Posted on July 5, 2018
As we ease into the summer, enjoy some highlights from our research sessions at HDSA’s 33rd Annual Convention in Los Angeles.
The Ionis/Roche ASO drug has gotten lots of press in the past year, but did you know there are additional strategies for lowering huntingtin? Multiple companies and academic laboratories are approaching this goal from other angles. We invited Dr. Pavlina Konstantinova to speak about her research at a company called UniQURE that is using a virus-based approach to deliver RNA therapies for lowering huntingtin: https://vimeo.com/275140952 Watch the video starting at 20:45 to hear from Dr. Elizabeth Doherty, Director of Medicinal Chemistry at CHDI, who spoke about efforts to lower levels of huntingtin protein using a small molecule drugs.
The 2018 HDSA Convention also featured a Clinical Trials Showcase in which researchers and company representatives spoke about big ongoing clinical trials: https://vimeo.com/274612855. First up (0:22) is Jamie Levy who leads the clinical platform team at CHDI and runs the Enroll-HD study. Next (9:42), Dr. Ed Wild talks about HDClarity, a worldwide sample collection initiative.