Phase 1-2

Preliminary AMT-191 phase 1/2a data shows sustained increases in α-Gal A enzyme activity in patients with Fabry Disease

uniQure has announced updated preliminary safety and exploratory efficacy data from 11 patients in its phase 1/2a trial of AMT-191, an investigational AAV gene therapy...

Positive longer-term phase 1 data of Nexiguran Ziclumeran (nex-z) in patients with Transthyretin Amyloidosis with cardiomyopathy

Intellia Therapeutics, a leading clinical-stage gene editing company focused on revolutionising medicine with CRISPR-based therapies, has announced positive follow-up data from the ongoing phase 1 clinical...

Amgen advances toward lupus redemption while gastric cancer candidate struggles

Amgen’s lupus efforts may be about to succeed. Daxdilimab, a monoclonal antibody acquired from Horizon Therapeutics in 2023, hit the primary endpoint in a phase 2...
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