Maze Therapeutics, a company focused on translating genetic insights into precision medicines, has announced a $190 million financing round to advance its pipeline of therapies for genetically defined diseases. The funding, led by Matrix Capital Management, will support the clinical development of MZE001 for Pompe disease, as well as programs targeting chronic kidney disease and amyotrophic lateral sclerosis (ALS).
The financing will enable Maze to initiate clinical trials for MZE001 in the first half of 2022. MZE001 is being developed as a novel treatment for Pompe disease, a rare and often fatal genetic disorder caused by a deficiency in the enzyme acid alpha-glucosidase (GAA). This deficiency leads to the accumulation of glycogen in various tissues, particularly muscle, resulting in progressive muscle weakness and respiratory problems.
Advancing Precision Medicine with Compass Platform
In addition to MZE001, the funding will support Maze's APOL1 program for chronic kidney disease and its ATXN2 program for ALS. A significant portion of the investment will also be directed towards expanding Maze Compass, the company's proprietary platform for drug discovery. The Compass platform integrates human genetic data, functional genomic tools, and data science technology to identify novel connections between genes and their influence on disease susceptibility and progression.
"We think Maze’s unique approach that fuses deep human genetics with a platform that can deliver end-to-end computational chemistry informed drug discovery has the potential to generate a continuous stream of precision therapies in complex disease areas that have long been underserved and lack meaningful treatment options," said Andy Tran, investor at Matrix Capital Management and incoming Maze board director.
Addressing Unmet Needs in Genetically Defined Diseases
Maze Therapeutics aims to address the significant unmet needs in genetically defined diseases, where current treatment options are limited or non-existent. By leveraging its Compass platform and focusing on variant functionalization, Maze is developing small molecule and biologic therapies that target the underlying genetic causes of these disorders.
"The foundational vision of Maze was focused on applying learnings from the evolving landscape of genetic insights and the role they play in disease and translating that knowledge into new medicines where other approaches have fallen short," said Charles Homcy, M.D., chairman of the board and partner at Third Rock Ventures.
