Up­dat­ed: Maze re­veals pos­i­tive first look at PhI Pompe dis­ease can­di­date

The sci­en­tists at Maze Ther­a­peu­tics call them­selves “ge­net­ic nav­i­ga­tors,” and on Thurs­day, they mapped out some pos­i­tive Phase I re­sults from their most ad­vanced pro­gram.

MZE001 was well-tol­er­at­ed in healthy vol­un­teers. The drug is de­signed for pa­tients with Pompe dis­ease, a rare mus­cle-wast­ing con­di­tion caused by the buildup of a com­plex sug­ar called glyco­gen.

Pompe pa­tients are miss­ing a func­tion­ing copy of the en­zyme re­spon­si­ble for dis­pos­ing of glyco­gen. While some drug­mak­ers have sought to in­fuse ar­ti­fi­cial copies of that en­zyme or de­liv­er a gene for a healthy one, MZE001 tar­gets GYS1, a gene re­spon­si­ble for mak­ing glyco­gen. The idea is to in­hib­it GYS1 to pre­vent the buildup from oc­cur­ring in the first place.

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