- Biotech company UniQure said Friday it has dosed the first patient with a gene therapy for Huntington's disease, a degenerative condition marked by destruction of nerve cells in the brain. Two patients have entered the trial, one who has received an injection of the therapy into the brain and one who has undergone an imitation procedure.
- The treatment, called AMT-130, is one of two gene therapies UniQure is testing in humans. Its hemophilia B candidate is nearing completion of a Phase 3 trial ahead of planned regulatory submission in 2021.
- In Huntington's disease, UniQure could soon be joined by Voyager Therapeutics, which is readying its therapy VY-HTT01 for clinical trials. Voyager has said it will update investors on its Huntington's program in mid-2020.
Huntington's disease is marked by physical and cognitive decline caused by cellular production of an abnormal version of a protein called huntingtin, which is essential for nerve cell health. About 30,000 people in the U.S. have the condition.
No treatments exist for the underlying disease, but Lundbeck's Xenazine and Teva's Austedo can help control the abnormal movements associated with Huntington's.
AMT-130, by contrast, uses a common virus to deliver strips of nucleic acid that can block the mutated genes that cause production of abnormal huntingtin. It is the first gene therapy to be tested in Huntington's disease patients.
UniQure plans to enroll 26 patients in this trial, with two groups receiving different doses of AMT-130 and a placebo arm that involves a partial surgery to make it appear patients have received the injection deep in the brain.
The main goal of the trial will be to determine if the therapy is safe, but as a secondary goal researchers will want to see how persistent AMT-130 is in the brain. Researchers will also be looking for signs that patients receiving the gene therapy decline more slowly than those who didn't, but the small trial may not be able to definitively answer that question.
UniQure likely won't be alone in this chase for long. Voyager, which has already advanced a Parkinson's disease gene therapy into the clinic in partnership with Neurocrine Biosciences, is nearing a decision on when to advance VY-HTT01 into the clinic.
At its first quarter corporate update, Voyager said it is "engaged in the ongoing conduct and review of preclinical studies" and would provide an update at mid-year.