This week, Novartis released a community update on VIBRANT-HD, the phase 2b study of branaplam in adults with HD. The statement recapped what we know about the temporary suspension of dosing in the study and confirmed that while dosing has halted, study visits will continue as planned. Additionally, the letter reaffirmed Novartis’ commitment to the HD community and gratitude for the dedication of study participants. Novartis plans to share more information when they are able in the coming months. To read the full community update, click here.
AskBio announces initiation of novel gene therapy trial
Earlier this week, AskBio announced they were approved to begin a Phase I/II trial for a novel HD gene therapy, BV-101. The trial will take place in France and be conducted through AskBio’s subsidiary, BrainVectis. BV-101 uses a harmless virus called an AAV to transport a genetic drug into brain tissue, which is delivered by an MRI-guided brain surgery procedure. The drug aims to improve the function of neurons in the brain by improving cholesterol metabolism, as well as improve clearance of mutant huntingtin protein. To read the full press release, click here.
ASGCT Webinar TODAY: Gene therapy administration techniques
Ground breaking science has made gene therapy a legitimate yet controversial treatment opportunity on the horizon for many genetic conditions. Delivery of these types of drugs is one of many challenges of their implementation. In this week’s lunch and learn from the American Society of Gene & Cell Therapy, Dr. Kimberly Goodspeed will walk through different administration techniques for gene therapies, their limitations, and how preclinical development of these techniques is translated into humans. To learn more and register to watch the webinar live at 12PM CT today, click here.
