Maze Therapeutics to Present Preclinical Data Highlighting MZE001 as a Potential Treatment for Pompe Disease at 2022 WORLD Symposium

January 31, 2022 |

2 min read

Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced the company will present new preclinical data supporting the advancement of MZE001, an oral, selective and potent muscle glycogen synthase (GYS1) inhibitor that aims to address the underlying cause of Pompe disease.

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced the company will present new preclinical data supporting the advancement of MZE001, an oral, selective and potent muscle glycogen synthase (GYS1) inhibitor that aims to address the underlying cause of Pompe disease. The data will be presented during two oral and two poster presentations at the 18^th Annual WORLD Symposium being held February 7-11, 2022, virtually and in San Diego.

“These presentations highlight the breadth of Maze’s capabilities in human genetics, biology, pharmacology and small molecule drug discovery,” said Jason Coloma, Ph.D., co-founder and chief executive officer of Maze. “Pompe disease is a challenging condition that leads to devastating outcomes, and despite the availability of enzyme replacement therapies, most patients continue to experience disease progression, highlighting the need for new treatment approaches. We are excited to present the first preclinical data that support our novel approach to treat Pompe disease by inhibiting GYS1. With MZE001, we have the potential to offer an oral treatment option for these patients, and we look forward to sharing these findings at the WORLD Symposium next week.”

Maze’s oral presentations will be part of a panel on February 10, beginning at 9 a.m. PT. Details of the presentations are as follows:

Oral Presentations

Title: In-vitro characterization of MZE001, an orally active GYS1 inhibitor to treat Pompe disease
Presenter: Rebeca Choy, Ph.D.
Date & Time: February 10, 2022, at 9 a.m. PT

Title: Substrate reduction therapy for Pompe disease: small molecule inhibition of glycogen synthase 1 in preclinical models
Presenter: Julie Ullman, Ph.D.
Date & Time: February 10, 2022, at 9:30 a.m. PT

Poster Presentations

Title: #122. Genetic reduction of muscle glycogen is well tolerated in UK Biobank participants
Presenter: Julian Homburger, Ph.D.
Date & time: Poster presentations will be available for viewing by conference attendees beginning on February 7, 2022, at 3 p.m. PT

Title: #327. Pharmacology of small molecule inhibitors of GYS1 in a mouse model of Pompe disease
Presenter: Yannan Xi, Ph.D.
Date & time: Poster presentations will be available for viewing by conference attendees beginning on February 7, 2022, at 3 p.m. PT

About Maze Therapeutics

Maze Therapeutics is a biopharmaceutical company applying advanced data science methods in tandem with a robust suite of research and development capabilities to advance a pipeline of novel precision medicines for patients with genetically defined diseases. Maze has developed the Maze Compass^TM platform, a proprietary, purpose-built platform that combines human genetic data, functional genomic tools and data science technology to map novel connections between known genes and their influence on susceptibility, timing of onset and rate of disease progression. Using Compass, Maze is building a broad portfolio of wholly owned and partnered programs Maze is based in South San Francisco. For more information, please visit mazetx.com, or follow us on LinkedIn and Twitter.