PPP1R3A

Maze Therapeutics Presents New Preclinical Data Supporting Advancement of MZE001 as a Potential Treatment for Pompe Disease

Retrieved on: 
Thursday, February 10, 2022

Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced new preclinical data supporting the advancement of MZE001, which aims to address Pompe disease by reducing pathologic glycogen accumulation through the inhibition of muscle glycogen synthase (GYS1).

Key Points: 
  • Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced new preclinical data supporting the advancement of MZE001, which aims to address Pompe disease by reducing pathologic glycogen accumulation through the inhibition of muscle glycogen synthase (GYS1).
  • Pompe disease affects both people who are living with the disease, as well as their caregivers.
  • This work demonstrates the potential value of our substrate reduction approach across the Pompe disease spectrum both as combination therapy and as monotherapy.
  • Taken together, the preclinical data support the potential for a GYS1 inhibitor, such as MZE001, as a treatment for Pompe disease.