The Huntington’s disease community celebrated a much-needed win last month when results for a gene therapy demonstrated slowed disease progression for the vexing neurological disorder.
The phase 1/2 readout for uniQure’s AMT-130 not only buoyed hopes for a disease-modifying option, but gave the larger field a boost by renewing confidence for a central hypothesis in Huntington’s research: that lowering levels of a toxic protein can help keep symptoms at bay.
“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing symptoms to effectively controlling the disease,” Amy Gray, president and CEO of the Huntington’s Disease Society of America, said in an email.
Huntington’s research has been beset by a series of demoralizing drug failures. For a time, it appeared that uniQure’s one-and-done gene…