Huntington’s Disease and The Triad of Therapeutic Conviction

By Eric Green, CEO of Trace Neuroscience, as part of the From The Trenches feature of LifeSciVC

Few neurodegenerative diseases have clearer genetic causation than Huntington’s disease (HD). And yet for decades, translating that genetic clarity into desperately needed effective therapies has proven frustratingly difficult.

This critical gap sets the backdrop for the recent public controversy surrounding uniQure and its investigational gene therapy, AMT-130. AMT-130 is an AAV5-delivered miRNA gene therapy for Huntington’s disease, administered via direct stereotactic injection into a patient’s striatum. It works by lowering huntingtin…