Nanoparticles for Precise & Targeted Treatment of Neurological Conditions

Problem:
Acute ischemic stroke (AIS) is the second leading cause of death worldwide, affecting around 600,000 Americans annually. However, pharmacological treatment of AIS is difficult due to ineffective and nonspecific targeting to blood brain barrier epitopes via intravenous administration. Over 100 drugs have been tried and failed in clinical trials over the past few decades. The leading cause of failure has been the inability to concentrate the drug at the injury site, indicating a strong need for novel delivery and targeting approaches.
Solution:
The inventors have developed a method of delivering a potent anti-inflammatory agent to the brain’s injury region after an AIS. The delivery method shows high specificity for the injured brain region and improved treatment outcomes in rodent models.
Technology:
The inventors designed lipid nanoparticles (LNPs) targeting vascular cellular adhesion molecules (VCAMs) that delivered mRNA encoding IL-10, an anti-inflammatory cytokine. They then injected these VCAM-targeting LNPs into a mouse model of AIS. Compared to non-targeted controls, the VCAM-targeting LNPs showed accumulation levels in the brain at 1-2 higher orders of magnitude. Additionally, compared to saline controls, mice injected with IL-10 carrying LNPs had much larger reductions in stroke volume and survival rates.
Advantages:

Reduces stroke volume by 65% in rodent model compared to 25% for the average clinical trial
20x higher drug delivery specificity to endothelial cells in the injured region of the brain compared to previous approaches
LNPs are easily injected intravenously
Treatment can be applied after injury and is not limited to prophylactic applications
Can be used to address other indications in the brain

Stage of Development: