In a press release published this morning, uniQure announced that AMT-130, an investigational gene therapy for treatment of Huntington’s disease, was well tolerated with no significant safety issues over the first year in the phase 1b/2a study’s earliest-dosed participants. The trial’s Data Safety Monitoring Board (DSMB) also gave a positive review for the first four participants in the high-dose cohort of the trial. Thus far, 19 participants in the US have successfully undergone surgery, with recruitment of the higher-dose cohort expected to be complete by mid-2022.
Additionally, at the November HSG conference, uniQure announced that a Phase 1b/2a study is beginning to seek participants in Europe and is planning to enroll 15 participants; the first patient has begun screening for enrollment in Warsaw, Poland. uniQure is also recruiting additional participants in the US and has opened new study sites for enrollment in the US and changed inclusion criteria from candidates with 44 or greater CAG repeats to candidates with 40 or greater CAG repeats. To learn more and see if you may be eligible to participate, visit the study listing at clinicaltrials.gov or match to sites on HDTrialfinder.org.
HDBuzz on KINECT HD: Valbenazine for treatment of chorea in HD
Neurocrine Biosciences recently announced positive topline data from the Phase 3 study, KINECT-HD, that tested the effects of the drug valbenazine on involuntary movements, chorea, in HD. The latest article from HDBuzz breaks down this news; valbenazine was found to significantly reduce involuntary chorea in study participants. However, we don’t yet know how valbenazine stacks up against other drugs approved to treat chorea in HD. Read the full article here.
What happens when huntingtin protein is mutated? Nature article explores HD’s effects
Mutant huntingtin protein (mHTT) aggregates have been linked to symptom onset in HD, but how the two connect is not well understood. An article recently published in nature explored how mHTT aggregates form and what their unique structural properties may tell us about the disease. Advanced imaging techniques allowed researchers at the Swiss Federal Institute of Technology to observe how mHTT clumps are formed in a mammalian cell model of HD. They found that while initial aggregation is dependent on the presence of CAG repeats that are characteristic of HD, other proteins, lipids, and cellular materials are pulled into mHTT aggregates, too. The cellular environment in which this aggregation occurs may also affect what materials are pulled in, which could contribute to different effects of mHTT in brain tissues vs elsewhere in the body. To read more about this study, click here.