uniQure (QURE) Soars 27% on Update of Huntington’s Disease Trial
uniQure has announced an update on their low-dose cohort in Phase I/II trial of AMT-130 Gene Therapy for the treatment of Huntington’s Disease, which has caused the stock to jump up 27% today.
The treatment was generally well-tolerated with no significant safety issues. And there was a mean reduction of 53.8% of mutant HTT observed in cerebral spinal fluid at 12 months in evaluable patients. More information about the trial can be found here – Press Release
“We are encouraged by this 12-month update on the patients enrolled in the low-dose cohort,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “Thus far in the clinical trial, AMT-130 has been well-tolerated, with no serious adverse events related to the gene therapy and NfL levels approaching baseline. We are also pleased to observe trends suggesting target engagement that are supported by the lowering of mHTT protein in evaluable patients receiving AMT-130. We look forward to presenting additional clinical data, including functional outcomes, on all patients from this important study next year.”
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington’s disease, refractory temporal lobe epilepsy, Fabry disease, and other diseases. www.uniQure.com