwide License Agreement for MZE001
This Substrate Reduction Therapy drug could be the first pill taken by mouth to treat Pompe disease.
Astellas Enters into Definitive Agreement to Acquire Audentes Therapeutics
This development should provide Audentes with additional resources for their gene therapy programs, including a Phase 1/2 study for Pompe disease.