HDSA Berman-Topper Fellow Dr. Rachel Harding has been working on understanding the shape of the huntingtin protein and how it interacts with other proteins. She has recently contributed to the creation of a high-resolution, interactive model. Rachel is committed to making all of her data public to promote open and collaborative science, soanyone can view (and play with) this model. It’s fun to drag huntingtin around on your computer screen and check it out from all sides in 3D!
Testing a New Huntingtin-Lowering Technology
NeuBase Therapeutics is a Pennsylvania-based company that is focusing on gene therapy for rare diseases using a proprietary form of drug. Their technology is called PATrOLTM, and like the drugs that Roche and Wave are testing, the NeuBase antisense oligonucleotide (ASO) is aimed at lowering huntingtin. The difference is that the PATrOLTM platform drugs can be given as an IV injection (into the arm) rather than a spinal injection. This week, NeuBase tweeted a press release from earlier this spring announcing some promising pre-clinical data. Using their novel ASO, they achieved huntingtin-lowering in cells grown in a dish, and showed that the drug could reach and travel throughout the brain when given as an IV injection to primates. Though this technology is not ready for prime time just yet, it’s an exciting prospect that huntingtin-lowering could be achieved less invasively.
Survey on COVID-19 and Clinical Trials
Have you or a loved one participated in a clinical trial during the COVID-19 pandemic? If so please consider filling out a survey about your experience and adding the voice of the HD community to this research project. The survey is being conducted by a biotech trade organization called BIO, to better understand how the pandemic has affected clinical research across a variety of industries. We know that some visits were paused or delayed, although many high-priority drug trials remained mostly on track despite the circumstances, huntingtin-lowering trials included.