Overview:
The National Institutes of Health (NIH) is seeking novel non-viral delivery vehicles to transport macromolecules (e.g., proteins, plasmid DNA, mRNA) across the blood-brain barrier. End applications include use in gene editing of brain cells.
Background:
While gene therapy and gene editing technologies are becoming more widely known, available and tested, there remain significant challenges to unlock their full potential. Of high interest to further this technology space are non-viral technologies that can cross the blood brain barrier and target a wide variety of brain cells.
Constraints:
Technologies will ideally:
- Have in vivo proof of concept in mammals
- Be non-viral
- Capable of delivering macromolecules such as genome editors
- Deliver cargo to multiple cell types in the brain
- Deliver cargo to a significant percentage of total cells in the brain (e.g., 5%+)
Possible Solution Areas:
- Gene editing
- Gene therapy
- Gene silencing
- CNS drug development
Desired outcome of the solution:
The NIH seeks to better understand the landscape of non-viral delivery technologies capable of crossing the blood-brain barrier to support delivery of genome editing technologies that might ultimately be used in vivo in the clinic.
Related Tech Needs:
- Automated Medical Inventory System
- Precision Health Techniques
- NIH Seeking: Programmable Delivery System for Genome Editing
