Brain-targeted drug delivery 2025-2026: convergence of focused ultrasound, lipid nanoparticles, and gene therapy vectors
Summary
A TechLifeSci annual neurotech review highlighted convergence in brain-targeted drug delivery between focused ultrasound BBB opening (Carthera SonoCloud), lipid nanoparticle CNS formulations, and AAV gene therapy vector engineering. The convergence creates combination delivery strategies: FUS-opened BBB + engineered LNPs that would normally be excluded from the brain; or FUS guidance for spatial precision of otherwise systemically administered gene therapy. Multiple clinical programs are testing these combinations in glioblastoma, Alzheimer's, Parkinson's, and SMA.
Why it matters
The convergence of FUS BBB opening with advanced drug delivery vehicles creates a new therapeutic paradigm: many drugs and biologics that are effective in cell culture or animal models but cannot cross the BBB may become clinically viable when paired with temporary FUS-mediated BBB opening. This convergence vastly expands the potential therapeutic applications of Carthera's and Insightec's FUS platforms.
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