enzyme disorders

BioMarin axes $270M asset after missing phase 3 endpoint

BioMarin Pharmaceutical has ended development of BMN 401 across all indications, months after reporting mixed phase 3 data on the rare-disease prospect.

Nick Paul Taylor Aug 7, 2026 9:10am

GSK won't pick up Wave's genetic disease RNA editor

Feb 2, 2026 11:14am

Denali ph. 1 Pompe plans hit with clinical hold

Dec 4, 2025 11:52am

Graphic image of two hands shaking with employeesexecutives on the forearms of each shaking hand

BioMarin inks $270M Inozyme buyout, bagging phase 3 prospect

May 16, 2025 8:46am

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BioMarin's R&D head hopes pipeline of good ideas won't 'dry out'

Feb 21, 2025 7:00am

Syntis launches with obesity pill to mimic gastric bypass

Jun 11, 2024 7:00am