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.

Compass platform

identifying and prioritizing genetically validated drug targets

Central to these initiatives is Compass, our 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 to create next-generation precision medicines.

The application of Compass assists in three core areas: target identification, drug discovery and drug development. For target identification, Compass allows us to prioritize, validate and reduce development risk of drug targets. For drug discovery, it aids in determining how best to drug the identified target and assists in estimating therapeutic effect size. For drug development, Compass helps to identify biomarkers and patient enrichment strategies, and provides insights about therapeutic window and tolerability.

We purposely built Compass to take advantage of these three disciplines to unlock the central tenet of precision medicine: deliver the right drug, to the right patient, at the right time.

By employing Compass, and its integration with our extensive drug discovery capabilities, we believe we can accelerate the pace of therapeutic development as well as increase the likelihood of producing therapies that provide meaningful clinical benefit. At Maze, we have leveraged Compass to build a broad pipeline of product candidates, including some that we are advancing ourselves and some that are partnered with external collaborators.

We are initially pursuing programs based on genetic insights in metabolic, cardiovascular and renal diseases, as well as neurological and ophthalmic disorders.

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

translating genetic insights into precision medicines

harnessing human genetics to build a next-generation precision medicine company

By integrating patient-level genetic data and paired clinical data, we are mapping novel connections between known genes and specific diseases. Therapeutic targets supported by human genetic evidence are more likely to yield efficacious treatments and we believe that through Compass, we can advance therapeutic candidates that offer enhanced clinical benefit and reduced development risk.

We currently use Compass to identify and prioritize potential drug targets and to assess the likelihood that a drug can be developed that interacts with the target to elicit the desired therapeutic effect. Additionally, Compass can accelerate the initial drug discovery process and inform clinical development considerations, including patient enrichment strategies and biomarker determination.

publications and presentations

published data and literature for Maze’s platform technology

maze programs


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

human genetics


A brief history of human disease genetics
Nature | Claussnitzer et al.


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


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


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

functional genomics


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


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.


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