by Isabella Ciccone, MPH – Nueurology Live –
Recently, the FDA granted regenerative medicine advanced therapy (RMAT) designation to uniQure’s investigational Huntington disease (HD) gene therapy AMT-130 based on interim data from a phase 1/2 trial (NCT04120493) and a comparison analysis of the data to a nonconcurrent criteria-matched natural history cohort.1 The company anticipates to provide an update of the interim data from the ongoing U.S. and European phase 1/2 studies investigating the agent in mid-2024, which will include up to 3 years of follow-up on 29 treated patients.
