Researchers at the Children’s Hospital of Philadelphia have created a system to better regulate gene editing by administration of a secondary activator drug. This novel system, dubbed Xon, acts like a dimmer switch; a genetic therapy is delivered in an inactive form, and an additional drug must be used to turn on the gene therapy. By changing the amount and frequency of the dose of the activator, scientists are able to control the action of the gene therapy after it has reached its target. The activator in this model is branaplam, a drug that Novartis is studying for use in adults with Huntington’s disease. Check out the latest article from HDBuzz for more details on this exciting new technology, how it works, and why it’s relevant to HD.
EHDN Annual Meeting: HD Clinical Trial Updates, Research Hot Topics, and More
Kicking off one week from today, the European Huntington’s Disease Network will host their annual meeting remotely over three, half-day sessions from September 9th to 11th. The virtual conference will cover topics ranging from genetic modifiers and cognitive changes in HD, to clinical trial updates, lessons learned from previous trials, and more. Although the conference is directed primarily at scientists and physicians, registration is free to all who are able to attend. Register to join and view the full program of events here.
HDSA & Me Returns September 9th
Also one week from today, HDSA’s fall programming will begin with the first session of HDSA & Me, a webinar series focusing in on unique topics affecting members of the HD community. To open the series, a group of HD experts will participate in a panel discussion on all things Long-Term Care for HD. Register to join the webinar, beginning September 9th at 2PM ET, here.