The Virtual 36th Annual HDSA Convention was a spectacular time for researchers, clinicians, family members, and affected individuals to come together for support through recent challenges and celebration of the progress we’ve made in the last year. If you weren’t able to join us for live sessions, don’t stress! Each presentation was recorded and is now available for viewing here. If you’re interested in revisiting our research sessions specifically, keep reading! Links to all research sessions from this year’s convention and brief recaps of their highlights can be found below.
Roche and Genentech Update and Q&A on Generation HD-1: Lauren Boak from Roche Pharmaceuticals gave us a recap of preliminary results from the GENERATION HD-1 trial, followed by a Q&A with webinar participants on what happened and where we go from here.
Wave Life Sciences and Azevan Pharmaceuticals HD Clinical Trial Results: Wave Life Sciences presented results from the PRECISION-HD trials in early manifest HD followed by a Q&A, and Azevan Pharmaceuticals presented promising results for a trial of SRX246 for irritability and aggression in HD that successfully met primary and secondary endpoints.
Ask the Scientist… Anything: No agenda, no slide deck, just questions from the community! HD Buzz editors, Drs. Ed Wild, Jeff Carroll, and Rachel Harding, participated in a live Q&A that covered everything from the role of huntingtin protein in humans to the importance of COVID vaccines for the HD community.
Demystifying Neurosurgical Approaches Required for Delivery of Gene Therapies to the Brain: Developing drugs for a brain disease is hard, but getting the to where they need to be is even harder. Expert neurosurgeon, Dr. Andres M Lozano, walked viewers through different strategies to deliver therapeutic molecules to the brain in patients with HD.
HDSA Research Update and Welcome: Dr. George Yohrling kicked off the 2021 research showcase with an update on HDSA’s fellowship programs and an introduction to HDSA’s new tailored online forum, launched in partnership with PatientsLikeMe, a virtual health community for those living with HD or caring for individuals with HD. Register to join here.
HD Research Year in Review: Drs. Ed Wild, Jeff Carroll, and Rachel Harding gave a community rundown on the ups and downs of the past year in HD research and exciting research in the pipeline—and all with a special Lord of the Rings spin!
Clinical Trials Showcase Part 1: Dr. Sarah Ying from uniQure provided updates on the single dose gene therapy, AMT-130, that is currently being administered to human trial participants, and Wave Life Sciences’ Dr. Vissia Viglietta covered SELECT-HD, a newly initiated clinical trial evaluating a new ASO therapeutic, WVE-003, for selective mHTT lowering.
Clinical Trials Showcase Part 2: The second installment of this year’s clinical trials showcase included updates from Dr. Erin Furr-Stimming on KINECT-HD, a study of Neurocrine Biosciences’ valbenazine for chorea, Dr. Lahar Metha on Annexon Biosciences’ ANX005 and targeting the immune system to combat HD, and, finally, from Dr. Andy Feigin on the PROOF-HD trial of pridopidine.
The Story of the Serendipitous Identification of a new Huntingtin Lowering Drug—Branaplam: Drs. Jang-Ho Cha, Beth Borowsky, and Rajeev Sivasankaran joined in for a discussion on branaplam, an oral, small molecule drug used to treat spinal muscular atrophy that was found to also lower HTT levels. The presentation walked us through the story of the discovery of branaplam, how it works, and how it might be beneficial to people affected by Huntington’s disease.
Insights into Huntington’s Disease Chorea: During this Teva sponsored presentation, neurologist and director of the Northwestern Huntington’s disease and Wilson’s disease clinics, Dr. Danny Bega, gave viewers a crash course on HD, chorea, and how drugs like Austedo can help. Dr. Bega was joined by HDSA’s Connecticut chapter president, Sue McGann, and her husband, James McGann, who shared their personal experiences with HD chorea, participating in clinical trials, and Austedo.
Novel HD Therapies in the Pipeline: Pharmaceutical leaders Dr Daniel Curtis from Atalanta Therapeutics, and Dr. Ron Batra and Micah Mackison from LocanaBio participated in a conversation on exciting new developments in the HD Therapeutics pipeline.
HDSA-supported scientists: Advancing huntingtin-targeted therapies for Huntington’s disease: In this workshop, two researchers supported by the Berman-Topper HD Career Development Fellowship, Dr. Nicholas Caron (University of British Columbia) and Dr. Yasaman Gholamalipour (UMass Medical School), spoke about their work towards the development of novel HD therapies and how collaborative efforts among young HD scientists are driving the field forward.