uniQure announced today that they will move forward with the Phase I/II Clinical Trial of AMT-130, a gene therapy candidate for treatment of HD administered in a single dose to deep brain tissues with MRI-guided neurosurgical delivery. After careful review of safety data for the first enrolled cohort of ten patients, the independent Data Safety Monitoring Board has recommended continued dosing in the study. uniQure will now begin to recruit patients for a higher-dose cohort of the study to continue evaluating the safety, tolerability, and efficacy of AMT-130. 

Patient Advocates Discuss Cognition in HD: Sage Science Spotlight  

Sage Therapeutics recently held an Investors’ Update for an in-depth review of a promising new drug candidate for HD, SAGE-718. The therapeutic is designed to increase activity of specific brain receptors, NMDA receptors, by mimicking the molecule that stimulates their function, 24(S)-HC, which is known to decline with onset of HD. NMDA receptors are critical for tasks like combining and linking memories, effective decision making, and others that fall under the umbrella of cognition.  

The spotlight of this discussion was not only the science behind SAGE-718, but also the voices of Director of HDSA Center of Excellence at Beth Israel Deaconess Medical Center, Dr. Samuel Frank, and patient advocate, Seth Rotberg. Dr. Frank gave listeners a clinical perspective on the role cognition plays in the life of someone living with HD. While motor symptoms are more outwardly apparent, cognition is central to memory and planning for people with HD. Additionally, in sharing the story of the onset of HD symptoms in his mother and testing positive for HD himself at age 20, Seth Rotberg explained the importance of the cognitive piece of the HD puzzle in maintaining independence, awareness, and a positive mental state. Seth highlighted the importance of evaluating quality of life for HD-affected individuals from a wholistic perspective, and developing treatments accordingly.

Compensated Interview Study for People Taking Deutetrabenazine (Austedo) 

M3 Global Research is interested in speaking with people who have been diagnosed with HD and recently began taking deutetrabenazine (Austedo). Participants will receive $125 for taking part in a 75-minute web-assisted telephone interview. To determine if you are eligible, please complete this short online survey: m3research.info/3s4ozwZ