February 16
@
12:00 PM
–
1:00 PM
Gene Therapy for Rare Neurodegenerative Diseases: RARE will get us there
Wally Block, PhD
Professor
Department of Biomedical Engineering
UW-Madison
Rare monogenic neurological diseases affect about 0.5% of Americans at birth and are estimated to account for up to 40% of the workload in hospital pediatric practice. Current drug delivery methods struggle to overcome the Blood Brain Barrier (BBB), with 98% of small molecule drugs and 99% of monoclonal antibody therapies failing to cross the BBB. This barrier, while protecting the brain, creates significant challenges for drug delivery and patient treatment.
Convection Enhanced Delivery (CED) is emerging as a promising solution, circumventing the BBB with direct, minimally invasive catheter-based infusion. Current CED surgical protocols distribute gene therapies are transforming the outlook for Huntington’s disease where only perhaps 1% of the brain needs to be altered genetically. In most rare neurodegenerative diseases however, much larger volumes of the brain require will require treatment.
New government initiatives like ARPA-H THRIVE are making a 9-figure investment in genetic correction, many of which will be focused on genetic correction for rare brain disorders. This talk will provide an overview of the biophysics technology being developed across a consortium centered at UW-Madison to get from 1% to 100% of brain coverage. The talk will present an argument why solving rare diseases will accelerate efforts to treat genetic approaches to much higher prevalence diseases such as Parkinson’s or Alzheimers.