What Maze Therapeutics does

Maze Therapeutics is a clinical-stage biopharmaceutical company founded in summer 2018 and headquartered in South San Francisco, California. The company is developing oral small-molecule precision medicines for renal, cardiovascular, and metabolic diseases, with a current focus on three clinical-stage programs: MZE829, an oral APOL1 inhibitor in Phase 2 for APOL1-mediated kidney disease (AMKD); MZE782, an oral SLC6A19 inhibitor in Phase 2 for both chronic kidney disease (CKD) and phenylketonuria (PKU); and S-606001 (formerly MZE001), an oral GYS1 inhibitor for late-onset Pompe disease that was licensed to Shionogi in 2024 and is now in Phase 2 under Shionogi's Esprit trial. Maze's discovery engine is its proprietary Maze Compass™ Platform, which applies variant functionalization — systematically identifying naturally occurring protective genetic variants in large population datasets and designing small molecules that mimic their biological effects — to de-risk target selection and predict safety before clinical development. The company went public on NASDAQ under ticker MAZE in 2025, with its stock appreciating 157% in its first year.

The business model is that of a clinical-stage biotech with no approved products and no recurring revenue. The single material revenue event to date is a $150 million upfront payment from the 2024 Shionogi licensing agreement for S-606001, with the broader deal potentially worth up to $735 million in upfront and milestones plus royalties. Future revenue is expected from a mix of direct commercialization of MZE829 and MZE782 (likely via specialty pharmacy and nephrology-focused distribution upon approval) and additional out-licensing transactions for non-core assets. The company serves genetically defined patient populations — approximately 1 million U.S. patients with AMKD, 37 million U.S. CKD patients, and roughly 60,000 PKU patients worldwide — through traditional pharmaceutical development executed via clinical trials, scientific publications, and investor/scientific conferences.

Maze has raised approximately $715 million cumulatively since inception across venture rounds (a $190M round led by Matrix Capital in 2022, a $115M Series D in 2024), a 2025 IPO, a $150M oversubscribed private placement in September 2025, and a $150M registered offering in April 2026. Cash position stood at $360 million as of Q4 2025, with the post-IPO raise explicitly intended to extend operational runway into 2029. The leadership team is anchored by CEO Jason Coloma (former Third Rock Ventures operating partner) and Chairman Hervé Hoppenot (former CEO of Incyte), with a board that includes Neil Kumar (BridgeBio Pharma CEO), Richard Scheller (former 23andMe CSO), and Daniel K. Spiegelman (former BioMarin CFO). The company had a net loss of $131.1 million for full year 2025 against zero product revenue.

Name

Maze Therapeutics

Legal name

Maze Therapeutics, Inc.

Website

https://mazetx.com

Company type

Public

Founded year

2018

Operating status

Operating

Headcount range

101–250 employees

Short description

Maze Therapeutics is a clinical-stage biopharmaceutical company developing oral small-molecule precision medicines for kidney and metabolic diseases, using its proprietary Compass Platform to translate protective genetic variants into therapies targeting APOL1-mediated kidney disease, chronic kidney disease, phenylketonuria, and Pompe disease.

Ownership category

akta.pro rank

Product category

Clinical-Stage Precision Medicine Pharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Rare Renal & Nephrology Disorder Therapies (HLAIAIAI)

akta.pro secondary industries

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN), Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI)

Keywords

• Precision medicine development
• Small molecule therapeutics
• Genetic variant drug discovery
• Kidney disease treatments
• Clinical-stage biopharmaceutical

Headquarters

HQ city

South San Francisco

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Product Revenue: Currently pre-revenue clinical-stage company with no approved products. All revenue in 2024 came from a $150 million upfront payment from licensing agreement with Shionogi for MZE001. Future revenue expected from drug approvals and commercialization.
2. Licensing and Milestone Payments: Licensing agreement with Shionogi provides potential future milestone payments for development and regulatory achievements for MZE001 (S-606001) in Pompe disease. Deal potentially worth up to $735 million in upfront and milestone payments.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels6 records

Core offering

Maze Therapeutics is a clinical-stage biopharmaceutical company developing small molecule precision medicines for renal, cardiovascular, and metabolic diseases. The company leverages its proprietary Maze Compass™ Platform, which applies variant functionalization to translate human genetic insights into targeted therapies. Its pipeline includes three clinical-stage programs: MZE829 (APOL1 inhibitor for APOL1-mediated kidney disease), MZE782 (SLC6A19 inhibitor for chronic kidney disease and phenylketonuria), and MZE001 (GYS1 inhibitor for Pompe disease, licensed to Shionogi).

Product overview

Maze Therapeutics is a clinical-stage biopharmaceutical company developing small molecule precision medicines for kidney and metabolic diseases. The company's portfolio consists of three clinical-stage programs: MZE829 (Phase 2, APOL1-mediated kidney disease), MZE782 (Phase 2, CKD and PKU), and MZE001 (licensed to Shionogi, Phase 2 for Pompe disease). The Maze Compass™ Platform underlies the company's drug discovery approach, leveraging variant functionalization to identify therapeutic targets based on genetic insights.

Differentiator

Problem solved

Functional benefit

Brands

• Maze Compass: Proprietary purpose-built platform to understand and integrate variant functionalization into drug development

Products and services

• MZE829 Oral small molecule inhibitor of APOL1 being evaluated in Phase 2 trials for the treatment of APOL1-mediated kidney disease (AMKD) and focal segmental glomerulosclerosis (FSGS). Targets patients with high-risk APOL1 gene variants, most prevalent in people of West African ancestry.
• MZE782 Investigational potent, selective oral inhibitor of SLC6A19 (sodium-dependent neutral amino acid transporter) being developed for both chronic kidney disease (CKD) and phenylketonuria (PKU). Provides a genotype-agnostic approach for PKU and a complementary mechanism for CKD.
• MZE001 (S-606001) Oral glycogen synthase 1 (GYS1) inhibitor developed as a substrate reduction therapy for Pompe disease, addressing limitations of enzyme replacement therapy by reducing glycogen production. Licensed exclusively to Shionogi & Co. for worldwide development and commercialization.
• Maze Compass™ Platform Proprietary purpose-built drug discovery platform leveraging variant functionalization to understand and integrate genetic insights into each stage of drug development. Used to identify protective genetic variants and design precision medicines that mimic their effects.

Quantifiable outcome

• 35.6% mean uACR reduction at week 12 for MZE829 in broad AMKD patients
• +4 more outcomes

Segments4 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Four partnerships are on record, tiered strategic, minor and core.

• Zifo TechnologiesstrategicTechnology or Integration · 19 February 2026Zifo and Maze Therapeutics partnered to provide AI-powered scientific workflows for managing and scaling biobank data to advance precision medicine research. The platform integrates genetic and phenotypic datasets into a unified system capable of rendering summary statistics in under five seconds. Maze uses Zifo's enterprise informatics platform to process data from UK Biobank and All of Us Research Program, aiming to accelerate target discovery and reduce manual effort and data fragmentation.
• Certara, Inc.minorStrategic or Co-development Partner · 18 February 2026Collaboration on pharmacokinetic/pharmacodynamic modeling for MZE782 treatment in phenylketonuria. Published research in March 2026 at ASCPT conference presenting mechanistic PK/PD model translating healthy volunteer data to PKU patients.
• Shionogi & Co., Ltd.coreOEM/ Whitelabel/ Licensing Partner · 1 January 2024Shionogi acquired exclusive worldwide rights to S-606001 (formerly MZE001) from Maze Therapeutics in 2024 in a deal potentially worth up to $735 million in upfront and milestone payments. Shionogi is conducting the global Phase 2 Esprit trial evaluating S-606001 as an oral substrate reduction therapy for late-onset Pompe disease. The drug received rare pediatric disease designation from FDA in 2025 and Orphan Drug Designation in 2022.
• SanofiminorStrategic or Co-development Partner · 1 January 2023Collaboration to develop novel glycogen synthase 1 (GYS1) therapies as strategic advancement in addressing root causes of glycogen accumulation in Pompe disease patients. Note: This appears in market research reporting about Nexviazyme; Maze's MZE001 is a separate GYS1 inhibitor program licensed to Shionogi.

Scale indicators10 records

Recent moves6 records

Expansion highlights6 records

Broad incumbents

• Vertex Pharmaceuticals: Specialty biopharma with established rare-disease franchises and a precision-medicine, genetics-driven discovery approach; comparable as a publicly traded rare/orphan disease developer with multiple wholly owned programs advancing through clinical development.
• BioMarin Pharmaceutical: Established rare-disease specialist with multiple approved orphan therapies across metabolic and genetic conditions; comparable business model and regulatory/commercial expertise relevant as Maze builds out its own rare-disease launch capabilities.
• Alnylam Pharmaceuticals: Genetic-medicine company pioneering RNA interference (RNAi) therapeutics with multiple approved rare-disease products; comparable as a platform-based precision-medicine company translating genetic insights into approved therapies, though using a different modality.
• Regeneron Pharmaceuticals: Large-cap biopharma with strong genetics-driven discovery (Regeneron Genetics Center) and rare-disease programs, including antibody-based therapies for genetic conditions; relevant as a mature genetic-medicine platform comparable to Maze Compass.

Direct peers

• BridgeBio Pharma: Genetic-medicine company developing therapies for diseases caused by genetic variants, with a meaningful focus on renal and cardiovascular diseases (e.g., acoramidis for ATTR-CM, infigratinib for ADPKD); directly comparable as a fellow genetically driven precision-medicine biotech and shares a board connection (Neil Kumar).
• Travere Therapeutics: Late-stage rare-disease company with approved Filspari (sparsentan) for IgA nephropathy and an FSGS program, directly competing with Maze in the genetically defined kidney disease space, particularly the FSGS subgroup where MZE829 showed 61.8% uACR reduction.
• Chinook Therapeutics: Pre-acquisition, Chinook was a clinical-stage nephrology-focused biotech developing precision medicines for rare kidney diseases (IgA nephropathy, ADPKD), acquired by Novartis in 2023 for ~$3.5B; directly comparable as a kidney-focused precision-medicine platform validated by a major strategic transaction.

Emerging players

• Ionis Pharmaceuticals: Genetic-medicine platform company developing antisense oligonucleotide therapies for rare and serious diseases; shares the precision-medicine ethos and rare-disease focus, with multiple wholly owned and partnered clinical assets.
• Disc Medicine: Clinical-stage rare-disease biotech developing precision therapies for hematologic disorders with genetic drivers; comparable as an emerging, publicly traded platform-based rare-disease company with multiple clinical assets and similar size/stage profile.
• Crinetics Pharmaceuticals: Clinical-stage rare-disease company developing oral small-molecule precision therapies for endocrine disorders; comparable as a clinical-stage rare/orphan disease biotech advancing multiple wholly owned programs toward approval.

Market position

Strengths5 records

Weaknesses4 records

Competitive moat4 records

Key risks5 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/mazetx
• Xx.com/MazeInBiotech

Revenue estimate

Valuation estimate

Number of profiles

Profiles16 records

Funding overview

Funding rounds6 records

Investors25 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Maze Therapeutics