Maze Therapeutics

charting the course of genetics

Human evolution has given rise to natural genetic diversity. Our inherited genetic makeup shapes who we are, and it also contributes to the risk of disease throughout our lifetimes. Some genetic variants can increase the risk of certain diseases, while others may provide protection from disease.

With the availability of genetic data across hundreds of thousands of individuals around the globe, it is now possible to study how variations in different genes contribute to disease, enabling the development of precision medicines that can be tailored to address the specific drivers of disease and optimize treatment outcomes.

The Maze Compass Platform™

Maze applies variant functionalization in tandem with advanced data science methods and a robust suite of research and development capabilities to advance a pipeline of novel precision medicines. Maze has developed the Maze Compass Platform™, a proprietary, purpose-built platform to understand and integrate the critical step of variant functionalization into each stage of drug development.

how it works
VIEW OUR PIPELINE

Thanks to CRISPR and other breakthroughs, we have seen a revolution in recent years in our ability to precisely alter cells and characterize how these perturbations impact function. This gives us an unprecedented ability to find ways to suppress disease states.

Jonathan Weissman

By studying human populations, we can uncover genetic variants that provide protection from genetic diseases. The scale of available human genetic data has now made it possible to begin systematically identifying these protective variants.

Mark Daly

publications and presentations

published data and literature for Maze’s platform technology

maze programs

March 2023 | MAZE THERAPEUTICS

Muscle glycogen reduction in healthy adults treated with MZE001, an oral inhibitor of GYS1 and potential substrate reduction therapy for Pompe Disease
Maze Therapeutics | Ullman, et al.

FEBRUARY 2023 | MAZE THERAPEUTICS

Results from a first in human study of MZE001, an orally bioavailable inhibitor of glycogen synthase 1 and potential substrate reduction therapy for Pompe Disease
Maze Therapeutics | Ullman.

FEBRUARY 2023 | MAZE THERAPEUTICS

Small molecule inhibition of glycogen synthase I restores autophagolysosomal and metabolic pathway dysfunction in a mouse model of Pompe Disease
Maze Therapeutics | Xi.

FEBRUARY 2023 | MAZE THERAPEUTICS

Results from a first in human study of MZE001, an orally bioavailable inhibitor of glycogen synthase 1 and potential substrate reduction therapy for Pompe Disease
Maze Therapeutics | Ullman et al.

FEBRUARY 2023 | MAZE THERAPEUTICS

Quantification of peripheral blood mononuclear cell (PBMC) glycogen as a novel biomarker for therapeutic intervention in Pompe Disease
Maze Therapeutics | Satterfield et al.

FEBRUARY 2023 | MAZE THERAPEUTICS

Small molecule inhibition of glycogen synthase I restores autophagolysosomal and metabolic pathway dysfunction in a mouse model of Pompe disease
Maze Therapeutics | Xi et al.

NOVEMBER 2022 | MAZE THERAPEUTICS

Genetic Inhibition of APOL1 Pore Forming Function Prevents APOL1 Kidney Disease
Maze Therapeutics | Hung et al.

NOVEMBER 2022 | MAZE THERAPEUTICS

MZ-301 Is a Small Molecule Inhibitor of APOL1 Pore Function That Attenuates Albuminuria in a Mouse Model of APOL1-Mediated Kidney Disease
Maze Therapeutics | Assimon et al.

JULY 2022 | MAZE THERAPEUTICS

Genetic Inhibition of APOL1 Pore Forming Function Prevents APOL1 Kidney Disease
Maze Therapeutics | Hung et al.

FEB 2022 | MAZE THERAPEUTICS

Genetic reduction of muscle glycogen is well tolerated in UK biobank participants
Maze Therapeutics | Homburger et al.

FEB 2022 | MAZE THERAPEUTICS

Pharmacology of small molecule inhibitors of GYS1 in canines and mouse model of Pompe Disease
Maze Therapeutics | Xi et al.

FEB 2022 | MAZE THERAPEUTICS

In vitro characterization of MZE001, an orally active GYS1 inhibitor to treat Pompe Disease
Maze Therapeutics | Choy

FEB 2022 | MAZE THERAPEUTICS

Substrate reduction therapy for Pompe Disease: Small molecule inhibition of Glycogen Synthase 1 in preclinical models
Maze Therapeutics | Ullman

JUNE 2021 | EXTERNAL EXPERTS

APOL1 at 10 years: progress and next steps
Kidney international | Freedman, Barry I., et al.

DECEMBER 2013 | EXTERNAL EXPERTS

APOL1 risk variants, race, and progression of chronic kidney disease.
New England Journal of Medicine | Parsa, Afshin, et al.

human genetics

APR 2021 | INTERNAL EXPERT

Identifying therapeutic drug targets using bidirectional effect genes
Nature Communications | Estrada et al.

JAN 2020| MAZE FOUNDER

A brief history of human disease genetics
PLOS Genetics | Claussnitzer et al

DEC 2019 | EXTERNAL EXPERTS

Are drug targets with genetic support twice as likely to be approved
PLOS Genetics | King et al.

JUL 2019 | EXTERNAL EXPERTS

Priority index for human genetics and drug discovery
Nature Genetics | Plenge et al.

AUG 2016 | MAZE FOUNDER

Leveraging human genetics to guide drug target discovery
Trends in Cardiovascular Medicine | Stitziel et al.

functional genomics

FEB 2022 | NATURE

TDP-43 represses cryptic exon inclusion in the FTD–ALS gene UNC13A
nature | Ma, X.R., Prudencio, M., Koike, Y. et al.

SEPT 2021 | EXTERNAL EXPERTS

From variant to function in human disease genetics
Science | Lappalainen, Tuuli, and Daniel G. MacArthur

OCT 2020 | MAZE THERAPEUTICS

A framework to integrate genome-wide CRISPR functional genomics screens with human
genetics to nominate novel therapeutic targets in ALS
American Society of Human Genetics 2020 Virtual Meeting | Cummings et al.

AUG 2018 | MAZE FOUNDER

Mapping the genetic landscape of human cells
Cell Press | Horlbeck et al.

JULY 2013 | EXTERNAL EXPERTS

Validating therapeutic targets through human genetics
Nature | Robert M. Plenge, Edward M. Scolnick, David Altshuler