Clinical Report: uniQure’s Huntington readout, Ideaya’s MAT2A data drive shares up

With new data showing a slowing of disease progression in Huntington disease patients, gene therapy pioneer uniQure intends to hold discussions with regulators this half about a path toward expedited approval.

Early Tuesday, uniQure N.V. (NASDAQ:QURE) said a single administration of gene therapy AMT-130 led to a slower decline in disease scores at 24 months among 21 evaluable patients in the study compared with external controls. The effect was statistically significant in the study’s high-dose arm, and did not reach significance in the low-dose arm despite a numerical trend favoring the therapy...