The Technology
Efficient delivery of nucleic acid therapeutics to the brain and muscle remains a major challenge. The blood-brain barrier restricts access to the central nervous system, while effective and selective gene delivery to muscle tissue also remains limited. Current lipid nanoparticle platforms therefore require improved targeting capabilities to achieve tissue-specific delivery and reduce off-target exposure.
This technology introduces a modular targeted lipid nanoparticle (LNP) platform for nucleic acid delivery to brain and muscle tissue. The LNP surface is functionalized with receptor-binding targeting moieties, including small molecules linked through PEG-lipids, enabling enhanced interaction with specific cell-surface receptors and improved tissue tropism. The platform is designed to encapsulate nucleic acid cargos such as mRNA and can be adapted for systemic or local administration.
A lead acetylcholine-functionalized LNP formulation demonstrated enhanced mRNA delivery and transgene expression in brain-targeting models, including human BBB and brain organoid systems, with evidence of receptor-mediated uptake and neuronal tropism. The same targeting strategy is also protected for muscle delivery, including skeletal muscle, supporting broader opportunities for gene replacement, gene modulation, and other nucleic acid-based therapeutics.
Advantages
- Targeted nucleic acid delivery to brain and muscle tissue Modular platform based on receptor-directed small-molecule targeting
- Compatible with mRNA and other polynucleotide cargos
- Supports systemic and local administration
- Receptor-mediated targeting with potential for improved tissue specificity
- Scalable LNP formulation using established microfluidic manufacturing approaches
- Validated activity in human-relevant brain models
Applications and Opportunities
- mRNA and nucleic acid delivery to skeletal muscle
- Genetic and inflammatory muscle disorders
- CNS and neurodegenerative diseases
- Neurodevelopmental and neuroinflammatory disorders
- Gene replacement and gene modulation strategies
- Precision delivery of RNA therapeutics
- Platform partnerships for targeted LNP development
Business Development Contacts
