Maze Therapeutics

a golden age of genetics

our strategy – applying variant functionalization to precision medicine

The scientific community has been discovering disease-gene associations at a remarkable pace, providing us with invaluable learnings and access to an unprecedented amount of genetic data. With the availability of these data, there has been exponential growth in our genetic understanding of both rare and common diseases, and while there is incredible potential, it has yet to be fully harnessed.

Maze was formed to solve this very problem - to determine which of these genetic associations can be translated into a therapy for patients.

Our strategy focuses on advancing precision medicines to address common diseases across renal and cardio-metabolic indications. To do this, we are applying insights in genetics, combined with our area expertise and Maze Compass™ platform to maximize the long-term impact we can have on worldwide human health.

Renal Disease

Estimated to impact -10% of the world population with higher estimates for patients of African ancestry

Cardio-Metabolic Disease

Cardio-Metabolic diseases, including cardiovascular disease, diabetes, obesity and chronic kidney failure, are the #1 cause of death worldwide, with >30% of risk attributable to genetics

Applying genetic medicine to large populations

Leveraging our area expertise, variant functionalization and Maze Compass™ platform to maximize impact on worldwide human health

Maze Compass™ Platform

Maze has developed the Maze Compass™, a proprietary, purpose-built platform to understand and integrate the critical step of variant functionalization into each stage of drug development.

Publications

Published data and literature for Maze’s platform technology

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

Muscular Dystrophy Association (MDA) Clinical and Scientific Conference | Ullman, et al.

OCTOBER 2023 | MAZE THERAPEUTICS Million Veteran Program and Vanderbilt University Medical Center

Genetic Inhibition of APOL1 Pore-Forming Function Prevents APOL1-Mediated Kidney Disease

Journal of the American Society of Nephrology | Hung et al.

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.

FEB 2022 | MAZE THERAPEUTICS

Genetic reduction of muscle glycogen is well tolerated in UK biobank participants

WORLDSymposium 2022 | Homburger 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

American Society of Nephrology’s 2022 Kidney Week | Assimon et al.

NOVEMBER 2022 | MAZE THERAPEUTICS

Genetic Inhibition of APOL1 Pore Forming Function Prevents APOL1 Kidney Disease

American Society of Nephrology’s 2022 Kidney Week | Hung et al.

APR 2021 | INTERNAL EXPERT

Identifying therapeutic drug targets using bidirectional effect genes

Nature Communications | Estrada et al.

JUNE 2021 | EXTERNAL EXPERTS

APOL1 at 10 years: progress and next steps

Kidney international | Freedman, Barry I., et al.

SEPT 2021 | EXTERNAL EXPERTS

From variant to function in human disease genetics

Science | Lappalainen, Tuuli, and Daniel G. MacArthur

JAN 2020| MAZE FOUNDER

A brief history of human disease genetics

PLOS Genetics | Claussnitzer et al

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.

JUL 2019 | EXTERNAL EXPERTS

Priority index for human genetics and drug discovery

Nature Genetics | Plenge et al.

DEC 2019 | EXTERNAL EXPERTS

Are drug targets with genetic support twice as likely to be approved

PLOS Genetics | King et al.

AUG 2018 | MAZE FOUNDER

Mapping the genetic landscape of human cells

Cell Press | Horlbeck et al.

AUG 2016 | MAZE FOUNDER

Leveraging human genetics to guide drug target discovery

Trends in Cardiovascular Medicine | Stitziel et al.

JULY 2013 | EXTERNAL EXPERTS

Validating therapeutic targets through human genetics

Nature | Robert M. Plenge, Edward M. Scolnick, David Altshuler

DECEMBER 2013 | EXTERNAL EXPERTS

APOL1 risk variants, race, and progression of chronic kidney disease.

New England Journal of Medicine | Parsa, Afshin, et al.